Q1 2025 Alnylam Pharmaceuticals Inc Earnings Call
Speaker Change: Good morning, ladies and gentlemen, and welcome to the Aniline Pharmaceuticals Q1 2025 Earnings Conference Call. At this time, all lines are in a listen-only mode. Following the presentation, we will conduct a question-and-answer session.
Christine Lindbloom: Good morning, I'm, Christine Lindbloom, Chief Corporate Communications Officer, Alan Island with me today are bond Greenstreet, Chief Executive Officer, Paul <unk>, Our Chief Commercial Officer, Chris spoke Garg, Chief Medical Officer, and Jeff Poulton Chief Financial Officer for those of you participating via conference call. The accompanying slides can be accessed by going to the events section.
Speaker Change: Investors page of our web site investors thought on island Dotcom flash events.
Speaker Change: During today's call as outlined in slide two Yvonne will offer introductory remarks and provide some general context total will provide an update on our global commercial progress, Portugal will review pipeline updates and clinical progress and Jeff will review, our financials and guidance followed by a summary of our upcoming milestones before we open the call to your questions.
Speaker Change: I would like to remind you that this call will contain remarks concerning allowance future expectations plans and prospects, which constitute forward looking statements for the purposes of the safe Harbor provision under the private Securities Litigation Reform Act of 1995 actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed in our most recent peer.
Speaker Change: Arctic report on file with the SEC. In addition, any forward looking statements represent our views only as of the date of this reporting and should not be relied upon as representing our views as of any subsequent date, we specifically disclaim any obligation to update such statements with that I'd like to turn the call over to Yvonne Goodbye.
Yvonne: Thanks, Christine and thank you everyone for joining the call today.
Yvonne: 2025 is off to a strong stance its owner.
Yvonne: In the first call. We made notable progress towards our goal of becoming the global Pizza, all either which will play a critical role in driving sustainable growth and value creation this year and beyond.
Commercially we delivered $469 million in combines and niche product revenues and this represents 28% growth year over year.
Yvonne: This is driven primarily by the robust 45% year over year growth. It's all U S. TCR franchise, which in the first quarter consisted solely of continued uptake in the hereditary polyneuropathy population.
Yvonne: We also have several exciting portfolio funds for the quarter, most notably the expanded indication from food truck and a T. T. All cardiomyopathy with approvals in the U S and Brazil and earlier. This week, we received a positive <unk> opinion in the EU.
Yvonne: We also saw approval at the six not undiscovered RNA therapeutic cure for.
Yvonne: So hemophilia, a or b secured by our partners incentive fee.
Yvonne: Lastly, we are reiterating our guidance for the year underscoring our confidence in continued growth and a strong launch in ATT. All cardiomyopathy. This also includes a goal of achieving sustainable non-GAAP profitability in 2025.
Yvonne: Now before I move on I'd like to take just a brief moment to acknowledge the uncertainty that exists related to potential evolutions with certain policies, including tariffs FDA personnel reorganization and other biotech industry initiatives being discussed.
Yvonne: So how much is yet to be clarified and finalized we've taken a comprehensive look across our business and we believe <unk> is.
Yvonne: Is well positioned and pretty well insulated from any potential impacts associated with the key initiatives that have been announced to date.
Yvonne: We feel that we are completely able to focus on the key tasks at hand.
Yvonne: Executing on our strong launch in <unk> cardiomyopathy, and continuing to advance our robust high value pipeline.
Yvonne: Yeah.
Yvonne: Our achievements in early 2025 underpin the three core elements of our business that we believe will drive sustainable growth and value creation for years to come.
Yvonne: The first is teaching our leadership.
Yvonne: <unk> will provide color on our launch of <unk> and <unk>, but at a high level. We're very pleased with our early progress which is focused on securing broad access driving awareness and supporting treatment initiation for patients in the initial few weeks since approval.
Yvonne: Next is growth through innovation, including a pipeline with numerous multibillion dollar opportunities and an R&D engine set up to deliver sustainable innovation and value creation.
Yvonne: The third element is strong financial performance with robust commercial delivery and a disciplined approach to capital allocation to enable us to sustained profitability going forward.
Yvonne: And of course all of this is underpinned by best in class team and award winning culture.
Yvonne: And this all represents tremendous progress against our own Ireland piece of fifth by 'twenty five goals, but we continue to work to turn these goals into reality by the end of the year.
Speaker Change: Doing so will further establish <unk> as a unique top tier biotech company delivering sustainable innovation to patients for years to come with that let me now turn the call over to talk for a review of our commercial performance toga.
Toga: Thanks, Ivan and good morning, everyone.
Toga: I'm, particularly excited to share our commercial update on this call today.
Toga: I am as encouraged as I have ever been not only by the impact we're already having poor patients affected by the conditions are medicines are indicated to treat <unk>.
Toga: Also by the potential that is just beginning to unfold.
Toga: Now that our ATT RCM launches underway in the U S.
Toga: I'll review our performance in Q1 and also provide some early perspective on our ATT RCM launch, which only began in the final days of this quarter.
Toga: We will take shape over the quarters ahead.
Toga: Our Q1 performance continues to be strong altogether, our portfolio delivered nearly half a billion dollars in combined net product revenues, representing 28% growth year over year, and 4% versus Q4 Q4 2024.
Toga: This is tremendous growth across our portfolio.
Toga: Several years after our initial launches.
Toga: Quite simply this means helping more patients affected by each of these devastating diseases.
Toga: Before we dive into our TCR franchise.
Toga: The exciting progress with our new Cm launch, let me briefly update you on our rare disease franchise.
Toga: Our air franchise, given the Orinoco zama.
Toga: Together delivered $109 million in combined product sales in the first quarter, representing 8% growth compared with the first quarter of 2024.
Toga: On a year over year basis, the growth in <unk> patients on therapy was approximately 15% and growth in <unk> patients on therapy was approximately 20%.
Toga: Revenue growth for Shlomo was less than growth in patients on therapy, primarily due to gross to net adjustments in European markets and the timing of orders in partner markets.
Toga: We continue to expect full year product sales growth of approximately 15% for a rare franchise costs.
