We provide a research subscription service with a free trial and cancel anytime. Try us out to see if we’re a good fit for your needs. See our service, plans and pricing, customer reviews and more by clicking here.
- Launch is going well; management reiterated the pent-up demand they’ve witnessed by patients
- CEO Jon Stonehouse reiterated that their goal is to meet or beat the Street’s revenue expectations for Orladeyo
- While the pandemic has impacted the ability of BioCryst’s sales team to engage physicians they have been successful nonetheless
- Orladeyo is well received by patients and physicians due to the pent-up demand for an oral preventative therapy for HAE
- List price for Orladeyo in Japan was set at $250K per patient per year
- Commercial strategy is different than in the U.S.
- BioCryst’s commercial partner in Japan Torii Pharmaceuticals will now need to go out and build the market (not many patients formally diagnosed)
- The approval triggered a $15M milestone payment to BioCryst
- Approval for Orladeyo in the EU expected this quarter. The market in the EU is concentrated in treatment centers so identifying patients is easier
- First launch will be in Germany in 2Q21
- More launches to come this year
- With regards to BCX9930 management reiterated that the goal is monotherapy. This means that they want all patients with PNH, regardless of their satisfaction/disease control with current injectable therapies.
- Of note, when asked about partnering to fund the program Bill Sheridan’s answer really resonated with us.
- “We want it all” referring of course to the rights/value of BCX9930
- Starting pivotal trials in PNH later this year. They may do multiple to support the broad label they have in mind
- Of note, when asked about partnering to fund the program Bill Sheridan’s answer really resonated with us.
- By broad label for BCX9930 Dr. Sheridan is referring to the fact that they want to develop and commercialize BCX9930 for a range of complement mediated disease and a label that reflects this broad potential utility
- CFO Anthony reaffirmed the company’s strong financial position with over $300M in the bank and access to an additional $75M upon hitting certain milestones for Orladeyo
Serg Belanger (analyst): Welcome to day three of the Needham healthcare conference. We have the biocryst team with us this morning. company has covered for for a few years now. They went through a lot of exciting changes in the last year first product approval transitioning to a commercial biopharma and recently announced some exciting data on the next asset they’re bringing forward in development. So with us this morning, we have the CEO john Stonehouse, we have the CFO, Anthony Doyle. We have the chief Commercial Officer Charlie Gayer. We have the new Chief r&d officer, Ellen thackray, Chief Business Officer, Megan Susan ski, and john Bluth, who handles IR functions for the company. So welcome, everybody. And I’ll hand it over to to john to give us an overview. And then maybe we’ll talk about the news that you guys announced this morning.
Jon P Stonehouse (CEO): Sure. Thanks, Sergen. Thanks for inviting us to the Needham healthcare conference. We can’t forget bill sharedin it our chief medical officer, as well as is with us today. So you know, the company is going through a major major transformation, we are building a company that discovers develops, and commercializes oral drugs for patients suffering from rare diseases. And we couldn’t be more excited with where we are, there are very few companies that are able to do all three. And when you are you create massive value. And we’re in that transformation as we speak. And we look forward to sharing more details with you today we’re going to be making some forward looking statements. And those statements have risks. And those risk factors can be found on our website. So as Serg said, we’ve got some really exciting news. I’ll give the headline and then Megan can answer any specific questions that you have surge on it. But this morning, it was announced that the MHLW in Japan, the Ministry of Health had made a decision on the pricing, which is the final step in the process of approval. So we now have a Japanese price. And our partner Tori is ready to go and we’ll be launching the drug very soon. So extremely exciting time for us. Both with the launch here in the US now the launch in Japan and soon we expect the approval in Europe as well and launching there.
Serg Belanger (analyst): Okay. So you had received approval, the Japanese approval a few months back, right. So where does the Japanese list price kind of compared to the US price?
Jon P Stonehouse (CEO): Megan you want to take that?
Megan Sniecinski (CBO): Sure, john. Thanks. Hi, Serge. So as we share with the news today, the price equates to about 250,000 US dollars per patient per year. And for us, we’re really pleased that we reach that agreement. I think the fact that now patients in Japan will have access to prophylaxis treatment. It will just revolutionize the standard of care and the treatment paradigm for these patients. So as john said this, you know, this is exciting news for us and Tori’s ready as we started the press release. They’d be launching shortly after the price listing occurs on the 21st. All in all, a really great day for, for the community.
