Q1 2020 Earnings Call

Hello, and welcome to the Biocryst first quarter 2020 earnings call.

Operator: Welcome to the BioCryst First Quarter 2020 Earnings Call. At this time, all participant lines are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. If anyone should require assistance during the conference, please press star, then zero on your touchtone telephone. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. John Bluth, at BioCryst.

At this time all the participant lines are in a listen only mode.

Later, we'll conduct a question answer session and instructions will follow at that time.

If anyone should require assistance.

Please press Star then do <unk> Touchtone telephone.

As a reminder, this conference call this being recorded.

I wouldn't know what's the time a conference over to your host Mr., John Bloop at Biocryst.

John D. Bluth: Thanks, Whitney. Good morning and welcome to BioCryst's First Quarter 2020 Corporate Update and Financial Results Conference Call. Today's press release and slides are available on our website. Participating with me today are CEO Jon Stonehouse, CFO Anthony Doyle, Chief Medical Officer Dr. Bill Sheridan, Chief Business Officer Megan Snizinski, and Chief Commercial Officer Charlie Geyer.

[music] banks Whitney good morning, and welcome to Biocryst first quarter 2020, corporate update and financial results Conference call.

Today's press release, the slides are available on our website.

Participating with me today are CEO, Jon Stonehouse, CFO, Anthony Doyle, Chief Medical Officer, Dr., Bill Sheridan Chief Business Officer, Megan Zinski, Chief Commercial officer, Charlie Guy or 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.

John D. Bluth: 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.

Forward looking financial information as well 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.

Factors. Please refer to the company's documents filed with the Securities Exchange Commission, which can be accessed on our website I'd now like to turn the call over to Jon Stonehouse. Thank you John and thank you all for joining US. This morning, I hope, you're all safe and doing well.

Jon P. Stonehouse: Thank you, Jon, and thank you all for joining us this morning. I hope you're all safe and doing well.

Jon P. Stonehouse: We are in an extraordinary position at BioCryst. The company is set to receive three approvals within the next 12 months for Bayer-Charleston, and we expect our first global approval in Japan in the second half of the year.

We are in an extraordinary position at Biocryst. The company is set to receive three approvals within the next 12 months for bear Charleston.

We expect our first global approval in Japan, and the second half the year.

Jon P. Stonehouse: We have a December 3rd PDUFA date from the FDA, and in Europe, our MAA was validated in March, and we expect approval about that time next year. Right alongside these approvals, we have a pipeline and a molecule with our oral factor D inhibitor, BCX9930, for complement-mediated diseases, including PNH. We will share exciting data for 99-30 and P&H patients with you for the first time today. Let's start with H-A-E and the value we expect to create with Baird-Charlson.

We have a December 3rd PDUFA date from the F.D.A.

And in Europe, our EMEA was validated in March and we expect approval about that time next year.

Right alongside these approvals we have a pipeline in a molecule with our oral factor Xa inhibitor BTX 90, 930 for complement mediated diseases, including P. an age.

We will share exciting data for 90 930 in pediatric patients with you for the first time today.

Let's start with H E and the value, we expect to create with Baird Charleston.

Jon P. Stonehouse: Patients are experiencing significant benefits in our clinical trial. Both physicians and HAA patients are consistently stating strong demand for our oral medicine in our market research. Based on the clinical response and customer demand, we expect Baratrolstat will generate peak sales of north of $500 million.

Patients are experiencing significant benefit in our clinical trials.

Both physicians and 80 patients are consistently stating strong demand for our oral medicine in our market research.

Based on the clinical response and the customer demand, we expect air troughs that will generate peak sales of north of $500 million.

Jon P. Stonehouse: And we announced yesterday that we have a new composition of matter patent that will extend our patent protection by four years to 2039. Add to that market potential of $99.30 in an established market of more than $4 billion for the treatment of complement-mediated diseases, and we see a significant opportunity for even greater value creation. We have built a company focused on discovering, developing, and commercializing oral drugs for rare diseases. We continue to advance our oral rare disease pipeline, which also includes BCX9250 for FOP, and additional discovery programs for other rare diseases. Beyond that, we've always believed our legacy antiviral programs play an important role in public health. Galidesivir is a broad-spectrum antiviral being studied in a NIAID-funded trial in COVID-19 patients. The experience and relationships we have developed across the U.S. government over the past decade to support our antiviral programs continue to add value. We have contracts totaling $82 million in government funding for Galadezivir, and we've been able to move quickly with Galadezivir into COVID-19 patients. Patient dosing has begun in our clinical trial in Brazil, and we look forward to generating data to determine if galidesivir could help in this global health emergency. The disruption of the coronavirus pandemic has impacted everything.

And we announced yesterday that we have a new composition of matter Pat.

That will extend our patent protection.

For years to 2039.

Add to that market potential for 90 930 in an established market up more than $4 billion for treatment of complement mediated diseases, and we see a significant opportunity for even greater value creation.

[music], we've built the company focused on discovering developing and commercializing oral drugs for rare diseases.

We continue to advance our oral rare disease pipeline, which also includes PCX 90, 250 for FOP and additional discovery programs for other rare diseases.

Beyond that we've always believed our legacy antiviral programs played an important role in public health.

Gallaudet severe is a broad spectrum antiviral in a NIAD funded trial in cobot 19 patients.

The experience in relationships, we have developed across the U.S. government over the past decade to support our any viral programs continue to add value.

We have contracts totaling $82 million of government funding for Gallaudet severe and we've been able to move quickly with gallaudet severe into cobot 19 patients.

Patient dosing has begun in our clinical trial in Brazil, and we look forward to generating data to determine if gallaudet severe could help in this global health emergency.

The disruption of the Corona virus pandemic has impacted every company.

Jon P. Stonehouse: While the situation is fluid, for the most part, BioCryst continues to maintain its progress and timeline. We're fortunate that the clinical trials and data to support our regulatory submissions for barotraustat were already completed at the time of the pandemic. As a result, our regulatory reviews are well underway, and our approval timelines and launch preparation activities remain on track. Charlie and Megan have been building the global commercial and medical affairs teams and drug supply to support our upcoming launches, and they'll provide an update. Then Bill will review our new data with 9930, Anthony will provide a financial review, and I will wrap up by sharing our progress with Gallaudet. With that, I'll turn the call over to Charlie.

Well the situation is fluid for the most part Biocryst continues to maintain its progress and timelines.

We're fortunate that the clinical trials in data to support our regulatory submissions for bear trials that were already completed at the time of the pandemic.

As a result, our regulatory reviews are well underway and our approval timelines and launch preparation activities remain on track.

Charlie and make it had been building that global commercial and medical affairs teams and drug supply to support our upcoming launches and they'll provide an update.

Then Bill will review, our new data with 90 930, Anthony will provide a financial review and I will wrap up by sharing our progress with Galidesivir here.

With that I'll turn the call over to Charlie.

Charles K. Gayer: Thanks, Jon. We've been busy since the start of 2020. Our U.S. marketing and market access teams are complete and in action, and our regional sales leaders are preparing to hire representatives for individual sales territories in the third quarter. We've also added an experienced and efficient commercial team to execute our launch in key European markets. Our launch preparations are moving forward smoothly because of the work we completed last year, plus the focused efforts of our growing team. COVID-19 has not slowed us down. Megan will describe our readiness in more detail, but first, I'd like to review our market research and clinical data. HEE attacks can be unpredictable and devastating, which explains why most patients in the United States have moved to prophylaxis. Several new injectable products have been launched in recent years, so patients and their physicians have experience switching to find the treatment that is best for them. What many patients want now is to switch to oral prophylaxis to control their disease and reduce the burden of treatment. A big part of our strategy is to focus on that switch.

Thanks, John we've been busy since the start of 2020 are U.S. marketing and market access teams are complete and inaction and our rail or regional sales leaders are preparing to higher representatives for individual sales territories in the third quarter. We've also added an experienced an efficient commercial team to execute our launching key European.

Markets.

Our launch preparations are moving forward smoothly because of the work we completed last year plus the focused efforts of our growing team covert 19 has not slowed us down.

Megan will describe our readiness in more detail, but first I'd like to review our market research and clinical data.

He attacks can be unpredictable in devastating which explains why most patients in the United States have moved to profile axis.

Several new injectable products have launched in recent years, so patients and their physicians have experienced switching to find the treatment that is best for them.

What many patients want now is to switch to oral purple access to control their disease and reduce the burden of treatment.

A big part of our strategy is to focus on that switch.

Charles K. Gayer: Our market research and clinical data give us confidence in this strategy. We surveyed 100 patients and 175 HAE treating physicians and presented a profile based on top-line data from APEX II. 59% of patients said they were very willing to use Barotrostat, growing to 71% with a physician recommendation. Notably, 79 of these 100 patients were already using Taxiro, Higarda, or Synrise, and most of them were also very willing to use Veritrol. Even among those very satisfied with their current injectable, half are very willing to use our oral drug. So why is that? One reason may be that even with current injectables, most patients experience breakthrough attacks. Patients on Taxiro, for example, reported that they still average about half an attack per month. The broader reason is that patients want to reduce treatment burdens, such as storage, preparation, and injection.

Our market research and clinical data give us confidence and this strategy.

We surveyed 100 patients and 175 E G treating physicians and presented a profile based on topline data from apex too.

59% of patients said, they were very willing to use bear a trial stat growing to 71% where the physician recommendation.

Notably 79 of these 100 patients were already using text IRO. He guard out were Sunrise and most of them. We're also very willing to use bear a trial staff.

Even among those very satisfied with their incurred with their current injectable half are very willing to use our oral drug so why is that.

One reason, maybe that even with current injectables most patients experienced breakthrough attacks patients on tax I wrote for example reported they still average about half an attack per month.