Toga: Consistent with the midpoint of our 2025 rare product sales guidance.
Toga: Moving now to our TCR franchise, we continue to see strong performance in Q1.
Toga: The franchise delivered $359 million in global net revenues, representing a 36% increase compared with the first quarter of 2020 for.
Toga: Importantly, this represents continued and robust growth from our base business.
Speaker Change: Hello, Terry Atti Pn since the CME indication approval came in the final days of the quarter.
Speaker Change: In the U S combined sales of all possible and Umbro truck in the first quarter increased by a robust 45% compared with the first quarter of 2024.
Speaker Change: Continue to drive strong growth momentum.
Speaker Change: 45% year over year growth was primarily driven by the following.
Speaker Change: At 32% increase in demand driven by the strength of ongoing <unk> patient uptake.
Speaker Change: We are pleased with the growth in demand, which has been consistent over the past five quarters. Despite new competition entering the market at the end of 2023.
Speaker Change: Which we believe is reflective of the leadership position, we are establishing a growing piece.
Speaker Change: <unk> end market over the last six years.
Speaker Change: The remaining growth in the quarter, 13% is related to favorable adjustments in gross to net deductions combined with stocking dynamics as inventory in the distribution channel modestly increased in Q1.
Speaker Change: I will share additional detail on our <unk> performance in the U S. On the next slide.
Speaker Change: Now, let me turn to our international markets for just a quick one moment, where the GTR franchise grew 24% compared with the first quarter of 2024.
Speaker Change: Similar to the U S. The year over year growth was primarily driven by increased demand for our withdraw as patient uptake remains robust.
Speaker Change: Bottom line.
Speaker Change: <unk> base business is very strong and continues to grow.
Speaker Change: Before I provide additional color on early days of our TT RCM launch, let me touch briefly on the dynamics, we're seeing in the U S <unk> markets.
Speaker Change: A full year after the launch of a new competitor a mutual remains the clear market leader in the U S. Maintaining a majority of new patient starts we downwards, capturing approximately 70% of new patient starts in the first quarter of 2025.
Speaker Change: This results from our strong brand value proposition.
Speaker Change: Coupled with continued disciplined execution by our commercial teams.
Speaker Change: What's more is it.
Speaker Change: Anticipated, we are finding that in categories, where the majority of patients remain on treatment.
Speaker Change: More treatment options mean, more voices, helping to raise disease awareness ultimately accelerating category growth and increasing the number of patients on therapy.
Speaker Change: In other words this continues to be a growth story.
Speaker Change: Patients are benefiting.
Speaker Change: With similar dynamics anticipated across <unk> <unk>.
Speaker Change: <unk> and <unk>, we believe about 80% of the global addressable population remains on <unk>.
Speaker Change: This bodes well for the opportunity ahead in <unk>.
Speaker Change: Now onto our new indication.
Speaker Change: Momentum is building fast and the team is laser focused on delivering a successful launch.
Speaker Change: On March 20th we received FDA approval for our truck is the first and only silencer approved in the U S for <unk> and the only treatment indicators in the U S for both the Polyneuropathy manifestations of heritage to the ATR and cardiomyopathy manifestation of ACTH.
Speaker Change: Sure.
Speaker Change: Though it's still early days, we are very encouraged by what we're seeing.
Speaker Change: Our first priority at Yates was the access setup.
Speaker Change: And we're pleased to say, it's progressing exactly to plan.
Speaker Change: Thus, enabling the second half growth story that we have described on our prior calls.
Speaker Change: Though it's of course early to quantify trends.
Speaker Change: I would like to share some examples of the very encouraging progress we're already seeing.
Speaker Change: Particularly with regard to the access and health system formulary reviews that we have shared would be the early priorities and key enablers of our second half launch momentum.
Speaker Change: Let me explain why we believe this launch is off an exceptional start with some specific examples.
Speaker Change: First we're seeing robust patient initiations across all payer segments.
Speaker Change: Medicare fee for service Medicare advantage and commercial.
Speaker Change: We are continuing payer engagements and a majority of contracted lives are covered by value agreements that include the cardiomyopathy line access.
Speaker Change: As expected the majority of which are patients to date paid zero dollar co pay in ATT RCM like.
Speaker Change: Like with <unk> P M.
Speaker Change: And we continue to have widespread payer coverage.
Speaker Change: Exactly in line with our expectations.
Speaker Change: Second and this is important.
Speaker Change: About 80% of TCR volume flows through approximately 170 health systems.
Speaker Change: Our team's focused efforts enabled inclusion of our mutual on formulary in more than half of these health systems within short four weeks off label.
Speaker Change: Expansion.
Speaker Change: In fact, the majority of these accounts have already initiated a much of a treatment for <unk> patients underscoring that with the proper setup. These accounts do indeed come online.
Speaker Change: And finally in terms of treatment choice.
Speaker Change: We're seeing healthy demand across both.
Speaker Change: Prior on which our prescribers as well as first time almost with prescribers.
Speaker Change: And what we are hearing from prescribers is that yes, mitra disruptive value proposition resonates.
Speaker Change: And is enabling this early and encouraging launch momentum.
Speaker Change: Early feedback from patients and physicians indicate clear excitement for a new mechanism of action in ATT RCM that kind of rapidly knockdown GTR.
Speaker Change: Hcp's further highlighted that our new trust clinically and statistically significant benefits in Standalone, all cause mortality, coupled with demonstrated preservation of functional capacity.
And quality quality of life, we're on which I make significant impact on <unk> and six minute walk distance are highly differentiating.
Speaker Change: Furthermore, physicians I appreciate how quarterly.
Speaker Change: <unk> administered dosing gifts peace of mind for verified adherence.
Speaker Change: In terms of early utilization here too we are very pleased by early signals, we see broad use in line with our expectations more specifically.
Speaker Change: We're seeing him which are used in first line. We're also seeing stabilizer progressive patients switch to ultra.
Speaker Change: We're seeing broad utilization, including an academic and community settings.
Speaker Change: Supported by the broad alternate site of care networks, that's been established.
Speaker Change: Simply put.
