Q1 2021 BioCryst Pharmaceuticals Inc Earnings Call

[music].

Ladies and gentlemen, this is the operator today's conference call is scheduled to begin shortly please continue to standby.

Again todays conference call scheduled to begin shortly please continue to standby we thank you for your patience.

[music].

Operator: Today's conference call is scheduled to begin shortly. Please continue to stand by. Again, today's conference call is scheduled to begin shortly; please continue to stand by. We thank you for your patience. The following sentences use commas to indicate a definite meaning:

Operator: and so on. The

If you require any further assistance please press for zero.

Operator: Ladies and gentlemen, thank you for standing by, and welcome to Biosch's first quarter, 2021 earnings call. Please note that today's call is being recorded. At this time, all participants are now listening only to the moat. After the speaker's presentation, there will be a question-and-answer session. To ask your question during the session, you will need to press star 1 on your telephone. If you require any further assistance, please press

I would not like to have the conference over to your speaker for today, Jon Bliss at by Chris Jon The floor is yours.

Thanks, Jay Good morning, and welcome to Biocryst first quarter of 2021, corporate update and financial results Conference call. Today's press releases available on our web site participating with me today or CEO, Jon Stonehouse, CFO, Anthony Doyle, Chief Commercial Officer, Charlie Guyer, Chief Medical Officer, Dr. Phil Sheridan Chief business office.

Operator: I would now like to hand the conference over to your speaker for today, John Bluth from Biker. John, the floor is yours. Thanks, Jay. Good morning and welcome to Biocris' First Quarter, 2021 Corporate Update and Financial Results Conference Call. Today's press release is available on our website. Participating with me today are CEO John Stonehouse, CFO Anthony Doyle, Chief Commercial Officer Charlie Geyer, Chief Medical Officer Dr. Bill Sheridan, Chief Business Officer Megan Zizinski, and Chief R&D Officer Dr. Helen Thackray.

Megan's Kaczynski, and Chief R&D Officer, Dr. Helen that Greg following on remarks, we will answer your questions. Before we begin. Please note that today's conference call will contain forward looking statements, including those statements regarding future results on audited and forward looking financial information as well as the company's future performance and or achievements. These statements are subject to known and unknown risks in on.

Operator: Following our remarks, we will answer your question. Before we begin, please note that today's conference call will contain forward-looking statements, including those statements regarding future results, unaudited, and forward-looking financial information, as well as the company's future performance and or achievements. These statements are subject to known and unknown risks and uncertainties, which may cause our actual results, performance, or achievements to be materially different from any future results or performance expressed or implied in this presentation.

Uncertainties, which may cause our actual results performance or achievements to be materially different from any future results for performance expressed or implied in this presentation you should not place undue reliance on these forward looking statements for additional information, including a detailed discussion of our risk factors. Please refer to the companies documents filed with the Securities and Exchange Commission, which can be accessed on our website I would know.

Operator: You should not place undue reliance on these forward-looking statements. For additional information, including a detailed discussion of our risk factors, please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website. I'd now like to turn the call over to John Stonehouse. Thanks, John.

To turn the call over to Jon Stonehouse.

Thanks, Jon.

I couldn't be more excited to have our first earnings call with revenue from a biocryst launch product.

This is the latest evidence that our company is going through a major transformation.

Sales were very encouraging in queue on especially considering this is our first quarter of the launch of Orla Dale.

This is a direct result of focused execution of our plan on the Biocryst team and the desire to build a great company that can discover develop and now successfully commercialize oral medicines for patient suffering from rare diseases.

John D. Bluth: I couldn't be more excited to have our first earnings call with revenue from a Biocrys launch product. This is the latest evidence that our company is going through a major transformation. Sales were very encouraging in Q1, especially considering this is our first quarter of the launch of Orledale. This is a direct result of the focused execution of our plan by the Biocris team and the desire to build a great company that can discover, develop, and now successfully commercialize oral medicines for patients suffering from rare diseases. Very few companies can do all three.

Very few companies can do all three.

The launches off to a great start because we have a great drug. It works patients are experiencing meaningful reductions in their attacks and they can achieve this by taking one capsule once a day.

This is leading the patient switches from injectable profane on on demand therapy.

Exactly what we saw on a market research and clinical trials and it's already playing out in the marketplace.

Great drugs don't sell themselves. We also have a fantastic team.

Jon P. Stonehouse: The launch is off to a great start because we have a great drug. It works. Patients are experiencing meaningful reductions in their attacks, and they can achieve this by taking one capsule once a day. This is leading to patient switches from injectable profine on-demand therapy. It's exactly what we saw in our market research and clinical trials, and it's already playing out in the marketplace. Great drugs don't sell themselves. We also have a fantastic team.

Jon P. Stonehouse: Charlie and Alan Hodge, our USGMs, have assembled an experienced team, but more importantly, they're working together, working through challenges and obstacles like COVID, and achieving great results. We couldn't be more pleased with the start, and the good news is that we're just getting going with so many more patients to reach in the U.S. While the U.S. is the largest market, we now have approval in other major territories around the world. Our partner, Tory, is launching in Japan, and we recently received approval in the EU. The same planning investments were made here too, but the launch ramp will take longer as these are different markets from the U.

Jon P. Stonehouse: But we expect these countries to contribute over time to our goal of achieving $500 million plus in global peak sales. And finally, our pipeline is full of the opportunity we have with our oral factor D inhibitor, B69930, for patients suffering from many different complement-mediated rare diseases. We're heading into pivotal studies in P&H and proof of concept in nephritis indications. We will apply all the learnings of our HAE program and build off that success to bring 9930 to market to create even greater value as a company that discovers, develops, and commercializes multiple oral drugs for patients suffering from rare diseases. Now I turn the call over to Charlie to go over more details about our early launch success. Charlie. Thanks, John.

Charles K. Gayer: The early data launch is off to a great start. We're pleased, but not surprised. We knew we had a great drug that patients want because they are tired of the physical, psychological, and logistical burdens of injectable therapy. Physicians also tell us they are confident in prescribing Orlideo for a broad range of patients when they see the long-term data on attack reduction, safety, and tolerability. Here's what we're seeing so far.

Patients are switching to orla Dale over half the patients starting on or starting early days for the first time are switching from injectable prophage.

The rest are switching from acute treatment only now that they have an oral once daily profi option.

In fact, the majority of patients on early day by the end of Q1, where those who switched from other products the rest where patients transitioning from our clinical trials and early access program.

Charles K. Gayer: In a very competitive market, patients are switching to Orideo. Over half the patients starting Orodeo for the first time are switching from injectable profiles. The rest are switching from acute treatment only now that they have an oral once daily prophylactic option. In fact, the majority of patients on Orideo by the end of Q1 were those who switched from other products. The rest were patients transitioning from our clinical trials in Early Access programs. The prescriber base also continues to expand. Former clinical trial investigators accounted for a minority of Orlodeo prescribers in Q1.

The prescriber base also continues to expand.

Former clinical trial investigators accounts accounted for a minority of oil a day O prescribers in Q1.

Roughly 500 physicians treat 50% of patients and our team has reached nearly all of them.

Most of these physicians tell us they intend to prescribe early day, but only a minority of them. So thus far so there is a lot of room for growth and.

And finally payers are reacting favorably to Orla Dale.

Of the reimburse product in Q1 came through medical exceptions, but many payers and Pbms also established coverage policies our momentum with payers is strong and we expect the great majority of patients to have access to coverage for early day by mid year.

Charles K. Gayer: Roughly 500 physicians treat 50% of HAA patients, and our team has reached nearly all of them. Most of these physicians tell us they intend to prescribe Orlideo, but only a minority have done so thus far, so there is a lot of room for growth. And finally, payers are reacting favorably to Orly. Most of the reimbursed product in Q1 came through medical exceptions, but many payers and PBMs also established coverage policies

The most important investment we made in this launch was building an experienced commercial team that knows how to execute our.

Charles K. Gayer: Our momentum with payers is strong, and we expect the great majority of patients to have access to coverage for Orlando by mid-year. The most important investment we made in this launch was building an experienced commercial team that knows how to execute. Our U.S. general manager, Alan Hodge, and our U.S. VP of sales, Ron Dullinger, launched Sinrise over a decade ago and successfully created the first market for H.A.E. They came to Biocris because they always knew that an oral drug is what patients really want.

Charles K. Gayer: They have attracted a talented and agile team that understands the importance of fighting for every patient in a competitive rare disease market like HAE. As excited as we are about the Q1 results, this team is just getting started.

Charles K. Gayer: COVID is limited to in-person sales calls, but vaccines are starting to change. Our marketing programs are just starting to kick in. And expanding reimbursement access is giving patients and prescribers even more comfort in switching to Oraladeo. We are very confident about the growth trajectory, and we believe Oralidao will reach peak global sales north of $500 million. The U.S. is the first and largest part of that opportunity, but we expect meaningful sales in Europe. Awareness and understanding of HAE in Europe are on par with the US, but use of targeted HAE prophylaxis has lagged based on a lack of options.

Megan Zizinski: Our work with European physicians and patients tells us that the availability of new options, and specifically an oral once daily therapy, will more than double the prophylactic treatment share to 60% or more of patients. We are taking the same approach in Europe as in the U.S., investing in experienced commercial teams that know how to launch rare disease products. Oraladaio will launch in Germany this quarter, and early access programs are active in France and the United Kingdom.

In early access programs are active in France, and the United Kingdom.

We look forward to sharing more about Europe in the coming quarters.

And there is yet another early day launched underway right now in Japan, I'll turn the call over to Megan to describe how our partners that Tori pharmaceutical are approaching this opportunity.

Eight Charlie we're excited the Japanese launch is now underway after successfully completing nanny side price negotiations last month.

For a few important points I want to emphasize again regarding the commercial opportunity in Japan, and what makes it different from the U S and Europe.

Megan Zizinski: We look forward to sharing more about Europe in the coming quarters. And there is yet another Oridea launch underway right now in Japan. I'll turn the call over to Megan to describe how our partners at Tori Pharmaceutical are approaching this opportunity. Thanks, Charlie.

First Orla day was the only approve prophylactic therapy in Japan.

With no competition Tori has a head start in building the protein market in our oral once daily medicine is well suited for a population that tends to prefer oral drugs over injections.

Megan Zizinski: After successfully completing the NIH-Night negotiations last year,

Megan Zizinski: There are a few important points I want to emphasize again regarding the commercial opportunity in Japan and what makes it different from the U.S. and Europe. First, Orlaideo is the only approved prophylactic therapy in Japan. With no competition, Tori has a head start in building the profi market, and our oral once daily medicine is well suited for a population that tends to prefer oral drugs over injections. Secondly, while the vast majority of H.A.

Secondly, while the vast majority of a K patients in the U S and you are already diagnosed Japan lags behind.

Only about 20 per cent of patients are identified in the registry today.

For he is focused on finding patients and helping to advance their standard of care by providing access to the first approved prophylactic medicine.

One reason, we chose Tori was based on what day accomplishment Gilead HIV franchise their strategy focused on driving these awareness amongst physicians and increasing patient identification.

Megan Zizinski: While patients in the U.S. and EU are already diagnosed, Japan lags behind. Only about 20% of patients are identified in a registry today. Tori is focused on finding patients and helping to advance their standard of care by providing access to the first approved prophylactic medicine. One reason we chose Tori was based on what they accomplished with Gilead's HIV franchise.

This strategy was very successful as they grew the business to almost $200 million annually.

This experienced serve as a strong foundation heading into Orla day on launch and similar to us Tories been preparing well in advance.

Are fully focused on supporting a successful launch.

Megan Zizinski: Their strategies focused on driving disease awareness among physicians and increasing patient identification.

Lastly, through our partnership economics, we have triggered the 15 billion dollar milestone payment following pricing and.

Megan Zizinski: This strategy was very successful as they grew the business to almost $200 million annually. This experience served as a strong foundation heading into Orla dey of Mott. And similar to us, Tori has been preparing well in advance and is fully focused on supporting a successful launch.

And we get to Sharon Tori success with a tiered royalty from 20 per cent to 40 per cent of net sales.

Overall, the Japanese market provides an outstanding longterm opportunity for.

We see a strong potential to follow up have similar to what we've seen in the U S and the last decade and expansion on V. H, a market driven by patient diagnosis and adoption of Profi treatment as part of standard of care.

Megan Zizinski: Lastly, through our partnership economics, we have stimulated

Megan Zizinski: has triggered the $15 million milestone payments following prices, and we get to share in Tories success with a tiered royalty from 20% to 40% of net sales. Overall, the Japanese market provides an outstanding long-term opportunity.

As Charlie shared there's a lot of early momentum in the U S launch and we're not surprised by this excellent start.

Over the course of this year, you will continue to see new clinical data highlighting how well patients do on Orland day over time.

Megan Zizinski: We see a strong potential to follow a path similar to what we've seen in the U.S. in the last decade, an expansion of the H.A.e market driven by patient diagnosis

Megan Zizinski: and adoption of Prophy treatment as part of standard of care. As Charlie shared, there's a lot of early momentum in the U.S. launch, and we're not

Megan Zizinski: Surprised by this excellent start? Over the course of this year, you will continue to see new clinical data highlighting how well patients do on Orla Day over time. Patients are getting their disease under control while reducing the impact treatment has on their lives

Megan Zizinski: reducing the impact treatment has on their lives and independence. Our work is also helping physicians understand how Orla Day is an ideal treatment choice for all patients. It offers patients the attack control they desire and the lifestyle freedom and benefits of a convenient, more discreet oral once daily pill. Our strategy remains focused on shifting this treatment paradigm. As Charlie spoke about earlier, patients are switching, which is early evidence our strategy is working. We see a very similar unmet need and opportunity in the complement space with our Factor D program. I'll now turn it over to Bill for more on our 9930 progress. Bill? Thanks, Megan.

William P. Sheridan: For everyone who discovered and developed Olodeo and our clinical trial collaborators around the world, it's just so great to see the Oladio launch going so well. For hereditary angioidema, all day, it was effective, it is safe, and because it is oral, it dramatically reduces the burden of therapy. A very similar shift is beginning for patients with paracizinal nocturnal hemoglobinuria, a very serious rare disease with no approved oral treatments, and its standard of care is lifelong intravenous infusion.

William P. Sheridan: BCX 9930 has a great opportunity to substantially improve disease control in PNH compared to the currently available intravenously infused C5 inhibitors. These do a good job controlling intravascular homolysis, but many patients remain anemy, are still transfusion-dependent, and continue to suffer from symptoms like fatigue. That's because C5 inhibitors cannot control extravascular homolice. These clinical advantages come on top of the obvious patient benefits of oral administration, and we are moving the program very quickly to get this medicine to patients with P&H.

We're moving the program very quickly to get this medicine to patients with teenage.

I am pleased to report there is net reached agreement with the FDA on both the design and the endpoints for F G and H pivotal trials.

The two pivotal trials each test oral VCX 90, 930, mono therapy and will support the indication of treatment of P. N H.

The dose will be 500 milligrams B I D.

One trial will include patients who had an inadequate response to see five inhibitors and the other trial will include patients not currently receiving complement inhibitors, including those naive to these drugs.

William P. Sheridan: I'm pleased to report that we have now reached agreement with the FTA on both the design and the endpoints for our P&H pivotal trials. The two pivotal trials will each test oral BCX 9930 monotherapy and will support the indication for treatment of P&H. The dose will be 500 milligrams BID. One trial will include patients who have had an inadequate response to C5 inhibitors, and the other trial will include patients not currently receiving complement inhibitors, including those naive to these drugs. Patients in both categories need release of anemia, freedom for transfusions, and release of a symptom.

Patients in both categories need relief with anemia freedom from transfusions and relief of symptoms.

The primary endpoint for both pivotal trials agreed with the FDA will be changed from baseline in hemoglobin, a direct clinical measure of relief of anemia.

In both trials, we will measure the impact on need for transfusions as a secondary endpoint and we will also catch on other important outcomes in T. H, such as fatigue schools teenage clone size and laboratory biomarker. So from all of this.

The outstanding results for <unk>, we reported in March with main hemoglobin changed from baseline of three three grams per deciliter in seaside inadequate response patients and three five grams per deciliter in treatment naive patients.

William P. Sheridan: The primary endpoints of both pivotal trials agreed with the FDA will be changed from baseline in hemoglobin, a direct clinical measure of release of anemia. In both trials, we will measure the impact on the needs for transfusions as a secondary endpoint, and we will also capture other important outcomes in TNH, such as fatigue scores, TNH clone size, and laboratory biomarkers of Femolysis. The outstanding results we reported in March, with mean hemoglobin changed from baseline of 3.3 grams per deciliter in C5 inadequate response patients and 3.5 grams per deciliter in treatment naive patients, position BCX-930 very well for success in our pivotal trial.

<unk> basically ex 19 on city very well for success in our pivotal trials.

Yeah.

As you heard in March Hematologists and patients with <unk> are very excited by the prospect of a treatment that could dramatically improve outcomes and to eliminate the need for IV infusions.

We are convinced that success in our pivotal studies will drive a paradigm shifting P&I age towards oral proximal complement inhibition with <unk> 90 930.

So whats next in this program, we are now ready to move directly into the pivotal trials in <unk> in the second half of the year using the designs now agreed with the FDA.

William P. Sheridan: As you heard in March, hematologists and patients with PNH are very excited by the prospect of a treatment that could dramatically improve outcomes and eliminate the needs for IV infusions. We are convinced that success in our pivotal studies will drive a paradigm shift in P&H towards oral proximal complement in addition to BCX9930. So, what's next in this program?

We will also moving to a proof of concept trial on selected nephritis indications in the second half for this year.

Yeah.

These kicks 90 930 represents a pipeline in a molecule. We are very excited to be moving this program. So quickly because we know patients are waiting now I'd like to hand, the call over to Anthony.

Thanks, Phil.

William P. Sheridan: We are now ready to move directly into the pivotal trials in PNH in the second half of this year, using the designs now agreed with the FDA. We will also move into a proof-of-concept trial and selected nephritis indications in the second half of this year. B69930 represents a pipeline in a molecule.

We've continually said that we are focusing our investments where they can drive the greatest value.

With early day on now approved in three key global territories. The commercial team in the U S getting the launch off to a great start on that.

Very positive data that we have a PC X 90, 930, we are executing the strategy.

Anthony J. Doyle: We're very excited to be moving this program so quickly because we know patients are waiting. Now, I'd like to hand the call over to Anthony. Thanks, Bill. We've continually said that we are focusing our investments where they can drive the greatest value. With Orladeo now approved in three key global territories, the commercial team in the US getting the launch off to a great start, and the very positive data that we have with BCX 9930, we are executing this strategy, and we are well positioned for future growth. You can find our detailed financials in today's earnings press release, and I'd like to call your attention to a few items. The net revenue for the quarter was $19.1 million.

We are well positioned for future growth.

