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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
Gena Wang: Good morning, everyone. My name is Gena Wang I’m snicker beltech analyst at the bar, please welcome to our second virtual global healthcare conference. First, I wish everyone stay healthy. And I would like to thank all the participants, investors, companies, and especially our event team and a corporate access team who made this virtual healthcare conference possible. With that, I would like to introduce our first presenter for the day, john stone house chief executive officer from biocryst. JOHN, I hand over for the opening remarks.
Jon Stonehouse: Thank you, Gena. Let me share my screen.Get it in presentation mode. Good morning, everyone.
Gena, thank you for the introduction and the invitation to your healthcare conference. It’s great to be with you. I’d rather be with you in
Miami, but virtual will do for now.
So I’m going to go over a few slides in introduction and then the rest of my team will join Gina for a fireside chat. I’ll be making some forward looking statements, those statements have risks that respecters can be found on our website.
So our goal or our aim is to deliver extraordinary and empower ordinary. And that’s based on a strategy that we have at biocryst of bringing novel oral medicines to patients suffering from rare diseases. The delivering extraordinary is that to be able to bring oral medicines to diseases like HAE, and PNH and C 3g, you’ve got to be able to not only have a potent enzyme blocker, but it has to be specific to that enzyme. And these enzymes are part of much larger families, in some cases, three 400 other similar enzymes in the family and the human genome. So that’s the real challenge. And that’s the extraordinary feat that our discovery team does through our structure based drug design capability.
And then we believe that by bringing forward oral medicines to patients with rare diseases, that we are able to give these patients something much more than just an incremental improvement and convenience in their therapy. But a shot at a normal life, an ordinary life, something the rest of us take for granted. Because there’s a significant burden with injectable therapy. And we’ve seen this in ha and we’re also starting to hear from patients suffering from complement mediated diseases as well.
So the result of that strategy is extremely full pipeline, and very exciting time here at biocryst. With a number of filings and now approvals and launches with our lead program or the daio, once a day capsule for HAE, and then behind that, a really exciting program, our oral factor D inhibitor BCX9930 complement mediated diseases and the first indication that we’re pursuing is PNH but there it’s a pipeline and a molecule so it’s almost like having several molecules that we can pursue in parallel, but yet with efficiencies because you only have to do one tox program, one CMC program and the like. So it’s a really unique opportunity and we think could create incredible value for the company. And then behind that and earlier stage program without oral out to ever BCX9250 are really difficult disease called fop that affects children. And then our discovery team continues to work on other exciting targets to bring more oral drugs forward for patients suffering from rare disease.
So as I said, Orladeyo is being launched. In the US. Starting on the fourth of December, we got the PDUFA FDA approved or got our approval on our PUDFA fifth date on December third, and we were out, calling out customers on December 4. And so far, you know, we’re really pleased with how the launch is going there. Charlie, I’m sure we’ll answer more questions in detail from Gena on that topic In the fireside chat, but we’re off to a good start, there appears to be a decent amount of pent up demand. And we believe that we’ve got a drug that works and that patients want and that’s always a good situation to be in when you’re going through a launch.
And our company’s changing and transforming and you’re gonna see a steady drumbeat of news flow coming out of our company that started in the fourth quarter of last year and will continue through the entire year 2021 with our r&d day on March 22, where we’ll be sharing additional data on 9930. But then approval decisions in Europe, reporting out our first full quarter of revenue in the US in our q1 earnings launches in Japan with Orladeyo, Germany and other parts of Europe, and then continuing to advance our pipeline. So really exciting time and a huge transformation for the company.
The other thing that’s really changed in our company is our cash position. The deal that we did at the end of last year where we brought in access to $325 million in capital, from a royalty deal with royalty pharma and a debt deal with a Ethyrium puts us in a position where we can now execute our plan rather than worrying about the next major milestone and how we’re gonna raise money off of that. So and then when you combine that with the ability to generate revenue with Orladeyo, you know, we’re in as good a financial position as we have ever been in the company’s history.
