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Operator: *Thank you all for standing by and welcome to the Biocryst second quarter 2021 earnings conference call. Please note that all participants will be in a listen-only mode until the question and answer session of today’s conference. To ask a question over the phone by that time, you may press the star key followed by the number one. Please also note that Today’s call is being recorded. I’ll now turn the call over to your host, John Bluth. Sir, you may now begin.
John Bluth (Communications): Thanks, Jesse. Good morning and welcome to Biocryst second quarter 2021 corporate update and financial results conference call. Today’s press release and accompanying slides are available on our website. participating with me today are CEO John Stonehouse, CFO Anthony Doyle, Chief Commercial Officer Charlie Gayer, Chief Medical Officer, Dr. Bill Sheridan, and Chief r&d officer Dr. Helen Thackary. Following remarks we will answer your questions. Before we begin. Please note that today’s conference call will contain forward-looking statements, including those statements regarding future results un-audited and forward-looking financial information, as well as the company’s future performance and or achievements. These statements are subject to known and unknown risks and uncertainties which may cause our actual results, performance or achievements to be materially different from any future results or performance expressed or implied in this presentation. You should not place undue reliance on these forward-looking statements. For additional information including a detailed discussion of our risk factors, please refer to the company’s documents filed with the Securities and Exchange Commission, which can be accessed on our website. I’d now like to turn the call over to john Stonehouse.
Jon Stonehouse (CEO): Thanks, John. As you can see in the numbers we reported today with $50 million of revenue, including $28.5 million of Orladeyo net revenue in the second quarter. The Orladeyo launch continues to go very well. We’re now in the ninth month of the launch, and have a consistent and growing body of evidence from the marketplace that most patients are benefiting and staying on the drug. They are switching from injectable therapies that control their disease because they’re achieving the combination of disease control and reduce the burden of therapy on Orladeyo, the commercial team has done a fantastic job executing despite extremely challenging circumstances, with COVID, limiting our ability for face to face interactions with our customers in the first half of the year. As you’ll hear in more detail from Charlie, our launch success is a direct result of having a drug with a profile patients want a great plan centered around patients switches and execution of that plan by an extremely talented and experienced team. This strong start to the Orladyeo launch in the US and approvals and launches starting outside the US to get Orladeyo to patients around the world. give us the confidence we’ll achieve sales this year of no less than $100 million and puts us on an excellent trajectory to exceed $500 million in global peak sales. A growing revenue stream for more Orladeyo enables us to continue to invest in our pipeline, advancing other important oral medicines for patients suffering from rare diseases. The best example of this is BCX9930. Where in the first half of the year we moved directly from phase one into pivotal trials and PNH which will begin enrolling later this year. Like HAE, patients with PNH tell us they want an effective therapy with a low burden of treatment. They tell us that the treatment burden with current therapies can be hours in an infusion center. We believe 9930 has the potential to offer a significant benefit to these patients. And we are thrilled that we’re entering pivotal trials in this disease. Bill will share an update and our phase three plans with you shortly. Our goal is to continue growing Orladeyo while in parallel pursuing approvals and launches of 9930 across PNH, renal complement diseases, and many other complement disorders. Biocryst has discovered and developed what is shaping up to be a very attractive pipeline of oral medicines for patients suffering from rare diseases. And we’re now demonstrating our commercial Rare Disease capabilities as well. I’ll turn the call over to Charlie to give you more details on the other day.
Charlie Gayer (CCO): Thanks, John. Quite simply, the Orladeyo launch is going great with q2 building on our fast start in q1. Long-term clinical data also confirmed what we knew; Orladeyo is a very effective drug. Data from our Apex two trial presented at yaqi in July showed that patients who started on Orladeyo on 150 milligrams had an 80% reduction in attacks compared to baseline after 96 weeks, the immediate attack rate most months being zero. The potential to have this outstanding level of attack control with just one pill a day is what motivates patients and prescribers. In fact, our prescriber base grew by 50% in Q2, and patient switches from injectable prophy agents and acute only therapy continue to drive the launch. We expected prophy switches to be the largest source of business for Orladeyo, and that is exactly what is happening. 60% of patients starting on Orladeyo today or in the second quarter came from other prophy therapies takhzyro represented over 40% of the Q2 Pro p switches followed in order by Hagarda into Cynrize and androgens. The HAE treatment paradigm in the US has moved beyond outdated classifications like a mild, moderate or severe disease. The goal of modern prophy therapy is to prevent as many attacks as possible and to reduce the impact of any breakthrough attacks. That’s why 60% of HAE patients were already treated with prophy at the time of the world Orladeyo launch. What patients want now is to control their attacks with the lowest possible burden of treatment. Oraldeyo is also contributing to prophy market expansion as use of acute treatment continues to decline. of the 40% of patients in q2 who did not come from other prophy therapies most switched from acute only. Some of the acute only switchers were patients returning to prophy after giving up on injectable products in the past, Orladeyo is also attracting a small but important population of patients new to HAE therapy. Orladeyo is transforming the HAE treatment landscape across all segments, allowing patients to prevent attacks with the lowest possible burden of treatment. Once patients switch, Orladeyo is meeting their expectations. With now eight months since launch, we see patient retention trending in line with a 75% one-year retention in our Apex two clinical trial. Importantly, we see no differences in retention related to prior treatments that patients used. Medication compliance rates are also very high, nearing what we saw in clinical trials because very simply, patients are self-motivated to prevent attacks, and they find it easy to take a daily or oral therapy. We also made significant progress with payers in Q2. Patient and physicians warned us that the hassle and uncertainty surrounding coverage for new HAE medications could limit willingness to switch. As of today, the payers for approximately 70% of the HAE market already have coverage policies for Orladeyo. This base plus additional policies we expect will allow many existing patients to transition to cover product over the rest of the year. The reassurance of broad coverage combined with a high level of service and support offered by our patient services program will also give new patients and their prescribers even more confidence to start on Oeladeyo. This launch is still in its early stages and we are bullish about the future. intend to prescribe as high across our target base including those ha traders who have not yet prescribed or Ladell. The gradual increase in face to face calls, promotional events and medical congresses all will add momentum to the launch. For all these reasons, we are very confident that early data will achieve no less than $100 million in sales this year. launches are also underway in Japan and Germany. And our European team is preparing to launch in the UK, France and several other markets over the next 12 months. We recently announced a regulatory filing in Israel and we are continuing to file around in the world. As I’ve shared with you today, the trends are strong in the Oraleyo launch as we seek to transform HAE care in the United States. We believe oraldeyo also has the potential to transform HAE treatment globally, with each new market contributing to what we expect to be $500 million plus in peak sales. I’ll now turn the call over to Anthony for the financials.
