Ladies and gentlemen, thank you for standing by and welcome to the biocryst third quarter 2021 earnings call. At this time all participants are in listen only mode. After the speaker’s presentation there will be a question and answer session. To ask a question during the session, you will need to press star one on your telephone keypad. Please be advised that Today’s conference is being recorded. If you require any further assistance, please press star zero. I would now like to hand the conference over to our speaker today. Mr. John Bluth at Biocryst. Sir, please go ahead.
John Bluth (Chief Communications Officer): 3:06
Thanks, Lorenz. Good morning and welcome to bio Chris third 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 our CEO Jon Stonehouse CFO Anthony Doyle, Chief Commercial Officer Charlie Gayer, chief medical officer, Dr. Bill sharedin and Chief r&d officer Dr. Helen thackray. Following our remarks, we will answer your questions. Before we begin, please note that today’s conference call will contain certain forward looking statements including those regarding future results on 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 wherever its 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 Jon Stonehouse.
Jon Stonehouse (CEO): 4:09
Thanks, John, three quarters of the way into 2021. And this has been an amazing year of execution for our company. The successful launch of Orladeyo and the rapid advancement of our pipelines are the evidence. Orladeyo is our first oral drug to get to the market. And by all measures, including that we did it in a pandemic, we significantly exceeded everyone’s expectations prior to launch. We now have a good line of sight to the trajectory of sales and have guided that for the full year we will achieve between 115 and $120 million in net revenue. What’s even more exciting is we’re just getting started. Charlie will share more color on the launch and what opportunities for continued growth lie ahead The bottom line is patients with HAE want a once daily oral medicine to prevent their attacks. The strongest evidence of that is they are switching from injectable therapy, even when they’re controlled and satisfied with these treatment options. Why? Because they want more, they now see an option to control their disease and the ability to reduce the burden of therapy. Our market research told us this two and a half years ago, we’re seeing it play out exactly the same way in the marketplace. And Charlie will share recent surveys that show those attitudes will increase in the future. As we continue the consistent steady growth of the launch in the US and continue to gain approvals and launch in other countries around the world. Our confidence that we will substantially exceed our global peak sales target of $500 million grows stronger every quarter. Oraldeyo is showing that oral drugs can have a huge impact on the lives of patients with rare disease. But what if we could repeat this over and over again in many different rare disease settings. That’s our strategy. And we have a world class discovery platform that differentiates us through its unique capability to develop potent, specific and orally bioavailable compounds against very difficult biologic targets, Orladeyo is the first to make it to market from our discovery engine. And our oral factor D inhibitor BCX9930. is next. Going after factor D as a target allows us not only to bring another oral drug to patients suffering from a rare disease, but there are multiple diseases to treat with an oral factor D inhibitor, as the alternative pathway plays a key role in many complement mediated diseases. After demonstrating proof of concept in PNH patients late last year with 9930. We are now in pivotal studies in PNH and a proof of concept study in three nephritis indications. Our pipeline is full and following quickly behind Orladeyo. There are many more rare diseases to pursue and many patients waiting for any drug let alone and oral drugs to treat their disease. 2021 continues to be an extraordinary year for biocryst. And the good news is we’re just getting started. Now I’ll turn the call over to Charlie for more details on the Orldaeyo Launch.
Charlier Gayer (Chief Commercial Officer): 7:30
Thanks, John. This launch got off to a great start 11 months ago, even better than our own high expectations. We knew going in that HAE patients really wanted an oral drug to prevent attacks. And that is exactly what we’re seeing as the number of patients taking Orladeyo grows steadily month after month. For the third straight quarter we saw the same strong volume of new patients starting on Orladeyo, more than half continued to switch from other prophy treatments. And patients are staying on therapy in line with our expectations because they’re doing really well. We also continue to see more physicians embracing Orldaeyo. The prescriber base grew by another 25% in q3 and now includes nearly half the top 500 HAE traders. Access also continues to improve nearly all new patients benefit from our Quickstart program, but the average new patient now receives paid product within 30 days. As strong as this launch has been, we see a trajectory ahead that will make Orladeyo the market leader in HAE prophylaxis. Comprehensive market research with large samples of HAE patients and physicians has long shaped our expectations and guided our strategy and is proven to be very accurate in predicting what we’re seeing in the launch. In August, we surveyed another 60 us physicians who treat an average of seven HAE patients each. They reported favorably on their clinical experience and see use of Orladeyo doubling to become their most prescribed product prophy treatment over the next 12 months. Our internal prescription data back this up. The number of repeat prescribers among the top 500 Ha physicians is substantial and has doubled since the first quarter. There are also multiple catalysts that will continue to fuel this launch, for example, the new 96 week data from Apex to showing sustained 80% reduction in attacks is persuasive to physicians. That level of long term attack control is what they expect from a prophy therapy and that data give them even more confidence to prescribe Orldaeyo. We also recently began in person patient education programs, which will be critical to activating patients and spreading word of mouth experience. And we’re very excited to attend our first in person major medical conference this weekend with the meeting in New Orleans. We will present data on how well HAE patients do when they switch to Orladeyo from other prophy products. So stay tuned. US sales will account for nearly all of what we report in 2021. But global launches will add future inflection points to Orladeyo growth. A key example is the favorable NICE recommendation for holidays received in q3 for patients in the UK. By the end of this month, Orladeyo will be covered for ha patients having a history of two attacks or more per month. In contrast, takhzyro and cynrise are covered for a much more limited group of patients experiencing two or more attacks per week. This coverage will allow patients in our Ames expanded access program to convert to reimburse product and will make Orladeyo the first line prophy treatment for the great majority of the UK market. We also recently secured reimbursement approval in Norway, a market where only androgens and cynnrise are currently available. Watches are underway in Germany, France, Japan and the UAE with more to come. We intend to bring Orladeyo to HAE patients all over the world. And with composition of matter patent protection through October 2039. We have many years of global growth ahead. It boils down to this Orladeyo is a drug that can change the lives of HAE patients by controlling attacks with a minimal burden of treatment. I’ll turn it over to Helen to expand on how our clinical data aligned with a real world experience we’re seeing.