Anthony J. Doyle: Of this, $10.9 million came from sales of Orla deo in the U.S. Operating expenses, not including non-cash compensation, for the quarter were 63 million, with the incremental investment from previous quarters focused on the development of BCX 9930. We ended Q1 with $244 million in cash. This cash, in addition to access to the additional 75 million from Ethereum and now revenue from Orlodeo, continues to give us cash runway into 2023. Since this is our first full quarter of Orlidaure revenue, I wanted to take a minute to remind you of our approach and a couple of key items. We recognize revenue when our sole source specialty pharmacy ships Orlando to patients for use. Each shipment contained a 28-day supply of Orlando.

You can find our detailed financials on today's earnings press release, and I'd like to call your attention to a few items.

Net revenue for the quarter was $19 $1 million of this $10 $9 million came from sales of oil a day on the U S. Our operating expenses not including non cash compensation.

For the quarter was $63 million with the incremental investment from previous quarters focused on the development of <unk> 90, 930, we ended Q1 with $244 million on cash. This cash in addition to access the additional $75 million from a theory and now revenue from Orlando continues to give us cash runway into 2020.

Three.

Since this is our first full quarter of all of their revenue I wanted to take a minute to remind you of our approach on a couple of key items.

We recognize revenue on a sole source specialty pharmacy ships on a day or to patients for use each shipment contains a 28 day supply of Orlando.

These are shipments directly from the specialty pharmacy pharmacy to patients. So they are true sales and you will not see any inventory or channel stocking in our revenue numbers.

When we look at growth to Nash the biggest impact at the moment will be driven by non reimburse shipments. Our quick start program has proven to be a real differentiator and is delighting customers and the ease and speed of getting access to or the day, sometimes within 24 hours of a start for them being submitted.

Anthony J. Doyle: These are shipments directly from the specialty pharmacy to patients, so they are true sales, and you will not see any inventory or channel stocking in our revenue numbers. When we look at Gross to Net, the biggest impact at the moment will be driven by non-reimbursed shipments. Our Quick Start program has proven to be a real differentiator and is delighting customers with the ease and speed of getting access to Oridea, sometimes within 24 hours of a start form being submitted.

The quick start program and our patient assistance program. Both resulted in a gross to net adjustment being higher now than it will be once the launches in a more mature phase.

Because gross to net is so fluid early in the launch we are not providing growth to net guidance, but as we continue to progress with the launch you should expect our gross to net adjustment to move in line with other rare disease products.

So what does our strong Q1 mean for future periods.

Anthony J. Doyle: The Quick Start program and our patient assistance program both resulted in the gross tonnet adjustment being higher now than it will be once the launch is in a more mature phase. Because Gross Tenet is so fluid early in the launch, we are not providing Gross to net guidance, but as we continue to progress with the launch, you should expect our Gross to net adjustment to move in line with other rare disease products.

Charlie's team did a great job of converting clinical trial patients, giving us a bolus of patients in the first quarter. Additionally, the team has achieved great success in helping many new patients switch from injectable prophylactic or acute only medications together. This gives us a really strong foundation for future periods.

We have not provided revenue guidance as there are still several variables that will impact the growth trajectory of revenue that we need more time to better understand first what is the steady state of monthly prescriptions, while we've been very encouraged with the numbers to date, we need more time to see it play out.

Anthony J. Doyle: So, what does our strong Q1 mean for future periods? Charlie's team did a great job of converting clinical trial patients, giving us a bolus of patients in the first quarter. Additionally, the team achieved great success in helping many new patients switch from injectable prophylactic or acute-only medications.

Next quarter's customer retention look like over a longer time.

Anthony J. Doyle: Together, this gives us a really strong foundation for future periods. However, we have not provided revenue guidance as there are still several variables that will impact the growth trajectory of revenue that we need more time to better understand. First, what is the steady state of monthly prescriptions? While we've been very encouraged with the numbers to date, we need more time to see it play out. Next, what does customer retention look like over a longer time?

In our clinical trials. This number was about 75% through 48 weeks, telling us that Orla day, it was well tolerated and is providing outstanding attack control.

We believe we will see this level of persistence in the market. It is still too early to confirm that this is the case.

And lastly, what does the trend look like when the vast majority of patients have reimbursement for $10 $9 million.

And net revenue we are reporting is only from patients whose early day of prescriptions are reimbursed as Charlie described we're making good progress getting early day on to policies, but the pace of reimbursement over especially the second quarter will be a key driver in the rate of revenue growth for us over the remainder of the year.

Anthony J. Doyle: In our clinical trials, this number was about 75% through 48 weeks, telling us that Orla deo is well tolerated and is providing outstanding attack control. While we believe we will see this level of persistence in the market, it's still too early to confirm that this is the case. And lastly, what does the trend look like when the vast majority of patients have reimbursement? The $10.9 million in net revenue we're reporting is only from patients whose Orla Dea prescriptions are reimbursed.

While we're early in the launch we are very encouraged by the results to date, we have a lot of work to do on a lot more patients to get this next generation of drug too.

We have a great product in Orlando for a strong patient demand for it and we have the team to execute on making our launch a success and getting us to our peak target of $500 million plus in the coming years with that I'll hand, it back over to Jon.

Anthony J. Doyle: As Charlie described, we're making good progress getting Orla deo onto policies, but the pace of reimbursement, especially in the second quarter, will be a key driver in the rate of revenue growth for us over the remainder of the year. While we're early in the launch, we are very encouraged by the results to date. We have a lot of work to do and a lot more patients to get this next generation of drug, too. But we have a great product in Orledale.

Thanks Anthony.

This is what execution looks like.

We're off to a great start with the launch of oil a day on the U S and starting to launch in other major parts of the world all contributing to what we believe will be a $500 million plus global peak sales product.

Anthony J. Doyle: There is strong patient demand for it, and we have the team to execute on making our launch a success and getting us to our peak target of $500 million plus in the coming years. With that, I'll hand it back over to John. Thanks, Anthony. This is what execution looks like.

Add to that our plan to advance into pivotal studies in <unk> with 90 930 and in parallel moving into other indications with this pipeline in one molecule.

And finally, having the financial flexibility that comes with a strong balance sheet and revenue generation allows us to focus on execution and value creation.

Jon P. Stonehouse: We're off to a great start with the launch of Orledeo in the U.S. and starting to launch it in other major parts of the world, all contributing to what we believe will be a $500 million-plus global peak sales product. Add to that our plan to advance into pivotal studies in P&H with 9930 and, in parallel, move into other indications with this pipeline in one molecule. And finally, having the financial flexibility that comes with a strong balance sheet and revenue generation allows us to focus on execution and value creation. The evidence is clear that Biocrys is transforming into a company with product revenue, a full pipeline, and a discovery engine that produces these compounds and will continue to produce more.

The evidence is clear that biocryst is transforming into a company with product revenue for.

Our pipeline in a discovery engine that produce these compounds and we will continue to produce more.

That concludes our prepared remarks, we'll now open it up for your questions.

Thank you and as a reminder, if you would like to ask a question. Please press Star then the number one on your telephone keypad once again Thats star one on your telephone keypad. If you would like to withdraw your question press the pound key.

<unk>.

Our first question comes from the line of Jessica Fye of Jpmorgan. Your line is open.

Operator: That concludes our prepared remarks. We'll now open it up for your question. Thank you. And as a reminder, if you would like to ask a question, please press star, then the number one on your telephone keypad. Once again, that's star one on your telephone keypad. If you would like to withdraw a question, please press the found key. Thank you. Our first question comes from the line of Jessica 5 of J.P. Morgan. Your line is open.

Hey, guys. Good morning, congratulations on a strong quarter.

I was curious if you could add some more color about specifically which agents.

Patients switching on to Orla switching from.

How do you want to take the yes, hey, good morning, Jeff. Thanks. Thanks for the question, we're seeing patients switch from all the different therapies really in proportion to what you'd expect from there.

Their market share so as I mentioned more than more than half. The patients are switching from injectable proceeds and that means tax iroh haegarda cinryze proportion to their market share.

Jessica Macomber Fye: Hey guys, good morning. Congratulations on a strong quarter. I was curious if you could add some more color about specifically which agents you see the patients switching on to Orla-AO switching from. Hell, do you want to pick that?

Okay. Thanks.

Unknown Executive: Heather, you want to take that? Yeah, hey, good morning, Jess.

You mentioned that from scripts were not reimbursed in the quarter can you say what proportion of first quarter scripts were not reimbursed.

Unknown Executive: Thanks for the question. We're seeing patients switch from all the different therapies, really in proportion to what you'd expect from their market share. So, as I mentioned, more than half the patients are switching from injectable prostheses, and that means tax Iro, Higarta, Sinrise, in proportion to their market share.

Yes, we haven't we haven't commented on the specific details, but as Anthony mentioned in his comments, what we're what we're doing with nearly all patients is starting off on our quick start program and then we work with patients and providers to go through prior authorization and in Q1, most patients get access through medical exception.

Unknown Executive: Thanks. You mentioned that some scripts were not reimbursed in the quarter.

<unk>.

Unknown Executive: reimbursed in the quarter. Can you say what proportion of first quarter scripts were not reimbursed?

But we're making great progress with payers and we expect to make continued progress through mid year and Jess Charlie made comments in his prepared remarks around the more policies that are in place where all of the day is on formulary, but the more interest there is going to be by both doctors and physicians.

Unknown Executive: Yeah, we haven't commented on the specific details, but as Anthony mentioned in his comments, what we're doing with nearly all patients is starting them off in our Quick Start program, and then we work with patients and providers to go through prior authorization. And in Q1, most patients got access through medical exception. But we're making great progress with payers, and we expect to make continued progress through mid-year. And Jess and Charlie made comments and their prepared remarks around, you know, the more policies that are in place where or the deoson formulary, the more interest there's going to be by both doctors and physicians.

Got it and just a last one on question you mentioned that longer term that will move in line with other rare disease drugs. So can you just characterize what typical gross to net are in rare disease.

Yes from a from a gross on a per.

Perspective, what we'd be looking at getting us kind of into the teens 20 type type of frame.

Great. Thank you.

Welcome.

Unknown Executive: Got it. And just the last one on growth to nest. You mentioned that, longer term, that'll move in line with other rare disease drugs. So can you just characterize what typical growth to nets are in rare disease? Yeah, from a gross enough

Thank you.

Question comes from the line of lease vehicles of Evercore ISI. Your line is open.

And then wanted to add my congratulations on a great quarter I'm, just I'm going to turn to the Pn study for a moment.

Unknown Executive: Yeah, from a gross and perspective, what we'd be looking at getting is kind of into the teens and 20 type of frame. Great, thank you. Welcome. Thank you. The next question comes from the line of Lisa Beico of Evercore ISI. Your line is open.

Can you maybe.

Yes.

Sort of control.

We will look like in both of the study.

Surely Olympic net.

Sure.

Hi, Lisa Thanks for the question.

Super excited about moving into these studies of course, and one study will be a superiority trial against the <unk> inhibitors.

Lisa Beico: And I wanted to add my congratulations on a great quarter. I'm just a minute; I'm going to turn to the PNH studies for a moment.

In patients who have made adequate response and the other study in patients who are not taking say five inhibitor.

Lisa Beico: Can you maybe discuss what sort of control arms will look like in both of them?

The control group will be placebo.

William P. Sheridan: Sure. Hi Lisa.

Okay and.

And can you maybe describe the.

The.

I guess, how youll be youll.

You'll be looking at the pure to see five will you be looking at combination therapy then for.

William P. Sheridan: Thanks for the question. I'm super excited about moving into these studies, of course, and one study will be a superiority trial against F5 inhibitors in patients who have an inadequate response, and the other study in patients who are not taking C5 inhibitors, the control group will be placebo.

For the patients that are.

Inadequate responders.

Moving on to monotherapy, how will that work for the inadequate response that's growth.

Sure Bose.

Studies are designed as tests for <unk> 90, 930 mono therapy.

William P. Sheridan: Okay, I see. And can you maybe describe the, um, the, uh, I guess how you'll be, you'll be looking at Superior to C5? Will you be looking at combinations?

For.

Patients will be randomized to <unk> in the C. Five inhibitor trial the randomized to continue their current treatment for.

William P. Sheridan: therapy, then, for the patients that are inadequate responders,

The 699 city mono therapy and discontinue their current treatment.

William P. Sheridan: Responders, or will people be moving on to monotherapy? How will that work for the inadequate response?

So this is the goal here is a label for monotherapy.

With a broad indication to treat.

William P. Sheridan: Sure. Both studies are designed as tests of BCX 9930 monotherapy. So, you know, patients will be randomized to, in the C5 inhibitor trial, be randomized to continue their current treatment or start BCX 9930 monotherapy and discontinue their current treatment. So this is, you know, the goal here is a label for monotherapy with a broad indication to treat patients with TNH. But the reason for the study designs, as we've just outlined, is to support that type of labeling.

Patients with <unk>.

The reason for those study designs as we've just outlined.

Support that.

Globally.

Okay, and how do patients go on to mono therapy from from being on the C. Five how does that work or is it just.

Penetration rates for their combination therapy for all of our network.

No.

The C five inhibitor has stopped.

The whole, Georgia Southern city stood at just a simple switch.

Okay.

We haven't seen the data yet in that population in terms of being on monotherapy can you give us a sense of when we might see that in your phase one phase two.

William P. Sheridan: Okay, and how do patients go on to my treatment?

William P. Sheridan: onto monotherapy from being on a C5. How does that work? Kind of a cold turkey switch, or is there a combination therapy and then a withdrawal? No.

Sure.

The protocol doesn't specify for particular.

Timeline.

So it's a physician judgment call that we expect that we'll be able to share data at the end of the year right.

William P. Sheridan: No, it's the

William P. Sheridan: The C5 inhibitor has stopped, and the oral drug has been started; 9930 started. It's a simple switch.

<unk>, nor us waiting for that data in order to start the study.

Because we are very confident in the drug on the basis of the monotherapy results from the naive patient population the divisions the disease.

William P. Sheridan: Okay. We haven't seen the data yet in terms of that population being on monotherapy.

William P. Sheridan: Can you give us a sense of what your phase one and phase two are like? Sure.

The effective day targets the same.

William P. Sheridan: Sure, you know, the protocol doesn't specify a particular timeline, and it's a physician judgment call, but we expect that we'd be able to share data at the end of the year. Neither the regulators nor us are waiting for that data in order to start the studies because we're very confident in the drug on the basis of the monotherapy results in the naive patient population. You know, the disease is the disease, the factor D targets the same, the dose is the same, so it's not necessary for us to see that data before we start the study.

The doses for <unk>.

Sure.

It's not necessary for us to see that 30 day before we start with COVID-19.

Okay.

Good and then I guess, just turning to Orla tail really great out of the day.

Is there any kind of additional color you can provide on either.

On the percentage of patients that were on your quick start program.

Receiving drug that way the percentage of prescriptions like an or any color on persistent goes on obviously the key.

Questions.

To help guide us as we think about the rest of the year launching off of this really great start.

William P. Sheridan: Okay, good. And then I guess just turning to Orla Deo, really great out of the gates. Is there any kind of additional call you can provide on either, you know, the percentage of patients that were on your Click Start program receiving the drug that way, the percentage of prescriptions, like, and or any color on persistence? Those are obviously the key questions to help guide us as we think about, you know, the rest of the year launching off of this. you know, a really great start. So I'm just hoping you can provide some additional color there. And that's my final question. Thank you.

Just hoping you can provide some additional color there and that's my final question. Thank you.

Sure Lisa so so on the percentage of quick start so in Q1, nearly all patients started on on quick start and Thats part of our strategy.

And as we work with them to get access and as I mentioned, most patients Scott <unk> got reimbursement through medical exception.

They were not able we were not able to do that in Q1 they'll continue on free good Intel the payers establish policies.

And then as far as your question about persistence as Anthony mentioned, we expect we had great results on our in our clinical trials was about 75% of patients staying on for at least a year. That's what we expected in the real world. So far the patient experience has been great, but it's too early to say what the long term persistence rate will be.

Unknown Executive: Sure, so on the percentage of Quick Start. So in Q1, nearly all patients started on Quick Start, and that's part of our strategy as we work with them to get access. And as I mentioned, most patients got reimbursement through medical exception. If we were not able to do that in Q1, they'll continue on free goods until the payer's established policies. And then as far as your question about persistence, as Anthony mentioned, we expect we'll have great results in our clinical trials with about 75% of patients staying on for at least a year. That's what we expected in the real world. So far, the patient experience has been great, but it's too early to say what the long-term persistence rate will be.

Okay and that patient quick start program is about is it on monthly prescription until didnt get on.

That's right.

It's a month and if there is not reimbursement in a month, where we will continue from either on quick start or on our patient assistance program.

Okay, I guess that just leads me to one final question approximately how long is it.

The gain on sale.

From our.

Reimburse drop at this point.

Unknown Executive: Okay, and that patient Quickstar program, is it a monthly prescription until you get on it?

So in Q1, it's all over the board everything from a few days to patients still being on the program and by mid year, we expect that to really stabilize as the great majority of patients will have access to coverage through their plants, yeah and Lisa the theme here is that as the year progresses, we will have more and more people on paid drug and fewer and fewer on.

Unknown Executive: Reimbursed, right? That's right. It's a month, and if there's not reimbursement in a month, we will continue them either on Quick Start or on our patient assistance program.

Unknown Executive: Okay, I guess that just leaves me with one final question. Approximately how long is it taking to get onto commercial, you know, reimbursed? In Kiwan, it's all over the board.

Quick start.

That's the plan.

Okay, alright, thanks, a lot going on.

Yeah.

Unknown Executive: So in Q1, it's all over the board, everything from a few days to, you know, patients still being on the program. And by mid-year, we expect that to really stabilize, as the great majority of patients will have access to coverage through their plans. Yeah, and Lisa, the theme here is that as the year progresses, we will have more and more people on paid drugs and fewer and fewer on Quick Start. That's the plan.

Thank you.

Thank you next question comes from the line of Ken Cacciatore from Cowen and co. Your line is open.

Hey, guys. Congratulations on the early progress just wondering.

Hear really good things about the interactions on both the clinician.

Clinician and patient level be it your hubs. So just wondering as it seems to be ramping a little bit faster do you feel you're staffed appropriately can you talk about some of the early learnings from this really kind of a more personalized approach and things that.

Unknown Executive: Okay. All right. Thanks a lot, guys. Thank you. Thank you. The next question comes from the line of Ken Kachatori, from Cohen & Co. Your line is open.

As things are getting underway that you may be changing or again, if you're staffing a little bit more than on EU.

Ken Kachatori: Hey, guys, congratulations on the early progress. Just wondering, we hear really good things about the interactions on both the clinician and patient level via your hub. So just wondering, as it seems to be ramping a little bit faster, do you feel you are staffed appropriately? Can you talk about some of the early learning?

Just wondering it looks like a potential really focus selling effort can you talk about where these patients are domiciled as it can be fairly easy to get at them fairly difficult maybe some of the learnings from the Injectables I know you feel and we feel as well that things should go better there, but maybe a little bit more nuance around.