Anthony Doyle (CFO): Thanks, Charlie. As you’ve just heard, the launch of Orladeyo has been going very well, especially when we consider the impact that COVID continues to have on a global basis, Orladeyo continues to create real value for patients, the company and shareholders alike. And we are confident that we have built the foundation for a strong second half of the year. You can find our detailed financials in today’s earnings press release, and I’d like to call your attention to a few items. net revenue for the quarter was $50 million. of this 28.5 million came from net global sales of Oladeyo and 15 million from our partner Tori. Following approval and completion of pricing negotiations in Japan. Our operating expenses, not including non cash stock compensation for the quarter were $72 million. And we ended Q2 with $223 million in cash. This cash on hand in addition to revenue generation, and access to the additional 75 million available from etherium continues to give us cash runway into 2023. Last quarter, we proactively addressed some questions that we had received, and I’d like to take the opportunity to do so again this quarter. As a reminder, we recognize revenue upon shipment from our specialty pharmacy to patients for use no inventory or channel stocking. Strong new patient uptake and the success of our quickstart and patient assistance programs continue to be the main driver of our gross to net adjustment. As we said previously, they should normalize in the 15 to 20% range as we get towards peak sales. As Charlie described, we continue to be very encouraged by the results today, we’re very confident that net revenue for Orladeyo over the year will be no less than $100 million. Plus there are both upside opportunities that could accelerate the launch trajectory and uncertainties that could slow us down. And we need more time to see them develop and play out before providing more specific guidance. Some of these areas that we need to be cognizant of as we go into the second half of the year are First, we’re still early in the launch. And as such, we need more time to let steady-state monthly prescriptions and customer retention develop further. We remain encouraged by what we’re seeing so far, but it’s still too early to accurately forecast or guide. Secondly, as Charlie said, we’ve made great strides and getting Orladeyo policy thus far, but we still have work to do to complete this effort and transition new and existing patients to reimburse product next summer and the end of your holidays historically are slower periods for switches for HAE patients. More data in the next couple of months will help us ascertain if this impacts their trajectory. And lastly, there is a continuing impact due to COVID. with doctors and patient visits naturally declining due to the impact of the Delta variant, we need more time to ascertain how this might impact the launch in the coming months. We’ll be watching these opportunities and uncertainties to see how they influence our launch trajectory. But as I said, based on our performance in the first half of the year, we are very confident that net revenue for today will be no less than $100 million this year. One thing that is for sure is that Orladeyo is a great product that is helping patients manage both attack reduction and the freedom that a reduced burden of treatment brings with the potential of this treatment combined with the opportunities for BCX9930 Bill will discuss. I’m very excited to see the progress that will make in the second half of the year and beyond. With that, I’ll hand it over to Bill.