Serg Belanger (analyst): And if I recall, this is the the first prophylactic he product for the Japanese market.
Megan Sniecinski (CBO): Correct.
Serg Belanger (analyst): Okay. And remind us again, the the economics, I know there’s a, there’s a $15 million milestone now with the pricing has been established. Just talk about the royalties or transfer pricing that’s involved.
Megan Sniecinski (CBO): Sure, I’d be happy to john. JOHN, would you like me to take that, please? Sure. So sir, you’re right. The news today triggers the $15 million milestone from Tory. And then the other aspects of the partnership agreement, we’re really excited that we structured this way, we do participate in their success through a tiered royalty on net sales ranging from 20% to 40%. So again, as they build this market, and you know, they’re we’re fully aligned on supporting this and making it as successful as possible, and we get to share in that that great work that we we expect them to do.
Jon P Stonehouse (CEO): And Serg one thing, you know, you pointed out that this is the first prophylactic in the market markets extremely different from both us, and Europe. And so there’s a real, I think, a really nice opportunity to help patients with a once a day oral prophylactic treatment. And, and I think it’s, you know, what we’ve seen around the world, is that when good therapies come forward in a country, the diagnosis and treatment of patients goes up dramatically. And so we expect that in Japan as well.
Serg Belanger (analyst): Okay. So let’s talk about the US launch. You launch a product in December. So, in the next guess, the next month or a few weeks, we’ll see the kind of the first full quarter of of sales. I don’t expect you to give us that number today. But maybe just talk about the you know, give us a launch update, you know, what you’ve seen so far, and what have been some of the COVID challenges that you know, you have to deal with? And in this environment?
Charlier Gayer (CCO): Yes, sure search. So we’re off to a very good start. We knew we invested early because we knew there was going to be a lot of demand from patients, we really invested in doing research to understand the marketplace. And so we got our Salesforce on early and they were ready to go. So COVID you know COVID has an impact. But the team’s been really creative in terms of how they get in touch with doctors, using zoom calls, other mechanisms has been very successful. And the fact that we’ve got an innovative product that patients and doctors want, means that the you know, the doctors and their their staff have been receptive to seeing us. So despite COVID, we’re, we’re off to a good start. And, you know, we’re seeing that or that patient pent up demand that we expected.
Serg Belanger (analyst): Okay, so we’re most reps able to have face to face meetings with the with physicians,
Charlier Gayer (CCO): you know, it really it’s it varies across the country. And some have been able to do that more. It’s it’s really a state by state, local, local kind of condition. Of course, most of the providers are vaccinated at this point, which is great, our team starting to get vaccinated. So we know more and more is going to open up and that that creates more opportunity for us to get to some of the providers that we haven’t seen and to have those face to face interactions that ultimately are, you know, the best way to, to meet with with customers. So we’re looking forward to being able to do more.
Serg Belanger (analyst): And then in terms of the patient physician interactions, I know from other products that, you know, in this environment, there’s a lack of face to face. There’s overall face to face interactions, and that’s been kind of a limiting factor to to switch patients to new products.
Is that the same kind of same kind of issue for HAE? Or are they a proponent of telemedicine use that kind of has address that problem? potential problem?
Charlier Gayer (CCO): Yeah, no, great, great question. And I think, you know, on the telemedicine side, what we see largely is that allergist immunologists, who are the main traders, they really embrace that they used a lot of that, particularly earlier in in COVID. But you know, I think increasingly patients are being able to see their doctors that has not COVID what we hear from doctors and patients was not a big limitation interacting, whether it was virtually or in person. And then the other thing is just with Oraldayeo being, you know, oral once daily. It’s easy to prescribe
And so doctors have been comfortable prescribing it remotely to patients where where the situation calls for that. And that, you know, the last thing I’ll say is, these doctors really tend to know their HAE patients well, because the average physician might only have 5,10,20 patients. And so the interaction, you know, sort of the history is, is there. And that made for the right patients and makes them very comfortable prescribing Oraladeyo, even with COVID.
