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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
- For our most recent research report, click here
- For a link to slides from the Cowen conference call, click here
Cowen 41st Annual Healthcare Conference
Ken Cacciatore: Thanks everyone for joining us for the biocryst fireside chat. We’re real happy to have john john and Bill joining us.
We at Cowen just picked up coverage on Monday, and with an outperform a much higher price target than it is today. We think there’s a lot of room here for value creation amongst the team. So we’ll get into some of that commentary before I turn it over to john, one comment I wanted to make is, in our initiation, if you look at our survey work, no pressure on the team here, but the consultants talk about ultimately, potential 50% penetration of the market an oral so real enthusiasm from our consultants. And then obviously, we have conversations outside of just doing the survey work with our consultants. And really what we love about the way they’re thinking about is the beauty is in the simplicity, why not try the world? We have so such fantastic injectable products as well. But why wouldn’t we try? And why wouldn’t we see if we have success and you you really get a situation as you kind of think through it. Sometimes when when we look at stories like this, what seems very obvious can be true. And so that’s at least our belief, your account. And I’m taking a little bit of the thunder away from the team. But letting the folks know that we couldn’t be more enthusiastic accounts. So with that as a backdrop, when I turn over to john for to run through some slides, and maybe set up a little bit broader perspective before we get going.
Jon Stonehouse: Well, thank you, Ken, with that kind of intro, I think we’re done. But
But anyway, thank you so much for that nice introduction and for inviting us to this year’s con healthcare conference. It’s been a while honestly, since we’ve been invited to Cowen. And so it’s great to be back and and really excited with the fact that can you and your team are are covering us as well. Next slide.
So we’re all going to be making some forward looking statements, those statements have risks, and the risk factors can be found on our website.
So I’m just going to go through a few slides to talk about where we are or where we’re going and where we are. And so let me start with our strategy. And what makes us different. It’s summarized in a pretty simple statement on a bit of a play on words delivering extraordinary by empowering ordinary and what we mean by that is, we go after really difficult targets to make medicines or making
Drugs, they’re validated targets. So we take the risk away by going after only validated targets. But these targets are often
enzymes that are in big families like kinases and serine proteases. And so what’s important is that you’re able to both make it a potent inhibitor and then bind tightly to the enzyme, but also be specific to that family, that member of the family and not other members of the family. And we’ve perfected structure based drug design for over the last almost three decades. And we’ve gotten really good at it. And so we’ve now been able to focus it on bringing forward oral drugs to patients that suffer from rare disease. And that’s where empowering the ordinary comes in. This isn’t some small, incremental convenience improvement. This is enabling patients not only to control their disease, but remove the burden of therapy off of them as well, so that they’re able to do ordinary things in life that the rest of us take for granted, but are life changing for these patients. So this is our strategy. And that’s our focus. And that’s what we aim to deliver. Next slide.
So the result is that we have a really full pipeline. If you had looked at our company a few years ago, this chart would look a lot different. And what’s even more interesting is this pipeline chart is going to change. And and it’ll continue to get more full and more interesting. And so you see at the top with Orlando, we have approvals in the US and Japan, we have filed in Europe and the UK, we’re expecting to get an answer in Europe in the second quarter. So we’ve got a marketed product. And that’s extremely exciting. And our first foray into bringing an oral drug into a space for patients with a rare disease. But behind that is equally as interesting. And our oral factor D inhibitor BCX9930. You see two lines for both P and H and renal diseases. But over time, this chart will change and you’ll see many arrows for many different indications. Because a factor D inhibitor can be applied to a number of different complement mediated diseases. And then after that, we’ve got b 692 50. And out to inhibitor for fop and our discovery team continues to work on other interesting and validated targets to bring more new discoveries forward and ultimately, into the clinic. So this is an exciting pipeline chart to look at, but it’s going to get even better. Next slide. Yeah.
And so the last point that I’d make, before we get into the q&a is, you know, if you’re one of the things that we hear from some investors is, you know, I knew biocryst a decade ago, I knew you guys, I was following you guys three years ago. Well, what I can tell you is, this is a very different company. And we are transforming at a very rapid pace. It started basically, the fourth quarter of last year with the approval of Orlando, additional financing more data readouts. But it’s going to continue throughout this year as well. We got the Japanese approval in January, we’ve got a really exciting r&d day focused on P and H, and data that will read out from 9930. On March 22, we’ll report out our first quarter sales in the earnings call for the first quarter. And then we’ll be reporting sales at each following earnings session. And then a number of additional launches and and and approvals. And so this is a very different company than the biocryst of a couple of years ago. And it’s going to get even more interesting as the year progresses. So that’s the high level overview. Ken I think we’re ready for your questions?