Helen Thackray (Chief R&D Officer): 11:38
Thanks, Charlie. The ability of Orladeyo to prevent HAE attacks has been truly impressive. And this is something we’re hearing consistently from physicians based on their own experience, prescribing Orladeyo. The pivotal trial experience in the first 24 weeks of the apex two trial was just the beginning of our understanding of the potential for Orladeyo’s impact on he attack rate. Now in the long term follow up of clinical trial patients, we’ve seen an average 80% reduction in attacks from baseline. This is in the 96 weeks data, which shows the patients have durable substantial reductions and attacks. The original demonstration of a 44% reduction in attack rate was in comparison to the placebo group. And it was at the early time point for the pivotal trial registration endpoint. Even then, at 24 weeks, we saw that there was a substantially greater drop in attacks when compared to an individual’s own baseline attack rate. We observe them 50% of patients were having 70% or greater reduction from their baseline attack rate. Now that there’s real world evidence from the launch in the US, that high rate, a 70% reduction or more that we observed is consistent with what we’re hearing from production from providers. They tell us patients are doing well, and continue to do so. Now, in our long term follow up from the trial, we can see why. With this understanding, we’re excited about the future opportunity here. We know that patients do well and have large sustained reductions in attack rates as they continue Orladeyo. To go into a little more detail on the newer information that we find so compelling. What we presented at the yaki conference in July, is that with long term treatment, attack rates are generally reduced to a greater extent, and this outcome is durable. Specifically, on slide five, you can see that in the patients who completed 96 weeks of treatments, or wideo, almost two years of treatment, and an 80% average reduction from the mean baseline attack rate per month was observed during parts two and three. And median attack rate decreased over time, from 2.7 attacks per month at baseline to zero attacks per month in 16 of 17 months during parts two and three. What’s notable here is that the response, the reduction in attack rate is durable. Patients are staying on treatment and experiencing an enduring result. Not only that, we see large reductions in attack rate, regardless of prior prophylactic treatment experience. And patients on Orladeyo have decreased needs for On Demand treatment. Finally, or they will continue to be generally well tolerated through the long term follow up treatment, which is also consistent with our real world experience observed through 11 months of launch. We are seeing most patients staying on therapy and having excellent experience on Orladeyo. More and more physicians are seeing for themselves the strength and durability of efficacy with Orladeyo, the excellent tolerability profile, and just how happy their patients are when they switched Orladeyo is Charlie mentioned, we are excited to be heading to New Orleans this weekend for the American College of Allergy Asthma and Immunology meeting, which will be our first live meeting since we launched. It will be an opportunity for doctors to come together and share their experiences with each other. We’ll also be presenting our new data on outcomes for patients who switched from injectable prophylaxis to Orladeyo. Of course, right behind or Ladell also coming from our Discovery Center of Excellence in Birmingham. We have our next oral medication for rare diseases BCX9930 rapidly advancing and development across multiple indications. Bill reported in our last earnings call that we have proceeded to pivotal trials with PNH, and the momentum continues to build. Today, Bill will give an update from that study, including the clinical changes we’ve seen in in patients. In total, the observations in the study provide robust evidence of clinical effect, and the impact on how patients feel when treated with BCX9930. Based on these data, we conclude there is exciting potential for 9930 to be an effective therapy for the treatment of PNH. And we have turned our full focus towards the pivotal program at PNH and expanding into subsequent indications. We are confident that this drug has the potential to deliver improvements in both the burden of disease and burden of treatment and to do so with durable benefit for patients, Bill,
Bill Sheridan (CMO): 16:30
So as Helen noted, the PNH program for BCX 9930 has moved directly from a proof of concept does transient trial to pivotal trial. With dose selection endpoints and trial designs agree with the regulator’s reminder of the primary and key secondary endpoints in two pivotal trials is shown on slide eight. In both trials, change from baseline and hemoglobin is the primary endpoint measure. Both pivotal trials include measures of red blood cell transfusions, and also an important quality of life metric that facet fatigue score is key secondary importance. Importantly, is importance reflect the goals of treatment that teenage patients would complement inhibitors to correct animia, reduce or eliminate transfusion burden and related symptoms. In the redeem 2 placebo controlled trial in patients with PNH not treated with C 5 inhibitors % change from those findings in LDH is also at the secondary endpoint. As shown on slide nine, the efficacy evaluation in redeem 2 is at week 12. In our long term rollover safety trial, for patients who continued from the proof of concept trial, we have nine patients need to see private inhibitors received BCX9930 monotherapy, up to 19 months, as of the end of September. Hemoglobin over time, and each patient is displayed in the panels in the fear on slide 10, we analyze the proof of concept data, the doses of 400 or 500 milligrams B ID according to the measures of benefits relevant to redeem 2 the outcome shown in the table on the same slide. As you can see, hemoglobin rose from a baseline mean of 3.7 grams per deciliter or nine patients for transfusion free, LDH fell by 65%. In fact fatigue scale, total score rose by 7.1 points. Those are three with this score the minimum clinically important difference is three. So that’s very impressive. This proof of concept results give us confidence that oral dosing with BCX9930 will perform well against placebo in patients not taking C five inhibitors in redeem 2. Six patients who had inadequate responses to C five inhibitors also into the long term or the child can the proof of concept trial. The BCX9930 initially added to continue to fight over the treatment. The displays of the data set up on slide 12 In the same way as the sequence in a cohort. In your gene one, the efficacy outcomes will be measured from week 12 through Week 24, represented the results both overall and excluding data from one patient who would be ineligible for redeem one. In that analysis. The mean change from baseline and invalid them from weeks 12 to 24 was 2.7 grams per deciliter. 80% of patients were transfusion free and the facet fatigue score score rose by 3.4 points. One patient who developed anemia after COVID-19 vaccination was recently discovered to have a warm antiibody catlytic disease. This immune reaction to vaccination was unrelated to tnh or to BCX9930. Clearly, the patient has transitioned to BCX9930 pt monotherapy, but withdrawn from long term follow up to this diagnosis and development of other unrelated illnesses. In three other patients transition to BCX9930. monotherapies benefits were maintained. This was results give us confidence that all those in DCX BCXwill perform well in the redeem one against contingency five inhibitors in patients with inadequate response to those medications. Most likely signals are being seen in long term dosing would be BCX99330. The accumulating favorable long term safety profile, together with the evidence of benefit that I have described, make us very excited to complete the clinical trials as quickly as we can, and primary regulatory applications for approval in the US and around the world. And then I’d like to hand the call over to Anthony.