Bill Sheridan (CMO): Thanks Anthony. The successful launch of Orladeyo is very exciting for Biocryst who discovered and developed this unique drug because we can see the profound impact that it is having on the lives of HAE patients. Next block in our development pipeline is BCX9930, factor D inhibitors are complement mediated diseases. At our March r&d day, we heard from a leading expert physician and a patient advocate who is living with PNH about the unmet needs of correcting anemia, relieving fatigue and reducing red blood cell transfusions. And we also heard about their enthusiasm for our oral factor D 99330 which has a great opportunity to substantially improve PNH Disease Control compared to the currently available intravenously infused c five because it is can address both extravascular and intravascular hemolysis. These clinical advantages Come on top of the expected dramatic reduction in the therapy. Based on the results of that dose-finding proof of concept study. We met with regulators reached an agreement on study design And have accelerated development of basics learning theory from phase one directly into pivotal trials. Today I’d like to share details about our pivotal trials in PNH and provide an update on our renal proof of concept trial and discuss next steps in broadening our research and other indications to pivotal trials of basic way of activity all monotherapy in patients with PNH redeem. One ever deemed to have been designed as rigorous randomized controlled trials, with the goal of supporting broad levels around the world. Details of these studies can be found in the slide set on our website. For both trials patients must be anemic and show donor response to the anemia with increased articular soft cap. The dose of BCX9930 in both trials is 500 milligrams B ID. Within one is a 24 week open label randomized clinical trial of the BCX9930 and approximately 81 PNH. Patients with inadequate response to see five inhibitors really into as a 12 week blinded randomized clinical trial with the 630 in approximately 57 patients PNH is not currently taking a couple inhibitor. In both studies after the control period, all patients received BCX9930 monotherapy through a formal week 52 safety evaluation and then rolled over into a long-term extension. The primary endpoint in both trials has changed from baseline in hemoglobin with pairing to detect the treatment effect of approximately two grams per deciliter. Given the much greater effect on hemoglobin that we have seen in a proof of concept study, we’re very confident of hitting the primary endpoint in both trials. The key secondary endpoints in both trials include assessment of transfusions, entity, and other endpoints include additional relevant clinical laboratory and health-related quality of life outcomes. Importantly, the blinding and redeem to allows us a unique opportunity to rigorously understand the impact of BCX9930 on fatigue scores and other health related quality of life outcomes. Finally, patients in redeem to must have a high on the urgent screening and percentage change from both finding ldh is an additional key secondary endpoint only in that trial 22 our ongoing rollover study for the dose ranging trial, patients have continued to take the BCX9930 blD with average dosing duration now exceeding nine months and the longest time on the drug of 17 months. The Nine patients who are naive to C five inhibitors will continue to benefit from the 9930 monotherapy. With maintenance of hemoglobin responses, no red blood cells transfusions, maintenance of improvement in biomarkers of hemolysis and teenage red blood cell clone size. all nine of these patients have continued on therapy. setting out to the 65 inhibitor inadequate response patients in our trial, five had high levels of C three opsonization on peanuts red blood cells at baseline. Remember, that’s a marker setting up extra vesicle analysis, and all five continue to benefit from BCX9930. Recently, investigators have decided to discontinue c five inhibitor in four of these patients including one patient who had required transfusions for hemolysis following COVID-19 vaccination while being treated with both revellers and BCX9930. This for patients and they’re continuing on BCX9930 monotherapy. Another patient with inadequate response to C five inhibitor who we discussed in March had a very large transfusion burden pre-trial associated with pre-existing harbor splinters and other medical conditions complicating tnh. In this patient at baseline the proportion of PNH red blood cells that were oxidized by competent was only 0.8%. indicating the complement-mediated from all sources is likely not the main factor in this individual’s anemia. And below the rate of red cell transfusion was reduced. With this, if nine out of 10 compared to pre-study, this patient has decided to withdraw from the trial. The 9930 has continued to be safe and well-tolerated with the increased duration of dosing. We’ve seen no safety signals with up to 17 months of exposure. Overall, the continued benefits and safety profile for our proof of concept trial patients are very encouraging and formed the basis for our confidence in moving to pivotal trials and PNH.
Although PNH is a competitive space for clinical trials, enthusiasm for our pivotal program for top investigators and PNH patient advocates based on different results from proof of concept trial provides us confidence that these trials can be recruited. So the startup activities are now underway around the world, and we expect to begin enrolling patients later this year. We’re also on track to begin our proof of concept trial in renal complement medium diseases by the end of the year. This weird basket study, including three cohorts of patients with cetriac monotherapy, or primary minister for apathy, each cohort will enroll up to approximately 14 patients. The trial will collect comprehensive clinical activity pathology and biomarker outcomes to estimate the treatment effect size and facilitate endpoint selection for pivotal studies for each of these indications. Because the BCX993 clearly inhibits activity, of working the alternative pathway Because this top rate is involved in many diseases, and because clinical evidence confirming this mechanism of action is compelling. We plan to drive development across multiple additional indications leading to renal to submissions, approvals and launches around the world. This is an exciting time. And BCX9930 truly represents a pipeline and emotional way of moving this program forward. We all do seed investing fully to complete the development of this unit medicine as fast as possible and bring it to patients who need it. With that, i’d like to hand the call over to john to wrap up.
Jon Stonehouse (CEO) Thanks bill. The launch of Orladeyo is off to a fantastic start. with even more success to come. We’re launching our pivotal program with 9930 and PNH, with more indications to follow. And our r&d team continues to advance additional molecules towards the clinic. It is an exciting time at Biocryst an exciting time to be a Biocryst shareholder. We look forward to keeping you updated on our progress as we continue to build our company. That’s it for the prepared remarks. operator, we’re now going to open it up for questions.
Operator: Speakers, first question is from the line of canned Ken Cacciatore of Cowen and Company, your line is now open.
Ken Cacciatore (Cowen): Congratulations team on the success of the rollout. It’s just fantastic to see it just wondering if you could give us some commentary around the pacing of patient starts through the quarter. So we can try to get an understanding of where we were exiting. And just to confirm I believe you said roughly 70% of patients were paid drug at this point, I just want to make sure we can confirm that in, you know on understandably good nuance about no less than 100 million to the balance of the year and all good reasons. I was just wondering about this initial start. And as we think about the pacing of patients in terms of how they see the clinician through the balance of the year. So they’re normally on average to the clinic, patient visits or one patient visit, just trying to understand your ability to capture how many kind of bites of the apple you get to convert patients. So just kind of any nuance on how these patients typically present themselves to the clinicians. And then also maybe this early commentary about Europe. I know it’s early in Germany, but any nuance around what we’re seeing there so far. Thanks so much.
