Q1 2025 Ionis Pharmaceuticals Inc Earnings Call
Good morning, and welcome to the Ion is first quarter 2025 financial results Conference call.
As a reminder.
Speaker Change: This call is being recorded at this time I would like to turn the call over to Wade Walke Senior Vice President of Investor Relations to lead off the coast.
Please begin.
Amy: Thank you Amy before.
Speaker Change: Before we begin I encourage everyone to go to the investors section of the website <unk>.
Speaker Change: The press release and related financial tables that we will be discussing today, including.
Speaker Change: Including a reconciliation of our GAAP to non-GAAP financials.
Speaker Change: We believe non-GAAP financial results better represent the economics of our business and how we manage our business.
Speaker Change: We've also posted slides on our website that accompany today's call.
Brent Maya: With me. This morning are Brent Maya, our Chief Executive Officer, Al G&A, Chief Global product strategy Officer, and Beth Hougen, Chief Financial Officer.
Brent Maya: Richard Gary Chief Development Officer, Eugene Schneider, Chief Clinical Development Officer, Eric Swayze Executive Vice President of research and Jonathan Virtual Chief Commercial Officer will also join us for the Q&A portion of our call.
I would like to draw your attention to slide three which contains our forward looking language statement. During this call we will be making forward looking statements that are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties and our actual results may differ materially.
Brent Maya: I encourage you to consider the risk factors contained in our SEC filings for additional detail.
Brett: Now I'll turn the call over to Brett.
Brett: Thanks, Wayne Good morning, everyone and thanks for joining us today.
Brett: Before we discuss our Q1 earnings and recent progress I want to take a moment to acknowledge the rapidly evolving environment, we're living in today.
Brett: Recent changes at the FDA and the introduction of new tariff policies have introduced a degree of uncertainty with the potential for disruption in our industry.
Brett: Despite these external uncertainties <unk> remains well positioned to execute on our strategic priorities, including all of our many value driving catalysts coming up this year and next.
Brett: We remain focused on our purpose to drive meaningful value for all stakeholders by successfully bringing better futures to people with serious diseases.
Brett: We are executing very well against all our strategic priorities.
Brett: A clear example of our ongoing success as the completion of two strategic licensing transactions recently.
Brett: Which enabled us to substantially increase our 2025 financial guidance.
Brett: This includes higher expected revenue in cash along with improved operating loss.
Brett: Raising guidance. This early in the year reflects our confidence in our ability to continue executing well, including our ability to deliver transformational medicines to patients even amid the current volatility and our commitment and expectation to drive long term value for shareholders.
Brett: Sure.
Brett: We achieved a major milestone priorities with our first independent commercial launch now successfully underway.
Brett: I am pleased to share that in its first full quarter on the market turn goals are the first and only FDA approved treatment for familial <unk> Micronesia syndrome exceeded expectations.
Brett: This encouraging start flex our commercial teams thoughtful planning outstanding execution and highlights the commercial capabilities that we have built.
Brett: Right behind <unk> is our second independent launched onto the worsened for hereditary angioedema or <unk>, which is rapidly approaching.
Brett: Sure.
Brett: The team is laser focused on ensuring success.
Brett: <unk> has the potential to become a preferred treatment for <unk> and we remain on track to launch in the third quarter.
Brett: These launches are just the beginning we are poised to report data from two <unk> one phase III programs later, this year, which if positive will enable us to continue delivering a steady cadence of important medicines to even more patients.
Brett: Our second indication for <unk> in severe hybrid triglycerides EMEA, our CTG, a large population with high unmet need and yoga nursing for Alexander disease is severe rare lipid dystrophy with no approved therapies.
Brett: Collectively these four programs represent needed patient breakthroughs and multibillion dollar revenue potential for our owners.
Brett: Additionally in the next three years, we expect four launches from our late stage partnered medicines currently in development to treat a range of serious life threatening diseases.
Brett: These medicines are poised to significantly increase our revenues by expanding the reach of ion just discovered medicines to more patients that are in need.
Brett: With the recent launch of <unk> and our upcoming independent and partnered launches I honest is on a path to bring important medicines to patients for years to come.
Brett: <unk> us to achieve substantial and sustained revenue growth and positive cash flow.
Kyle: With that I'll turn the call over to Kyle.
Kyle: Thank you Brett with our first independent launch underway in three additional independent launches planned in the next two years, our commercial team is executing on a focused agenda with substantial growth potential.
Kyle: We're well positioned to build on the early success of <unk>, while we prepare for our other upcoming launches Danita Lorson for HCA is on track for anticipated approval in August followed by <unk>, which is on pace for phase III data in the third quarter and a potential launch next year.
Kyle: Let's start with <unk>, which had an encouraging debut in the first quarter, our commercial team executed seamlessly delivering on all initial launch objectives and exceeding expectations with over $6 million and net product sales in the first quarter.
Kyle: In this first full quarter on the market, we saw strong patient uptake driven by multiple factors.
Kyle: First we successfully converted nearly all of the U S expanded access and clinical trial patients to commercial product.
Kyle: Second as triangles as the first and only approved FDA treatments for FCS early uptake benefited from patients who had been previously diagnosed and we're waiting for treatment.
Kyle: And third our patient finding efforts ahead of launch paid off contributing to scripts from newly identified and diagnosed Fcs patients.
Kyle: Engagement has also gained meaningful traction we've been encouraged by the initial breadth of physicians prescribing touring golda with scripts from a mix of specialists across the country.
Kyle: At the end of Q1, the initial prescriber mix included approximately 50% cardiologist and 25% endocrinologist with lipid apologists in internal medicine hcp's comprising the rest.
Kyle: Physicians have also reported positive and deeply moving feedback, including one doctor who shared that he was quote over the moon that this was the lowest as patients TG levels have ever been.
Kyle: On the access front coverage and reimbursement dynamics have been favorable coverage split for patients answering goals are to date is just over 60% commercial and just under 40% government.
Kyle: Importantly, patients whether clinically diagnosed or genetically confirmed have received timely access with most receiving coverage through the medical exception process based on clinical diagnosis.
