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JMP Webcast Transcript
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Jon Wolleben (JMP Analyst): Good afternoon, everybody. JOHN Wolleben here. I’m a senior analyst at JMP securities and welcome to day two of our annual Life Sciences conference. I’m pleased to have the team from biocyrst Pharmaceuticals joining us here for a fireside chat. And we got the whole team here today. So I’d like to welcome Jon Stonehouse (CEO), Anthony Doyle (CFO), Bill Sheridan (CMO), Charlie Gayer (CCO), and chief communication officer, Officer, John Bluth. Thanks, everybody, for coming and joining us today
Jon Stonehouse (CEO): Thanks for having us, Jon.
Jon Wolleben (JMP Analyst): And I think, for people dialed in just as a piece of housekeeping, if you have any questions you’d like me to ask on your behalf, feel free to enter in via the chat and I’ll pepper them to the team, as we have our discussion today. But maybe to start, john, for those who may not be as familiar with the biochar story, it’s been a heck of a 12 months for the team. I’m hoping you could tell us a little bit about what you’re working on and your overall strategy, and then we could dig in to dig into the details.
Jon Stonehouse (CEO): Sure. So let me let me start by saying we’re all going to be making forward looking statements and those statements at risks and risk factors can be found on our website. But john, we’re thrilled to be here with you. And you’re exactly right, the company has really transformed in the last 12 months. And for those that aren’t familiar with that company, you know, the goal here is that we’re building a great biotech company, one that brings oral drugs to patients who are suffering from rare diseases, and we are well on our way to building this great company. And the evidence of that is we’ve got our first oral drug for a rare disease launched, and the launch is going great that, you know, we have the first quarter results, where we tripled your wall street expectations and, you know, continue to see great progress on that front, and Charlie will be giving you more color on that, but we couldn’t be more excited. And the reason is that it’s a drug that patients really want and they’ve been doing well on. And we have expectations that this drug could be at global peak sales north of $500 million. So we’re really excited to have that as kind of the anchor of our company. But then beyond that, we’ve got a full pipeline with BCX9930. And it’s a pipeline and a molecule factor D inhibitor for complement mediated rare diseases. And we’re moving into pivotal trials in PNH and and proof of concept in nephritis indications and it almost instantly fills up our pipeline. And then we have now two inhibitor 9250 that we completed phase one. And then there’s more stuff, the great news is Babu and his team in Birmingham, Alabama continue to be efficient and effective in coming up with new discoveries. And so will continue to fill the pipeline. And then the last piece. And this is a big transformation for us as well as financially, we’re in such a solid spot compared to previous years with revenue coming in from Orladeyo, and a number of different options to bring additional capital, we couldn’t be in a more solid financial situation. So you put all those ingredients together, and you have the makings of a really great biotech company.
Jon Wolleben (JMP Analyst): It’s a great overview. And I think we should start with Orlando. That’s what everyone wants to talk and learn more about. And you mentioned early launch is going well, I was quite optimistic. And you guys did better than even I thought in the first quarter. Can you tell us kind of what’s going well, what have you learned? And where can you do better in these early days?
Jon Stonehouse (CEO): Yeah, Charlie, I’ll start and then I’ll pitch it to you. I think the big headline is it’s a switch market. And we you know, Charlie, and his team told you that two years ago at the market research, we saw it in the people that came into our clinical trial. And guess what, that’s what’s happening in the marketplace. And we’re having a great result in and I’ll Pat Charlie and his team on the back. I mean, and they’re doing it in COVID getting these fantastic results. So you know, just imagine what we could do when we get out of COVID. And with that, Charlie, I’ll pass it to you to give a little bit more color.
Charlie Gayer (CCO): Sure. Thanks, John. So as John Stonehouse mentioned, it is a switch market and we expected that that’s that’s exactly what we’re seeing. So, you know, in the first quarter, what we saw was more than half the patients switching from injectable prophylaxis products and then the rest of the patients switching over from acute only now that they have an option to treat with an oral once daily prophylaxis product. So so that’s going really well, and we’re seeing good consistent demands, you know, as we expand as we hear back from doctors, the intent to prescribe is really high. You know, amongst our target physicians, only the minority have had a chance to prescribe so far but the intent across the vast majority of them is is really high and so you know, we’re just getting started.