Jon Stonehouse (CEO): Charlie, I’ll take the last two, you can take the first two.
Charlie Gayer (CCO):Thanks, Kevin. So on the pacing of new patient starts since launch, it’s been really strong and consistent right from the beginning. month over month. And so you know, I think that’s part of a reflection of the 50% increase in prescribers that we’ve we’ve seen so very strong. You’re asking about the coverage. What we said today is that 70% of payers now have coverage policies established. In Q2, the majority of our patients were, were already receiving the reimbursed product. But as both Anthony and I said, we’ll have some of our existing patients now with the new coverage policies, rolling over to paid product in the second half of the year,
Jon Stonehouse (CEO): and then Ken, with regard to the no less than 100 million, and the pacing and clinic visits, I mean, you’re directionally right, in terms of, you know, roughly two visits per year. But I think Charlie, and Anthony did a really good job of telling you that things that could go, you know, very positively that could increase, you know, beyond the 100 million and those things that will keep us closer to the 100 million, the uncertainties that you know, like the summer months, we just this is the first July in August that we have of launching this drug. So we just want to see that play out before we give more finite guidance. And then on Europe, as Charlie said in his prepared remarks, you know, you should expect that the uptake is different than in the US that the ramp is slower. Germany, you know, the launch is underway. They had some pretty heavy COVID restrictions that they just released, I think in July. Now with the Delta variant, I’m not sure that they’re going to put those restrictions back to limit patients coming into the universities, clinics, and the likes. We just have to see how that plays out over time. I think those will be bigger contributors next year, and as Charlie said in his prepared remarks, we’ll be seeing additional countries launching as we get pricing agreements.
Brian Cheng (Cantor Fitzgerald): Goodmorning team, congratulations on a great quarter. Thanks for taking my question. After you see patients adopting, switching to Orladeyo, we’re curious how you’re thinking about demand versus prophy use will shake out over the next two years. And can you comment on how the patient access through medical exception versus formulary reach has changed versus last quarter and add extra color. Thanks.
Jon Stonehouse (CEO): Charlie I’ll take the first one you take the second. So Brian with regard to prophy in on-demand market, it’s shrinking every day. And it’s not just us that are shrinking it to Qaeda CSL with their prophylactic therapy. So, honestly, you know, we said 80/20, ultimately, when it settles out, it could be it could be 90/10, Just given how, you know, patients really, I think they always wanted, prophylactic therapy, they want to prevent attacks, and you know, that there’s an oral option. You know, we’re just seeing a lot of enthusiasm.
Charlie Gayer (CCO): Yeah. And then, Brian, on your question about the the medical exemption trend, we had a lot of success in the first half that we’ve talked about before getting patients covered via medical exemption. What’s great now with with 70% of the market, having access to Orladeyo coverage is that it as I said in my remarks, that gives patients and prescribers a lot of confidence coming in. And what it’s going to mean is that for new patients coming on to product, they’re going to start getting to pay product a lot faster, a lot sooner in the process. So we still pursue medical exceptions when needed, we still have successes there. But increasingly coverage will be through formal coverage policies.
Okay, and related 9930 and PNH. Just curious if you can tell us the rationale behind the difference in the primary endpoint for redeem one versus redeem two. And then will there be a change in ldh include as a part of the secondary endpoints in Iridium one? Thank you for taking my question.
Bill Sheridan (CMO): Sure. The primary endpoint in both studies is the same, it’s changed for baseline and hemoglobin. The pairing ability of what you can detect is a little bit different because of the difference in sample size between the two studies. And quite frankly, we’re very confident of exceeding what we’ve put in for the pair in terms of to approximately two grams of change in baseline hemoglobin based on the proof of concept results. Remember, these studies also have to provide a safety database. So we know we could have had a small sample size and redeem one perfectly easily with a slightly different hemoglobin target preparing purposes. But we need the additional subjects for safety data. And one, just repeating your second question
regarding LDH change, whether as a part of redeem one study
Bill Sheridan (CMO): No both. So I think that the the issue for most patients who have an inadequate response to see five inhibitors is extra vesicle analysis. And ldh is really a biomarker intravascular hemolysis. So we measure all the biomarkers but it’s not a key secondary important in in reading one.
Got it? Thank you.
Liisa Bayko (Evercore ISI): Hi, guys, great quarter. And thanks for taking the question. Can you provide a little more color on what kind of gross to net you’re seeing this point? I know, you said the majority of patients in the second quarter were receiving reimbursed drug but kind of what is what does that mean, as we think about gross to net adjustment?
Anthony Doyle (CFO): Yeah, thanks Liisa. So, you know, they continue to improve quarter and quarter so as more patients move off over from free product on to reimburse and as Charlie said, as more new patients come directly on to our quicker on to reimburse product. You know, we’ve absolutely seen them get better, every quarter. We’re not going to give specific details as they are in both, but we did say was we still feel really strongly about that idea that we’re going to get to 15 or 20% in line with other small molecules as we approach peak sales. I’m honestly really happy with the way things are playing out from a gross to net.
Liisa Bayko (Evercore ISI): Okay. And I guess were you at the majority last quarter just kind of thinking about how things have evolved.