Ken Cacciatore: Yeah, that’s great. Thanks, john, for that, and I start the conversation, making everything seem so simple. But obviously, when it comes to execution, and drug launches, you know, nothing is such right. There’s usually a lot of interesting complications, we like to call it the plumbing in the system. And, you know, not all companies approach launches the same way. Not all launches are built the same way. And in this case, I guess it would be interesting to take a little bit of time to talk about how you’ve prepped for a launch of a product like this, we often find from a clinician who wants to use it to a patient who eventually takes it. There’s some complications in between. and in a orphan launch like this, and obviously an expensive one. Oftentimes, a little bit of things get get lost in the mix. So can you talk about the way in which you’ve created for this launch, you talk about the individual nature of how you’re approaching, dealing with clinicians, and then dealing with patients, helping them through the through the process, we can understand the the oil that you’re putting in the system to to ensure a good launch?
Jon Stonehouse: That’s a really interesting question. Because one could say that, you know, it started shortly before we got approval, but it goes much further back, we made investments in this space, goodness, going back into 2010. And really one of the top priorities was to get to build relationships and understanding with physicians and patients. And so since 2013, we’ve been going to patient summits in the US and, and overseas. And, you know, Bill, and I could spend the whole half hour just telling you stories that patients have shared with us about their desire for a more normal life and wanting an oral drug. But the investment continued, in really developing relationships with the clinical sites and the key opinion leaders, and that started with Bill and his team, and then was passed over to our medical affairs team, about a year and a half ago. And and I would argue that we have one of the best relationships of any company in this space, with both the patient community and with physicians. And then we made investments in advance of the menoufia date. That was that some companies don’t do, right, they, they, they say I will wait until it’s certain, and then we’ll make offers to our sales force, we didn’t do that we made offers to our sales force back last summer, to get them trained and ready, especially in this COVID world where, you know, promotion is very different than it is when we weren’t in a pandemic, and you made significant investment in them. And then they would get around their territories, get to meet their customers, you know, adjust things, you know, because they can’t be face to face or figuring out where they can be face to face. And, and, and I think it paid off in us being able to hit the ground running. And then the last thing I’d say is, you know, we we’ve been spending probably the better part of five years trying to gather insights into how patients are switching from one medicine to the other. And we invested a lot of money in, in market research, really trying to get inside the heads of both physicians and patients on what makes a good switch, what makes a bad switch? Right. And, and I think, as a result, you know, we we again, we hit the ground running way faster. And as Charlie said, in our earnings call last week, you know, there was some real pent up demand. And, and we’re trying to capitalize on that.
Ken Cacciatore: So I know, I’m gonna make you chuckle a little bit when I say this, but on a product like this, and the the number of patients that are out there, and the number of clinicians who are dealing with these patients, you know, we’ve seen this happen before, we’re literally the inability to have one to one interactions, to be able to walk a patient through the managed care be able to help the clinician through the process. And I always joke around with a patient size like this, does everyone have the CEOs phone number, if there’s if there’s a problem, if there is something happening to what degree is the sensitivity within the organization, so not looking to harp on it too much, or to know to what degree your phone number is out there for folks to be able to access you, but to just ensure to the investor base that the level of kind of individual care that both patients and clinicians are getting during a launch like this.
Jon Stonehouse: It’s really high, I mean, and it goes all the way to the top in the company. I mean, we all treat this, like every single patient is precious to us. And so yeah, you’re right, there’s a lot of things that could go wrong. And and no, no launch is perfect. You know, we’ve hit some speed bumps along the way. But in general, it’s going extremely well. And I think, you know, this is the beauty of being a small company, too is how quickly do you pivot? When you do run into a speed bump? How quickly do you adjust? And and I’ve been really impressed with the team. And and I haven’t been able to or haven’t been needed to be pulled into too many things, but I have been pulled into a few. And, and it’s helpful when you’re a small company and patients know me physicians know me. I mean, I had a doc, we were in a call with a doc last Friday, and he ended it by saying, you know, I’ve never met the CEO of a company before. And so we take great pride in that.
