BioCryst Pharmaceuticals, Inc.

Fourth Quarter 2020 Earnings Call. At this time, all participants are in a listen-only mode. After the speaker's presentation, there will be a question and answer session. To ask a question during the session, you will need to press star 1 on your telephone. Please be advised that today's conference is being recorded. If you require any further assistance, please press star 0. I would now like to hand the conference over to your speaker today, Mr. John Bluth with Biochrist. You may begin.

Thanks, Daphne. Good morning, and welcome to Biochrist's fourth quarter 2020 corporate update and financial results conference call. Today's press release is available on our website. Participating with me today are CEO John Stonehouse, CFO Anthony Doyle, Chief Medical Officer Dr. Bill Sheridan, Chief Business Officer Megan Snizinski, and Chief Commercial Officer Charlie Geyer. 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 John Stonehouse.

Thanks, John. Good morning to all of you, and thanks for joining us. Meaningful value is created by companies that translate great science into clinical benefit and leading to approvals and successful market launches of new medicines that patients are waiting for. And the most successful biotech companies are able to repeat this process of discovery, development, and commercialization across multiple drugs in multiple disease areas. Building this capability doesn't happen overnight, but when you make this transformation, you see real change and real value. That transformation is happening right now at Biochrist. We know patients with rare diseases are waiting for oral treatments despite having injectable therapy to manage their disease. We've heard that for years in HAE and are hearing the same thing in PNH. We've built an exceptional drug discovery platform to go after challenging targets like calocrine inhibitors, factor D inhibitors, and L2 inhibitors. so that we can take this great science, get drug approvals, and finally to the market to put an end to the patients waiting. The approval of Orlodeo in the U.S. and Japan is evidence of this change. Charlie and Megan will describe what we are doing and what we are hearing from customers in the marketplace. And while it's early days in the U.S. launch, they will share why we believe we're off to a good start. Megan will then share with you the even bigger unmet need in Japan for patients suffering from HAE and how Orlodeo can have a big impact as the first approved treatment for preventing attacks. We believe Orlodeo has the potential to generate north of $500 million in global peak sales. Next up is European approval. We expect that approval in Q2, and Charlie will describe how his team in Europe has been preparing for the launch so we can hit the ground running just like we have in the U.S. We also have an increasing body of evidence that we can repeat the success in complement-mediated diseases with our oral factor de-inhibitor. Last year, we shared proof-of-concept data in four P&H patients treated with BCX9930. Bill will update you on the progress of the study and what to expect when we announce the data from a full set of 16 patients, including both treatment-naive patients and C5 inadequate responders. What makes our Oral Factor D program even more valuable and exciting is we plan to go after many complement-mediated rare diseases. Having one molecule to go after many indications could enable us to have several different approvals for many more patients. And finally, you can see the change in our balance sheet. As you will hear from Anthony, capital from our December financing has transformed our company and allows us to put our head down to successfully launch Orladeo around the world and rapidly advance our pipeline. So this transformation is no longer aspirational. It's happening right now, and we're focused on going fast. because we know patients are waiting. Now I'll turn the call over to Charlie to share early insights into our launch. Charlie?