Anthony Doyle (CFO): 21:15
Thanks Bill. Having both significant revenue from Orladeyo and an even bigger, fast follower across multiple indications with 9930 presents a fantastic opportunity to deliver for patients and drive value for shareholders. You can find our detailed third quarter financials in today’s earnings press release, I’d like to call your attention to a few items. revenue for the quarter was $41 million, of which 37 came from net sales of Orladeyo. Operating expenses not including non cash compensation for the quarter were $72.5 million. Our gross and net adjustments including access to free drug continues to improve and is on track toward their target of 15 to 20% of peak sales. As Jon said with our expectation that Orladeyo will generate net revenue for full year 2021 of 115 to $120 million and revenue from international regions set to become more meaningful, we are very well positioned for a strong 2022 and beyond. We ended Q3 with $204 million in cash, cash on hand. Continued revenue growth from Orladeyo and access to added to the additional 75 million available from Arthryium gives us cash runway into 2023. This cash position and continued access to multiple different sources of capital enhanced by our strong results and execution puts us in an outstanding financial position to fully invest in launching Orladeyo globally and to advance our Factor D program. Based on the trends that we see, we are very confident that the strong launch of Orladeyo in the US will continue and be enhanced as international markets come online with 9930 as a fast follower. And with more to come from our r&d engine. Few biotechs are as well positioned for significant and sustained growth and value creation. As we are here at biocryst. It is a very exciting time to be a Biocryst shareholder. Operator. We’d now like to open it up for q&a.
Thank you, sir. At this time, if you would like to ask a question, please press star one on your telephone keypad. Again, that star one on your telephone keypad. Your first question comes from the line of the zine Ahmed from Bank of America, your line is open.
Tazeen Ahmad (BofA) 23:36
Thanks for taking my questions. A couple, maybe one on Orladeyo. So John, can you give us a sense of where in Europe right now you’re starting to get sales? Presumably, Germany would be one of those countries? And can you give us a sense about how the dynamics in Europe might in any way be different than the dynamics that you’ve seen here in the US? Meaning you’ve seen a fast uptake here? Would there be any reason to think that you wouldn’t see a fast uptake in Europe? And then secondly, for PNH? Maybe the questions for Bill based on your inclusion criteria? How is enrollment in your mind likely to proceed? And when do you think you’d be in a position to have top line data? Thank you.
Christopher Raymond (Piper Sandler) 24:25
Thanks for the question to Tazeen, Charlie, just make sure I get this right. So in Europe, the contributors where we’re launching and selling drugs are in Germany, we actually have a cohort atu in France. So we’re selling in France and as Charlie mentioned in the UK by the end of the month. Patients will have access and we’ll be selling drugs in the UK. The difference? I can tell you that COVID has had a bigger impact. There’s less telemedicine going on in Europe than there had been in the US So patient doctor interaction has been less. So I think the ramp is a little bit slower. But you know, we expect a pretty good contribution for 2022. And the team is, you know, really been working hard at getting set up. And, Charlie, you might just want to talk a little bit more about NICE and the EMS and why you’re so excited about the UK.
Charlier Gayer (Chief Commercial Officer): 25:25
Yeah. They Thanks, Jon, the the UK is very exciting. And it’s, it’s two things. One, anytime you get a positive recommendation from NICE for any rare disease, that’s a big deal. And the fact that modern prophy has been very limited to that niche of patients with two attacks per week or more. But with Orladeyo, it’s going to it’s going to open up much wider with two attacks per month or more. So there, there are a lot the majority of the market, frankly, that Orladeyo be eligible for. And then we have those EMS patients that we can convert. And we expect to do that over the next the next few months. And so the UK is probably the one place like the US where we can get out of the gates a little faster to see.
Christopher Raymond (Piper Sandler) 26:10
And then to the last point that I’ll make before I hand it over to Bill, as you know, as we add a country, you know, it’s an opportunity to get more patients and generate more revenue. And and Norway’s an example, you know, and we’ll continue to do that over the course of 2022 and beyond. So Bill, do you want to take Tazeen question around entry criteria and impact on enrollment? And?
Bill Sheridan (CMO): 26:33
Sure, yeah, hi Tazeen. Thanks for the questions, I think the eligibility and other characteristics of these studies, and the opportunity to investigate approximate compliment inhibitive characteristics of 9930 make both studies very attractive, actually. And there’s plenty of excitement from the hematology community, in participating in these studies around the world. So I don’t see particular barriers with regards to the eligibility criteria. The both studies are on track for startup. We’re very busy doing that right now. It’s really hard to predict when we’ll finish enrollment, because the field is so competitive. So until we have the first few months under our belt and see what the trajectory is like, I think it’d be hard to predict exactly when we’ll finish. Okay,
Tazeen Ahmad (BofA) 27:27
and do you think that there would still be any kind of COVID impact, at least in the developed world?