Unknown Executive: from this really kind of more personalized approach and things that, as things are getting underway, that you're maybe changing, or again, if you're staffing a little bit more, then on EU, just wondering, it looks like a potential really focused selling effort. Do you talk about where these patients are domiciled? Is it going to be fairly easy to get at them, fairly difficult?

Europe, and then lastly, maybe for Bill I know, it's really early just announcing you're going to start these pivotal for <unk>.

A shot at timing of results.

Maybe a little bit of nuance here in terms of the kind of competitive landscape to enroll patients, but any kind of shot at when we might expect to see data would be fantastic. Thank you.

Unknown Executive: Maybe some of the learnings from the injectables. I know you feel, and we feel as well, that things should go better there, but maybe a little bit more nuance around Europe. And then lastly, maybe for Bill, I know it's really early just announcing you're going to start these pivotals, but if you take a shot at the timing of results, maybe a little bit of nuance here in terms of the kind of competitive landscape to enroll patients, but any kind of shot at when we might expect to see data would be fantastic. Thank you.

Great. Thanks for the questions Ken.

The first question you had just about our specialty pharmacy patient services program in staffing.

One of the great things about having a sole sources, we really prepared for the right staffing and then Alan Hodge and his team have done a great job working with ESP to pivot as we see this demand from patients so increasing staffing appropriately increasing procedures.

Unknown Executive: Great, thanks for the questions, Ken. So the first question you had was about our specialty pharmacy, patient services program, and staffing. One of the great things about having a sole source is that we are really prepared for the right staffing, and then Alan Hodge and his team have done a great job working with the SP to pivot as we see this demand from patients. So increasing staffing appropriately, increasing procedures, they're really quick in making those changes to serve the patients.

They're really quick and making those those changes to serve the patients well and it's making a difference.

As far as the EU really good question about access to patients and how easy or difficult is that.

Unknown Executive: patients well, and it's making a difference. As far as the EU is concerned, a really good question about access to patients and how easier or difficult that is. The great thing about the EU is that treatment centers are very concentrated, so you can literally have hundreds of patients within a given center.

Unknown Executive: And so what our teams are doing is working with the HAA experts at those centers to make them aware that Orlodeo is coming and to reach out to their patients and bring them into the centers. So we're really enthusiastic about the opportunity there. And Ken, I'd add that, you know, Bioschrist is a known entity to these docs that treat H.A.E. in Europe.

Unknown Executive: I mean, we've been doing clinical trials all the way back to our first generation program, and so we've got fantastic relationships. And they know the company. They've been involved in our trials, and so the excitement level is high. And it may be in building the, yeah, go ahead. Yeah, sure. So Ken, what a great question. When will the studies finish?

Unknown Executive: I wish I could be specific and give you a prediction. I think it's really difficult at this stage, and we need to get them up and running. What I can tell you is that we have a great clinical execution team here at Birochrist, and we are going to approach this on the basis that patients really need this drug. It's going to be a major advance in P&H, that's what we believe. And so we'll be ramping up as fast as possible, and just like in H.A., we'll go to the best centers all around the world to get these studies done. And just like H.A.E.

The patient getting patients on clinical trials is a competitive activity and that we believe we have a a great offering here with on our effective day into but let's say, we look forward to seeing the study.

Study start on the accrual moving and they will they will be in a bit of position too.

Like an estimate appointment on finish.

Alright. Thanks again can I did yeah, just add it's it's hard to say because we just don't know what the rate of enrollment it'll be right in a competitive space and and you know much larger study than what we've studied so far on P and H. It just super hard to predict but one thing I will say is bill's team is.

Unknown Executive: In most rare diseases, getting patients on clinical trials is a competitive activity, and we believe we have a great offering here with an oral factor D. We look forward to seeing the study start and the accrual moving, and then we'll be in a better position to make an estimate of when they might finish. Can I just add that it's hard to say because we just don't know what the rate of enrollment will be in a competitive space in a, you know, much larger study than what we've studied so far in P&H. It's just super hard to predict, but one thing I will say is Bill's team is excellent, and their aim is to be the sponsor of choice.

Excellent and their aim is to be the sponsor of choice and this is where big doesn't necessarily help you small companies that pay attention that listen that give the extra TLC make a difference in enrollment and so I am super confident that bells those team will do that.

Very helpful.

Thank you next question comes from the line of search Bellinger from he'd come in company. Your line is open.

Unknown Executive: And this is where big doesn't necessarily help you. Small companies that pay attention, that listen, that give the extra TLC make a difference in enrollment.

Hi, good morning.

Thanks for taking my questions.

Few on Aurulent Dale Firstly apologies if you cover this before.

Did you just call the number of patients that were on drugs a day.

The first quarter and I guess, how many of them for patients transitioning from clinical trials or the.

Unknown Executive: And so I am super confident that those people will do that. Very helpful. Thank you. Next question comes from the line of Serge Ballinger from Needham and Company. Your line is open.

Access programs.

Hey search good morning that we have not disclosed the detail on that and what I can say is the number of patients on rodeo continues to grow every day is patient switch to Orlando.

Serge D. Belanger: Hey, good morning, and thanks for taking my questions. A few on Aura Lideo. First, apologies if you covered this before. Did you disclose the number of patients that were on the drug at the end of the first quarter, and I guess how many of them are patients transitioning from clinical trials or the early access programs?

Q1, what we did say is that the majority of patients by the end of the quarter, where those new patients switching and then.

Unknown Executive: Hey, Sirich, good morning. We have not disclosed the details on that. What I can say is the number of patients on Orlydeo continues to grow every day as patients switch to Orleao. In Q1, what we did say was that the majority of patients by the end of the quarter were new patients switching, and then to Orideo, and then the rest of them were those transitioning from our clinical trials and EAP. And that transition program finished in Q1.

Unknown Executive: And so that bolus that Anthony talked about is complete, and then everything going forward is growth as patients switch to early day. Yeah, and just one point of clarification on that: complete means that they're switched from clinical trials to Quick Start or patient assistants. They have to go through the same process of reimbursement like any other patient. And so some have been reimbursed, some have not.

Unknown Executive: Okay, and I think you talked about Position based on about 500, serving about 50% of

Unknown Executive: of the H.E. How big is the next set of positions serving the other 15% of the population?

Unknown Executive: need to address. Sir, there are easily another thousand-plus physicians out there, and we're reaching them as well. We're really concentrated on that top 500 just because they are, you know, they have more patients and they really know H.E. So that's our priority, but we're reaching other doctors as well, and we're getting prescribing from them too. And, Serge, I'd add, you know, what makes this achievement in the first quarter with the sales that Charlie and his team have generated even more remarkable is that they did it in COVID, right? And so, you know, we're already seeing things starting to open up with vaccination. That's only going to get better as the course of the year goes on.

Unknown Executive: Okay, and then while we're on COVID, you know, what has been the overall impact with restrictions, or are you seeing limitations on the number of patient switches, and is that something that you expect will increase as restrictions lift?

It's something that many physicians have been comfortable prescribing oral a day out.

Unknown Executive: I think first of all, surge, as John was talking about, COVID limited our in-person interaction with providers; fewer than 50% of our calls were in-person. That's going to make a big difference as we're able to do more in-person visits. And we had a great quarter despite all of this.

In a remote environment to their patients.

With more in person visits, though it's just it's more opportunity and we're excited for what's to come.

Great. Thank you congrats on the progress.

Thanks, so much.

Thank you next question comes from the line of smaller very cross of <unk>.

Unknown Executive: Another piece is that oral Adio is easy for doctors to prescribe. So it's something that many physicians have been comfortable prescribing oral Adio in a remote environment to their patients. Patients. With more in-person visits, though, it's just more opportunity, and we're excited for what's to come.

For you your line is open.

Hi, This is Kenny Chen on for Maury Raycroft I have two questions one on Orla Dale.

The progress on the formulary adoption versus.

Reimbursement by medical exception have you encountered any pushback on the cost effectiveness from insurances and.

Unknown Executive: Great. Thank you for us on the progress.

How are the reimbursement conversations going on reinsurance is looking at ICI reports.

Unknown Executive: Thank you. The next question comes from the line of Moire aircraft. Of Jeffries, your line is open. Hi, this is Kenny Chan on behalf of Moray-Croft. I have two questions, one on Orla Deo.

And for the second question on the.

The <unk> program.

As the FDA feedback on LDH levels and is there a specified.

Unknown Executive: How's the progress on the formulary adoption versus reimbursement by medical exception? Have you encountered any pushback on the cost effectiveness from insurances? How are the reimbursement conversations going? Are insurance companies looking at ISA reports? And for the second question on the P&H program, what was the FDA feedback on LDH levels, and is there a specified LDH threshold that would trigger a safety concern? Fairly, you want to...

Threshold that would trigger a safety concern.

For the Olympic Yes, thanks, Thanks, Kenny as far as the payer progress for policy progress as we said in our comments, it's going really well.

In Q1, it was mainly medical exception that we were getting access, but several payers and pbms put oil a day on on formulary and we accept we expect a lot of acceleration of that over the over Q2.

Cost effectiveness no. This is the lowest price protein on the market.

Unknown Executive: Yes, thanks, Kenny. As far as payer progress, policy progress, as we said in our comments, it's going really well. In Q1, it was mainly medical exception that we were getting access, but several payers and PBMs put Orlydeo on formulary, and we expect a lot of acceleration of that over Q2. Cost-effectiveness is, no, this is the lowest price profile on the market, and payers have reacted really well to our pricing strategy as well as the profile of the drug.

And payers have reacted really well to our pricing strategy as well as the profile of the drug and then as they see the demand coming from patients switching to oral a day Oh, it's really encouraging.

Payers to establish coverage policies quickly. So we're very pleased with where we are and let me stress one point I made in the prepared remarks. This drug works right.

People are switching that are controlled on appropriate therapy to our drug and they are staying on our drug. So this idea of effectiveness. This drug works I, just really want to stress that.

Unknown Executive: And then, as they see the demand coming from patients switching to Oraleo, it's really encouraging payers to establish coverage policies quickly. So we're very pleased with where we are. And let me stress one point I made in my prepared remarks. This drug works, right? People that are controlled on prophy therapy are switching to our drug, and they're staying on our drug. So this idea of effectiveness this drug works. I just really want to stress that.

Bill do you want to take the FDA feedback on LDH.

Sure.

Thanks for the question.

Do have a secondary endpoint.

Vintage is changed from baseline in LDH.

There is no threshold, either as an endpoint or as a safety concerns for that matter.

Sure.

There'll be a useful biomarker.

Improvement in anemia, with the change from baseline in hemoglobin hemoglobin going up and the improvement in transfusion burdened with transfusion avoidance being of course, the most important number.

Unknown Executive: Bill, do you want to take the FDA feedback on LDH? Sure, Kenny, thanks for the question. We do have a secondary endpoint of percentage change from baseline in LDH. There is no threshold either as an endpoint or as a safety concern, so that matters.

Transfusion going down.

Very important clinical outcomes.

It's also worth stressing that another very important secondary endpoint.

William P. Sheridan: So, LBH is a useful biomarker, the improvement in anemia with the change from baseline in hemoglobin, hemoglobin going up, and the improvement in transfusion burdened with transfusion avoidance being, of course, the most important, but the number of transfusions going down. A very important clinical lab. You know, we're also stressing that another very important secondary end point is the evaluation of quality of life with tools such as the Sasset fatigue score.

<unk>.

Valuation of quality of life with tools, such as the assessment to take school, so measuring fatigue is important too.

And bill.

You might want to just talk about the interaction with the FDA and that it was focused on clinical benefit biomarkers that are important but clinical benefits more important.

Right so.

Regulators of course want to see clinical outcomes being treated as important endpoints in clinical trials.

That's why we have changed from baseline hemoglobin as the primary endpoint and transfusion avoidance as the key secondary endpoint.

William P. Sheridan: So measuring fatigue is important too. And Bill, you might want to just talk about the interaction with the FDA and how it was focused on clinical benefits, right? Biomarkers are important, but clinical benefits are more important. Right, you know, so the regulators, of course, want to see clinical outcomes being treated as important endpoints in clinical trials. So that's why we have changed from baseline hemoglobin as the primary endpoint and transfusion avoidance as a key secondary endpoint.

That's really the linchpin of evaluating a treatment of anemia.

So there's a whole range of interesting biomarker as we can into Michigan.

So the <unk> is not viewed as important enough to be a primary endpoint, it's really very simple.

Thanks.

Thank you once again, if you would like to ask a question. Please press Star then the number one on your telephone keypad.

William P. Sheridan: And, you know, that's a little linchpin of evaluating a treatment for anemia after all. So, you know, there's a whole range of interesting biomarkers we're going to measure. But OVH is not viewed as important enough to be a primary input. It's really very simple.

Next question comes from the line of Brian Abrahams of RBC capital markets. Your line is open.

Hey, good morning, Thanks for taking my questions and congrats as well on the early launch.

My first question is on the launch I was wondering if you could maybe parse out the pent up demand for patients new to Orla day, or I guess I'm wondering how long into the year might you expect first quarters rate of switching from existing acute or prophylactic therapies to continue.

William P. Sheridan: Thank you. Once again, if you would like to ask a question, please press Star, then the number one on your telephone keypad. The next question comes from the line of Brian Abrams of RBC Capital Markets. Your line is open.

Hey, Brian just trial and good good good question and yes, there was pent up demand and we expected that.

Brian Corey Abrahams: Hey, good morning, thanks for taking my questions and congratulations as well on the early launch. My first question is about the launch. I was wondering if you could maybe parse out the pent-up demand for patients new to Orlodeo. I guess I'm wondering how long into the year might you expect the first quarter's rate of switching from existing acuter prophylactic therapies to continue? Hey, Brian's Charlie, good question.

There are always early adopters, what we're really pleased about is the demand is continuing so we're seeing patients switch to early day literally every day. So and then as I mentioned in my remarks, there's a lot of opportunity to come as all of these physicians, who we've reached but haven't yet prescribed theyre, telling us that they are planning to have the <unk>.

<unk> with their patients and prescribed in the future. So a lot of opportunity to come throughout the year.

Unknown Executive: And, yeah, there was pent-up demand, and we expected that. So they're always early adopters. What we're really pleased about is the demand is continuing, so we're seeing patients switch to Erle deo literally every day. And then, as I mentioned in my remarks, there's a lot of opportunity to come, as all these physicians who we've reached but haven't yet prescribed. They're telling us that they're planning to have the conversation with their patients and prescribe them in the future. So there is a lot of opportunity for them to come throughout the year.

Yeah. We don't think this is just.

On one quarter pop and then it's going to Peter out Brian that we expect that this is going to continue to be strong through the course of the year and we said, it's a $500 million plus global peak sales product and we're even more confident in that.

Unknown Executive: Yeah, we don't think this is just a, you know, one-quarter pop, and then it's going to peter out, Brian. We expect that this is going to continue to be strong through the course of the year, and we've said it's a $500 million-plus global peak sales product, and we're even more confident in that. Got it, that's really helpful. And then I'm curious what sort of feedback you're getting on how real-world efficacy, safety, and tolerability are comparing to the clinical trial setting.

Unknown Executive: Any surprises there? And then, I know it's early days, but I'm curious what you're seeing with respect to persistence and compliance for new patients to therapy versus the rollovers from the expanded access and quick start programs. Yeah, I'll take the first part, Charlie; if you could take the second, that'd be great.

Unknown Executive: So I'm smiling as you asked that question because the doctors and patients told us that these drugs do way better in the real world than they do in clinical trials. And so I think our clinical trials don't really represent the real benefit that we're seeing from patients. Megan mentioned that, you know, we're looking at the data from Apex 2, you know, out to now 96 weeks, and it just keeps looking better, right?

Chose to continue on early day on the commercial world at because they are having such a great experience in the early word back from patients. So we hear back from our specialty pharmacy, and we hear back through physicians. The Ah reps. The early feedback is very strong patients newly switching to oral a day or having a great experiences as.

Unknown Executive: And so, you know, I'll say it again, this drug works. People are getting a real benefit. They're getting control of their disease. And, oh, by the way, they're doing it on one capsule once a day. So it's just a huge, huge benefit.

Unknown Executive: And then Brian, your question about the experience of rollovers versus new patients. The great majority of the patients who are in our clinical trials chose to continue on Orlydeo in the commercial world because they're having such a great experience. And the early word back from patients, and we hear back from our specialty pharmacy, and we hear back from physicians via our reps, the early feedback is very strong. Patients newly switching to Orla Deo are having a great experience. as we expected. So, we're pleased, and to my point earlier, we expect more patients to continue to switch to Oralideo based on what they hear from their peers about their experience.

We expected. So we're we're pleased and and my point earlier, we we expect more patients to continue to switch to early down.

Based on what they hear from their peers about their experience.

Great. Thanks, so much.

You're welcome.

Q next question comes from the lines Enamoured of Bank of America, you're in 97.

Hi, Good morning day, Thanks for taking my questions.

I don't really Danielle I wasn't really too.

I guess treatment naive patient.

And what is your latest market data, telling you about the percentage of patients.

K E who are currently not on any therapy.

Unknown Executive: Great, thanks so much. You're welcome. Thank you. The next question comes from the line of Tessin Ahmed of Bank of America. You're on the line is open.

And related to that I'm, just wondering do you think.

COVID-19 starts to clear and more offices start to reopen at the percentage way of new patients or kitten. The naive patients that are receiving scripts will normalize more relative to what you're seeing in the early stages of for lunch and then I have a couple of follow ups.

Tazeen Ahmad: Hi, good morning, guys, thanks for taking my questions. On Oradeo, as it relates to, I guess, treatment-naive patients, what is your latest market data telling you about the percentage of patients who are having to

Unknown Executive: who are currently not on any therapy. And related to that, I'm just wondering, do you think, once COVID starts to clear and more offices start to reopen, that the percentage of weight of new patients or treatment I use patients that are receiving scripts will normalize more relative to what you're seeing in the early stages of the launch, and then I have a couple of questions that follow it.

See I think I think I've got your question, but let me let me address it in a couple of different parts.

What we saw prior to the launch of Orla day, though it's about 60% of patients are on prophylaxis and so in the first quarter, we're seeing more than half of the patients coming to early day I was switching from those other profi products.

Just a the remainder of patients switching to Orla day or switching from you only treatment. So they may be naive to prophylaxis, but they've been treated with acute only.

Unknown Executive: See, I think I've got your question, but let me address it in a couple of different ways. What we saw prior to the launch of Oradeo is that about 60% of patients are on prophylaxis. And so in the first quarter, we're seeing more than half of the patients coming to Oralideo switching from those other profi products. Most of the remainder of patients switching to Oralideo are switching from acute-only treatment, so they may be naive to prophylaxis, but they've been treated with acute only.

Our research tells us that the number of truly itchy treatment naive patience, they're not on acute not on Profi is small.

That's a small opportunity in the in the future, but but most of these patients are treated well by their doctors. They have at least an acute therapy and those patients are deciding to switch to profi now that they can do it with an oral once daily option. So this is Ah I can't stress. This enough. This is a switch strategy and it's working right.

