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- Orladeyo approved in the U.S. and Japan, EU approval expected in Q2
- “Dose-ranging trial of BCX9930 has fully enrolled 16 PNH patients; Data to be presented at R&D day March 22″
- Year end cash of $303 million which provides runway into 2023
- R&D day March 22, 2021
Q4 2020 Conference Call Transcript
Ladies and gentlemen, thank you for standing by and welcome to the biocryst 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 one on your telephone. Please be advised that Today’s conference is being recorded. If you require any further assistance, please press star zero. I would now like to hand the conference over to your speaker today, Mr. JOHN Bluth with bio Chris, you may begin.
Thanks, Daphne. Good morning and welcome to bio Chris fourth quarter 2020 corporate update and financial results conference call. Today’s press releases available on our website. participating with me today are CEO john stone house, CFO Anthony Doyle, Chief Medical Officer Dr. Bill Sheridan, Chief Business Officer officer Megan sinskey, and chief Commercial Officer Charlie Guyer. 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 on audited and forward looking financial information, as well as the company’s future performance and or achievements. These statements are subject to known and unknown risks 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, leading to approvals and successful market launches of new medicines that patients are waiting for.
In 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 biocryst. 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 and he and I are hearing the same thing and P and H.
We’ve built an exceptional drug discovery platform to go after challenging targets like kallikrein inhibitors, factor D inhibitors, and elptoo inhibitors. So that we can take this great science get drug approvals and finally to the market to put an end to the patient’s waiting.
The approval of Orla de Mayo in the US 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 US 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 HIV and how Orlando can have a big impact as the first approved treatment for preventing attacks.
We believe Orlando 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 US.
We also have an increasing body of evidence that we can repeat the success in compliment mediated diseases with our oral factory inhibitor.
Last year we shared proof of concept data and for P and H patients treated with bc x 9930.
Bill we’ll update you on the progress of the study and what to expect when we announced
The data from a full set of 16 patients, including both treatment naive patients, and C five, 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 for the day all 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 Berlinale. 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 Orlando. We’re also seeing strong early demand from new patients. So far, patients on therapy are equally split between clinical conversions, and those new to Orlando. 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 Orlando from injectable prophy products as those starting prophy on Orlando after previously treating their Hae with only acute medications. This is right in line with our strategy because we believe Orla do offer 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 off to a good start and addressing this strong pent up demand is that ha traders are embracing Orlando, we have significantly expanded the prescriber base beyond those involved in the apex clinical trials.
Once patients decide to switch to Orlando, our empower patient services team works with them to get started on product right away. So far, most are starting on our quickstart program, while in power 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 Orla data 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 were and we prepared to launch in this environment. Face to face meetings with customers had been limited in many areas of the country. But physicians have been receptive to zoom calls because they want to learn about willowdale.
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 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 to 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 Orla do remotely because starting an oral medic medicine does not require training.
Our us launch is off to a strong start and meeting the pent up demand from patients in ha traders. Looking forward to Europe. A market research research tells us there is also a 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 profi market with Orlando. Just as we did in the US 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’re starting to change the HIV treat 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 Orlando. overall healthcare providers are embracing the strength of our clinical data and product profile. In particular is the comparison of how patients do before and while on treatment, like the main attack reduction from a baseline of three per month to one per month, sustained over time, and the attack three periods many patients are experiencing in our longer term safety study. We continue to generate additional supportive data, including what we’ll share a quad AI which starts tomorrow will 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 Orlando, including from patients who’ve been on other profiles before. Our medical strategies also focused on the shift towards individualized treatment plans that best meets the needs of patients with respect to reducing both disease and treatment burden. The recent publication from Dr. bannerjee at Mass General outlining a model for shared decision making and he 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 prophecies. And patients and caregivers want a treatment that is easier to administer or research. 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 Orlando 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 a seeing a balance of switch patients from injectable prophy products and those previously only on acute therapy, we are making early progress and shifting the paradigm. JOHN shared how we’re supporting multiple launches globally. We were thrilled to receive MHL w approval last month, marking Orlando’s historic milestone as the first approved prophy 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 ha patients are identified today with prevalence estimates upwards of 2500. With Orlando Tory 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 US over the last decade with the introductions of new HIV therapies. With approval towards medical representatives are now able to meet with physicians. They’re hearing interest and excitement from physicians who see Orlando as a major advancement in care for their patients.
or any type 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’d receive a $15 million milestone, and we share employee 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.
