- Speakers from BCRX: Jon P. Stonehouse (CEO), Helen Thackray (Chief R&D), Charlie Gayer (CCO), Megan Sniecinski (CBO)
- Speakers from Jefferies: Maury Raycroft (analyst)
Maury Raycroft (analyst): Hi everyone, my name is Maury Raycroft and I’m one of the biotech analysts at Jefferies with great pleasure that I like to welcome the the biocryst team today. We’ve got Jon Stonehouse, Charlie Gayer, John Bluth (IR), Helen Thackray, and Megan Sniecinski. And Anthony Doyle with us. Thanks, everyone for joining us.
Jon P. Stonehouse (CEO): Thanks for having us
Maury Raycroft (analyst): And it’s going to be a fireside chat format. So maybe to start off if you want to provide a one minute intro to biocryst.
Jon P. Stonehouse (CEO): Sure, I’ll take that one. Before we get started though, we’re all going to be making some forward looking statements and they have risks and they can be found. Risk factors can be found on our website.
So the one minute overview is our goal is to be one of the next great biotech companies. And there was a time in our history where that was a dream or an aspiration. And now we’re well on our way. And it starts with a strategy of coming up with oral drugs for patients with rare diseases, that fills a huge unmet medical need. And it differentiates us from the competition. And the evidence that we’re making progress is we have our first approved oral drug for a rare disease in HAE with Orladeyo. We’re off to a great start with the first quarter sales. And that great start continues. And we believe this is a $500 million plus peak sales drug. And I think with that success, especially in COVID, when companies that really struggled with launches, it’s just a validation of the team that we have, and the great molecules that we have and unmet need in the marketplace for these. And so it all stems from our discovery team down in Birmingham, Alabama, that builds potent and specific enzyme blockers to give us these oral drugs for rare diseases, and then our clinical and regulatory anomalies, CMC and all the other functions that got us for approvals in them and major territories around the world in six months. So we’re the real deal.
Maury Raycroft (analyst): Very good. And it’s a great intro. And you reported solid 1Q for Orladeyo. And just wanted to check on the latest you’re saying about the commercial launch. And if there are any new metrics or qualitative updates on Orladeyo that you can share recently, especially as COVID is starting to help things reopen with x vaccinations.
Charlie Gayer (CCO): Sure, Maury. So yeah, the launch is going great. And we were not surprised but we’re we’re really pleased about seeing the patients switching. This is this is the big thing that we’ve been talking about, which is patients who are new to Orladeyo in q1 More than half of them are switching from other prophy, injectable prophy products. The rest were switching from treating with acute only. And that’s a trend we see continuing. You know, our commercial team is doing a great job out there carrying the message about orladeyo. To to prescribers. And then I think the other the other key piece about this is this early demand that the payers are seeing. Most of the reimbursement in q1 was through medical exceptions. But that puts a lot of pressure on payers, and it’s bringing them to the policy table sooner. And so by the end of this quarter, we expect the majority of patients will have access to early data coverage through policy. And so that that’s that’s a part that bodes really well, for the second half of this year.
Maury Raycroft (analyst): And for to Q2 estimates, are you guys providing any more qualitative or even quantitative guidance on that?
Jon P. Stonehouse (CEO): Yeah, at some point, we’re going to give revenue guidance, we just want to make sure that when we give it that we can give accurate guidance, and we just need some more time, I can’t tell you what in the year that will happen. But as soon as we feel that we can give you accurate guidance, we will. In the meantime, the consensus forecast doubled. After we show the first quarter sales went from 30, something to 65 or something like that. And our goal continues to be to meet or beat so couldn’t do that if it was a one quarter fluke. So we believe that that trend here is really strong. And that launch will continue to be strong through the course of the year.
Maury Raycroft (analyst): Got it. So as far as consensus numbers go, you’re comfortable with worth where they’re at right now.
Jon P. Stonehouse (CEO): Yeah, our goal is to meet or beat.
Maury Raycroft (analyst): Got it. Okay. And for EU in Japan, if you can provide kind of a status update on on those regions.