Jon Stonehouse (CEO): The majority paid, is that what you’re asking?
Charlie Gayer (CCO): in Q1 Lisa, we were The majority were on free product and Q2, the majority were paid. So the payment that the trend is that, yep.
Liisa Bayko (Evercore ISI): Okay, thank you. And then, um, in terms of compliance persistence, I realized that’s kind of a, you know, an unknown factor, you know, for the future. But can you maybe tell us what you’re seeing so far? And these first six months as launch? What kind of compliance persistence Are you seeing?
Jon Stonehouse (CEO): Charlie, you might want to just reference what we saw in the clinical trial, and then how it relates to that.
Charlie Gayer (CCO): Yeah, so so Leeson in Apex two, we saw 75%, one year, patient retention. And what we’re seeing so far, eight months into the launch, is we’re trending within that. So we’re, the patient potential is really good. And I think this is a big part of this is reflection, this drug works really well. And, you know, the data we presented at yaqi, with 80% reduction and attacks versus baseline, shows that patients do really well on this drug. And that as far as compliance, I mentioned, it’s been very high, close for our clinical trials rate, which were about 90% range,
Jon Stonehouse (CEO): It was high 90s.
Charlie Gayer (CCO): It was high 90s in clinical trials. And I think that’s just a reflection of patients know that. When they take one pill a day, it’s easy, and it’s going to help them prevent attacks. So they’re self-motivated to do so.
Liisa Bayko (Evercore ISI): Okay, great. And then and turning now to oral factor D, kind of really interested in this basket study that you’re doing have kind of renal complement diseases. Can you talk about, you know, how you were thinking about that study? I’m assuming proteinuria? is going to be the endpoint? When would you measure that? And like, is that kind of important across all these diseases? And what sort of levels you know, do you think you need to do to reduce to be competitive, maybe just give us a little more color around things like that? If that’s a good approach,thanks.
Jon Stonehouse (CEO): Yeah, a bill, maybe focus on the, you know, the quality of, selecting patients that really truly have the disease, what we’re doing to make sure that we have that, and then, you know, clarity around the endpoints.
Bill Sheridan (CMO): So, I think, in previous courses mentioned that we’ve had fantastic discussions with external nephrology experts in a series of meetings, to help us think about all of these questions. And what’s really critical, is making sure that we have patients who have active disease in the first place and not burnt out disease. So that’s, that’s why there’s, as I mentioned, on the call, we’ll be doing kidney pathology. So there’ll be a baseline and a follow-up biopsy to assess that. That’s very important. And we’ll be doing, you know, very well, standardized and comprehensive assessments of the kidney pathology and the role of the company’s system in each of these diseases. That’s number one. They are in a proof of concept study like this. I don’t like the idea of a single endpoint. I like to measure everything, right. So we’re measuring everything, we’re measuring this whole suite of biomarkers. You’re absolutely correct, that proteinuria is a very attractive endpoint. And that and by the way, in the FDA initiative with industry, and academia, around this whole topic of endpoints in kidney diseases, the combination of reduction in protein urea, stabilization of the neural filtration rate is the direction that the field has headed quite independently of states regular walk with, comparable mediated diseases. So that’ll be a discussion with regulators after we’ve got the proof of concept results in terms of choice of primary endpoint for a face for a pivotal study. And each disease is separate. But, you know, I think that the existing evidence out there on the effective complement inhibitors, for example, in BCX330, makes it pretty clear that you can see the benefits pretty quickly.
Jon Stonehouse (CEO): Yeah, Lisa, that the other thing I’d point out is, you know, we’ve gone from a study where we were, you know, phase one, looking at PNH patients, to, you know, in the second half of the year, we’re going to be in pivotal studies and PNH patients and then three more indications in renal disease. So for indications, we’re going to be pursuing by the end of the year, which is very, very exciting for us.
Liisa Bayko (Evercore ISI): Okay, great. Thank you.
Jon Stonehouse (CEO):Thank you.
Jessica Fye (JP Morgan): Thanks. Good morning. Congrats on the quarter. Not sure if I missed this but the 28.5 million Orladeyo sales, can you just break out what if any contribution came from Europe? Or basically anything? You know, any Japan in that number? And then is there any way we should think about how much of a tailwind, you might see in the back half? From gross to net due to improving coverage and less free drugs?
Anthony Doyle (CFO): Yeah, thanks. The best way to think about the 28.5 is the vast majority, almost all of it came from the US. You know, John said Europe, had just kicked off Germany to kick off in June, Japan and Q2. So I think there’ll be more valuable in terms of revenue generation in 2022. So start for Q2, and then for this year that the majority of the revenue is going to come from the US. In terms of the gross and nets in the second half of the year, I’d say the trend will continue. Right. So as we continue to get more and more patients on, and as Charlie said, we continue to get more payer coverage, I would expect that the gross to nets themselves will tighten. Now, as we move off medical exception, and on to reimbursed via coverage, there will be some adjustments, but it will be overweighted by new patients coming on, and then the transition of existing patients on to that reimburse product. So my expectation is that’s really right, the rest of the year, that gross and that’ll continue to lower.
Jessica Fye (JP Morgan): Okay, great. And I think you mentioned that patient retention is 75% similar to the trial, can you help us better understand the time course over which patients figure out if they’re going to stay on drugs? Because this happened early on? Or is it more kind of steady over time?