Ken Cacciatore: Yeah, it’s, it’s good to hear. Obviously keeping you keeping the system running as smoothly as possible early launch you frame out for us, obviously there’s a price and the competitors are priced as well. You talk about maybe how we should think of as we think of net value per patient going forward how you’ve tried to frame that for investors,
Jon Stonehouse: yeah, so the list prices in the public domain $485,000 per patient per year. But we’re the lowest of the prophylactic therapies and quite a bit lower than not most expensive. And so the way we haven’t given any guidance on gross to net, but here’s how you should think about it. So first off, it’ll probably be the highest in our first year. And for these reasons, first off, one of the things that’s really important in our strategy is fill the funnel with as many patients as possible. And then as quickly as possible, get drug tool, when a doctor and a patient make a decision that they want this patient to go on our drug, we want to capitalize on that and get the medicine to them quickly. So we have a program called quickstart, where they immediately get free drug, and then we work with them through the process of getting their insurance coverage along the way, so that free goods upfront effects, some of the gross to net, and then there’s not a ton of, of rebaiting and negotiating in this space, but there’s some. And so that has some effect as well. And then we also are assuming that with COVID, and the number of people that are laid off, that there’s going to be more people that don’t have insurance. And so that’ll affect the gross to net to so Charlie’s advice to people who have asked is, you know, take a more conservative view on gross to net in this first year, and then it’ll probably level out to what would be a normal range for a rare disease drug.
Ken Cacciatore: As we think about and obviously we’re going to be getting the real metrics when you report the the numbers in q1. But can you talk about other metrics that you’re looking at? Do you have enrollment forms? Or is there some level of patient engagement that gives you a leading indicator? and to what degree are we going to be turning on, you know, more direct to the patient promotional effort, obviously a small population that they’re they’re captured in smaller groups, but can you just talk about that metrics and an engagement that you’re already seeing a pre even prescribing?
Jon Stonehouse: Yeah, well, let me let me describe what we’re, we’re shooting for. So again, get as many patients into the funnel, the source of that comes from two different places, we have our Apex s study, which was an expanded study for safety, and then we have an expanded access program as well. So that’s about 50% of what’s filling the funnel is getting those folks converted off of clinical trial material or the expanded access program and into quickstart and commercial supply. The other half is coming from patients that are brand new to Orladeyo, and and you know, that will be the sustainable part going forward. You know, at some point when we convert everybody that was in our clinical trial and unexpanded access will be solely focused on patients nuda Orladeyo. So that’s, that’s one piece of color. I think the other piece is for those people that were in our clinical trial, we said about half of them came from prior prophy. And the other half came from an acute therapy. Well, believe it or not, that’s what we’re seeing in the new patients as well, from the information that we’ve gathered in these, these early days is about 50/50. Again, switching from prophylactic therapy, or switching from acute on demand therapy, you know, in terms of other metrics, of course, we know start forms, and we have a sole source, specialty pharmacy, so we get, you know, really good information quickly. But we also did a lot of digging, and what’s the best way to guide Wall Street in our analysts. And and here’s how we look at it, there’s a consensus view out there by all of our analysts. And our view is, if we can meet or beat that, probably what we’re going to give you is just revenue on a quarterly basis, if it needs more explaining, because we haven’t met it, or beat it, it then it’s probably things like start forms of that to give you a better idea of how the launch is going. But we think revenue is the best indicator and what one of the beauties of a sole source Specialty Pharmacy is the numbers we’re going to give you are not, you know, stocking warehouses and supply right it’s real time go into patience.
Ken Cacciatore: Yeah. So I hate this part of my job where I push a little bit further than I even would like, but when you talk about or think about looking at the analysts models, and obviously, you know, we feel you’re referencing the near term, which which you are, can you give some sense of as you look at the long term and you hear folks like me, and Stacy and your Georgi say, look, our survey work is saying we think ultimately this could be 50% coming from obviously our thought leaders, is there any pushback you want to give or any encouragement you would want to provide in terms of the way you view the out yourself?Are you view kind of a very bullish commentary from folks like us?
Jon Stonehouse: I agree with you. I, I look, I mean, we’ve said this publicly, we believe this drug is north of $500 million peak, globally. And, and, you know, we expect that we will deliver on that. So I agree with you.