The broader reason is patients want to reduce treatment burden such as storage preparation that injection.

Charles K. Gayer: Physicians understand the benefits of oral prophylaxis and expect to treat 41% of current patients with our oral drug in the future. Our clinical data also show that many patients on injectable prophylaxis are likely to switch. As you can see on slide 13, 44% of patients who enrolled in Apex-2 previously used C1 inhibitor prophylaxis. Since Apex S opened in the U.S. last year, about 50% of newly enrolled patients were previously treated with Taxiro, Hegarta, or Synrite. These numbers align with physician expectations reported in our research. Of the 41 percent share they anticipate for barotraustat, half comes from switching from current prophylaxis.

Physicians understand the benefits of oral profile axis and expect to treat 41% of current patients with our oral drug in the future.

Our clinical data also showed that many patients on injectable purple access are likely to switch as you can see on slide 13, 44% of patients who enrolled in apex. Two previously you see one inhibitor profile access.

Since apex S opened in the U.S. last year about 50% of newly enrolled patients were previously treating with tech side, Okay, Garda or sunrise.

These numbers aligned with physician expectations reported in our research.

The 41% share they anticipate per barrel trial staff has come from switches from current prophylaxis.

Charles K. Gayer: Most patients in our trials are staying on Barotrauma Step because they really experience a benefit. For example, patients on 150 mg for a year in Apex-2 had a baseline average of 3 attacks per month but averaged just 1 attack per month on treatment. Those switching from placebo to 150 milligrams after 24 weeks average only about half an attack per month. Patients taking 150mg in Apex-S have similar long-term results, and in 6 out of the 12 months, half or more are attack free. The experience from both these trials shows the drug is safe and generally well-tolerated. The main adverse events are gastrointestinal symptoms, but most of these are mild, self-limited, and resolve within the first two months of treatment. We recently interviewed 20 U.S. patients who have been enrolled in APEX II for over a year. The quotes on slide 14 represent, in their own words, how Baratrostat is helping them. You can see what a dramatic impact oral once-daily barotraustat is having on their lives, and we are excited to be so close to bringing our drug to HAE patients. Now I'll turn it over to Megan to describe other important areas of our global launch readiness.

Most patients in our trials are staying on Barrick trolls step because they really experience a benefit patients on 150 milligrams for a year in apex too had a baseline average of three attacks per month, but average just one attack per month on treatment.

Those switching from placebo to 150 milligrams. After 24 weeks average only about half an attack or months.

Patients, taking 150 milligrams and apex s. have similar long term results and six out of the 12 months half or more or a tax free.

The experience from both these trials shows that shows the drug is safe and generally well tolerated the main adverse events or gastrointestinal symptoms, but most of these are mild self limited and resolved within the first two months of treatment.

We recently interviewed 20 U.S. patients in the United States, United States, who had been enrolled in apex two for over a year.

The quotes on slide 14 represent in their own words, how bear interest at is helping them you can see what a dramatic impact oral once daily barrel trolls stat is having on their lives and we're excited to be so close to bringing our drug ha patients.

Now I'll turn it over to Meg into described other other important areas of our global launch readiness.

Thanks, Charlie and good morning, we're excited to be preparing for the launch of bird Charles that.

Megan Snizinski: Thanks, Charlie, and good morning. We're excited to be preparing for the launch of Veritrolstat. As Jon shared earlier, we have regulatory reviews ongoing by the three major agencies with approval timelines on track as planned. Building on what Charlie highlighted, I'd like to touch on a few additional aspects of our launch readiness. First, I want to touch on our KOL engagement, which is a fundamental part of our pre-launch activities.

As John shared earlier, we had regulatory reviews ongoing by the three major agencies with approval timelines on track as planned.

Building on what Charlie highlighted I'd like to touch on a few additional aspects of our launch readiness.

First turning to our cable engagement, which is a fundamental part of our prelaunch activities.

Megan Snizinski: Across the U.S. and the E.U., we continue to interact with the H.A.E. medical community. These are important opportunities for scientific exchange and education on the clinical evidence supporting barotrauma statistics.

Across the U.S. and meet you we continued to interact with the 80 medical community.

These are important opportunities for scientific exchange in education on the clinical evidence supporting bear Charles that.

Recently as you may have seen the quite a high annual meeting one of the major annual Congress has for the AG physician community was cancelled in March due to cobot.

Megan Snizinski: Recently, as you may have seen, the Quad AI Annual Meeting, one of the major annual congresses for the HAE physician community, was canceled in March due to COVID. The Congress shifted to hosting a virtual poster hall, and in response, our medical affairs team conducted a series of virtual sessions, which were a great opportunity to present and share the data from our accepted posters with the scientific community. In Europe, we also continue to interact with KOLs across the region, as well as various patient organizations. Overall, HAE clinicians continue to be accessible via virtual calls in light of COVID, and we're really pleased by the continued strong interest in learning more about our Oral Once Daily treatment and its clinical program. From this work, we continue to see how barotraustat will meet what is still a significant unmet need in the area of prophylactic treatment for HAE patients.

Congress shifted so hosting a virtual poster hall and in response, our medical Affairs team conducted a series of virtual sessions, which were a great opportunity to present and share the data from our accepted posters, but the scientific community.

In Europe, we also continue to interact with K wells across the region as well as various patient organizations.

Overall AG conditions continue to be accessible via virtual called in light of cobot.

We're really pleased by the continued strong interest in learning more about our oral once daily treatment and its clinical program.

From this work we continue to see how Bertram that will meet what is still a significant unmet need in the area of prophylactic treatment for HIV patients.

Now moving to our supply readiness.

Megan Snizinski: Next, moving to our supply readiness. Having seen supply shortages for other H.A. treatments in the past, BioCryst committed early on to ensuring that would not happen for Baritolstat. We have dual source redundancy across the supply chain, with two manufacturing sites for each state.

Having seen supply shortages for other Hh treatments in the past Biocryst committed early on to ensuring that would not happen for beverage off that.

We have dual source redundancy across the supply chain, what's your manufacturing sites for each stage.

Megan Snizinski: We are working with well-established CMO partners and are well-positioned in terms of supply today because of our early investment. We already have more than ample product manufactured for final packaging, and at this time, I'm happy to share that we don't foresee any COVID impact on supply for a commercial lawn. Lastly, as Charlie mentioned, we've been fortunate to continue our APEX II and APEX S clinical studies despite the current pandemic. Site Monitoring Transitions from On-Site Visits to Virtual Consultations, and while many companies have stopped clinical trial operations, APEX-S screening continues, and we even enrolled several patients in the U.S. last month alone. We think this continues to speak to the unmet need and demand for our oral once daily prophy treatment options.

We are working with well established CMO partners and are well positioned in terms of supply today because of our early investment.

We already have more than ample product manufactured for final packaging and at this time I'm happy to share that we don't foresee any cobot impact to supply for our commercial launch.

Lastly, as Charlie mentioned, we've been fortunate to continue our apex to an excess clinical studies. Despite the current pandemic site monitoring transition from on site visits to virtual consultations and while many companies have stopped clinical trial operations apex S. screening continues and we even enroll.

Several patients in the U.S. last month alone.

We think this continues to speak to the unmet need and demand for our oral once daily Prophy treatment option.

Our teams remain focused on preparing for successful launches in the U.S. Yoo and through our partner Tory in Japan.

Megan Snizinski: Our teams remain focused on preparing for successful launches in the U.S., the EU, and through our partner, TORI, in Japan. With the potential to receive our first global approval in Japan, Tory launch preparations are actively underway, including building disease awareness and education. And as a reminder, with the PMDA approval, we stand to receive a $20 million milestone payment contingent upon clearing a minimum price threshold following our MHLW pricing discussion. It's clearly a transformative year for BioCryst as we look to what's ahead of us in the next 12 months with our launch. In addition, we're also focused on advancing our 9930 program, and Bill will walk you through that now. Okay, Bill?

With the potential to receive our first global approval in Japan, Tory launch preparations are actively underway, including building disease awareness and education.

As a reminder, with the PMDA approval, we stands receive a $20 million milestone payment contingent upon clearing a minimum price threshold following our NHL w. pricing discussions.

It's clearly a transformative year for Biocryst as we look to whats ahead of us in the next 12 months with our launches.

In addition, we're also focused on advancing our nine 930 program and Bill will walk you through that now bill.

Thanks, very much Megan.

William P. Sheridan: Thanks very much Megan. We are very excited to share early data from the lowest dose cohort of treatment naive patients in our ongoing PNH proof-of-concept study with our oral factor D inhibitor BCX9930. You will see from the 50 and 100 mg twice a day data that we are well on our way to achieving monotherapy. We are seeing dose-related effects on control of hemolysis and clinical benefits, and in the Healthy Subjects Multiple Last Ending Dose Study, The Effect of 9930 on Alternative Pathway Activity was superior at 200mg and 400mg twice a day compared to the lower doses. We've seen no safety signals.

We're very excited to ship early data from the lowest dose cohort of treatment naive patients ongoing PDH proof of concept study with oral factor Xa inhibitor Bcfs 90 930.

You will see from the 50 and 100 milligrams twice a day data that we are well under way to a goal of achieving monotherapy.

We are seeing dose related to fix on controlling for mosys and clinical benefit.

And in the healthy subjects multiple ascending dose study the effect of 99 city on alternative.

Alternative pathway activity.

With superior a 200 milligrams and 400 milligrams twice a day compared to the lower doses, we've seen no safety signals.