Bill Sheridan (CMO): 27:35
So I think people have started to figure out how to continue clinical trials during COVID them and we did that in the proof of concept trial. You have to make adjustments, of course. But medical practice has had to go on telemedicine as an example in the US. I think that their their base supply chain disruptions, as we all know, are there all sorts of impacts, but I think the community in general, is figuring out how to continue important clinical trials.
Tazeen Ahmad (BofA) 28:06
Okay, great. Thank you.
Christopher Raymond (Piper Sandler) 28:09
One One other point that I like to make the bill probably won’t brag on, but I’ll brag on for him is the quality of execution that he’s he and his team have demonstrated with the HAE program, you know, large numbers of sites around many different countries in you know, and that can add risk. But Bill’s team has done a great job of ensuring absolute quality and then really high touch connection with every single site medical monitors talking to investigators about, you know, the patients they’re planning to enroll. And so that that oversight and high quality high touch makes a big difference not only in speed, but in the quality outcome of the trial. So that’s an important piece to success in a pivotal study.
Tazeen Ahmad (BofA) 28:56
Okay, thank you, John.
Your next question comes from the line of Brian Chang from Cantor Fitzgerald, your line is open.
Brian Cheng (Cantor) 29:09
Good morning team. Thank you for taking my question. So maybe for Charlie, I wonder if you can provide a bit more color on the growth of the prescriber base. It seems that the growth rate has fluctuated a bit this quarter compared to the last. How much of the fluctuation do you think is due to the summer holiday and the Delta area and headwinds that you mentioned previously on the second quarter call? And do you have any color on the pace of adoption in your tier one versus tier two prescriber base? Have you seen any significant difference between the two and then I have one more follow up? Thank you.
Charlier Gayer (Chief Commercial Officer): 29:49
Great, thanks. Thanks, Brian. So on on the overall prescriber base. It’s growing really strongly and it’s so I think you’re referring back to in Q2 We said that the base grew by 50%, this quarter grew by 25%. But on a much larger base, I think that’s the key thing to pay attention to. And as far as tier one versus tier two, as I said in some of my comments, what we’re particularly excited about is the penetration and growth in the tier one top 500 physicians. So we’re up to about almost 50% of them have prescribed, we’re seeing a doubling of repeat prescribing amongst those Doc’s. And then our forward looking market research tells us with Doc’s just like that, that they expect to double again over the next 12 months. So it’s going really well, we still get business from the next tier. And we’re still going to explore every corner of this market because their patients everywhere, but Tier one is going fantastically well.
Brian Cheng (Cantor) 30:44
Great. And then one more on access. Can you remind us how your access looking like that, like now on commercial insurer patients? And then how should we think about access for all day out in the new year?
Charlier Gayer (Chief Commercial Officer): 31:01
Thanks. Yeah, pay payer access continues to go well. So first off, patients are getting the average patient gets the paid product really quickly. So within 30 days, that’s what we expect, and we expect it to continue to improve. We’ve had great progress throughout the year in terms of coverage policies, we had a couple more wins in in Q3, but, you know, we still have some payers to go. And we expect continued progress through the rest of this year and into early next year. As we turn into the second full year, that’ll be a key time for us to as patients switch around plans. And we’re ready for that. So we feel really good about where we are with access.
Bill Sheridan (CMO): 31:45
Great, thank you.
next question comes from the line of Lisa Bayko from Evercore
Liisa Bayko (Evercore ISI) 31:56
Hi, thanks for taking the questions. just clarify what you include in net revenue guidance for Orladeyo
Anthony Doyle (CFO): 32:04
Yeah, so included in the net revenue is, you know, like we said, it’s the vast majority of is going to come from the US. And so included in is the, I think 74.5 year to date. And then in Q4, it will be the US plus the European countries that we have that we’ve added. And just again to bear in mind from a revenue perspective here in the US. It’s based on shipments, no stocking, no stockpile orders, just you know, when it gets direct to patient, that’s when we’ll recognize the revenue.
Liisa Bayko (Evercore ISI) 32:40
Okay, and that’s not net of your royalty to rp and it’s not it doesn’t it doesn’t include the Japanese royalty
Anthony Doyle (CFO): 32:49
royalty. Yeah. So it it does include the Japanese it doesn’t include the full amount of the Japanese revenue, because that’s borne by Tori, what you’ll see is a it’ll hit our royalty line, the portion that we take from Tori, it does not include the royalty that’s below the line.
Liisa Bayko (Evercore ISI) 33:07
Yeah. Okay. And can you give us a sense of what gross nets were for the quarter?
Anthony Doyle (CFO): 33:14
Yeah, what we said is that gross does continue to get better. So Charlie’s talked about access and how the access has continued to improve the files majority of the adjustment for the gross and continues to be free products. You know, we are seeing adjustments as and when we guess payers added. You know, it’ll continue to get better quarter over quarter. And Lisa, we’re still on track to get to about 15 to 20% that we’ve talked about when we hit our peak sales.
Liisa Bayko (Evercore ISI) 33:44
Okay, great. And then what kind of compliance persistence Are you seeing, thus far
Charlier Gayer (Chief Commercial Officer): 33:50
as compliance if you’re talking about compliance are people taking their medicine one pill a day has been really high. So it’s been north of 90%, just like we saw in clinical trials, and then persistent patient retention is in very strong as well was in line with our expectations.