Jon P Stonehouse (CEO): I think something else search that that Charlie’s team’s done a really good job of is all the investment and preparation work that we did leading up to the launch. So all the market research that we gathered four years in advance of the launch, and really understanding the switching that was going on in the marketplace, even before we got approved, right, there were other new drugs that came into the market, then hiring the sales organization, you know, the six months before the launch, and getting them to get, you know, used to doing zoom calls with their new customers and, and getting, you know, prepared for whatever obstacles they face and how to navigate through that. And I think that served us very well.
Serg Belanger (analyst): And then, you know, the US markets, the prophylactic therapies are pretty well established in the USA market. So what has been the initial focus of the launch really, on new patients, or switching from existing products? Whereas What do you focus on at this point?
Charlier Gayer (CCO): Good question. Well, what I what I’ll say is, it’s really it’s both of those, and you’re right the market has, it’s great for patients, you know, about 60% of the patients are already on prophylaxis. And what we heard consistently from both doctors and patients in that market research was that page, a lot of these patients really wanted to reduce their burden of treatment, because the existing therapies are injectable and infused. And so, you know, a big portion of our opportunity is switching. And then the other thing that doctors told us is, with Oraladeyo out there, they see that 60% billing to 80% of their patients over time. And in the early days, what we’ve what we’ve reported is, you know, that’s exactly what’s happening, we’re getting, you know, about half the business from switches, and then the other half patients newly coming to prophylaxis. Now they can do it with a once a day pill.
Jon P Stonehouse (CEO): So it’s just see that just one clarifying point, it’s not newly diagnosed patients, those are not no fractions. These are patients that are either on demand therapy, acute treatment, or prophylactic, and they’re switching from either one.
Charlier Gayer (CCO): That’s right. It’s a very difficult concept. In contrast to Japan, this is a very developed market, you know, prophy is used to patients or patients are known that Japan strategies, going out finding the patients and then Introducing prophy for the first time, so it’s a great opportunity in all places,
Serg Belanger (analyst): for products for, for patients that have been on an injectable product for a while, is the willingness to switch just based on convenience, or, you know, just the willingness to try something new and that, you know, maybe helpful.
Charlier Gayer (CCO): Yeah, so, you know, I think convenience is a big part of it. But I think it goes way beyond convenience. It’s what I was saying that that is that real burden. So there’s a there’s preparation that goes into these injections, there’s limitation on travel, because some of these products have to be refrigerated. There’s the scheduling of those infusions, that it adds physical time burden and mental burden. There’s, of course, you know, the needle fatigue that many patients face, all of that is a big burden that is solved with for many patients with an oral, and so it’s those patients who are experiencing that burden. The those are the ones who really want to switch.
Jon P Stonehouse (CEO): And that’s another key piece to our strategy. The omegon in the MSL team has been out for a while. And we published last December, a number of posters around the burden of therapy. And it’s a paradigm shift, instead of just treating attacks, which is what the old paradigm was. Doc, you patient would talk to the doc and say, You know how well controlled are your attacks? How many attacks have you had now they have a broader conversation that’s around the burden of their therapy in addition to the burden of their disease, and having those conversations is really opening the eyes of both patients and physicians that there’s more to be gained here.
Serg Belanger (analyst): Okay. Um, maybe give us an update on reimbursements. You know, for for new products, at least in some other markets. Sometimes it takes up to a year to get kind of the full reimbursement coverage by by payers. Maybe How does it compare for Orladeyo and when do you expect to be at a kind of a steady state level.
Charlier Gayer (CCO): Yeah, very, very important part of the process. So we there’s kind of three parts to our strategy there. The first is, you know, when a patient and doctor have decided that oral The day was right for that patient, we want to get them started right away, regardless of the reimbursement. So we’ve got a very comprehensive quickstart program for patients so that everybody can get going right away. Well, then, the second part is we help the providers and the patients through the access process. In the early days, for many patients, what that means is medical exceptions, we’re having some success getting patients paid for a medical exemption. But what we’re trying to do there is show the payers that there’s a real demand from from patients, and that that kind of puts the pressure on them for the third part, which is getting to the formal policies for Orladeyo. So quite a number of plans have already put more Orladeyo on policy, we expect good acceleration of that, this quarter acute in q2, and by about mid year, we expect the great majority of payers to have coverage policies for for Orladeyo, there will be stragglers that go on throughout. But showing them this showing the payers the demand, and really, really helping the providers push on that is is paying off in early days.