Unknown Executive: Our research tells us that the number of truly H-A-E treatment naive patients, you know, they're not on acute, not on profi, is small. That's a small opportunity in the future, but most of these patients are treated well by their doctors. They have at least an acute therapy, and those patients are deciding to switch to profi now that they can do it with an oral once daily option. So this is a, I can't stress this enough, this is a switch strategy, and it's working, right?

That is if you walk away with anything today walk away with this as a marketplace where people are on profi therapy, and acute on demand therapy, and they're switching to an oral drop right. So that's what we we saw that in the market research. We saw it in the clinical trials now is playing out in the marketplace, which is great and it's only the first quarter.

Into your point about COVID-19 I think there's opportunity here I think access of our reps to Dax has been challenging in some parts of the country. They have been really villages and resourceful.

Unknown Executive: That is, if you walk away with anything today, walk away with this: this is a marketplace where people are on prophytherapy and acute on-demand therapy, and they're switching to an oral drop, right? So that's what we saw in the market research, we saw it in the clinical trials, and now it's playing out in the marketplace, which is great. And it's only the first quarter.

And they produce a great result in the first quarter, but I think as things open up board is going to get better and I think the same goes for dachshund patients right.

It's just been harder it's been telemedicine, maybe they are putting off their visit and so I think that gets better as more people get vaccinated and more people start to go see the doctor.

Unknown Executive: And to your point about COVID, I think there's opportunity here, right? I think, you know, access for our reps to docs has been challenging in some parts of the country. Yeah, they've been really diligent and resourceful, and they produced a great result in the first quarter, but I think as things open up more, it's going to get better.

Okay. Thanks for that color, Jon and then furniture Pan I know, it's very very early in the launch but in terms of the trajectory what to expect it to be similar to what we would expect a day in the U S.

Unknown Executive: And I think the same goes for docs and patients, right? It's just been harder. It's been telemedicine. Maybe they're putting off their visit. And so I think that will get better as more people get vaccinated and more people start to go see their doctor. Okay, thank you.

And then do you have any visibility on what typing in Japan.

Making me want to take then.

Sure sure so on the trajectory to be made I think.

Unknown Executive: Okay, thanks to that caller, John. And then for Japan, I know it's just very, very early in the launch, but in terms of the trajectory, which you expect it to be similar to what we would expect to see in the U.S., and do you have any visibility on what pricing in Japan is?

And sharing in Japan is a very different market in the U S and Europe and as a decade behind and so we're absolutely thrilled about the opportunity that Tori has to really build in shape. It. So I think our our trajectory in the near term is more tempered we need also to Japan is in a state of emergency currently with.

COVID-19 and.

Unknown Executive: Sure, so on the trajectories to Vime, I think, as we've been sharing, Japan is a very different market.

Ah different rollout with a vaccine for that May also.

Slow that initial ramp, but as I share to my remarks, we see this is a really strong alone return potential is tori uhm drive the adoption of profit, but also the expansion on the market through for more diagnose within treatment.

Unknown Executive: The U.S. and Europe are a decade behind, and so we're absolutely thrilled about the opportunity that Tori has to really build and shape it. So I think our trajectory in the near term is more tempered. Also, too, Japan is in a state of emergency with COVID and a different rollout with the vaccine. So that may also slow that initial ramp. But as I shared in my remarks, we see this as a really strong long-term potential, as Tori drives the adoption of Prophi but also the expansion of the market through more diagnosis and treatment. And then to your second question, with our pricing negotiations completing last week, last month, excuse me, this week.

Uhm and then into your second question.

With our pricing negotiations completing last week last month excuse me, we net it out with a price on around 250000 U S dollars per patient per year and for asking for really proud and excited about that price point and and the opportunity it represents for patients and for Tori. So.

All in all their their equally excited to now be advocates like the U S has been for the last few months and we're excited to see what day they can deliver.

Okay. Thank you and then maybe just one question if I could on on T N H.

Unknown Executive: netted out at a price of around 250,000 US dollars per patient per year.

Two trials at your running I know, it's difficult T T.

Unknown Executive: per year. And for us, we were really proud and excited about that price point and the opportunity it represents for patients and for Tori. So all in all, they're equally excited to now be out of the gates like the U.S. has been for the last few months, and we're excited to see what they do.

A gas on how long, it's gonna take to enroll but for.

For the 19th Birthdays see placebo inadequate responder trial excuse me would you expect both to read out at around the same time or do you think one could enroll faster than the other.

Oh, you on for either of those for sure so hard to say.

Unknown Executive: Okay, thank you. And then maybe just one question, if I could, on PNH. So the two trials that you're running, I know it's difficult to, you know, take a guess on how long it's going to take to enroll, but for the naive versus the placebo, the inadequate responder trial,

It's very difficult to say, if you search either of those possibilities could come to pass for it but it could read up personally at the same time or one could move much quicker than the other I think it's too early tomorrow.

Okay. Thank you.

Unknown Executive: Excuse me, would you expect both to read out at around the same time, or do you think one could enroll faster?

Thanks disease.

Thank you next question comes from the lineup channel wrong.

Barclays share line, you tube and.

Unknown Executive: Bill, you want to take that one? Yes, sure. Hi Tizene.

Hi, Good morning. This is Shaun <unk> on for Gina. So just one question on on P. N H.

Unknown Executive: It's very difficult to say at this stage, and either of those possibilities could come to pass, right? They could read out at approximately the same time, or one could move much quicker than the other. I think it's too early to know.

So for me can you tell us on <unk> or what the size of the price could be for these please the and then like what specific P. K P. D day towers look pet to go ahead with the 500, maybe idea dosing, what's the closest 400.

Unknown Executive: Thank you. The next question comes from the line of Channel Wong. Off Barclays, your line is open.

Mmm. Thanks for your questions. So we're going to resist additional details about the studies for later this year.

Channel Wong: Hey, good morning. This is Swapnil on behalf of Gina. So just one question on P&H. Can you tell us about what the size of the trials could be for this phase three and then, like, what specific PKPD data was looked at to go ahead with the 500 microgram BID dose versus versus 400.

Might be at a medical Bay day or something like that so we'll we'll we'll be happy to share with a those details at this stage with your day at the selection of the does the Pico P D modeling.

Uses all of the information you have for example on <unk>.

Unknown Executive: Thanks for your questions. We're going to reserve additional details about the studies for later this year, maybe at a medical meeting or something like that. So we'll be happy to share all of those details at that stage. Now, with regard to the selection of the dose, the PKPD modeling uses all of the information you have, for example, on in-vitro, complementary. assays and, you know, things like clinical outcomes and biomarkers like LDH.

Is the true calling from other essays and.

Things like clinical outcomes, but I'm on it cause <unk> <unk>. This is where it'll be I check please quite useful.

Is it really is a fairly rapidly responsive button on Monica that for.

It was for dosing.

For that you know disclosures through last year about.

The the higher doses, obviously superior to load boasted so I think that it's that type of information that goes into the motor those were the growth of course, the the drug levels.

And let me let me just add on the size I mean, these are rare disease trials right and so.

Unknown Executive: This is where LDH actually is quite useful as a fairly rapidly responsive biomarker that follows the dosing. And we saw that in our disclosures through last year about, you know, how the higher doses are obviously superior to the lower doses. So I think that it's that type of information that goes into the model as well as, of course, the drug level. And let me just add on the size.

Typically in rare disease trials, you need a couple of hundred patients on the drug for a year for safety, if it's a chronic therapy and the.

The expectation is it'll be something like that I think we ended up bill with over 300, and the Haa program when we filed so.

Similarly, but yeah yeah.

Okay. Thank you and then one follow up questions for for for the slightly higher.

Unknown Executive: I mean, these are rare disease trials, right? Typically, in rare disease trials, you need a couple hundred patients on the drug for a year for safety if it's a chronic therapy. And, you know, the expectation is it'll be something like that. I think we ended up, Bill, with over 300 in the HAA program when we filed.

<unk> that we see for the N D H that was previously.

Cause it <unk>, maybe <unk>, a shark or duration of pigment and make small sample size.

So do you expect 500, maybe Ikea goes being a little bit of time to get to who billowed at 1.5, I'll put liberal from <unk>.

For.

Unknown Executive: So, you know, roughly similar. Yeah. Okay, thank you. And then one follow-up question.

The that particular target is not a named pointing on this study.

Unknown Executive: So for the slightly higher levels that we see for LDH that was previously presented, maybe it was due to a shorter duration of treatment and a small sample size. So do you expect 500 MECD dosing over a period of time to get you below that 1.5 upper limit of normal? So, that particular target is not an endpoint in either study. So, you know, I think that's the most important response to the question, in fact.

So you know the that day.

That's the most important.

Response to the question for it so.

They're actually he's nothing magical about one point for October the appointment available available <unk>.

And so you know it'll be I should put me useful by Monica.

And <unk> and it's included as a secondary and point, it's more relevant in the city when you're starting out with Ohio D. H and you know it on and see if I can do for the therapy Uhm.

But it's not as important as <unk>.

Increasing the hemoglobin and reducing the transfusions on improving the fatigue.

Unknown Executive: So there actually is nothing magical about 1.5 times the upper limit of normal for the LD8, and you know, LDH is certainly a useful biomarker, and we're measuring it, and it's included as a secondary endpoint. It's more relevant in the setting when you're starting out with a high LDH and you're not on C5 in the epitherepherapy. But it's not as important as increasing the hemoglobin and reducing the transfusions and improving the fatigue.

For you know specifically and asked you. The question I think uhm, what we've seen in all of the other studies of all of the other component inhibitors.

In sponsors have published individual patient day, though over a long period of time, you see that there'll be ice for choice.

And that'll be typical in this disease, where you expect to see the same thing for you.

Pick a full on and off the whole day, which is kind of fluctuate for the time, but.

Unknown Executive: So, you know, specifically in answer to your question, I think what we've seen in all of the other studies of all of the complement inhibitors when sponsors have published individual patient data over a long period of time, you see that the LBH fluctuates, and that'll be typical in this disease; we expect to see the same thing. So, you know, observe people for long enough, and the LDH is going to fluctuate over time.

If people on on them.

Nick and not being transfused on feeling well then we've achieved all of the principal objectives of treatment of Taylor.

Yeah, and what's most exciting as we now have agreement with the FDA that hemoglobin is the primary endpoint changed from baseline, which is really exciting.

<unk>. Thank you for taking on questions.

Okay welcome.

Unknown Executive: But if people are not e-nic and not being transfused and feeling well, then we've achieved all of the principal objectives of treatment for TNH. Yeah, and what's most exciting is we now have, you know, agreement with the FDA that hemoglobin is the primary endpoint, you know, change from baseline, which is really exciting.

Thank you. Your final question comes from the line is Jonathan <unk>.

Oh for G. M P security sure line you Susan.

Hey, good morning, and sharing my congrats on strong lunch just just a few for me I'm wondering if the current point 9 million figure for Ledoux, if that's net of you're royalty payments and if there's any meaningful contribution from the H U in France.

Unknown Executive: All right. Thank you for taking on this.

Yeah, so the $10.9 million is not Nash.

Jonathan Wollebin: Thank you. Your final question comes from the line of Jonathan Wollebin of JMP securities. Your line is over. Hey, good morning, and I'm sharing my congratulations on the strong launch. Just a few for me.

The royalty payments from royalty payments are just as a reminder, $8 75% of those net revenues of two.

Two 350, and then once we get over 350, it drops down to $2 75.

Unknown Executive: I'm wondering if the $10.9 million figure four, Ladeo, is net of your royalty payment and if there's any meaningful contribution from the ATU and Frank. Yeah, so the $10.9 million is not net of the royalty payments. The royalty payments are, just as a reminder, 8.75% of those net revenues up to 350, and then once we get over 350, it drops down to 2.75. Where that hits is, you know, below the line between operating income and net income, basically as an interest expense, John. In terms of the ATU, no, nothing, really nothing meaningful. That was all U.S. sales at 10.9.

Worry about hits is.

The line between operating income and met income basically has an interest expense Jon.

In terms of the H U no nothing nothing nothing for that was that was all U S sales for $10 nine.

Great and then I'm just one question on the market with you're seeing patients switch from a cute. If you have any sense on the percentage of patients now all HIV patients who are on prophylaxis, and where do you think they're kind of levels out in the future now with you guys on the market and you're saying more switches over to prophylaxis.

Good question, Jon So as I mentioned, what we see we're very confident that at the time of the rodeo on to about 60% of U S. H E patients were on prophylaxis and so his patients also switch from acute therapy only to Orlando that number is going to grow and what's physicians tell us is that in a few.

Unknown Executive: Great. And then I've just got one question on the market. If you're seeing patients switch from acute, if you have any sense of the percentage of patients now of all H.A. patients who are on prophylaxis, and where do you think that kind of levels out in the future now that you guys are on the market and you're seeing more switches over to prophylaxis? Good question, John. As I mentioned, what we see, we're very confident that at the time of the Oraldeo launch, about 60% of U.S. H.A.E. Patients were on prophylaxus.

Sure.

About 80% of patients they expect 80% of patients to be treated with proceeds. So the acute market is shrinking every day and we expect that same kind of trend to occur in Europe. After our launch there and it's turn it up that the market research that Charlie has been doing is playing on the marketplace.

Perfect well, thanks for taking my questions and congrats again.

Unknown Executive: And so as patients also switch from acute therapy only to Oraleo, that number is going to grow. And what physicians tell us is that in the future, about 80% of patients; they expect 80% of patients to be treated with prophecy. So the acute market is shrinking every day.

Thanks, Jon Thanks, Jon.

<unk> there are no further question at this time I will now turn to call back to Jon Stonehouse for closing remarks.

Unknown Executive: And we expect that same kind of trend to occur in Europe after our launch there. And it's turning out that the market research that Charlie has been doing is playing out in the marketplace. Perfect. Well, thanks for taking the questions and congratulations again. Thanks, John. Thanks, John. Thank you. There are no further questions at this time.

So I've done a lot our earnings calls and my 14 years at Biocryst and I got to say this is one of the most fun and exciting ones that I've been a part of we've got a great launch with a great product and a great team and we're just getting started and so it's really exciting.

Unknown Executive: I will now turn the call back to John Stonehouse for his closing remarks. So, I've done a lot of earnings calls in my 14 years at Biochrist, and I've got to say this is one of the most fun and exciting ones that I've been a part of. You know, we've got a great launch with a great product and a great team, and we're just getting started. And so it's really exciting to have a product that's generating real revenue. We have the green light to go into Pivotal Studies with our next program. That's an entire pipeline and a molecule.

To have a product that's generating real revenue.

We have a green light to go into pivotal studies with our next program, that's an entire pipeline and a molecule and so we're going to be starting up pivotal studies later this year.

So it doesn't get a whole lot better than that and our focus now is execution I'm. Just so proud of the team and just incredible focus and execution that they've delivered on and will continue to deliver on so thanks for your interest in our company and have a great day.

Ladies and gentlemen. This concludes today's conference call. Thank you for joining you may now disconnect have a great day.

Jon P. Stonehouse: And so we're going to be starting up pivotal studies later this year. So it doesn't get a whole lot better than that. And, you know, our focus now is execution. I'm just so proud of the team and the incredible focus and execution that they've delivered on and will continue to deliver on.

[music].

Operator: So thanks for your interest in our company and have a great day. Ladies and gentlemen, this concludes today's conference call. Thank you for joining us. You will now disconnect.

Operator: Oh!...I don't know... and I'm gonna, and... I'm gonna be able to be. And... I'm gonna......you know...

Operator: I'm gonna... and the and so on the way, and so. Thank you. Thank you. Thee and and and and You know, Thank you, and and so on the other. Thank you, Thank you.

Alright.

[music].

Operator: Thank you. Thank you. Thank you. Thank you. Thank you, and Thank you. Thank you. Thank you, and the and Thee, You know, The You know, I'm

Operator: Welcome to the Biocryst First Quarter, 2021 Earnings Call. Please note that today's call is being recorded. At this time, all participants are now listening only to the words. After the speaker's presentation, there will be a question-and-answer session. To ask your question during the session, you will need to press star 1 on your telephone. If you require any further assistance, please press bar zero.

[music].

Operator: I would now like to hand the conference over to your speaker for today, John Bluth from Biker. John, the floor is yours. Thanks, Jay. Good morning and welcome to Biocris' First Quarter, 2021 Corporate Update and Financial Results Conference Call. Today's press release is available on our website. Participating with me today are CEO John Stonehouse, CFO Anthony Doyle, Chief Commercial Officer Charlie Geyer, Chief Medical Officer Dr. Bill Sheridan, Chief Business Officer Megan Sosinski, and Chief R&D Officer Dr. Helen Thackray.

[music].

Welcome to the Biocryst first quarter 2021 earnings call.

Note that today's call is being recorded.

At this time all participants are in a listen only mode. After the speaker's presentation. There will be a question and answer session to ask your question. During the session you will need to press star one on your telephone if you require any further assistance. Please press star zero.

I would now like to hand, the conference over to your speaker for today, John Bluth at Biocryst Jon.

Operator: Following our remarks, we will answer your question. Before we begin, please note that today's conference call will contain forward-looking statements, including those statements regarding future results, unaudited, and forward-looking financial information, as well as the company's future performance and or achievements. These statements are subject to known and unknown risks and uncertainties, which may cause our actual results, performance, or achievements to be materially different from any future results or performance expressed or implied in this presentation.

Jon the floor is yours.

Thanks, Jay Good morning, and welcome to Biocryst first quarter 2021, corporate update and financial results Conference call. Today's press release is available on our website participating with me today are CEO, Jon Stonehouse CFO, Anthony Doyle, Chief Commercial Officer, Charlie Gayer, Chief Medical Officer, Dr. Bill Sheridan Chief Business Officer Megan.

As inscape and Chief R&D Officer, Dr. Helen <unk> correct. Following our remarks, we will answer your questions before we begin. Please note that today's conference call will contain forward looking statements, including those statements regarding future results unaudited and forward looking financial information as well as the Companys future performance <unk> achievements. These statements are subject to known and unknown risks and uncertainty.

Fees, which may cause our actual results performance or achievements to be materially different from any future results or performance expressed or implied in this presentation you should not place undue reliance on these forward looking statements for additional information, including a detailed discussion of our risk factors. Please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website I'd now like to turn the.

Operator: You should not place undue reliance on these forward-looking statements. For additional information, including a detailed discussion of our risk factors, please refer to the company's documents filed with the Securities and Exchange Commission, which can be accessed on our website. I'd now like to turn the call over to John Stonehouse. Thanks, John.

Call over to Jon Stonehouse.

Thanks, Jon I Couldnt be more excited to have our first earnings call with revenue from a biocryst launch product.

This is the latest evidence that our company is going through a major transformation.

Sales were very encouraging in Q1, especially considering this is our first quarter of the launch of Orla Dale.

This is a direct result of focused execution of our plan by the Biocryst team and the desire to build a great company that can discover develop and now successfully commercialize oral medicines for patients suffering from rare diseases.