Speaker Change: The mechanism of action and the well established safety and Tolerability of Ultra hits Hall our.
Speaker Change: Our consistent data speaks volumes.
Speaker Change: <unk> administered verify it tears, Israel and patients are getting on therapy on <unk>.
Speaker Change: Im good truck most with.
Speaker Change: Zero copay.
Speaker Change: Conclusion, the launch is off to an exceptional start.
Speaker Change: These are of course, the early days of launch.
Speaker Change: We will continue to share more as the launch progresses.
Speaker Change: Including bought qualitative and quantitative launch indicators.
Speaker Change: We have provided full year guidance projecting robust 36% year over year growth in our TTS franchise revenues at the midpoint of the guidance.
Speaker Change: This reflects our confidence in the launch and the future potential of this franchise.
Speaker Change: Now with a few early weeks of experience since launch.
Speaker Change: We remain as confident as ever.
Speaker Change: In conclusion.
Speaker Change: We are pleased with the results in the first quarter with both our <unk> and rare franchises delivering strong growth in patients on therapy.
Speaker Change: As well as delivering robust year over year growth in revenues, giving us confidence.
Speaker Change: Ability to deliver substantial growth at.
Speaker Change: Cheap profitability and most importantly, deliver our highly differentiated and transformative medicines to more patients in need around the world.
Speaker Change: With that I will now turn it over to push call to review, our recent R&D and pipeline progress push call.
Speaker Change: Thanks, Tony and good morning, everyone.
Speaker Change: So my colleagues I'm very excited.
Speaker Change: The launch of <unk> and ATT RCM at the end of Q1, and then we've hit the ground running to educate physicians and patients and drive awareness and I'm delighted by the early progress that told you just described which is based on great execution by our commercial and medical teams and the impressive Helios B results summarized here in.
Speaker Change: In a population representative of today's <unk> cm patients nearly half of whom were already being treated with a stabilizer. We saw a substantial benefit in boots on cardiac outcomes.
Speaker Change: Putting a 35% to 36% reduction in the risk of all cause mortality alone.
Speaker Change: We saw broad benefits on a series of other important clinical parameters, such as cardiac biomarkers and good credit graphic measures and disease progression.
Speaker Change: These together with the encouraging safety profile.
Speaker Change: Frequent HCP administered dosing regimen that enables a high degree of adherence support the potential for <unk> to become a first line therapy in this disease.
Speaker Change: Based on these exceptional data we quickly submitted filings to regulatory agencies in key geographies and we're pleased with the pace of progress. Thus far we of course achieved U S approval of <unk> in March followed shortly thereafter by approval in Brazil and earlier. This week, we announced that the committee for medicinal products for human use of the European.
Speaker Change: Medicines agency has adopted a positive opinion recommending approval of <unk> in <unk>. This should enable the European commission to approve and Butcher in Europe in Q3, and possibly in Q2.
Speaker Change: So things are progressing swiftly and we look forward to launching in other important territories in the coming quarters.
Speaker Change: Now beyond those primary results that I discussed a moment ago, we continue to share additional insights from the Helios B dataset.
Speaker Change: At ACC last month, we shared a few new analyses that further support <unk> compelling profile.
Speaker Change: And one we highlighted echocardiographic data, which demonstrated that treatment with <unk> led to significant improvements in diastolic function and attenuation of declines in left ventricular and right ventricular systolic function at month 18 compared to placebo.
Speaker Change: These indicate that <unk> is favorably impacting cardiac function, which is truly remarkable.
Speaker Change: We also shared in exploratory subgroup analysis, which demonstrated <unk> reduced all cause mortality and recurrent CV events across a range of baseline heart failure severities in patients with this disease and.
Speaker Change: And lastly, a separate analysis confirmed that <unk> significantly maintained or improved functional capacity and patient reported health status and quality of life compared to placebo over 30 months. These last two analyses were also published in the journal of the American College of Cardiology.
Speaker Change: We look forward to continuing our analysis of this landmark study and educating the <unk> amyloidosis community about the differentiated profile of in vitro.
Speaker Change: Further to our leadership and commitment to innovation.
Speaker Change: In this disease into these patients at R&D day in February we shared updated plans for pivotal studies of <unk>. Our next generation RNA therapeutics targeting <unk> with the potential for deeper knockdown and biannual dosing.
Speaker Change: <unk> will be a randomized double blind events driven.
Speaker Change: Outcome study in <unk> patients with either hereditary or wild type disease, and a medical history of symptomatic heart failure Nyj class one through three.
Speaker Change: All patients will be allowed to be on background stabilizer therapy, approximately 200 patients will be randomized and the primary endpoint will be a composite of all cause mortality and CV events.
Speaker Change: Primary analysis will be event driven.
Speaker Change: A minimum 24 months of the last patient enrolled.
Speaker Change: If successful we target launching <unk> and <unk> around 2030.
Speaker Change: In parallel we have the goal of bringing decrease ran to patients as quickly as possible and see an opportunity to do so in hereditary <unk>.
Speaker Change: Not yet prepared to discuss the full details of Triton PM. We plan to initiate the pivotal study by the end of this year and anticipate the potential to launch in this indication. Several years ahead of <unk>, we look forward to sharing more details in due course.
Speaker Change: R&D date also showcased a number of exciting advancements earlier in our pipeline that underscore the bright future for RNA therapeutics across a broad range of diseases.
Speaker Change: Key among these updates we disclosed two new clinical programs.
Speaker Change: And $43 24 targets <unk> 14 for the treatment of type two diabetes with a novel mechanism of action and acts as an insulin sensitizing.
Speaker Change: Addressing a tremendous unmet need in diabetes, where insulin resistance is a major issue with.
Speaker Change: We're pleased to announce today that we've initiated the phase one study for <unk> hundred $43 24.
Speaker Change: The second program under it.
Speaker Change: The targets liver derived plasminogen and.
Speaker Change: And this molecule represents a potential universal hemostatic agent for the treatment of bleeding disorders without the risk of thrombosis.
Speaker Change: Another key highlight was our update on delivery solutions with best in class potential for adipose muscle heart and kidney tissue as well as a new approach to crossing the blood brain barrier as.
