Q2 2019 Earnings Call

Operator: This conference is being recorded. I would now like to turn the call over to Jon Bluth at BioCryst. Sir, you may begin.

John D. Bluth: Thank you, Sydney. Good morning, and welcome to BioCryst's second quarter 2019 Corporate Update and Financial Results conference call. Today's press release is available on our website. Participating with me today are CEO Jon Stonehouse, Chief Medical Officer Dr. Bill Sheridan, CFO Tom Staub, and Chief Business Officer Megan Sneezin-Sklar.

Thank you Sandy good morning, and welcome to Biocryst second quarter, 2019, corporate update and financial results Conference call.

Today's press release is available on our web site.

Participating with me today are CEO , Jon Stonehouse, Chief Medical Officer, Dr., Bill Sheridan CFO , Tom Staab, Chief business Officer, Megan see Stansky.

Unknown Executive: 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 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. 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 Jon Stonehouse.

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 into our 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.

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 Jon Stonehouse.

Jon P. Stonehouse: Thank you, Jon, and thank you all for joining us this morning. We're excited to update you on our progress since announcing our positive phase 3 results for our HAE prophylaxis program and share some important advancements in our pipeline. Since May, we've been engaging directly with treating physicians and HAE patients to gather important insights to build out our commercialization strategy. The demand and excitement they are expressing for our once-daily oral prophylactic therapy, BCX7353, have been resounding and consistent. Ten days ago, we had the privilege of attending the HAEA Patient Summit in Atlanta, which gathered over a thousand attendees from the HAE community. Over the course of the summit weekend, we had one-on-one interactions with hundreds of patients. So what did we learn?

Thank you John and thank you all for joining us this morning.

We're excited to update you on our progress since announcing our positive phase three results for our AG profile access program.

And share share some important advancements in our pipeline.

Since may weve been engaging directly with treating physicians and HIV patients together important insights to build out our commercialization strategy.

The demand and excitement they are expressing for our once daily oral prophylactic therapy PCX 70, 353 has been resoundingly inconsistent.

10 days ago, we had the privilege of attending the H E patient summit in Atlanta, which gathered over a thousand attendees from DHL community.

Over the course of the summit weekend, we had one on one interactions with hundreds of patients.

So what did we learn.

Jon P. Stonehouse: Many patients are doing better preventing or treating their attacks, but they want more. Patients are tired of sticking themselves with needles. During the conference, patients were pulling BioCryst team members aside to remind us that they're waiting for our oral therapy, and we need to go fast. Some are experiencing challenges with access. It causes stress and anxiety for these patients, which is a known trigger for HAE attacks.

Many patients are doing better preventing or treating their attacks, but they want more.

Patients are tired of sticking themselves with needles. During the conference patients were pulling biocryst team members aside to remind us they're waiting for our oral therapy and we need to go fast.

Some are experiencing challenges with access it causes stress innings I'd for these patients which is a known trigger for AG attacks.

Unknown Executive: Access is an important topic we are in the process of planning for, and we will be prepared when we launch so that we are able to knock down as many of these barriers as we can. There is a strong desire for our product's profile and the benefits it can provide. An oral drug that reduces attacks and brings the convenience and a lifestyle patients want. Patients told us they were excited. Their wait for a once-daily oral prophylactic therapy is nearly over, and they want our product. So, what have we concluded?

Access is an important topic, we are in the process of planning for and we will be prepared when we launch so that we are able to knock down as many of these barriers as we can.

There's a strong desire for our products profile in the benefits. It can provide an oral drug that reduces attacks and brings the convenience and a lifestyle patients one.

Patients told us they're excited they're wait for once daily oral prophylactic therapy is nearly over and they want our product.

So what do we concluded.

Unknown Executive: We have an amazing drug, and they want it approved. This is a direct quote from a mother of a teenager in our clinical trial. They want more than they have now with injectables. Being controlled is not enough for many.

We have an amazing drug and they want it approved this is a direct quote from a mother of a teenager in our clinical trial.

They want more than they have now with injectables being controlled is not enough for many.

Unknown Executive: While there are new treatment options available to them, patients tell us they are still having breakthrough attacks. No drug is perfect. With the finish line now in sight, the excitement building among patients is, The enthusiasm for 7353 is not confined to the U.S. Representatives from outside the U.S. attended as well, and their response was the same. No real prophylactic market exists outside the U.S., and they want to build it with $73.50 and Oral Therapy in Europe, Japan, Latin America, and across Asia Pacific. This global commercial opportunity is one of the many reasons we're happy to add Megan Snezinski, our Chief Business Officer, to our leadership. In her leadership role at PTC Therapeutics, Megan helped drive significant global revenue for multiple products through a mix of their own commercialization and partnership, and she will add tremendous value to BioCryst as we evaluate and then execute a similar strategy. Our patient and physician interactions, including our ongoing market research, are aligned. Overall, 7353 represents a significant market opportunity, and we are actively preparing for its launch next year. So what's next?

Well there are new treatment options available to them patients tell us they are still having breakthrough attacks.

No drug is perfect.

With the finish line now in sight.

The excitement building among patients is amazing.

The enthusiasm for 70 353 is not confined to the U.S.

Representatives from outside the U.S. attended as well and their response was the same.

No real prophylactic market exists outside the U.S. and they want to build it was 70 353, an oral therapy in Europe , Japan, Latin America and across Asia Pacific.

This global commercial opportunity is one of the many reasons, we're happy to add Megan sneezing ski our chief business officer to our leadership team.

In her leadership role at PTC Therapeutics, Megan helped drive significant global revenue for multiple products through a mix of their own commercialization and partnerships.

And she will add tremendous value to biocryst as we evaluate and then execute a similar strategy.

Our patient and physician interactions, including our ongoing market research are aligning overall 70 353 represents a significant market opportunity and we are actively preparing for the launch next year.

So what's next we are in the process of completing patient physician and payer market research to refine our go to market strategy.

Jon P. Stonehouse: We're in the process of completing patient, physician, and payer market research to refine our go-to-market strategy. Bill's making great strides in filling out his medical affairs team, which is holding regional advisory boards to build physician relationships and gather their feedback much more broadly than we've been able to do in the past. We are also developing the U.S. and ex-U.S. commercial launch and resource and are filling critical roles to support a successful launch. We look forward to sharing more on our commercial launch strategy with you this fall.

Bills, making great strides in filling out as medical affairs team, that's holding regional advisory boards to build physician relationships and gather their feedback much more broadly than we've been able to do in the past.

We are also developing the U.S., an ex us commercial launch and resource plan.

And our filling critical roles to support a successful launch.

We look forward to sharing more on a commercial launch strategy with you. This fall.

Let me now move to our oral factor D program, which is another exciting priority for us.