Charlie Gayer (CCO): Yeah, just the what we said is we’re trending toward that 75% for one year. So typically, you know, patients make that decision in the first few months. That’s what we saw in clinical trials. And that’s what we’re seeing in the real world.
Jon Stonehouse (CEO): And the other thing I’d say, Jess, is, you know, the this Yaqi data that we have, where you see patients doing better over time, you know, one of the things that we’re really encouraging is that patients really give it a fair shot to get the full benefit. Right. So, you know, as Charlie said, it was 80% reduction from baseline over 96 weeks, which is incredibly, you know, effective and, and so patients need to give time to get to that point.
Jessica Fye (JP Morgan): Great, thank you.
Serge Belanger (Needham): Good morning, and thanks for taking my questions. Congrats on the progress. First question on Orladeyo, I know you haven’t disclose the number of patients on drug, but can you give us an idea of how many new patients started early their therapy in the second quarter?
Charlie Gayer (CCO): So you’re right Serge, we haven’t commented on the specific number. What I can say is, you know, again, the demand has been strong and consistent, and new patients are now the great majority of our patient base and since launch, and then we also if we continue to, to expand our prescriber base. So we had 50% more prescribers in Q2, than we did in Q1. And that trend continues. So we have a lot of growth in front of us.
Jon Stonehouse (CEO): Yeah. And Serge. If this question is around, you know, is this a bolus or you know, just a point in time it is there it is very, if we leave you with anything, it’s very consistent growth. very consistent.
Serge Belanger (Needham): And then for Charlie, you mentioned that 70% of HAE patients now we’re on a reimbursement policy. Can you maybe talk about how that policy compares to coverage of other products? And when do you expect the remaining 30%?
Charlie Gayer (CCO): Oh, thanks for the good question Serge. So the all of the policies established thus far, are in parity with other prophy products. So that’s our strategy. We want to make sure that patients and their prescribers can make the treatment choice. And I think it’s a reflection of how well payers are receiving oral health. We’re not going to stop it at 70% and we’re actually having really good continuing discussions. With additional payers, so we expect that number to go up. And coverage will be even stronger by the end of the year.
Serge Belanger (Needham): Okay, and let me squeeze in one last one. For Anthony. I know there’s not you’re not providing opex guidance. But can you give us some maybe directional color for the second half after? You know, both r&d and SGA? saw some ramp up in the second quarter? So how should we think about the second half for those line items?
Jon Stonehouse (CEO): So I think Anthony, just focus on kind of where are we making the investments.
Anthony Doyle (CFO): Certainly a good question. And we continue to invest in the rollout of Orladeyo on a global basis. We’ve talked about the launch going on in the US, we’ve talked about expansion into Europe, and then markets beyond that. So we’ll continue to invest in the commercial side of the house. And then Bill talked about BCX9930, which is, you know, earn a big deal for us in terms of both the pivots and then the proof of concepts that we have. So we will continue to invest in those areas. You know, and so if I, if I look at where we are at the moment, can you take that as a run rate, and then expect additional investment? Yeah, I mean, we there’s value to be unlocked and created in those areas. And we will continue to invest as long as it makes sense to create that value.
Serge Belanger (Needham): Thank you.
Tazeen Ahmad (Bofa): Hi, guys. Good morning. Thanks for taking my question. And congratulations, from me as well, on a really spectacular quarter. I just wanted to get a little bit of color. So you’ve been really thoughtful about providing your sense about how the rest of the year could look with at least 100 million in sales projected, and some caveats to that. But I wanted to just maybe get a little bit of color on a couple of those caveats, namely, summer vacation schedules, and COVID. So as we kind of entered the middle of August, I’m just wondering how those two factors are trending so far this quarter, relative to how you exit into Q and then I have a couple of follow up.
Charlie Gayer (CCO): So it’s a good question, Tazeen. And, you know, as John said, this is our first summer. And yeah, people are going on vacation doctors and patients. And so that’s something you know, we’re watching. But demand has been consistent and strong thus far. As far as COVID, we were able to get, we launched in COVID. We’ve been prepared for this, we’ve done well, throughout COVID, the team, our sales team was able to make more in person calls in Q2. And you know that that makes a difference. But they’re prepared for anything that comes. So we’re watching it, but with the Delta variant, it’s something that could affect things in the rest of the year, just in terms of slowing in-person medical meetings, etc. But we’re very confident in our 100 million no less than 100 million for the year.
Jon Stonehouse (CEO): Yeah, I would just add to that Tazeen, that if the lockdown, you know, if there’s less face to face stuff, right, like medical meetings, you know, we were hoping that the college meeting, or is it the call? Yeah, the college meeting in the fall in November is face to face. And we’ve heard that attendance will be fantastic. But if that doesn’t happen, and it’s virtual, again, that’s a missed opportunity. You know, getting patients together, getting physicians together, face to face is an opportunity. If COVID prevents that from happening, that’ll be a missed opportunity. But we’re very, very confident that no less than $100 million for the year.
Tazeen Ahmad (Bofa): Okay, thanks for that Jon. And, you know, maybe another question on COVID. Is there any impact that you’re seeing geography-wise on the uptake? So I guess we are to believe the reports that we see that cases are surging and highly populated states like Texas, Florida, California, are you seeing any kind of correlation between the higher rates of delta in those states and your ability to get traction there?