Bc x 9930 Development Program is making excellent progress. And we are excited to advance this novel thekkady inhibitor into advanced development trials across multiple indications in 2021. We’re looking forward to a big year.
Our overall goal and development of 9930 is very clear to bring forward an oral monotherapy complement inhibitor treatment for both P and H an additional rare, serious and potentially life threatening diseases driven by the alternative pathway.
inhibiting factor D and P and h is important for patients, no matter their prior experience with C five inhibitors to go to clinical program to support a label for monotherapy treatment in all plh patients. This means P and H patients who are naive to see five inhibitors patients who have had an inadequate response to T five inhibitors. Patients doing well medically, but who want to eliminate the burden of therapy from injections or infusions. In 2020, we shared data from four c five inhibitor naive patients treated with 9930 monotherapy, with doses escalated to 400 milligrams twice a day. This data showed that controller thermolysis was related to the safety profile this next one.
We have since completed enrollment in the study, with a total of 16 P and H patients 10 retreatment naive, who received 9930 as monotherapy, and six for patients with inadequate responses to see five inhibitors who received 9930. In addition to the C five available treatment, and our upcoming r&d day, we look forward to sharing results. The complete phase one dose ranging trial and panitch is what we plan to have in the data readout. First, we will have data from all 16 T and H patients. It tends to five integrally naive and 65 inadequate responders, through at least six weeks of treatment at either 400 milligrams or 500 milligrams B ID and the range of both clinical outcomes and laboratory outcomes, including for example, hemoglobin transfusions for particular sites, P and H client 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 P and H patients. What you’ll see will feel very familiar as the insights are very similar to what we saw in HIV patients. There’s a tremendous burden of treatment associated with injectables, and these patients want an oral treatment. In 2020, we discussed that plant that does selection and design considerations for future studies with both US and European regulators. The selection of a cx 9930 is based on precedent for PK PD modeling and responses in plh. With completion of this tnh 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 and plh. With the upcoming data readout, we will be ready to finalize our study plans and stop pivotal trials and clh and proof of concept trial in selected the Friday syndication later this year. We’ll announce the details of those trials as we start them.
A strong balance sheet allows them to fully invest in this program. We’re very excited by the progress we made in 2020. And the terrific opportunity we have in 2021 to bring an oral mono therapy closer to approval. Seriously, your patients will complement mediated diseases. Now hand the call over to Anthony.
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 holiday revenues from the US 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 and return. The main areas that we’re investing in continue to be supporting the launch of holiday or globally and investing in the development of BC x 9930. 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 parlodel 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 faculty also continues to evolve and are 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 us to draw down the additional 75 million from our existing credit credit facility with a cerium. And as we shared 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 Ladell, 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 biocryst biocryst is transforming and so to is our value proposition. We are commercial stage Rare Disease company and already the data will generate meaningful revenue across multiple regions. With bs x 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 our prepared remarks will now open it up for your questions.
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 standby while we compile the q&a roster.
Your first question comes from the line of Jessica buy with JP Morgan.
Hey, guys, good morning. Hope all’s well, I had a few financial questions.
First on Bloomberg, Orlando consensus for this year is around 35 million. Are you comfortable with that number? Second, how should we think about gross nets 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 growth to net goes, you know, first, first of all, just a reminder for everyone that Orlando has the lowest whack price in the market. And we kind of we expect gross to net to change over time, as I said in my remarks, early on, we’re using the Quickstart program and and we’re getting patients on therapy via medical exception primarily. And so we expect this to the gross to net to evolve in the air 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 Orlando onto onto policy. And we’re getting very good feedback from payers in terms of the value proposition of Orlando, and understanding the patient demand for Orlando, so we’re really expecting coverage to accelerate this quarter. It’s going to be a year long process for some payers. So it’ll 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, what are we able to do to get paid? Yeah, so absolutely. So the first thing we’re trying to do is make sure patients get right on therapy right away. And then we’re working through medical exception processes with with payers. And we’ve had some early success with that. It’s a more labor intensive process. But it’s 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, just the more we fill the funnel with new start. So that’s a that’s a primary focus of Charlie’s team.