Jon P. Stonehouse: Let me now move to our Oral Factor D program, which is another exciting priority. We are just months away from reporting clinical data from our phase one trial for our oral factor D inhibitor, BCX9930, which represents an even larger commercial opportunity for our company. The current market for complement-mediated disease therapy is $4 billion, double that of HAE, and could double again as treatments and development target more indications. The well-established development path and widely available and accepted biomarkers enable rapid advancement of 9930 and allow us to show meaningful clinical differences in a small number of patients over a short period of time. The timeline to show progress is very attractive with this program. Bill will go into more detail here, but the takeaway is that you get a much faster answer to understand if you have a drug, and we'll get an important answer soon when we report out the PK and PD results of Phase 1.

We're just months away from reporting clinical data from our phase one trial for oral factor Xa inhibitor, BCS 99, 30, which represents an even larger commercial opportunity for our company.

The current market for complement mediated disease therapy is $4 billion double that of HIV and could double again as treatments in development target more indications.

The well established development path and widely available in excepted Biomarkers enable rapid advancement of 99 30.

And allow us to show meaningful clinical differences in a small number of patients over a short period of time.

The timeline to show progress is it is very attractive with this program.

Bill will go into more detail here, but the takeaway is that you get to a much faster answer to understand if you have a drug.

And we will get an important answer soon when we report out the PK and PD results of the phase one.

Following our phase one read out we will move to a pre proof of concept trial in a small number of ph patients.

Jon P. Stonehouse: Following our Phase I readout, we will move to a proof-of-concept trial in a small number of P&H patients. This patient data will provide further proof and set us on a path to bring another valuable oral drug to many patients with rare diseases. And finally, another important piece to the puzzle is the capital to fund all of this. The good news is that we have roughly $100 million on the balance sheet and a number of different options to bring in more capital. And I'd like to turn the call over to Tom to discuss this in more detail. Thank you, Jon.

This patient data will provide further proof and set us on a path to bring another valuable oral drug to many patients with rare disease.

And finally, another important piece of the puzzle is the capital to fund all of this the good news is we have roughly a $100 million on the balance sheet in a number of different options to bring in more capital and I'd like to turn the call over to Tom to discuss this in more detail.

Tom.

Thank you John .

Tom Staub: With the upcoming commercial launch of 7353 and the predictive clinical data coming from our Oral Factor D program, we are evaluating the best approach to fund these opportunities and have a range of options to consider. These options cover both equity and debt approaches, as well as global business development opportunities. We are pleased that we have multiple alternatives to support our program.

With the upcoming commercial launch of 70, 353, and the predictive clinical data coming from our oral factor D program. We are evaluating the best approach to fund these opportunities and have a range of options to consider.

These options cover both equity and debt approaches as well as global business development opportunities.

We are pleased that we have multiple alternatives to support our progress.

Tom Staub: As you may recall, earlier this year, we extended our cash runway and enhanced our financial flexibility by closing a $100 million secured credit facility. Based on our successful APEX II trial, we currently have the ability to draw $30 million of non-dilutive capital at our option, that would be additive to our current cash balance, as well as an additional $20 million that would be available to us following FDA approval of 7350. I am thrilled to have Megan's experience on the team to help us evaluate the global market opportunities for 7353 in order to assess where we want to commercialize it ourselves and where we want to explore partnerships that provide non-dilutive capital. In order to gauge our future capital requirements to support all of our programs, we need to complete our launch planning and understand BCX9930 Phase 1 data. We can then pair that information with our commercial investment, clinical development, and partnership plans to develop the right financing mix.

As you May recall earlier this year, we extended our cash runway and enhanced our financial flexibility by closing $100 million secured credit facility.

Based on our successful apex to trial, we currently have the ability to drop $30 million of non dilutive capital at our option that would be additive to our current cash balance as well as an additional $20 million that would be available to us following FDA approval of 70 353.

I am thrilled to have megan's experience on the team to help us evaluate the global market opportunity opportunities for 70 353 in order to assess where we will want to commercialize it ourselves and where we want to explore partnerships that provide non dilutive capital.

In order to gauge our future capital requirements to support all of our programs, we need to complete our launch planning and understand BCS 99, 30 phase one data.

We can then pair that information with our commercial investment clinical development and partnership plans to develop the right financing mix.

Tom Staub: We look forward to sharing more details on our capital plans with you in the fall. Our detailed second quarter 2019 financial results can be found in the press release we issued this morning, but there are a few items I need to highlight. We incurred a non-cash charge of $2 million in the second quarter of 2019 associated with realizing compensation expense on two performance-based tranches of stock options. Although a cashless event and outside our guidance ranges, it is important to note there will be a continuing charge of approximately $3.5 million as these options continue to vest throughout the remainder of the year, and thus, these continuing charges will increase our loss per share in the third and fourth quarters of 2019 beyond our normal run rate.

We look forward to sharing more details on our capital plans with you in the fall.

Our detailed second quarter 2019 financial results can be found in the press release, we issued this morning.

But there are few items I need to highlight.

We incurred a non cash charge of $2 million in the second quarter of 2019 associated with realizing compensation expense on to performance based traunches of stock options.

Although a cashless event and outside our guidance ranges. It is important to note there will be a continuing charge of approximately $3.5 million as these options continue to vest throughout the remainder of the year and thus these continuing charges will increase our loss per share in the third and fourth quarters of 2019 beyond our normal run rate.

Tom Staub: This charge has no impact on our cash utilization or our operating expense guidance for future quarters and the year. With two quarters under our belt, I also wanted to call your attention to cash utilization. We ended the second quarter with approximately $98 million in cash. Our operating cash use for the first half of 2019 was $53 million and is annualizing at the lower end of our projected range of $105 to $130 million. Additionally, we continue to expect our 2019 operating expenses will be comfortably in the previously guided range of $120 to $145 million. As we consider the best options for raising additional capital, it is very rewarding to see the strong patient and physician enthusiasm for 7353 and the rapid advancement of our oral Factor D inhibitor program. Now, I'd like to turn the call over to Bill.

This charge has no impact on our cash utilization, where our operating expense guidance for future quarters in the year.

With two quarters under our belt I also wanted to call your attention to cash utilization.

We ended the second quarter with approximately $98 million in cash our operating cash use for the first half of 2019 was $53 million and is annualizing at the lower end of our projected range of $105 million to $130 million.

Additionally, we continue to expect our 2019 operating expenses will be comfortably in the previously guided range of $120 million to $145 million.

As we consider the best options for raising additional capital. It is very rewarding to see the strong patient and physician enthusiasm for 70 353.

And the rapid advancement of our oral factor Xa inhibitor program.

Now I'd like to turn the call over to Bill.

William P. Sheridan: Thanks Tom. Like Jon, I attended the HAE Association Patient Summit in Atlanta, and it was wonderful to see the engagement of patients and caregivers with our team and the tremendous encouragement that they gave to all of us in launching our oral once-a-day calocrine inhibitor. They came up to our booth or found us in the halls to tell us just how much this treatment option means to them and their families after years of IV and subcutaneous injections. Many patients, even patients who are well controlled on these therapies, told us that an oral drug is a dream come true. Since we announced the APEX2 data and first shared it with our clinical trial investigators in the United States, Canada, and Europe, the response to the data has been very positive, with growing momentum.