Kyle: This underscores both the urgency of the high unmet need and payer willingness to support access ahead of establishing formal policies.
Kyle: As payers establish coverage policies over the next several months it will be important to these policies support both clinical and genetic diagnosis pathways to this end our market access team is actively engaging with payers to enable continued patient access through the first quarter. The team had already engaged with payers representing the vast majority of covered lives in the U.
Kyle: And are working toward getting additional policies in place.
Kyle: We're also seeing positive trends beyond coverage, including reimbursement timelines that have exceeded our internal benchmarks and in the first full quarter of launch nearly 90% of patients had zero out of pocket costs, highlighting the effectiveness of our ion US every step support program.
Kyle: I almost every step has been instrumental in delivering high quality experience for both patients and providers more than 90% of patients have opted in receiving support that includes disease and nutrition education.
Kyle: Auto injector administration training reimbursement support and more.
Kyle: For health care providers, the program streamlines prescribing by handling insurance authorization and coverage assistance as well as coordinating delivery re authorizations and refills.
Kyle: We're proud of metering goals as early momentum, but we know we're still in the early innings. The vast majority of the estimated 3000 people living with FCS in the U S remain unidentified to close that gap, we're continuing to focus on our patient finding efforts and HCP education targeting physicians, who treat patients with severe hyper triglyceride EMEA to increase.
FCS awareness at the same time, our payer engagement efforts will continue to focus on establishing broad durable access.
Kyle: Backed by an experienced and high performing team, we are well positioned to build on our first mover advantage to bring Turing goals it patients in need and keep them on treatment.
Kyle: Building on our foundation in FCS, we're advancing towards the blockbuster opportunity in <unk> with <unk>.
Kyle: Mike FCS SHT G is a serious condition defined by triglyceride levels over 500 milligrams per deciliter, well above the normal level of less than 150.
Kyle: These levels are easily determined through routine fasting blood tests.
Kyle: <unk> effects are broad population with many people unable to adequately manage their triglycerides with currently available treatments.
Kyle: This is particularly true for individuals with high risk <unk> defined as triglyceride levels greater than 880 milligrams per deciliter or greater than 500, with a history of acute pancreatitis or other comorbidities.
Kyle: There are more than $1 million of these especially vulnerable people in the U S, which represents our initial target population for OLED <unk> assuming approval.
Speaker Change: We heard during our recent Kols panel.
Speaker Change: <unk> represents a significant unmet medical need and physicians are eager for better treatment options, especially those that can reduce tg's down below pancreatitis risk levels for.
Speaker Change: For example, Dr. Allan Brown, a leading cardiologist focused on lipid management shared that <unk> can be devastating with patients experiencing recurrent pancreatitis attacks. Despite best efforts. His primary goal is to reduce the risk of acute pancreatitis, which carry significant mortality and morbidity.
Speaker Change: Importantly, all three panelist agreed that <unk> three inhibition offers a promising new therapeutic pathway with the potential to help patients dramatically reduce their triglycerides and the associated risk of acute pancreatitis.
Speaker Change: View is also shared by other physicians, who we've engaged with including recent discussions at the American Academy of Cardiology annual conference with Kols and community cardiologists.
Speaker Change: With this conviction to treat we believe <unk> is well positioned to meet the needs of people with <unk>, and we're making excellent progress across clinical and commercial fronts. As we prepare for an anticipated launch next year, where we also have first mover advantage.
Speaker Change: We expect data from our supportive safety study essence in the second quarter. We then expect to report data in the third quarter from our global pivotal <unk> studies. These pivotal studies core and core to our being conducted on our target <unk> population defined by triglycerides greater than 500 milligrams per desk.
<unk>.
Speaker Change: Baseline characteristics were recently published in the American Heart Journal, showing that fasting median triglycerides at baseline and core and core two were 836 and 749 milligrams per deciliter, respectively and.
Speaker Change: And across the two studies nearly half of participants had baseline <unk> levels of greater than $8 80, even though all participants were receiving standard of care therapy.
Speaker Change: Additionally, 22% and 13% of participants had a history of pancreatitis in the last 10 years prior to enrollment in core and core two respectively.
Speaker Change: Importantly, both studies are well powered to show a significant reduction in triglycerides and it's this reduction that we expect will lead to improvements in clinical outcomes for patients.
Speaker Change: Assuming positive data, we plan to submit an S NDA before year end.
Speaker Change: We're also expanding <unk> reach we recently broadened our partnership with Sobey, who will commercialize <unk> in most markets outside the U S, including in Europe, which is on pace for an FCS approval decision later this year.
Speaker Change: This agreement expands the reach of touring goals at a more patients globally, while providing us the opportunity to earn milestone payments and royalties up to the mid 20% range.
Speaker Change: With a significant first mover advantage in both FCS and SHT G compelling real world data and phase III results in FCS and confidence in the upcoming data for <unk>. We believe <unk> is well positioned to become the standard of care in both indications transforming treatment for thousands of people and driving <unk>.
Speaker Change: Long term growth for eye on us.
Speaker Change: Turning to <unk>, our second anticipated independent launch, we see compelling opportunity to advance the prophylactic treatment for people with hereditary angioedema, we're applying the same discipline and focus that is driving a successful <unk> launch as we prepare to bring down into <unk> in the market.
Speaker Change: More than 20000 people in the U S and Europe are estimated to have HCA.
Speaker Change: In the U S. Most of these patients are on prophylactic treatment.
Speaker Change: As this market continues to evolve up to 20% of patients annually have switched treatments in search of more effective and convenient options signaling that current therapies aren't adequately addressing patient needs.
Speaker Change: Additionally, a recent Harris poll found that 9% and 10 Hae's patient surveyed are interested in trying new prophylactic therapies and we believe <unk> is uniquely positioned to meet that demand if approved with.
Speaker Change: With strong clinical data patient friendly auto injector and monthly or every other month dosing.
Speaker Change: We believe <unk> checks all the boxes that people with HIV are looking for in our next generation prophylactic treatment.