Serg Belanger (analyst): And what kind of step throughs? Or priorities Do you expect, once these the these policies are implemented?
Charlier Gayer (CCO): So we expected that in the vast majority of cases, it’s going to be the prior off that they use for all prophy products. And so there’ll be equal access for prophy. But patients will still have to go we have there’s lab tests, your patient histories, usual stuff, but the providers are very familiar with that. And then our patient Services Program helps them through that process.
Serg Belanger (analyst): Okay. And as we talked before, we’re gonna see the first sales numbers soon. You haven’t given guidance, and obviously, that’s usually not expected and for an initial launch, but, you know, are you comfortable with the current street expectations for first year sales?
Jon P Stonehouse (CEO): I’ll take that one, Charlie. So, you know, what we’ve said repeatedly is our goal is to meet or beat the expectations. And so you’ll get the answer to that very soon
Serg Belanger (analyst): good. Um, I guess the overall he markets but a 2 million worldwide market, half of it is in the US. He said 60% or so was prophylactic? How do you think things change as a new product like oral adeo comes on the market?
Charlier Gayer (CCO): Yeah, so you know, what we expected the us what the doctors have told us and all signs point, this direction is getting more towards 80% of patients. Ha traders really believe that prophy is the way to go. And now that they have more and better options, that just that accelerates that process, what we’re really excited about in Europe, which will be kind of our next market that we were going to launch ourselves
is that, you know, historically prophy use has been very limited. But the doctors and the patients have kind of equal excitement about an oral once daily coming out. And so they’re expecting that prophy use is going to expand significantly in Europe. And then, you know, as Megan was talking about prophy use in Japan. So really, it’s it’s the way to go. And we see it, we see it growing worldwide.
Jon P Stonehouse (CEO): And I would add, you know, so we have the market research that Charlie talked about, but now we have real world experience. We’re seeing people switching from on demand therapy during the launch in the US. So this is no longer hypothetical. This is that market is shrinking. Right.
Serg Belanger (analyst): Yeah. And then, you know, we touched upon Europe a little bit. Once again, as a product been approved in Europe, and what are your current launch plans?
Jon P Stonehouse (CEO): So we’ve gotten the CHMP positive opinion. And we’re awaiting the European Commission final sign off. And you know, we’re expecting that soon. And I’ll let Charlie describe how how ready we are we made the same investments that I talked about in the US and Europe in advance, and I’ll let him describe it.
Charlier Gayer (CCO): Yeah, so that means the strategy searches, hire really great experience people in rare disease, get them ready, you know, be fully prepared from a commercial perspective to launch. So our first our first launch will be Germany, right after we get that AEC approval. And then you know, what we’re really excited about too is that we’ve got early access programs going in the UK, with the Ames program that we started last in q4 of last year. And then recently we announced the cohort at EU in France. So patients are starting to get access already in, in your in Europe today. And so we look forward to multiple country launches in the coming months and years.
Serg Belanger (analyst): Okay. You expect pricing to be, obviously, it’s not going to be at the same us level, but maybe something similar to what you were able to achieve in Japan.
Charlier Gayer (CCO): Yeah, I think you got it, it’s not going to be quite the same in European markets is always more limited. But it’s, it’s a great opportunity for us, because there, the thing about Europe is the patients tend to be diagnosed. And so they’re there. And so like I was saying that opportunity to grow the pro free market means it’s, you know, and we’re able to do it with really strong teams, but very efficient teams. Because the scent, the treatment centers, tend to be very concentrated, you know, sometimes hundreds of patients in one center. And so it’s going to be a really profitable opportunity for us.
Serg Belanger (analyst): Okay. Before we move on to pipeline assets, I think in the past, you’ve talked about peak sales potential for Orladeyo 500 million plus. You still reiterating that? That target?
Jon P Stonehouse (CEO): Yeah, make sure you remember the plus!