Thanks, Tom.

Like John I attended the HIV Association patients summit in Atlanta.

And it was wonderful to see the engagement of patients and caregivers with that team and a tremendous encouragement that I gave to all of us in launching an oral once a day kallikrein inhibitor.

They came up to add food fantasy into holes.

Tell us just how much this treatment option means to them and their families.

After years of Ivy and subcutaneous injections.

Many patients even patients who are well control them. These therapies told us that an oral drug is a dream come true.

Since we announced that it takes to data and finish it with that clinical trial investigators in the United States, Canada and Europe .

Response to the data has been very positive with growing momentum.

In the last few months and medical team has conducted a series of regional advisory boards comprised to viewers allergists, an immunologist, who treat patients with HIV.

William P. Sheridan: In the last few months, our medical team has conducted a series of regional advisory boards comprised of U.S. allergists and immunologists who treat patients with H.A.E. Universally, these physicians have responded enthusiastically to the efficacy, safety profile, and approvability of 7353 based on the APEX II results and tell us that the patient experience in the study with 50% of patients experiencing at least a 70% reduction in attack frequency with a once-daily oral drug will absolutely translate into patients using this medicine. Last week, I also had the opportunity to visit with staff of an experienced HEE clinical trial site that has been a major contributor to trials of every new treatment introduced for HEE, and I was told this drug will be life-changing for the HEE community.

Individually. These physicians have responded enthusiastically to the efficacy safety profile and Approvability of 70 353 based on the apex to results and tell us that the patient experience on study with 50% of patients experiencing at least a 7% reduction in attack frequency with a once daily oral drug will absolutely translate into patients using this medicine.

Lastly, I also had the opportunity to visit with stuff is an experienced HG clinical trial site.

That has been a major contributor to trials of every new treatment introduce HIV and was told this drug will be less changing for the ha community.

William P. Sheridan: So, I'm very happy to report that all the activities needed to wrap up the 48-week dosing and analyses in Apex-2 and Apex-S are on track, and we are very confident in our Q4 target to submit the US NDA and Q1 2020 target to submit both the European MAA and JNDA in Japan. Moving on to our Oral Factor D Inhibitor Program for Complement-Mediated Diseases, I'm very pleased to say that we have also had strong support from U.S. and international expert physicians for progressing 9930 in the clinic as fast as possible because of the medical need across multiple different disease indications where the alternative pathway of complement is the dominant bad actor, and because they've been hoping for a factor D inhibitor and especially an oral complement inhibitor The non-clinical profile of 99.30 is extremely promising. The key findings... were absolutely disproportional exposure over a wide range.

So I'm very happy to report that only activities needed to ramp up the 48 week dosing and analyses Ics to exist are on track.

And we are very confident in our Q4 target to submit to us in da.

In Q1, 2020 target to submit both the European M&A and JNDA in Japan.

Moving onto our effected inhibited programs complement mediated diseases I'm very pleased to say that we have also had strong support from us and international ex that physicians are progressing 99 city in the clinic fast as possible.

Because of the medical need across multiple different disease indications with the alternative pathway of complement is the dominant bad actor.

Because I've been hoping for effected inhibitor, and especially an oral complement inhibitor drug.

The Nonclinical profile, if not done 30 is extremely promising.

The key findings.

Well, absolutely does proportional exposure over a big range.

William P. Sheridan: Human Equivalent Dose of the Noel Dose Level of 5 grams per day prompt and sustained suppression of the alternative pathway of complement after oral dosing at a fraction of the no oral dose in non-human primates. What these findings mean is that we should have a very wide margin of safe dosing in the clinic to achieve our therapeutic goals in complement inhibition. The Phase 1 Clinical Trial in Healthy Subjects is off to a great start and is progressing rapidly. We look forward to sharing the safety, tolerability, PK, and PD results of this study in the fourth quarter. As we've seen with other programs in the field, phase one healthy subject studies with complement inhibitors provide vastly more information than the usual phase one clinical trial because we can measure suppression of this pathway in healthy subjects using alternative pathway biomarkers that are predictive of clinical effects, and because this greatly facilitates dose selection for trials in the target diseases.

Human equivalent doses of the Noel dose level of five grams per day.

Prompt and sustained suppression of the alternative pathway of complement of oral dosing at a fraction of the Noel dose in non human primates.

What these findings name is that we should have a very wide margin of site dosing in the clinic to achieve that therapeutic goals in complement inhibition.

The phase one clinical trial in healthy subjects is off to a great start and is progressing rapidly.

Look forward to sharing the safety Tolerability PK and PD results of this study in the fourth quarter.

As we've seen with other programs in the steel phase one healthy subjects studies with complement inhibitors provide vastly more information than the usual phase one clinical trial, because we can measure suppression of this pathway in healthy subjects using alternative pathway biomarkers that are predictive of clinical effects and because this greatly facilitates dose selection for trials into target diseases.

Also very important and unlike many other disease areas. They are commercially available standardized size what was led by PS site that enable comparison across programs.

William P. Sheridan: Also very important, and unlike many other disease areas, there are commercially available standardized assays like the Weisslab AP assay that enable comparison across programs. Progression of 99.30 from discovery project to non-clinical development to phase one in the clinic has gone very fast, and we have closed most of the gap with the oral factor D inhibitor competition. We look forward to moving quickly to clinical proof-of-concept in patients with PNH in 2020. That will be very exciting as we can easily measure multiple biomarkers like LDH, reticulocytes, and bilirubin that are both predictive of clinical benefit and very quick to obtain after very brief durations of dosing. You can look forward to seeing data on 99.13 PNH subjects in 2020.

Progression of 99 30 from Discovery project Nonclinical development phase one in the clinic.

Has gone very fast and we have closed most of the get with the oral effectively inhibit a competition.

We look forward to moving quickly to clinical proof of concept in patients with an aging 2020 .

That will be very exciting as we can easily mission multiple biomarkers like LDH particular sites that we've proven that about predictive of clinical benefit and very quick to obtain very brief durations of dosing.

You can look forward to seeing data on 99 city subjects in 2020.

Megan Sneezin-Sklar: Thanks, Bill. For all the reasons Jon, Tom, and Bill have laid out, I'm really excited to be here at this pivotal stage for BioCryst. It's a privilege to be part of a company that's fully dedicated to bringing life-changing oral treatments to patients with rare diseases. Looking at what's ahead, I'm eager to bring my experience and energy to building the capabilities and infrastructure we need to commercialize 7353 and to advance our pipeline with the ultimate goal of creating value for our patients. In this role, I have accountability for business development and corporate strategy, supply chain, and program management.