Great, thank you. Welcome.
Your next question comes from the line of wine chain, with Bank of America.
Hi, Tim, thanks for taking my questions this morning. My first question is on holiday. Oh, can you give me an update on the rate of conversion from your EAP and extension studies to the commercial truck? When do you expect the conversion to to come on to the pulley company based on what you’re seeing so far? And I have one follow up?
Sure. Brian, this is Charlie, I’ll take I’ll take that question. So the conversion is is just about complete at this point are 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 or Ladell in the commercial world. And totally there’s two steps in that process. Right. There’s converting them to commercial drug and then there’s they have to go through the same prior authorization and the insurance.
That’s right. That’s right. So what we 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 exemptions and ultimately policies for those patients. But we’re really encouraged with the progress on the clinical conversions.
Okay, great. Um, so maybe it’s just one more on 9930. So as we’ll be getting data, with patients getting the high dose at 405 100, for at least six weeks next month, Can you remind us that the trial allows the inadequate responders to wean off sci fi since you’re using 9930? As an add on? Is there potential for us to see the effect of patient weaning down sci fi while they’re on 9930?
Thank you. Bill. Do you want to pick that one?
Sure. Good morning. Thanks for the question. Yes, the protocol does allow patients who are inadequate responders to have the CCI inhibitor withdrawn. So we’d like to see everything be stable, you know, 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’ll be that opportunity. It’s too soon in the study to see that yet. And it’s possible we might have that data later this year. And and it’s a great question, Brian, because again, the goal is mono therapy, as Bill said in his his prepared remarks.
Great. Yeah, I agree. Thank you so much for the further answers. Looking forward to that data read out. Right.
Here. Next question comes from the lineup, Gina Wang, with Barclays.
Hi, this is David for for Gina. So I have a couple of questions. The first one is on the pH 930. Assets. So it seems to be that all 16 patients being treated, is there any new cases of a rash? And that’s being observed? And I can just give us some additional thoughts around the mechanisms of the downside of rash.
Do you want to take that? Sure.
So the you know, as we noted last year, we’ve had some cases of inconsequential rash. And these events disappear. As the 699 30 treatments continued infective doses increased on day 15 for the protocol. So we’ll update that, you know, the r&d day call with the new data cup is coming up. It hasn’t been an issue. And with regard to the mechanism of action, you know, if you look in the literature, and we’ve done extensive consulting, actually, this started way back in the bear trailside Development Program, when we saw a few similar cases. It’s seldom that you actually work these things out for these benign crashes exactly what the mechanism is.
So we don’t expect that we’ll know that we’ll have no laboratory 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 it’s really non consequential.
Yeah, that’s really helpful. So another question is around your registration or trial for the notary zero, understand that you’re going to share some additional color on the RMD day, I’m just wondering if you can share some, some initial thoughts around the execution child would likely be a single arm trial, or do you need to have a arm with the five inhibitor to show noninferiority or superiority?
Hey, Bill, you might want to just focus on what’s the goal of the label that we’re shooting for with with the drug, and then that can help give them a central what we need to study?
Sure. So just just to clarify one thing on the r&d day, I won’t be going into designs of future studies, when we start studies will, but we’ll talk about that later this year. 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 oral drug available for every patient with P and H 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 C five inhibitors for various reasons and there are people who are currently on C five inhibitors. Some of them are not doing so well and having inadequate responses and some of them are doing okay. But you know, oral drug has powerful attraction. So we’d like to be able to cover all the bases. You know, you can have a look at other sponsors, studies that are published, and there’s a history of controlled clinical trials in the field, I think
that will 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 pivotal and building this team have done a fantastic job of being creative and, and getting us to accelerate that’s going fast.
Thank you for the color.
Your next question comes from the line of Brian Abrams with RBC capital markets.
Good, thanks for taking my questions. Stephen. For Brian, those early days in the rollout, can you share whether or Ladell uptake is 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, a status? Charlie. You know, as I said in my in my prepared remarks, the the we’re really pleased with the breakdown of patients thus far, we’re getting an equal split with people coming from switching from injectable proxies, and those who are treated with acute only and then coming over to prophy for the first time now that Orladeyo’s available, you know, as far as the profi switches, you know, we can’t comment specifically on the severity of those they were on prophy already. So they’re pretty, pretty severe. But what our clinical data shows is that regardless of background therapy, regardless of treatment raid, patients across the board do well on Orladeyo.