William P. Sheridan: As we move next to the 200- and 400-milligram twice-a-day cohort in treatment-naive PNH patients, these data tell us we should see complete control of hemolysis. As a reminder, the design of the P&H study is shown on slide 20. Cohort 1 is testing 50 and 100 milligrams twice a day. Cohort 2 will test 200 and 400 milligrams twice a day. So where are we

As you move next to the 200 400 milligram twice a day cohort in treatment naive teenage patients. These data tell us we should see complete control of Melissa.

As a reminder, the design of the P. an IDE study is shown on slide 21 is testing 50, and 100 milligrams twice a day cohort two to 200 400 milligrams twice a day.

So where are we today so far we have enrolled treatment naive ph patients that means they have not had cfive inhibitor drugs.

William P. Sheridan: So far, we have enrolled treatment-naive PNH patients. That means they have not had C5 inhibitor drugs. 99.30 is administered orally, twice a day as monotherapy.

99 cities administered orally twice a day as monotherapy Threepi an edge patients have completed 14 days dosing at 50 milligrams twice a day followed by 14 dies dosing at 100 milligrams twice a day at the day 28 visit all three clinical benefit assist by the investigators from add drug. So all three continues.

William P. Sheridan: Three PNH patients completed 14 days of dosing at 50 mg twice a day, followed by 14 days of dosing at 100 mg twice a day. At the day 28 visit, all three had clinical benefit assessed by the investigators from our drug, so all three continued on 100 mg twice a day in the long-term extension. On slide 21, you can see that these patients were seriously ill with PNH.

On 100 milligrams twice a day in the long term extension.

On slide 21, you can see that these patients with seriously ill with P. at age one had previously had a terrible bank thrombosis from the disease. The second required red cell transfusion and the third at a plastic in any advantage.

William P. Sheridan: One had previously had cerebral vein thrombosis from the disease, the second required red cell transfusions, and the third had aplastic anemia at PNH. In PNH, patients show quite variable degrees of hemolysis and anemia. Before treatment, among our three patients, the LDH, or lactate dehydrogenase level, a sensitive marker of hemolysis, ranged from over 800 to over 2400 units per liter, or 3.7 to 11 times the upper limit of normal, and the degree of anemia was severe with a hemoglobin of 6.0 to 8.2 grams per deciliter. All three patients had elevated reticular site counts, reflecting the bone marrow working over time to try to get the hemoglobin level up.

In P. NIH patients showed quite variable degrees from analysis at anemia before treatment among our three patients. The LDH all electric dehydrogenation level sensitive knockers from analysis ranged from 800 to over 2400 units per liter were 3.7 to 11 times you up limited normal.

A degree of anemia associated with the hemoglobin of 6.0 to 8.2 grams per deciliter.

We'll treat patients had elevated particular site counts, reflecting the primary working overtime to try to get the hemoglobin up.

We're very encouraged but look brushing clinical responses that we are seeing with that lowest doses of 90, 930, 50 milligrams twice a day and 100 milligrams twice a day the key Biomarkers interim analysis. All improved you can see the individual tighter on slide 22 Wolf tree at clinically meaningful in dose dependent drops LDH.

William P. Sheridan: We are very encouraged by the laboratory and clinical responses that we are seeing with our lowest doses of 9930. 50 mg twice a day and 100 mg twice a day, the key biomarkers of hemolysis all improved. You can see the individual data on slide 22.

The magnitude of effect is impressive given that these doses are lower and not optimized.

Ticket was site counts fill in all three patients.

Total blue ribbon another marker if I'm all assessing PIH was elevated into patients at baseline and normalized on 90 930.

[noise] previous studies of complement inhibitors to shut it takes about eight weeks to see stabilization in hemoglobin with optimized doses hemoglobin is already increasing at a four week study window at our lowest dose is subject to for example in at the study dependent on transfusions with a day one hemoglobin a 7.0.

William P. Sheridan: All three had clinically meaningful and dose-dependent drops in LDH. The magnitude of the effect is impressive given that these doses are low and not optimized. Particular side counts fell in all three patients. Total bilirubin, another marker of hemolysis in PNH, was elevated in two patients at baseline and normalized at 99.30. Previous studies of complement inhibitors have shown it takes about 8 weeks to see stabilization in hemoglobin with optimized doses. However, hemoglobin is already increasing in our four-week study window at our lowest doses. Subject 2, for example, entered the study dependent on transfusions, with a day 1 hemoglobin of 7.0. Following a two-unit red cell transfusion on day 15, this patient has now been transfusion-free for six weeks, and the hemoglobin has risen from 8.9 post-transfusion to 11.1 at week 8 of the study, while on 99.30 at 100 milligrams twice a day.

Following achieving at Red cell transfusion on day 15. This patient has never been transfusion freight six weeks and the hemoglobin has risen from 8.9 post transfusion to 11.1 at week eight of study while on 90 930 at 100 milligrams twice a day.

The safety and Tolerability profile at 90 930 during the 28 day evaluation period is shown on slide 23.

Unlike our earliest days when experience in healthy volunteers no patients develop to drug rash no drug related serious adverse events.

The most common observation was transient headache early in dosing, which is a well recognize class effect of complement inhibitor treatment in PIH.

One unrelated serious adverse events occurred in the extension period disseminated fair a seller infection that led to a patient did this patient whose PDH was treated with chronic corticosteroids and is a timeframe was a frontline healthkit worker, who is exposed to and subsequently contracted farah so.

William P. Sheridan: The safety and tolerability profile of 9930 during the 28-day evaluation period is shown on slide 23. Unlike our earlier Phase 1 experience in healthy volunteers, no patients developed a drug rash. There were no drug-related serious adverse events. The most common observation was transient headache early in dosing, which is a well-recognized class effect of complement inhibitor treatment in PNH. One unrelated serious adverse event occurred in the extension period, a disseminated varicella infection that led to a patient death. This patient, whose PNH was treated with chronic corticosteroids and azathioprine, was a frontline healthcare worker who was exposed to and subsequently contracted varicella. Based on this clinical history, the investigator determined the event was unrelated to 9930.

There are still or is known to be especially dangerous in patients taking steroids and other drugs that suppress lymphocytes.

Based on this clinical history investigated determined the event was unrelated to 90 930.

At fourth treatment naive patients in cohort one was recently enrolled in South Africa.

Following completion of cohort one we expect to begin enrollment of Cfive inhibitor nave patients in cohort two testing 204 hundred milligrams twice a day.

And despite the current challenges we continue to receive strong interest from investigators in patient advocates to enroll ph patients who have poor responders to see five inhibitors.

We expect to begin enrolling poor responding patients in the third quarter and report data from these patients by the end of the it.

We're very excited about this early data at 50, and 100 milligrams twice a day in pediatric patients.

Also we have completed the med cohorts for 200 400 milligrams twice a day in healthy subjects.

William P. Sheridan: Our fourth treatment-naive patient in cohort one was recently enrolled in South Africa. Following completion of Cohort 1, we expect to begin enrolment of C5-inhibitor-naive patients in Cohort 2, testing 200 and 400 mg twice a day. And despite the COVID challenges, we continue to receive strong interest from investigators and patient advocates to enroll PNH patients who are poor responders to C5 inhibitors. We expect to begin enrolling responding patients in the third quarter and report data from these patients by the end of the year.

The Pharmacodynamic profile at these doses is clearly clearly superior to the PD profile of 5100 milligrams twice a day and there were no safety signals.

The steady state results individual healthy subjects in the med as shown on slide 26 for both the IP hemolysis and IP. This web essays.

Note that the essays were continued for 24 hours after the loss does.

Importantly for peanuts treatment, the higher doses provide more consistent coverage, especially in the period beyond 12 hours after the dose.

The main values as shown on slide 27.

200, 400 milligrams twice a day IP activity was blocked by more than 98% in both at sites throughout the dosing interval at steady state.

William P. Sheridan: We are very excited about this early data at 50 and 100 milligrams twice a day in PNH patients. Also, we have completed the MADD cohorts at 200 and 400 milligrams twice a day in healthy subjects. The pharmacodynamic profile of these doses is clearly superior to the PD profile of 50 and 100 milligrams twice a day, and there were no safety signals. The steady state results for individual healthy subjects in the MAD are shown on slide 26 for both the AP Haemolysis and AP Vooselab assays. Note that the assays were continued for 24 hours after the last dose. Importantly, for PNH treatment, higher doses provide more consistent coverage, especially in the period beyond 12 hours after the dose. The mean values are shown on slide 27.

When you see the level of complement suppression, we have a 200 400 milligrams you may ask if this could be the profile of a once a day drug it might be and we do plan to explore once daily dosing in the healthy subject Mad study as well as wrapping up the study by characterization of clinical pharmacology of 90 930 with additional cohorts testing.

Super therapeutic doses.

So what are we learned about those.

First there is a clear dose response at 15 100 milligrams twice a day in treatment nave ph patients with clinical benefit second PD results at the 200 400 milligram twice a day doses in healthy subjects with superior to the low doses.

Before we plan to begin the C correspond to cohort at that dose level 200 milligram 400 milligram.

William P. Sheridan: At about 200 and 400 milligrams twice a day, AP activity was blocked by more than 98% in both assays throughout the dosing interval at steady state. When you see the level of complement suppression we have at 200 and 400 milligrams, you may ask if this could be the profile of a once-a-day drug. It might be, and we do plan to explore once-daily dosing in the Healthy Subject MAD Study, as well as wrap up the study by characterization of clinical pharmacology of 9930 with additional cohorts testing super-therapeutic doses. So what have we learned about those?

And our goal is to develop BCS 99 city as an oral monotherapy for PNM and other complement mediated diseases.

The pediatric patient and med healthy subject data, we shared today strongly support that goal. We excited to complete a proof of concept study to speak with rigs, but it's a bit on next steps in p. NIH and other diseases caused by Dysregulation to complement.