Liisa Bayko (Evercore ISI) 34:10
What does that mean? What are your expectations?
Charlier Gayer (Chief Commercial Officer): 34:14
So right right now, about this through Q3 80% of patients who started on Orladeyo or are still on Orladeyo, and overall a one year period, we see that settling period, we see that settling out at around 70%. So about 70% of patients staying on Orladeyo across the year.
Liisa Bayko (Evercore ISI) 34:34
Great, thanks. Okay. And then. So for the third quarter, can you just describe, you know, when you talk about net revenue, is that include? There’s some there’s some x us revenue in there, I assume. Could you maybe talk about it or they’re mostly
Christopher Raymond (Piper Sandler) 34:52
Yeah, it is nearly all us revenue.
Liisa Bayko (Evercore ISI) 34:57
Jon Stonehouse (CEO): 35:01
because the contribution outside of the US will will, you’ll see more in 2022.
Liisa Bayko (Evercore ISI) 35:09
Okay. And then I’m just gonna question for me, you kind of talked about looking, you know, forward to, you know, more developments from your research engines. Can you talk a little bit about that? And then, along those lines, any update on the FOP program? Thanks.
Christopher Raymond (Piper Sandler) 35:30
Sure. You know, the beauty of the strategy is we have this discovery platform where we can build, as I said, in the prepared remarks, potent, specific, and orally bioavailable molecules on really hard biologic targets, like serine, proteases, and kinases. And so, and we’ve seen that others have tried it and have been unsuccessful, right. And, and, and stumbled, and so you got to be able to do all three, to bring an oral medicine to these patients. And, you know, so we’re working with our oral factor D inhibitors, you know, as you know, and we’re, you know, Helen and others are working hard. At what indications do we go beyond the four we’ve already chosen and are starting to study. And so that’s really exciting. But then as you said, they ought to inhibitor for fop, you know, we’re playing a bit of catch up on that front, with, you know, getting drug supply and toxa work, but nobody’s going fast. In this space, it’s a hard disease to tackle. And, and, and we still believe there’s a lot of unmet need. And the data we see thus far in our phase one trial gives us a lot of confidence that 9250 has a shot at being a real therapy for these patients that have nothing. And then there’ll be more to come, you know, Babu and team are working in Birmingham on other rare disease targets. And when they’re ready for primetime, we will be sharing what else we’re going after, and what we’re investing in for more drugs for patients with rare disease.
Liisa Bayko (Evercore ISI) 37:09
So when when we hear more about fop, like what’s precluding you from moving it forward now, or what are the next steps? Timing?
Christopher Raymond (Piper Sandler) 37:17
Yeah, the the bulk of this year has all been about the drug supply and tox. So there was really no opportunity to get into any kind of clinical development, then another important step is talking to the regulators about what the path is. And so making, you know, this is a disease that the earlier you treat, the more impact you have. And so what’s required, you know, to get to those, those age children that have that kind of impact, so we got to work through that. And then also, you know, continue to figure out the design of the trials moving forward. So right now, I can’t give you a timeframe on when we plan to start clinical trials. But you know, we’re trying to go we’re trying to speed that up a lot. And you’ll hear more about it next year.
Liisa Bayko (Evercore ISI) 38:01
Okay, great. And then sorry, just to clarify, I didn’t catch everything Charlie said about, he kind of gave some color on, you know, some something doubling over the next six to 12 months. I didn’t catch everything. I was wondering if you could just repeat as you were talking about kind of like patient ads or market research. Yeah,
Charlier Gayer (Chief Commercial Officer): 38:18
yet. Surely said, you know, we do. We do large surveys with doctors. We did another 60 physician survey average of seven hae patients for each of those Doc’s. And what they said is they’re they’re really happy with what they’ve seen so far. And they see their prescribing doubling over the next 12 months to become their most prescribed. Hae prophy therapy.
Liisa Bayko (Evercore ISI) 38:42
Jon Stonehouse (CEO): 38:44
Your next question comes from the line of Stacy Cooper from Cowen and company, your line is open.
Stacy Ku (Cowen) 38:55
Congratulations on the progress. And thanks for taking your questions. I have a few. First, given your commentary about the q3, tailwinds in the last few months, very encouraging to see continued solid growth. So can you provide some any early thoughts on your current expectations for next year? And then can you also comment on the percent of sales calls that have been face to face at this point? How much growth would you expect without this impact on COVID? Could you provide some level of magnitude and follow up?
Jon Stonehouse (CEO): 39:32
But surely Why don’t you talk about the things that you think are going to kick in to Addy value in 2022? And I’ll handle the the question about what we expect.
Charlier Gayer (Chief Commercial Officer): 39:41
Yeah, absolutely. So Stacy, some of the things I mentioned on the call there. First off the the 96 week data that both that I talked about. Helen talked about showing sustained 80% reduction that’s been really persuasive to physicians and we’re just starting to roll that out. So that’s, that’s a big one, we are starting to do live patient meetings, which is another big thing, because patients don’t like to do things on Zoom. And that face to face contact and word of mouth is really important. And then just live meetings with doctors. This weekend, the college meeting in New Orleans, a lot of the key prescribers to the HA space are going to be there, we’re going to be meeting with them, we have new data. And we we expect to have many more of those kind of live meetings in the coming year. All of this is is in contribute to the strong and steady growth in patients that we’re already seeing
Christopher Raymond (Piper Sandler) 40:39
and Stacey, with regard to what we expect. Next year, we’re not in a position to guide we just guided for this year today. So but we will, you know, we have Charlie has a very good line of sight now on the trajectory of this launch. You know, I don’t know if you caught but he said that, from what we see and what we hear from physicians, we expect that this will be the market leader in the prophylactic treatment of HIV patients. And so as you said, there’s a really solid base that we’re working on. And that Charlie’s team is adding a steady and consistent amount of new patients into the funnel every single month. And as he told you, when Lisa asked the question about discontinuations. And the rate of this discontinuations in line with our expectations, which is much slower than the rate, that we’re filling the funnel, right. So that just shows you a curve, hopefully, I’m painting a picture of a curve. That’s very steady growth. So you know, I would expect that sometime in the new year, we’ll give guidance for 2022. And at some point, we’ll also give an update on our adjusted peak global peak sales because the 500 number is no longer accurate. And it’s definitely on the plus side
Charlier Gayer (Chief Commercial Officer): 41:56
And stacy. I don’t think I answered your question about sales calls. But we’ve reached 100% of our top prescribers to top tier one physicians. In Q2, we got into a lot more in person calls in Q3. I think that dropped just a little bit. But in general, we’re making more in person calls than virtual calls. And we expect that to really continue going forward.