So before I open it up to the fireside chat with Gena and the rest of the team, I just wanted to highlight a lot of attention is going towards Orladeyo with the the launch in the US, but I want to highlight BCX9930. As I’ve mentioned before, we’re really excited about this program because of the broad application across multiple indications for complement mediated diseases. And we have an upcoming data readout that I want to remind investors about. And that’s the readout of our PNH, phase one dose ranging study, where we’ll now have a total of 16 patients worth of data. Today, we’ve only showed you four patients worth of data, some of these patients will have been treated up to nearly a year. And we’re going to have both treatment naive patients and patients that have been poor responders to C five inhibitors. And we’re going to look at data at the higher doses of 405 100 milligrams B ID upwards of eight weeks or greater. And so I strongly encourage you to participate in our R&D day on March 22, because we’re really excited about sharing the data. And also spending time talking to physicians and patients about the challenges of dealing with PNH and nephrology diseases related to compliment.
So that’s my remarks. And now I’d ask the team to come back and Gena and we can get into the q&a session. Let me just introduce who from the team is joining me so we’ve got john Bluth, our chief communications officer Charlie Guyer, our chief Commercial Officer, bill Sheiedan and our chief medical officer, and Megan Sniecinkski, our Chief Business Officer. So Gena, I’ll turn it back to you. Thank you
Gena Wang: Thank you for a great introduction, overview of the company. So maybe I will start with Orladeyo’s launch and starting with us.
So wondering if you can give a little bit more color regarding, you know, the initial patient, like, what is the breakdown between naive patient versus switching from the subacute? population?
Jon Stonehouse: Charlie, you want to take that?
Charlie Gayer: Sure. Yeah. Thanks, Gina. So we’re, as john said, we’re experiencing a lot of pent up demand. So we’re, the launch is going well, and thus far, this is what we expected, and we’re really pleased to see it, but about 50% of the patients are coming from switches from injectable prophy. And then the other 50% are patients who were previously on acute only, and are now stepping up to two prophy. So we’re really pleased to see that and because that’s what we saw in our market research.
Gena Wang: And then what is the patient characteristics for this switch.
Charlie Gayer: So the characteristics, you know, I think the fact that a lot of the patients are switching from prophy means, you know, that they’re we’re seeing patients from from all different types, you know, and that’s one of the things that we saw on our clinical trials, there’s no phenotype for patients that respond to to Orladeyo, all all patients of all different types do well on this product.
Jon Stonehouse: I just want to add one other thing, which is, you know, these are patients that are largely pretty well controlled on their prophy therapy, right. So they’re, it’s not because of lack of efficacy, that they’re coming to our drug, they want more, and this burden of therapy is a really big deal. And so we’re seeing that with
the patients that are going on our therapy in the early days.
Gena Wange: Yeah, so I think that’s why I’m asking like, you know, in terms of their any particular population, like older or, you know, certain it’s more related to the disease more like, the age or other characteristics that you can identify?
Charlie Gayer: No, no, it’s it’s, it’s, it’s patients with HAE. It looks like all of them.
Gena Wang: Yeah. And I believe, you know, everyone has, like, maybe that’s irrelevant to the age or whatever. It’s, it’s, it’s about what you consider as the burden.
That’s right. Yeah.
Gena Wang: Okay. And then the other part is the compliance. I know, it’s very short, you know, and then you may not have enough data. But so far, you know, how’s the patient compliance look like? You know, I’m not sure how much data you can collect that usually is really few. And here, I don’t know, if you have a real world experience, like a how, how’s the compliance ongoing now?
Charlie Gayer: Okay. Well, like you said, it is it is early, we’re just a few months in here. But we’re not seeing anything unexpected. We saw great compliance in our clinical trials. And the early read is, we’re patients who have great compliance. So.