Now I'd like to turn the call over to Megan.

Thanks Bill.

For all the reasons, John Tom and Bill have laid out I'm really excited to be here at this pivotal stage for Biocryst, it's a privilege to be part of a company that's fully dedicated to bringing life changing oral treatments to patients with rare diseases.

Looking at what's ahead I'm eager to bring my experience and energy the building the capabilities and infrastructure, we need to commercialize 70, 353 and to advance our pipeline with the ultimate goal is to create value for our patients.

In this role I have accountability for business development, and corporate strategy supply chain and program management.

Megan Sneezin-Sklar: Right now, my focus is on execution across three key areas: launch readiness, advancing our development programs, and exploring BD opportunities that fit with our strategy. First, for launch readiness, these activities include finalizing our launch plans based on the physician, patient, and market insights Jon mentioned earlier, actively building out our supply and distribution channel both in the U.S. and outside the U.S. to be well positioned to support the strong patient demand we anticipate, and concentrating our effort on the key resources and investments we need to support a highly successful launch. The regulatory submissions are also the team's top priority, and we remain on track for the FDA submission in the fourth quarter and the EMA and Japanese submissions in the first quarter of 2020.

Right now my focus is on execution across three key areas.

Launch readiness advancing our development programs and exploring BD opportunities that fit with our strategy.

First for launch readiness. These activities include finalizing our launch plans based on the physician patient and market insights John mentioned earlier.

Actively building out our supply and distribution channel both in the U.S. and ex us to be well positioned to support the strong patient demand we anticipate.

And concentrating our efforts on the key resources and investments we need to support a highly successful launch.

The regulatory submissions are also the teams top priority and we remain on track for the FDA submission in the fourth quarter and the May in Japanese submission in the first quarter of 2020.

Megan Sneezin-Sklar: Knowing that every day matters to the patients we serve, we will be fully focused on ways to rapidly advance our HAE programs, as well as our early stage assets, like 9930. Lastly, on the BD front, as Tom noted, we're actively exploring potential partnering opportunities to maximize the value of our assets. For example, the Accelerated Regulatory Path with the 7353 Sakagaki designation in Japan presents a real opportunity to consider a local partner, one who has the capabilities and strength to bring our product to a market which currently has no approved prophylaxis treatment. I, too, was overwhelmed by the reception BioCryst received at the recent HAEA patient summit. This is a highly engaged, aligned, and driven patient organization, and I'm very excited to be here and to partner with this amazing community to bring our product to patients in need. Now, let me turn it back over to Jon.

Knowing every day matters to the patients we serve we will be fully focused on ways to rapidly advance our ha E programs as well as our early stage assets like 99 30.

Lastly on the BD front as Tom noted, we are actively exploring potential partnering opportunities to maximize the value of our assets.

For example, the accelerated regulatory path with 70 353 seconds Jackie designation in Japan presents a real opportunity to consider a local partner.

One who has the capabilities and strength to bring our product to market, which currently has no approved prophylactic treatment.

I too was overwhelmed by the reception Biocryst received at the recent ha patient summit.

This is a highly engaged aligned in driven patient organization and I'm very excited to be here and to partner with this amazing community to bring our product to patients in need.

Now, let me turn it back over to John .

Jon P. Stonehouse: Thanks, Megan. Let me wrap it up with this. In my 12 years at BioCryst, the company has never been in a stronger position. We are hearing extraordinary customer demand for BCX7353. Next year, we will launch the first oral prophyHAE treatment, and proof-of-concept data on a potential blockbuster oral factor D inhibitor is coming soon. And finally, we have the financial flexibility to get the capital we need to do so. That concludes our prepared remarks. Operator, we're now ready to open it up for questions.

Thanks Megan.

Let me wrap up with this in my 12 years at Biocryst. The company has never been in a stronger position.

We are hearing extraordinary customer demand for BCS 70 353.

Next year, we will launch the first oral prophy HIV treatment.

In a proof of concept data on a potential blockbuster oral factor xa inhibitors coming soon.

And finally, we have the financial flexibility to get the capital we need to get there.

That concludes our prepared remarks, operator, we're now ready to open it up for questions.

Operator: Thank you. Ladies and gentlemen, if you have a question at this time, please press the star and the number one key on your telephone keypad. If your question has been answered or you wish to remove yourself from the queue, please press the pound key. To prevent any background noise, we ask you please place your line on mute once your question has been stated. Once again, that is star and number one. Our first question comes from Jessica Fye with JP Morgan. Your line is open. Hi, this is Daniel speaking on behalf of Jessica. Thanks for taking our question. There are a couple of questions here.

Thank you ladies and gentlemen, if you have a question at this time please.

Star.

And the number one key on your telephone keypad.

My question has been answered from move yourself from the queue. Please press the pound key.

To prevent any background noise. They ask you. Please please your line on mute. Once your question has been stated once again that is star and the number one.

And our first question comes from Jessica Fey with JP Morgan Your line is open.

Hi, This is Daniel for just Scott. Thanks for taking your question a couple of questions here.

Jessica Macomber Fye: First, have you met with the FDA to discuss the results of EPICS-2? Second, given that EPICS-S is an open-label trial, can you speak at a high level about whether the safety in that study is consistent with or meaningfully different from what you have described in EPICS-2? And last, with the Phase III acute study now postponed to 2020 for initiation, is it possible that EPICS-5 goes on the back burner for a period of time to preserve cash and focus on the filing for prophylactic? Thank you.

First have you met the ft to discuss the results of Opex too.

Second given that FX is an open label trial can you speak at a high level to whether the safety in that study is consistent with or meaningfully different from what you have described in opex to last with the phase three acute study now postponed to 2024 initiation is it possible that goes on the back burner for a period of time to preserve cash and focus on the filing for prophylactic. Thank you.

Yes, Hi, Danielle ill take the first couple with regard to regularly regulatory interactions. The NDA plan, we've had a pre NDA meeting.

William P. Sheridan: Hi Danielle, I'll take the first couple. With regard to regulatory interactions on the NDA plan, we've had our pre-NDA meeting. That went fine. So, you know, all of the elements that we need to submit for the NDA are on track in the fourth quarter, including the rate-limiting elements, which are the long-term safety of Apex-S and Apex-2. On that front, there's really no news really. We haven't learned anything new that we haven't seen previously in the Phase 2 and the Phase 3 programs. As I have mentioned on previous calls, we have an independent data monitoring committee that meets regularly to review cumulative data, and they have not suggested any changes to the protocols. So that's ongoing, and we look forward to completing that work later this year.

That went fine sorry, it all of the elements that we need to submit for the India are on track in the fourth quarter.

Including the rate limiting elements, which the long term safety from Texas and a fixed.

On that front.

There's no news really we haven't learned anything new that we haven't seen previously.

In the phase two in the phase three program.

As I have mentioned on previous calls we have an independent data monitoring committee that meets regularly to review cumulative data and they have.