Stacy Ku (Cowen) 42:19
Thanks. It’s really helpful. And one last question on on pipeline for 9930. For further renal basket study, when did we start seeing initial or interim data?
Jon Stonehouse (CEO): 42:32
Bill, you want to tackle that one?
Bill Sheridan (CMO): 42:35
Sure, really, basket study set up as a real learning experiment in three, three specific nephritis conditions that are driven by the alternative pathway compliment, see treatment, or a lot of the primary membranous, nephropathy and agenda property. So what we want to do here is learn as much as possible, we also want to make sure that patients have active illness, that the time that they enter, and to do that we require a recent biopsy. The screening period is up to 66 days to allow for all of that to happen in the central pathology review. And the sites are getting set up right now. Moving forward with getting this study started, I look forward to seeing results evolve over the course of the next month. I think because these diseases are really rare. It’s hard to predict when we’re going to have enough data to take a look at and see what the results look like as a reminder of the design exporting patients to cohort. So there’s going to be an exciting experiment. Field of complementary research in nephrology has exploded as you know, there’s a lot of anticipation and expectation of benefit for these patients. Nothing approved.
Stacy Ku (Cowen) 44:00
Thanks so much.
Your next question comes from the line of Chris Raymond from Piper Sandler. Your line is open.
Christopher Raymond (Piper Sandler) 44:20
Hey, thanks. Um, just a couple of questions on the commercial side. So, um, I think you guys know so you know, today, I think that more than half of patients are new to the drug news Orladeyo or switching from injectables. And I think I read that your your commentary last year or last quarter, sorry, was about 60%. I know you’re not guiding to 2022. But just kind of, can you give us a sense of those, you know, two patient pools of switchers versus new to therapy. Can you give us a sense of of how you expect that to evolve? You know, thinking out maybe a A year from now, two years from now. And then maybe the second question I think I heard you say to a previous question around discontinuations, that your one year sort of steady state is around 70%. Do you have a sense now of those patients that discontinue? What’s the most common driver? Is the drug or you know, efficacy slash tolerability? Or is it? Is it more access related? Thanks.
Charlier Gayer (Chief Commercial Officer): 45:34
Hey, Chris, thanks for the questions. First of all, the the evolution of the switching. Yeah, in Q3, we saw it was greater than 50%, it was actually a little closer to 60 than 50. But, you know, we see that trend continuing. And I back to the market research that I referenced, doctors see that continuing over the next 12 months, they see patients coming from all the different prophy products and acute only. So eventually, we see the the trend has been towards prophylaxis in general, more and more of the patients being treated with proceeds the best for patients. And we see that number growing to 80% plus maybe over 90% over the long term. So a longer term growth will probably come from growing the prophy market. But over the next year, we see the same trends of switches from prophy. And then the rest being acute only and some, usually less than 10% are kind of newly diagnosed patients or newly treated patients that start the therapy on Orladeyo. As far as the 70%, retention and the drivers for that. As we said before, the biggest drivers are going to be if some patients do have adverse events, and we gi adverse events in particular, we set expectations. So patients know that those are likely to go away relatively early to therapy, and that helps. And then the other is just going to be perceived efficacy, no drug is perfect. And no drug is for every patient so that that’ll be the other big thing. It really has not been access. And that’s because we’ve we’ve done a really good job of helping patients access Orladeyo, and if the payer doesn’t cover it, we give them access anywhere if they’re doing well on on therapy. And so patients and doctors are responding really well to that.
Christopher Raymond (Piper Sandler) 47:22
And Chris, I’d add to what Charlie said at the beginning. And there is still a huge opportunity of patients that are well controlled on their prophy therapy that haven’t made the decision to switch yet that we believe, you know, will get over time. And you know, when patients talk to patients, when doctors talk to each other when you know we get more access to them. We think that’s going to be a continuous steady flow of new patients onto our drug.
Great, thank you very much.
Your next question comes from the line of JJonathan Wolleben from JMP securities, your line is open.
Jonathan Wolleben (JMP) 48:13
Hey, good morning. Thanks for taking the question. And congrats on all the progress. Couple for the day for me. You mentioned a couple times you expect Orladeyo now to be the market leader for HAE prophylaxis. I’m wondering if that’s primarily supported from the survey you discussed or is it also trends you’re seeing with demand out in the real world? And then Jon, you mentioned this also, you’re looking to change of peak sales assumptions and wondering where that could land I think takhzyro still expected to be about a billion dollar product. So if you could discuss kind of how you think that dynamic plays out over time, that’d be helpful.