Jon P. Stonehouse: I couldn't be more excited to have our first earnings call with revenue from a Biocrys launch product. This is the latest evidence that our company is going through a major transformation. Sales were very encouraging in Q1, especially considering this is our first quarter of the launch of Orledale. This is a direct result of the focused execution of our plan by the Biocris team and the desire to build a great company that can discover, develop, and now successfully commercialize oral medicines for patients suffering from rare diseases. Very few companies can do all three.

Very few companies can do all three.

The launch is off to a great start because we have a great drug. It works patients are experiencing meaningful reductions in their attacks and they can achieve this by taking one capsule once a day.

This is leading to patient switches from injectable protein on demand therapy.

Exactly what we saw on our market research and clinical trials and it is already playing out on marketplace.

Great drugs don't sell themselves. We also have a fantastic team.

Jon P. Stonehouse: The launch is off to a great start because we have a great drug. It works. Patients are experiencing meaningful reductions in their attacks, and they can achieve this by taking one capsule once a day. This is leading to patient switches from injectable profine on-demand therapy. It's exactly what we saw in our market research and clinical trials, and it's already playing out in the marketplace. Great drugs don't sell themselves. We also have a fantastic team.

Charlie and Alan Hi, Joe U S. GM have assembled an experienced team, but more importantly, they are working together working through challenges and obstacles like COVID-19 and.

And achieving great results.

We couldnt be more pleased with the start and the good news.

We're just getting going with so many more patients to reach in the U S.

While the U S is the largest market we now have approval in other major territories around the world.

Our partner Torii is launching in Japan, and we recently received approval in the EU.

Jon P. Stonehouse: Charlie and Alan Hodge, our USGM, have assembled an experienced team, but more importantly, they're working together, working through challenges and obstacles like COVID, and achieving great results. We couldn't be more pleased with the start and the good news. We're just getting going with so many more patients to reach in the U.S. While the U.S. is the largest market, we now have approval in other major territories around the world. Our partner, Tory, is launching in Japan, and we recently received approval in the EU. The same planning investments were made here, too. The launch ramp will take longer as these are different markets from the U.

The same planning investments were made here too for <unk>.

Launch ramp will take longer as these are different markets from the U S. But we expect these countries to contribute over time to our goal of achieving $500 million plus in global peak sales.

And finally, our pipeline is full with the opportunity we have with our oral factor D inhibitor <unk> hundred 90, 930 for patient suffering from many different complement mediated rare diseases.

We're heading into pivotal studies in <unk> and proof of concept in nephritis indications.

We will apply all of the learnings of our program and build off that success to bring 90 930 day market to create even greater value as a company that discovers develops and commercializes multiple oral drugs for patients suffering from rare diseases.

Jon P. Stonehouse: But we expect these countries to contribute over time to our goal of achieving $500 million plus in global peak sales. And finally, our pipeline is full of the opportunity we have with our oral factor D inhibitor, B6, 9930, for patients suffering from many different complement-mediated rare diseases. We're heading into pivotal studies in P&H and proof of concept in Ephritis indications. We will apply all the learnings of our HAE program and build off that success to bring 9930 to market to create even greater value as a company that discovers, develops, and commercializes multiple oral drugs for patients suffering from rare diseases. Now I turn the call over to Charlie to go over more details about our early launch success. Charlie. Thanks, John.

Now I'll turn the call over to Charlie to go over more details about our early launch success Charlie.

Thanks, Jon.

Early day to launch is off to a great start we're pleased but not surprised we knew we had a great drug for patients want because they are tired of the physical and psychological and logistical burdens of injectable therapies.

Physicians also tell us they are confident in prescribing early day out for a broad range of patients when they see the long term data on attack reduction safety and Tolerability.

Here's what we're seeing so far in.

Charles K. Gayer: The early data launch is off to a great start. We're pleased, but not surprised. We knew we had a great drug that patients want because they are tired of the physical, psychological, and logistical burdens of injectable therapy. Physicians also tell us they are confident in prescribing orlodeo for a broad range of patients when they see the long-term data on attack reduction, safety, and tolerability. Here's what we're seeing so far.

In a very competitive market patients are switching to <unk> over half the patients starting on starting on rodeo for the first time are switching from injectable proceed.

The rest for switching from acute treatment only now that they have an oral once daily profi option.

In fact, the majority of patients on early day by the end of Q1, where those who switched from other products the rest where patients transitioning from our clinical trials and early access program.

Charles K. Gayer: In a very competitive market, patients are switching to Orideo. Over half the patients starting Orodeo for the first time are switching from injectable profiles. The rest are switching from acute treatment only now that they have an oral once daily prophylactic option. In fact, the majority of patients on Orlydeo by the end of Q1 were those who switched from other products. The rest were patients transitioning from our clinical trials in Early Access programs. The prescriber base also continues to expand. Former clinical trial investigators accounted for a minority of Orlyo prescribers in Q1.

The prescriber base also continues to expand.

For our clinical trial investigators accounts accounted for a minority of oil a day of prescribers in Q1.

Roughly 500 physicians treat 50% of HAE patients and our team has reached nearly all of them.

Most of these physicians tell us they intend to prescribe early day, but only a minority have done so thus far so there is a lot of room for growth.

And finally payers are reacting favorably to Orlando.

Most of the reimburse product in Q1 came through medical exceptions, but many payers and Pbms also established coverage policies our momentum with payers is strong and we expect the great majority of patients to have access to coverage for early day by mid year.

Charles K. Gayer: Roughly 500 physicians treat 50% of HAA patients, and our team has reached nearly all of them. Most of these physicians tell us they intend to prescribe Orlaea, but only a minority have done so thus far, so there is a lot of room for growth. And finally, payers are reacting favorably to Orlaea. Most of the reimbursed product in Q1 came through medical exceptions.

The most important investment we made in this launch was building an experienced commercial team that knows how to execute our.

Our U S General manager, Alan Hodge, and our U S. VP of sales Ron Dellinger launched Cinryze over a decade ago and successfully created the first market for <unk> proceeds.

Charles K. Gayer: But many payers and PBMs have also established coverage policies. Our momentum with payers is strong, and we expect a great majority of patients to have access to coverage for Orlando by mid-year. The most important investment we made in this launch was building an experienced commercial team that knows how to execute. Our U.S. general manager, Alan Hodge, and our U.S. VP of sales, Ron Dullinger, launched Sinrise over a decade ago and successfully created the first market for H.A.E. Profit.

They came to biocryst because they always knew that an oral drug is what patients really wanted they.

They attracted a talented and agile team that understands the importance of fighting for every patient and a competitive rare disease market like HPE.

As excited as we are about the Q1 results. This team is just getting started.

COVID-19 is limited in person sales calls, but vaccines are starting to change. This on marketing programs are just starting to kick in and expanding reimbursement access is giving patients and prescribers, even more comfort in switching to oil at al.

Charles K. Gayer: They came to Biocris because they always knew that an oral drug is what patients really want. They attracted a talented and agile team that understands the importance of fighting for every patient in a competitive rare disease market like HAE. As excited as we are about the Q1 results, this team is just getting started.

We are very confident about the growth trajectory and we believe oral aveo will reach peak global sales north of $500 million.

The U S. As the first and largest part of that our opportunity, but we expect meaningful sales in Europe.

Charles K. Gayer: COVID is limited to in-person sales calls, but vaccines are starting to change this. Our marketing programs are just starting to kick in. And expanding reimbursement access is giving patients and prescribers even more comfort in switching to Oraladeo. We are very confident about the growth trajectory, and we believe Oral will reach peak global sales north of $500 million. The U.S. is the first and largest part of that opportunity, but we expect meaningful sales in Europe. Awareness and understanding of HAE in Europe are on par with the US, but use of targeted H.A.E. Prophylaxis has lagged based on a lack of options.

Awareness and understanding of <unk> in Europe is on par with U S. But use of targeted HAE prophylaxis is lagged based on lack of options.

Our work with European physicians and patients tells us that the availability of new options and specifically on oral once daily therapy will more than double prophylactic treatment share to 60% or more of patients.

We are taking the same approach in Europe as in the U S investing in experienced commercial teams that know how to launch rare disease products.

Oil a day on will launch in Germany. This quarter and early access programs are active in France, and the United Kingdom, We look forward to sharing more about Europe in the coming quarters.

Charles K. Gayer: Our work with European physicians and patients tells us that the availability of new options, and specifically an oral once daily therapy, will more than double the prophylactic treatment share to 60% or more of patients. We are taking the same approach in Europe as in the U.S., investing in experienced commercial teams that know how to launch rare disease products. Orlando will launch in Germany this quarter, and early access programs are active in France and the United Kingdom.

And there is yet another orla day launch underway right now in Japan, I will turn the call over to Meghan to describe how our partners at Torrey pharmaceutical are approaching this opportunity.

Thanks, Charlie we're excited the Japanese launch is now underway after successfully completing the NHI price negotiations last month.

A few important points I want to emphasize again regarding the commercial opportunity in Japan, and what makes it different from the U S and Europe.

Charles K. Gayer: We look forward to sharing more about Europe in the coming quarters. And there is yet another Oralale launch underway right now in Japan. I'll turn the call over to Megan to describe how our partners at Tori Pharmaceutical are approaching this opportunity. Thanks, Charlie. We're excited the Japanese launch is now underway after successfully completing the NIH price negotiations last month. But there are a few important points I want to emphasize again.

First early day it was the only approved prophylactic therapy in Japan with <unk>.

No competition Towry has a head start in building the protein market and our oral once daily medicine is well suited for a population that tends to prefer oral drugs over injection.

Secondly, while the vast majority of <unk> patients in the U S and EU are already diagnosed Japan lag behind.

Megan Zizinski: Again, regarding the commercial opportunities in Japan and what makes it different from the US

Megan Zizinski: First, Orlaideo is the only approved prophylactic therapy in Japan. With no competition, Tori has a head start in building the profi market, and our oral once daily medicine is well suited for a population that tends to prefer oral drugs over injections. Secondly, while the vast majority of H.A.

Only about 20% of patients are identified in a registry today.

Sure. He is focused on finding patients and helping to advance their standard of care by providing access to the first approved prophylactic medicine.

One reason, we chose torii was based on what they accomplished the Gilead HIV franchise there.

Megan Zizinski: While patients in the U.S. and EU are already diagnosed, Japan lags behind. Only about 20% of patients are identified in a registry today. Tori is focused on finding patients and helping to advance their standard of care by providing access to the first approved prophylactic medicine. One reason we chose Tori was based on what they accomplished with Gilead's HIV franchise. Their strategies focused on driving disease awareness among physicians,

<unk> strategy focused on driving these awareness amongst physicians and increasing patient identification.

This strategy was very successful as they grew the business to almost $200 million annually.

This experienced served as a strong foundation heading into oil a day of launch and similar to US Tory has been preparing well in advance.

Our fully focused on supporting a successful launch.

Lastly, through our partnership economics, we have triggered the $15 million milestone payment following pricing and.

Megan Zizinski: and increase patient identification

Megan Zizinski: This strategy was very successful as they grew the business to almost $200 million annually. This experience served as a strong foundation heading into Orla deus Mon. And similar to us, Tori has been preparing well in advance and is fully focused on supporting a successful launch.

And we get to share inquiry success with a tiered royalty from 8% to 40% of net sales.

Overall, the Japanese market provides an outstanding long term opportunity.

We see a strong potential to follow a path similar to what we've seen in the U S. In the last decade and expansion of the <unk> market driven by patient diagnosis and adoption of Profi treatment is part of standard of care.

Megan Zizinski: Lastly, through our partnership economics, we have triggered

Megan Zizinski: triggered the $15 million milestone payment following prices, and we get to share in Tories's success with a tiered royalty from 20% to 40% of nest sales. Overall, the Japanese market provides an outstanding long-term opportunity.

As Charlie share Theres, a lot of early momentum in the U S launch and we're not surprised by this excellent start.

Over the course of this year, you will continue to see new clinical data highlighting how well patients do on Orlando over time.

Megan Zizinski: We see a strong potential to follow a path similar to what we've seen in the U.S. in the last decade, an expansion of the H.A.E market driven by patient diagnosis and a

Patients are getting their disease under control, while reducing the impact treatment has on their lives and independent.

Megan Zizinski: and adoption of Prophi treatment as part of standard of care. As Charlie shared, there's a lot of early momentum in the U.S. launch, and we're not surprised by this excellent start.

Our work is also helping physicians understand how orla day or as an ideal treatment choice for all patients.

It offers patients the attack control, they desire and the lifestyle freedom and benefits of our convenient more discrete oral once daily pill.

Megan Zizinski: Over the course of this year, you will continue to see new clinical data highlighting how well patients do on Orlydeo over time. Patients are getting their disease under control while reducing the impact of treatment.

Our strategy remains focused on shifting this treatment paradigm as Charlie spoke about earlier patients are switching which is early evidence our strategy is working.

Megan Zizinski: has on their lives and independence. Our work is also helping physicians understand how Orla Day is an ideal treatment choice for all patients. It offers patients the attack control they desire and the lifestyle freedom and benefits of a convenient treatment.

We see a very similar unmet need and opportunity in the complement space with our factor D program.

I'll now turn it over to Bill for more on our 90 930 progress sales.

Megan Zizinski: of a convenient, more discreet oral once-daily pill.

Yes.

Thanks Megan.

So everyone, who discovered and developed for all of data and the clinical trial collaborators around the world.

Megan Zizinski: Our strategy remains focused on shifting this treatment paradigm. As Charlie spoke about earlier, patients are switching, which is early evidence our strategy is working. We see a very similar unmet need and opportunity in the complement space with our Factor D program. I'll now turn it over to Bill for more on our 9930 progress. Bill? Thanks, Megan.

So Greg for safety all of their launch doing so well.

For hereditary angioedema over day, it was effective and because it is <unk> dramatic.

Dramatically reduces the burden of therapy.

Very similar shift is beginning for patients with paroxysmal nocturnal hemoglobinuria.

Very serious rare disease with no approved oral treatments and to standard of care is lost loan intravenous infusions.

William P. Sheridan: For everyone who discovered and developed Olodeo and our clinical trial collaborators around the world, it's just so great to see the Oladio launch going so well. For hereditary angioidema, Oridio is effective, it is safe, and because it is oral, it dramatically reduces the burden of therapy. A very similar shift is beginning for patients with paracizinal nocturnal hemoglobinuria, a very serious rare disease with no approved oral treatments, and the standard of care is lifelong intravenous infusion.

<unk> 99, <unk> has a great opportunity to substantially improve disease control and P&I itch compared to currently available intravenously infused <unk> inhibitors.

These do a good job controlling intravascular hemolysis, but many patients remained anemic.

Our steel transfusion dependent and continue to suffer from symptoms like fatigue.

That's because C five inhibitors cannot control extravascular hemolysis.

William P. Sheridan: BCX 9930 has a great opportunity to substantially improve disease control in PNH compared to the currently available intravenously infused C5 inhibitors. These do a good job controlling intravascular homolysis, but many patients remain anemic, are still transfuse independent, and continue to suffer from symptoms like fatigue. That's because C5 inhibitors cannot control extravascular homolose. These clinical advantages come on top of the obvious patient benefits of oral administration, and we are moving the program very quickly to get this medicine to patients with P&A.

The clinical advantages come on top of the obvious patient benefits of oral administration and we are moving the program very quickly to get this medicine to patients with <unk>.

I am pleased to report there is net reached agreement with the FDA on both the design and the endpoints for FG and H pivotal trials.

The two pivotal trials each test Aro <unk> 90, 930, mono therapy and will support the indication of treatment of P&I itch.

Dose will be 500 milligrams B I D.

One trial will include patients who had an inadequate response to <unk> inhibitors and the other trial will include patients not currently receiving complement inhibitors, including those naive to these drugs.

William P. Sheridan: I'm pleased to report that we've now reached agreement with the FTA on both the design and the endpoints for our P&H pivotal trials. The two pivotal trials will each test oral BCX9930 monotherapy and will support the indication for treatment of PNH. The dose will be 500 milligrams BID. One trial will include patients who have had an inadequate response to C5 inhibitors, and the other trial will include patients not currently receiving complement inhibitors, including those naive to these drugs. Patients in both categories need release of anemia, freedom for transfusions, and release of a symptom.

Patients in both categories made releases of anemia.

Freedom from transfusions and relief of symptoms.

The primary endpoints for both pivotal trials agreed with the FDA will be changed from baseline in hemoglobin direct clinical measure of release of anemia.

In both trials, we will measure the impact on needs of transfusions as a secondary endpoint from.

We will also catch on other important outcomes in <unk> H, such as fatigue schools, Gnrh clone size and laboratory biomarker because of the losses.

The outstanding results for <unk>, we reported in March with main hemoglobin change from baseline of three three grams per deciliter in <unk> inadequate.

William P. Sheridan: The primary endpoints of both pivotal trials agreed with the FDA will be changed from baseline in hemoglobin, a direct clinical measure of relief of anemia. In both trials, we will measure the impact on the need for transfusions as a secondary endpoint, and we will also capture other important outcomes in P&H, such as fatigue scores, PNH clone size, and laboratory biomarkers from OLLOS. The outstanding results we reported in March, with mean hemoglobin change from baseline of 3.3 grams per deciliter in C5's inadequate response patients and 3.5 grams per deciliter in treatment naive patients, position BCX 9930 very well for success in our pivotal trial.

Response patients and three five grams per deciliter in treatment naive patients.

<unk> <unk> 90 on city very well for success in our pivotal trials.

As you heard in March Hematologists and patients with <unk> are very excited by the prospect of a treatment that could dramatically improve outcomes and to eliminate the need for on IV infusions.

We are convinced that success in our pivotal studies will drive a paradigm shifting P&I edge towards oral proximal complement inhibition with <unk> 90 930.

So whats next in this program, we are now ready to move directly into pivotal trials in <unk> on a second half of the year using the designs now agreed with the FDA.

William P. Sheridan: As you heard in March, hematologists and patients with TNH are very excited by the prospect of a treatment that could dramatically improve outcomes and eliminate the need for IV infusions. We are convinced that success in our pivotal studies will drive a paradigm shift in P&H towards oral proximal complement inhibition with BCX 9930.

We will also move into a proof of concept trial and selected nephritis indications in the second half for this year.

This kicks 90 930 represents a pipeline in a molecule. We are very excited to be moving this program. So quickly because we know patients are waiting now I'd like to hand, the call over to Anthony.

William P. Sheridan: So, what's next in this program? We are now ready to move directly into the pivotal trials in PNH in the second half of this year, using the designs now agreed with the FDA. We will also move into a proof-of-concept trial in selected nephritis indications in the second half of this year. B69930 represents a pipeline in a molecule.

Thanks, Phil.

We've continually said that we are focusing our investments where they can drive the greatest value.

With early day on now approved in three key global territories. The commercial team in the U S getting the launch off to a great start on the very positive data that we have a <unk> 90 930, we are executing the strategy.