Speaker Change: We're building momentum ahead of launch by drawing on learnings and infrastructure from our ongoing trend GOSE than we knew of launches. This includes expanding our I honest every step patient support program to address the unique needs of <unk> patients to ensure they have a seamless treatment experience with.
Speaker Change: We've already hired a substantial portion of our field facing team and remain on track to complete hiring and training ahead of the anticipated approval.
Speaker Change: Our market access team is actively engaging with payers and our medical affairs team has also been laying the groundwork for a successful launch their efforts include showcasing our robust <unk> and clinical data and I honest this technology at key medical conferences.
Speaker Change: For example at the recent Quad AI meeting, we delivered 11 poster presentations and had robust engagement at our booth, including a dedicated space for unbranded disease awareness campaign.
Speaker Change: We also capitalized on the conferences, San Diego location by welcoming physicians to our campus for deeper in person education about I honest as science and technology.
Speaker Change: Physician feedback continues to be positive, particularly regarding our switch study, which we believe could be a key differentiator in a competitive space.
Speaker Change: Study showed that people switching to <unk> from other prophylactic treatments experienced a further reduction in attacks and 84% of those surveyed said they preferred danita lorson compared to their previous prophylactic treatment.
Speaker Change: While we recognize the converting people from existing therapies will take time were confident that <unk> represents a compelling opportunity to be the preferred prophylactic treatment for many patients.
Speaker Change: We're excited about the road ahead, and well positioned to bring down into Larsen to people who need it assuming approval.
Speaker Change: Our highly experienced efficient and scalable commercial organization is delivering tangible results with the trend goals of FCS launch underway.
Speaker Change: And as we build on these efforts were focused on maximizing <unk> full potential while preparing to successfully execute on our three additional launches planned over the next two years with more to follow positioning I honest for sustained growth and long term success with that I'll now turn the call over to Beth. Thank.
Beth: Thank you Kyle.
Beth: I am pleased to share today that we are increasing our 2025 financial guidance across all metrics, including raising revenue by more than 20% due to our strong first quarter results and recent successful licensing transaction.
Beth: We earned $132 million of revenue in the first quarter, which increased 10% year over year.
Beth: And then half of our revenue came from commercial products, which grew 28% compared to the same period last year.
Beth: Our increasing revenue reflected the encouraging early performance of the twin goals of lots, marking the first time in our company's history, we reported product revenue.
Beth: And the first full quarter at the lines to Ingalls has generated over $6 million in product sales exceeding expectation.
Beth: As Kyle discussed we saw good uptake in patients awaiting treatment and from newly identified patients.
Beth: Our commercial revenue also included $48 million since been lots of royalties from substantial and rise of product sales, which increased 25% year over year.
Beth: Additionally, we earned $9 million and weigh newer royalty, we and our partner Astrazeneca expect weigh newer revenue to grow this year driven by strong U S demand and an expanding global footprint. Following the recent EU approval.
Beth: The remainder of our revenue came from programs under our R&D collaboration underscoring the important financial contributions of our partners pipeline.
Beth: You may have noticed we've streamlined the revenue section of our P&L. We did this to reflect our new chapter as a commercial stage biotech and to plan for numerous independent launches in the coming years.
Beth: Total non-GAAP operating expenses increased less than 5%, reflecting our commitment to disciplined investment and driving operating leverage as planned our sales and marketing expenses increased year over year, reflecting our investments in the U S launch of <unk>.
Beth: And preparations for the upcoming lines have gone into Larson.
Beth: Our SG&A expenses also included our minority portion of we knew with sales and marketing expenses.
Beth: R&D expenses decreased year over year as several of our late stage studies recently concluded.
Beth: Fortunately, we continue to appropriately fund our advancing pipeline with a large majority of our total R&D expenses funding our late stage program.
Beth: Building on our first our strong first quarter results. We recently completed licensing transactions for Stat book, Larson and ex U S commercialization rights for <unk> that meaningfully enhanced our financial outlook.
Beth: This is the reason we are substantially increasing at 2025 guidance.
Beth: Last week, we finalized the licensing of <unk> and <unk>.
Beth: <unk> outside of our core disease areas of cardiology and neurology.
Beth: And generating $280 million.
Beth: This transaction enables us to remain focused on realizing the potential of the medicines that we plan to independently and to patients.
Beth: While further strengthening our financial position.
Beth: Additionally, we are eligible to earn significant milestone payments established in advances together with royalties up to the mid teen percentage range on future product sales.
Beth: As a result of the substantial license fee, which exceeded the <unk> revenue included in our original guidance, we are increasing our revenue guidance by more than 20%.
Beth: We now expect to earn revenue in the 725 million to $750 million range for the year.
Beth: Our revenue guidance also includes sizable commercial revenue anchored by stable state RASM royalties and growing <unk> royalties.
Beth: And with our first independent launch underway, we expect increasing product revenues as the year progresses.
Beth: As the <unk> launch progresses, it's important to remember that FCS is a rare disease with most patients still unidentified and undiagnosed.
Beth: Therefore, our focus remains on disease education, and patient identification, both of which will take time.
Beth: We're also on track to add initial product revenue from our second lines with the FDA action date predominantly set for August 21st.
Beth: In addition to the <unk> revenue we recognized in Q2, we expect to earn additional R&D revenue from partnered programs later this year.
Beth: We continue to project our full year 2025 operating expenses to increase in the high single digit percentage range compared to last year.
Beth: This increase will be driven by SG&A expenses as we invest to support the success of our multiple ongoing and planned launches.
Beth: We project, our R&D expenses to remain steady this year similar to last year.
Beth: As several of our late stage studies have recently.
Beth: Recently concluded or are wrapping up this year, we are able to reallocate our resources toward our next wave of opportunities.
Beth: Including <unk> for Angelman syndrome, which we expect to start phase III development shortly.
Beth: Since our licensee license fee revenue dropped entirely through our bottom line, we are improving our non-GAAP operating loss guidance by nearly 25%.
Beth: Less than $375 million.
Beth: And we now expect to end the year with $1 9 billion in cash.