Thanks, John. We invested early to be ready for a fast launch, to get patients on therapy quickly, and to secure reimbursement access for Orladeo. What we're seeing so far is very encouraging. We are wrapping up clinical trial conversions, and the great majority of these patients are choosing to continue with Orladeo. We are also seeing strong early demand from new patients, So far, patients on therapy are equally split between clinical conversions and those new to Orladeo, and our sales and marketing efforts are filling the funnel quickly. What's notable is we're also seeing as many new patients switching to Orladeo from injectable Profi products as those starting Profi on Orladeo after previously treating their HAE with only acute medications. This is right in line with our strategy. because we believe Orlideo offers significant and sustained attack reduction with a reduced burden of treatment regardless of a patient's background therapy or attack rate. More early evidence that we're off to a good start in addressing this strong pent-up demand is that HAE treaters are embracing Orlideo. We have significantly expanded the prescriber base beyond those involved in the APEX clinical trials. Once patients decide to switch to Orladeo, our Empower patient services team works with them to get started on product right away. So far, most are starting on our Quick Start program, while Empower helps them through the prior authorization process. Reimbursement approvals are coming mostly through medical exceptions at this stage, but our market access team is making significant progress as payers see the strong demand from patients. Payers are starting to add Orladeo to their coverage policies, and we expect this process to accelerate over the next quarter. We knew that COVID would require some adjustments, and we prepared to launch in this environment. Face-to-face meetings with customers have been limited in many areas of the country, but physicians have been receptive to Zoom calls because they want to learn about Orladeo. Our team has been very efficient with these interactions by transferring calls directly to colleagues when market access or medical questions come up. Live meetings and congresses are not quite the same in a virtual format, but we've seen a lot of interest in our virtual education events for healthcare providers and patients. For example, over 200 patients attended two virtual events earlier this month. We also see that physicians and their patients are communicating via telemedicine when in-person visits are not possible. And many physicians have been comfortable prescribing oral Adeo remotely because starting an oral medicine does not require training. Our U.S. launch is off to a strong start in meeting the pent-up demand from patients and HAE treaters. Looking forward to Europe, our market research tells us there is also significant pent-up demand for the first targeted oral treatment. The difference in Europe is that prophy use has been limited by lack of options, so we have a great opportunity to grow the prophy market with Orlodea. Just as we did in the U.S., we have built a team with deep rare disease experience and passion for launching innovative drugs. Germany will be our first commercial launch in Q2, and we look forward to reporting on multiple global launches in the coming quarters. Now I'll pass it to Megan to provide more color on how we are starting to change the HAE treatment paradigm and to describe launch preparations in Japan.

Thanks, Charlie. Medical Affairs is partnering closely with Charlie's team to support a successful launch. Our efforts are aimed at increasing physician knowledge and understanding of Orlodeo. Overall, healthcare providers are embracing the strength of our clinical data and product profiles. In particular, the comparisons of how patients do before and while on treatment, like the mean attack reduction from a baseline of three per month to one per month sustained over time, and the attack-free periods many patients are experiencing in our longer-term safety study. We continue to generate additional supportive data, including what we'll share at Quad AI, which starts tomorrow. We'll show the positive outcomes of attack reductions and less on-demand use regardless of prior prophylactic experience or baseline attack frequency. This adds to the body of evidence, helping physicians and patients understand the breadth of experience and improved outcomes while on Orlodeo, including from patients who've been on other prophies before. Our medical strategies also focused on the shift towards individualized treatment plans that best meet the needs of patients with respect to reducing both disease and treatment burden. The recent publication from Dr. Banerjee at Mass General outlining a model for shared decision-making in HAE is evidence of this movement in the field. In addition, our burden of treatment data is resonating. Physicians acknowledge injection fatigue and needle phobia is real. Breakthrough attacks still occur while on other trophies, and patients and caregivers want a treatment that is easier to administer. Our research shows patients want more than only attack reductions. This is why it's so important for physicians to ask about each patient's needs, even if they believe the attacks are under control. Our work is helping physicians see how Orladeo offers patients both the attack control they desire and the lifestyle freedom and benefits of a convenient, more discreet oral once-daily pill. As Charlie mentioned, with us seeing a balance of switch patients from injectable prophy products and those previously only on acute therapy, we are making early progress in shifting the paradigm. John shared how we're supporting multiple launches globally. We were thrilled to receive MHLW approval last month, marking Orlodeo's historic milestone as the first approved profi therapy in Japan. We chose Tori, our commercial partner, given their performance in building the HIV market. They proved they could increase disease awareness and patient identification to grow their HIV business into a $200 million franchise. As a reminder, only about 500 HAE patients are identified today, with prevalent estimates upwards of 2,500. With Orlodeo, Tori has the opportunity to apply their past experience to build the prophylactic market, driving patient identification and prophy adoption similar to what we've seen in the U.S. over the last decade with the introductions of new HAE therapies. With approval, Tori's medical representatives are now able to meet with physicians. They're hearing interest and excitement from physicians who see Orladeo as a major advancement in care for their patients. Our NHI pricing discussions are in progress, and we expect to complete them in early Q2. After price listing, doctors can begin prescribing, and Tori's full launch promotion and marketing activities will kick off. Upon successful completion of the pricing discussion, we receive a $15 million milestone, and we share in Tori's success with a tiered royalty from 20% to 40% of net sales. In addition to the commercial launches, we're equally focused on advancing our Factor D program and pipeline. I'll turn the call over to Bill for more on our clinical progress.