William P. Sheridan: We're very excited to be moving this program so quickly because we know patients are waiting. Now, I'd like to hand the call over to Anthony. Thanks, Bill. We've continually said that we are focusing our investments where they can drive the greatest value. With Orladeo now approved in three key global territories, the commercial team in the US getting the launch off to a great start, and the very positive data that we have with BTX 9930, we are executing this strategy, and we are well positioned for future growth. You can find our detailed financials in today's earnings press release, and I'd like to call your attention to a few items. The net revenue for the quarter was $19.1 million.

We are well positioned for future growth.

Anthony J. Doyle: Of this, $10.9 million came from sales of Orla Dayo in the U.S. Operating expenses, not including non-cash compensation, for the quarter were 63 million, with the incremental investment from previous quarters focused on the development of BCX 9930. We ended Q1 with $244 million in cash. This cash, in addition to access to the additional $75 million from Ethereum and now revenue from Orledo, continues to give us cash runway into 2023. Since this is our first full quarter of Orlidaire revenue, I want to take a minute to remind you of our approach and a couple of key items. We recognize revenue when our sole source specialty pharmacy ships Orlando to patients for use. Each shipment contains a 28-day supply of Orlando.

You can find our detailed financials in today's earnings press release, and I'd like to call your attention to a few items.

Net revenue for the quarter was $19 $1 million of this $10 9 million came from sales of oil a day on the U S. Our operating expenses not including noncash compensation for.

For the quarter was $63 million with the incremental investment from previous quarters focused on the development of <unk>. We ended Q1 with $244 million on cash. This cash in addition to access the additional $75 million from a theory on now revenue from Orlando continues to give us cash runway into 2020.

Three.

Since this is our first full quarter of all of their revenue I wanted to take a minute to remind you of our approach on a couple of key items.

We recognize revenue on a sole source specialty pharmacy ships on a day or for patients for us. Each shipment contains a 28 day supply of all the data.

These are shipments directly from the specialty pharmacy pharmacy to patients. So they are true sales and you will not see any inventory or channel stocking in our revenue numbers.

When we look at growth to Nash the biggest impact at the moment will be driven by non reimburse shipments. Our quick start program has proven to be a real differentiator and is delighting customers and the ease and speed of getting access to or the day, sometimes within 24 hours of a start for them being submitted.

Anthony J. Doyle: These are shipments directly from the specialty pharmacy to patients, so they are true sales, and you will not see any inventory or channel stocking in our revenue numbers. When we look at Gross to Net, the biggest impact at the moment will be driven by non-reimbursed shipments. Our Quick Start program has proven to be a real differentiator and is delighting customers with the ease and speed of getting access to Oridea, sometimes within 24 hours of a start form being submitted.

The quick start program and our patient assistance program. Both resulted in a growth to net adjustment being higher now than it will be once the launches in a more mature phase.

Because gross to net is so fluid early on the launch we are not providing gross to net guidance, but as we continue to progress with the launch you should expect our gross to net adjustment to move in line with other rare disease products.

So what does our strong Q1, meaning for future periods.

Anthony J. Doyle: The Quick Start program and our patient assistance program both resulted in the gross to net adjustment being higher now than it will be once the launch is in a more mature phase. Because gross to net is so fluid early in the launch, we are not providing gross to net guidance, but as we continue to progress with the launch, you should expect our gross to net adjustment to move in line with other rare disease products.

Charlie's team did a great job of converting clinical trial patients, giving us a bolus of patients in the first quarter. Additionally, the team has achieved great success in helping many new patients switch from injectable prophylactic or acute only medications together. This gives us a really strong foundation for future periods.

We have not provided revenue guidance as there are still several variables that will impact the growth trajectory of revenue that we need more time to better understand first what is the steady state of monthly prescriptions, while we've been very encouraged with the numbers to date, we need more time to see it play out.

Anthony J. Doyle: So, what does our strong Q1 mean for future periods? Charlie's team did a great job of converting clinical trial patients, giving us a bolus of patients in the first quarter. Additionally, the team has achieved great success in helping many new patients switch from injectable prophylactic or acute-only medications. Together, this gives us a really strong foundation for future periods. We have not provided revenue guidance as there are still several variables that will impact the growth trajectory of revenue that we need more time to better understand.

Next quarter's customer retention looks like over a longer time.

In our clinical trials. This number was about 75% through 48 weeks, telling us that early day. It was well tolerated and is providing outstanding attack control.

We believe we will see this level of persistence in the market. It's still too early to confirm that this is the case.

Anthony J. Doyle: First, what is the steady state of monthly prescriptions? While we've been very encouraged with the numbers to date, we need more time to see it play out. Next, what does customer retention look like over a longer time?

And lastly, what does the trend look like when the vast majority of patients have reimbursement the $10 $9 million.

And net revenue we are reporting is only from patients whose early their prescriptions are reimbursed as Charlie described we're making good progress getting early day on want to policies, but the pace of reimbursement over especially the second quarter will be a key driver in the rate of revenue growth for.

Anthony J. Doyle: In our clinical trials, this number was about 75% through 48 weeks, telling us that Orlidaio is well tolerated and is providing outstanding attack control. While we believe we will see this level of persistence in the market, it's still too early to confirm that this is the case. And lastly, what does the trend look like when the vast majority of patients have reimbursement? The $10.9 million in net revenue we're reporting is only from patients whose orlidaeo prescriptions are reimbursed.

For us over the remainder of the year.

While we're early in the launch we are very encouraged by the results to date, we have a lot of work to do on a lot more patients to get this next generation of drug too.

We have a great product in Orlando, there's strong patient demand for it and we have the team to execute on making our launch a success and getting us to our peak target of $500 million plus in the coming years with that I'll hand, it back over to Jon.

Anthony J. Doyle: As Charlie described, we're making good progress getting Orlidao onto policies, but the pace of reimbursement, especially in the second quarter, will be a key driver in the rate of revenue growth for us over the remainder of the year. While we're early in the launch, we are very encouraged by the results to date. We have a lot of work to do, and a lot more patients need to get this next generation of drugs, too.

Thanks Anthony.

This is what execution looks like.

We're off to a great start with the launch of oil a day on the U S and starting to launch in other major parts of the world all contributing to what we believe will be a $500 million plus global peak sales product.

Anthony J. Doyle: We have a great product in Orledale. There is strong patient demand for it, and we have the team to execute on making our launch a success and getting us to our peak target of $500 million plus in the coming years. With that, I'll end up back over to John. Thanks, Anthony. This is what execution looks like.

Add to that our plan to advance into pivotal studies in <unk> with 90 930 and in parallel moving into other indications with this pipeline in one molecule.

And finally, having the financial flexibility that comes with a strong balance sheet and revenue generation allows us to focus on execution and value creation.

Jon P. Stonehouse: We're off to a great start with the launch of Orladeo in the U.S. and starting to launch it in other major parts of the world, all contributing to what we believe will be a $500 million plus global peak sales product. Add to that our plan to advance into pivotal studies in P&H with 9930 and, in parallel, move into other indications with this pipeline in one molecule. And finally, having the financial flexibility that comes with a strong balance sheet and revenue generation allows us to focus on execution and value creation.

Evidence is clear that biocryst is transforming into a company with product revenue.

Full pipeline in a discovery engine that produce these compounds and we will continue to produce more.

That concludes our prepared remarks, we will now open it up for your questions.

Thank you and as a reminder, if you would like to ask a question. Please press Star then the number one on your telephone keypad once again Thats star one on your telephone keypad. If you would like to withdraw your question press the pound key.

Jon P. Stonehouse: The evidence is clear that Biocrys is transforming into a company with product revenue, a full pipeline, and a discovery engine that produces these compounds and will continue to produce more. That concludes our prepared remarks. We'll now open it up for your questions. Thank you. And as a reminder, if you would like to ask a question, please press star, then number one on your telephone keypad. Once again, that's star one on your telephone keypad. If you would like to withdraw a question, please press the found key. Thank you. Our first question comes from the line of Jessica Fye of JPM's. Your line is open.

Thank you.

Our first question comes from the line of Jessica Fye of Jpmorgan. Your line is open.

Hey, guys. Good morning, congratulations on a strong quarter.

I was curious if you could add some more color about specifically which agencies.

Patient switching on to oral with switching from.

Or do you want to take that yes, hey, good morning, Jeff. Thanks for the question, we're seeing patients switched from all the different therapies really in proportionate to what you would expect from.

Their market share so as I mentioned more than more than half the patients for switching from injectable proceeds and that means tax iroh haegarda cinryze proportion to their market share.

Jessica Macomber Fye: Hey guys, good morning, congratulations on a strong quarter. I was curious if you could add some more color about specifically which agents you see the patients switching on to Orlydeo switching from.

Okay. Thanks.

You mentioned that from scripts were not reimbursed in the quarter can you say what proportion of first quarter expense were not reimbursed.

Unknown Executive: Yeah, good morning, Jess, thanks for the question. We're seeing patients switch from all the different therapies really in proportion to what you'd expect from their market share. So, as I mentioned, more than half the patients are switching from injectable prostheses, and that means tax Iro, Hagarda, Sinrise, in proportion to their market share.

Yes, we haven't we haven't commented on the specific details, but as Anthony mentioned in his comments, what we're what we're doing with nearly all patients is starting off on our quick start program and then we work with patients and providers to go through prior authorization and in Q1, most patients get access through medical exception.

Unknown Executive: Okay, thanks. And you mentioned that some scripts were not reimbursed in the quarter.

<unk>.

Unknown Executive: In the quarter, can you say what proportion of first quarter scripts were not reimbursed?

But we're making great progress with payers and we expect to make continued progress through mid year and just Charlie made comments in his prepared remarks around the more policies that are in place where all the day is on formulary, but the more interest there is going to be by both doctors and physicians.

Unknown Executive: Yeah, we haven't commented on the specific details, but as Anthony mentioned in his comments, what we're doing with nearly all patients is starting them off in our Quick Start program, and then we work with patients and providers to go through prior authorization. And in Q1, most patients got access through medical exception. But we're making great progress with payers, and we expect to make continued progress through mid-year. And Jess and Charlie made comments and their prepared remarks around, you know, the more policies that are in place where or the deoson formulary, the more interest there's going to be by both doctors and physicians.

Got it and just a last one on gross to net you mentioned that longer term that will move in line with other rare disease drugs. So can you just characterize what typical gross to net are in rare disease.

Yes from a from a growth.

Prospective what we'd be looking at getting us kind of into the teens 20 tie type of frame.

Great. Thank you.

Welcome.

Unknown Executive: Got it. And just the last one on gross to nets. You mentioned that, longer term, that'll move in line with other rare disease drugs. So can you just characterize what typical growth to nets are in rare diseases? Yeah, from a gross enough

Thank you.

Question comes from the line of Fleece vehicle of Evercore ISI. Your line is open.

And then wanted to add my congratulations on a great quarter.

Unknown Executive: Yeah, from a gross enough perspective, what we'd be looking at getting is kind of into the teens 20 type of frame. Great, thank you. Thank you. Next question comes from the line of Lisa Beko of Evercore ISI. Your line is open.

I'm going to turn to the <unk> study for a moment.

The book.

Yes.

What sort of control.

On will look like in both of the study.

Paul.

Chile limping net.

Sure.

Hi, Lisa Thanks for the question.

Super excited about moving in today's studies of course, and one study will be a superiority trial against the <unk> inhibitors.

Liisa Ann Bayko: And I wanted to add my congratulations on a great quarter. I'm just a quick, I'm going to turn to the PNH studies for a moment. Can you maybe discuss what sort of control arms will look like in both of them?

In patients who have made adequate response and the other study in patients who are not taking a C. Five inhibitor.

For the control group will be placebo.

William P. Sheridan: Sure. Hi Lisa.

Okay I see.

And can you maybe describe the.

On the.

I guess, how youll be youll.

You'll be looking at the period five will you be looking at combination therapy then for.

William P. Sheridan: Thanks for the question. I'm super excited about moving into these studies, of course, and one study will be a superiority trial against C5 inhibitors in patients who have an inadequate response, and the other study in patients who are not taking C5 inhibitors, the control group will be placebo.

For the patients that are.

Inadequate responders.

Moving on to monotherapy, how will that work for the inadequate response group.

Sure.

Studies are designed for <unk> 90, 930 mono therapy.

William P. Sheridan: Okay, I see. And can you maybe describe the, um, the, uh, I guess how you'll be looking at Superior to C5? Will you be looking at combination therapy then for the patients that are inadequate responders, or will people be moving?

Patients will be randomized to <unk> in the C. Five inhibitor trial, the randomized to continue their current treatment for stuff.

Joining me on city mono therapy, and discontinue their current treatment.

So this is the goal here is a label for monotherapy.

William P. Sheridan: Moving on to Monotherapy, how will that work for the inadequate response group?

The broad indication to treat.

William P. Sheridan: Sure, both studies are designed as tests for BCX 9930 monotherapy. So, you know, patients will be randomized to, in the C5 inhibitor trial, be randomized to continue their current treatment or start BCX 9930 monotherapy and discontinue their current treatment. So this is, you know, the goal here is a label for monotherapy with a broad indication to treat patients with PNH. But the reason for the study designs, as we just outlined, is to support that type of labeling.

Patients with <unk>, but the reason for those study designs.

The outlined.

That's on globally.

Okay.

How do patients go on to monotherapy from from being on the C. Five how does that work is it just.

Kind of very simple for their.

Their combination therapy for all.

All of our work.

No.

The C five inhibitor has stopped.

For the oral Georgia southern stood.

Just a simple switch.

Okay.

We haven't seen the data yet in that population in terms of being on monotherapy can you give us a sense of when we might see that in your phase one phase two.

William P. Sheridan: Okay, and how do patients go on to monotherapy from being on a C5? How does that work? Is it just a kind of cold turkey switch, or is there combination therapy and then a withdrawal, or does it work?

Sure.

The protocol doesn't specify for particular timeline.

For the physician judgment call that we expect that we'll be able to share data at the end of the year right now the regulators nor are still waiting for that data in order to start the study.

William P. Sheridan: No, the C5 is not there.

William P. Sheridan: The C5 inhibitor is stopped, and the oral drug is started; 9930 starts. It's just a simple switch.

We are very confident in the drug on the basis of the monotherapy results from the naive patient population the diseases the disease.

William P. Sheridan: Okay, we haven't seen the data yet on that population in terms of being on monotaph.

William P. Sheridan: on monotherapy. Can you give us a sense of what is white, see that in your phase one, phase two? Sure.

The effective day targets per se.

William P. Sheridan: Sure, you know, the protocol doesn't specify a particular timeline, and it's a physician judgment call, but we expect that we'd be able to share data at the end of the year. Neither the regulators nor us are waiting for that data in order to start the studies because we're very confident in the drug on the basis of the monotherapy results in the naive patient population. You know, the disease is the disease, the factor D targets the same, the dose is the same, So it's not necessary for us to see that data before we start the study.

The doses for soon.

Sure.

It's not necessary for us to say that day that before we start with COVID-19.

Okay.

Good and then I guess, just turning to Orla Dale.

Really great out of the gate.

Is there any kind of additional color you can provide on either.

The percentage of patients that were on your quick start program.

Receiving drug that way.

The percentage of prescriptions.

And are any color on persistence those are obviously the key.

Questions.

Help guide us as we think about the rest of the year launching off of this really great start.

Unknown Executive: Okay, good. And then I guess just turning to Orlodeo, really great out of the gates. Is there any kind of additional call you can provide on either?

Just hoping you can provide some additional color there and that's my final question. Thank you.

Unknown Executive: on either, you know, the percentage of patients that were on your Click Start program, receiving the drug that way, you know, the percentage of prescriptions, like, and or any color on persistence, those are obviously the key questions to help guide us as we think about, you know, the rest of the year launching off of this, you know, really great start. So just hoping you can provide some additional color there. And that's my final question. Thank you.

Sure Lisa so so on the percentage of quick start so.

In Q1, nearly all patients started on quick start and Thats part of our strategy.

And as we work with them to get access and as I mentioned, most patient Scott expert got reimbursement through medical exception. If they were not able we were not able to do that in Q1 they'll continue on free good until the payers establish policies.

And then as far as your question about persistence.

Unknown Executive: Sure, so on the percentage of Quick Start, so in Q1, nearly all patients started on Quick Start, and that's part of our strategy, as we work with them to get access. And as I mentioned, most patients got reimbursement through medical exception. If we are not able to do that in Q1, they'll continue on free goods until the payers establish policies. And then, as far as your question about persistence, as Anthony mentioned, we expect we had great results in our clinical trials with about 75% of patients staying on for at least a year. That's what we expected in the real world. So far, the patient experience has been great, but it's too early to say what the long-term persistence rate will be.

Anthony had mentioned, we expect we had great results on our and our clinical trials was about 75% of patients staying on for at least a year. That's what we expected in the real world. So far the patient experience has been great, but it's too early to say what the long term persistence rate will be.

Okay and that patient quick start program is about is it on a monthly prescription until didnt get on.

That's right.

It's a month and if there is not reimbursement in a month, where we will continue from either on quick start or on our patient assistance program.

Okay, I guess that just leaves me on one final question approximately how long is it taking.

To get on to come on.

Now remember a strike at this point.

Unknown Executive: Okay, and that patient Quickstar program, is it a monthly prescription until you get on it?

So in Q1, it's all over the board everything from a few days to patients still being on the program and by midyear, we expect that to really stabilize as the great majority of patients will have access to coverage through their plans.

Unknown Executive: Reimbursed drugs? That's right. It's a month, and if there's not reimbursement in a month, we will continue them either on Quick Start or through our patient assistance program.

The theme here is that as the year progresses, we will have more and more people on paid drug and fewer and fewer on quick start.

Unknown Executive: Okay, I guess that just leaves me with one final question. Approximately how long is it?

Unknown Executive: to get on to reimburse records. So in Q1, it's all over the board.

That's the plan.

Okay.

Alright, Thanks, a lot going on.

Unknown Executive: So in Q1, it's all over the board, everything from a few days to, you know, patients still being on the program. And by mid-year, we expect that to really stabilize, as the great majority of patients will have access to coverage through their plans. Yeah, and Lisa, the theme here is that as the year progresses, we will have more and more people on paid drugs and fewer and fewer on Quick Start. That's the plan. Okay. All right.

Thank you.

Thank you next question comes from the line of Ken Cacciatore from Cowen and co. Your line is open hey.

Guys. Congratulations on the early progress just wondering we hear really good things about the interactions on both though.

Clinician and patient level via your hubs. So just wondering as it seems to be ramping a little bit faster do you feel you're staffed appropriately can you talk about some of the early learnings from this really kind of a more personalized approach and things that.

Unknown Executive: Thanks a lot, guys. Thank you. Thank you. The next question comes from the line of Ken Kachatori from Cohen & Co. Your line is open.

As things are getting underway that you may be changing or again, if you're staffing a little bit more than on EU.

Ken Kachatori: Hey, guys, congratulations on the early progress. Just wondering, we hear really good things about the interactions on both the clinician and patient level via your hub. So just wondering, as it seems to be ramping a little bit faster, do you feel you are staffed appropriately? Can you talk about some of the early learning?