Jon P. Stonehouse (CEO): Megan you want to start with Japan, and then you can pass it to Charlie for EU
Megan Sniecinski (CBO): So with Japan, we’re excited that our launch is underway with our partner, Torii it’s is extremely exciting there because unlike the US and Europe Orladeyo’s the first approved prophy for patients and so it’s really a build the market opportunity and Torii was very successful with their newly added HIV franchise and we look forward to them applying to the same principles to building out the pro free market. An important point just to note too, with the structure of our arrangement, we get to participate in their upside and their success through the tiered royalty on net sales ranging from 20% to 40%. So certainly was an exciting milestone back in April to announce that launch.
Charlie Gayer (CCO): Yeah, and then in in Europe, we’re really excited in April. And may we get, we received approvals for Orladeyo in EU and then UK. So that’s our third and fourth major approval in the last six months. And we’ve built out just like we did in the US, we built out really experienced commercial teams, our German team will launch first, they’re already promoting and we’ll have product to launch this quarter. We get to set our price. And so we’ll start seeing revenues soon in Germany. And then we have early access programs going in the UK, and then in France. And then the longer term goal is to launch in all countries around Europe over the next couple of years.
Maury Raycroft (analyst): Can you say what the price is going to be in Germany?
Charlie Gayer (CCO): We will we’ll let you know when we would actually launch. Well, we’ll announce it then.
Maury Raycroft (analyst): Okay. And, Charlie, going back to the policy decisions and I guess, and going from medical exceptions to commercial reimbursement, I guess, how will that impact price?
Charlie Gayer (CCO): So in in in the US and Anthony said this before to you know, long term, we expect rose to net from off of whack to be 15 to 20%. Once we reach steady state, there’s not huge discounting going on in the marketplace. You know, for some payers and pbms. There, there will be rebates, but we’ll get to about a 15 to 20% Gross to net.
Maury Raycroft (analyst): Got it and I guess just to clarify as is medical exception, you higher reimbursement than the contract.
Charlie Gayer (CCO): Yeah. So Medicare medical exemption is typically more at list price. So it’s in payers interest to establish policies.
Maury Raycroft (analyst): Got it. Okay. And also, another question on AGI. So we talked a little bit about Japan. Are there plans to go into other regions outside of Japan such as India, potentially?
Jon P. Stonehouse (CEO): Yeah, I won’t. I won’t comment on specific countries, but the goal is to bring it to patients around the world. And we, you know, one of the beauties of doing a global study is that the package that you have has global application. And so our intent is to go throughout the rest of the European Union to go to Latin America to go to Asia, you know, all around the world. And we believe that we’ll be able to do that with the package that we have. And think about it every country after you get past the big ones that add five or $10 million in revenue, you add a bunch of those up, and that’s a real contribution to our peak sales number.
Maury Raycroft (analyst): Right. And on your 4Q call, you mentioned that real world efficacy is higher than trial efficacy.
What is your current hypothesis? What could be driving the the higher real world efficacy relative to that clinical trials?
Jon P. Stonehouse (CEO): Well, I think it’s in the clinical trial, you’re comparing yourself to patients that are on placebo, and there was a big placebo effect in our pivotal study, right. And then what the pivotal, or the endpoint, primary endpoint was, was the difference between the two. In reality in that study, so not even in the marketplace, we saw patients go from three attacks per month baseline down to one, right, that’s a, that’s a big drop, right. And what we also saw, when we added on the apex s data, the long term safety data, is that there were many months where 50% or more of the patients were attack free. Guess what, that’s what we’re seeing in the real world, that so anybody out there who’s thinking that people don’t get well controlled on our drug that, you know, they’re sacrificing efficacy for convenience, the wrong, right, it’s this drug isn’t for everybody, right? It won’t work in everybody, but it is working in a good chunk of the patient population. And 75% of the patients in our clinical study stayed on the drug for a year. And you know, they wouldn’t do that if they weren’t controlled. Right. So I think that’s a, that’s a wall street comparison that’s not faced in the real world. And what we’re seeing in the real world is real efficacy, and control.
Maury Raycroft (analyst): Got it? Just to clarify that. I think that comment around the real world versus trial was made. I know what you call. I had another question. come in on a just I guess with, um, well, if there’s a way to expand relationship with Tori, and what are your if that’s something that you could potentially do in the future?