Speaker Change: As we announced at our R&D day, our goal is to achieve delivery of RNA Therapeutics every major tissue by 2030.
Speaker Change: Lastly, I'd like to congratulate our partners at Santa fee for securing FDA approval for <unk> for <unk> for hemophilia, a or b with or without factor eight or nine inhibitors with this approval <unk> becomes the 6000 island discovered RNA therapeutic approved by the food and <unk>.
Speaker Change: Doug Administration.
Speaker Change: In summary, this remarkable and unique pace of innovation puts us in a great position to have a deep self sustainable pipeline that can continue to deliver meaningful impact to patients for many years to come.
Speaker Change: And with that let me now turn it over to Jeff to review, our financial results and upcoming milestones Jeff.
Jeff: Push call and good morning, everyone I am pleased to be presenting the summary about the island Q1, 2025 financial results and discussing our full year guidance.
Speaker Change: Before turning to the details on our Q1 financial performance I would like to further some of <unk> comments on the early observations of our cardiomyopathy launch.
Speaker Change: <unk> pleased with the commercial execution demonstrated by the U S GTR team thus far.
Speaker Change: <unk> revenue being a second half story with the second quarter, focusing on continuing to build awareness unlocking patient access and supporting the trees and supporting treatment initiation for patients given.
Speaker Change: Given the early progress the toga described I am confident in reiterating our financial guidance, including GTR product sales, which I will discuss later.
Speaker Change: With that let's begin with a summary of our P&L results for Q1 2025 compared with prior year.
Speaker Change: Total product revenues for the quarter were $469 million or 28% growth versus last year, driven by the 36% growth in our TTS franchise with particularly strong performance in the U S. Market has told you described.
Speaker Change: <unk> also noted in vitro was approved for <unk> cardiomyopathy with only one week remaining in the first quarter. So did not contribute to our Q1 results.
Speaker Change: Collaboration revenue for the quarter was $99 million or 16% decrease compared to the first quarter of last year.
Speaker Change: Decrease was primarily driven by the $65 million of Roche milestone achieved Q1 dollars 24 related to the first patient being dosed in the cardiac <unk> III study.
Speaker Change: Partially offset by the $30 million milestone achieved from beer and reimbursements from our collaboration with Regeneron in Q1 25.
Speaker Change: Royalty revenue for the quarter was $26 million, representing a $16 million increase compared to last year driven by Novartis has increased <unk> sales.
Speaker Change: Gross margin on product sales was 85% for the quarter, which was consistent with the first quarter of 2024 is the in vitro royalty rate payable to <unk> resets at the beginning of each calendar year for.
Speaker Change: For the balance of the year, our gross margin on product sales is expected to decrease as the in vitro average royalty rate escalates, driven by annual sales, reaching higher royalty tiers.
Speaker Change: Our non-GAAP R&D expenses were $241 million were in line with prior year as increases in clinical trial expenses associated with our cardiac III study for <unk> and then <unk> phase II <unk> study were offset by reductions related to wind down of the Helios B Phase III study, we do expect that R&D expense.
Speaker Change: This will increase compared to 2024 for the balance of the year as phase III studies for both Dolby surround an increase around ramp up.
Speaker Change: Our non-GAAP SG&A expenses increased 12% in the first quarter compared to the same period last year, primarily driven by increased investments in support of <unk> cardiomyopathy launch.
Speaker Change: Our non-GAAP operating profit for the quarter was $75 million, representing a $73 million improvement compared to last year, driven by the strength of our topline growth combined with more moderate growth in operating expenses.
Speaker Change: Finally, we ended the quarter with cash cash equivalents in marketable securities of $2 6 billion compared to $2 7 billion at the end of 2024.
Speaker Change: Now turning to our guidance, whereas I previously mentioned, we are reiterating our 2025 guidance as presented during our last earnings call and is summarized in our guidance slide.
Speaker Change: Additionally, I'd like to provide two additional points of perspective our.
Speaker Change: Our guidance continues to assume foreign exchange rates as of December 31, 2024, which are noted in the footnote of our guidance slide.
Speaker Change: As you are aware the U S. Dollar has weakened considerably in recent weeks.
Speaker Change: The current FX rates continues through the remainder of the year, we expect currency upside relative to our current product sales guidance of approximately $50 million.
Speaker Change: We will reevaluate our full year guidance for FX in Q2.
Speaker Change: Secondly, I'd like to make some brief remarks on the potential impact of recently announced tariffs as well as pharma tariffs that are currently being contemplated.
Speaker Change: As it relates to the tariffs that are now in place, we do not see a material impact on our business and believe we can absorb any resulting cost increases within our existing 2025 financial guidance.
Speaker Change: As it relates to the pharma tariffs that are being considered there is obviously still uncertainty as we await the drafting of the final rules. However, we are comfortable sharing some high level perspective that reflects our positioning as a company.
Speaker Change: Our commercial supply chain is global in nature with the majority of our products are manufactured through third party contract manufacturing organizations in the U S. Additionally.
Speaker Change: Additionally, our intellectual property is also primarily based in the U S. We think both of these factors position us well in terms of limiting exposure to potential future pharma tariffs. We will provide further updates as we get more clarity on tariff policy.
Speaker Change: Let me now turn from financials and discuss some key goals and upcoming 2025 milestones.
Speaker Change: We intend to initiate the Triton cm phase III study of <unk> in <unk> in the first half of 2025 Triton.
Speaker Change: <unk> is expected to initiate by the end of 2025.
Speaker Change: We expect additional approvals of <unk> and <unk> cardiomyopathy in Japan in Q2, and the European Union in Q3.
Speaker Change: And to report data from the cardio <unk> phase III trials as I'll be surround the second half of the year.
Speaker Change: These results will help inform the final design for the phase III, Steve out if there'll be Saran, which we also expect to initiate in the second half of 2025.
Speaker Change: Let me now turn it back to Christine to coordinate our Q&A session. Christine. Thank you, Jeff Operator, we will now open the call for your questions to those dialed in we would like to ask you to limit yourself to one question each and then get back in the queue. If you have additional questions.