You know, as Jon mentioned, we were doing this in COVID. And I think one of the things is you talked about what could go better. Being able to visit our customers live is really going to make a difference. You know, building those deeper relationships with the doctors and the rest of the staff is important. And we haven’t had a chance yet to do live patient programs. But that’s, that’s getting underway now, too. And that’s gonna be really important to just continue the excitement from the patient side.
Jon Wolleben (JMP Analyst): You mentioned the switch market. And obviously, we’ve seen your market research, and it’s very consistent with what we’ve done with our due diligence that people really want an oral prophylactic. Is there any other common characteristic you’re seeing in patients who have switched? Or is it too early or not enough patients to get kind of big trends as a patient who still have breakthrough attacks on the prophylactic or on a specific, prophylactic injectable? Kind of any learnings there on what kind of patients are coming over?
Charlie Gayer (CCO): You know, it’s a it’s a good question, john. But honestly, no, the consistent thing is they have HAE, you know, more of the patients are coming from from prophy. That’s, that’s what we saw on our clinical trials as well. There’s there’s no phenotype in terms of where does Orladeyo work really well. And there’s no typical patient. It’s just like the it looks like the market as far as people who are switching their their switching from prophy in proportion to their market share. So the number one prophy switching is coming from Takhyzro. It’s kind of Tak, then Haygarda then Synrise. So like I said, it’s, it’s, it’s going well, and it’s what we expected.
Jon Wolleben (JMP Analyst): And you mentioned your target physicians, what is the target profile? The docs are going after? First? How many are there? And you mentioned a minority of eprescribe? Have you said, what percentage of the habit have been prescribing so far?
Charlie Gayer (CCO): We haven’t, we haven’t specified the percentage. But what I have said is that there are about 500, doctors, mostly allergist immunologists, who we know from our targeting treat a 50% of the patients in the US. So that’s our number one focus. And that’s where we’re having great uptake there. But we still have plenty of runway to go. And then we’re also having uptake deeper in our target list as well from from doctors who might only treat two or three HAE patients, but they’re they’re starting to prescribe Orladeyo as well.
Jon Wolleben (JMP Analyst): Can you give us an update on how reimbursement coverage is going and the availability and the use of your patient access program? Because I know how important that is to their early lives. So can you give us some details in color there?
Charlie Gayer (CCO): Yeah, the reimbursement processes is going well, the payers are receiving Orladeyo favorably. And I think a lot of that is because they’re seeing the demand coming in from patients. So in the first quarter, the majority of reimbursement that we got was through medical exceptions. But that puts pressure on the payers, it’s it’s work for them, cost them money in time and effort. And that brings them to the policy making table sooner. So what we expect is by the middle of the year, the majority, the great majority of patients will have access to Orladeyo coverage via policy through their planner or PBM.
And as far as you talked about the the Access Program, part of our strategy was making sure that we could get patients on drug really quickly. So as soon as that start form comes in, usually within a day or a couple of days, we get a quick start a month of free therapy out to the patient so they can start experiencing Orladeyo while we go through the access process. And we really want to take that burden off of patients so that they don’t have to worry about will the payer cover, they can just focus on the the experience with the drug.
Jon Wolleben (JMP Analyst): Any feedback so far on kind of persistence and adherence or are patients are getting on multiple months of dosing now so what does that look like so far?
Charlie Gayer (CCO): It’s looking good. What we expected based on our Apex to clinical trial, we saw 75% of patients staying on for at least a year. So that was our target. And through six plus months. What we’re seeing is the trends are, are well within our expectations. So patients are staying on. They’re having a good experience with the drug and we expect that to continue.
Jon Wolleben (JMP Analyst): Fantastic. And we’ve got some positive developments across the pond in the EU the past couple months. Can you talk to us a little bit about how you view that opportunity, the versus the US? Are there learnings between what’s gone well in the US that you can use or is it a completely different ballgame over there? How should we think about that opportunity
Charlie Gayer (CCO): there absolutely are learnings it’s a it’s a little bit of a different playing field to continue that analogy. So in Europe, the the market is mature, but it’s less of to date. It’s been less of a prophylaxis market. So us is about 60% prophylaxis, Europe is 30% or less. So the opportunity is to grow there. The the feedback from patients and physicians though is they want prophylaxis, they’re starting to get access to modern prophy therapies. And an oral once daily is, is really what they want. So there’s a lot of enthusiasm.
