Q1 2025 BioCryst Pharmaceuticals Inc Earnings Call
Dr. Wolleben, Dr. Wolleben, Dr. Wolleben
Speaker Change: Good day and welcome to the BioCryst first quarter of 2025 Warnings Conference call. All participants will be in lesson only mode. Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star then one on your telephone keypad.
To withdraw your question, please press star, then to... [inaudible]
Please note that the 7th is being recorded [inaudible]
Speaker Change: I would now like to turn the conference over to Jon Bluth with investor relations with BioCryst. Please go ahead.
Speaker Change: Thank you. Good morning and welcome to BioCryst's first quarter 2025 corporate update and financial results conference call. Today's press release and accompanying slides are available on our website.
Speaker Change: Participating with me today are CEO Jon Stonehouse, Chief Commercial Officer Charlie Gayer, and Chief R.D. Officer Dr. Helen Thackray. Following our remarks, we will answer your questions.
Speaker Change: 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 four looking statements.
Speaker Change: 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.
Jon Stonehouse: I'd now like to turn the call over to Jon Stonehouse.
Speaker Change: Thank you, John . We've started 2025 with another quarter of outstanding performance. As you will hear from Charlie in more detail, the US commercial team made tremendous progress in moving patients on our ladale from free drug to paid at a much faster rate than we expected.
The result is quarterly revenue of $134 million for $134 million.
Speaker Change: This improvement in the paid rate impacts revenue performance in Q1, but also through the rest of the year leading us to raise annual revenue guidance for Orlodeo to between $580 and $600 million in dollars.
Speaker Change: Our improved revenue growth significantly increases our margins and has also accelerated our cash flow and profitability goal by a year.
Speaker Change: We now expect to be profitable on a full year basis this year. This increased financial strength and the positive cash flow it generates enabled us to pay down $75 million of our debt in April while continuing to invest in and advance our pipeline.
Speaker Change: In addition, we are now moving into Orlidaeal revenue levels where we no longer pay a royalty on sales above $550 million and we're getting closer to our peak sales for Orlidaeal of $1 million $1 million.
Speaker Change: In the current environment of uncertainty, having a company with growing sustainable revenue,
Speaker Change: An advancing pipeline, and financial strength to be profitable, paid on debt, and be independent of the markets is hard to find in our industry, but that's exactly what we have at BioCryst we have at BioCryst Pharmaceuticals, we have at BioCryst
Speaker Change: With that, I'll turn it over to Charlie to discuss our fantastic first quarter results. Charlie?
Charlie Gayer: Thanks, Jon. The launch trajectory for Orlando has been consistently strong for the past four years, but the first quarter of 2025 was our best yet because of the combination of great demand generation and amazing progress, helping patients gain access to therapy.
Charlie Gayer: As we described previously, US patient demand in 2024 matched the first year of the launch. Our team continued this momentum as first quarter new prescriptions slightly exceeded our best quarter from last year.
Charlie Gayer: In addition, our latest patient survey showed that, regardless of their current treatment status, the percentage of USHA patients who strongly prefer oral prophylaxis [inaudible]
Charlie Gayer: Group to 70% up from 51% in 2023, as you can see on slide 7 in today's presentation.
Charlie Gayer: And HAA Treaters are increasingly convinced that Orlodeo is very effective and convenient for patients based on their individual experience and the rapidly expanding body of real-world evidence that they have seen. Orlodeo is becoming their treatment of choice.
Charlie Gayer: What really drove Q1 performance, though, was a 10%-page point jump in the rate of paid patients in the U.S. over where we ended 2024.
We expected that level of improvement to take three years. [inaudible]
Charlie Gayer: We did it in four months. The Inflation Reduction Act drove about two-thirds of the improvement because the IRA is achieving its intent of helping Medicare patients affore their prescription copayments.
Charlie Gayer: Medicare patients were not only able to afford their early day of prescriptions, but they were able to do so earlier in the quarter than expected.
Charlie Gayer: And we also continue to make great progress among the roughly 60% of oral adeotations who have commercial insurance.
The paid rate in that segment increased to 84%
Charlie Gayer: As we further improved our ability to convert patients from long-term free product to paid product By the end of April , we were nearly through the reauthorization season and approximately 84% of all established patients on Orlodeo were receiving paid therapy
Charlie Gayer: That's close to our long-term goal of 85% on the past $800 million in US revenue, a rate we forecast would take at least three years to hit.
Jon Stonehouse: This acceleration and paid rate improvement means a lot more revenue this year, allowing us to increase guidance significantly as Jon noted.
Jon Stonehouse: But it also positions us to capture more Orlando revenue over the next several years, as our patient base continues to grow. In other words, we're still on a path to a billion dollars in global revenue in 2029, and the path is now even more profitable.
Jon Stonehouse: Slide five shows visually how we expect this year to look different
Jon Stonehouse: We anticipate this pattern shift because we captured more revenue opportunity per patient in Q1, so the Q2 increase will move closer to the underlying patient growth trend.
I'll provide a bit more color to explain.
Jon Stonehouse: As I mentioned earlier, patients moved to paid status more quickly than expected in Q1, which means we gave away less free product.
Jon Stonehouse: Our team also continued to improve growth to net, which in Q1 allowed us to keep it closer to the lower end of our typical range of 15 to 20 percent.