Not suggested any changes to the protocols.

So thats ongoing and we look forward to completing that work later this year.

William P. Sheridan: And then with regard to your question about HAE priorities, the PROFI program and the filing and the approval are absolutely the number one priority. You heard Megan talk about it; one of her main priorities and focus is, you know, launch and execution, the successful execution of the launch. So that takes resources, that takes focus, that is the priority. The acute program has been delayed a bit due to, you know, continued regulatory interactions and figuring through the CMC pieces to get to our phase three. And as you said, we plan to get that started sometime in 2020.

And then with regard to your question about how the priorities.

Prophy program and the filing and the approval is absolutely the number one priority heard Megan talk about her.

One of her main priorities and focus is launch and execution successful execution of the launch so that takes the resources that takes the focus that is the priority. The acute program has been delayed a bit due to continued.

Regulatory interactions and figuring through the CMC pieces to get to our phase three and as you said, we plan to get that started sometime in 2020.

Thanks.

Operator: Thank you, and our next question comes from the line of Maury Raycroft with Jeffries. Your line is open.

Thank you and our next question comes from the line of Maury Raycroft with Jefferies. Your line is open.

Hi, This is 12 million for Murray.

Maurice Thomas Raycroft: Hi, this is Swapnil on behalf of Morrie. Thank you for taking our questions. So just one for 7353. Do you anticipate any unique risks with the approval process, especially due to the availability of such strong potent prophylactic therapies out there? And then I have a follow up.

Thank you for taking my questions. So just one for 75 piece or do you like anticipate any.

Unique risks with ruined process, especially due to.

So strong.

Prophylactic therapy is on that and then I have a follow up.

William P. Sheridan: I didn't completely catch your question. I think it was a market competition question, but could you just restate it?

I Didnt completely catch your question I think it was a.

Market competition question, but could you just could you restate it yes. So it's.

Maurice Thomas Raycroft: Yeah, so do you think there are any unique risks with the approval process for this 7353, especially given the availability of potent prophylactic therapies?

And do you like thing that unlike any unique risks with the approval process for the bus.

75 for the.

Especially given.

Visibility of Portland Prophylactic puppies.

William P. Sheridan: None. So, no, we don't anticipate any unique regulatory assessments with regard to competition. It's all about this drug in front of the regulators and its safety and efficacy profile and the accumulated database that we have in Phase I, Phase II, and Phase III studies. So, no, I don't think competition will play into that.

None so no we don't anticipate any unique regulatory.

Assessments for it with regard to competition. It's all about this drug in front of the regulators and its safety and efficacy profile.

And the accumulated data base that we have in phase one phase two and phase three studies are.

No I don't think competition will play into that at all Yeah I'll point you to.

William P. Sheridan: Yeah, and I'll point you to, I can't remember if it was 2018 or 17, 2018 I guess, the FDA had their patient meeting with the HAE Patient Association, and it was crystal clear from the audience response when they were asked the question, "what's your number, or what's your top priority with regard to new therapies?" And convenience was number one. So they've heard loud and clear that patients want more convenient therapies.

I can't remember if it was 2018 or 17.

2018, I guess, the FDA had their patient meeting with the AG patient Association and it was crystal clear from the audience response when they were asked a question what's your number or whats your top priorities with regard to new therapies and convenience was was number one so they've heard loud and clear that patients want more convenient therapies.

Okay. Thank you and I have one follow up question on other programs so for.

Maurice Thomas Raycroft: Okay, thank you. And I have one follow-up question on other programs. So for the FOP molecule, which is especially targeting ALK2, do you see any off-target preclinical signals, especially because of closely related targets such as TGF-beta or active in type 1 receptors?

FOP molecule, which is especially positive thing too.

Do you see any like off target preclinical signals, especially because of closing the analytic targets such as PGM.

Active in pipeline to support us.

William P. Sheridan: Thanks for the question. So the FOP program is on track to enter the clinic in the coming months this year. As you may recall, just to manage regulatory and clinical resources, earlier in the year we made the decision to prioritize 9930, so that's going great. FOP is on track.

Thanks for the question. So this program is on track to enter the clinic.

In coming months this year.

As you may recall, just to manage regulatory and clinical resources earlier in the year. We made the decision to prioritize 99 cities. So thats going great. If it stays on track.

William P. Sheridan: All kinase inhibitors are multi-kinase inhibitors. As you're familiar with the field, it's been very interesting to see how that's evolved, but there's no such thing as a mono kinase inhibitor. So at some dose level, all kinase inhibitors will have off-target effects. But we're very comfortable with the non-clinical profile of the drug supporting entry into the clinic, and I don't anticipate that that'll be an issue. Obviously, all of our Phase I studies are done very carefully with single-assenting dose and multiple-assenting dose components, and the Phase I trial for this particular drug will be conducted in healthy subjects.

All kinase inhibitors and multi kinase inhibitors as your revenue with the field, it's been very interesting to see how thats evolved there's no such thing as a mono kinase inhibitor.

So at some dose level kinase inhibitors will have off target effects.

We are very comfortable with the nonclinical profile of the drug supporting.

Entry into the clinic and iden anticipate that that will be an issue.

Obviously all of that phase one studies have done very carefully with single ascending dose and multiple ascending dose components.

The phase one trial for this particular drug.

We conducted in healthy subjects.

Okay. Thank you. Thank you for taking my questions you are welcome.

Maurice Thomas Raycroft: Okay. Thank you. Thank you for taking my questions. You're welcome.

Thank you.

Brian Corey Abrahams: Thank you. And our next question comes from Brian Abrahams with RBC Capital Markets. Your line is open. Hi, this is Bert on behalf of Brian. Thanks for taking our question. Just wanted to get kind of an update on your current thoughts on the 7353 pricing, in terms of what you may be able to charge compared to the competitors in the space to capture share and or to be tried in earlier lines.

Question comes from Brian Abrahams with RBC capital markets. Your line is open.

Hi, this is burden on for Brian Thanks for taking our question.

Just wanted to get kind of an update on your current thoughts on the 70 353 pricing in terms of what you may be able to charge compared to the competitors in the space to capture share and or to be tried in earlier lines.

Yes, so we're still conducting the payer research and doing the pricing analysis.

Jon P. Stonehouse: Yeah, so we're still conducting payer research and doing the pricing analysis. You know, the key, and I've said this before, is to be able to price it at a point where you knock down the barriers, but you don't leave a lot of money on the table. And we haven't gotten the data to make those decisions at this point in time, but what I will tell you is that with our competitors having drugs costing upwards of $600,000 per patient per year, we have a lot of flexibility and a lot of room, especially with a small molecule cost of goods. So, you know, we'll know more about that later. But the key here, and this is really important, is with some of the early market research that we're getting showing that the majority of patients want to use an oral medicine with our profile, and the feedback we got from hundreds of patients at the HAE patient summit, there is a significant opportunity with this drug.