Ken Cacciatore: I guess, lastly, then maybe in your work, which would be better than ours, the the hesitation of some patients to engage in a prophylactic treatment, because of the options that they had available that maybe is not being captured, as we look at the kind of the current treatment base and try to always do our metrics off of what we’re seeing, you talk about those patients that likely now would want to join into the prevention market, that previously had not yet
Jon Stonehouse: I’ve met patients that, you know, are managing their disease by, you know, injections of Brady kind of blocker. And you know, what you hit and they don’t want to go on to the injectables, because of their, you know, their concern about, you know, back in the days of sunrise, you know, finding a vein and the frequency, you know, it’s got better with, with tax iro and the frequency there, but there’s still people that, you know, the pain of that injection is just something that they dread. So, yeah, you know, the, we’ve said it before, and I’ll say it, again, the acute market is going to shrink. And I mean, every day, it’s shrinking more, because we’re getting scripts from people that are switching from on demand therapy, and, and they’ve been waiting, you know, to be able to be able to take a capsule once a day to prevent their attacks. So that market will get smaller and smaller. And we’ve said, you know, ultimately could be as low as 20%, or lower.
Ken Cacciatore: And I would think of a managed care perspective, good that fears yours, generic now only helps you in terms of causing a little bit less pain, by being able to use you and still have kind of the on demand treatments.
Jon Stonehouse:Yeah, none of these drugs are perfect. Can you know, all of them have some level of breakthrough attacks, we’ve shown that in some of our patient survey work. And so and they all work pretty well. Right. And so, you know, this burden of therapy is a big deal. And, and, you know, and what we’re seeing is that people would prefer an oral it’s, it’s like you said at the beginning, why wouldn’t you try it to see if it worked for you?
Ken Cacciatore: Well, good, why don’t we talk about then next to us before we go to the pipeline? And, you know, you touched on in Japan, and then hopefully, we’re gonna have Europe, just walk us through expectations or how we should be viewing those those markets.
Jon Stonehouse: Yeah, two distinctly different markets. So let’s start with Japan, you know, we’re we’ve got approval, we’re in the process of negotiating the pricing with Ministry of Health. And that takes about 60 to 90 days post approval. So we’re saying second quarters, when you should expect that we’ll complete that process.This market is just the best way to describe it is it’s about 10 years behind the United States market, both in the number of drugs that are available, the diagnosis and and the organization of the patient community. But it’s getting better if you’d asked me that, to give you that number, five years ago, it’d be more like 50 patients diagnosed instead of 500. And it should be 2500. When you apply the prevalence numbers to the population, so so it is a find patient market. And one of the things that we’ve been told by the advocates is, you know, you need you need a good patient advocate group. And they’re building that in Japan. And it’s really, really growing and getting network, unique KOL’s, that really pay attention to this disease and understand it, and the third is drugs. And so they tell us with an oral that that’s gonna, that’s really going to help in identifying patients. And we chose Torri pharmaceuticals as our partner because they showed back and their partnership with Gilead and HIV that they could find patients. And so so it’ll be interesting to see how quickly and and how that evolves. And but we’re the only prophylactic treatment at at this time in that market. So there could be real opportunity there.
And then on the European front. So a diagnosis is much more similar to the US where patients have been identified. But the on demand therapy is the majority of use. prophylactic therapy had some struggles back in the day when prophy therapy was being injured introduced. And so there are way more people on on demand. So we hope with an oral that we can convince doctors and patients to as you said again, why not try it right and and really switch the market from a largely on demand to a largely prophylactic market.
Ken Cacciatore: Right. Okay with that one, I turn it over to Stacy to talk a little bit about the pipeline.
Stacy Ku: So for factor d 9930. Could we first talk about the decision to first look and P and H, and then maybe the potential opportunity you see there as it relates to the current FDA approved options and the other products and development?
Jon Stonehouse: Bill, you want to take that?
Bill Sheridan: Sure. Stacy, the decision to look at p and H first was a combination of medical need, and opportunity, as well as how quickly the biomarkers change, especially in patients who are naive to see five inhibitor therapy when you start a complement inhibitor. that latter point allows us to rapidly step through a dose ranging study and figure out doses or a dose that we would like to take forward into pivotal trials. There’s certainly a medical need. So in the original licensure studies for eco lism, Ed, half the patients were still transfusion dependent. And we’ve learned since then, that extravascular hemolysis, is a really important complication of the disease after it’s been treated with C five inhibitors. And you know, we think that the opportunity there to control extravascular hemolysis, as well as intravascular hemolysis, with B 699 30, is really strong.
Stacy Ku: So maybe you could provide a teaser for the upcoming r&d day, in terms of the those that are c five, experienced and the naive patients, and we should expect sure types of disclosures.