Charlier Gayer (Chief Commercial Officer): 48:46
On the market leader perspective, what we’re seeing is, as I’ve described, really strong and steady growth month over month in patients, and so that’s the primary segment we see in our own internal data, that steady growth, and then the market research just supports that. The fact that physicians see it as well, and we know, the market opportunity and these physician groups, and we see the repeat prescribing happening among the physicians, you put that all together with the patient desire for a once daily oral to prevent attacks. And that’s what that drives OUR outlook on that.
Christopher Raymond (Piper Sandler) 49:28
Yeah, and then, you know, the market leader and, and where we think the peak will net out, you know, the market leader Charlie’s referring to is the most prescribed, prophylactic drug and then that’s what Doc’s are telling him, and we’re seeing the switching in the marketplace and have a high degree of confidence now to say that allow and then we really believe we have the evidence to back it up. In terms of what that number is. It’s significantly north of 500 But we’re not at a point yet to tell you so But you know, we will be soon we, you know, I think the biggest part is, how does x us evolve? And how does it contribute to that number?
Jonathan Wolleben (JMP) 50:10
That’s helpful. And just one last one for me. I think I don’t think you’ve given this out before. But can you tell us what percentage of patients are on free drug? You mentioned the consistent patient ads per month, and it’s about 30 days on so are we expecting a steady flow? That just kind of nets out? quarter over quarter? But today, can you tell us is it 10% 25% 40%? Just in terms, so we have a sense of when those patients roll over to pay drug?
Charlier Gayer (Chief Commercial Officer): 50:41
Yeah, John. So as as of Q3, it’s about a third of the patients who are on free drug. And they you know, over time, we expect more than half of those patients on free drug to to move over to paid product, it’s not going to happen all at once. It’s going to happen as we get additional payer wins. It’s going to happen as you know, a new year starts with government payers. So it won’t be it’s not something that’s going to happen in Q4. But over time, we expect to to get more more than half of those patients over to say and we’re excited about that. It’s an it’s another opportunity for growth.
Jon Stonehouse (CEO): 51:17
Yeah. And a big chunk of the source of those free drug patients. ARE the new patient said his team as every month.
Jonathan Wolleben (JMP) 51:26
Thats very helpful. Thanks for the color and congrats again.
Jon Stonehouse (CEO): 51:30
Here next question comes from the line of Jessica Fye from JPMorgan, your line is open you answer question.
Jessica Fye (JP Morgan) 51:42
Hi, Daniel, for Jessica’s question. With the average new patient receiving paid drug within about 30 days now. Should we think of the conversion of patients from unpaid to paid, representing less of a tailwind for sales going forward?
Charlier Gayer (Chief Commercial Officer): 52:02
I think Daniel the suit to two part two parts there. One is the fact that patients are getting paid so quickly is gives patients and doctors a lot of confidence. You know, that’s something that they worried about. We’re really pleased with the progress there. And then the you know, the the what I just mentioned about patients converting from free product to to paid product that’s going to happen over time. And it is it is a tailwind. It’s not an instant tailwind. But it’s something that we expect to make progress over over the coming months contributing to that steady growth in patients and revenue going forward.
Jon Stonehouse (CEO): 52:38
Yeah. And again, I don’t want to sound like a broken record, Daniel, but it’s also a function of how many new patients do we add every month, and Charlie’s team has done a phenomenal job of that.
Jessica Fye (JP Morgan) 52:49
Okay, great. And then, in terms of cash, following the attempted equity offering earlier in the queue that didn’t go through, and given you have cash for cash runway into 2023. Can you talk about how you think about funding for the company going forward?
Anthony Doyle (CFO): 21:15
Yeah, yeah, I can, I can take it. So you know, I think we’re in a really strong cash position. Right. And I think it’s, it’s primarily driven by two parts of the cash on hand that we have the access to capital through the Arthryiium deals, the revenue that we have coming in from an order from Orladeyo, calculating how we get to that kind of net cash burn. And then in terms of a future perspective, it’s the access to numerous sources of capital. So you know, I think gone are the days when we’d be solely reliant on equity. And we proved that last December with the debt and the royalty deals that we have multiple other avenues that are available for us to raise. So I feel like we’re in a really strong cash position. If and when we do make a raise, you know, we’ll make sure it’s strong. Capital, he referenced the equity raise that we were through in Q3. I mean, that’s honestly, unsurprisingly, probably the least attractive at this point in time for us to use from a source of capital perspective.
Jessica Fye (JP Morgan) 54:10
Great. And then one last question on 9930. Maybe for Bill, with this new update for those patients. For the six patients who are C five inadequate responders, you see change in hemoglobin plus 2.3 grams per deciliter. Maybe can you elaborate that and frame it in comparison to the r&d update? Where we saw a change of 2.2 grams perdeciliter?
Bill Sheridan (CMO): 54:32
Sure, thanks for the question. So the way we approached today’s update is to think about the endpoints in the pivotal trials and how they’re going to be calculated. So specifically for the inadequate respond to study, we will be randomizing patients who are still anemic despite a C five inhibitor with a stable dose to either continue to C five inhibitor, or to take basic running on 30, the primary endpoint and the tacit fatigue, both measured as change from baseline by looking at week 12, the visits from week 12 through 24. So that’s 12,16 20,24. So the average of that. So that’s a difference to the way we’ve talked about the data before, which was just whatever the last last treatment visit was compared to baseline. That’s the primary difference. The second difference here is that we’ve had a couple of, you know, we’ve had some events, like the one I described with the development of an unrelated illness, which happened to be in hemolytic anemia, and one of the subjects that obviously affected the hemoglobin, you can see right in the middle panel in the fingers on the bottom row. So David, the the key differences, I think that what’s important here is that this is, this is what we’ve seen in in the context of COVID. Around the world, right now, there are literature reports of those types of complications. Obviously, they’re just as likely to happen on the control arm as they are on the active arm so nothing has changed in terms of enthusiasm and positivity around the ability to show a clear and clinically meaningful difference compared to continuing to C5 inhibitors in that study.