The key and I've said this before is to be able to price. It at a point, where you knock down the barriers, but you don't leave a lot of money on the table and we havent.

Gotten the data to make those decisions at this point in time, but what I will tell you is with our competitors, having drugs of upwards of $600000 per patient per year, we have a lot of flexibility and a lot of room.

Especially with the small molecule cost of goods so.

Well, we'll know more about that later, but the key here and I think this is really important is with some of the early market research that we are getting showing that the majority of patients want to use an oral medicine with our profile. The feedback we got from hundreds of patients at the patient summit. There is a significant opportunity with this drug.

Jon P. Stonehouse: Great, thank you. You're welcome.

Great. Thank you.

You're welcome.

Thank you.

Liisa Ann Bayko: Thank you, and our next question comes from Liisa Bayko with DAMP Securities. Your line is open. Hi, great, thanks.

Our next question comes from Liisa Bayko with JMP Securities. Your line is open.

Hi, great. Thanks for taking my question can you maybe eliminate us as to when we might see.

William P. Sheridan: Thanks for taking the question. Can you maybe illuminate us as to when we might see the presentation of the data and a little bit more on quality of life and those kind of metrics? And, I guess, how important is that latter point?

Presentation of the data and.

A little bit more on quality of life and those kind of metrics and I guess, how important is that latter point.

William P. Sheridan: Sure, Liisa, it's Bill. We're working hard on producing materials to present at scientific meetings and also publication planning for the main manuscript for APEX II. It's always a bit hard to predict when those things are coming out, but our objective is, of course, to support our launch with high-quality publications in a great journal, and you know the data is terrific, so we feel pretty confident that we can do that. We've got complete engagement from the principal investigator and the other key investigators on the study to help us get those publications out. The upcoming meetings are the college allergy meeting later this year and Quad AI early next year in Antioch, and we'll be trying to have materials at all of those meetings.

Sure Hi, Lisa it's bill it we're working hard on producing materials to present at scientific meetings and also publication planning for the main manuscripts for apex too.

It's always a bit hard to predict when those things are coming out but our objective is of course to support our launch with high quality publication in a great channel and.

I think the data is terrific. So we feel pretty confident that we can do that.

Complete engagement from the principal investigator and the other key investigators on the study to help us get those publications at the upcoming meetings at the College LNG meeting later this year.

And quite early next year, and any Yankee and we'll be trying to have materials at all of those meetings.

William P. Sheridan: On the last point, you know, with regard to, you know, other elements, you know, I think that one of the interesting sets of conversations or a theme of conversation at the patient summit was also over the long term what happens and improvement in patient's well-being after months of therapy, and that's probably true for all of these drugs, so I think that story is still evolving, and it'll be interesting to see what type of quality of life data we get out of our long-term safety study as well with Apex2.

On the lottery point with regard to other elements.

I think that.

One of the interesting.

Sets of conversations or theme of conversation at the patient summit was also over the long term, what happens and improvement in patient well being.

After months of therapy, and that's probably true for all of these drugs. So I think Thats story is still evolving and it will be interesting to see what type of quality of life data, we get out of long term safety study as well as like X or.

Okay, great. Thanks, and then.

Liisa Ann Bayko: Okay, great. Thanks.

William P. Sheridan: And then for the Factor D program, can you maybe comment on recent data from the competitive landscape? I know there was some data released recently from Achilleon, and then how you anticipate your content might compare and contrast. What are the key differences, even from a kind of chemistry perspective, just to help us understand where you see the niche after what you've seen, what we've seen so far with Achillean's program? Sure.

After D. program can you maybe comment on recent data from.

From the competitive landscape I know there were some data release.

Chile on and then.

How how your comp.

How you anticipate your content might compare contrast, what are the key differences even from a.

Kind of chemistry perspective, just help us understand where do you see kind of the.

The net charge than what you've seen and what we've seen so far with that Julien Shirley.

William P. Sheridan: So, you know, a lot of respect, of course, for all of our competition. The data presented, I think, is encouraging for the field and indicates that, you know, they're making some progress with their programs. But I think it's very difficult at this stage to give you a direct answer in terms of head-to-head comparisons.

Lot of respective cost for all of that competition.

The data presented I think is encouraging for the field and indicates that they are making some progress on the programs I think it's very difficult at this stage to give you a direct answer in terms of head to head comparisons we will have more information that everybody will be able to assist once we have.

William P. Sheridan: We'll have more information that everybody will be able to assess once we have our safety PTAM-PD profile from Phase 1. In general, it's worth pointing out that the serine protease field and serine protease inhibitors are difficult targets. That's why there are only two companies that we know of, most likely, you know, actually entering the space of oral factor D inhibitors. We're really thrilled with our preclinical data, and we're closing the gap on our competition. So, given the market size, I'm not worried that there are, you know, there's more than one player.

Safety, PK and PD profile from the phase one.

Just in general it's worth pointing out that the searing protease field, considering protease inhibitors are difficult targets.

That's why there are only two companies that we know of.

Most likely actually entering the space of oral factor Xa inhibitors.

We're really thrilled to have preclinical data and way to closing the gap on.

Competition start given the market size on not worried that there.

There's more than one player.

Okay, great. Thanks.

Liisa Ann Bayko: Okay, great. Thanks.

Thank you and our following question comes from Sergey Belanger with Needham Your line is open.

Serge D. Belanger: Thank you. And our next question comes from Serge Belanger with Needham & Co. Your line is open.

Hi, good morning.

Serge D. Belanger: Hi, good morning. First, a question on the prophylactic program. I think the last time you talked to us back in May, you talked about a potential marketing strategy that would emphasize sampling in a lower-priced product. So after the patient summit and some of these other HAE meetings and the feedback you've had with patients and physicians, is that still the marketing strategy going forward, or has that evolved?

First a question on.

The prophylactic program I think the last time, you talk to us.

Back in May.

You're talking about a potential marketing strategy that would emphasize sampling and a lower priced product. So.

After the patients coming in and some of these other HCCI meetings and the feedback you've had with patients and physicians.

Is that still the marketing strategy going forward or is that involved.

Jon P. Stonehouse: Yeah.

Yes, I think well the strategy is the same that we want to position ourselves by making it easy for patients to get our drug and to move to the front of the line.

Jon P. Stonehouse: I think, well, the strategy is the same, that we want to position ourselves by making it easy for patients to get our drug and move to the front of the line. But I think what's different is that the opportunity here is much, much, much bigger. You know, price discounts. I'm not going to talk about that until we get the pricing research done, but we really see the demand for this drug to be huge. And so I think the opportunity is very big.

I think what's different is the opportunity here is much much much bigger.

Price discounts I'm not going to talk about that until we get the pricing research done, but we really see the demand for this drug to be huge and so I think the opportunity is very big.