Gena Wang: Okay, and then the the other part is, regarding the safety record, we have some safety, you know, that confuse the symptom from the actually toxicity. And then, how’s that look like in the real world now?
Charlie Gayer: So far, like looking good, nothing unexpected, you know, we’re really pleased with our product label. And anything we see is kind of consistent with with the product label, there’s there’s nothing major there. So patients are generally having a really good experience early on. Oh, cool.
Jon Stonehouse: Anything you want to add to that?
Bill Sheridan: No, you know, that we’re really happy with the label, and with the safety profile, and the efficacy profile that drove the label. So this is
everything we learned in the clinical program, is that this is an important drug for patients with HAE, and that’s what’s going to happen in the marketplace.
Gena Wang: Okay, and then, you know, last component for the US part is, you know, the payer part, you know, any, you know, feedback or pushback from the peers, any additional color and then if you can give a little bit more color, you know, was how many pairs or life insurance you know, the that you already signed the agreement?
Charlie Gayer: Sure, and yeah, what I’ll say is the, you know, the early payer feedback has been good, the whole payer of getting access is a process, but Orladeyo’s the lowest price prophy on the market, so the feedback from that with payers has been very positive. We’ve had success thus far getting Orladeyo on policy in quite a number of plans. But like I said, it’s a process that will continue expect acceleration with major plans in in the next quarter. And then you know, your your long as new patients come on, we’ll continue to work on it but it’s going well.
Gena Wang: Will you be able to share some additional color all in terms of you know, stock form are those in the coming quarters?
Jon Stonehouse: I’ll take that one. Surely. So the guidance, we’ve given Geno’s, we’re not giving guidance number one. And what we’ll report out on in the first quarter is if if our sales are at or above expectations, you’ll get revenue. If they aren’t, we’ll give more to explain what’s going on. So people have a better idea of how things are going.
Gena Wang: Okay. Okay. And then, where do you see the major driver, see for the next quarters, is that from, you know, the acute converted to profi, or is the switcher
Charlie Gayer: so, so our, you know, our sales and efforts in our marketing efforts are just really filling the funnel. Now, as john mentioned, we’ve got that pent up demand. So what I’d expect is we’ll continue to see the same, the same thing, we’re gonna have patients coming from prophy switches, and patients coming from a queue, particularly as more patients are able to get in and see their see their physicians. And that’s what we’re really trying to drive that physician patient conversation.
Jon Stonehouse: And I’m really impressed with the sales and marketing team that Charlie has brought into the company. I mean, they’re off to a great start, they’re really focusing on the top prescribing clinics and physicians at this point in time, you know, there’s a whole plan over the course of the year to expand out more broadly, but I’m really, really impressed with this team and the start that they’ve gotten off to.
Gena Wang: Okay. And then that, you know, switch gear to Europe. How’s the launch?
Charlie Gayer: Yes, the team is ready. Yeah, we got our ch MP, positive opinion at the end of February. And so our first launch will be in Germany, in q2. And just like the US, we hired a really experienced Rare Disease team, the difference in in Europe is that the center’s are really concentrated, you can have literally hundreds of patients under treatment at a given center. So it’s a it’s a very efficient team, and they’re, they’re ready to go. And then in some of the other major markets, we have Early Access Programs underway, that will help drive the launch as well.
Gena Wang: Okay. And, you know, Europe market is predominantly acute. So what efforts are being taken, you know, undertaken that to, you know, potentially transform the market to prophy treatment, and I understand TAK just launched maybe they will also help pave the Wait, what additional effort you will do?
Charlie Gayer: Yeah, I think you’re right, I think that, you know, the availability of new targeted prophy therapies really makes a difference. And so what we see is, you know, in all the work we’re doing with physicians and with with patients, that there’s pent up demand there as well, and there’s, there’s a readiness to switch to prophy now that new options are coming. And, you know, particularly a an oral once daily is very attractive to the market. And so we expect, you know, in the future, use of prophy will start to look more and more like it does in the US 60% of the patients in the US are on prophy today, and that’s still growing. And we expect the same trends to happen in the coming years in Europe.