Speaker Change: Thank you.
Speaker Change: Ladies and gentlemen, we will now begin the question and answer session. So do you have a question. Please press the star followed by the one on your Touchtone phone you will hear a pump that you had has been raised should you wish to come from the polling process. Please press star followed by the two and if Youre, losing a speaker phone. Please lift the handset before pressing any case.
Speaker Change: Our first question comes from Kristine Amit at Bank of America. Please go ahead.
Kristine Amit: Hi, guys. Thanks for taking my question I'm, sorry, if I missed this on the prepared remarks.
Kristine Amit: Wanted to get a little bit of it.
Speaker Change: Additional color.
Speaker Change: Initial patients that are starting treatment.
Speaker Change: The prescriber base.
Speaker Change: Will that have already had previous experience with an ultra or are you seeing a mix of doctors that are also completely Nina.
Speaker Change: Yes, that's a great question and one for talk but I think what's really encouraging.
Speaker Change: About about the launch of <unk> cardiomyopathy is it really kind of all the indicators are flashing green and we're really seeing.
Speaker Change: Broad and balanced use across all aspects, but <unk>, perhaps you can speak specifically to the <unk>.
Speaker Change: Prescriber base.
Speaker Change: The way we see this as a really good broad uptake, we see cardiologists that are as familiar with the product, but we're also seeing a pretty good uptick in terms of those cardiologist that have now tried downwards. So that's really really encouraging but the most of our thinking encouraging part is that.
Speaker Change: What I highlighted our number one priority, which was at the gate to setup and enables second half momentum by actually setting up these institutions to be formulary ready and within a short four weeks, we've been able to get to into formulary of half of those that caused that to me is really opening up.
Speaker Change: An incredible opportunity for us.
Speaker Change: Thanks, <unk> next question.
Speaker Change: Thank you. The next question comes from Gena Wang of Barclays. Please go ahead.
Gena Wang: Thank you.
Speaker Change: So I can only.
Gena Wang: So our first full quarter of revenue.
Gena Wang: I think a 79 million for to sadness and a $37 million for Tobey youre cutting out.
Gena Wang: So which one do you think it's a good benchmark for industrial first quarter revenue and also my quick calculation for you.
Gena Wang: Franchise.
Gena Wang: First in the last quarter of roughly $17 million over quite a close any revenue contribution from <unk>.
Gena Wang: <unk> cardiomyopathy.
Gena Wang: So I think Thats really for you it was actually quite hard to hear you seen it but I'll try I think there was one question around how do we think about benchmarks for the.
Gena Wang: CMS cm goals.
Gena Wang: Yes.
Gena Wang: Happy to take that Gina.
Gena Wang: First and foremost we've already laid out.
Gena Wang: Our year end guidance, so thats really important to demonstrate that the mid year point.
Gena Wang: The midpoint of the total growth is going to be about 36%. That's obviously our goal is to meet and hopefully exceed that year end guidance now in respect to the.
Gena Wang: First quarter results of those two stabilizers I think it's really important to highlight the categories growing if you take out the IRI impact off of the Fabulous.
Gena Wang: Both products have grown in fact, I think the Fabulous had led that growth. So that's a clear good early sign of how this category remains.
Gena Wang: Untapped in terms of growth.
Gena Wang: So I mean thats great.
Gena Wang: Look we really are excited to be in a class of our own in this market with a single focal.
Gena Wang: Mechanism and a differentiated profile.
Gena Wang: Just come back to <unk> I'm not sure I heard all of your question.
Gena Wang: Sorry, yes, so I can get.
Speaker Change: Yes, sure can you hear me.
Gena Wang: Better now.
Gena Wang: Okay. So.
Gena Wang: Yes. The second question is regarding the this quarter number quarter over quarter growth. When we look at the ETF franchise. It's about 17 million just wondering any revenue contribution from <unk>.
Gena Wang: <unk> cardiomyopathy.
Gena Wang: No no actually made those points in our prepared remarks, it's it's all it's all <unk> polyneuropathy.
Gena Wang: This speaks to again the profile advantage here that we have in place of the fee and.
Gena Wang: The quality of commercial execution, I'm really delighted actually by the strength of our play neuropathy business with terrific growth target you want to add anything.
Gena Wang: Thank you and our next question.
Speaker Change: Thank you. Your next question comes from Greg Harrison at Scotia Bank. Please go ahead.
Speaker Change: Hi, everyone. Good morning. This is curious vitale I'm, sorry, Greg Harrison Congrats on all the progress this quarter and thanks for taking our question.
Speaker Change: Im curious to hear how you're leveraging your commercial expertise from thinking about additional strategic efforts are you, making on the commercial front to be competitive in capturing <unk>.
Speaker Change: <unk>. Thanks.
Speaker Change: Great question. So look I think it's fair go straight to you yes.
Speaker Change: First and foremost as <unk> indicated we come with an entirely new mechanism of action that clearly sets us apart in terms of.
Speaker Change: Any any stabilizers are out there given that we work on upstream and rapid knockdown clearly.
Speaker Change: They manifested itself with Helios B results were helped to patients where bolt on placebo as well as on on.
Speaker Change: The study arm were actually already on a stabilizer.
Speaker Change: And the results are obviously.
Speaker Change: Compelling.
Speaker Change: But frankly, when you think about the disease itself given that it's a rapidly progressing fatal disease physicians are first and foremost interested in.
Speaker Change: Treating the disease early.
Speaker Change: And actually trading with the best possible option they have and in that case, we believe actually withdraw provides a real clear first line option.
Speaker Change: Now in terms of those physicians who are considering.
Speaker Change: Switch, we're already seeing that in our broad patient uptake and we believe that's going to continue to be the case given that half the patients.
Speaker Change: The most recently published.
Speaker Change: ACC.
Speaker Change: Study that shows that patients do progress our stabilizer. So we believe actually we're going to remain a very important option for those patients are progressing on any stabilizer.
Speaker Change: Thank you <unk> next question.
Speaker Change: Thank you. Your next question comes from Ellie Merle at UBS. Please go ahead.