Thanks, Megan, and good morning, everyone. Our BCX9930 development program is making excellent progress. and we are excited to advance this novel factor D inhibitor into advanced development trials across multiple indications in 2021. We're looking forward to a big year. Our overall goal in development of 9930 is very clear, to bring forward an oral monotherapy complement inhibitor treatment for both PNH and additional rare, serious, and potentially life-threatening diseases driven by the alternative pathway. Inhibiting factor D in PNH is important for patients, no matter their prior experience with C5 inhibitors. So our goal with the clinical program is to support a label for monotherapy treatment in all PNH patients. This means PNH patients who are naive to C5 inhibitors, patients who have had an inadequate response to C5 inhibitors, and patients doing well medically, but who want to eliminate the burden of therapy from injections or infusions. In 2020, We shared data from four C5 inhibitor naive patients treated with 9930 monotherapy with doses escalated through 400 milligrams twice a day. This data showed that control of hemolysis was dose related and that the safety profile was excellent. We have since completed enrollment in the study with a total of 16 PNH patients. 10 were treatment naive who received 9930 as monotherapy and six were patients with inadequate responses to C5 inhibitors who received 9930 in addition to their C5 inhibitor treatment. At our upcoming R&D day, we look forward to sharing the results of the complete phase 1 dose ranging trial in PNH. Here's what we plan to have in the data readout. First, we will have data from all 16 PNH patients. The 10 C5 inhibitor naive and 6 C5 inhibitor inadequate responders through at least six weeks of treatment at either 400 milligrams or 500 milligrams BRD. And a range of both clinical outcomes and laboratory outcomes, including, for example, hemoglobin transfusions, reticulocytes, PNH clone size, and LDH, and safety data from dosing for up to 48 weeks. At the R&D day, we'll also share new market research with you from PNH patients, What you'll see will feel very familiar as the insights are very similar to what we saw in HAE patients. There is a tremendous burden of treatment associated with injectables, and these patients want an oral treatment. In 2020, we discussed our plan for dose selection and design considerations for future studies with both US and European regulators. Dose selection for BCX9930 is based on precedent for PK-PD modeling and responses in PNH. With completion of this PNH study, we will have the information we need to choose the dose for accelerated advanced development programs across all indications, and we believe we will be ready to move straight from phase one to pivotal trials in PNH. With the upcoming data readout, we will be ready to finalize our study plans and start pivotal trials in PNH and proof-in-concept trials in selected nephritis indications later this year. We'll announce the details of those trials as we start them. Our strong balance sheet allows us to fully invest in this program. We're very excited by the progress we've made in 2020 and the terrific opportunity we have in 2021 to bring an oral monotherapy closer to approval for seriously ill patients with complement mediatic diseases. I now hand the call over to Anthony.

Thanks, Bill. What a difference a year makes. We ended 2019 with $138 million of cash, and we ended 2020 with $303 million. access to another $75 million, and upcoming Orlodeo revenues from the U.S., Japan, and Europe. The strengthening of our balance sheet, which takes us into 2023, allows us to focus on creating value for the company and shareholders by investing in areas that will provide maximum efficiency in return. The main areas that we are investing in continue to be supporting the launch of Orlodeo globally and investing in the development of BCX9930 across multiple indications. while, of course, making sure that we have the infrastructure in place to support the pace of progress that we expect. As the CFO, it's also nice to have significant financial flexibilities and levers to pull as we move forward. As the launch for Aladeo continues to progress, we can make strategic and financial adjustments that correlate with the pace of the revenues that are being generated. Our investment in Factor D also continues to evolve, and our continuing desires to move quickly and broadly with the investment in this program's development. As I noted, we will also have the option, if we need it, to draw down the additional $75 million from our existing credit facility with Ethereum. And as we showed with the financings that we announced in December, we have future opportunities to access capital with our growing portfolio of assets. Because of these many variables, we are not providing specific revenue or operating expense guidance in the launch period for Orladeo. But based on our expectations for revenue, operating expenses, and our option to access the additional $75 million, we believe our current cash runway takes us into 2023. This position of financial strength allows us to focus on execution, to focus on value creation, and not on near-term cash needs. That's a new and exciting spot for Biochrist. Biochrist is transforming, and so too is our value proposition. We are a commercial-stage rare disease company, and Orlodeo will generate meaningful revenue across multiple regions. With BCX 9930, the development team is working on a drug that has huge potential across multiple indications. The team in Birmingham continues to discover our next generation of medicines for rare diseases, And we are now in a strong financial position to invest in driving value creation across all of these areas. That's it for prepared remarks. We'll now open it up for your questions.