And then a second question was the doc prescribing as a specialty?
Yeah, I mean, at this point is this is predominantly a market that’s treated by allergist, immunologist, 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 on the big top traders. Yeah, your question is a good one. And one of the benefits of COVID is the fact that you’re not behind the steering wheel driving to the next clinic or you’re not in an airplane. And so diving deeper into this, 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 and doing it remotely or virtually is a real plus.
As a reminder, to ask a question, press star one on your telephone.
And your next question comes from the line of Mari Ray cross with Jefferies.
Hi, good morning, everyone. Congrats on the progress. And thanks for taking my questions.
My first question was on BCX9930 data. It’s your r&d day, I guess how should we think about hemoglobin variability and the bar for success on hemoglobin measures for the naive and experienced patient populations?
Do you want to take that? Sure. All right. Thank you. Interesting question. You know, the, the leading physician to treat tnh 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 controlling the transfusions, and having people not be dependent on transfusions is a big goal. And to do that, you need to stabilize the hemoglobin. And it’s clearly benefit people, if they’re an Emir is relieved and the fatigue improves. So I think the controlling transfusions and having the hemoglobin go up in that, in that publication, early publication as a good startup. You know, there were various metrics included for hemoglobin including eight grams, 10 grams and 12 grams per deciliter. So I don’t think there is a single magic number with the answer. The goals here are control of transfusions and improve the anemia and improve the symptoms of the disease. Got it?
Okay, that’s helpful and, and then on clinical trials.gov. The estimated enrollment for P and H was relatively high. Just wondering if you can provide any insight into the enrollment rate for this study, and remind rationale for why you didn’t add more sites for thisgraph type one.
So we know this was an innovative design that included P and H patients. In phase one person human study, we started off with healthy subjects, single ascending dose, therapy, subjects, multiple ascending dose. And part three, the study, redesigned in incredibly flexible way. Because you know, when we started, we didn’t know how many subjects we would need to complete those ranges. So what we did there was have potential for multiple cohorts studying at different doses that we worked out along the way. And we wanted also to study a number of patients with C five and hc inhibitor, inadequate response history, and a number with no history of ever having a C two c five inhibitor. So that flexibility was basically, you know, there wasn’t a particular number that we had in mind, you know, we had enough, you know, in the trial design that we had a good envelope, and we needed, we needed no more than 16 to fit our objectives. Yeah, that that piece is really important. We always put numbers. So we don’t have that 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 don’t you can’t use these patients in the next study. So if you over enroll in early studies, you have a more challenge in 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 two 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, I’m moving directly into base twos?
Bill, you want to take that? Sure. On the first question, with regard to the new products, indications and other indications, Darren D day, we will go over the fields, and how exciting it is and what the opportunities might be. We won’t be specifying exactly what we’re including in our new product studies until we start them because we’ve seen no very competitive fields, 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 PK PD 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 the alignment around those general considerations. So we need to finalize that designs, wrap that up. And then later this year, we’ll be in a position to stop. This is another efficiency Mori, in terms of being able to do a dose ranging study and P and H patients in phase one, and then have the dose to be able to go into other indications that really accelerate the program.
That’s a key point, right? So factor D, is not the targeted target of mutation in any of these diseases. It is a whole range of different things can happen that disturb the complement system and activate the alternative pathway all the way from picking mutations in P and H in bone marrow stem cells, to germline factor, h mutations, and so on. So none of those are mutations, in fact, to do so effectively, it’s just happens to be the enzyme that starts the alternative pathway and the dose that treats that is adequate to give it back to b and plh.
The dose is adequate to effectively everything else. Got it?
Thank you very much for the perspective. And congrats again. Thanks, Mark.
At this time, there are no further questions. And I will now turn the call over to Mr. Stone house for concluding remarks.
So first off, let me again, thank you for joining us. This is a really exciting time at biocryst. And so we look forward first off to be sharing the 9930 phase one dose ranging data with you at our r&d day on March 22. And we also look forward to sharing the first full quarter of Orlando 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 you know 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 you
Thank you for participating and you may now disconnect