Gena Wang: Okay, and the, you know, one question. So, like, with all the sales for a force build out, you know, how’s the burn? That will look like, you know, what will be the expectation? How much, you know, the burn for the, for the Salesforce, you know, just launching frappe? You know, how much that will be?
Jon Stonehouse: Yeah, we, we have a, we’ve said that, you know, the Salesforce started around the middle of the year, so in the summer, so in terms of its impact on the last year’s burn, you know, it’s it’s probably more like 50% of the the normal run rate, and then Europe was building up around the same time and build up even more over the course of this year. So what we’ve said publicly is that kind of that the normal run rate going forward for both US and Europe is between 50 and $70 million.
Gena Wang: Okay. Very helpful. So that’s very good. And then Europe regarding the pricing compared to the US how they will look like
Charlie Gayer: well, so European price prices are always a little bit lower than the US but I think some of that starting to change. So if you look at the tax iro price in Europe now and in dollars, it’s about 350 $360,000. Compared to 590, about 590 in the US. So it’s it’s it’s lower, but it’s still very favorable pricing and a great opportunity. For all the data, we’ve got all of our market access processes underway, all the different dossiers have been submitted, and that’s going well.
Gena Wang: So will you also be doing similar proportion like, TAK in Europe? If we think about the discount that you pulled it? Like, would that be similar apply to?
Charlie Gayer: I think it’s a reasonable guy. You can never never say for sure, because each each market is a different process. And you know, it’s very, it’s a very different pricing dynamic than the US but I think that’s a reasonable guy to start. Okay.
Gena Wang: Okay, good. Okay, now switch here to effect the D.
Jon Stonehouse: Gena, can we can we just touch on Japan real quickly? Because it’s also a really important. Yeah. Megan, would you would you just give a quick update?
Megan Sniecinski: Sure. So with Japan, we’re eager to be launching the set in the second quarter. So with our January approval, we just need to complete pricing negotiations. But our partner Torii there is extremely excited to be bringing the first prophy treatment to the patients in Japan. So similar to Europe, we’re ready to go and just waiting for the second quarter.
Gena Wang: Okay, that’s good. So when do you think it will be like meaningful revenue contribution from Japan?
Megan Sniecinski: Well, certainly, Torii will be ready to launch after the pricing is completed and for the first year, and there’s sort of a different pattern of prescribing where the patients have to go in and visit every few weeks. But for us with a market that has a prevalence of about 2500 patients and 500 patients identified now. And the first treatment, we see a real opportunity for Torii to build this market over the coming years. So for us, it’s just an amazing opportunity. And with our deal that we entered into we get to share in their success with royalties coming up 20%, upwards of 40% based on tiered net revenues. So for us, it represents a real opportunity for the business.
Gena Wang: Yeah. So like will later this year, like see the week q for Q should we expect some, you know, revenue stop, you know, some meaningful revenue started contribute?
Jon Stonehouse: my guidance there would be you know, I wouldn’t put a whole lot in until we see how things progress. It’s such a different market Gena, where it’s a find the patient market, that I think we’ve got to get a couple quarters under our belt first before we can really tell you what to expect.
Gena Wang: Okay, that’s fair. And actually, Charlie, you know, wanted to ask the Europe market, you know, what is that? When should we expect meaningful revenue contribution?
Charlie Gayer: To get, you know, the beauty of in Europe is with Germany launching, we can get out of the gates right away. So we’ll see some revenue this year. But I think it’s the same thing. You know, as we look at, we got to get a few quarters in each until we get any kind of perspective on what to expect going forward. But we’re, we’re positive about the opportunity in Europe.