Jon P. Stonehouse (CEO): Yeah, listen, we’re focused. And Megan, you can chime in here, we’re focused on making sure they’re successful with or the day Oh, to start beyond that, you know, I’m not going to comment, I think it’s really important that we support them in every way. Because as Megan said, we share in their success. I don’t have anything else to add.
Megan Sniecinski (CBO): No, I think you I think you hit it too. I think right now it’s supporting them in their execution with with or the de automate for most of that opportunity. And for where they are, and where we are with the launch are excited about what seeing what they’re able to bring in this patient population. And the real unmet need there. So they’re excited.
Jon P. Stonehouse (CEO): I’ll never forget the trip. It was like Megan’s first week on the job. And we went to Tokyo, and we met with Tori. And they presented the business case and what they did with Gileads HIV franchise, and we were blown blown away. We’re both in the Uber on the way back to their hotel, and we’re like, wow, that’s impressive. And so that’s a big reason why we chose.
Maury Raycroft (analyst): Interesting. Um, well, moving to PNH. Ah,
well, I guess maybe one other question for an Orladeyo, just if you’re planning on recording any additional data on Orladeyo at upcoming medical conferences?
Jon P. Stonehouse (CEO): Megan, you want to touch on just the kind of the general philosophy of what we’re doing?
Megan Sniecinski (CBO): Yeah, absolutely. Certainly one of our commitments from a medical perspective is to continue to add to what we think is already a very rich data set. Looking at the efficacy and the profile, I think Charlie hit on putting that data early. And so certainly there are upcoming congresses this year. And again, our data set and clinical trials have really matured, as we’re transitioning to commercial stage. So we haven’t said specifically but certainly a key priority is for us to continue to generate that that data as well as the longer term data right now that we’ve had patients in our clinical trials for an extended period of time. And, again, we look forward to that supporting just what’s already in this in the data set around just how impactful and meaningful the clinical outcomes are for patients on Orladeyo.
Jon P. Stonehouse (CEO): I think I think another thing that’ll be a benefit when we’re at a COVID is just go into these conferences and see and docs and having interactions that are more than just an electronic poster on a virtual zoom, you know, thing for one of the meetings that, you know, we hear a lot of people talking about the college meeting, which is probably one of the lighter attendance, meetings of the allergy meetings. And they’re all like, you know, it’s in the fall, it’s in November, we’re going we’re going live because they all want to go to one of these. That’s a big miss that that I think we have, and so it makes the results so far even more outstanding.
Maury Raycroft (analyst): Right. Okay. Well, shifting gears to PNH. BCX9930. Just wondering if there’s an update on the phase one response durability in a number of patients still on trial since the r&d day that you can share?
Jon P. Stonehouse (CEO): Helen, you want to take that
Helen Thackray (Chief R&D): Yeah, sure. We’re not providing specific data updates, but we do have patients continue on therapy, that the responses that we reported earlier, I think are representative and the durability has also given us confidence with that. So
there’s, it’s been on the basis of those that were proceeding to the pivotal trials.
Maury Raycroft (analyst): Got it in house prep going for the bnh, phase threes. And can you imagine any new specifics on trial design?
Helen Thackray (Chief R&D): Sure. How’s the process going? Well, we’re preparing for global trials, pivotal trials, we’re excited to begin in the second half of the year. As we had mentioned before, we have agreement with the agency on the primary endpoint, which has changed from baseline and hemoglobin for those trials. We’re very happy with that. We also have agreement on dose and key secondary endpoint, which will be around transfusion. We’ve shared that we’re initiating two trials. One in each population, this will be for monotherapy. Both trials are concerned or controlled. And with this program with those trials, we do anticipate being in a good position for global registration, so we don’t have additional details on the protocols to share today, but just that we’re making excellent progress. We’re making excellent progress also with sites and investigators as compared to start and make sense to start those in the second half of this year.
Maury Raycroft (analyst): Got it. And also wanted to check to get your perspective on how policies reason PNH approval in label in treatment C5 experienced could affect 9930 strategy and market positioning.