Beth: Our strengthened balance sheet and commitment to drive operating leverage positions us well to advance our strategic priorities and this dynamic macroeconomic environment.
Beth: Looking beyond 2025, we are confident in our ability to deliver sustained revenue growth.
Beth: The late stage programs, we have in our pipeline today represent a significant opportunity with combined peak sales potential in the multibillion dollar range.
Beth: We expect our eye on its own medicines to generate more than $3 billion in peak annual product sales. This of course means that we will continue making investments to bring these medicines to patients.
Beth: On top of our substantial product revenue opportunity.
Beth: Late stage partnered medicines could contribute over $2 billion annually and peak royalties.
Beth: Notably nearly all of these medicines are on track to deliver phase III data either this year or next year setting the stage for potential launches soon after.
Beth: Together these upcoming launches or place to reach hundreds of thousands of patients worldwide.
Beth: And in turn positioned us to deliver significant topline growth and reach sustainable positive cash flow.
Brett: Turning scientific innovation into long term value for shareholders and with that I'll turn the call back over to Brett.
Brett: Thank you Beth.
Brett: We've accomplished a great deal to get us where we are today, a fully integrated commercial stage biotech company with our first independent launch well underway.
Speaker Change: 125 is off to an excellent start including the U S <unk> launch.
Speaker Change: With additional upcoming launches anticipated, including down Laurus in for a J and a deep commitment to innovation, we're well positioned to drive accelerating value.
Speaker Change: It's through <unk> innovation that we've established a proven discovery and development engine, which has provided us with a pipeline of medicines with transformational potential.
Speaker Change: Our pipeline continues to deliver we are on track for Ozarks in yoga nurse and phase III data later this year that if positive would expand the reach of our medicines to even more patients.
Speaker Change: And with many additional phase II data Readouts expected next year, we are well positioned to continue bringing a steady cadence of medicines to patients.
Speaker Change: Our scalable and experienced commercial organization is executing well as demonstrated by the encouraging start to the <unk> launch.
Speaker Change: The more our commercial organization has established a solid foundation to support multiple planned product launches in the future.
Speaker Change: And based on recent successful licensing transactions, along with our first quarter results and disciplined financial management, we significantly increased our 2025 financial guidance.
Speaker Change: Importantly, the progress we've made puts us on a clear path to achieving positive cash flow supported by our expectations for substantial top line revenue growth and with that we'll now open the call up for questions.
Speaker Change: Thank you.
Speaker Change: To ask a question you May Press Star then one on your tariff from Cooper.
Speaker Change: Ladies and speakerphone, please pick up your handset before pressing the keys.
Speaker Change: Please limit yourself to one question and one follow up if you have further questions you may bring into question Keith.
Speaker Change: Okay.
Speaker Change: So try your question my question. Thank you.
Speaker Change: At this time with personal Mccarron from November actually we will discuss today.
Allison: Allison from Oppenheimer.
Speaker Change: Oh, Hey, congrats on all the progress.
Speaker Change: Thank you for taking our question.
Speaker Change: Curious about <unk> and then any overlap between physician prescribers for FCS and S. H TV.
Speaker Change: How you plan to leverage that and then related to that would approximately 20% pancreatitis history for the patients enrolled in your pivotal <unk> studies provide sufficient power to show a reduction in what level of reduction are you targeting thank you.
Speaker Change: Thanks, Jade Karl we'll take the the.
Speaker Change: Overlap of prescribers for FCS Hcg, and I'll touch on the AP.
Speaker Change: Question, yet, yes, thanks, Jay first.
Speaker Change: <unk> launch is really off to an encouraging start the execution by the commercial team that has been very strong.
Speaker Change: Very pleased with where we're at right now as we discussed we have been able to convert the EAP in OA patients.
Speaker Change: Those that have been waiting for a therapy have begun getting treatment and we're also identifying new patients.
Speaker Change: The current targets that we have are a few thousand physicians as you would expect based on the type that are treating Fcs today.
Speaker Change: These are a cardiology cardiologists and endocrinologists.
Speaker Change: And theyre seeing <unk> patients, but theyre also seeing FCS patients. So that's kind of the the runway to get us.
Speaker Change: Two <unk> when you get to <unk>. The population expands obviously upwards of north of 1 million patients.
Speaker Change: And as we go there the overlap is quite significant theyre still cardiologists endocrinologists and lipid apologist treating these these patients.
Speaker Change: They are currently being treated with suboptimal therapies and not getting the goal.
Speaker Change: And what we're hearing and we heard this from the Kols panel that we did on April 14th is that there is the realization that reducing triglycerides is directly correlated to AP and physicians are well aware of that and theyre looking for a better treatment option and we're looking forward to potentially having positive data and an approval for <unk> to be able to help those patients.
Kyle: Thanks Kyle.
Speaker Change: Obviously, you're referring to the AP baseline.
Speaker Change: Event data that we reported in our base and our demographics baseline demographics clinical trial design study that we published last month.
Speaker Change: Earlier this month.
Speaker Change: Where we had 22% and core and 13%.
Speaker Change: And core to patients with a history of AP over the last 10 years.
Speaker Change: Hi.
Speaker Change: I want to emphasize before I get into.
Speaker Change: Expectations for IP data from those studies that this is a substantial unmet medical need large patient population that hcp's.
Speaker Change: Healthcare providers are ready to treat patients with.
Speaker Change: S H T G. Even without <unk> data in hand, this is a large market opportunity large unmet need and what we heard in our HCP panel and we've heard from other hcp's that they are looking for.
Speaker Change: A medicine that will substantially reduce.
Speaker Change: Lissa rides on top of standard of care, which is exactly how our trial design that is the opportunity there.
Speaker Change: Now with that said AP is of course as a secondary endpoint in our study as a secondary endpoints not powered for AP.
Speaker Change: But we're doing everything we can to increase the powering for the study for example.
Speaker Change: We are combining <unk> in quarter two in our secondary analysis of AP.
Speaker Change: Events, and we're looking at AAP events at 12 months, whereas the triglyceride primary event primary endpoint is at six months. So we're extending that we're looking at the maximum amount of.