Jon Stonehouse: When in prior years, it was closer to 20% in the first quarter. We expect growth to net to improve throughout the rest of the year, but more gradually, so that the full year average will be right around 15% So that's the end of the year, so that's the end of the year, so that's the end of the year
Jon Stonehouse: Our great executions to start the year and accelerated Orlando Outlook for the rest of 2025 come at an exciting time because our clinical pipeline is mirroring important early milestones. [inaudible]
Jon Stonehouse: The path to another differentiated rare disease product like Orlodeo could soon be increasingly clear. I'll turn it over to Helen to describe our progress.
Helen Thackray: Thank you, Charlie. Good morning. Today, I'm pleased to share an update on recent significant milestones for our pipeline programs.
Helen Thackray: First, we submitted our pediatric NDA for LaDale to FDA, introducing an oral granule formulation for patients aged 2 to 11, with additional filings in Europe , Japan, and Canada also this year.
Helen Thackray: This would be a significant advance in the treatment of children with HAE, as it would be the first targeted oral prophylaxis therapy for this age group.
Helen Thackray: Next, I'm pleased to report that we received authorization to initiate patient enrollment for both of the pipeline programs following Warlodeo.
Helen Thackray: BCX 1775 in Netherton Syndrome and a Voral Stat in Diabetic Macular EZMA, which is an important step on our path to having initial clinical data in patients for both programs by the end of the year.
Helen Thackray: Today, I'll focus on the Netherton Syndrome Program in detail and provide more information about the trial design, what we're looking for, and what to expect by the end of the year.
Helen Thackray: First, I'll describe why we're so excited about the potential for 1775 as a transformative treatment for people living with nether's syndrome. This is a devastating illness. It's consequences are very serious and leads a lifelong impact on health and well-being .
Helen Thackray: We've heard from patients that the lack of treatment leads them to withdraw from medical care. We recently heard exactly the same feedback from our clinical site investigators.
Helen Thackray: Just as it's been for other rare diseases like HAE, we expect introducing a new potential treatment that could revolutionize care will attract patients back into the care system and into clinical trials.
Helen Thackray: We designed 1775 to address the fundamental pathology that causes Nethershind syndrome.
Helen Thackray: Every aspect of which is ultimately caused by a genetically-determined lack of an essential skin protein called spink-5, also known as lecti.
Helen Thackray: Normally, this protein stops cells in the outer layer of the skin from prematurely separating from the cells below. It achieves this by controlling the activity of a key callocrine, KLK-5, which digest bridging proteins that glue the outer layer of skin cells together. [inaudible]
Helen Thackray: In Netherton Syndrome, there is no breaking mechanism for this because KLK-5 is continuously on, and so skin cells quickly separate and are lost.
Helen Thackray: What that causes is a massive disruption of the essential barrier functions of the skin, which normally keeps warmth and moisture inside and microorganisms outside, but not in Netherton syndrome.
Helen Thackray: And in addition, KLK-5 directly activates the rest of the calcrine cascade in the skin, via KLK-7 and KLK-14.
Helen Thackray: Through cascading, pathologic inflammatory and allergic pathways, the effect is magnified down into the deeper skin and tissue layers, ultimately also resulting in systemic inflammatory and atopic effects like asthma and food allergies.
Helen Thackray: All this stems from one faulty gene leading to the lack of one protein, think five, the regulator for KOK 5.
Helen Thackray: This means KLK5 is the key pharmacologic target in this disease because it's the pinnacle protein, the one at the very top of the cascade that drives the whole process as we see in slide 13 in today's presentation.
Helen Thackray: Our scientists, engineered 1775 to replace the function of that missing protein in its control of KLK-5.
Helen Thackray: So we have tremendous confidence in the target and the pharmacological approach we are taking based on the significant experience of our scientists and many others who have published their clinical and preclinical work.
Helen Thackray: A big challenge with a protein therapeutic in this disease is to get enough drug into the epidermis and have it stay put.
Helen Thackray: The epidermis, or the outer layer of the skin, is where it needs to act.
Helen Thackray: So, to give us the best chance at success, our scientists designed this molecule with far greater potency compared to the natural inhibitor and very high affinity or stickiness for KLK-5.
Helen Thackray: These features give us the best chance to minimize the dose necessary and make every molecule that finds the target count by binding tightly and blocking KLK-5.
Helen Thackray: Today, we're reporting that the IMD has cleared in the U.S. for enrolling patients with Nethershin Syndrome into our phase one trial. This clinical trial is set up to tell us quickly whether we've met our objectives and design of the drug. The trial plan is outlined on
Helen Thackray: We're currently dosing healthy volunteers to evaluate basic information on exposure and safety and we're now identifying sites in both the US and Australia for the patient arm of the study. Patients will be given four weeks of treatment and about two months of continuing evaluation
Helen Thackray: Building on this, we also plan to expand the trial later in the year to evaluate a longer 12-week course of treatment as we learn more about the drug.
Helen Thackray: Cohort Size will be small as we expect to gain a significant amount of information from just a handful of patients and perhaps from every patient.
Helen Thackray: To best inform the design of future trials, and to provide early insights into the therapeutic potential of 17-725, we need to understand how the drug behaves in patients living with Netherton Syndrome.
Helen Thackray: To do this, we'll be looking at drug levels in the epidermis, both by using adhesive tape to collect skin epidermal cell samples and with small skin biopsies.
Helen Thackray: We would be thrilled to see the presence of 1775 in those samples and normal levels of skin, calichrine, enzymatic activity achieved in patients who have nethertian syndrome, which would provide a strong signal of the potential for clinical efficacy.
Helen Thackray: In addition, we'll be looking at clinical impact. If we don't see clinical benefit at a studied dose level and dosing it safe, we intend to progress it to higher doses.