Ellie Merle: Hey, guys. Thanks for taking my question and congrats on the early launch progress just in terms of <unk> I guess.
Ellie Merle: What do you expect or what are you seeing in terms of the use of the first injection free versus guidance straight to paid track.
Ellie Merle: Maybe assuming that more with to go straight to pay attract Greg your comments on the formulary.
Ellie Merle: And then just a follow up.
Ellie Merle: On your commentary on formulary.
Ellie Merle: I guess since youre already on formulary and more than half of the year.
Ellie Merle: 70 priority centers are you seeing any change to the price.
Ellie Merle: Yes for Polyneuropathy from these centers and just your latest expectation on how those price level pro out between Pinar apathy versus cardiomyopathy. Thanks.
Ellie Merle: So two questions I think one about our quick start program in the second around formulary and yes, I mean, maybe I'll take the second question first we have a single price and we don't see any price difference between our both of our indications which are both rare rare conditions now in terms of our I think you are.
Ellie Merle: You're referring to are our quick start program.
Ellie Merle: Look I mean, if you take a step back.
Ellie Merle: We have plethora of services.
Ellie Merle: That is actually developed by our in house patient support program, helping patients to actually navigate.
Ellie Merle: Aerie comps ex U S health care system.
Ellie Merle: And frankly.
Ellie Merle: If you look at our track record our conversion rates.
Ellie Merle: When a patient comes into those patient out programs is is actually very fast and is very effective as patients stay on our <unk> therapy now that program itself.
Ellie Merle: Includes our quick start program.
Speaker Change: And we had it frankly for that program for years and so far we're seeing a very limited use of this quick start program as we had seen in polyneuropathy.
Speaker Change: And what we're really pleased this frankly those patients that are coming into the program across the different payer mix or getting on therapy, whether its through Medicare fee for service Medicare advantage as well as in commercial.
Speaker Change: And I must also add.
Speaker Change: As we anticipated those most of those patients are paying zero co pay as we had seen in polyneuropathy as well.
Speaker Change: Importantly, we're getting good good patient access.
Speaker Change: Next question.
Speaker Change: Next question comes from Gary Nachman at Raymond James. Please go ahead.
Gary Nachman: Great. Thanks for taking the question and congrats on the progress.
Gary Nachman: So with the <unk> in the early launch phase and getting some traction is that creating a headwind, Brian which was launched in cm or is it helping create more noise for the entire category.
Gary Nachman: What's the early read on physician receptivity to our new <unk>.
Gary Nachman: <unk> versus a new stabilized are out there.
And then just in May.
Gary Nachman: Specifically on the reimbursement side, how are the dynamics playing out as a part b drug versus the stabilizers that are part D drugs. Thank you very much.
Gary Nachman: Yes.
Gary Nachman: So I think yes.
Gary Nachman: Toga in it.
Gary Nachman: Made the point that we really arent, particularly growth story and what we're just seeing is actually continued evidence of a large growing and frankly unsatisfied market and I think we're very.
Gary Nachman: We're excited to be able to participate in this market with the recent launch of <unk>.
Speaker Change: Andrew It shrink cardiomyopathy I think the second question was around the.
Gary Nachman: Pub.
Gary Nachman: Parties in that mix and how that plays III. So look I mean, I think a good example of how we've been managing part D. Part D dynamics frankly is what we have already demonstrated in polyneuropathy.
Gary Nachman: Just shared with you in.
Gary Nachman: This past quarter after one year of launch.
Gary Nachman: As a part D product, we've actually been able to achieve 70% of all new patients and continue to grow.
Gary Nachman: Actually in an accelerated faith based from a larger base of business now in terms of what we're seeing in this new line extension, we have we're seeing no headwinds and thankfully with the value based agreements, we have and we're seeing this uptake across all payer mixes. So there are no.
Gary Nachman: Signals that we can.
Gary Nachman: Iterate right now that would suggest otherwise.
Gary Nachman: And in terms of.
Gary Nachman: In terms of differentiation and whether we're actually seeing any headwinds look what really sets us apart.
Gary Nachman: Is the strength of our data.
Gary Nachman: It's complete consistent and cohesive.
Gary Nachman: We're presenting the full picture with scientific rigor.
Gary Nachman: And in contrast, we are seeing some some in this space selectively highlighting data points with creative interpretations.
Gary Nachman: And we already see and believe investors and physicians alike value declarative and integrity of our comprehensive dataset and that's exactly what we're delivering.
Gary Nachman: And yes it is.
Gary Nachman: We've been doing for the past several years as we focused on meeting the needs of patients.
Gary Nachman: Next question.
Jessica Fye: Next question from Jessica Fye of Jpmorgan. Please go ahead.
Jessica Fye: Hey, guys. Good morning, Thanks for taking my question.
Jessica Fye: You mentioned, providing additional launch indicators with Q2 results can you just touch on what those will be and then of the 170 priority Health systems. I think you mentioned more than half already have <unk> on formulary can you just speak to your goal of where you want that number to stand maybe next quarter and by year end. Thank you.
Jessica Fye: Yes, yes.
Yes look I mean, I think we've been very consistent about how we are describing Linda.
Jessica Fye: And the way we are updating the <unk>.
Jessica Fye: Investors and in this case, our goal frankly was to complete.
Jessica Fye: The inclusion and formulary by year end.
Jessica Fye: You can see we're taking our progress has been faster and we're very pleased with that so.
Jessica Fye: So it's absolutely a day by year end, we want to be able to get there. So that we have access what's most encouraging frankly about the most recent development is a big majority of these accounts not only included in the formulary, but those are costs that have included the formulary already actually.
Jessica Fye: Initiating patients on therapy, and that's incredibly encouraging now in terms of our acuity Q2 data.
Jessica Fye: I think the biggest update is going to be our our revenue uptake.
Jessica Fye: And the pull through and how we're actually seeing denim and obviously, we're committed to providing additional color in terms of.
Jessica Fye: The broad patient uptake and what that really looks like and and prescriber update and soon and so forth. That's great. We're looking forward to sharing much more so on next earnings call.
Jessica Fye: Next question.