Speaker Change: Time and patients to <unk>.
Speaker Change: Look at data.
Speaker Change: And of course, the FCS data is also encouraging.
Speaker Change: Our twin goals, where we reported statistically significant reductions in acute pancreatitis events in our balance study. So take all that into account, but really what we are laser focused on is to demonstrate substantial reduction of triglycerides in <unk> patients, which is what hep's are looking for to prescribe <unk>.
Michael: Michael that's awesome.
Gary Nachman: The next question comes from Gary Nachman of gaming.
Speaker Change: Hey, guys. Thanks for taking the questions and nice quarter, so on Don at the Los Angeles.
Speaker Change: More about what you're doing ahead of the producer date internally to prepare for that launch.
For patients that are going to be switching how youre thinking about that transition I guess in the first year of launch.
Speaker Change: And just in terms of access how long do you think that process is going to take.
Speaker Change: And then.
Speaker Change: Just as a follow up Brett on the macro dynamics you just highlighted it briefly but just talk a bit more what you think the expected impact from tariffs is going to be.
Speaker Change: For all business just based on the current trade policy and just in terms of your conversations with FDA, if youre seeing any sort of signs of potential delays, whether with <unk> dates we're starting clinical studies. Thank you.
Speaker Change: Great.
Speaker Change: Some of those launch yes.
Speaker Change: No about the HLA market is this is largely an unsatisfied market.
Speaker Change: See approximately 20% of patients that are moving between prophylactic treatments in the U S on an annual basis.
Speaker Change: We also recently released the Harris poll that showed that nine out of 10 patients are interested in trying new therapies. In this space. So there is definitely an opportunity here in the dining <unk> profile in terms of efficacy tolerability and convenience.
Speaker Change: Offered all in one single treatment and when we start talking about switching patients and the dynamic of why patients want to switch. It's one of those three reasons that theyre looking for something new and different.
Speaker Change: We're going to be able to provide value to <unk>.
Speaker Change: The way that demonstrates reductions in attacks improved tolerability and there are only going to have to take the treatment every four or every eight weeks. So the profile is very very strong there in.
Speaker Change: In terms of switching patients.
Speaker Change: What we need to do is we need patients to advocate for themselves and we need physicians to understand what these patients are experiencing and thats. The work that we're doing right now.
Speaker Change: We are being informed by patients and we're also beginning to communicate directly with Hcp's. What the need is in this market and setting ourselves up to be able to talk about how and why to switch these patients and why it's important for that to happen.
Speaker Change: But this isn't a switch market, we will begin the ramp hopefully in Q4, and we anticipate peak sales being greater than $500 million for dining tillerson and with a concentrated prescriber base. We can have a very targeted sales force to go out and do that which is currently being hired and trained and going to be deployed well in advance of the <unk>.
Speaker Change: Right.
Kyle: Thanks Kyle.
Kyle: To your other question, Gary obviously carefully monitoring.
Kyle: Changes that the new administration is bringing both at the FDA.
Kyle: At the macro level on tariffs et cetera.
Kyle: At the FDA.
Kyle: Today.
Kyle: We're pleased to report that we've had no.
Kyle: Changes.
Kyle: And any of that in any of the programs that we've been discussing with the FDA.
Kyle: Everything is on track as mentioned earlier <unk>.
Kyle: Is on track for August 20, <unk> and the discussions we're having are.
Kyle: The discussions we shouldn't be having at this stage of the review process same is true for our other discussions with the FDA R&D and so on so everything appears to be going.
Kyle: Well with the FDA at this time, despite the changes that are occurring there will of course keep on monitoring that.
Kyle: With respect to other changes tariffs et cetera.
Kyle: That too we have not seen any meaningful impact on our business to date that includes <unk>.
Kyle: <unk> or that we have to potentially absorb for drugs that are already launched.
Kyle: Our future drugs with.
Kyle: With respect to commercial supply and clinical supply.
Kyle: It is also going very well.
Kyle: We have sufficient supplies in place to support the triangle from Golar <unk> launch and have done in divorce and launch.
Kyle: And they are ready to go.
Kyle: And to support those launches.
Kyle: For the near term.
Kyle: And for our clinical trials.
Kyle: Have we.
Kyle: We make drug here <unk> as well as with some CMO is but we have a lot of redundancies and in the supply chain to support our clinical trial.
Kyle: Studies.
Kyle: With respect to supply so.
Kyle: It's obviously a fluid situation that we're monitoring very carefully and building contingencies to manage however to date, we have not had any impact with respect to tariffs or with the FDA on our on our business.
Kyle: Okay.
Speaker Change: The next question comes from Jackie counterparty.
Kyle: Hi.
Kyle: Hey, good morning, and thanks for taking my question.
Kyle: So on the FCS launch how confident is the team on the 2000 plus FCA.
Kyle: FCS patient estimates and how quickly can you convert these patients to commercial therapy before you have significant price erosion with the launch in SSD Chi. Thank you.
Kyle: Jonathan take that please yeah happy to take that I think while the epidemiology varies between $1 13 per million, we fairly confident in those three towers in the Genesis of your question really is how quickly can we identify them I think is Kyle outlined we.
Kyle: Obviously had clinical trial patients that we've been able to comment on sort of covered.
Kyle: Very quickly in the prelaunch phase, we were definitely very successfully engaging with our target audience and identifying Fcs patients.
Kyle: And that helps drive initial uptake in the product.
Kyle: But in the every month as more and more physicians become aware, we're finding more and more patients.
Kyle: And that's very encouraging for the outlook that we've got.
Kyle: For FCS I definitely think this is a.
Kyle: Underserved community with a very debilitating disease and as that awareness grows.
Kyle: Both amongst hcp's, but importantly through a lot of the work we're doing with Omnichannel, we're going to find more and more patients in the next 18 months prior to the <unk> launch.
Kyle: Obviously with positive data and potential approval from the FDA.
Kyle: Soon is that indication expands.
Kyle: We'll be addressing the far larger population the specifics of FCS become less apparent.