Thanks, Bill as you can imagine we are very excited about these results and getting closer to our goal of having an oral factor de inhibitor that has great efficacy as monotherapy.

William P. Sheridan: First, there is a clear dose response at 50 and 100 milligrams twice a day in treatment nave PNH patients with clinical benefit. Second, PD results at the 200 and 400 milligrams twice a day doses in healthy subjects were superior to the lower doses. Therefore, we plan to begin the C5 core responder cohort at that dose level, i.e., 200mg and 400mg.

With the $115 million, we reported at the end of Q1.

We have sufficient capital to get us through this year and into the early part of next year. This capital funds completing our proof of concept study with 90 930 and fully investing in the launch preparation for bear Charleston.

We also plan. We also have a plan that gives us flexibility to bringing additional capital into the company and I'm very pleased to introduce our new CFO Anthony Doyle to describe that for you.

William P. Sheridan: Our goal is to develop BCX9930 as an oral monotherapy for PNH and other complement-mediated diseases. The PNH patient and MAD-healthy subject data we shared today strongly support that goal. We're excited to complete our proof of concept study and to speak with regulators about our next steps in PNH and other diseases caused by dysregulation of complement.

We conducted a comprehensive nationwide search with some exceptional candidates and Anthony stood out among them.

He spent the past six years as the CFO of a global Sea Arrow and spent the majority of his career prior to that rising through the ranks at GE.

Jon P. Stonehouse: Thanks Bill. As you can imagine, we are very excited about these results and getting closer to our goal of having an oral factor D inhibitor that has great efficacy as monotherapy with the $115 million we reported at the end of Q1. We have sufficient capital to get us through this year and into the early part of next year. This capital will fund completing our proof-of-concept study with 9930 and fully investing in the launch preparation for barotrauma. We also have a plan that gives us the flexibility to bring in additional capital into the company. And I'm very pleased to introduce our new CFO, Anthony Doyle, to describe that for you. We conducted a comprehensive nationwide search with some exceptional candidates, and Anthony stood out among them. He spent the past six years as the CFO of a global CRO and spent the majority of his career prior to that rising through the ranks at GE. With that introduction, I'll now turn the call over to Anthony.

That intro I'll now turn the call over to answer.

Thanks, John.

Certainly as an exciting opportunity for me in a great time to be joining biocryst with the upcoming commercial entre bear trough strong pipeline behind it including an oral factor xa inhibitor and opportunities to help in the Corona virus pandemic with Canada severe the company has tremendous runway for success in the near future.

You can find a financial results from the first quarter detailed in our press release, but I did want to highlight where we are with the balance sheet and our approach to capital in the upcoming months as John noted on the cash side. We ended Q1 was $115 million based on the outlook that we provided this gives us runway through 2020 and into early 2021.

We have several additional potential capital sources to provide financial flexibility as we progress through the year.

We expect to trigger up to $20 million milestone with from Tory also our data from BTX 90, 930 provides options to add capital such as a partnership to advance that program.

Additionally, we are evaluating royalty and or debt financing for better trial stuff that we're bringing capital at approval to fund the launch.

Anthony J. Doyle: Thanks, Jon. It certainly is an exciting opportunity for me and a great time to be joining BioCryst. With the upcoming commercial launch of Bertralstat, a strong pipeline behind it, including an oral factor de-inhibitor, and opportunities to help in the coronavirus pandemic with Galidesivir, the company has tremendous runway for success in the near future. You can find the financial results from the first quarter detailed in the press release, but I did want to highlight where we are with the balance sheet and our approach to capital in the upcoming... As Based on the outlook that we've provided, this gives us runway through 2020 and into early 2021. We have several additional potential capital sources to provide financial flexibility as we progress through the year.

Stepping into this role I'm very much looking forward to generating revenue starting early next year with a product that we believe we'll have peak sales now extended through 2013 with their new patents of greater than $500 million and a very dynamic pipeline behind us.

Thanks Anthony.

I also want to update you on our progress with Gallaudet severe our nuclear side are any polymerase inhibitor, which we are testing as a potential treatment for cobot 19.

In April we announced that we had opened a randomized double blind placebo controlled clinical trial of Gallaudet severe in cobot 19 patients in Brazil. This study is funded by not yet the trial. It started with patients currently enrolling into part one the dose ranging part of the trial. We look forward to updating you on what we see in part one and how that.

Data informs our dose selection and progress in our too.

The rationale for studying gallaudet severe in cobot 19 is that it's Dennis seen nucleus side analog Arnie polymerase inhibitor that demonstrated broad spectrum antiviral activity. We've conducted in vitro test against more than 20, R&D viruses in nine different families, including the Corona viruses.

Anthony J. Doyle: We expect to trigger up to the $20 million milestone from Tory. Also, our data from BCX9930 provides options to add capital, such as a partnership to advance that program. And additionally, we're evaluating royalty and or debt financing for Baratralstat that would bring in capital at approval to fund the launch. Stepping into this role, I'm very much looking forward to generating revenue starting early next year with a product that we believe will have peak sales now extended through 2013 with our new patent of greater than $500 million and a very dynamic pipeline behind it. Ciao.

That caused mers and Sars.

In vitro testing of Gallaudet severe against Sars koby to the virus that causes cobot 19 is also underway.

And we're working with our government partners and collaborators to identify potential animal models that could provide additional data against experimental Sars koby too.

At the end of the day clinical data from a randomized placebo controlled trial will provide the best information on the benefit the drug has for coated patients and we're looking forward to getting that data as quickly as possible.

So let me wrap up where I started.

Jon P. Stonehouse: Thanks, Anthony. I also want to update you on our progress with Galidesivir, our nucleoside RNA polymerase inhibitor, which we are testing as a potential treatment for COVID-19. In April, we announced that we had opened a randomized, double-blind, placebo-controlled clinical trial of Galidesivir in COVID-19 patients in Brazil. This study is funded by NIAID.

Biocryst is in an extraordinary position, we have three approvals coming within the next 12 months for bear Charleston, the strong clinical data end market demand from ha patients and physicians have led us to a forecast north of $500 million in peak sales for this product.

In addition, we have a pipeline in a molecule with 99 dirty and the early data we shared today adds to our confidence in the success of this program across multiple complement mediated diseases.

Jon P. Stonehouse: The trial has started with patients currently enrolling into Part 1, the dose-ranging part of the trial. We look forward to updating you on what we see in Part 1 and how that data informs our dose selection and progress into Part 2. The rationale for studying galidesivir in COVID-19 is that it's an adenosine nucleoside analog RNA polymerase inhibitor that's demonstrated broad-spectrum antiviral activity. We've conducted in vitro tests against more than 20 RNA viruses in nine different families, including the coronaviruses that cause MERS and SARS. In vitro testing of galidesvir against SARS-CoV-2, the virus that causes COVID-19, is also underway, and we're working with our government partners and collaborators to identify potential animal models that could provide additional data against the experimental SARS-CoV-2.

And our anti viral programs are positioned to help address a global health emergency and add additional value.

I want to close by thanking our team at Biocryst and all of our investigators patients and collaborators around the world who have made this progress possible. Despite the significant current disruptions and challenges in their own daily lives.

We wouldn't be where we are today without you. So thank you.

With that we'll turn it over to the operator for questions.

Ladies and gentlemen, if you have a questions at this time. Please first the stars and then be number one key on your Touchtone telephone. If your question has been answered.

So from the Q.

Keith.

Your first question is from the line of Jessica Fye with JP Morgan.

Jon P. Stonehouse: At the end of the day, clinical data from a randomized placebo-controlled trial will provide the best information on the benefit the drug has for COVID patients, and we're looking forward to getting that data as quickly as possible. So, let me wrap up where I started. BioCryst is in an extraordinary position. We have three approvals coming within the next 12 months for barotrauma. The strong clinical data and market demand from HAE patients and physicians have led us to a forecast north of $500 million in peak sales for this product. In addition, we have a pipeline and a molecule with 9930, and the yearly data we shared today adds to our confidence in the success of this program across multiple complement-mediated diseases, and our antiviral programs are positioned to help address a global health emergency and add additional value. I want to close by thanking our team at BioCryst and all of our investigators, patients, and collaborators around the world who have made this progress possible despite the significant current disruptions and challenges in their own daily lives. We wouldn't be where we are today without you, so thank you. With that, we'll turn it over to the operator for questions.

Hey, guys. Good morning, Thanks for taking my questions and a couple on the 90 930 data.

First why do you think ridiculous sites appeared a rebound after the initially Paul on treatment.

And second sounds like there were no rush the observed in the first three patients was or even any transient rash and I'm curious if you have a hypothesis for why that was not seen here one healthy volunteer data would have suggested you might.

Yes, Hi, Jessica it's bill Thanks for the question that particular sites.

Turning to remain.

Active at elevated wireless subjects are anemic, sorry, as we see the data mature in the subsequent weeks and see the hemoglobin come up you'd expected to come come and stay in to the normal range and with regards to rash.

And see any mile brasher and not rational in the first three subjects.

Okay.

That's an interesting question on the bare trails that program, we saw a similar phenomenon, where we had higher incidence of ration healthy subjects compared to people getting ha.

We'll see how doubles.

Okay, Great and case, just a couple on bill it does appear as well.

Jessica Macomber Fye: Ladies and gentlemen, if you have a question at this time, please press the star and then the number one key on your touchtone telephone. If your question has been answered or you wish to remove yourself from the queue, please press the pound key. Your first question is from the line of Jessica Fye with JPMorgan. Hey guys, good morning.

How many sites are opened in Brazil, and when should we anticipate that data and I think theres also been some reports on the web seeing gallaudet severe has shown activity in vitro against the current Corona virus press release makes it sound like you're still evaluating that.