Same as in us, we hired really great teams really experienced with rare disease launches. And so our launches underway in in Germany. And then we’ll, we’ll expect to have further launches in the coming months and into next year and the UK and France and the rest of rest of Europe.
Jon Wolleben (JMP Analyst): And, john, when you when you talk about a $500 million product that is that worldwide sales, I’m wondering if you’ve given out kind of what you think that geographic split is.
Jon Stonehouse (CEO): Yeah, it’s global sales. And remember, it’s $500 million plus, so don’t don’t forget the plus. And yeah, you know, it’s gonna mostly be US. But the way we look at it, we’re bringing this drug to every country around the world, we’re going around the globe, and every country that contributes five or $10 million dollars, you know, when you add that stuff up, it really is meaningful. So we’re really excited about the progress we’re making moving this drug around the world.
Jon Wolleben (JMP Analyst): It is early days, it’ll probably take a couple quarters to get a sense of trends. But when when do you think you might be able to provide guidance to the street? And maybe at the top line? Or if not, so what metrics might be helpful if it’s start forms, or prescribing Doc’s something along those lines? And I think I looked this morning consensus for the year seems to be about 58 million, we’d love to kind of see how you feel about that range as of today.
Jon Stonehouse (CEO): Yeah, you know, we’ll give guidance when we feel we have enough information that we that the trends are clear to us, and that we can give you accurate guidance. We don’t want to guide when we’re still not clear on what the trends are. So after a quarter, that’s pretty tough. Right? I think, you know, it’s likely over the course of the year that we’ll be in a position where we’ll have a better sense of the trends. And when we do we’ll, we’ll put out that guidance. But in the meantime, I mean, you said after we put out the first quarter results, you know, the consensus doubled. And our view is that, you know, we our goal is to meet or beat that even though it doubled and so we’re real confident that we’ll be able to do that.
Jon Wolleben (JMP Analyst): Given how well things have gone, are you gonna need additional investment to meet demand? Is the sales organization right sized or if COVID lightens up, and people are getting out and seeing more physicians? Are you thinking there might be additional investment? Kind of on the sales organization?
Jon Stonehouse (CEO): Charlie, you can you can take that one.
Charlie Gayer (CCO): Yeah. So no, I think that our sales organization we did a lot of targeting work to in building that team. And, you know, I mentioned the 500 doctors that treat 50%. And so I think we’ve got we’ve got a great team and we’ve got the right sighs team. I think we’re we’re constantly looking to invest more. It’s just you know, what’s working really well. And you know, are there certain programs we can do more of so we’ll, at the margins, we’ll we’ll definitely we’re very data focused, analytically focused. And so we’re always going to look for what what can we do that’s more, but there’s not a massive investment of, you know, building the team or anything that we need anytime soon.
Jon Wolleben (JMP Analyst): It’s to your earlier intro, john, it’s not just Orladeyo for you guys. 9930 is a really interesting asset. You’re all effector D inhibitor. We saw compelling data for PNH earlier. And I was hoping you could give us a rundown on why this is an exciting opportunity for you guys.
Jon Stonehouse (CEO): So I’ll start bill and then you may want to jump in as well. You know, it’s a really interesting target number one, and, you know, like, plasma kalakrine is a serine protease, factor D is a serine protease, but the difference here is that it applies to many, many different diseases. So it’s like an entire pipeline in a molecule. And so one of the things that bill and his team and others have been doing is starting to identify beyond PNH because now we’re moving into pivotal trials, what other indications Can we go into, and being in that stronger financial position I talked about, you know, we have the resources to be able to, you know, go into other indications, and I’ll let bill talk about what’s interesting to us.
Bill Sheridan (CMO): Thanks, John, this is such an exciting opportunity to help patients with a whole variety of different diseases that are driven by upset or dysregulation of the alternative pathway of complement. You know, the research from academia over the last few decades has been fantastically productive. And in one of our slides in our standard corporate deck, you know, we’ve got six or seven indications that have very good evidence of involvement of that pathway in driving the pathology of the disease, and that spans hematology, nephrology, rheumatology. So this is I think it’s a really big deal in medicine. those diseases are uncommon to rare. And by and large, they don’t have any licensed effective therapies right now, some do, but most of them don’t. Some of them affect children in a big way, and can lead to end stage renal disease and the need for kidney transplants. So the medical need here is huge. And scientific validation is extremely strong. So we look forward to having a very broad program across a whole variety of different indications for Factor D the inhibitor program at biocryst.