Helen Thackray: If we do see clinical benefit as indicated by improvement in measures such as itch, score, and physician, global assessment of severity, we'll have excellent information to inform dose selection for a future pivotal program, and even higher confidence in the opportunity for 1775 to be a truly disease modifying therapy for people living with Netherton syndrome.
Helen Thackray: Although it's always a challenge to conduct clinical trials and rare diseases, we have plenty of experience with this and we're very encouraged by the clear enthusiasm for our trial that we heard during our recent clinical flight visits. We look forward to sharing our initial phase one trial data in patients from Tethersine Syndrome by year end.
Jon Stonehouse: and now I'll hand the call back to Jon for a financial review.
[inaudible]
Thanks, Helen.
Jon Stonehouse: We are exiting the first quarter in a great financial position with stronger than expected revenue growth increasing full-year revenue guidance, accelerating full-year profitability to this year and last month we've reduced our outstanding debt.
Jon Stonehouse: While you can find our detailed first quarter financials in today's press release, I'd like to draw your attention to a few items.
Jon Stonehouse: Total revenue for the quarter came in at $145.5 million, $134.2 million of which came from Orliteo.
Jon Stonehouse: Of that Orla Dayo Total Revenue, $120.2 million or 89.5% is coming from the US.
Thank you.
Jon Stonehouse: Operating Expenses, Excluding Stock-based Compensation, were $102.9 million for the quarter, up from 93.6 million in the same quarter last year.
Jon Stonehouse: This was primarily driven by an increase in commercial expense to support our growing or the day of revenue, our newly launched regions like Spain and Italy, and expanded international operations including global commercial support activities across finance, HR, IT, and supply [inaudible]
Jon Stonehouse: Operating profit for the first quarter of 2025 was $21.2 million, and net income was slightly positive.
Jon Stonehouse: Cash, at the end of the quarter, was $317 million
Thank you.
Charlie Gayer: As a result of the significant and durable improvements to revenue that Charlie discussed, we are revising our revenue guidance by $45 to $50 million above our prior guidance.
Charlie Gayer: in which represents, as I mentioned before, a 33-37% growth over last year.
Charlie Gayer: We now expect non-GAAP operating expense for the year to be $440 to $450 million, a $15 million increase over our prior guidance driven by expenses supporting our commercial growth and an increase in cogs as sales of RepoLab increased.
Charlie Gayer: The result of this strong revenue performance combined with our continued focus on discipline capital allocation is that we now expect to be profitable for the full year on a net income and positive cash flow basis this year. This is one year earlier than we previously planned.
Charlie Gayer: Further, based on this improved financial strength in April , we made a paydown of $75 million on our debt with Pharmacon and reduced our outstanding debt to $249 million.
Charlie Gayer: As a result, we expect to save approximately $23 million in interest payments over the life of the debt net of the early prepayment penalty.
Charlie Gayer: I am immensely proud of the continued focus of our employees who are delivering meaningful improvements in patients' lives, both today and in the future.
Charlie Gayer: Our commercial and support teams are driving access and usage of Orlodeo. And our R&D team is advancing our pipeline to address challenges in new disease areas where patients are underserved.
Charlie Gayer: Financially, we are driving revenue growth and well on our way to $1 billion at peak, efficiently allocating capital in R&D to create sustainable and long-term revenue growth, accelerating our profitability and reducing our outstanding debt.
Charlie Gayer: We have never been in a stronger financial position and I am excited for how this sets us up for a very bright future.
Operator, we are now ready for your questions.
Thank you.
We will now begin the question and answer session
Charlie Gayer: To ask a question, you may press star then one on your telephone keypad.
Charlie Gayer: If you are using a speakerphone, please pick up your handset before pressing the keys.
Charlie Gayer: If at any time your question has been addressed and you would like to withdraw your question, please press star then two.
Charlie Gayer: At this time, we will pause momentarily to assemble our roster
Thank you.
Speaker Change: Our first question comes from Steven Seedhouse, from Cantor, please go ahead.
Good morning. Thanks so much for digging the question.
Just looking ahead to another potential tailwind.
Speaker Change: for Orlidae, which is the pediatric launch. Do you have a sense of
Speaker Change: How many pediatric HAE patients are on TaxiRot today in the U.S.? And do you think you'll mainly be switching children?
Speaker Change: from TXIRO, or can you quantify how many patients maybe just don't want a needle but would be a new start opportunities for Orlidae of Prophylaxis altogether?
Speaker Change: Charlie, you want to take that one? Sure. We certainly do expect pediatrics to be a tailwind for us and just as a reminder, any future revenues for pediatrics are not included in our 800 million in the US or a billion dollar global peak sales.
Speaker Change: Tancy Recursor, I don't know exactly how many patients are on taxiral. We do think that there are about 500 patients, pediatric patients in the U.S.
Speaker Change: and at least 200 of whom could be appropriate for prophylactic therapy. But once an oral is available, we actually think that that could grow. And so I think our where we'll get patients is a mix of...
those switching from injectable therapies like TaxiRO.
Speaker Change: But we also think that the whole landscape for treating kids under age 12 could change.
Speaker Change: and that more kids could start prophylaxis at a younger age to prevent attacks and really change the course of their life with the condition. And an oral therapy really makes that happen because I would say there's literally no preference for an injectable for children.
Speaker Change: So we think we'll be very well positioned, we're excited about it.