Kyle: And price.
Kyle: Price erosion play out in terms of price.
Kyle: Priced at the FCS launch appropriately with the 3000 patient population out there right and Thats. How we ended up at the price point that we did and the value that <unk> brings to that patient population.
Kyle: As we think about moving forward for <unk> that population is going to be exponentially higher right.
Kyle: Dollars plus addressable in terms of the population if we look at.
Kyle: The marketplace typically drugs that have a $1 million or so patients addressable or in the specialty pricing range of 10 to $20000. For example, we.
Kyle: We've got some work to do to figure out what the pricing will be ultimately in this category based on the data that supports that.
Kyle: The program itself, which will note later this year and we will go back and do some more research and then obviously announced the price when we receive approval for the <unk> indication.
Kyle: And again, we're very encouraged by the <unk> launch to date. It is early days of course, but we expect to be.
Kyle: Commercializing <unk> and FCS for a year and a half to two years before that's http commercialization will kick in.
Kyle: The next question is from Jessica Fye.
Jessica Fye: J P Morgan.
Speaker Change: Hey, guys. Good morning, Thanks for taking my questions I just had a couple.
Speaker Change: I missed this but was there any channel stocking for triangle, Kathryn that <unk> number and if so can you quantify that.
Speaker Change: Second one can you just elaborate a little bit more on the sources of upside to the revenue guidance and how much of that is driven by commercial product performance versus the licensing deal.
Speaker Change: And lastly, kind of touched on this but maybe just a little more specifics can you just talk about the manufacturing footprint for Trimble the Donegal arson.
Speaker Change: And how do you think about possible exposure to tariffs.
Speaker Change: Sure.
Speaker Change: So channel stack and pile for interim goals.
Speaker Change: Yes. The <unk> sales is currently directly correlated to demand there was not a large stock and inventory.
Speaker Change: It's adequately being managed for the contract that we have in place with a specialty pharmacy.
Speaker Change: It's really reflected by demand.
Speaker Change: You want to add anything to that.
Speaker Change: As well as then talk about the revenue guidance sure.
Speaker Change: Secondly, Kyle said that the team has done a wonderful job at managing inventories and of course with a rare disease.
Speaker Change: A drug like <unk>, you want to make sure that you are keeping their inventory levels as low as you, possibly can and driving.
Speaker Change: Inventory in the channel from demand, which is exactly what the team is doing.
Speaker Change: On the revenue guidance.
Speaker Change: That is that it.
Speaker Change: Based on obviously, a strong Q1, but the vast majority of that raise is related to the licensing transactions, we did for our <unk> with Ono and the.
Speaker Change: U S commercial licensing to assay will be for all of the Tyson.
Speaker Change: <unk>.
Speaker Change: Those transactions, where you can imagine probably lies in our initial guidance of more than $600 million in revenue.
Speaker Change: And with the now completion of those transactions, we are increasing our guidance to reflect 100% probability.
Speaker Change: And flowing that through not only revenue, but two.
Speaker Change: Our operating loss into our cash guidance as well.
Speaker Change: Regarding manufacturing Jess.
Speaker Change: We have.
Speaker Change: A number of sources for supplying our drugs for both clinical trials as well as for the commercial launches, including South Korea, Europe, and the U S. As I mentioned earlier to one of the other questions.
Speaker Change: We're well stocked.
Speaker Change: Supply already to support the <unk> launches as well as.
Speaker Change: The anticipated launch predominate worse and so the oil that's in place, we're continuing to monitor any potential impact.
Speaker Change: Right now there has not been any.
Speaker Change: And that includes on tariffs as well really we haven't we're watching it very carefully but we're not we have not experienced any meaningful impacts.
Speaker Change: And in our guidance stays the same we just reduced.
Speaker Change: <unk>.
Speaker Change: <unk> improved our guidance and that's also factoring in any potential impact of tariffs.
Jason <unk>: The next question is from Jason <unk> of Bank of America.
Speaker Change: Okay.
Speaker Change: Hey.
Speaker Change: <unk> are correct, Jason Thanks for taking our question.
Speaker Change: Maybe just a follow up on <unk> and.
Speaker Change: Unpack a little bit between.
Speaker Change: The conversion that you see from the expanded access.
Speaker Change: <unk>.
Speaker Change: Newly identified patients that were previously not erode it in any of the prior clinical trials.
Speaker Change: Understood that this is a market that requires some market <unk> are.
Speaker Change: Are you seeing this dedication of new starts and.
Speaker Change: And just a quick follow up.
Speaker Change: <unk>.
Speaker Change: You guys have it on track for our <unk> phase III start are there any outstanding staff will be to execute before the phase III start. Thanks, so much.
Speaker Change: Okay.
Speaker Change: Jim.
Speaker Change: Take the <unk> question is a brief answer and then.
Kyle: Kyle I'll take the.
Kyle: Question with respect to conversion.
Speaker Change: EAP patients and new starts so the Angelman study phase II study is on track for time on time initiation.
Speaker Change: As a reminder, we had excellent.
Speaker Change: Discussions and.
Speaker Change: Completion of conversations with the FDA last year, we aligned on our phase III trial design. We think we have the right trial design, we have the right drug the right chemical platform.
Speaker Change: To get this study started in the first half of this year, which means that we'll get started in this quarter. So very soon.
Speaker Change: On our way and what we've also said is that we'll.
Speaker Change: Our expectation is to complete enrollment.
Speaker Change: In 2026, so we expect to enroll fairly quickly.
Speaker Change: Yeah. Thanks, Brett.
Speaker Change: In terms of the <unk> launch.
Speaker Change: Strong patient uptake in Q1, as we were referencing.
Speaker Change: It's demand driven.
Speaker Change: Inversion of EAP in OA patients was obviously, a main focus here and moving those patients onto commercial drug.
Speaker Change: As we mentioned the majority of those patients converted over.
Speaker Change: It was exactly what we expected to have happen.
Speaker Change: Area of patient population, we talked about are those that were waiting for treatment right. This was kind of the pent up patients that have been treated for years and unfortunately.