Bill Sheridan: So we’re, you know, we’re excited about the opportunity to have an r&d day and talk about the way we discover specific potent enzyme inhibitors, and bringing them forward into development. And most of the focus will be on BCX9930 program. We’ll have some leading experts in hematology and nephrology, as well as a patient advocate to help us explain the types of questions that you’re asking around the landscape and the opportunity, the medical need, and what it’s like to suffer with one of these diseases. And with regard to the data, we’ll go through the summary data for all 16 subjects, 10 of whom have never had a complement inhibitor before. So they need to complement inhibitor therapy, six of whom haven’t done so well on the existing c five inhibitors. So the response has been inadequate. And you know, the type of information that is the most relevant here is, you know, this is an anemia, right? So the type of information that’s most relevant, is the patient still needing transfusions, and what happened to their hemoglobin? And, you know, there’s also symptoms to take into account. And they’re also biomarkers. So of course, we’ll also be talking about the safety and tolerability. And on that front, you know, we now have patients that actually pretty soon they’ll be coming up to almost a year. So quite a lot of time on therapy with patients who came on to the study from the start. So I think it’s going to be a really rich set of data and should answer a lot of questions.
Stacy Ku: as it relates to the highest dose. What’s the range of time that you expect these patients to have already been on?therapy? And maybe if, if another teaser on the onset of action?
Bill Sheridan: Oh, sure. During the data we presented last year, actually, with regard to last question, you can see that the onset of action is quite quick.You know, that also applies actually pretty early on in things like change from baseline and hemoglobin. And there’s a chart in our deck for today, that, you know, is the same as the one we presented in September last year that plots the hemoglobin, for example. So you can see it quite quickly. With regard to the length of time on the higher doses. Many patients have had, you know, in that set have had many months. And you know, the doses of interest here are 400 milligrams twice a day and 500 milligrams twice a day, the minimum that we’ll be looking at there is about six weeks.
Stacy Ku: Got it. And moving on to the nephrology and nephrology indications. Will these be similar diseases that some of the other alternative complement inhibitors are going to be looking at? And what are you guys thinking in terms of a basket trial, or if it’s gonna be a very specific indication as proof of concept.
Bill Sheridan: So what I really love about the whole idea of an alternative pathway inhibitor and effective D inhibitor in particular, is just the breadth of indications we can go after. And, you know, I think that there are certainly approaches to look at more than one disease in proof of concept studies. And, you know, we won’t be getting into the details of the design of that or indeed of the pivotal trials in pivotal in PNH until we start those studies later this year. But, you know, the opportunity there is absolutely remarkable because of just the growth of knowledge about the role of dysregulation of the alternative pathway. The role that plays in the pathology of these diseases and driving ill health and bad, bad patient outcomes. A lot of these diseases have no treatment at all. And many are very serious and life threatening diseases.
Jon Stonehouse: And Bill of sorry, got very Stacy, it bill, isn’t there a lot of good precedent to learn from on what to do or not to do and rosaries as well.
Bill Sheridan: So everybody reads the same literature. So yeah, you can, you can have a look at what other sponsors have done. And, you know, I don’t think every, not every individual disease has been studied with a proximal complement inhibitor yet. But, you know, I think that there’s pretty broad agreement that there’s a really great opportunity across lots of indications.
Stacy Ku: In the last few moments, a final question on nine to five zero. What’s the timing for results?
Bill Sheridan: So 9250, we completed a phase one study, we’re very happy with the results. So that’s also included in our deck today. And we saw linear dose proportional increase in exposure with increase in dose. So when we double the dose, we doubled the exposure. And it’s also true with repeated doses. And there was really nothing to see in terms of an adverse effect profile that, you know, there weren’t any safety signals. And so that’s a good result, especially for a kinase inhibitor. In its first in human study, exposure, he got to at the highest dose of 20 milligrams once a day is similar to the exposure we saw in rodent models, where we artificially stimulate heterotopic ossification. So that’s abnormal bone formation. So there’s quite a lot of work to do now, excuse me in making drug supply and conducting longer term toxicology studies to set up the next phase of development. So that’s, that’ll be occupying us this year.
Ken Cacciatore: Okay, with that, thanks for this discussion. We really appreciate it. JOHN, Bill and john. It’s very helpful, a lot going on. Obviously, still early in the launch, and pipeline broadening just a lot of good things coming together at the same time. So continued Good luck, as you introduce or the day Oh, and, and obviously, we’ll be tuning in for the r&d day. We really appreciate this discussion. Thanks. So thanks again. Thank you. Have a great day. Thanks. Bye bye.