Speaker Change: The trauma of sticking a needle into a toddler, we should be the market leader. There's no doubt in our minds that that's going to be the case and I think the other thing that Charlie's referenced in the past.
is this halo effect. We still have some parts. [inaudible]
Speaker Change: HAE Physicians that haven't started switching patients and we know they have pediatric patients and there's no reason for them to not start using our drug for pediatric patients and when they see how well it works [inaudible]
Speaker Change: Our expectation is that that's going to expand their usage, so we're really excited about this. We haven't put any hard numbers on it yet publicly, but we're really excited about it.
Great.
Speaker Change: also on DME program, so it looks like you're guiding for data this year, I'd be just curious if you could frame what you would do is a successful outcome for that first first readout, given the precedent data, given your, you know, obviously the novel delivery method just
Speaker Change: What do you hope and see there in the first group of patients?
Speaker Change: Yeah, so we'll have our first patients on drug. As I said, we have authorization to proceed with enrolling patients in that trial. And what we're looking for is, you know, the obvious safety and tolerability, but more importantly, to your question, we're looking for the effect on the thickness of the retina, the demon in the retina. So we'll be able to, in those first patients, watch over several months of exposure to assess that retinal thickness.
Speaker Change: and whether that is changing. We do expect it will take a little bit of time to change but not very long and I point to some of our...
Speaker Change: And DME patients could tell us a lot, and so we're excited to see what we see at the end of the year.
Thank you.
Thanks so much. You're welcome.
Thank you.
Speaker Change: A next question comes from the line of Mari Raycroft from Jeffries, please go ahead.
Maury Raycroft: Hi, good morning. Congrats on the update today, and thanks for taking my questions.
Maury Raycroft: I'm wondering if you just talked more about getting patients onto paid drug and what you did there that worked better than expected and should we expect the rate to be maintained or improved upon going forward or what are some of the drivers you're focused on that could cause the rate to decrease again over time.
Speaker Change: Sure, Murray. The number one thing that we did, and we've talked about this before, is we've, over the last couple of years, made investments in our team to make sure that we're really prepared and really expert in this reauthorization process. Let's go.
Speaker Change: and we've gotten better every year and we knew that there was this potential tailwind with Medicare this year. We also knew there was more space that we could improve in the very important commercial segment.
Speaker Change: and so I would just say that the team has just... [inaudible]
Speaker Change: reached a level of excellence that allowed them to execute this year.
and we're looking forward to further improvements, going forward.
Speaker Change: Our paid rate has driven more by new prescriptions as opposed to reauthorizations.
Speaker Change: But where we ended Q1, where we ended April becomes the floor for what I would expect in the future. And I'm very confident we'll reach our 85% goal. We'll reach the head of when we expect it to originally. And we'll be looking to exceed it if we can. We'll reach the head of when we expect it to originally. We'll reach the head of when we expect it to originally.
Speaker Change: The other thing I'd say is, you know, you should take a look at that slide that Charlie referred to around the pattern of revenue this year. It's different, right? So we pulled forward revenue that would usually be a big bump from first quarter to second quarter into the first quarter. So you're going to see less of a bump. [inaudible]
Speaker Change: I think the other thing though that's exciting is that the underlying growth continues to be really strong and so you know that's where we're going to see incremental improvement over the course of the year and we're really excited to share that with you.
Speaker Change: Got it. That's helpful and maybe just as a follow up, if you could just provide more specifics on what you did to improve Gerstinette in first quarter and what you're doing to better improve that over the course of the year as well.
Maury Raycroft: Yeah, so a couple of things more, one big, big impact was just the fact that we got so many more Medicare patients paid.
and that changed the overall growth to that mix. [inaudible]
Maury Raycroft: I'm really pleased that we made that kind of a progress in the first quarter, and I think that will be durable through the rest of the year
Got it. Okay, thanks for taking my questions.
Welcome.
Speaker Change: Thank you. Your next question comes from Jessica Fye from JP Morgan, please go ahead.
Jessica Fye: Hey guys, good morning. Thanks for taking a question. I was curious more specific to the guidance bump for Orlidaeal. Can you quantify how much of that increase is related to partly redesigned and higher proportion of paid patients?
Speaker Change: How does that impact how you think about investing in the business? And I guess more specifically could that allow you to accelerate any development plans for your earlier stage pipeline? Thank you.
Thank you.
I can take the the first part of that. [inaudible]
Speaker Change: As I mentioned in my remarks of the 10% improvement in paid reign.
Speaker Change: About two-thirds of that was related to the IRA helping patients afford their copay, so that's great.
Speaker Change: But what I'm really excited about is how much progress we made in the 60% of patients who are on commercial.
Speaker Change: Insurance, where we improved by close to 5% over where we ended last year ending up at 84%, so that was the other third of that improvement.
Speaker Change: Clearly, in terms of raising our guidance for the year, that increase in paid rate overall was the number one factor.
Speaker Change: But we have an underlying patient growth year over year close to 20% and as I mentioned we had one of our best years ever last year and in the first quarter we were a little bit better than that and so that underlying demand is what's going to drive this brand going forward. [inaudible]
Speaker Change: And then, John , yeah, I'll take the profitability. So yeah, it's territory we haven't been in before and are really excited to be in it.
Jon Stonehouse: And then in terms of what are we investing, I think we gave you a pretty good clue by paying, making a paydown of 75 million of the pharmacondes Cleaning up the balance sheet not having to pay significant amounts of interest over time is something that we'll continue to look at as cash flow comes into the company. [inaudible]
Thank you.