Speaker Change: Didn't have a therapeutic option available those have also many of them have started on treatment as well and then the identification of new patients is really what we're going to be focused on moving forward.
Speaker Change: That's the main focus of our work but in Q1.
Speaker Change: We had patients from all three of those buckets, but the predominant patient populations were coming from the EAP OLED and the pent up demand patients that had already been identified so our focus really is getting new patients diagnosed new patients treated and working them through the payer channel, which we're doing very effectively right now Jonathan.
You can add a little bit perhaps a little bit of color were definitely having success engaging with hcp's to actually find both patients.
Speaker Change: There were identified prior to launch and converting them to covered product number one but also number two is the awareness gets out there finding new patients and becoming aware of.
Speaker Change: Patients that.
Speaker Change: That we werent aware of launch so that's super encouraging in the first quarter of our launch.
Speaker Change: Thanks, so much.
Speaker Change: Thanks, Jim.
Speaker Change: Your next question comes from Yaron Werber activity Cowen.
Yaron Werber: Alright, thanks, so much congrats on the nice progress.
Yaron Werber: Maybe just a couple of questions from me the first one on the way Newark can you give us a little bit of a sense of the part D redesign.
Yaron Werber: Now that it's in effect and its kept cutting patient two grand out of pocket.
Yaron Werber: Does that going to do you think sort of impact uptake.
Yaron Werber: Under part D and then.
Speaker Change: <unk> do you have when you said some of the 6 million was it really a great number of that sounds like it's a good baseline from here onwards.
Speaker Change: From Hereon do you already have patients that you think are going to come onboard pretty quickly that are diagnosed or.
Speaker Change: As we kind of think ahead about growth in patient identification and maybe give us some qualitative view on how fast that can be.
Speaker Change: Yes, let me touch on our first Astrazeneca continue to see very strong U S demand for wind.
Speaker Change: The redesign of Medicare part D impacted some of the Q1 revenue, but there are a lot of positives that you just emphasized in terms of the out of pocket change that's happening.
Speaker Change: First of all I think youre going to see an increased volume from new patient starts because of this.
Speaker Change: We're seeing cardiologists and neurologists prescribed way new up and what they are telling us is that it's going to number one increase the number of patients. They can treat because of affordability and number two they feel like it's going to increase the compliance rates for those patients as well because they are going to be able to afford their medication moving forward.
Speaker Change: I think that has a significant advantage and opportunity for patients overall, and we knew of specifically to be able to demonstrate revenue growth quarter over quarter. As we move forward in 2025, I think we're very optimistic about that.
Speaker Change: In terms of the structuring goals at question.
Speaker Change: Again greater than $6 million in the first quarter.
Talked about the three patient buckets that that came from.
Speaker Change: But we do have additional patients that are being identified currently that are potentially going to go on too.
Speaker Change: <unk>.
Speaker Change: This takes a lot of a lot of work great 3000 potential patients in the U S.
Speaker Change: We're doing a lot of work not only with our customer facing teams, but also with our omnichannel efforts.
Speaker Change: In the education that we're doing with our medical affairs groups at Congresses in different meetings. So those are the things that we're doing to get the word out about the new treatment option available how to diagnose Fcs patients, but it's going to take some time and some work, but we are encouraged by by the number of patients that are coming forward and we will work to get them on treatment. This <unk>.
Speaker Change: And moving forward throughout the year.
Kyle: Thanks, Kyle remember your own.
Speaker Change: The.
Speaker Change: <unk>.
Speaker Change: <unk> is now launched outside the U S and Europe as well. So we're expecting continued revenue growth and based on our strong patient demand not only in the U S. But also in other geographies as well for 2025 and going forward.
Luca: The next question is from Luca <unk> at RBC capital markets.
Speaker Change: Hi, Good morning, Kim This is kelsey on sorry, Luca. Thanks, so much for taking my question and congrats on the impressive launch to take friction goes up and this question will be answer TR cardiomyopathy. We appreciate it's still early days as cardio transform has yet to read out but were you surprised.
Speaker Change: By an item not stellar in pricing when they got label expansion for cardiomyopathy and are you planning to follow the same playbook or is there a scenario, where you compete with them by lowering the price.
Speaker Change: Other therapy much appreciate it thank you.
Speaker Change: Yes in terms of our <unk> strategy.
Speaker Change: I wasn't too surprised by that.
Speaker Change: I believe that they've got a balance of patients that are hereditary polyneuropathy patients.
Speaker Change: I think they are trying to maintain a price in order to manage their revenues.
Speaker Change: Now and moving forward.
Speaker Change: And it's up to them kind of how that market evolves, I think and what theyre able to get coverage on for cardiomyopathy, I think thats yet to be determined.
Speaker Change: In terms of the strategy for where that is.
Speaker Change: Ultimately astrazeneca is decision in terms of how they navigate that I'm sure. There are a lot of learnings in this market and as the market continues to evolve we'll take a look in terms of reimbursement and coverage and physician and patient feedback and figure out what's the right price point is for <unk> based on the benefit and value that it delivers.
David Lebowitz: The next question comes from David Lebowitz.
David Lebowitz: Thank you very much for taking my question.
Speaker Change: When you look at the core of trials and the baseline data.
David Lebowitz: Data.
David Lebowitz: I'm curious as to how the analysis in the trials will actually carried out is it is it going to be strictly based on baseline for the overall population relative to reductions whether it be additional studies or analyses on patients that actually had baseline AP events.
David Lebowitz: To reduce but over the smaller denominator.
David Lebowitz: Curious to whats pre specified and what could possibly be accepted by the FDA for labeling considerations.
David Lebowitz: Eugene.
David Lebowitz: Yes.
David Lebowitz: Thanks for the question. So certainly the analysis of AP events. The Prespecified analysis, we will look at all events happening during the 12 months exposure.
David Lebowitz: In a trial, obviously, comparing the active arm versus placebo.
David Lebowitz: We will look at population that had a baseline or at baseline reported historical events.
David Lebowitz: It's a smaller much smaller subgroup.