Serg Belanger (analyst): Okay. Will do. All right. So wanted to talk about the next development asset that you’ve been talking about 9930 factor d? can you just give us an overview of that, that asset and the mechanism of action?
Jon P Stonehouse (CEO): Bill, you want to take that? Sure.
Serg Belanger (analyst): Bill I didn’t forget about you, you were just on the other, the other page of my zoom screen
Bill Sheridan (CMO): won’t hold it against you. So that would be BCX9930 is really terrifically exciting project. This is a partner specific factor D inhibitor. So the mechanism of action is to block the enzymatic activity of that complement enzyme. That enzyme sits at the very start of the alternative pathway of complement. So that opens up a whole set of opportunities across different diseases that are driven by dysregulation of that alternative pathway of complement, and the amplification loop that’s embedded in that pathway. So our first experiment was in paroxysmal, nocturnal hemoglobinuria, we’ve presented serially the accumulating results in that study in our r&d day, on the 22nd of March, we had a patient advocate, and leading investigator who’s collaborating with us on the study, describe what patients need, and what physicians want for their patients. And we went over the results that we’ve generated today. And in summary, this is an anemia, disease, patients feel terrible, and they have complications from the meiosis. And so what patients want is to not feel terrible and not have transfusions. And what doctors want is to see the hemoglobin go up and the transfusions go down. That’s exactly what we’ve generated in our data. We’ve treated patients now for longer than a year in that study, and we’re skipping further dose ranging, we’ve completed dose ranging, they go straight to pivotal studies, starting later this year. So it’s a very exciting program.
Jon P Stonehouse (CEO): I’d add one one other thing is, you know, the market research that Charlie’s team has done so far, is really eye opening in this space, for a couple of reasons. One, while there are you know, great improvements with the injectable therapy or infused therapy, these patients can spend a half a day in the infusion center, getting their therapy, and that I mean, good god, that’s a bird right of treatment. And I think the other piece is the all these patients remember when they weren’t sick with PNH, right? So they remember life before PNH. And the idea of having twice day, twice a day oral drug to treat their disease that manages all the things that bill just described that we see in our study is a shot at having that life that they had before they got diagnosed with PNH.
Serg Belanger (analyst): So you have data now in treatment, naive PNH patients, as well as kind of inadequate responders so that people have inhibitors. So where do you see the product fitting in terms of, you know, the data you had so far?
Bill Sheridan (CMO): Sure. So at all, great question at that goal is monotherapy. So there’s really no fundamental reason why you would need to add a distal complement inhibitor, if you’re already using a proximal complement inhibitor, like BCX9930. And we’ve shown that very well, in the C five inhibitor, naive subjects getting treated with monotherapy already specked out really, it’s the spectacular improvements. In our C five inhibitor, inadequate response subjects, the first thing to do is to add a drug to their existing treatment, their existing treatment, by definition is inadequate. And again, the responses in those subjects were very, very good. So over the course of the next period in the study, we expect that the investigators will be able to withdraw the C five inhibitor, and we look forward to sometime later this year. Our plan is to you know, share the data from monotherapy in that group. But the the ultimate goal here, just to be really, really clear, is a broad label, single Agent monotherapy would be BCX99330. For every patient with PNH, whether they had a C5, and whether or not they’re doing well on it now or doing badly,
Jon P Stonehouse (CEO): I would underline everyone in bill statement, because it’s the people that don’t have access to a C five inhibitor or for some reason choose not to go on it, the people that are on it, but aren’t well controlled. And the people that are well controlled, that wants something more and or don’t want to the burden of therapy, right. And so they want that idea of a less of a burden with an oral treatment that we so we want everyone.
Serg Belanger (analyst): Okay. And as you go forward, the key endpoints here, really transfusion independence. Anything else that you’re really keyed on?
Bill Sheridan (CMO): So the regulator’s put a lot of trust in totally objective endpoints like increasing the hemoglobin in the absence of transfusions. So that’s really important. So they’re the two key things.
Serg Belanger (analyst): Got it. And then in terms of adverse events, I think the profile was, you know, it was well tolerated. There were a few incidences of rash. It doesn’t look like it was anything severe that led to discontinuations. But it always brings about some questions, so maybe just talk a little bit about that.