Jon Stonehouse (CEO): 56:29
Yeah, one thing I’d add, Daniel, is that the whole intent behind Bill showing you the data, the way he did is to tell you that, you know, if we’ve measured, if this was our pivotal study, we would we would succeed, right? And so, you know, that’s why we did calculations based on how the endpoints are calculated in pivotal studies. So, you know, with six in nine patients, it’s incredibly encouraging to see this data and that probability of success in the pivotal is very high.
Jessica Fye (JP Morgan) 57:03
Great, that makes sense. Thank you again.
Your next question comes from the line of Brian Abraham’s from RBC Capital Markets. Your line is open.
Brian Abrahams (RBC) 57:18
Hi, good morning. This is Steve on for Brian. Congrats on the progress. And thanks for taking our question. spoke a bit about the pipeline earlier and fop program in particular. But I’m curious if you can share a bit more about your plans for Galidesivir of your moving forward.
Jon Stonehouse (CEO): 57:32
Thanks. Yeah, we continue to work with the government. You know, you know that antivirals are an important part of dealing with COVID. COVID doesn’t look like it’s going to be going away anytime soon. And so a treatment and an antiviral is still important. You know, these programs go at the speed that the government moves them. And so that’s where we’re at right now. But you know, we’re continuing to, to get funding and continuing to do work to advance that program.
Your final question comes from the line of Gena Wang from Barclays Your line is open, you may ask your question.
Gena Wang (Barclays) 58:19
Thank you for taking my questions. I have a few regarding all the data you launch. The first one is, you mentioned that, you know, we’ll be less than 10% patient, our newly diagnosed patient in your patient pool, wondering what would that number will be the percentage in the context of overall newly diagnosed patients? And my next question is regarding your guidance, when we look at a guidance, the full queue seems largely flat. Is that due to the conservatism and then lastly, the house the pricing X US versus us?
Jon Stonehouse (CEO): 59:03
Charley one, you take the first and the third. I’ll pick the second but you can take them both, and then I’ll do it.
Charlier Gayer (Chief Commercial Officer): 59:09
Gotcha. Okay. Hi Gena. Thanks. So as far as the less than 10% newly diagnosed, I should be specific about that. We don’t know if they’re all newly diagnosed. We just know that they’re newly they to us. They appear to be newly treated with Ha’s therapy. So it’s some patients, we actually expect most of those patients who are sitting on the sidelines but with an oral are coming forward, and now treating their their therapy. In the US at least we think that the newly diagnosed every year there are patients who come out, but it’s a relative, it’s a mature market, and that’s a relatively small percentage. As far as the price of X US versus us, it’s going to be lower. Clearly. There are a launch price in just to give you a sense. Our launch price in Germany is 200,000 euros, that’s the price that we get at launch, not the final price per year, compared to 485,000 US Dollars whack. And then our price or list price in the UK is 133,000 British pounds. And so that’ll give you a sense. In general, the European prices can be 50%, or less than the US price, but we see the volume opportunity makes it really attractive to us.
Jon Stonehouse (CEO): 1:00:24
And with regard to guidance, we feel we’re at a point where you know, and we told you, we wouldn’t give you guidance until we could accurately guide you, we feel we can now there is growth in that number. And it’s steady growth off of a really nice base. And, you know, this is our first fourth quarter, by the way, it’s the holidays, you know, we don’t know exactly how the holidays affect, you know, shipments and prescribing and patient visits and the like. And, and so, you know, we’re factoring that into this as well.
Gena Wang (Barclays) 1:00:59
Okay, I’m just wondering, you know, does that mean like, in 2022, do you anticipate a main growth driver will be x us and that how do you see the contribution X US versus us in terms of growth?
Jon Stonehouse (CEO): 1:01:15
Yeah, no, I, the the growth is going to come largely from the US and there will be additional inflection points coming from the sales that’ll start to heat up X us. And let me just remind you what Charlie’s doing that will help that. It’s the patient patient direct interactions, those those meetings face to face meetings that Charlie said, we’ve started to implement, those are going to make a big difference. Face to face Doctor meetings, like the conference, we’re about to go to doctors talking to each other, they’re not even aware, because they’re not talking to each other about how the launch is going. Other than what they’re hearing from us, the 96 week data that Helen talked about, we’re really just getting going on getting docs to understand that. And then there’s switch data that we’re going to be sharing at the college meeting that we’ll be able to use as well. So all of that is going to have an impact on you know, the trajectory of the US sales going forward. And then the last piece, you know, Charlie’s market research has been pretty accurate, thus far, and that he just did another big study. And it says that physicians that are prescribing are going to double their prescribing in the future. So we expect that that’ll contribute as well. So there’s there’s definitely continued growth in the US no doubt about that.
There are no more phone questions. Mr. Stonehouse, any concluding remarks?
Jon Stonehouse (CEO): 1:02:44
Yeah, thank you. So as Charlie and Helen said, the three of us and the commercial team are heading to New Orleans tomorrow. This is incredibly exciting for us. Because, you know, it’s nearly 12 months since our approval and our producer date. And this is our first you know, true face to face medical meeting. And, and we’re just so excited. Our schedules are packed. We’re really looking forward to you. We’ve got posters, we’ve got an oral session. We’ve got, you know, sessions with physicians and and other meetings. And so we couldn’t be more excited to be heading down to New Orleans to our first face to face major medical conference. And it’s the first time we will actually have a real booth in the exhibit hall. So if you’re at the meeting, I would encourage you to stop by and say hello. So as always, thank you for your interest in our company and have a great day.
This concludes today’s conference call. You may now disconnect