Operator, I think we can go to the next question
Speaker Change: Yes, thank you. The next question comes from the line of Brian Abrahams from RBC Capital
Speaker Change: Hi, everyone. This is Nevin on For Brian . Congrats on the quarter and thanks for taking a question. So just following up from some of the prior questions. So
Speaker Change: Given that you're on around 84% paid fraud and also that you're on the lower end if you're grossed in net rains for the year.
Speaker Change: Does this potentially indicate that there was a dip in the total patients that were on Orliteio and like how do you see that kind of evolving into the rest of the year. [inaudible]
Speaker Change: Well, I can tell that there wasn't a death in patients on all the day, so I'm maybe not quite understanding your question, Nevin. We've had extremely consistent demands. So, you know, again, last year...
Speaker Change: We had as many new patient starts as we did in the first year of the launch and then we did a little bit better than that in the first quarter.
Speaker Change: The retention rate of patients has also been extremely consistent the last couple of years where anybody who starts on Orladeo, we get about 60% of those patients to stay on therapy through the first year and then very few drop off after that.
Speaker Change: So what we're seeing in patient demand is as strong, if not stronger than we've ever seen it. And then you add on top the paid rate and the improved growth to net. It just means that we're more profitable.
Speaker Change: and so we still see reaching a billion dollars in 2029 but the path to get there every year with that underlying demand is just going to be more profitable than we previously expected. So it's a great position to be in. [inaudible]
Speaker Change: Yeah, I think, Nevin, I don't know if you're referring to that slide of the pattern of sales, which, you know, because of the...
Speaker Change: Fast, Change in free drug to pay, the patterns different than previous years and it'll probably go back to the same pattern as previous years next year, but there is nothing.
Speaker Change: that is slowing down and we're really excited about the underlying demand and continue and there's more patience to get.
Speaker Change: Quite frankly, I think the last update Charlie said is around 3,000 patients right at the end of last year And there's 10,000 available so there's plenty more to get so no signs of slowing down [inaudible]
Trump Exchange and Pattern [inaudible]
Speaker Change: Got it. And then I guess on the uptick in patients that you're seeing for preference for oral prophylactics, what do you think is kind of driving that underlying dynamic there have rights of maybe needle phobia change sure it's just
Speaker Change: A convenience factor that's being appreciated more, I say, kind of the, the, you know, more maturing launch of all of the day, and the convenience of the offers.
Speaker Change: Thanks for that question. I think what we've known since before launch that fundamentally the great majority of patients prefer oral therapy to injectable.
Speaker Change: But what's really important to patients is that they get control of their HE attacks so they'll never sacrifice efficacy for convenience against them.
Speaker Change: But now four plus years into the launch, what they're seeing is with all of our efforts is with Orlando, you can have both. You can have great efficacy.
Speaker Change: You can have convenience, and so the patients have seen it themselves, they've heard...
Speaker Change: from their peers. It's a very connected community within HAE. And then their healthcare providers are just that much more confident, which always is a critical factor that the physicians. [inaudible]
Speaker Change: Have confidence in a drug. And so you put all this together with our the efforts that we've made from a sales and marketing perspective. It's natural that patience.
Charlie Gayer: Underlying Preferences is coming out and I think that's why we've seen it grow by about 20% over the last couple of years and I you know I wouldn't be surprised if it continues to grow. I think it was around a year ago we we told you Charlie told you that. [inaudible]
Charlie Gayer: There was a momentum change and there was a confidence build up and that was because more and more physicians were having positive experiences with their patients.
Charlie Gayer: Patients Talk, and doctors talk about other patients to their patients saying, you know, I have a patient that does really well on Oralidaea, you might want to consider trying it. And so it's that confidence in the product that I think is reflected in the market research.
Charlie Gayer: Ray, thank you all so much and I can grab the end of the quarter.
Speaker Change: Thank you. Thank you. Your next question comes from Stacy Ku from T.D. Corwin. Please go ahead.
Stacy Khoo: Thanks so much for taking your questions and congratulations on the really impressive quarter.
Um, so first, as we think about these...
Additional patient additions and strong patient demands.
Speaker Change: You've previously added around 60 prescribers per quarter. What are your expectations for this year? And then some follow-ups on the other hands.
Speaker Change: Are you able to make any disclosures on the phase one early sad matter results? Are you able to disclose anything at this one in time? Or can you narrow the timeline for any updates in the vision?
and then I have a follow-up on that agenda.
Stacy Khoo: Okay, I'll start with the healthcare providers. Good memory, Stacy. Last year we actually averaged 61 new healthcare prescribers for Orlando per quarter.
and in Q1 we had 59, so it's incredible. Thank you very much for your time.
Consistency at this stage in the launch that we're still
Stacy Khoo: We're still convincing new prescribers, our team will always go out and find new prescribers. The other thing though is, and Jon touched on this earlier, but we've made progress in the pure one position. So 81% of those doctors have now prescribed.
Stacy Khoo: and we're seeing more and more physicians become repeat prescribers, and even some returning who prescribed some years ago, now returning now that they better understand the product. So the physician dynamics are excellent along with the overall patient dynamics.
Helen Thackray: And then, Helen, you might want to talk about how we're thinking about packaging all the data towards the end of the year. Yeah, so in terms of the results on the said mad Stacy, we've been following that trial. We have it's intent with initial safety and PK data. We have the confidence now from that to open the dosing and patients. You should see that as a sign of generally what we're seeing. And then what's most important and we've been clear about looking for the skin outcomes and patients. [inaudible]
Helen Thackray: with most important is what happens in patients. So we'll bundle it all together. It'll be by the end of the year. It will have the updates from the healthy volunteer data, but then more importantly that outcome in patients than are we at the dose against skin healing. So in healthy volunteers, no news is good news.