Just wondering it looks like a potential really focus selling effort can you talk about where these patients are domiciled is it going to be fairly easy to get at them fairly difficult maybe some of the learnings from the Injectables I know you feel and we feel as well that things should go better there, but maybe a little bit more nuance around.

Unknown Executive: from this really kind of more personalized approach and things that, as things are getting underway, that you're maybe changing, or again, if you're staffing a little bit more, then on EU, just wondering, it looks like a potential really focused selling effort. Do you talk about where these patients are domiciled? Is it going to be fairly easy to get at them, fairly difficult?

Europe, and then lastly, maybe for Bill I know, it's really early just announcing you're going to start. These pivotal if you take a shot at timing of results.

Maybe a little bit of nuance here in terms of the kind of competitive landscape to enroll patients, but any kind of shot at when we might expect to see data would be fantastic. Thank you.

Unknown Executive: Maybe some of the learnings from the injectables. I know you feel, and we feel as well, that things should go better there, but maybe a little bit more nuance around Europe. And then lastly, maybe for Bill, I know it's really early just announcing you're going to start these pivotals, but if you take a shot at the timing of results, maybe a little bit of nuance here in terms of the kind of competitive landscape to enroll patients, but any kind of shot at when we might expect to see data would be fantastic. Thank you.

Great. Thanks for the questions Ken So the first question you had just about our specialty pharmacy patient services program in staffing.

One of the great things about having a sole sources, we really prepared for the right staffing and then Alan Hodge and his team have done a great job working with ESP to pivot as we see this demand from patients so increasing staffing appropriately increasing procedures.

Unknown Executive: Great, thanks for the questions, Ken. So the first question you had was about our specialty pharmacy patient services program and staffing. One of the great things about having a sole source is that we are really prepared for the right staffing, and then Alan Hodge and his team have done a great job working with the SP to pivot as we see this demand from patients. So increasing staffing appropriately, increasing procedures. They're really quick in making those changes to serve the patients.

They're really quick and making those those changes to serve the patients well and it's making a difference.

As far as the EU really good question about access to patients and how easy or difficult is that.

The great thing about the EU is that treatment centers are very concentrated so you can literally have hundreds of patients within a given center and so what our teams are doing is working with the experts at those centers to make them aware that Orla day, it was coming and to reach out to their patients and bring them into the centers. So we're.

Unknown Executive: patients well, and it's making a difference. As far as the EU is concerned, a really good question about access to patients and how easier or difficult that is. The great thing about the EU is that treatment centers are very concentrated, so you can literally have hundreds of patients within a given center.

Really enthusiastic about the opportunity there and Ken I would add that.

Biocryst is a known entity to these docs that treat <unk> in Europe, I mean, we've been doing clinical trials all the way back to our first generation program and so we've got fantastic relationships and they know the company they've been involved in our trials and so the excitement level is high.

Unknown Executive: And so what our teams are doing is working with the HAA experts at those centers to make them aware that Orlodeo is coming and to reach out to their patients and bring them into the centers. So we're really enthusiastic about the opportunity. And Ken, I'd add that, you know, Bioschrist is a known entity to these docs that treat H.A.E. in Europe.

And then maybe Phil the Yeah go ahead, yeah sure.

So again, what a great question when will the study is finished.

Unknown Executive: I mean, we've been doing clinical trials all the way back to our first generation program, and so we've got fantastic relationships. And they know the company. They've been involved in our trials, and so the excitement level is high. And it may be in the, yeah, go ahead. Yeah, sure. So Ken, what a great question.

I wish I could be specific.

For a prediction I think it's really difficult at this stage, we need to get them up and running on what I can tell you is that we have a great clinical execution team here at Biocryst and.

We are going to approach this on the basis for patients really need this drug is going to be a major advance in <unk>. That's what we believe and so it will be.

Unknown Executive: When will the studies finish? I wish I could be specific and give you a prediction. I think it's really difficult at this stage, and we need to get them up and running. What I can tell you is that we have a great clinical execution team here at Birochrist, and we are going to approach this on the basis that patients really need this drug. It's going to be a major advance in P&H, that's what we believe. And so we'll be ramping as fast as possible, and just like in H.A., we'll go to the drug. The best standards all around the world to get these studies done.

Ramping as fast as possible.

Just like we will go to the best centers all around the World typically studies done.

And just like HP, Inc.

Most weighted basis patient.

Getting patients on clinical trials is the competitive active.

Activity.

We believe we have a great offering here with the SEC.

<unk> inhibitor. So we look forward to seeing the.

Study start on the accrual moving.

And then we'll be in a better position to make an estimate of what that might finish.

Unknown Executive: And just like HAE, in most rare diseases, a patient, getting patients on clinical trials is a competitive activity, and we believe we have a great offering here with an oral factor D&JB. So we look forward to seeing the study start and the accrual moving, and then we'll be in a better position to make an estimate of when they might finish. Can I just add, it's hard to say because we just don't know what the rate of enrollment will be in a competitive space in a, you know, much larger study than what we've studied so far in P&H. It's just super hard to predict, but one thing I will say is Bill's team is excellent, and their aim is to be the sponsor of choice.

Great. Thanks, Ken Ken, Yes, I'd just add.

It's hard to say because we just don't know what the rate of enrollment will be right in a competitive space.

Much larger study than what we've studied so far in <unk>.

It's super hard to predict but one thing I will say as Bill's team is excellent and their aim is to be the sponsor of choice and this is where big doesn't necessarily help you small companies that pay attention that listen that give the extra.

<unk> make a difference in enrollment and so I am Super confident that those sales team will do that.

Very helpful.

Thank you next question comes from the line of Serge Belanger from Needham and company. Your line is open.

Unknown Executive: And this is where big doesn't necessarily help you. Small companies that pay attention, that listen, that give the extra TLC make a difference in enrollment. And so I am super confident that those team members will do that.

Okay.

Hey, good morning, and.

Thanks for taking my questions.

Few on or deal.

First and I apologize if you've covered this before.

Did you disclose the number of patients that were on drug.

Serge D. Belanger: Very helpful. Thank you. The next question comes from the line of Serge Ballinger from Needham and Company. Your line is open.

At the end of the first quarter and I guess, how many of them.

For patients transitioning from clinical trials or the.

Early access programs.

Hey, Serge good morning that we have not disclosed for detail on that and what I can say is the number of patients on early day will continues to grow every day as patients switched to oral <unk>.

Unknown Executive: Hey, good morning, and thanks for taking my questions. A few on Orola-Dale. First, apologies if you covered this before. Did you disclose the number of patients that were on the drug at the end of the first quarter? And, I guess, how many of them are patients transitioning from clinical trials or the early access programs?

In Q1, what we did say is that.

The majority of patients by the end of the quarter, where those were for new patient switching and then two early day on and then the rest of them were those of transitioning from our clinical trials and EAP and that transition program finished in Q1, so that bolus that Anthony talked about is is complete and then everything going forward is.

Unknown Executive: Hey, Sirch, good morning. We have not disclosed the details on that. What I can say is the number of patients on Orlydeo continues to grow every day as patients switch to Orleao. In Q1, what we did say was that the majority of patients by the end of the quarter were those new patients switching, and then to Orideo, and then the rest of them were those transitioning from our clinical trials and DAP.

Growth is patient switched oil a day.

Just one point of clarification on that the complete means that the switch from clinical trials to quick start or our patient assistance. They have to go through the same process of reimbursement like any other patient and so some have been reimbursed summit not.

Okay.

Unknown Executive: And that transition program finished in Q1. And so that bolus that Anthony talked about is complete, and then everything going forward is growth, and patients switched to early day. Yeah, just one point of clarification on that. The complete means that they're switched from clinical trials to Quick Start or patient assistants. They have to go through the same process of reimbursement like any other patient. And so some have been reimbursed; some have not.

And I think you talked about.

Yeah.

Based on the 500.

Serving about 50% of.

Hey patients.

How big is the next set of physicians, serving the other 50% that you need to address.

Search theyre easily another thousand plus physicians out there and we're reaching them as well we're really concentrated on that top 500, just because they are they have more patients and they really know so thats our priority, but we're reaching the other doctors as well and we're getting prescribing from from them too.

Unknown Executive: Okay, and I think you talked about position based on about 500, serving about 50%.

Unknown Executive: of the H. How big is the next set of physicians?

And Sir Jai to add what makes this achievement in the first quarter with the sales that Charlie and his team have generate even more remarkable as they did it in COVID-19 right.

Unknown Executive: fed a position serving the other 50% that you need to address.

Unknown Executive: Sirge, there are easily another thousand-plus physicians out there, and we're reaching them as well. We're really concentrated on that top 500 just because they are, you know, they have more patients and they really know H.E. So that's our priority, but we're reaching other doctors as well, and we're getting prescribing from them too. And, Serge, I'd add, you know, what makes this achievement in the first quarter with the sales that Charlie and his team have generated even more remarkable is they did it in COVID, right? And so, you know, we're already seeing things starting to open up with vaccination, and that's only going to get better as the course of the year goes on.

And so we're already seeing things starting to open up with vaccination, that's only going to get better as the course of the year goes on.

And then more around COVID-19.

<unk>.

What has been the overall impact with restrictions.

Are you seeing the limitations on the on the number of patient switches and is that something that you expect will increase as restrictions lift.

I think first first of all surge as Jon was talking about COVID-19 limited our in person interaction with providers fewer than 50% of our calls were in person that's going to make a big difference as we're able to do more in person visits.

Unknown Executive: Okay, and while we're on COVID, you know, what has been the overall impact with restrictions, or are you seeing limitations on the number of patient switches, and is that something that you expect will increase as restrictions lift?

And we.

We had a great quarter. Despite all of this.

Another piece is that oral is easy for doctors to prescribe. So it's something that many physicians have been comfortable prescribing oral a day.

Unknown Executive: I think first of all, surge, as John was talking about, COVID limited our in-person interaction with providers; fewer than 50% of our calls were in-person. That's going to make a big difference as we're able to do more in-person visits. And we had a great quarter despite all of this.

Our remote environment to their patients.

More in person visits so it's just it's more opportunity and we're excited for what's to come.

Great. Thank you congrats on the progress.

Thanks.

Thank you next question comes from the line of smaller Raycroft of Jefferies. Your line is open.

Unknown Executive: Another piece is that oral Adio is easy for doctors to prescribe. So it's something that many physicians have been comfortable prescribing oral Adio in a remote environment to their patients. Patients. With more in-person visits, though, it's just more opportunity, and we're excited for what's to come.

Hi, This is Kevin Chen on for Maury Raycroft I have two questions one on <unk>.

Unknown Executive: Great. Thank you. That's in the progress.

Healthy progress on the formulary adoption versus.

Reimbursement by medical exception have you encountered any pushback on the cost effectiveness from insurances and.

How are the reimbursement conversations going on insurance is looking at IAC reports.

Unknown Executive: Thank you. The next question comes from the line of Moirey Craft of Jeffries. Your line is open. Hi, this is Kenny Chan on behalf of Moray-Croft. I have two questions, one on Orla Deo.

For the second question on the.

The <unk> program.

As the FDA feedback on LDH levels and is there a specified.

Unknown Executive: How's the progress on the formulary adoption versus reimbursement by medical exception? Have you encountered any pushback on the cost effectiveness from insurances? How are the reimbursement conversations going? Are insurance companies looking at ISA reports? And for the second question on the P&H program, what was the FDA feedback on LDH levels and if there is a specified LDH threshold that would trigger a safety concern? Fairly, you want to...

Threshold that would trigger a safety concern.

Barry you want to yes.

Ken as far as the payer progress for policy progress as we said in our comments, it's going really well.

In Q1, it was mainly medical exception that we were getting access, but several payers and pbms put oil a day on on formulary and we accept we expect a lot of acceleration of that over the over Q2.

Cost effectiveness no. This is the lowest price brophy on on the market.

Unknown Executive: Yes, thanks, Kenny. As far as payer progress, policy progress, as we said in our comments, it's going really well. In Q1, it was mainly medical exception that we were getting access, but several payers and PBMs put Orlydeo on formulary, and we expect a lot of acceleration of that over Q2. Cost-effectiveness, no, this is the lowest price profy on the market, and payers have reacted really well to our pricing strategy as well as the profile of the drug. And then, as they see the demand coming from patients switching to Oraleo, it's really encouraging payers to establish coverage policies quickly. So we're very pleased with where we are.

And payers have reacted really well to our pricing strategy as well as the profile of the drug and then as they see the demand coming from patients switching to oil a day Oh, it's really encouraging.

Payers to establish coverage policies quickly. So we're very pleased with where we are and let me stress one point I made in the prepared remarks. This drug works right people are switching that are controlled on appropriate therapy to our drug and they're staying on our drug. So this idea of effectiveness. This drug works.

Just really want to stress that.

Bill do you want to take the FDA feedback on LDH.

Sure.

Thanks for the question.

Do have a secondary endpoint.

Unknown Executive: And let me stress one point I made in my prepared remarks. This drug works, right? People that are controlled on prophy therapy are switching to our drug, and they're staying on our drug. So this idea of effectiveness, this drug works. I just really want to stress that. Bill, do you want to take the FDA feedback on LDH? Sure, Kenny, thanks for the question. We do have a secondary endpoint of percentage change from baseline in LDH. There is no threshold either as an endpoint or as a safety concern for that matter. Thus, LBH is a useful biomarker.

Vintage is changed from baseline in LDH.

There is no threshold, either as an endpoint or safety concerns for that matter.

Sure.

There'll be a useful biomarker.

The.

Improvement in anemia, with the change from baseline in hemoglobin hemoglobin going up and the <unk>.

Improvement in transfusion burdened with the transfusion avoidance <unk> cost for the most important number.

Transfusions going down.

Very important clinical outcomes.

It's also worth stressing, but another very important secondary endpoint.

<unk>.

Valuation of quality of life with tools, such as the assessment to take school, so measuring fatigue is important too.

Unknown Executive: The, you know, incursment in anemia with the change.

Unknown Executive: with the change from baseline in hemoglobin, hemoglobin going up, and the improvement in transfusion burden with transfusion avoidance being, of course, the most important, but the number of transfusions going down. A very important clinical app. You know, we're also stressing that another very important, second, end point is, you know, evaluation of quality of life with tools such as the Fasset Fatigue score. So measuring fatigue is important too. And Bill, you might want to just talk about the interaction with the FDA and that it was focused on clinical benefits, right? Biomarkers are important, but clinical benefits are more important.

And bill.

You might want to just talk about the interaction with the FDA and that it was focused on clinical benefit biomarkers that are important but clinical benefit is more important.

Right so.

Regulators of course want to see clinical outcomes being treated as important endpoints in clinical trials.

Thats why we have changed from baseline hemoglobin as the primary endpoint and transfusion avoidance as the key secondary endpoint.

That's really the linchpin of evaluating treatment of anemia.

So there's a whole range of interesting biomarker as we can into Michigan.

Unknown Executive: Right, you know, so the regulators, of course, want to see clinical outcomes being treated as important endpoints in clinical trials. So that's why we have changed from baseline hemoglobin as the primary endpoint and transfusion avoidance as a key secondary endpoint. And, you know, that's really a linchpin for evaluating a treatment for anemia after all. So, you know, there's a whole range of interesting biomarkers we're going to measure. You know, OVH is not viewed as important enough to be a primary endpoint. It's really very simple.

It is not viewed as important enough to be a primary endpoint its really very simple.

Thanks.

Thank you once again, if you would like to ask a question. Please press Star then the number one on your telephone keypad.

Next question comes from the line of Brian Abrahams of RBC capital markets. Your line is open.

Hey, good morning, Thanks for taking my questions and congrats as well on the early launch.

On my first question is on the launch I was wondering if you could maybe parse out the pent up demand for patients new to Orla day, or I guess I'm wondering how long into the year might you expect first quarters rate of switching from existing acute or prophylactic therapies to continue.

Brian Corey Abrahams: Thank you. Thank you. Once again, if you would like to ask a question, please press star, then the number one on your telephone keypad. The next question comes from the line of Brian Abrams of RBC Capital Markets. Your line is open.

Hey, Brian just trial and good good good question and yes, there was pent up demand and we expected that.

Unknown Executive: Hey, good morning, thanks for taking my questions and congratulations as well on the early launch. My first question is about the launch. I was wondering if you could maybe parse out the pent-up demand for patients new to Orlodeo. I guess I'm wondering how long into the year might you expect the first quarter's rate of switching from existing acuter prophylactic therapies to continue? Hey, Brian's Charlie, good question.

There are always early adopters, what we're really pleased about is the demand is continuing so we're seeing patients switch to early day literally every day. So and then as I mentioned in my remarks, there's a lot of opportunity to come as all of these physicians, who we've reached but haven't yet prescribed theyre, telling us that they are planning to have the <unk>.

Conversations with their patients and prescribed in the future. So a lot of opportunity to come throughout the year.

Unknown Executive: And yeah, there was pent-up demand, and we expected that. So they're always early adopters. What we're really pleased about is the demand is continuing, so we're seeing patients switch to Erle deo literally every day. And then, as I mentioned in my remarks, there's a lot of opportunity to come, as all these physicians who we've reached but haven't yet prescribed. They're telling us that they're planning to have the conversation with their patients and prescribe them in the future. So there's a lot of opportunity for them to come throughout the year.

Yeah. We don't think this is just.

One quarter pop and then it's going to Peter out Brian that we expect that this is going to continue to be strong through the course of the year and we said, it's a $500 million plus global peak sales product and we're even more confident in that.

Got it that's really helpful. And then I am curious what sort of feedback you're getting on how real world efficacy and safety and Tolerability.

Unknown Executive: Yeah, we don't think this is just a, you know, one-quarter pop, and then it's going to peter out, Brian. We expect that this is going to continue to be strong through the course of the year, and we've said it's a $500 million-plus global peak sales product, and we're even more confident in that. Got it, that's really helpful. And then I'm curious what sort of feedback you're getting on how real-world efficacy, safety, and tolerability are comparing to the clinical trial setting.

Is comparing to the clinical trial setting any surprises there and then I know, it's early days, but I'm curious, what you're seeing with respect to persistence and compliance for.

For the new patients to therapy versus the rollovers from the expanded access and quick start.

Yes, I'll take the first part Charlie if you could take the second that that'd be great. So I'm smiling.

You asked that question, because the docs and patients told us that these drugs do way better in the real world than they do in clinical trials and so I think our clinical trials doesn't really represent the real benefit that we're seeing from patients Megan mentioned that we're looking at the data from apex two.

Unknown Executive: Any surprises there? And then, I know it's early days, but I'm curious what you're seeing with respect to persistence and compliance for new patients to therapy versus the rollovers from the expanded access and quick start programs. Yeah, I'll take the first part, Charlie; if you could take the second, that'd be great.

Now 96 weeks and it just keeps looking better right and so.

Unknown Executive: So I'm smiling, as you asked that question, because the doctors and patients told us that these drugs do way better in the real world than they do in clinical trials. And so I think our clinical trials don't really represent the real benefit that we're seeing from patients. Megan mentioned that, you know, we're looking at the data from Apex 2, you know, out to now 96 weeks, and it just keeps looking better, right?