Okay. At this time, if you would like to ask a question, press star followed by the number one on your telephone keypad. To withdraw your question, press the pound key. Please stand by while we compile the Q&A roster. Your first question comes from the line of Jessica Phi with JP Morgan.

Hey, guys. Good morning. Hope all's well. I had a few financial questions. First, on Bloomberg's Orlodeo consensus for this year is around 35 million. Are you comfortable with that number? Second, how should we think about gross to net in the early part of the launch, and could that evolve as you get coverage ramped up? And third, where does formulary coverage stand now? What's your goal for coverage, and when do you expect to achieve it?

All right. Thanks, Jess. I'll take the first one, and Charlie, you can take the next two. So the consensus, as we said, we're not going to give guidance. Our goal is to meet or beat the expectations of Wall Street. And, you know, we're excited to, you know, approach the first quarter, give you a first full quarter in the next earnings call. So that's that answer. And you want to take the gross to net?

Yeah, absolutely. Hi, Jeff. So as far as gross to net goes, you know, first of all, just a reminder for everyone that Oradeo has the lowest WAC price in the market. And we expect gross to net to change over time. As I said in my remarks, early on we're using the Quick Start program and we're getting patients on therapy via medical exception primarily. And so we expect the gross to net to evolve in the year as we get more policies out there. So we're being conservative in how we look at gross to net for year one, and we encourage other people to be conservative in your estimates for year one. As far as coverage, as I mentioned, we're starting to have some early successes with plans putting Orladeo onto policy, and we're getting very good feedback from payers in terms of the value proposition of Orladeo and understanding the patient demand for Orladeo. So we're really expecting coverage to accelerate this quarter. It's going to be a year-long process for some payers, so it will continue to evolve, but we expect to make a lot of progress in the next quarter.

And, Charlie, the other thing you might want to just mention is, you know, in the interim while we're working through negotiating for policy, what are we able to do to get paid?

Yeah, so absolutely. So the first thing we're trying to do is make sure the patients get right on therapy right away. And then we're working through medical exception processes with payers, and we've had some early success with that. It's a more labor-intensive process, but it's one that – we're working closely with physicians and patients on, and so that's working for many patients.

And we get more leverage, Jess, the more we fill the funnel with new starts. So that's a primary focus of Charlie's team.

Great. Thank you. You're welcome.

Your next question comes from the line of Ryan Chang with Bank of America.

Hi, team. Thanks for taking my questions this morning. My first question is on Orladeo. Can you give me an update on the rate of conversion from your EAP and extension studies to the commercial drug? When do you expect the conversion to fully complete based on what you're seeing so far? And I have one follow-up.

Sure, Brian. This is Charlie. I'll take that question. So the conversion is just about complete at this point. We set out to make that conversion this quarter, and we're really wrapping that up at this point. And as I mentioned in my remarks, we're seeing the great majority of patients who are on the two clinical trials plus our EAP deciding to continue on Orlaneo in the commercial world.

And, Charlie, there's two steps in that process, right? There's converting them to commercial drug, and then they have to go through the same prior authorization and the insurance. That's right.

That's right. So what we worked with the clinical sites is getting the start forms for patients, and then they go through the prior authorization. Some of those patients, just like the brand-new ones, will go on QuickStart. And then, as I just mentioned, we're working through medical exceptions and ultimately policies for those patients. But we're really encouraged with the progress on the clinical conversions.

Okay, great. So maybe just one more on 9930. So as we'll be getting data with patients getting the high dose at 400 and 500 for at least six weeks next month, can you remind us if the trial allows the inadequate responders to wean off C5 since You're using 9930 as an add-on. Is there potential for us to see the effect of patients weaning down C5 while they're on 9930? Thank you.

Bill, you want to take that one?

Sure. Good morning. Thanks for the question. Yes, the protocol does allow the patients who are inadequate responders to have the C5 inhibitor withdrawn. So we'd like to see everything be stable. It can take quite a while for the hemoglobin to plateau out, for example. It takes a long time for the bone marrow to get to a new steady state. So in each individual, there will be that opportunity. It's too soon in the study to see that data yet, and it's possible we might have that data later this year.

And it's a great question, Brian, because, again, the goal is monotherapy, as Bill said in his prepared remarks.

Great. Yeah, I agree. Thank you so much for the answers. Looking forward to the data readout.