Jon Wolleben (JMP Analyst): And when we take a look at the PNH data you presented already, we’ve seen data and treatment naive patients, as well as those who are inadequately controlled on C five inhibitors. I thought it was pretty compelling data set. And throughout the year, you mentioned that you you’re going to start weaning off patients from the C five inhibitors. So they’re going to be on 9930, mono therapy, can you give us an update on where you are on that front?
Bill Sheridan (CMO): Yeah, the physicians will make that decision as the principal investigators at the sites, we’ll make that decision as things progress in the year, we didn’t need to wait for any of that data. The monotherapy data in PNH, subjects who were not taking c five inhibitors was so strong, that the regulatory agencies agreed that we can move straight into pivotal studies in both c five inhibitor inadequate response patients, as well as C five inhibitor naive patients. And you know, the fact that the target is the target, it’s the same target in both in both patient populations. And you know, the beautiful thing about proximal complement inhibition is that its effects are predictable. And that we can deal with the opsonization, that happens as a result of C five inhibition in those people who have inadequate response. So we’re not waiting for that. You know, that everything’s well advanced. We’re in implementation mode, and doing all of the things required to start those studies in the second half.
Jon Wolleben (JMP Analyst): I’m just excited to see it, I think it’d be particularly interesting. When we, when we look down the road to ASH, later this year, we’re going to see some more data from the ongoing study, maybe longer follow up, or what could be the next kind of data cut, because obviously, you’re gonna be kicking off your pivotal trials, it will be a little bit of time from data from there. So yeah, what additional data might we see from 9930 Between now and then?
Bill Sheridan (CMO): certainly consider it. You know, I think that big focus right now is implementing the pivotal studies. You know, I think that once all of the i’s are dotted and the T’s crossed, you know, we’ll find an opportunity to go into the design of those studies in a great in a lot of details. And, you know, we might also take the opportunity to do that at an academic conference as well, because there’s such a lot of interest in, you know, study designs in this field.
Jon Wolleben (JMP Analyst): You’ve given some details, correct. As far as dosing and endpoints and patient populations, what can you tell us today about the pivotal?
Bill Sheridan (CMO): Well, I think that the regulatory discussions were very productive. The primary endpoint of both studies is change from baseline and hemoglobin, showing that there’s a reduction in transfusion requirements is a key secondary endpoint. We’re measuring the relevant biomarkers. But you know, the, the bio, and the surrogate biomarkers in this disease are not as important as correcting the anemia and relieving the transfusion burden. So they’re the two most important things that study designs are straightforward. There’s a head to head comparison with C five inhibitors in the patient population that has an inadequate response to C five inhibitors. That’s one of the studies. And the other study is a placebo controlled trial in patients not taking c five inhibitors. And again, it’s a head to head comparison of 9930 with placebo in that case, you know, you can expect that the duration of these studies will be similar, in general to the duration of other studies in the field. And, you know, our goal is to offer BCX993o to every patient in those studies, who wants to continue, you know, beyond the study duration. So, that’s exactly what we did in the viral step program and, and the berotralstat step program. So now we have people on all the day all around the world, waiting for commercial drug in those countries, which is really good for a generation of long term safety data as well. So we’ll do the same thing you know, with the BCX9930 program,
Jon Wolleben (JMP Analyst): how much more difficult is it to enroll and run the treatment naive PNH study versus the inadequate responders? And what is timing look like between these two do you think?
Bill Sheridan (CMO): I think it’s a really hard thing to handicap You would think, you know that offering a placebo controlled study would be harder. I’m not so sure that that’s true. And we don’t know yet. So once we, you know, once we get cranking with site activations and see how the enrollment actually is going, then we’ll be in a better position to, you know, understand that overall duration of those studies for enrollment. But I’m actually quite optimistic that, you know, we’ll be able to recruit the study. So obviously, there’s competition. But you know, our, our objective is to become the sponsor of choice and do a really great job listening to the principal investigators at the sites and patient advocacy groups about where to go and what the need is and generating enthusiasm.