David Lebowitz: So I guess the answer is that we.
David Lebowitz: The overall analysis, we will look at all the rounds happening during the trial comparing all of ours in patients.
David Lebowitz: I believe if I recall Eugene we're also will be looking at.
David Lebowitz: AP and patients above and below AAV.
David Lebowitz: In the analysis as well right.
David Lebowitz: Yes.
David Lebowitz: So we will be carving up the data based on history.
David Lebowitz: Of AP and without and so on as far as the labeling discussions and getting a pea in the label I think thats a data driven.
David Lebowitz:
David Lebowitz: Outcome that will have to talk about with the FDA.
David Lebowitz: But.
David Lebowitz: Again as I emphasized earlier, we believe that this is a very substantial unmet need and a very sizable market opportunity for our owners based on triglyceride lowering.
David Lebowitz: But these patients are suffering from acute pancreatitis and.
David Lebowitz: We have a substantial number of patients with a history of AP.
David Lebowitz: Enrolled enrolled in our studies.
David Lebowitz: We think we have the right trial design.
David Lebowitz: If we can see it.
David Lebowitz: The potential to see it yes, I'll just add we have a pea in the label now for train GOSE, which is on the FCS population.
David Lebowitz: But that's that's very beneficial and then the read through in terms of the connection between triglycerides and lowering of acute pancreatitis. We know from the recent <unk> panel that we just had that that's well understood as Brett Eugene just described.
David Lebowitz: Thanks, David.
Speaker Change: Question comes from Manny <unk>.
David Lebowitz: Frank.
David Lebowitz: Hi, This is really on some money first.
David Lebowitz: First question digging a little more into the <unk>.
David Lebowitz: The script.
Speaker Change: First one loser.
Speaker Change: So you mentioned that the majority of patients with from the open label extension is where does the EAP program, but in terms of the remaining patients or the Uni diagnose and troubleshoot diagnose patients could you give a little more color on the distribution there between newly diagnosed have you seen diagnosed as well as your expectations.
Speaker Change: How it will evolve quarter to quarter.
Speaker Change: I don't have too much additional color to add.
Speaker Change: Not going to disclose the exact patient numbers or the percentages at this point in time.
Speaker Change: But we are encouraged at the patient finding work that we've done the identification of new patients. So far and we believe we'll be able to add incremental patients quarter over quarter as we move forward throughout the year. The launch is going very well as we talked about a strong patient uptake and strong demand and good payer coverage. So I think we are.
Speaker Change: And a great place, yes, and just to clarify.
Speaker Change: We said that the majority of patients are from the open label extension what.
Speaker Change: What we said was that we have that.
Speaker Change: We have obviously patients in the open label extension study from our Phase III trial, which had 66 patients in balance.
Speaker Change: In the U S. And then of course, we are focused on the U S segment of that in.
Speaker Change: Many of those patients the majority of those patients have converted over to commercial but that doesn't necessarily represent the overall.
Speaker Change: Patient population.
Speaker Change: Tringle commercially today going forward, it's going to be newly identified patients. That's that's what we've emphasized because those patients that were ready to diagnosed.
Speaker Change: As well as the clinical trial patients.
Speaker Change: We did a good job we are doing a good job converting those patients, but going forward, it's all about patient identification.
Speaker Change: So a lot of work to do.
Speaker Change: One thing I'll add is we are definitely finding new patients just just to be.
Speaker Change: To be transparent around that I mean.
Speaker Change: Difficult.
Speaker Change: Sika diagnosis for a condition, where it doesn't ultimately change the treatment so as awareness builds.
Speaker Change: Clinical experience bills. This far more motivation to see these patients and they definitely definitely exists.
Speaker Change: I think we have time for one more question.
Speaker Change: The last question comes from Mike <unk> at Morgan Stanley.
Speaker Change: Hey, guys. Thanks for taking the question and for squeezing me in maybe just a follow up on <unk> S. H T G.
Speaker Change: Looks like you've narrowed the timing of that data slightly for the corn Corps two studies the <unk> from the second half previously just curious.
Speaker Change: Any reason behind that and then secondly, maybe just to clarify will that initial update from those studies include the 12 months AEP data. Thanks.
Speaker Change: Thanks, Mike.
Speaker Change: No nothing really behind it just as we get closer to the data Readouts and we.
Speaker Change: We get through everything we need to get through all the blocking and tackling to read out the top line data we provide more clarity. So yes. We are we are we have provided more precision Q2.
Speaker Change: Since data Q3 <unk>.
Speaker Change: Core and core to top line data.
Speaker Change: With respect to.
Speaker Change: AP Readouts, we don't expect AP in the essence study of course, just as just to confirm that.
Speaker Change: These are mildly elevated for patients with triglycerides. They aren't they don't have many apu events. This is a safety study to support these exposure database that's necessary for a highly prevalent diseases in the eyes of the FDA.
Speaker Change: So really we're focused on triglyceride lowering.
Speaker Change: In that study and <unk>.
Speaker Change: Safety.
Speaker Change: I also want to emphasize that the percent reductions in triglycerides that we'd expect in essence is not a reflection of what we would expect in core <unk> and thats because these patients are only mildly elevated.
Speaker Change: So again, we're expecting good substantial reductions in <unk>, but.
Speaker Change: Not to the magnitude of what we would expect in core.
Speaker Change: In the topline data for core and core to us they're working that that al whether we will have the data in time for that topline data, but we will be focused on there principally.
Speaker Change: Triglyceride reductions.
Speaker Change: Safety for core on core too.
Speaker Change: And if we have the IP data at that time, we will certainly share it.
Speaker Change: And our and our top line.
Speaker Change: Announcements later this year so thanks.
Mike: Thanks for the question, Mike Thanks, everybody for.
Speaker Change: Participating in all the great questions.
Mike: Really emphasize that looking forward.
Mike: We're excited about building on our momentum throughout the year and sharing our additional achievements.
Mike: With all of you along the way so thanks again for participating and.
Mike: We will talk soon have a great day.
Mike: Goodbye.