Gena Wang: Okay, great. Now switching gears, in fact, the D. So, Joe, you You said you know, March 22, at R&D you will share some data, maybe just high level? What would you be consider as a win? for, you know, both trimmer naive alleged population and inadequate population responders?
Jon Stonehouse: Yeah, let me start filling in, I’ll pass it to you with, you know, what to look for in the data. I mean, our goal is really simple. You know, we want to have monotherapy, for PNH patients, and all of them, right. So it doesn’t matter if you’ve never been on a C five inhibitor, if you’ve been on a C five inhibitor, and you’re not getting the response you want or you’re controlled, but you you know, have a burden of therapy with your injections or infusions. And you want to switch to an oral we want all of them. And so you know what, so really the way investors and how we look at it is, what’s the control that’s necessary to be able to get people to switch and go on to our drug and achieve that goal. And I’ll turn it over to Bill and what data we’ll look at.
Bill Sheridan: So, Gena, I think the first thing I would say is we’re really excited about the data we showed already last year. And in general, with more patients longer follow up and the addition of C five inhibitor inadequate response to patients. If we see similar data, that’s a win. Right? So that’s the simplest way to think about it. You know, the when we talk to the hematologists and also the patient advocates with PNH they tell us that the goals here are reduce the transfusions if possible, avoid transfusions altogether. red cells because it’s a big burden. And nobody wants to have a red cell transfusion in order to achieve that relieve the anemia, so the hemoglobin coming up compared to what it was before. And you know, there are a whole bunch of biomarkers, you can also measure that indicate that your drug is working as advertised in controlling hemolysis. Things like improving the reticular site count, getting it back to the normal range, and so on. But, you know, the, at the end of the day, improving the transfusion burden and relieving the anemia leads to relief of symptoms in the disease, and longer term, maintaining that control. So that’s on the efficacy front. On the safety front, of course, we now have a lot more experience with the drug patients are coming up to almost a year from when we started the study, and the most important safety parameters. Has anybody discontinued the drug because of adverse event? And had there been any significant safety signal? So that’s the sort of way I think about it for for evaluation of Is there a win. Importantly, this is a phase one dose ranging study. And a big goal here for us is pick a dose to move straight into pivotal trials. So that we’re very confident of that. And you know, later this year, we look forward to studying those pivotal studies.
Gena Wang: So regarding the dose, is it possible to move to Qt or it will be likely the IV.
Bill Sheridan: The PK profile of this drug supports twice daily dosing. PNH is almost a unique disease. And it’s very important to maintain high drug levels to try to prevent break through hemolysis. Because you know, and then people aren’t perfect. Occasionally they’ll forget to take a dose. So if we spread the dosing out to once a day, that missing a dose becomes more problematic. So it might be possible to does the drug once a day in other indications. We don’t know that yet. But in PNH, we’re very, very happy with the twisted endosome. Schedule.
Gena Wang: Okay. And then, regarding safety, I think that we are running out of time, but I do want to ask that safety question. I think, although, you know, can you give a little bit more color on the rash? It seems like investors are very focusing on safety. I wanted you to give a little bit more color any, you know, yeah.
Bill Sheridan: So I think that I’m not quite sure why investors are so focused on the rash. It’s a tolerability issue that goes away. And it’s very short term. So you know, what we talked about last year was that in the PNH subjects, those individuals where we saw the rash, there were no dosing interruptions. In fact, according to the protocol, people have the dose increased on day 15 doubled on day 15 and the rash goes away. So this is a short term tolerability phenomenon, not a safety issue.
Gena Wang: Did you continue to see rash across all different doses?
Bill Sheridan: we will give a full report on March 22. And, you know, the, I think that we’re very, very comfortable with the safety profile with the drug.
Gena Wang: Okay. Well, thank you very much football team. We are looking forward to the on D day to learn more and never shoot a great launch for the drug. And looking forward to our continued discussion. Thank you.
Jon Stonehouse: Thanks for having us. Okay.