Jessica Macomber Fye: Thanks for taking my questions. I had a couple on the 9930 data. First, why do you think reticulocytes appear to rebound after they initially fall on treatment? And second, it sounds like there were no rashes observed in the first three patients. Was there even any transient rash? And I'm curious if you have a hypothesis for why that was not seen here when the Healthy Volunteer data would have suggested you might.

Have you seen any early indications of activity.

Yes, so on the sites a off the top of my head I think theres, three but I'll have to confirm that get get back to you I think there's three sites in Brazil, and we're working on getting a force with regard to the Sars koby two in vitro testing.

Don't want to comment until that work is fully completed and a and it isn't and when it is we will report that data.

And is there any timeline for the clinical data.

William P. Sheridan: Hi Jessica, it's Bill. Thanks for the question. The reticular sites are going to remain active and elevated while the subjects are anemic. So as we see the data mature in the subsequent weeks and see the hemoglobin come up, you'd expect it to come and stay in the normal range. And with regard to the rash, no, we didn't see any mild rash or any rash at all in the first three subjects. Why? That's an interesting question. You know, in the Baratralstat program, we saw a similar phenomenon where we had a higher incidence of rash in healthy subjects compared to people getting HAE. And, you know, we'll see how it evolves.

That's a hard one to predict.

The.

Pandemic in Brazil is pretty widespread in pretty active right now and so were we were dosing patients were in part one, but it's really really hard to predict the more sites that we have opened in Brazil, the faster, we'll be able to enroll and we're doing as much as we can to move as quickly as.

Okay.

Great. Thank you.

Welcome.

Your next question is from Gino way with Barclays.

Thank you for taking my questions.

What do you follow the Rosh question why did come from this cohort data, which patient cohort, we do now you penicillin.

Jon P. Stonehouse: Okay, great. And can I just ask a couple of questions on galidesivir as well? How many sites are open in Brazil and when should we anticipate that data? And I think there's also been some reports on the web saying galidesivir has shown activity in vitro against the current coronavirus. The press release makes it sound like you're still evaluating that. Have you seen any early indications of activity?

That's right.

Prophylaxis against last year infections with vaccination and.

Looking at the whole body of evidence here with thrilled with the data that we have in the first three subjects. In this study not just the absence of rash.

In a serious disease like this evening patients did get a rash would treat through it.

Benefit here is an outstanding.

Jon P. Stonehouse: Yeah, so on the sites, off the top of my head, I think there's three, but I'll have to confirm that and get back to you. I think there are three sites in Brazil, and we're working on getting a fourth. With regard to the SARS-CoV-2 in vitro testing, I don't want to comment until that work is fully completed, and it isn't, and when it is, we will report that

Sorry.

There was not penicillin, Nigeria prophylaxis with vaccination.

Okay and arbitrage.

Yes.

Right.

All the proposed.

New cohorts.

She's asking yes.

I think that.

Completely relaxed about the co administration of penicillin or any other antibiotic, but this drug by the way sorry, that's it's not really an issue for us we figured out that there was a drug fresh in the healthy subjects, which was benign, but clinically and pathologically and we treated through a couple of cases.

Jon P. Stonehouse: And is there any timeline for the clinical data?

Jon P. Stonehouse: That's a hard one to predict. The pandemic in Brazil is pretty widespread and pretty active right now. And so, you know, we're, we're dosing patients. We're in part one. Um, but it's really, really hard to predict, you know, the more sites that we have open in Brazil, the faster we'll be able to enroll. And we're doing as much as we can to move as quickly as possible.

And there's no protocol requirement to use penicillin at all if people need antibiotics for whatever reason they can get them, but we'll use the vaccine. The vaccine is the apart next year, yes.

I think the leaving I'm asking just wanted to see how likely the ration.

Hello.

Dan.

Debbie.

Eliminate any.

Jon P. Stonehouse: Thank you. You're welcome. Your next question is from Gina Wang with Barclays.

Sorry.

Yes, so yes.

Yeah no.

Huidong Wang: Thank you for taking my questions. Just want to follow up on the Roche question, want to confirm for this cohort data, the PMH patient cohort, that you do not use penicillin.

We can't say that it's the penicillin difference between the healthy volunteers in the ph patients.

The fact of the matter is we've had three patients with PNM and we've seen no rash and as Bill mentioned in his comments. This is a phenomenon that we saw in ha with bear troughs that as well that we saw rash at a higher incidence in healthy volunteers and way lower incidence and ha patients will see as we go.

William P. Sheridan: That's right, the prophylaxis against Neisseria infections was vaccination, and, you know, looking at the whole body of evidence here, we're thrilled with the data that we have in the first three subjects in this study, not just the absence of rash. In a serious disease like this, even if patients did get a rash, we would treat through it. The benefit here is, you know, outstanding, so there was no penicillin at Neisseria prophylaxis with vaccination.

Okay and then another data question.

Slide 20.

Just wondering do you have one station on the left side.

Thank you Melissa Sachs you have one patients.

Huidong Wang: Okay, and for all the trials, you will not use penicillin, right, for all the proposed new cohorts?

That's the regarding 200 milligram to 400 milligram.

William P. Sheridan: She's only asking for a few.

Patients typically has increased.

William P. Sheridan: Yeah, I think that we're completely relaxed about the co-administration of penicillin or any other antibiotic with this drug. So that's it's not really an issue for us, you know we figured out that there was a drug rash in the healthy subjects which was benign clinically and pathologically, and we treated through a couple of cases, and you know there's no protocol requirement to use penicillin at all if people need antibiotics for whatever reason, they can get them. But we'll use the vaccine anyway. Yeah, the vaccine is the approach.

Yes.

Hemolysis.

During the rebound.

He is one all lines there.

And then.

Right side, when we using were flat.

You have solution location will solution with us.

All lines later, just wondering if you can give a little bit more color on PCB.

In addition, we're calling.

Baseline Amy.

Contribute to this.

Jon P. Stonehouse: I think the reason I'm asking is just wanted to see how likely the rash is due to penicillin and, you know, would that be any, you know, eliminate any unnecessary...

Paul.

Remodels.

Sure.

Sure.

So the question I really love this Chuck.

Jon P. Stonehouse: Yeah.

It shows a spectacular.

Unknown Speaker: Unknown Speaker.

Jon P. Stonehouse: Yeah, no, we can't say that it's the penicillin difference between the healthy volunteers and the PNH patients. The fact of the matter is, we've had three patients with PNH, and we've seen no rash. And as Bill mentioned in his comments, this is a phenomenon that we saw in HAE with Barrett-Tralsted as well, where we saw rash at a higher incidence in the healthy volunteers and a way lower incidence in HAE patients.

Data consistency, comparing 200 milligrams and 400 milligrams versus 50 milligrams and 100 milligrams and the reason that we've shown that data this way is because.

We've done the essays through 24 hour sauces lost those were the twice daily dosing regimen. So all the way through 16 hours. There is almost complete suppression of IP activity, whether or not you measuring it into these plant that say what are the most assess say of course as the drug disappears from the system.

Jon P. Stonehouse: We'll see as we go.

Huidong Wang: Okay, and then another data question on slide 26. I'm just wondering if you do have one patient on the left side when you're using AC hemolysis. You have one patient that's ranging from 200 milligrams to 400 milligrams. You have one patient that basically had an increase in hemolysis inhibition and rebound. There's one outlier there. And then on the right side, when we're using the Westlab assay, you have an additional patient who also showed up, outlining later. Just wondering if you can give a little bit more color on basically these two outliers, any additional color on the baseline or anything that could contribute to this rebound of hemolysis inhibition.

The next period eventually it will get to levels, where it's not surprising.

Complement enough and eventually you will get positive results in the saying, we're starting to see that in the on individual here and there.

This is a great job, but at the twice a day dialogue coverage out the 16 with everybody is fantastic. Yeah. This is a great job, yes. So I think thats. The reason I am asking is.

Likely that could be beachwalk.

Yes.

These patients.

William P. Sheridan: Sure. Thanks for the question.

Actually.

William P. Sheridan: I really love this chart. It shows a spectacular beta consistency comparing 200mg and 400mg versus 50mg and 100mg. And the reason that we've shown the data this way is because we've done the assay through 24 hours after the last dose on a twice daily dosing regimen. So all the way through 16 hours, there's almost complete suppression of AP activity, whether or not you're measuring it in the VFLAB assay or the hemolysis assay. Of course, as the drug disappears from the system over the next period, eventually, it will get to levels where it's not suppressing complement enough, and eventually, you'll get positive results in the assay, and we're starting to see that in the odd individual here and there.

Right.

Yes, any any differences in terms of baseline yes.

Color.

I think it's.

With this data, we don't know yet, but it's obviously encouraging us to study once daily dosing, which is what exactly were going to doing in the med and I will have to figure out what doses must be able to achieve that.

Okay. Thank you.

Your next question is from Tyler Van Buren with Piper Sandler.

Hey, guys. Good morning, Thanks for taking the questions. It's exciting to see the initial 90 930 pianists data I guess I just wanted to ask you guys too.

Comparisons potentially to the phase two dinner and data for the or other.

William P. Sheridan: This is a twice a day drug, so coverage is up to 16 hours.

Oral factor D.

William P. Sheridan: This is a great job.

It's early days of course, and small number of patients, but they seem to compare favorably are there any noticeable differences that you guys would point out and then the second question on the once daily dosing what do you see in the Mad study in order to have confidence that you can use that in patients in the corporate into clinical profile.

Huidong Wang: I think it's, you know, with this data, we don't know yet, but it's obviously encouraging us to study once-daily dosing, which is what we're going to do in the MAD. And we'll have to figure out, you know, what doses might be able to achieve that.