Helen Thackray (Chief R&D): Yeah. So we think the approval highlights the value of proximal compliment inhibition, we’re actually really pleased to see that validation. Many, many patients are not getting adequate control of their disease with the C5 inhibitors. And this validates the remaining need, and the ability to meet it with a proximal complement inhibitor. It will be nice to have a palace highlighting this value ahead of our completion of pivotal trials. We do you also note that their product is a twice weekly subcutaneous infusion with a large volume. And so we think there’s remaining need there for an oral and an earlier and easier treatment method. So we anticipate bringing not only a change in the burden of disease with maximal compliment inhibition, but also then improvement in the burden of therapy with 9930 and an oral
Jon P. Stonehouse (CEO): Maury this is where the strategy of oral drugs for rare diseases, you know, just really creates value, right? It’s going to be another switch, right? Another switch strategy where we’re going after model therapy for all PNH patients.
Maury Raycroft (analyst): Right? Makes sense. And you guys just had your R&D day event around 9930. And at the 13 week time point, patients were still on background complement inhibitor. Whereas at a similar time point, Novartis on p, OT three factor B, those patients were able to discontinue use to complement inhibitor. So just wondering if the comparison is relevant, and if so what explains the difference?
Helen Thackray (Chief R&D): So our protocol does call for investigators in that trial to withdraw the C five inhibitor and to get to monotherapies. We do expect them to do that exactly when isn’t really important to the assessment of the drug. It’s a reflection rather a physician preference at the timing of visits and the concurrent medical status for the patient. So it is anticipated in our protocol. And the timing is not really a critical factor. I would make one other point though, on that question, which is that the there’s no difference in disease state in b&h whether patients been treated with a drug or not previously, the PNH, the underlying disease is the same, and in the patients getting monotherapy In the previously untreated population. We saw great control of anemia and transfusions with monotherapy in our population, so you’d expect to see the same issue inpatients on mono therapy after having had a C five inhibitor. So I think that’s probably the most important information. And with this great response in hemoglobin and no longer needing transfusions, we have everything we need to go to different trials.
Jon P. Stonehouse (CEO): And Helen, I think in the Novartis study, there was like a set timeframe went where they had to switch in their protocol where ours was physician choice. So that’s the difference Maury.
Maury Raycroft (analyst): Okay. That makes sense. And I guess, to dig in and dig into it a little bit more, though, for when physicians decide to take patients off of compiliment inhibitor. Guess, can you provide any more color on what factors go into that?
Helen Thackray (Chief R&D): So they are making a decision, I think, based on their individual patient, but one important point there’s in the, in the context of a trial, the investigators making that decision are the same KOL’s who are retreating PNH in clinical practice. And so it’ll be the same judgment. But again, I would, I would point out that the pivotal trial, and this may be more important to real world practice the pivotal trial, we’ll be looking at monotherapy and the data set, then we’ll be on monotherapy. And registration data set will reflect that. So we’d expect that physicians would proceed straight to monotherapy for at 9930 based on that registration data set when we have it.
Maury Raycroft (analyst): Got it. Okay. And for PNH. You mentioned having an additional data update by year end 21. Are you saying anything additional about what data we should expect in that update?
Helen Thackray (Chief R&D): No, but you can’t expect that we will be attending scientific meetings and have presentations on the data when when we have more say.
Maury Raycroft (analyst): Got it. And for the phase three studies, are you I guess, getting some early enrollment signals? What are you guys doing to identify patients before you start those studies?
Helen Thackray (Chief R&D): So those studies, as I said, it will be extensive to start in the second half of the year. So at the moment, it’s it’s site identification, investigator recruitment. This is something we do really well. It’s a competitive space for enrollments and for recruitment of patients and trials. We’re also a company with expertise in rare disease, trial initiation and recruitment. And so getting to know the centers, getting to the right centers, getting getting to the investigators, and helping them understand that we’re listening to them, we’re listening to what they say about what they want to see for their patients. We’re listening to them also and what they say about what makes a trial easy to execute, and get to the data you need, so that we can become their sponsor of choice. That’s our goal. And this will be a global trial, where we’re these two will be global trials where we’re doing that on a global basis. So we’re just executing on that plan.