Jon Wolleben (JMP Analyst): And you mentioned the pipeline in a molecule you plan on starting, I think, a couple different proof of concept studies later this year as well In the renal indications. I don’t think you’re gonna tell us today what those are. But maybe can you tell us how you think about these different opportunities? I’m guessing it’s some balance between opportunity competitive landscape, and maybe biology and regulatory risk. But at a high level, there’s a lot of things you could go after what what makes an attractive opportunity strategy wise?
Bill Sheridan (CMO): Well, I think you nailed that, I would add one more thing, which is the opportunity to really understand the effects of the drug in those indications, in an incredibly thorough way. And so you know, selected naphryatis syndications is where we’re going, and we’ll talk more about that in the second half. But you know, it’s going to be an incredibly thorough evaluation of the biomarkers, complement biomarkers, disease, biomarkers, kidney biopsies, and the like. So that we can get a really solid handle on what the next step is in each of those indications that we’re looking at. So I think that’s going to be very exciting.
Jon Wolleben (JMP Analyst): And any update, or can you give us a reminder of the current status of 9250 your op two inhibitor for fop.
Bill Sheridan (CMO): Sure, john, you want me to take that? Yeah. So we were very pleased with the phase one study results in humans, I mean, you always have to be very cautious putting a kinase inhibitor into first in human studies of it there. In fact, were no significant safety findings at all. And we were able to show linear and dose proportional exposure in the dose range we studied. You know, we’re now in the process of scaling up manufacturer to progress that program, and continuing with longer term toxicology studies to support evaluations in patients with fop, the medical need, there remains extreme with no approved products. And, you know, we look forward to getting to the next step.
Jon Wolleben (JMP Analyst): And I’m getting a lot of inbound questions here on there’s some news today that the government wants to invest more money into COVID antivirals. We haven’t talked about Gallaudet severe in quite some time. I’m wondering if you’ve had any interactions or any updates there?
Jon Stonehouse (CEO): Yeah, I don’t think there’s a whole lot to update, you know, we continue to talk to niad and other parts of the government around, you know, the need for broad spectrum antivirals. And you know, we’re continuing to move forward on the Marburg animal rule path. But you know, I think an important piece is we collected some clinical data in this COVID outbreak, and there’s still a huge unmet need. I believe, you know, that we make great progress on the vaccine front, which is fantastic. But the world still needs more antivirals.
Jon Wolleben (JMP Analyst): Yeah, I agree. And then I guess, in the last couple of minutes, we have, Can you remind us of your current cash position, and kind of where that gets you runway wise? And then I know, everyone’s gonna be focused on the Orladeyo numbers each quarter. But john, if you could also lay out the key catalysts for the company over the next year, year and a half, maybe to get us through 2022?
Jon Stonehouse (CEO): Anthony you want to take the first part, and then I’ll wrap it up.
Anthony Doyle (CFO): Yeah, yeah. So john, john stone has talked about the strong financial position that we have. So we ended the quarter one was about 244 million in cash, that cash and then in addition, we have access to about 75 million for to draw down with financing we did with a theorem late last year. And then revenue, you know, revenue coming in from from Orladeyo, which is a huge deal for us. So all of that gives us cash runway into 2023.
Jon Stonehouse (CEO): And then, in terms of the catalyst, John, you know, later this year and into next year, it’s all about the launch and the revenue, right? Each quarter. Our intention is to show everyone that this is a great drug, and it’s got a great trajectory. And we’re going to do that by showing revenue, quarter by quarter and then As I said before, we’re going to continue to look at countries around the world where we can bring this drug to market. And so filings and approvals. And then lastly, the progress on our pipeline in a molecule with 9930. Getting the pivotal studies up and running, getting the proof of concept studies up and running and the nephritis indications. So there’s a lot going on in this company. It’s incredibly exciting.
Jon Wolleben (JMP Analyst): I agree and congrats on all the progress. The success has been unprecedented so far this year. So I’m looking forward to tracking all the progress. And I wish everybody well on the line at the team at BioCryst, thanks for joining us today.
Jon Stonehouse (CEO): Thanks for having us, john