Great. Your next question comes from the line of Gina Wang with Barclays.

Hi. This is David for Gina. I have a couple of questions. The first one is on the PNH 9930 asset. So it seems to me that all of six patients are being treated. Is there any new cases of rash that's being observed? And can you just give us some additional thoughts around the mechanisms of the onset of rash?

Bill, you want to take that? Sure. So as we noted last year, we've had some cases of inconsequential rash, and these events disappear as BCX9930 treatment is continued, in fact, even if the dose is increased on day 15 for the protocol. So we'll update that, you know, at the R&D day call with the new data cut that's coming up. It hasn't been an issue. And with regard to mechanism of action, You know, if you look in the literature and we've done extensive consulting, actually this started way back in the Barrett Health State Development Program when we saw a few similar cases. It's seldom that you actually work these things out for these benign rashes, exactly what the mechanism is. So we don't expect that we'll, you know, that we'll have, you know, laboratory, you know, investigations that work out the mechanism exactly. That almost never happens.

But the main point is it's benign, it goes away, and you can keep dosing up. So in our view, it's really non-consequential.

Yeah, that's really helpful. So another question is around your registrational trial for 9930. I understand that you're probably going to share some additional color on the R&D day. I'm just wondering if you can share some initial thoughts around the registration trial. Would it likely be a single-arm trial, or do you need to have an arm with C5 inhibitor to show non-inferiority or superiority?

Bill, you might want to just focus on what's the goal of the label that we're shooting for with the drug, and then that can help give them a sense of what we need to study.

Sure, so just to clarify one thing. At the R&D day, I won't be going into designs of future studies. When we start studies, we'll talk about that. That's later this year. At the R&D day, we'll go over the data from the ongoing study, which we're very excited about to share with you. And the goal for treatment here is to make our oral drug available for every patient with PNH as a monotherapy. So that means that we'll probably have to do more than one study because there are people who are not currently on C5 inhibitors for various reasons, and there are people who are currently on C5 inhibitors. Some of them are not doing so well and having inadequate responses, and some of them are doing okay. But an oral drug has powerful attraction, so we'd like to be able to cover all the bases. You can have a look at other sponsors' studies that are published, and there's a history of control clinical trials in the field, I think that would give you some guidance as to where the standards are.

I think the most exciting part is we're going from a phase one study into pivotals, and Bill and his team have done a fantastic job of being creative and getting us to accelerate. That's going fast.

Thank you for the color. You're welcome.

Your next question comes from the line of Brian Abrams with RBC Capital Markets.

Great. Thanks for taking my question. This is Steve Vaughn for Brian. Though it's early days in the rollout, can you share whether Orladeo uptake is even across severity with mild and severe patients equally represented there? And any breakdown on whether new patients are coming from general practitioners or specialists? Thanks.

Sure. Hey, Steve. It's Charlie. You know, as I said in my prepared remarks, we're really pleased with the breakdown of patients thus far. We're getting an equal split with people coming from switching from injectable prophies and those who are treated with acute only and then coming over to prophy for the first time now that Orlodeo is available. You know, as far as the prophy switches, you know, we can't comment specifically on the severity of those. They were on prophy already, so they were pretty severe. But what our clinical data shows is that regardless of background therapy, regardless of treatment rate, patients across the board do well on Orladeo.

And then his second question was the doc prescribing is a GP specialty.

Yeah, I mean, at this point, this is predominantly a market that's treated by allergists, immunologists. And we know where those doctors are, and we're really excited because we're moving well beyond the group that did our clinical trials, and we're seeing real uptake in a broadening base of those specialists. Eventually, we'll get some GPs and others too, but right now we're focusing on the big top treaters.

Yeah, your question's a good one. And one of the benefits of COVID is the fact that you're not behind a steering wheel driving to the next clinic or you're not on an airplane. And so diving deeper into the list, I mean, as Charlie says, the initial part of the launch is focused on the high prescribers, but we can work our way through the list over time, and doing it remotely or virtually is a real plus. Thanks. You're welcome.

As a reminder, to ask a question, press star 1 on your telephone. And your next question comes from the line of Mari Raycross with Jefferies.

Hi, good morning, everyone. Congrats on the progress, and thanks for taking my questions. My first question was on 9930 data. It's your R&D data. I guess, how should we think about hemoglobin variability and the bar for success on hemoglobin measures for the naive and experienced patient populations?

Bill, you want to take that?