Unknown Executive: Okay, thank you. Your next question is from Tyler Van Buren with Piper Sandler.

Unknown Executive: Hey, guys. Good morning. Thanks for taking the questions.

Brian.

Okay. So.

First of all I'd say, we're incredibly happy with the data we have in the first three subjects you.

William P. Sheridan: It's exciting to see the initial 9930 PNH data. I guess I just wanted to ask you guys to, you know, make some comparisons, potentially, to the phase 2 Denexpan data for the other oral factor D. It's early days, of course, and a small number of patients, but they seem to compare favorably. Are there any noticeable differences that you guys would point out? And then, on the once-daily dosing, what do you see in the MAD study in order to have confidence that you can use that in patients and incorporate it into the clinical program?

The.

LDH particular sides, Billy Rubin will going the right direction really really strong drops from pre treatment and in a short period, the hemoglobin starting to rise and I Didnt mention this on the call but.

Ph client size in the two subjects, where it was fairly low has come up pretty dramatically even in the first two weeks on 50 milligrams twice a day, it's pending for satellite but in terms of comparisons with other studies.

I would direct people to look at the very early studies with other agents and add data is at least as good as anybody else's, especially when you're looking at people who are severely and they make a baseline. So this is a this is a tremendous result, hey, bill I would add on the comparisons the one of the challenges in comparisons is where to patients start as bill said in his.

Jon P. Stonehouse: Okay. So, first of all, I'd say we're incredibly happy with the data we have on the first three subjects here. The LDH reticulocytes, bilirubin, all going in the right direction and really, really big drops from pretreatment. And in a short period, hemoglobin is starting to rise. And I didn't mention this on the call, but the PNH clone size in the two subjects where it was fairly low has come up pretty dramatically, even in the first two weeks on 50 milligrams twice a day. That's pending for later. But in terms of comparisons with other studies, I would direct people to look at the very earliest studies with other agents, and our data is at least as good as anybody else's, especially when you're looking at people who are severely anemic at baseline. So this is a tremendous result.

Remarks, these were really sick people and so instead of looking at the absolute number I think a percentage change is important to compare and I think we did fantastic on that front.

And with regard to once a day dosing, it's the persistence of Pharmacodynamic effect through 24 hours in the great majority of people, who had 200 milligrams. Every 12 hours were 400 milligrams that reached 12 hours.

It is striking and gives us absolutely good.

From Koji reason to go in test once daily dosing and we'll do that and see what we get a NIM will be in a position to understand whether we want to include that in a teenage cohort.

Jon P. Stonehouse: Hey Bill, I would add to the comparisons. One of the challenges in comparisons is where do the patients start? As Bill said in his remarks, these were really sick people, and so, you know, instead of looking at the absolute number, you know, I think a percentage change is important to compare, and I think we did fantastic.

Great. Thank you.

Well.

Your next question is from Elisa.

MP Securities.

Hi, there thanks for taking my question.

Go through some certain comparing to call gallaudet severe versus.

William P. Sheridan: With regard to once-a-day dosing, it's the persistence of the pharmacodynamic effect through 24 hours in the great majority of people who had 200mg every 12 hours or 400mg every 12 hours that is striking and gives us absolutely a good clinical pharmacology reason to go and test once-a-day dosing. Great, thank you.

In terms of kind of.

Where they're similar where you see opportunities to differentiate I know that molecule themselves are quite similar maybe talk about exposure other attribute.

Yeah, I'll start and then bill can get into some of the specifics. This is not like a normal market, where you've taken market share from one product to another.

Unknown Executive: You're next.

The government and we've seen this in smallpox and other areas the government needs multiple weapons in the Arsenal to combat.

Liisa Ann Bayko: Your next question is from Liisa Bayko with JMP Securities. Hi there, thanks for taking the question. Um, can you maybe just go through some sort of comparing and contrasting Galidesivir versus Rumbesivir in terms of kind of... Where they're similar, where you see opportunities to differentiate, I know the molecules themselves are quite similar. Maybe you can talk about exposure and other attributes.

Viral outbreaks like the one we're currently experiencing and so.

We see the ability to have both ramdev severe and gallaudet severe in strategic national stockpile is around the globe and you've even heard from some of the government officials that more needs to be done cocktails of drugs need to be done studies need to be done and so there's plenty of room for another.

Jon P. Stonehouse: Yeah, I'll start, and then Bill can get into some of the specifics. You know, this is not like a normal market where you're taking market share from one product to another.

Our an eighth limmer ace inhibitor like Galidesivir, yes.

Jon P. Stonehouse: The government, and we've seen this in smallpox and other areas, the government needs multiple weapons in the arsenal to combat viral outbreaks like the one we're currently experiencing. And so we see the ability to have both remdesivir and galidesivir in strategic national stockpiles around the globe. And you've even heard from some of the government officials that, you know, more needs to be done, cocktails of drugs need to be done, you know, studies need to be done. And so there's plenty of room for another RNA polymerase inhibitor like galidesivir.

In terms of comparison to both kellett, this severe and rare disease nucleoside analogs.

Both adenosine analog structurally obviously the different.

It's really good to see the emerging data on rim disappointed coming at positive Thats good for the world and it's good for the field and it's good for nuclear side analog soon.

Very happy that we started this study.

Other respects I think it's just not enough information to make any detail comparisons.

William P. Sheridan: In terms of comparison, both Galadezivir and Remdesivir are nucleoside analogues; they're both adenosine analogues, but structurally, obviously, they're different. You know, it's really good to see the emerging data on Remdesivir coming out positive. That's good for the world, and it's good for the field, and it's good for nucleoside In other respects, I think there's just not enough information to make any detailed comparisons.

Okay.

[music].

Talking further down the line about prelaunch activities.

[music].

For a change he can you maybe talk about kind of what your plans are how much.

Heavy lifting in terms of hiring and building out the Salesforce infrastructure do you plan to do a head of.

Approval.

Charlie Yes.

Thanks for the question and as I mentioned in my comments were.

Our our in in office team is complete at this point our sales leadership team regional regional sales leaders are complete and we're getting ready for the hiring the field force. So we'll be doing that in Q3.

Charles K. Gayer: Okay, in terms of talking further down the line about pre-launch activities for HAE, can you maybe talk about kind of what your plans are? How much heavy lifting in terms of hiring and building out the Salesforce infrastructure do you plan to do ahead of approval?

And making you want to hit the medical affairs piece.

Sure John I think from medical affairs, prospective and sort of shared in my remarks, we got us a full team deployed that are actively engaging with the care wells than.

Charles K. Gayer: Charlie? Yes, thanks for the question, and as I mentioned in my comments, our in-office team is complete at this point, our sales leadership team, and regional sales leaders are complete, and we're getting ready for the hiring of the field force, so we'll be doing that in Q3.

Similar to Charlie.

We've been really encouraged by the talent, we brought into our teams and their experience in the prelaunch launch phase rare diseases.

Megan Snizinski: And Megan, do you want to hit the medical affairs piece?

Jon P. Stonehouse: Sure, Jon. I think from a medical affairs perspective, and as I sort of shared in my remarks, we've got a full team deployed that is actively engaging with the KOLs. And similar to Charlie, you know, we've been really encouraged by the talents we've brought into our teams and their experience in the pre-launch and launch phase of rare diseases, as well as specifically HAE. So I know he and I are just feeling really excited about where we are today and looking to continue to do the important work in the coming months and ready for a launch later this year.

As well as specifically.

So I know he and I are just feeling really excited about where we are today and looking forward to continue do the important work in the coming months and ready for launch later this year.

Yeah, I can't tell you make his point to really make its points are really important one I can't tell you. The number of people that have come into the commercial organization and medical Affairs and said they came in because we have an oral drug for AG. So that tells you something.

Liisa Ann Bayko: Yeah, I can't tell you Megan's point is a really important one. I can't tell you the number of people that have come into the Commercial Organization of Medical Affairs and said they came in because we have an oral drug for HAE. So that tells you something.

Thank you talk about.

Drug supply, there, where where do you manufacture and then just kind of to Sta are they on track with scheduled actions in that kind of thing I'm, just thinking about due to travel restrictions I'd be curious about some color and how that's.

Liisa Ann Bayko: I think you talk about drug supply there, you know, where, you know, where you manufacture and then just kind of to FDA. Are they on track with scheduled inspections and that kind of thing? I'm just thinking due to travel restrictions. I'd just be curious about some color on how that's all tracking.

Tracking.

Making you want to take the manufacturing question.

Sure sure Sobi side, so couple of things to highlight.

The fact that Biocrysts me that early investment in the dual source redundancy throughout the chain has positioned us well and.

Megan Snizinski: Megan, do you want to take the manufacturing question?

We were able to again do a lot of work before.

Megan Snizinski: Sure, sure. So, Liisa, I have a couple of things to highlight. I think the fact that BioCryst made that early investment in the dual source redundancy throughout the chain has positioned us well, and we were able to, again, do a lot of work before COVID, so we really feel like we've got ample supply and are in great shape with what we need for a successful launch, and everything in terms of what we need would be on track for the PDUFA date in December.

Before covance, so we really feel like Youve got ample supply and are in great shape with what we need a for a successful launch and everything in terms of what we need would be on track for for the PDUFA date in December.

From a supply perspective.

And one other thing that we've started to see even with the FDA. We saw with a the Japanese PMDA is virtual inspections are starting to take place by both agencies.

So that's encouraging too.

Huh.

That's interesting how does how does that work.

Jon P. Stonehouse: And one other thing that we've started to see, even with FDA, as we saw with the Japanese PMDA, is virtual inspections are starting to take place by both agencies. So that's encouraging too.

Through technology.

Okay [laughter] [laughter].