Helen Thackray: Okay, wonderful. Well, I guess that leads to my then follow-up about in other terms. And he's talked about kind of the early interactions of the investigators in the activations of sites, but
Maybe nuanced Part Four of the Facetime Study.
Speaker Change: Sounds like you're getting at least some sense of safety. Could you broaden the POC and the
Part 4 is the extension of treatment. The treatment, it is...
Speaker Change: Design so that it's additional patients. So what we're hearing at the sites, we're hearing clear enthusiasm, we're hearing that a potent KLK5 inhibitor that could target at the top of the cascade is something that physicians want to try in their patients with an other syndrome. It'll be the first few patients who are on that part three and then we're sort of continuing. We'll open part four and continue and broaden enrollment. Thank you very much.
Speaker Change: to get a longer term exposure. I would think of them as sort of going together rather than being separate. And the result is, by the end of the year, we'll have patients on, we think, both sections with a longer term follow-up on those who've been doced in part three, and then more recent follow-up in those doced in part four. [inaudible]
I think one other thing, Stacy, that... [inaudible]
Speaker Change: I'm certainly learning as we learn more about this community and both the physicians and the patients is there are some significant differences from HA right there's no therapies in place
Speaker Change: The organization for advocacy isn't nearly as advanced as HAE was so those are challenges and finding patients but the flip side is the hope we're offering these patients is [inaudible]
Off The Charts
Speaker Change: Right? They were hopeless or they have been hopeless and and coping
Speaker Change: because they have nothing to treat the underlying cause of their disease. And when we start talking to patients...
Speaker Change: and tell them what we're trying to work on. I mean, it brings them to tears and so that's powerful. And so if we can find these patients and I'm confident we will and this drug gets to the skin, we've got something really powerful to help them and give them hope.
Wonderful, thanks so much [inaudible]
You're welcome.
Speaker Change: Thank you. The next question comes from Gina Wang from Barclays, please go ahead.
Speaker Change: Gina Wang, your line is unmuted, please proceed with your question.
Oh, thank you. Thank you for taking my questions. I have two questions.
Speaker Change: I think that I join a little bit late, so if you've already discussed, and I heard some of the answers, if I already discussed, I apologize. So for your paid...
Ray, truly very impressive, like 10% over 10% increase from...
Speaker Change: The last quarter is 73.5, now improved to 84%. You did a comment when you answer numerous questions, various parts.
But can you help us understand? And, like,
Speaker Change: We'll make it in such a short time that has such a big improvement. Can you walk us through the actual process where you did make it possible in a short time? We'll make it possible in a short time that has such a big improvement.
Speaker Change: to see such a high increase regarding the pay rate. And then the second question is regarding
Speaker Change: You will have a patient data later this year. Could you give us some benchmark you are looking for that you will consider that will be encouraging to move forward?
Speaker Change: Yeah, hey Charlie, maybe I'll start first on this first one and then you can answer the rest I want to remind everybody that when the Medicare negative thing happened it snapped the other direction very quickly like within a quarter so this is a snap back but I'll let Charlie explain it more detail. [inaudible]
Charlie Gayer: Yeah, so again, Jean, one thing I explained is about two-thirds of the improvement was Medicare, and as Jon said, that was a quick snapback.
Charlie Gayer: But it was because our team prepared, and so the snapback is, and I've always said this, that Medicare plans
Charlie Gayer: Approved Orlando at the highest rate amongst all insurers that we see. It was just an affordability thing for patients. So it's great that the IRA is helping patients afford.
Charlie Gayer: and our paid rate amongst the 20% of our patients who are in Medicare is now 89%, so that kind of underlines what I just said about it being the top segment for approvals.
Charlie Gayer: The other piece, though, is the team really prepared on the commercial side.
Charlie Gayer: And one of the preparations is we have all this long-term real world evidence. So what we know about the drug and the efficacy today is very different from what we knew four years ago.
Charlie Gayer: and so the team is able to use that more proactively with payers and it's convincing more and more payers that this is a very effective drug and therefore they're choosing to pay for it for the patients. That's where I think we can still make progress.
Helen Thackray: But it's about the data, the preparation, and then also the intended impact of the IRA coming true. The other thing before I turn it over to Helen on the DME is...
Speaker Change: Back when patients were struggling to make their copay, we made a very conscious decision to give them free drug.
Speaker Change: And we kept the patients and we actually gained patients as a result of that strategy. So then when the affordability got back to normal, those patients snapped back quickly. And I think strategically that was in hindsight a good move to me.
Speaker Change: Helen, you want to talk about DME? Yeah, so because we have authorization now to dose in patients this is this is a big milestone for this program. We are looking this year to have exposure and experience with a few patients with that single dose as John mentioned earlier. This is a this is a drug with longer ability of exposure and we're looking for that the outcome the retinolidema. Yeah.
Speaker Change: Lessening, reduction in the size of the retinal thickness. We're looking for that and we'll be looking for that for the end of the year. In terms of what then is the benchmark to move forward we're looking for a really big effect. [inaudible]
Speaker Change: We're looking for a clear and uncontroversial reduction in edema that would then tell us that we have a dose level that's got the exposure necessary to have that effect. If we don't see that, we will increase the dose so that we'll either see that a very significant reduction in edema know that we have a dose we can take forward or will not have to achieve that yet and we'll expect to increase the dose until we do.
Speaker Change: Yeah, I think we've always said we're looking for signals of activity and starting to get a sense around dose and that's exactly what Helen said when you have three patients per cohort . . .