Speaker Change: Next question from Lee to borrow at TD Cowen. Please go ahead.
Speaker Change: Good morning, guys.
Jessica Fye: Hi.
Jessica Fye: You mentioned that you're seeing first line use already.
Speaker Change: Can you speak a little to that type of patient or profile of patient.
Jessica Fye: It is.
Speaker Change: He is making up that first line use.
Speaker Change: And then I.
Speaker Change: Verification on formulary inclusion.
Speaker Change: When you have them.
Speaker Change: Formulary inclusion is that where any.
Speaker Change: Prior authorization requirements sort of gets set for use in the health system.
Speaker Change: As well in <unk> and <unk>.
Speaker Change: If so how should we be thinking about what you're seeing from a prior off same formulary inclusion.
Speaker Change: Yes.
Speaker Change: We too high I'm grateful for your.
Speaker Change: Question, Let me take this last one first meaning the type of formulary and whether that will require actually prior off. So we're talking about two types of axis here. One is the access where youre dealing with the payers that Medicare fee for service Medicare advantage as well as <unk>.
Speaker Change: Sure.
Speaker Change: They are actually all products are go through medical exception process and and it's usually.
Speaker Change: Essentially if you are a part D product. The prior authorization is it almost all sorts of circumstances is required what makes US unique is in Medicare fee for service there are no prior offs request.
Speaker Change: The second part, which I was alluding to is the 170 health care systems and <unk> systems is just a question of the organization, saying, we're actually going to be buying this product because now it's actually included with this new indication that requires no. Prior authorization. So I just wanted to.
Speaker Change: Make that very clear now in terms of.
Speaker Change: The type of patients that we've seen the first slide whats really exciting about this is when doctors believe actually that I'll withdraw and understand the <unk> profile that has clearly differentiated as a rapid knockdown agents. They don't actually make a lot of differences in terms of what patients.
Speaker Change: I want to put it what they see is if the patient is progressing on a stabilizer. They immediately put that patient on on an upward track if they see a new patient whether thats early offsets or actually there already progressive they immediately put that patient on on the first slide so it's not so much about the types.
Speaker Change: Of patients, but it's also whether the doctor have clearly understood. The compelling data set that we get to presenting with ESPN and additional real world evidence.
Speaker Change: Thanks, Paul next question.
Speaker Change: Next question from Celgene Richter of Goldman Sachs. Please go ahead.
Speaker Change: Hi, This is Tommy on for Zalviso. Thank you for taking our question.
We just want to confirm some of the metrics that you gave out 170 health systems over half on formularies and over 75 initiated one per system around 65 patients give or take is that accurate to think about and just a follow up.
Speaker Change: Im wondering if youre seeing some more standardized guidelines for identifying stabilizer progressing as among hcp's now that we have new options. Thank you.
Speaker Change: So let's talk a why don't you take the first question and then I think Pascal will take 10 seconds, yes.
Speaker Change: Just just to be clear, we clearly laid those out on the slide which essentially says <unk>.
Speaker Change: 80% of the GTR.
Speaker Change: The treatments are initiated through these 170 U S health care systems. These are some regional networks, maybe local networks as well as national networks.
Speaker Change: Once those institutions includes a product because it is a buy and bill product.
Speaker Change: In their formulary that means actually that institution can acquire and can initiate therapy and while we're alluding to is.
Speaker Change: Of those 170 within three to four weeks those systems are already set up as online now big majority of those systems are already initiating treatment in this setting that's what we're referring to.
Speaker Change: I don't know if that captures I think.
Speaker Change: So maybe to get onto their second question for Scott, Yes, Tony in terms of the <unk>.
Speaker Change: Stabilizer progresses, but maybe just a couple of points on this because it does come up not infrequently doctors are well trained cardiologists to evaluate patients with heart failure, and whether they are doing well or not well on therapy and that's why you've seen over the years. Many studies that have said hey, our patients doing on stabilizers and they report 30% to 50% of patients.
Speaker Change: <unk>.
Speaker Change: Do progress on those therapies.
Speaker Change: And so that's I think that's where that's coming from it. So I think they are well trained I think if you look for example at ESC heart failure guidelines that were set up for EQT RCM. They show the doctors look at factors like clinical symptoms or they're hospitalized towards they are walking ability whats their shortness of breath, what's happening on imaging like Echocardiograms for example, let's have.
Speaker Change: Turning with critical Biomarkers like empty pro BNP and troponin.
Speaker Change: And so I think doctors are well attuned to looking at those factors and examine and I think once again really hurt.
Speaker Change: <unk> to us about the profile of <unk> is that on actually every one of those parameters.
Speaker Change: We see a benefit in terms of delaying or favorably impacting these progression I think thats a core aspect of this disease as I mentioned, we just published data showing that he actually theres actually improvements in aspects of cardiac function with this drug which indicate the potential for a disease modifying profile. So.
Speaker Change: That's what's compelling about this I think there will be more guidelines et cetera around this but I think I think really what we're seeing is that.
Speaker Change: Doctors know how to recognize heart failure patients, who aren't doing well and then reaching for the best therapy for their patients.
Speaker Change: Great. Thanks Thats correct.
Speaker Change: Next question.
Speaker Change: Next question from cost Us Polaris at BMO capital markets. Please go ahead.
Speaker Change: Thanks for taking my question and congrats on the progress one question from Us on a booth Altai to push my luck here, a little bit costar market at <unk>.
Speaker Change: But it's by a recently mentioned that they saw an impact from <unk> launch on the number of actual discrete and this impact was particularly from suites base. Yes. So that said I wonder whether youll see the majority of the <unk> coming from sweepstakes.
Speaker Change: Our new pace.
Speaker Change: Any quantitative or directional color that would be very helpful. Thank you.
Speaker Change: I'll take that question and high cost US look I think the way we best describe it given that it's still early days as broad what I'm really really excited as again, we're taking all kinds of patients, including definitely first line patients as well as well as switch patients and.
Speaker Change: And look.
Speaker Change: Those physicians that are.
Speaker Change: Choose to switch they also start new patients on <unk> and that's definitely.
Speaker Change: And early trends that we start seeing.