Charlie Gayer (CCO): What we’ve seen throughout the year is the kind of how COVID is it? What’s the prevalence of COVID? And what are the local policies affect, you know, our ability to do face to face but we’re not seeing any trends in terms of patient uptake that we could read.
Jon Stonehouse (CEO): Texas is a great example. The state doesn’t require mass but the city of Austin requires mass, right? So there’s just high variability even within a state.
Tazeen Ahmad (Bofa): Okay, thanks. And then the last question for me is maybe on the PNH market, so there was a recent approval from the pellets for their c three inhibitor. As you think about how this market is evolving, Does that change the way you think about the market in any way and how your drug could be positioned there?
Jon Stonehouse (CEO): Yeah, I think the really simple answer is, you know, we’ve got a twice a day oral drug. And that is Charlie remind me every three or four day, twice,
Charlie Gayer (CCO): twice a week, 20 milliliters per injection.
Jon Stonehouse (CEO): So yeah, so from a, from a burden of therapy to Xen, and, you know, if we’ve learned anything in HAE, that, you know, it’s about control disease, and it’s about reduced burden of therapy. And that combo gives patients a real opportunity to have, you know, a treatment regime that they’ve been dreaming about. Right. So So I think we could be very competitive with all injectables.
Tazeen Ahmad (Bofa): Okay, thanks, John.
John Wolleben (JPM): Hey, good morning, and thanks for taking the questions and congrats on the progress. A couple on Orladeyo, you mentioned that there’s 50% new prescribers. Can you tell us how many prescribers you have now and how your targeting approach has been going. And if you have any color on kind of the average number of patient purpose grabbers that are on Orladeyo.
Charlie Gayer (CCO): Good questions, John. So what I’ve said before is our number one tier of targets is about 500, doctors that treat about 50% of existing HAE patients. And we’ve made great progress, great penetration into that group, but still, the majority of them have not prescribed, but the intent to prescribe is really high amongst them. So we have a lot of opportunities there that we’re very bullish about and then we’ve, we’ve made a lot of progress into the next year of our prescriber list, what I’ll say about the kind of the number of patients per doctor, we’ve had prescribers, who have prescribed for multiple patients with Orladeyo. And that’s, that’s the goal, we want to go broad across the prescriber base and then deep within the prescribers. But, you know, the average doctor is still getting started with Orladeyo at this point. So we expect a lot more of those multiple prescribers in the future.
Jon Stonehouse (CEO): Yeah, and all of that leads to upside, john, at the end of the day, right that, you know, doctors who maybe put one patient on, you know, have a good experience and start to put more on over the course of the rest of the year.
John Wolleben (JPM): That’s helpful, and maybe one or two on 9930. Can you comment on how you’re thinking about enrollment timing for both per Redeem one and Redeem two. And given that we’re starting at a higher dose versus what you did in the proof of concept, you expect any difference in outcomes or responses without the hydration that you did earlier? Thanks,
Jon Stonehouse (CEO): Bill, you might want to clarify that dosing first, because we did have 500 in that study.
Bill Sheridan (CMO): Yeah. So we had several cohorts of different dose levels in the proven concept, and we had individual intro patients escalation to that we’ve had plenty of experience in the range of 500 milligrams twice a day, and also a foreigner, whereas twice a day, we chose to go with the higher dose, there were no safety differences. We haven’t had any safety signals. 400 milligrams twice a day, very comfortable. And we expect to see very good responses. And I suppose in regard to the enrollment until we’ve really started enrolling, it’s sort of hard to predict in rare diseases, how you’re going to go. And we worked in HAE programs that we did, for how do we cope with uncertainty about that, what we do is select terrific sites all around the world and cast a broad net. And that’s the way we approach it. So as I mentioned in my remarks, we’ve had fantastic interactions with leading hematologists and patient advocates about the data that we shared in March. They’re very enthusiastic about testing this drug for all of the reasons that I mentioned, to improve patient care. And the fact that it’s an oral medicine is a big deal. So it’s really hard to predict. And so we look forward to having a study’s start up and seeing that the enrollment goes and then we’ll be in a better position.
Jon Stonehouse (CEO): Yeah, john, I’d add to that, you know, with the appelles drug on the market in the US and other competing studies going on and it’s a rare disease. You know, it’s challenging, but one thing that I’ll say, and Bill and Helen won’t brag about this, but they should, which is their team’s work to be sponsor of choice. So the relationship ships that they build, you know, with the study coordinators with the site staff with the site Pi is the best, honestly. And so that makes a difference in terms of enthusiasm for working with us.
John Wolleben (JPM): Got it. Thanks. And congrats again on the great quarter. Thanks, john.
Maury Raycroft (Jefferies): Morning, everyone, and I’ll add my congrats on the quarter. For Orladeyo. What is the latest update on real-world efficacy you’re seeing compared to the phase three studies, and you have a way to collect some of the real-world data to potentially publish it at some point for reference, I guess, what’s your plans there? I know you’re not providing a lot of specifics but can you maybe provide any more perspective into the discovery deal that you’re seeing at this point
Charlie Gayer (CCO): Yeah so as I mentioned on the call, 80% reduction in attacks over 96 weeks shows that this drug really works over the long term and so real world that’s what we’re hearing. Patients are doing really well on this drug, regardless of whether they’re coming from other prophy products which is 60% of our patients or if they’re coming from acute only which is about 40%. And we do plan to keep looking at our long-term clinical data and our additional real-world data from the US and around the world and you can expect us to look to publish things in the future. And so as far as compliance rate we saw 90%. And we’re seeing closed very close to that in the real world. And that’s a reflection of this drug works. patients know what it does. And it’s really easy to take one pill a day. And they’re they’re being very compliant.