Bill Sheridan (CMO): Sure. So the safety profile that’s emerging with this drug so far has been really very satisfactory. We’ve had no discontinuations no drug interruptions. So that says a lot. And you know, with chronic treatment now, accumulating every week, in 15 subjects who continue on the study. The rash is a non event fundamentally, it’s, it’s an inconvenience, it happens it goes away by itself. Patients continue to take the drug twice a day, and it hasn’t been a problem. So so far, so good. Like with all complex inhibitors, we have to be very careful about protecting patients against infection with organisms like know Nisyria that was discovered with a colorism airborne. So we vaccinate make sure people are vaccinated, but the safety profile of this truck so far, and the benefit profile that’s emerging is strongly positive.
Serg Belanger (analyst): Then, this was a few weeks ago, you announced plans for a pivotal trial in in PNH. Have you had a chance to sit down with the FDA to kind of finalize the design of that trial? Or is that upcoming at this point?
Bill Sheridan (CMO): Sure. So last year, we had very productive interactions with US and European regulators around our preliminary thoughts on pivotal study designs and dose selection. And we’re now executing the completion of those regulatory interactions. We fully anticipate that that, you know, better battle come to pass in due course, and we’ll be able to start our pivotal programs in the second half of this year in PNH. I should add also that, you know, going back to the very start here on opportunity across many diseases, we also having regulatory interactions around a selected nephritis indications. And we plan to start a proof of concept study in those indications again in the second half of the year.
Jon P Stonehouse (CEO): And Bill won’t do this. So I’ll do it for him. I’ll brag on him and his team. You know, we were in a phase one study. And now you know, in the second half of this year, we’re going to be in a pivotal pivotal studies in PNH and proof of concept in renal diseases. So that’s warp speed. And that’s really going fast.
Serg Belanger (analyst): Okay. As you think of the development path for the PNH indication, so you know, what would a pivotal trial look like just in terms of size and maybe some of the the, the endpoints and what you need to do to conduct to them To satisfy FDA requirements for approval?
Bill Sheridan (CMO): So, you know, that’s a very interesting question. It’s a rare disease space, a serious and potentially life threatening disease, there’s definite unmet medical need, I don’t know whether one or more than one is required for approval. However, to give our commercial team the best opportunity to promote the drug across the board, for every patient with PNH, we may choose to do more than one study to support a broad level. In terms of size and design, we’ll go into that in more detail once we’ve completed the regulatory interactions and everything’s nailed down. But, you know, you can look at what’s happened with the other sponsors, and, you know, less than a few 100 patients is adequate, or has been adequate to, you know, get drugs approved and dispose. And it’s a very rare disease. So, you know, I think that you can expect something of that order of magnitude, durations have not been hugely long, right? So these studies can be done in terms of patient experience in a reasonable timeframe.
Serg Belanger (analyst): Okay. And since these trials, like you said, are, aren’t overly large. There’s no, you know, need to really partner this product at this point. company is able to move it forward on its own.
Bill Sheridan (CMO): You want me to take that, john? Yeah, sure. This is an asset that we want it all permanently. So that means Run, run the clinical program, launch the drug, and keep, you know, maximize the value in the future, the way that value is maximized. You know, it can take different forms in different regions, obviously, but the goal here is to maximize the value for patients and therefore for the company. Now, we can certainly run the clinical programs without partnering.
Jon P Stonehouse (CEO): Yet, it’s all about scale search. And, you know, we’ve learned a lot from the HAE program. And so it’s a matter of, can we do multiple indications in parallel, right, the P and H programs, it’s different from HAE, but it’s also very similar in certain ways, in terms of size and scope. And so it’s a matter of scale. And the financing that Anthony did at the tail end of last year, gives us the opportunity to hang on to the rights. And so we’ve got the capital, and we’re you know, it’s one of these things, we’re just gonna keep repeating what we were doing really well at which is developed discovering these drugs, developing them getting them approved, and then commercializing them. And that creates massive value.
Serg Belanger (analyst): Yeah. And, like Bill said, this is really looks like a kind of a pipeline and a molecule. opportunity here. You talked about the renal complement mediated diseases. You know, maybe why did you choose that indication, as as the first next one, when there’s a pretty open green field of other opportunities.