Sure. Hi, Murray. Interesting question. You know, the leading physicians who treat PNH patients have started to think about, you know, what are the goals of treatment in the era of introduction of proximal complement inhibitors into clinical research? And, you know, there's a nice publication on that that talks about grades of benefits. So, you know, controlling the transfusions having people not be dependent on transfusions is a big goal and to do that you need to stabilize the hemoglobin and it's you know clearly better for people if their anemia is relieved and their fatigue improves so i think that controlling transfusions and having the hemoglobin go up in that you know in that publication early publication as a thought starter you know there were various metrics included for hemoglobin, including 8 grams, 10 grams, and 12 grams per deciliter. So I don't think there is a single magic number is the answer. You know, the goals here are to control the transfusions and improve the anemia and improve the symptoms of the disease.

Got it. Okay. That's helpful. And then on clinicaltrials.gov, the estimated enrollment for PNH was relatively high. Just wondering if you can provide any insight into the enrollment rate for this study and remind on rationale for why you didn't add more sites for this study?

For our phase one? So we, so, you know, this was an innovative design that included P and H patients, you know, phase one first in human study. We started off with healthy subjects, single S ending dose, healthy subjects, multiple S ending dose, and then part three of the study, we designed in an incredibly flexible way because when we started, we didn't know how many subjects we would need to complete dose ranging. So what we did there was have potential for multiple cohorts starting at different doses that we worked out along the way, and we wanted also to study a number of patients with C5 inhibitor inadequate response history and a number with no history of ever having received a C5 inhibitor. So that flexibility was basically, there wasn't a particular number that we had in mind. We had enough in the trial design that we had a good envelope and we needed no more than 16 to hit our objectives.

Yeah, that piece is really important. We always put numbers so we don't have to add amendments to increase the study size. 16 was plenty to figure out the dose. And the other thing is it's a rare disease, so you can't use these patients in the next study. So if you over-enroll in early studies, you have a more challenging recruiting for the later studies.

Got it. That makes sense. And maybe last quick question. So you've talked about providing clarity on additional indications to pursue with 9930 that you could move directly into Phase 2 with. And it sounds like we could learn more about this at your R&D day. I guess just clarifying if that's the case, if we should expect an update on that. And then can you say if you've already reached some alignment with regulators on moving directly into Phase 2s?

Bill, you want to take that?

Sure. On the first question with regard to the nephritis indications and other indications. At the R&D day, we will go over the field and how exciting it is and what the opportunities might be. We won't be specifying exactly what we're including in our nephritis studies until we start them because we've seen it's a very competitive field and don't advertise exactly what we're doing before it's absolutely necessary. With regard to the second question, on interactions with regulators, we had very good interactions last year. And the two key topics were, how do we pick a dose for pivotal trials? And that's based on PKPD modeling, and we got alignment on that. And the second was just general design considerations around pivotal studies and moving directly from the phase one dose ranging into pivotal studies, and we got alignment around those general considerations. So we need to finalize that design, wrap that up, and then later this year we'll be in a position to start.

This is another efficiency, Maury, in terms of being able to do a dose-ranging study in P&H patients in Phase 1 and then have the dose to be able to go into other indications. That really accelerates the program.

That's also a key point, right? So Factor D is not the targeted target of mutation in any of these diseases you know it there's a whole range of different things can happen that disturb the complement system and activate the alternative pathway all the way from pigot mutations in pnh in bone marrow stem cells to germline factor h mutations and so on so none of those are mutations in fact to do so factor d is just happens to be the enzyme that starts the alternative pathway and The dose of treats that is adequate to inhibit factor D and P and H is the dose that's adequate to inhibit factor D and everything else.

Got it. Thank you very much for the perspective, and congrats again. Thanks, Morris.

At this time, there are no further questions, and I will now turn the call over to Mr. Stonehouse for concluding remarks. Thanks.

So first off, let me again thank you for joining us. This is a really exciting time at BioChrist, and so we look forward, first off, to be sharing the 9930 Phase I dose-ranging data with you at our R&D day on March 22nd, and we also look forward to sharing the first full-quarter data of Orla Deo Sales at our next earnings call. So as I said before, the transformation in this company is happening now, and we hope that we've gotten your interest. And if you'd like to reach out to us, we're happy to connect with you in the meantime. So thanks again, and have a great day.

This concludes today's conference call. Thank you for participating, and you may now disconnect.