Speaker Change: You know, there's a limited amount of information that you can get, but I think-
Speaker Change: If we can see some sort of effect and duration of effect that gives us encouragement, nobody else with a chronic inhibitor has done that yet, right? And so that would be very exciting for the program.
Speaker Change: Sorry if I can follow up. So how many micron reduction you will consider clear and uncontroversial?
Speaker Change: So I would use the within the literature for what is, you know, generally accepted as something that's going to be predictive of likely response and visual acuity. I can't give you a number today, but I can tell you we're looking for uncontroversial difference, so not something that's small.
Okay. Thank you.
Thank you.
Thank you.
Speaker Change: Thank you. Your next question comes from Laura Chico from Red Bush Securities, please go ahead.
Speaker Change: Good morning. Thanks very much for taking the questions. First one is just going back to the Pediatric Formulation for Orlodeo. Can you just remind us on maybe some of the timelines after a pediatric launch arrives? Would there be a lag in deployment to the channel upon approval just getting material out for supply? And then as we're thinking about the cadence about taking a pediatric population versus the adult, just kind of curious what dynamics we should be thinking about that might impact that up? Take a little bit differently. So what we're seeing in
with the adults and I have just one quick follow up.
Sure.
Speaker Change: We will try to make the leg as small as possible.
Speaker Change: When we launched capsules back in 2020, it was only two weeks from approval to being in the channel. That was amazing. I don't know if we'll go quite that fast, but we're looking to get it out as quickly as possible. The other thing we'll do is prepare our team.
Speaker Change: Prophylaxis really hasn't been the standard of care for kids under 12
Speaker Change: The historical conventional wisdom is that kids become symptomatic mainly around puberty. I think what we saw in our clinical trial is...
Speaker Change: For many of these kids, that is very not true. Many of these kids were, in fact, I think the average age of symptoms was age 2.
Speaker Change: And so changing the way that the whole treatment paradigm for children, if you could prevent attacks.
Speaker Change: So that patients could avoid that fear, so parents could avoid that fear. It could really change the course of a kid's life living with HAE and so I think that'll be the biggest change and as Jon and I both said earlier, I think an oral formulation is going to be the natural choice. [inaudible]
Speaker Change: for that starting prophylaxis with the kids. So we'll see how quickly it goes, but we're hearing a lot of enthusiasm from both healthcare providers.
Speaker Change: and the patient community for the change that I'm describing. One of the things we learned with adults.
Speaker Change: is it takes longer than you think and so that's why we're not getting out over our skis here on you know giving you guidance on the uptake because we just don't know yet we were confident that we will be the market leader because we believe we have a formulation that is way more acceptable to little kids than injectable.
Speaker Change: but the pattern to get there is, it's just too early to have predict.
Speaker Change: And I said, okay, thank you for that one. And then one quick follow-up on medicine. Can you remind us the dosing range you're going to be starting with for 1775? And then I just wanted to clarify, I know you mentioned.
Speaker Change: Advancing later to 12-week-go thing, but what is the actual trigger for that? Is that dependent on accruing sufficient four-week data?
Speaker Change: and the initial cohorts. Any clarity there? It would be helpful. Thank you. Yeah, sure. So in terms of the dosing range, we have experience with a fairly broad dosing range now in healthy volunteers, and we'll be dosing patients somewhere in the middle of that range.
Speaker Change: Dosing initially in part three, learning from that as we initiate part four, both going in parallel with ongoing observations over time. So, as I said earlier, it's more like a continuous set of two arms that go together.
Speaker Change: Yeah, and if the drug works, we'll have an ability to keep patients on drugs.
Thank you.
Thank you guys, congrats on the quarter.
Thank you [inaudible]
Speaker Change: Thank you. An ex-question comes from Liisa Bayko from Errico ISI. Please go ahead.
Lisa Baker: Hi there, thanks for taking the question and congratulations on a strong quarter. Two parts for my question. First is...
Lisa Baker: I just want to make sure I understand that the paid rate, improvement that you saw, you've talked about it kind of kind of being like a switch-up flipside. Is there any chance this flip's back? Can you talk about the sustainability?
Lisa Baker: of this paid rate improvement as we head into next year and future years. And then part two is really, can you talk about the patients on treatment today and then walk us through the kind of remaining pool of patients out there, at least in the U.S.
Lisa Baker: Who have not yet tried Oralida on your plans to kind of increase utilization there? Thanks.
Sure. Thanks Lisa. The paid rate...
Lisa Baker: I believe will be very sustainable, based on the evidence that we've shown for Oral Dayout.
Lisa Baker: It's really changing our conversations with payers, and so I think that 84% is kind of our new floors, we look forward over time.
Lisa Baker: The patients on therapy, so as I mentioned earlier at the end of last year, 3,000 patients in the U.S. have tried out of a wolf about 10,000.
wants to date. [inaudible]
52% of our patients have switched from other prophylaxis [inaudible]
Lisa Baker: About 30% just over 30% have switched from acute only and the remaining 16, 18% whatever it is our patients best we can tell were naive to therapy.
Lisa Baker: And so that last piece I'm excited about because what it shows is that this is a market that is still growing. We've known that the market for prophylaxis has been growing over time, but we're finding new patients out there and they're coming to prophylaxis.
Lisa Baker: And so we have at least another 7,000 or so patients who have not yet experienced Orlidao and many of them will be appropriate for prophylaxis.
and the the growing healthcare provider enthusiasm.
Lisa Baker: and the fact that we're getting paid at such a high rate, it just increases confidence overall and I think we'll continue to drive or the day out going forward.