Jon P. Stonehouse: How would you compare the European opportunity and how that market has evolved over the last few years with some of these new products?

Okay, and how would you compare against the European opportunity and how that market has.

Evolved over the last few years with some of these new products.

Jon P. Stonehouse: Yeah, so there really isn't a prophylaxis market, to be honest with you. I think that's the key difference.

So there really isn't a profile access market to be honest with you.

I think thats the key difference pricing pressure as we know in any therapeutic area is harder and heavier in Europe .

Jon P. Stonehouse: You know, pricing pressure, as we know, in any therapeutic area is harder and heavier in Europe. But our goal is to build a prophylactic market. And so, as I've mentioned before, the drugs that have been successful in this space have had 90% of their sales come from the U.S. and 10% from abroad. I see a real opportunity to build value outside the U.S. as well. So, you know, I think it's not that the U.S. won't bring us great value. We think there's significant value in the U.S., but we think there's significant value abroad as well. And the feedback, our experience with patients, and the KOLs in Europe, I mean, remember, we've done a lot of our trials in Europe, and we've gotten a lot of excitement. We were approached by people from Brazil, Europe, other parts of South America, Japan, and just a lot of enthusiasm from around the world to get access to our drugs.

But our goal is to build prophylactic market and so as I've mentioned before.

The drugs that have been successful in this space had 90% of their sales come from us and 10% from abroad.

I see a real opportunity to build value in outside the us as well. So I think it's not that the U.S. will bring us great value. We think there's significant value in the us, but we think theres significant value abroad, as well and the feedback our experience with patients and and the Kolašin in Europe I mean remember we've done a lot of our trials in Europe , and we've got a lot of excitement we were approached by people from Brazil, Europe . Other parts of South America, Japan, and just a lot of enthusiasm from around the world to get access to our drug.

Okay.

Serge D. Belanger: A couple of questions for the ACUTE HAE program. Any feedback, I guess, from your meetings at these recent meetings about the appetite for an oral product? And then can you also talk about how extensive the additional CMC formulation work that needs to be done before you undertake a Phase III trial?

A couple of questions for the acute HCV program.

Any feedback I guess from from your meetings that is right.

At these recent meetings about the the appetite for an oral product and then can you also talk about.

How extensive the additional CMC formulation more vendors.

That needs to be done before you undertake the phase three trial.

Jon P. Stonehouse: So, in terms of the feedback, I don't think I got a single question from a patient on ACUTE. It was all about prophylaxis, and you can understand that they'd much rather prevent attacks than treat them.

So in terms of the feedback I don't think I got a single question from a patient non acute it was all about prophylaxis and you can understand that that they'd much rather prevent attacks and treated tax so that answers. Your first question on your second question.

Jon P. Stonehouse: So, that answers your first question. On your second question, you know, we just want to have a formulation in Phase 3 that's better than what we had in Phase 2, and we're still working through that. We're, you know, confident we'll get there, but we're still working through it.

Yes, we just we want to have a formulation that's.

In phase three that's better than what we had in phase two and we're still working through that we're confident we'll get there, but we're still working through it.

Serge D. Belanger: Go. Alright, thanks for taking my questions.

Got it.

All right. Thanks for taking my questions.

Jon P. Stonehouse: You're welcome.

You're welcome.

Huidong Wang: Thank you. Once again, ladies and gentlemen, if you have a question at this time, please press the star and the number one key on your telephone keypad. Our next question comes from Gina Wang with Barclays. Your line is now open. We can't hear you.

Thank you once again, ladies and gentlemen, if you have a question at this time. Please press the star and the number one key on your telephone keypad. Our next question comes from Gena Wang with Barclays. Your line is now open.

Thanks.

We can't hear you.

Hi can you hear me yeah, better. Thank you Hi, Hi. This is this is Peter for Gina. Thanks for taking my question just two quick ones from US a one is for Bcr <unk> seven to 70 353 program do you expect to have any outcome meeting and mice and our second question is on the the CMC formulation.

Huidong Wang: Hi, can you hear me?

Operator: Yeah, it's better, thank you.

Peter Fujina: Hi, this is Peter Fujina. Thanks for taking our questions. Just two quick ones from us. One is, for the BCR-7353 program, do you expect to have any outcome meetings? And our second question is, on the CMC formulation for Zenith II, was that a request by FDA, or was that more initiated on your end? Thank you.

For those in it to was that a request by F.T.A. or was it more initiated on your end.

William P. Sheridan: Yeah, hi Peter, it's Bill. It's always hard to handicap whether or not you're going to have an adcom, but you always need to be prepared, so we'll be prepared.

Thank you.

Oh, Yes, hi, Peter it's Phil.

It's always hard to handicap, whether or not you guys have an AD com, but you always need to be prepared so we will be prepared.

William P. Sheridan: On the formulation front, it was ours, not FDA's. As I said before, we wanted a better formulation in Phase 3 than in Phase 2, and so it was ours.

On the formulation front there was ours not FDA, we just as I said before we want a better formulation phase threes in phase two and so it was ours.

Great. Thanks, Thank you very much.

Peter Fujina: Great, thank you very much. You're welcome. Thank you. And our next question comes from Tazeen Ahmad with BOFA Merrill Lynch. Your line is now open. Hi, good morning, guys. Thanks for taking my question. Um, Jon, just want me to follow.

You're welcome.

Thank you.

Following question comes from disease.

With Bofa Merrill Lynch. Your line is now open.

Hi, Good morning, guys. Thanks for taking my question John Just wanted me to follow up on your plans for commercial organizations. So you've you've got your application.

Tazeen Ahmad: I just wanted to follow up on your plans for commercial organizations. So, you know, you've got your application underway and almost ready to submit. How are you thinking about the size of the sales force that you might need? Obviously, you're a rare disease-focused company, so you won't need a large infrastructure. But have you started the interview process? Do you think that you might be open to hiring folks from competing companies? Can I also ask what your current cash runway is? Thanks.

Underway and almost ready to submit how are you thinking about.

The size of the sales force that you might need obviously youre a rare disease focused companies. So you will need a large infrastructure, but have you started the interview process. You think you might be opened the hiring.

Folks from competing companies and Tonight.

Also ask what your current cash runway us. Thanks.

Jon P. Stonehouse: Sure. So, um, with regard to... You know, the commercial infrastructure, you know, we're hiring, and we're filling jobs as we speak. So, in fact, we're, and I think it's posted, we've got a head of sales that we're currently recruiting. In terms of the size of the sales force, you rightly pointed out that these are really small sales forces in rare diseases. We haven't nailed down exactly what that is until we complete the market research and have a better idea of what the segments are, and what the physician population is. We have a pretty good idea now, but we haven't completed it, and we'll share more of that in the fall.

Sure so.

With regard to.

The commercial infrastructure, we're hiring and we're filling jobs as we speak so in fact were.