Speaker Change: By the way before before.
Speaker Change: Before we move on I also want to take a quick moment.
Speaker Change: Since I know the team is always.
Speaker Change: Listen again to call out their phenomenal work what they've done.
Speaker Change: Honestly, what I witnessed leading up to this large entities early days has been nothing short of world class are customer facing and patient support teams are really firing on all cylinders, bringing the exceptional profile on what's right to the patient community Thats long waited for a breakthrough medicine.
Speaker Change: And the early momentum cost us as I shared is strong and all signs point to us being right on track to deliver something truly impactful.
Speaker Change: Okay. Thanks for those perspectives.
Speaker Change: Next question please.
Next question from Luca <unk> at RBC capital markets. Please go ahead.
Speaker Change: Oh, great. Thanks, so much for taking my question Congrats on the progress maybe talk a quick one here on access.
Speaker Change: At least based on our checks it sounds like step edits are likely going to be implemented for commercial patients, but are much much less likely on the Medicare fee for service station, one is that as Sierra characterizations and too.
Speaker Change: So how should we think about the Medicare advantage patients. The one kind of in between the two buckets are they conceptually closer to commercial patients where step edits are likely or are they closer to fee for service patients where step edits are less likely any thoughts there much appreciate it.
Speaker Change: Yes.
Speaker Change: I appreciate the question look.
Speaker Change: First and foremost what we've already seen is a very nice broad uptake on all payer mix. So we're really not seeing any any headwinds in terms of.
Speaker Change: Any restrictions on the product now when it comes to fee for service.
Speaker Change: So it's Charlie there won't be any limitations, so definitely half of their Medicare patients, we will be able to have access very seamless and we're seeing that with.
Speaker Change: Zero Copay I must say in.
Speaker Change: In most of the patients.
Speaker Change: In terms of commercial and Medicare advantage.
Speaker Change: Not seeing any step edits so far.
Speaker Change: And we obviously have established that great.
Speaker Change: Infrastructure through our value based agreements that enables us to be able to actually demonstrate clinical value and that clinical value provides us that similar similar advantage now if there were to be any step edits. We certainly have the tools to be able to address that.
Speaker Change: If there were to be the case, but look we have seen it in polyneuropathy. We're now seeing is CN, we have the patience.
Speaker Change: Getting on treatments.
Speaker Change: And a very quick short amount of time and staying on treatment.
Speaker Change: Thanks to again, the product profile and the support that we provide.
Speaker Change: Okay.
Speaker Change: Sure Tim got one snick.
Speaker Change: Next question please.
Speaker Change: Next question from Thomas <unk> at on the line. Please go ahead.
Paul: Alright, Thanks, Paul from Stifel. I appreciate you taking the time.
As it relates to uptake so far tolga when we identify weeks ago, you talked about how there are certain physicians, who they actually can use the drug without going through a P&C as many.
Speaker Change: Any metrics there like what percent of the prescriber base is that and what are you seeing from that population and then as it relates to Medicare fee for service patients given the ease of access is this the kind of thing where over time, you take a patient who has who has insurance covered by Medicare fee for service could actually get drug the first dose of drug the same day that it's prescribed.
Paul: Thank you.
Paul: Yes, I mean, maybe I'll take care of your last question first look at the end of the day. This is a.
Speaker Change: Healthcare professional administered product, which requires actually injection and buy and bill and that requires some level of color.
Speaker Change: Coordination so it will never be a one day.
Speaker Change: Administration, but it's certainly.
Speaker Change: A very quick given given the kind of support that we provide and we obviously track those patients. While it's also very encouraging is.
Speaker Change: Whether you're a fee for service advantage or commercial patients. These patients come in through a start form where we don't actually see any distinction.
Speaker Change: Just on the insurance coverage we are.
Speaker Change: Neutral to the to the.
Speaker Change: To the payer mix and despite that those patients start getting on therapy and the main reason is that I really want to underlying this.
Speaker Change: Whether it's step at it or prior art. This is a rare and a very very severe disease that rapidly progressing.
Speaker Change: And because of this we see really great coverage of this category as a whole and given what we have to offer and based on the value based agreements that we've established we believe actually we have the ability to to have the appropriate patients put on therapy.
Speaker Change: As a physician requires.
Speaker Change: Right Okay.
Speaker Change: I think we've got time for one last question.
Speaker Change: Next question from Michael <unk> at Morgan Stanley. Please go ahead.
Michael: Good morning, Thanks for taking the question maybe just another one in cardiomyopathy.
Michael: I Wonder if you can talk about the rate of diagnosis and may be if you've noticed a shift since the beginning of the year now that theres two additional products on the market.
And then maybe just secondly, with about 20% of patients currently diagnosed what do you think that could look like at the end of the year. Thanks.
Michael: Two great questions.
Speaker Change: No. Thank you look I think we actually have empirical evidence now what you see in call neuropathy.
Speaker Change: From 50% growth rate to 60% just with another.
Speaker Change: Product in Polyneuropathy already accelerated debt and we remain the market leader now when you look at actually the Q1 results of <unk>.
Speaker Change: Both stabilizers exited in the U S.
Speaker Change: The stabilizer as grown the fastest ever in the quarter I think that's somewhat somewhat also supported by IRA.
Speaker Change: Changes as well, but we see this category grows only accelerating we used to provide analogies with multiple sclerosis and other products now we see it in our own category. So theres definitely evidence of that now in terms of how large the category at how much.
Speaker Change: Diagnosis rates going to go up.
Speaker Change: It's going to be very difficult for us to predict that now, but I think we will.
Speaker Change: And I am sure all voices in this category is going to help accelerate that in the future.
Speaker Change: Good okay.
Speaker Change: Thank you everyone for joining us on the call. So you can here 2025 is off to a great start around on them and we look forward to providing with further updates as the year progresses. Thanks everybody.
Speaker Change: Ladies and gentlemen, Goodbye conference call for today, we thank you for participating and we ask that you. Please disconnect your lines.
Speaker Change: [noise].
Speaker Change: Sure.
Speaker Change: Okay.
Speaker Change: Yes.