I'll say it again this drug works people are getting a real benefit they're getting control of their disease and oh by the way they're doing it on one capsule once a day. So it's just a huge huge benefit.

And then Brian as your question about the experience of rollovers versus versus new patients. The great majority of for patients who are on our clinical trials chose to continue on oral a day on the commercial world at because they are having such a great experience in the early word back from patients. So we hear back from our spur.

Unknown Executive: And so, you know, I'll say it again, this drug works. People are getting a real benefit. They're getting control of their disease. And, oh, by the way, they're doing it on one capsule once a day. So it's just a huge, huge benefit.

The pharmacy, and we hear back through physicians via our reps. The early feedback is very strong patients newly switching to oil a day are having a great experience as we expected. So we're we're pleased.

Unknown Executive: And then Brian, your question about the experience of rollovers versus new patients. The great majority of the patients who are in our clinical trials chose to continue on Orlydeo in the commercial world because they're having such a great experience. And the early word back from patients, and we hear back from our specialty pharmacy, and we hear back from physicians via our reps, the early feedback is very strong. Patients newly switching to Orladeo are having a great experience. as we expected. So we're pleased, and to my point earlier, we expect more patients to continue to switch to Orlydeo based on what they hear from their peers about their experience.

And to my point earlier, we X, we expect more patients to continue to switch to early day out.

Just on what they hear from their peers about their experience.

Great. Thanks, so much.

Youre welcome.

Thank you next question comes from the line of Stefan <unk> of Bank of America. Your line is open.

Hey, good morning, guys. Thanks for taking my questions.

I don't know a day as it relates to.

I guess treatment naive patients.

What is your latest market data, telling you about the percentage of patients.

Jay who are currently not on any therapy and related to that Im just wondering do you think.

Unknown Executive: Great, thanks so much. You're welcome. Thank you. The next question comes from the line of Desi Namad of Bank of America. You're on the line is open.

Once COVID-19 starts to clear and more offices start to reopen that the percentage.

Desi Namad: Hi, good morning, guys; thanks for taking my questions. On Oradeo, as it relates to, I guess, treatment-naive patients, what is your latest market data telling you about the percentage of patients who have H.E. Who are currently not on any therapy? And related to that, I'm just wondering, do you think, once COVID starts to clear and more offices start to reopen, that the percentage of weight of new patients or treatment I use patients that are receiving scripts will normalize more relative to what you're seeing in the early stages? Early in the morning

New patients for treatment naive patients that are receiving scripts.

It will normalize more relative to what youre seeing in the early stages of for lunch and then I have a couple of follow ups.

So yes, I think I think I got your question, but let me let me address it in a couple of different parts.

What we saw prior to the launch of for the day I was about 60% of patients are on prophylaxis and so in the first quarter, we're seeing more than half for the patients coming to oil a day of switching from those other protein products.

Unknown Executive: You're seeing this in the early stages of the launch, and then I have a couple of follow-ups.

Most of the remainder of patients switching to early day are switching from acute only treatment. So they may be naive to prophylaxis, but they've been treated with acute only our research tells us that the number of truly each day treatment naive patients they're not on acute not on Profi is small.

Unknown Executive: Yeah, I think I've got your question, but let me address it in a couple of different ways. What we saw prior to the launch of Oradeo is that about 60% of patients are on prophylaxis. And so in the first quarter, we're seeing more than half of the patients coming to Oralideo switching from those other profi products. Most of the remainder of patients switching to Oralideo are switching from acute-only treatment, so they may be naive to prophylaxis, but they've been treated with acute only.

That's a small opportunity and in the future, but but most of these patients are treated well by their doctors. They have at least an acute therapy and those patients are deciding to switch to proceed now that they can do it with an oral once daily option. So this is <unk>.

Can't stress. This enough. This is a switch strategy and its working right that is if you walk away with anything today walk away with this is a marketplace where people are on appropriate therapy and acute on demand therapy and they are switching to an oral drop right. So that's what we.

Unknown Executive: Our research tells us that the number of truly H-A-E treatment naive patients, you know, they're not on acute, not on profi, is small. That's a small opportunity in the future, but most of these patients are treated well by their doctors. They have at least an acute therapy, and those patients are deciding to switch to profi now that they can do it with an oral once daily option. So this is a, I can't stress this enough, this is a switch strategy, and it's working, right?

We saw that in the market research we saw it in the clinical trials and now it's playing out in the marketplace, which is great and it's only the first quarter and to your point about COVID-19 I think theres opportunity here right I think access.

Unknown Executive: That is, if you walk away with anything today, walk away with this: this is a marketplace where people are on prophesy therapy and acute on-demand therapy, and they're switching to an oral drug, right? So that's what we saw in the market research. We saw it in the clinical trials, and now it's playing out in the marketplace, which is great. And it's only the first quarter.

Of our reps to docs has been challenging in some parts of the country, they've been really diligent and resourceful.

<unk>.

Produce a great result in the first quarter, but I think as things open up more its going to get better and I think the same goes for docs and patients right.

Just been harder it's been telemedicine, maybe they are putting off their visit and so I think that gets better as more people get back <unk> and more people start to go see their doctor.

Unknown Executive: And to your point about COVID, I think there's an opportunity here, right? I think, you know, access for our reps to doctors has been challenging in some parts of the country. But they've been really diligent and resourceful, and they produced a great result in the first quarter.

Okay. Thanks for that color, Jon and then for.

Japan I know it's.

Very very early on the launch but in terms of the trajectory with two expected to be similar to what we would expect to see in the U S.

Unknown Executive: But I think as things open up more, it's going to get better. And I think the same goes for docs and patients, right? It's just been harder. It's been telemedicine. Maybe they're putting off their visit. And so I think that will get better as more people get vaccinated and more people start to go see their doctor.

And do you have any visibility on what pricing in Japan.

Again, you want to take that.

Sure sure so.

On the trajectory Praveen I think.

Unknown Executive: Okay, thanks for that color, John. And then for Japan, I know it's just very, very early in the launch, but in terms of the trajectory, which is expected to be similar to what we would expect to see in the U.S., and do you have any visibility on what pricing in Japan is?

Been sharing Japan is a very different market than the U S and Europe and is a decade behind them. So we're absolutely thrilled about the opportunity that Tory has to really build and shape. It. So I think our trajectory in the near term is more tempered on.

Also to Japan is in.

State of emergency currently with COVID-19.

Megan: Megan, you want to take that? Sure, so on the trajectory, Sazim, I think, as we've been sharing, Japan is a very different market than the U.S. and Europe.

And different rollout with a vaccine for that May also.

Flow that initial ramp, but as I shared in my remarks, we see this as a really strong long term potential as torry.

Megan: and is a decade behind. And so we're absolutely thrilled about the opportunity

Drive the adoption of proceeds but also the expansion on the market turmoil on diagnosis and treatment.

Megan: that Tori has to really build and shape it, so I think our trajectory in the near term is more tempered. Also, Japan is in a state of emergency currently with COVID and a different rollout with the vaccine, so that may also slow that initial ramp. But as I shared in my remarks, we see this as a really strong long-term potential as Tori drives it.

And then thank you for your second question.

With our pricing negotiations completing last week last month excuse me.

Net it out with a price of around 250000 U S dollars per patient per year and for asking we're really proud and excited about that price point and the opportunity. It represents for patients and for Torrey. So all in all there. They are equally excited to now be out of the gates like the U S has been for the last.

Megan: adoption of Profi, but also the expansion of the market through more diagnosis and treatment. And then, to your second question, with our pricing negotiations completing last week, last month, excuse me, we netted out with a price of around $250,000 U.S. dollars per patient per year. And for us, we were really proud and excited about that price point and the opportunity it represents for patients and for TORI. So, all in all, they're equally expensive.

A few months and we're excited to see what they can deliver.

Okay. Thank you and then maybe just one question if I could on on <unk>.

The trials that you're running I know it's difficult.

Take a GAAP on how long, it's going to take to enroll but for.

For the Navy for versus placebo.

Adequate responder trial excuse me would you expect both key readout at around the same time or do you think one could enroll faster than the other.

Bill you want for either of those.

Megan: Okay, thank you. And then maybe just one question, if I could, on PNH. For the two trials that you're running, I know it's difficult to, you know, take a guess on how long it's going to take to enroll, but

Sure it's hard to say.

It's very difficult to say at this stage on either of those possibilities could come to pass or I could read at approximately the same time or one could move much quicker than the other I think it's too early to know.

Unknown Executive: For the naives versus the placebo, the inadequates,

Okay. Thank you.

Unknown Executive: Adicit Responder Trial Excuse me, would you expect both to read out at around the same time, or do you think one could enroll

Yeah.

Thanks Dizzy.

Thank you next question comes from the line of channel one.

Unknown Executive: could enroll faster

Of Barclays. Your line is open.

Bill: Bill, you want to take that one? Yes, sure. Hi Tizene.

Hey, good morning. This is supplemental on for Gena. So just one question on on.

Bill: It's very difficult to say at this stage, and either of those possibilities could come to pass, right? They could read out at approximately the same time, or one could move much quicker than the other. I think it's too early to know.

<unk> edge.

So far on can you tell us about for water.

The size of the price could be for this space.

And then like what specific PK PD data was looked at to go ahead with the 500 B I D dosing versus close this 400.

Unknown Executive: Thank you. The next question comes from the line on Channel 1. Of Berkeley, the share line is open.

Alright, thanks for your questions.

So we're going to reserve additional details about the studies for.

Channel 1: Hey, good morning. This is Swap Mill on for Gina. So just one question on P&H. Can you tell us about what the size of the trials could be for this phase three and then, like, what specific PKPD data was looked at to go

Later this year.

Might be at a medical meeting or something like that so.

So.

We'll be happy to share all of those details at this stage.

Now with regard to selection of the dose the PK PD modeling.

Unknown Executive: ahead with the 500 mic BID dosing versus 400. Thanks for your questions.

Uses all of the information you have for example on.

In vitro.

Complement assays and.

Unknown Executive: So we're going to reserve additional details about the studies for later this year, maybe at a medical meeting or something like that. And we'll be happy to share all of those details at that stage. Now, with regard to selection of the dose, the PKPD modeling uses all of the information you have, for example, on in-vitro, consummate assays and, you know, things like clinical outcomes and biomarkers like LDH. This is where LDH actually is quite useful as a fairly rapidly responsive biomarker that follows the dosing.

Things like clinical outcome Biomarkers like LDH. This is where it actually is quite useful.

As a.

As a fairly rapidly responsive biomarker that follows the dosing.

So that.

Disclosures.

Through last year.

Yeah.

The higher doses, obviously superior to the low doses. So I think that it's that type of information that goes into the model as well as growth of <unk>.

Cost of the drug levels.

And let me let me just add on the size I mean, these are rare disease trials right and so.

Typically in rare disease trials, you need a couple of hundred patients on the drug for a year for safety, if it's a chronic therapy and.

Unknown Executive: And we saw that in our disclosures through last year about, you know, how the higher doses are obviously superior to the lower doses. So I think that it's that type of information that goes into the model as well as, of course, the drug level. And let me just add on the size.

The expectation is it will be something like that I think we ended up bill with over 300 in the Hai program when we filed so.

Roughly similar yes.

Yep Yep.

Okay. Thank you and then one follow up question so forth for the slightly higher.

Unknown Executive: I mean, these are rare disease trials, right? Typically, in rare disease trials, you need a couple hundred patients on the drug for a year for safety if it's a chronic therapy. And, you know, the expectation is it'll be something like that. I think we ended up, Bill, with over 300 in the HAA program when we filed. So, you know, roughly similar. Yeah.

A level that we see for India that goes for.

Previously.

Because it maybe was due to a shorter duration of pigment and make small sample size.

So do you expect <unk> via IV dosing over a period of time to get you below that $1 five up for Liberty.

Unknown Executive: Yeah. Okay, thank you. And then one follow-up question. So for the slightly higher price

So.

That particular target is not an endpoint in either study.

Unknown Executive: levels that we see for LDH.

So I think that's the most important.

Unknown Executive: That was previously presented, maybe due to a shorter duration of treatment and a small sample size. So do you expect 500 MbID dosing over a period of time to get you below that 1.5 upper limit of normal?

Response for the question in fact.

Sure.

Theyre actually there's nothing magical about one five times the upper limit of normal of the LDH.

And so it'll be I should certainly a useful biomarker.

And we're measuring it.

This included as a secondary endpoint, it's more relevant in the sitting when you're starting out with a high LDH not on.

Unknown Executive: So that particular target is not an endpoint in either study. So, you know, I think that's the most important response to the question, in fact.

C five inhibitor therapy.

But it's not as important.

Increase in hemoglobin and reducing the transfusions on improving the fatigue.

Unknown Executive: So there actually is nothing magical about 1.5 times the upper limit of normal for the LDA, and you know, LDH is certainly a useful biomarker, and we're measuring it, and it's included as a secondary endpoint. It's more relevant in the setting when you're starting out with a high LDH and you're not on C5 in therapy, but it's not as important as increasing the hemoglobin and reducing the transfusions and improving the fatigue.

Sorry.

Specifically on this you the question I think.

What we've seen in all of the other studies of all of the other complement inhibitors.

On the sponsors have published individual patient data over a long period of time.

The day Opi's fluctuates.

That would be typical in this disease, we expect to see the same thing.

People for long enough Youll day, I, just kind of fluctuate.

Todd.

Unknown Executive: So, you know, specifically in asking you the question, I think what we've seen in all of the other studies of all of the other studies of complement inhibitors when sponsors have published individual patient data over a long period of time, you see that the LBH fluctuates, and that'll be typical in this disease; we expect to see the same thing. So, you know, observe people for long enough, and the LDH is going to fluctuate over time.

If people on the other day, Nick and not being transfused on feeling well.

We've achieved all of the principal objectives for treatment of Taylor.

What's most exciting is we now have agreement with the FDA that hemoglobin as the primary end point change from baseline, which is really exciting.

Well, thank you for taking our questions.

Okay welcome.

Unknown Executive: But if people are not amemic and not being transfused and feeling well, then we've achieved all of the principal objectives of treatment for TNH. Yeah, and what's most exciting is we now have, you know, agreement with the FDA that hemoglobin is the primary endpoint, you know, change from baseline, which is really exciting.

Thank you Youre for.

Final question comes from the line of Jonathan <unk> of.

Of GMP Securities. Your line is open.

Hey, good morning, and sharing my congrats on strong launch just just a few for me I'm wondering if the $10 9 million figure for the day. If that's net of your royalty payment and if theres any meaningful contribution from the HEU on France.

Unknown Executive: Thank you for taking up.

Yes, so the $10 $9 million is not in Nash.

Unknown Executive: Thank you. Your final question comes from the line of Jonathan Wollabin.

The royalty payments from royalty payments are just as a reminder, 875% of those net revenues up to 350, and then once we get over 350, it drops down to 275.

Jonathan Patrick Wolleben: Of JMP securities, your line is over. Hey, good morning, and I'm sharing my congrats on a strong launch. Just a few for me.

Unknown Executive: I'm wondering if the $10.9 million figure four, Ladeo, is net of your royalty payment and if there's any meaningful contribution from the ATU and Frank. Yeah, so the $10.9 million is not net of the royalty payments. The royalty payments are, just as a reminder, 8.75% of those net revenues up to 350, and then once we get over 350, it drops down to 2.75. Where that hits is, you know, below the line between operating income and net income, basically as an interest expense, John.

Weighted that hits is below the line between operating income and net income.

And basically is on interest expense Jon.

In terms of the HEU no nothing nothing nothing meaningful in that was that was all U S sales of $10 nine.

Great and then just one.

One question on the market, whether youre seeing patients switched from acute if you have any sense on the percentage of patients now of all HBV patients who are on prophylaxis, and where do you think that kind of levels out in the future now with you guys on the market and Youre seeing more switches over to prophylaxis.

Unknown Executive: In terms of the ATU, no, nothing, nothing, nothing meaningful. That was all U.S. sales, the 10.9. Great. And then I've just got one question on the market. If you're seeing patients switch from acute, if you have any sense of the percentage of patients now of all H.A. patients who are on prophylaxis, and where do you think that kind of levels out in the future now that you guys are on the market and you're seeing more switches over to prophylaxis? Good question, John. As I mentioned, what we see, we're very confident that at the time of the Orlydeo launch, about 60% of U.S. H.A.E. Patients were on prophylaxus.

Good question, Jon So as I mentioned, what we see we're very confident that at the time of the order the day launch about 60% of U S. HIV patients are on prophylaxis and so as patients also switch from acute therapy only two oral <unk> that number is going to grow and what physicians tell us is that in the few.

Sure.

About 80% of patients they expect 80% of patients to be treated with prophage. So the acute market is shrinking every day and we expect that same kind of trend to occur in Europe. After our launch there and it's turning out that the market research that Charlie has been doing is playing out in the marketplace.

Unknown Executive: And so as patients also switch from acute therapy only to orale, that number is going to grow. And what physicians tell us is that in the future, that it's about 80% of patients; they expect 80% of patients to be treated with prophecy. So the acute market is shrinking every day.

Okay.

Perfect well, thanks for taking the questions and congrats again.

Thanks, Jon Thanks, Jon.

Thank you there are no further question at this time I will now turn the call back to Jon Stonehouse for closing remarks.

Unknown Executive: And we expect that the same kind of trend to occur in Europe after our launch there. Yeah, and it's turning out that the market research that Charlie has been doing is playing out in the marketplace. Perfect. Well, thanks for taking the questions and congratulations again. Thanks, John. Thank you. There are no further questions at this time.

So I've done a lot on earnings calls in my 14 years at Biocryst and I got to say this is one of the most fun and exciting ones that I've been a part of we've got a great launch with a great product and a great team and we're just getting started and so it's really exciting.

Unknown Executive: I will now turn the call back to John Stonehouse for his closing remarks. So, I've done a lot of earnings calls in my 14 years at Biocrist, and I've got to say this is one of the most fun and exciting ones that I've been a part of. You know, we've got a great launch with a great product and a great team, and we're just getting started. And so it's really exciting to have a product that's generating real revenue. We have the green light to go into Pivotal Studies with our next program. That's an entire pipeline in a molecule.

To have a product that's generating real revenue.

We have a green light to go into pivotal studies with our next program. That's an entire pipeline in a molecule and so we're going to be starting up pivotal studies later this year.

So it doesn't get a whole lot better than that and.

Our focus now is execution I'm, just so proud of the team and just incredible focus and execution that they've delivered on and we will continue to deliver on so thanks for your interest in our company and have a great day.

Ladies and gentlemen, this concludes today's conference call. Thank you for joining you may now disconnect.

Jon P. Stonehouse: And so we're going to be starting up pivotal studies later this year. So it doesn't get a whole lot better than that. And, you know, our focus now is execution. I'm just so proud of the team and the incredible focus and execution that they've delivered on and will continue to deliver on.

Great day.

Jon P. Stonehouse: So thanks for your interest in our company and have a great day. Ladies and gentlemen, this concludes today's conference call. Thank you for joining us. You will now disconnect.