And I think it's posted we got ahead of sales that were currently recruiting in terms of the size of the sales force you rightly pointed out that these are really small sales forces in rare disease, we haven't nailed down exactly what that is until we complete the market research and have a better.

Do you have what the segments are what the physician population is we've got a pretty good idea now, but we haven't completed it and we'll share more of that in the fall.

And then on cash runway.

Jon P. Stonehouse: and then on Cash OnWay.

Tom Staub: Hey Tazeen, it's Tom. Thanks for the question. So on cash runway, it continues to be into 2020. And like I said in my prepared remarks, we have a lot of financial flexibility. You know, obviously, our cash runway is largely dependent on the commercial outlay and some of the research that Jon mentioned, as well as our plans with the 9930 program. And so it will continue to be in 2020. And we'll refine the cash runway as we get more information.

Hey, dizziness, Tom Thanks for the question. So on cash runway continues to be into 2020 and like I said in my prepared remarks, we have a lot of financial flexibility.

Obviously, our cash runway is largely dependent on the commercial outlay in some of the research that John mentioned as well as our plans with the 99 30 program and so it continues to be in 2020, and we will refine the cash runway as we get more information.

Okay. Thanks, Tom and that includes your plans for commercial structure built that correct, yes. It does.

Tom Staub: Okay, thanks Tom. And that includes your plans for the commercial structure.

Okay. Thank you.

Thank you and the last question comes from Tyler Van Buren with Piper Jaffray. Your line is now open.

Tom Staub: ...

Unknown Executive: And the last question comes from Tyler Van Buren. Your line is now. Hey, good morning.

Hey, good morning.

William P. Sheridan: I had a question on 9930 in the initial data year end. Very interesting that you guys mentioned that it influences financing options given how early it is. Obviously, safety is important, but can you just, you spoke about PK PD, suppression of the pathway, and standardized assays. So could you give a little bit more specificity on what type of PK PD measurements you'll be looking at to measure suppression of the pathway and what magnitude of suppression would be promising as you guys think about moving into PNH patients?

The question on 99 30 in the initial data yearend that interesting.

You guys mentioned that.

Influences financing options given our early it is.

Obviously safety is important but can you just you spoke about PK PD suppression of the pathway and standardize assays. So could you give a little bit more specificity on what type of.

PK PD measurements will be looking at to measure suppression of the pathway and what magnitude of suppression.

Would be promising as you guys think about moving into PMIC teenage patients.

William P. Sheridan: Sure. So, I would frame it up as follows, and a lot of this you can get from the literature on complement and the development of other products and investigational drugs in the field of complement inhibitors. So, there are some commercial standardized assays that interrogate the alternative pathway, which is, you know, factor D is the critical enzyme in the alternative pathway, and one of those is the Weisblad AP assay. So, that's the first thing, and the reason that it's important is that you can buy the kit and follow the instructions. So if everybody buys the kit and follows the instructions, that makes the data a bit more interpretable, one program to the next, to the next. The remaining assays are more bespoke, but they're well-published, and there is a whole range of them, including AP hemolysis assays, factor BB assays, and the like, and a lot of that is in the literature.

Sure sorry.

I would frame it up as follows and a lot of this you can get from the literature on.

On complement and the development of other products and investigational drugs in the field of complement inhibitors sorry.

There is some commercial standardize essays that interrogate the alternative pathway, which is effectively is the critical enzyme in the alternative pathway and one of those is to watch, but they pay US site. So thats. The first thing and the reason that's important is because you can buy the kit and follow the instructions.

So everybody buys the kit and follow the instructions that makes the data a bit more interpretable one one program to the next to the next are the remaining essays.

More.

The spark.

But they are well published and there are a whole range of them, including IP Mosys essays effected the assays and the like and a lot of that is in the literature. So we have a comprehensive set in healthy subjects that you can measure.

William P. Sheridan: So we have a comprehensive set in healthy subjects that you can measure. You know, you might ask, how can you measure these things in healthy people that don't have a disease? The reason is the alternative pathway is always switched on. It's always ticking over. So you can measure suppression of that in healthy people. So that's, you know, it's a very comprehensive set.

You might ask how can you measure these things in healthy people I don't have a disease reason is the alternative pathways always switch donuts always ticking over so you can measure suppression of that in healthy people.

So thats.

That's very comprehensive said.

William P. Sheridan: And what we're really looking for here is very strong suppression of the alternative pathway. If we don't get very strong suppression with our drug, that would be a big surprise on the basis of the data we've shown with oral dosing in the monkey that's in our public slide decks. So we're looking forward to seeing very strong suppression of the alternative pathway, and that'll be the, you know, that'll be a key element in interpreting phase one.

And what we're really looking for here is very strong suppression of the alternative pathway.

If we don't get very strong suppression with that drug that would be a big surprise on the basis of.

That would be a big surprise on the basis of the data we've shown.

With oral dosing in the monkey.

Public slide decks. So we're looking forward to seeing very strong suppression of the alternative pathway and that'll be the.

That will be a kit.

A key element in interpreting the phase one.

William P. Sheridan: Great, thanks so much. You're welcome. Thank you. And I'm showing no further questions at this time. I would now like to turn the call back to Jon Stonehouse for closing remarks.

Great. Thanks, so much.

You're welcome.

Thank you and I'm showing no further questions at this time I would now like to turn the call back to Jon Stonehouse for closing remarks.

Jon P. Stonehouse: Yeah, thank you. As I said in my prepared remarks, I don't think we've been in a stronger position at BioCryst. We're about to file and launch a drug that, having been at the HAE Patient Summit, patients really want. The demand is extremely high, and that leads us to conclude that the opportunity here is significant. And the great news is we'll be filing at the end of this year and launch next year. On top of that, we have an oral Factor D inhibitor with 9930. That's a pipeline in a molecule.

Yes. Thank you so as I said in the prepared remarks.

I don't think we've been in a stronger position at Biocryst, we're about to file and launch.

Drug that I can tell you having been at the patient summit patients really want the demand is extremely high and that leads us to conclude that the opportunity here is significant.

And the Great news is filing the end of this year and launch next year on top of that we've got.

Oral factor de inhibitor with 99 30, that's a pipeline in a molecule as I said, the current market double that of AG and it could double from there with additional indications and so advancing that and Betsy that quickly. So that we get another great oral drug out to patients many patients with different rare diseases is another great opportunity.

Jon P. Stonehouse: As I said, the current market doubles that of HAE, and it could double from there with additional indications. And so advancing that and advancing that quickly so that we get another great oral drug out to patients, many patients with different rare diseases, is another great opportunity. And we look forward to starting to update you on all the progress that we make this fall. So, thanks again for your interest in BioCryst, and have a great day.

And we look forward to starting to update you on all the progress that we make this fall. So thanks again for your interest in Biocryst and have great day.

Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program and you may all disconnect everyone have a great day.

Operator: Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program, and you may all disconnect. Everyone have a great day. Thanks for watching!