Maury Raycroft (Jefferies): Got it? That’s helpful. And then for the PNH study, just wondering if you can provide any more specifics into when the fuor patients on 9930, discontinued C five, in any more perspective into the other one patient that remains on C five combo.
Bill Sheridan (CMO): Sure. You know, I think that you know, as we’ve mentioned, in previous calls, the decision to move to discontinue the C five inhibitor in each individual patient is made by the site investigator. And if you look at the existing evidence to the effect of the inhibitor program, from another sponsor, it took many, many months. They reported better up to a year and still three of the 10 patients hadn’t been switched to monotherapy by that time. So no, we’re not in any hurry here. We don’t need the data for a particular reason right now. We’re moving ahead with pivotal studies. And it was, as I mentioned in my remarks, these the discontinuation of the C five inhibitor in the pool of patients that were investigated that made that decision already was recent. For PNH we liked to see like to see the data mature over time. So it was very recent. other part of your question was about the individual hasn’t switched yet. Just like in the other study, it’s just a matter of time. For the investigator to make the decision, I think.
Maury Raycroft (Jefferies): Got it. Okay. That’s very helpful. Thanks for taking my questions.
Brian Abrahams (RBC): Hi, this is Steve on from Brian, thanks for taking our question. And congrats again on the quarter. So looking through this week’s FDA adverse event report, it looks like there are a few gi events for Orladeyo that were reported. And can you comment on any feedback you’ve received from physicians on any reports of dose reduction due to gi issues? Thanks.
Jon Stonehouse (CEO): Yeah, I’ll take that one, Steven, and maybe bill if if you need to add any color to it. I mean, that’s a known adverse event that’s in our packet circular, and we’ve seen it in the clinical trials, it’s mild, it typically goes away fairly quickly and we’ve had 75% retention. You know, I think the in the clinical study, I’m not even sure if we had a drop off maybe one due to gi adverse events. So anyway, you know, we’re, you know, nine months into the launch, and it’s absolutely meeting our expectations in terms of, you know, what we’re seeing and then you know, I think the other thing That’s really important is, you know, our salespeople, making sure that they’re setting expectations with the physician to counsel the patient properly. And then our patient Services Group, also working very carefully. Because really, when you think about it, if you have the opportunity to really have no tolerability issues and be well controlled on the drug, if you give it enough time, and it’s a once a day oral, why wouldn’t you try? Right? And so that’s the key to this, and so far, so good.
Charlie Gayer (CCO): we’re hearing that the patients are really happy on this drug.
Bill Sheridan (CMO):So what we’re seeing in the FDA fears database, for example, is totally consistent with their labeled adverse event description. We have a safe effective drug.
Brian Abrahams (RBC): Great, thanks.
Chris Raymond (Piper Sandler): Good morning. This is Nicole breast standing in for Chris. Congrats on the quarter. And thanks for squeezing us in. So I guess just two quick ones on Orladeyo kind of just going back to patient retention. I know you just commented on the 80s. But I guess just with more time on the market, what’s been the most common driver of discontinuation so far? has it been response or tolerability, related issues or, or access or potentially something else? And then just the second one, can you just remind us how active the HAE patient community is? Just trying to gauge how much word of mouth initial patients have had success on Orladeyo, will lead to future patient growth and switching. Just wondering if you have any feedback there. Thanks.
Charlie Gayer (CCO): Sure. You know, as far as the active the HAE community in the US this has been very well organized, a lot of communication between patients, we see this as a big opportunity. What patients haven’t been able to do due to Covid is have in person meetings and as John was talking about earlier that becomes a missed opportunity but we expect is really going to start to become very strong as patients talk to each other, and as more and more patients have a good experience with Orladeyo.
Jon Stonehouse (CEO): Yeah, and on the discontinuation. So let me just stress first that Charlie has said, you know, these three times today, we’re within the range of what we saw on the clinical trial. So 75% or greater, are staying on our drugs. So the discontinuation rate is pretty low. And, you know, the mix is all over the board. Right? That, you know, it can be adverse events that no drug works in everybody. So could be lack of efficacy and some, but it’s a mix. It’s a really broad mix.
Chris Raymond (Piper Sandler): Okay, thanks very helpful.
Operator: Thank you participants. I’ll now turn the call over to John Stonehouse for final remarks.
Jon Stonehouse (CEO): Great. Let me wrap up the call with a huge, huge thank you to all of our employees. I mean, the commitment that they’ve shown to be able to discover, develop, and now bring to market innovative drugs for patients suffering from rare diseases is absolutely amazing to me. And they do it with a sense of urgency, they know that patients are waiting. So thank you so much to all the employees of biocryst. And thanks to our shareholders, for those of you in particular that have stuck with us. Just you know, we’re really excited about the future. That company I hope you are too, and we look forward to keeping you updated as the rest of the year unfolds. Thanks and have a great day.
That concludes today’s conference. Thank you all for joining. You may now disconnect