Bill Sheridan (CMO): It’s very exciting. So that our approach is to is to rank indications by how well validated the alternative pathway is as a pathologic driver of the disease. And so the reason for doing that is to minimize efficacy, failure risk in the program, right. So if we if we have abundant evidence, that dysregulation of the alternative pathways driving the disease, and our goal is to dose to an adequate safe dose to block, factor D and block the alternative pathway and expectations. You know, if you want to be a Bayesian statistician, your Bayesian prior is really very strong here in terms of getting efficacy and minimizing the risk of efficacy failure. So that’s the, that’s our approach. I think, over time, it’s likely that we’ll be able to take a bit more risk on that front. There are other diseases that might start off as proven by the classical or lectin pathway, but we know for various experiments with the alternative pathway accounts for about 80% of the activity of those other pathways. So, you know, it might be useful to investigate those other diseases at the right time.
Serg Belanger (analyst): Right. And then wanted to spend a little bit of time on on the other asset, probably the one that gets less attention, mostly because, you know, we’ve gotten a lot, a lot more news on 9930 lately, but the 9250 molecule, and you know, we’ve, we’ve seen some data was it earlier this year, like late last year? So maybe just give us an overview and like, what you’re, what you’re thinking about bringing that program forward.
Bill Sheridan (CMO): So now 250s, an ALC, two kinase inhibitor ALC. Two is the enzyme that’s mutated in a very particular way, in every individual who suffers from this horrible disease. And there’s a huge unmet medical need, there are no approved treatments, that progress of the disease is awful to see. And typical patients are crippled by the time they’re in adolescent years. So it’s very unfortunate that the programs that were ahead of us have met with huge obstacles. That’s not a good outcome for FOP community. So our data last year was a phase one single ascending dose and multiple ascending dose experiment in healthy subjects. And we’re very, very pleased with the exposures we got. There were no safety issues at all. And we were able to achieve exposures with once daily dosing that look like that they are similar to exposures in rodent models, where we force this new bone formation to occur heterotopic ossification. So we could inhibit that at similar drug levels that we achieved in people, we now need to make a lot of drug and start chronic animal safety experiments in order to support progression in the clinical program. So that’s the focus this year, pretty clearly this asset should be brought forward.
Jon P Stonehouse (CEO): Yeah, I think the bottom line is, there’s a huge unmet medical need. And yeah, this, this program was on a slower track. But the data was really impressive that we got at the end of the year. And so now that we have the capital, we’re going to speed the program up. And as bill says, it’s all about making drug getting the tox so that we can be in a position to start clinical trials.
Serg Belanger (analyst): Okay, so maybe the next clinical trial gets underway in 2022.
Jon P Stonehouse (CEO): Probably probably.
Serg Belanger (analyst): Alright. And then just to end, maybe have Anthony talk a little bit about financials, the current cash balance, and I don’t think you’ve provided guidance for for this year in terms of capex, they maybe just talk about their cash runway. And what’s embedded in that?
Anthony Doyle (CFO): Yeah, sure. So, from a financial position, we’re in a really strong position. So like john talked about the financing that we did in December, put us in a great spot, we ended the year with about $303 million in cash access to another 75 upon meeting certain milestones for Orladeyo. I think what it does is allows us to focus on executing on our strategy, I think it gives us a lot of freedom to invest in areas that are going to drive a lot of value creation. So specifically investing in the Orladeyo area launch globally, investing, like Bill had talked about in 9930, across multiple indications and then investing in 9250. And yeah, during the launch period, we’ve said that we won’t provide guidance. But what we have said is from a cash runway perspective, the cash on hand that we have, in addition to the the available options, through the additional financing that we did in December, gets us into 2023. So, you know, revenue, being able to generate revenue in the us being able to generate revenue now in Japan and hopefully soon in Europe. That, in addition to our cash bonds puts us in a really great spot.
Serg Belanger (analyst): Well, I think we’re up on our time here. So want to thank all of you. It was great seeing you on my computer screen here. Hopefully we can meet in person soon. Thanks for the great overview this morning.
Jon P Stonehouse (CEO): Yeah, thank you Serg