Jon P. Stonehouse (CEO): Yeah, well, Maury, we won’t have a sense of pace until we’re actually recruiting. And so that’s why we’re not giving any direction on one will finish. But, you know, as we get some rhythm of recruitment, we’ll have a better sense, you know, that like Helen said, That’s not going to start up until a second.
Maury Raycroft (analyst): Right. Okay. And just wondering if you can recap what you said about pursuing additional kidney indications with 9930? And what details about phase two design and strategy can you share at this point?
Helen Thackray (Chief R&D): I think we’ve shared that we expect to begin proof of concept in the second half of this year, and we are looking at select nephritis indications to complement mediated nephritis indications. We we have, I think a lot of examples in the field for to learn from in terms of design, but we’ve not stated yet what that design would be we do have a dose level for factor D In addition, and so the dose that will take into those trials is is fairly straightforward. And I would add one other point there while we’re starting working towards starting a trial in select nephritis indications the alternative pathway, and proximal complement inhibition is relevant to many other diseases. And so this is just the first step both in PNN is is pivotal trial program and then nephritis as a second step, and our third step would be to continue to add disease indications were treatment with the factor D inhibitor is relevant and they control disease. And I think there’ll be more to come.
Jon P. Stonehouse (CEO): And Maury, I’d add. One of the reasons we brought Helen on board is to really scale up the r&d capability that includes running multiple indications in parallel. We’re exploring other indications outside of nephritis indications and PNH and where we want to go and then also building the discovery capability beyond what we’ve done. I mean, financially We were in a hand to mouth situation for quite a few years. And that’s not the case anymore with revenue coming in and you know, the balance sheet as strong as it is. So, you know, Helen’s main focus is to really figure out how do we scale this up to really run a lot in parallel.
Maury Raycroft (analyst): And for your phase two study for HAE that you guys ran, and it’s kind of a nice study design, because it enabled you to do some interim data cuts along the way. For PNH. How do you think about that with, with these phase two indications, I guess, would you try to do something similar where you can provide data updates and keep investors interested?
Jon P. Stonehouse (CEO): I think that was more dose ranging right. And we did certain cohorts, and we had we read out, we’re done with those ranging for 9930. That’s the beauty. So the proof of concept data that you’ll get in nephritis indications, for example, will be at a set dose that will then go into pivotal studies. So that that’s exciting.
Maury Raycroft (analyst): Got it? Well, maybe maybe, to clarify that question. So for the phase two’s, how do you think about timelines for for those studies?
Helen Thackray (Chief R&D): So proof of concept studies starting this year in select nephritis indications and other studies we’ll be assessing. We’ll have more as we arrive at timing for this
Jon P. Stonehouse (CEO): And the beauty is it doesn’t it doesn’t take like years to get an answer on are you having an effect either. So, you know, so we should get readouts in a reasonable timeframe on these proof of concepts.
Maury Raycroft (analyst): Got it. Okay. And for your r&d day, you guys highlighted the biocryst drug design capabilities, expertise, do you have plans to reveal any new drug candidates this year.
Jon P. Stonehouse (CEO): I’m not sure that will unveil any this year, you can bet that we’re working on them in the background. And when they’re mature enough to share more broadly, we’ll do that but you know, early discovery stuff, it’s never to our advantage to the show a lot to the competition and others. But know that we’re working on and as I said, you know, how I came on board to help us scale up and, and produce more out of what we think is a world class discovery group.
Maury Raycroft (analyst): Makes sense. And I think we’re almost out of time. So it’s been a great conversation. But with with the remaining time maybe if you want to just highlight some of the key catalysts ahead that investors should be focused on
Jon P. Stonehouse (CEO): revenue revenue revenue. I think the launch of Orladeyo is is really the focus right and in terms of what you’ll see come in, we feel real bullish about our ability to continue to have a successful launch and then getting studies up and running. Right getting the pivotal studies with PNH up and running and getting the proof of concept studies. up and running are key.
Maury Raycroft (analyst): Got it. Okay. Well, it was great seeing you all today and thanks for joining for the fireside discussion. Thanks,