Lisa Baker: Yeah, and if you think about it, if at the end of the fifth year since we got approval, we're at about a third of the patients that have tried it and we're and we're at 580 to 600 million, we are definitely on our path to $1 billion.
Okay, great. Thank you so much.
Welcome.
Thank you.
Speaker Change: The next question comes from Serge Belanger from Needham & Company, please go ahead
[inaudible]
Speaker Change: Good morning and congrats on the nice quarter. A couple or a day, or a day of questions for Charlie. Now that you've reached your
Speaker Change: Target for a paid rate of 85%. Should we expect continued improvements on that number? Is there more to do on a commercial or Medicare side? And then just to follow up on the patient ad questions.
Speaker Change: Celtic 1-2 was one of your best quarters ever in terms of new patient ads. Just curious if you're noticing a trend on...
Speaker Change: Where these patients are coming from is, if it's still 50 percent...
Speaker Change: Profi, and 30% acute, or is that next changing with this new batch of new patient ads? Thanks.
Sure, thanks, sir.
Yeah, I think we're almost to our long-term goal [inaudible]
Speaker Change: 85% little, little more room there, but what we'll do is we'll probably up that number, you know, we'll keep striving to get better. I mentioned just a minute ago that in the Medicare segment, there's 20% of our patients, the paid rate is now 89%.
Speaker Change: and so that shows that we can improve past the 85% and we'll do everything we can to keep getting better. I don't expect another big tailwind like we had this year, but it's just now going to be incremental improvements.
Speaker Change: In the first quarter, yeah, fundamentally, the patient base was very similar to what we've seen since the beginning of launch. The other thing I'd add is...
Speaker Change: The mix of prescribers that I haven't completely discussed today, but in the first quarter, 55% of the prescriptions were from our top tier of doctors, those physicians that we know treat about 50% of all HE patients.
Speaker Change: and then the other 45% came from everyone else. So really balanced across the board. I mentioned earlier we've had 59 new prescribers in the quarter and so we just keep capturing more opportunity and we keep capturing it from all the different segments.
Speaker Change: which gives us that extra confidence in the sustainability of this growth over the next several years.
Thank you.
Thank you. Bye.
Operator, we can go to the next question
Perfect. Thank you.
Speaker Change: Your final question comes from Jon Wolleben from the cities and please go ahead.
Speaker Change: Thanks for taking the questions. A couple of them, Netherton, for me.
Speaker Change: Can you remind us of your pre-clinical talks work that you've completed so far? And then, Helen, when you talk about seeing a clinical impact, determining, you know, dosing higher, what metrics will you be looking at and what's your bar entirely to determine whether or not you're in a sufficient spot in terms of efficacy?
Yeah, sure. It's a pre-comical talks.
Speaker Change: So this is the biological protein therapeutic. There's a fairly standard safety profile for protein therapeutics, and we're seeing a very consistent profile with that. I think the other thing to look at with pre-conical talks is we have the authorization now to proceed not only in healthy volunteers but also in patients, and that includes authorization in the US and Australia. So just indicative of that.
the range of... [inaudible]
Speaker Change: Confidence, coming from preclinical talks data. Yeah, and Helen, I would add we have an open I&D and we have the ability to dose in part three up to four weeks and part four up to 12. Yeah, so that gives you some sense of
Speaker Change: The duration of toxin in the life. Right, so standard toxicology to support that.
Speaker Change: In terms of the clinical impact, so what we're looking for is really normalization of the skin.
Speaker Change: So that means we're assessing by how the patient feels, father's skin, is there itch still? And we want to see the itch really go away. We're looking also to physician assessment of the skin and the severity index for sort of thickening and scaling of the skin. And so we're looking for that redness to go down and for the scaling to stop. I expect that we'll see some of that initially. I expect that it will probably improve over time.
Thank you.
Speaker Change: How many patients per cohort, can patients in part three be included in part four, and then how do you think about the time dependent nature of these improvements?
So I...
Speaker Change: It's a small, part three is smaller than part four. We could see what we need to see in single-digit patients, so it's going to be fewer than ten in part three. Part four will be slightly larger, but it's still ultra rare disease, and every patient will inform whether we're at the dose and seeing what we need. So it'll be larger, but not much larger in part four, maybe more than ten.
In terms of the time dependency, we just don't know. We do know that
Speaker Change: We expect the skin turnover and other skin syndrome to be about two weeks. We do know that we would expect healing to occur as the skin is turning over so we may be seeing
Speaker Change: Clinical Finds, as quickly as four or eight weeks in this. We also may be seeing continuing improvement and healing of the skin because the inflammatory components take a little bit longer to heal. But our goal is to see whether we are getting that initial quick results in the short term for a week time frame after end of dosing, and then observe over time as we continue to expand part four and be able to dose patients for longer period of time.
Super helpful. Thanks, Helen.
Thank you.
Jon Stonehouse: This concludes our question and answer session. I would now like to turn the conference back over to Mr. Stonehouse for closing remarks.
Jon Stonehouse: Thank you. So, I mean, it was a great quarter. I don't know what else to say. And I'll repeat what I said earlier. I am immensely proud of the employees of BioCryst for delivering the results of this quarter. But count on us to continue to be executing. We're not going to rest on our laurels. You know, there's a lot more to do, a lot more to deliver over the course of the year. And you can count on us for continuing to work as hard as we can to do that. So thank you for your interest and we'll keep you updated along the way. Have a great day. Thank you.
Thank you.
Speaker Change: The conference has now concluded. Thank you for attending today's presentation,
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