Next gen or some yeah, yeah, yeah, well remember a lot of its document sharing and answering questions and okay.

Understood, Okay and then.

Just to follow up on Gina question.

Liisa Ann Bayko: Huh. That's interesting. How does that work?

Jon P. Stonehouse: Oh.

Operator: Transcripts provided by Transcription Outsourcing, LLC.

QD looks like a real possibility you don't really haven't on slide 21.

Jon P. Stonehouse: Well, remember, a lot of it is document sharing and answering questions and... Okay. Okay, and then just to follow up on Gina's question, QD looks like a real possibility. You don't really have it on slide 20, a kind of outline of when you might explore QD. Can you maybe speak to that? And then, and this is my last question, for 90 and 30, as you think about other indications, what makes sense, maybe, as a second and a third indication to explore, and when might you start working on that? Thank you.

When you buy the lower acuity can you maybe speak to that and then as and then my last question for 1930 as you're thinking about other indications what make sense, maybe at the second and third.

Please turn to explore and when might you start working on that thank you.

Sure.

So the multiple ascending dose healthy subjects study is still open. So we plan to study QD as the rest of year unfolds and then we'll look at the data and decide whether it justifies looking at editing piano in the pitch study one way or another.

William P. Sheridan: Sure. The Multiple Life Threatening Dose Healthy Subjects Study is still open, so we plan to study QD as the rest of the year unfolds, and then we'll look at the data and decide whether it justifies looking at it in the PNH study one way or another.

Yeah, there was on indications and so.

The indications landscape here is rich so one of the.

Incredible things about affected de inhibitor that's put into specific like this is that.

William P. Sheridan: The other was on indications and...

The number of diseases. This can treat and make a huge impact on patients lives is fantastic. So for example.

William P. Sheridan: So, the indication landscape here is rich, so one of the incredible things about a factor D inhibitor that's potent and specific like this is that the number of diseases that this can treat and make a huge impact on patients' lives is fantastic. So, for example, you know, there's C-tracheal marijuana, nephritis, dense deposit disease, membranous nephropathy, and IGA nephro There are a bunch of things in the field of nephritis, and we'll make those decisions as we meet with regulators and develop the program.

They are a seats this sequel, Maryland fraud distance deposit disease.

Membranous nephropathy nephropathy I changed from the there a bunch of things in the field of the fried us.

We'll make those decisions as as we meet with regulators and develop the program and you could very well see that we do a broad clinical development program off of around multiple indications and when we talk about the excitement of this data it's not just p. an age with the data that we have we're excited about all the.

Jon P. Stonehouse: Yeah, and you can very well see that we have a broad clinical development program around multiple indications. And when we talk about the excitement of this data, it's not just P and H. With the data that we have, we're excited about all the kinds of mediated diseases.

Diseases. This one particular difference between PNM and all of the other diseases is that PDH is masked by the some mosys right none of the others.

And the scheduling in the fried us I can easily see as wants to date with the data that we currently have.

William P. Sheridan: There's one particular difference between PNH and all of the other diseases is that PNH is marked by this hemolysis, right; none of the others are. And the scheduling in nephritis I can easily see is once a day with the data that we currently have.

Very exciting thanks, a lot, France and my question.

You're welcome.

Yes. Good question is from Brian Abrams with RBC capital.

Liisa Ann Bayko: Very exciting. Thanks a lot for answering my questions.

Hi, Hello. This is Leo on for Brian I just had.

Jon P. Stonehouse: You're welcome.

Operator: Your next question is from Brian Abrahams with RBC Capital. Hi, hello, this is Leo on behalf of Brian.

Another question on the age profile.

The drug I'm just curious so the patient that had died where they still on.

Brian Corey Abrahams: I just had another question on the profile. The drug, I'm just curious. So the patient that died, were they still on the drug post 28 days? And were they also the same patient that required a transfusion? And can you remind us if factor D inhibition can also increase susceptibility to viral infections? And if it does, how might that play out in terms of impact on clinical trial recruitment in the middle of a pandemic?

The the drug post 28, and where they all the things patient that required transfusion and can you remind us if.

The factor Dean inhibition can also increases up the ability to viral infections and if it does how that might play out in terms of impact the clinical trial.

The middle endemic.

William P. Sheridan: Sure. But, you know, the patient who unfortunately contracted varicella had never had a vaccination and had never had chicken pox, and she was a healthcare worker. So that, you know, that is an unfortunate combination of circumstances, and this person was taking corticosteroids. Chronic corticosteroids is the number one risk factor for getting disseminated varicella. Of course, we've done very extensive diligence around this, including extensive literature searches looking at congenital complement deficiencies. There is not a single case of disseminated varicella with any sort of congenital complement deficiency.

Sure.

So this is patient who unfortunately contracted vericel had never had a vaccination and had never had chicken pox and was a healthcare worker.

So that is an unfortunate combination of circumstances and this person was taking corticosteroids a chronic corticosteroids is the number one risk factor for getting disseminated Vera Sela of course, we've done very extensive diligence around this including extensive literature searches.

Yes.

Looking at.

Congenital complement deficiencies, there's not a single case of disseminated ferrous selling with any sort of congenital complement deficiency extensive which associates radicalism meb.

William P. Sheridan: Extensive literature searches around ecolizumab. There was one reported case, not of a death, but of a young boy who had hemolytic uremic syndrome from Shiga toxin and got varicella and recovered, who was getting ecolizumab. And then in the FDA adverse event reporting system, we've extensively gone through that looking at a whole variety of drugs, but specifically, of course, ecolizumab. And there is one individual who was also getting corticosteroids and mycophenolate mofetil. So all of the evidence here points to corticosteroids. Of course, we want to play this very safe, so we're changing the protocol to make sure that people are immune to chickenpox.

There was one reported case.

None of this bit of young boys.

Ahead, hemolytic Uremic syndrome.

She could talk soon and.

Vera seller on recovered.

Who is getting Eculizumab and then in the FDA adverse event reporting system Weve extensively come through that looking at a whole variety of drugs, but specifically of course eculizumab.

And.

There is one individual who was also getting corticosteroids and microsatellite Mcmaster tool. So all of the evidence here at points towards corticosteroids.

Cautious.

I want to play this very safe. So we're changing the protocol to make sure that people are immune against chicken pox, Yeah, and then your question about recruitment we had a fourth patient as Bill said in his prepared remarks coming in the study last week.

William P. Sheridan: Yeah, and then your question about recruitment. We had a fourth patient, as Bill said in his preparing remarks, come into the study last week. So, you know, there's still a lot of enthusiasm. Bill, the investigators are still really enthusiastic about the drug and the...

So theres still lot of enthusiasm build the investigators are still really enthusiastic about the drug in the trial. It we don't expect it to impact recruitment at all so in summary, all of that diligence says that if you seriously damaged lymphocytes, that's what sets up the risk.

Jon P. Stonehouse: So in summary, all of that diligence says that if you seriously damage lymphocytes, that's what sets up the risk, and the complement system, you know, inhibiting the complement system doesn't do that. Okay, thank you, and if you would like to ask a question,

And the complements system inhibiting the complements system doesn't do that.

Okay. Thank you.

Okay, and if you would like to ask a question. Please press star.

William P. Sheridan: Again, if you would like to ask a question, please press star and then the number 1 on your telephone keypad. Your next question is from the line of Serge Belanger with Liedemann Company.

On your telephone keypad.

Your next question is from the line of search Ballenger.

Company.

Operator: Whitney, we may want to go to the next one.

Surge.

Whitney we may want to go to the next.

Serge D. Belanger: There you are. Go ahead. The mute button was not my friend.

Yes. It does this.

The new button was on my friend.

Serge D. Belanger: A couple questions on 9930. First, Bill, you reported, I think, on slide 26 that you achieved over 98% sustained Alternative pathway suppression at the doses of 200 and 400 milligrams and the multiple ascending dose. In the trial in healthy volunteers, how does that compare to the lower dose of 50 and 100?

Couple of questions on 99 Dirty.

First Bill you reported I think on slide 26 that you achieve over 90% sustained.

Alternative pathway suppression.

The doses of 200, 400 milligrams and the multiple ascending dose.

Part of the trial in healthy volunteers, how does that compare to the lower dose of 50 in 100.

So if we look at the Chuck.

Serge D. Belanger: So, if we look at the chart, what matters is the consistency. So, if you look at the 12-hour time point, you can see that there are many individuals at 50 and a couple of individuals at 100 who have more than 5% residual activity in the alternative pathway in those assays. So, what we want to achieve here is 100% of the subjects having more than 98% suppression, which is exactly what you get at 200 and 400 milligrams. And the slide number on that, Bill? Yeah, slide 26. So it's all about the consistency of effects. So, the second thing here is, as we mentioned a couple of times on the call already, the persistence of effect beyond 12 hours is especially important in PNH because you want to make sure that you have round-the-clock coverage.

What matters is the consistency. So if you look at the 12 hour time point you can see that there are many individuals at 50 and a couple of individuals at 100, who have more than 5% residual activity at the alternative pathway in those areas. So what we want to achieve here is 100% at the subjects having.

More than 98% suppression, which is exactly what you get at 200 400 milligrams in the slide number on that 26.

Slide 26 server so it's all about the consistency of effect.

So the second thing here is as we mentioned a couple of times on the call already the persistence of effect beyond 12, Atlas is especially important in P.H. because you want to you want to make sure that you have round the clock coverage.

Okay.

And then on Gilead severe.

Jon P. Stonehouse: And then on Geli Desevier, you talked about an $82 million contract with BARDA and NIAD. Where are you vis-à-vis the span of that contract? Are there any ongoing efforts to expand that as a... The trial in Brazil gets underway here.

You talked about.

82 million client 82 million dollar contract with BARDA and ads.

Where are you visit the spend the that contract and.

Are there any ongoing efforts to to expand that as a.

The trial and Brazil gets underway here.

Jon P. Stonehouse: So we're about halfway through that total money. I think it's been more consumed on the NIAID side than the BARDA side, and of course, you know, we'll continue to look at other opportunities to get proposals to the government to add additional money, either to this contract or a new contract.

So we're about halfway through that total money I think it's.

More largely consumed on the NIAD side than the BARDA side and of course will continue to look at other opportunities to get proposals to the government to add additional money either to this contract or new Patrick.

Charles K. Gayer: And then I guess just one on barotrauma stat for Charlie. As you start thinking of negotiations for a label, what are the key aspects of... The label you'll be looking for, as well as key aspects of the APEX data that you'll want to see on the Ferro-Telstat label?

Okay.

And then I guess just want to purchase that for a for Charlie.

As you start thinking of negotiations for a label.

What are the key aspects of.

The label, you'll be looking for as well can you aspects of the apex data.

You want to see on the.

Charles K. Gayer: Yeah, thanks. So, thanks.

Search else that label.

Charles K. Gayer: Thanks for the question. So obviously, we've submitted a draft label, and we expect to have a label that's consistent with the other products in the marketplace. And so, you know, we're, and based on what we've seen so far, we're confident that that's what we'll get.

Yes. Thanks. So thanks for the question. So obviously, we have submitted a draft label and we kind of we expect to have a label. That's that's consistent with the other products in the marketplace. So yes, we're and based on what we've seen so far we're confident that that's what we'll get.

Okay. Thank you.

Maurice Thomas Raycroft: Thank you. You're welcome. Your next question is from the line of Maurice Raycroft with Jefferies.

Welcome.

Your next question is from the line of Maury Raycroft ordered with Jefferies.

Maurice Thomas Raycroft: Good morning everyone, and thanks for taking my questions. I just had a quick one on 9930. For patient two, it looks like that patient was getting transfusions at baseline and needed one while on treatment. Do you expect that the patient may need fewer transfusions over time as the patient stays on 9930?

Hi, good morning, everyone and thanks for taking my question I just had a quick one on nine I Rio per patient to it looks like that patient was getting for infusions that baseline and needed one while on treatment you expect the patient maybe you are treated over time.

Based on 90 930.

William P. Sheridan: That individual had a transfusion on day 15, Maury, and at week 8, the hemoglobin has now risen from a post-transfusion of 8.9 or something like that to 11.1. Anecdotally, you know, when the patients have made clinic visits, we've had comments relayed by the principal investigators at the site that this is the best they've ever felt. So, you've got to remember that in South Africa, the C5 inhibitors are not approved. So, you know, the symptoms of the disease have been severe, and they feel fantastic on the drug, which is, you know, it's anecdotal, but it's really great to hear. And the fact that we're seeing this at low doses that are not optimized yet is really, really encouraging.

That individual headed transfusion on day 15, Maury and at week eight the hemoglobin is now arisen from post transfusion is 8.9 or something like that to 11.1.

Dave.

Anecdotally when that patience. It may clinic visits we've had comments relate by the principal investigators at the site. So this is the best type business. So.

You got to remember that in South Africa.

The C inhibitors are not approved so the symptoms of the disease obtained a severe and.

I feel fantastic on the drug which is any it's anecdotal, but it's really great to hear and the fact that we're seeing this at low doses.

That are not optimized shit is.

Really really encouraging.

Got it Okay, and then for patient three it seems like the if your response was not as robust as the other patients.

William P. Sheridan: For patient 3, it seems like the efficacy response was not as robust as the other patients. Just wondering if that's related to the patient's baseline aplastic anemia, and do you think the higher dose could overcome that and get this type of patient to respond better?

I'm wondering if thats related to the patients baseline a classic anemia, and do you think the higher dose could overcome and get the patient get this type of patients respond better.

William P. Sheridan: I think that's a really interesting question. This patient does have combination aplastic anemia and PNH, so the ability of the bone marrow to respond is going to be more limited under those circumstances, but also at week 8 in that patient, the hemoglobin has continued to rise, and I can't predict where it's going to go ultimately, but we are amending the protocol to allow dose escalation in the extension phase for these subjects who are completing cohort 1, so we will be able to get the opportunity to see what happens when we increase the dose.

I think that's a really interesting question. This patient does have a combination I plastic in EMEA and ph. So the ability of the bone marrow to respond is going to be more limited under those circumstances, but also we cutting that patient. The hemoglobin has continued to rise and I can't predict where it's going to go ultimately, but we.

Our amending the protocol to allow.

Dose escalation in the extension phase for the subjects, who are going through a completing cobalt. One. So we will we will be able to get the opportunity to see what happens when we increase the dose.

Jon P. Stonehouse: Got it. And last quick question just regarding the new patent for the crystalline salt forms, Veritulisat. Will any of the new forms be used in the commercial setting? And have you done bioequivalency testing with the new forms?

Got it and last quick question regarding the new Pat for that occurs points all forms.

Total.

When you the new form for use in the commercial setting and you've done bio equivalency testing, which can be forms.

Jon P. Stonehouse: It's the API. So it's in the commercial formulation as we speak, and so it's automatic.

It's the CPI so.

It's in the commercial formulation as we speak and so it's automatic.

Maurice Thomas Raycroft: Got it. Okay. Okay, thanks for taking my question.

Got it okay.

Okay. Thanks for taking my question.

Okay.

Your final question quick question is from the line of Gino way with Barclays.

Huidong Wang: Your final question is from the line of Gina Way with Barclays.

Huidong Wang: Thank you for taking my follow-up. I think I forgot to ask you about the headache question. So the three, like all three subjects, had a moderate headache. We understand this likely is a drug class since we saw also 4471 had a headache. Just wondering if you can give a little bit more color regarding, you know, the onset and then you did mention a little bit, you know, less than one to three days. Like if you could give a little bit more color on the headache regarding its onset, how long it lasts, and how it is resolved.

Thank you for taking my follow up.

Ask about how the question.

Michael.

Hello.

Then just unlikely to walk Clos.

Also.

Kevin Hovick, just wondering if you can give a little bit more color regarding.

Mhm.

Thanks.

Launch.

If you can deliver more color on Hattie Bobby on so how long long haul.

William P. Sheridan: Sure. The onset is very quick in the first day or two. The duration is a few hours to less than a couple of days. And what's really striking about this is three out of three, that means that in every single individual, we're immediately starting to control hemolysis because the reason these people get headaches when they start taking complement inhibitors is because you release nitric oxide from being scavenged. And without going into all of the details, the investigators in the field have worked this out. So it's a class effect of getting on top of intravascular hemolysis with the drug. And then it disappears very quickly.

[music].

Sure.

Yes. It is very quick in the first data to the duration is a few hours.

To less than a couple days and what's really striking about this is three at a three.

That means hitting every single individual where immediately starting to control the most us because the reason these people get hit like when I start complement inhibitors.

Because you.

Release, nitric oxide from being scavenged without going into all of the details.

The investigators in the field that work this out so it's a it's a cost effective getting on top of Intervest Calamos us with the drug and did it disappears very quickly.

William P. Sheridan: So any other drug, you know, to alleviate the headache?

Good luck.

Okay.

Hi.

William P. Sheridan: This has been reported previously with Ecolizumab and Altamiris and other complement inhibitors that are investigational. So it's a sign that the drugs work. Exactly. What it boils down to is that...

So this has been reported previously we take a limit and ultra mirrors and other complement inhibitors that are investigational.

So it's a sign that probably weren't exactly that were both down too is that it is in vivo evidence that the drug is working yes, yes.

Thank you.

William P. Sheridan: It is the in vivo evidence.

William P. Sheridan: It is in vivo evidence that the drug is working, yes. Transcripts provided by Transcription Outsourcing, LLC.

C.

In any.

Like prescription drug or anything to making Hattie now this is the tylenol.

Jon P. Stonehouse: Okay. Okay. Okay, thank you. I am showing no further questions at this time. I will now turn the call back to Mr. Stonehouse for any closing remarks.

Okay. Okay.

Okay. Thank you.

I'm showing no further questions at this time.

Call back to Mr. Stonehouse for any closing remarks.

Operator: Thank you, Whitney. As I said at the beginning, we are transforming this company with three launches coming, two of them coming this year, and starting to generate real revenue starting next year. The data that we have with 9930 is something that we couldn't be more excited about, and that gives proof that we have a drug that can be used in complement-mediated diseases across the board beyond PNH. So we've got a tremendous pipeline. And then add to that the opportunity to play a role in the global pandemic with our antiviral. We just couldn't be in a better spot. And so we're super excited about where we sit. We're going to be working really hard to continue to move our programs forward, get ready for the launches and approvals, and we will keep you posted along the way. Thank you for your interest, and stay safe and have a great day.

Thank you Whitney.

As I said at the beginning we are transforming this company with three launches coming two of them coming this year generating starting to generate real revenues starting next year.

The data that we have with 90 930, we couldn't be more excited about and that gives proof that we have a drug that can be used in complement mediated diseases across the board beyond P., an h. So we got it.

Tremendous pipeline and then add to that the opportunity to play a role in the global pandemic with our antiviral yet we just couldn't be in a better spot and so we're super excited about where we sit we're going to be working really hard to continue to move our programs for get ready for the launches and approvals and we will keep you posted a.

Along the way. Thank you for your interest and stay safe and have a great day.

Ladies and gentlemen. This concludes today's conference. Thank you for participation you have a wonderful day you may now disconnect.

Operator: Ladies and gentlemen, this concludes today's conference. Thank you for your participation, and you have a wonderful day. You may all disconnect.