Q1 2025 Scholar Rock Holding Corp Earnings Call
Okay.
Speaker Change: Ladies and gentlemen, thank you for standing by and welcome to scholar Rock's first quarter financial results and business update call.
Speaker Change: At this time all participants are in a listen only mode.
Speaker Change: Following the presentation, we will conduct a question and answer session.
Speaker Change: To ask a question. During this session you will need to press star one on your telephone you wouldn't hear an automated message of biting the hand its rate.
Speaker Change: Draw. Your question. Please press star one again.
Speaker Change: Please be advised that today's conference is being recorded.
Rock: I'd like now to turn the conference over to scholar Rock. Please go ahead.
Wrestling: Good morning, I'm Wrestling, not senior Vice President of corporate Affairs, and Investor Relations at scholar Rock with me today are David Halal Chief Executive Officer Akshay. That's now President of R&D. He was chief operating officer, and Vikas Sinha, Our Chief Financial Officer for those of you.
Wrestling: Participating via conference call. The accompanying slides can be accessed by going to the event section of the investors page on our website.
Wrestling: During today's call as outlined on slide two David will provide introductory remarks and provide a general business update.
Wrestling: Jay will review, our clinical and regulatory progress.
vico: He will provide an update on our commercial readiness activities and vico will provide commentary on our company's financial and the summary of our 2025 priorities.
Wrestling: And then we will open the call for questions.
Wrestling: Before we begin I'd like to remind you that during this call we will be making various statements about scholar rock expectation plans and prospects that constitute forward looking statements for the purposes of the safe Harbor provision under the private Securities Litigation Reform Act of 1995.
Any forward looking statements represent our views only as of today and should not be relied upon as representing our views as of any future date I encourage you to go to the investors and media section of our website for our most up to date SEC statements and filings.
David: With that I'd like to turn the call over to David David Thanks, Ross and good morning.
David: Thanks to everyone for joining us on today's call.
David: 2025 is off to a strong start at scholar rock in the first quarter, we made significant progress against our top priority.
David: Bringing a critical math of patients and families living with and suffering from SMA in the U S Europe and around the world.
David: We were very pleased with the positive phase III Sapphire trial, where we showed a statistically significant and clinically meaningful improvement in motor function as measured by the primary endpoint the gold standard Hammersmith scale.
David: And in Q1 to underscore the body of evidence for our clinical map for patients with SMA. We were gratified that our BLA was granted priority review by the FDA with a September 20 <unk> date.
David: The team at scholar rock is working urgently to prepare for our U S commercial launch.
David: Which will be the first country in AR.
David: Series of launches over the coming years.
David: I am confident under Keith's leadership, we will assemble and deploy and experience.
David: Talented and patient centric team committed to the SMA community.
David: Along with key Akshay and because we now have the responsibility to bolster scholar rock's capabilities as we advance our mission to deliver a category map for patients with SMA.
David: This is what we know well and what we do well.
David: We join scholar rock at a time of great strength and opportunity as we scale for the next phase of growth to a commercial stage fully integrated global biopharmaceutical company.
David: Globally, nearly 35000 patients with SMA have received SMN targeted therapies and at scholar rock, we are working with urgency to prepare for a global launch.
David: Which we anticipate will commence in the U S in Q3.
David: We are also beginning to set the table to serve patients with SMA in Europe Asia Pacific and Latin America.
David: Our ambition at scholar rock is that any patient with SMA that can benefit from our political map should have access to a critical math.
David: I am confident that the global opportunity with <unk> in SMA alone offers the potential for many years of sustainable growth that will power our company through the end of this decade and into the next.
While we are very focused on our large opportunity to serve patients with SMA. We are continuing to evaluate expanding that study of <unk> into other rare severe and debilitating neuromuscular disorders Akshay will touch upon this shortly.
David: Now I'd like to turn to our exploratory cardio metabolic program the embrace proof of concept study.
David: Our effort here is to understand the role we may play in the treatment of obesity.
David: We are the world leaders in Myostatin biology, and know well the G. L. P ones have brought needed innovation to patients with obesity and cardio metabolic disorders.
David: While the benefits outweigh the risks muscle wasting and lean mass loss is something that the medical community is trying to address the embrace study is our initial effort in the cardio metabolic space and will provide insights that will guide us moving forward, we remain on track to share top line results.
David: In June.
Our achievements in 2025 to date underpin the opportunity before us to bring the potentially life transforming benefits of a political map to patients and families with SMA.
David: We are focused on delivering for patients with akshay, leading efforts in collaborating with U S and EU regulators.
David: Key applying a similar approach from <unk> Garten Mg to the commercial opportunity for our political map and be cost focused on disciplined capital allocation to fuel near and long term growth.
David: With that I'll turn the call over to Akshay to provide a more detailed update on our R&D progress akshay. Thanks.
Akshay: Thanks, David.
Akshay: I'm delighted to be here after six years on the board of scholar rock as a physician scientist I could not be more excited to have seen the concept of a better map our highly innovative anti myostatin antibody go from bench to bedside without remarkable satellite data.
Speaker Change: Sapphire assured, but a bit of a map has the potential to reverse the progression of SMA from a loss of motor function to a gain of function specifically the study demonstrated a statistically significant improvement in Hammersmith most patients on placebo listened.
Certainly patients treated with Paragon map and approximately three fold high chance of a three point or greater increase in Hammersmith versus those on placebo.
Speaker Change: Along with the very encouraging safety profile. The Sapphire data suggests that our Pet-sitter map has great potential to provide clinically significant benefit to patients with SMA. Despite the use of background SMA target therapies.
Speaker Change: Additionally, at the muscular Dystrophy Association conference in Dallas in March the detailed data from the Sapphire trial were presented for the first time.
Speaker Change: These data demonstrated that treatment with a headroom app achieved clinically meaningful and consistent benefit in motor function across Prespecified SMA patient subgroups, including the type of SM and targeted therapy age age of initiation of SMA therapy and geography.
Speaker Change: Efficacy was also supported by additional analyses of outcome measures of motor function, such as the revised upper limb module and the World Health organization Motor Development Index.
Speaker Change: As David mentioned, our BLA was accepted under priority review by the FDA and the MAA was validated by the EMA in Europe.
Speaker Change: Turning first to the FDA, we were gratified that our BLA for our critical map was granted priority review by the agency with a September 22nd producer date.
Speaker Change: We feel the potential clinical benefits of repetitive map as demonstrated by a phase III trial underscored by the Fda's priority review designation.
Speaker Change: By definition, a priority review designation by the agency conveys the capacity of our critical map to potentially impact unmet need in SMA by either being a treatment for a serious or life threatening condition or provide a significant improvement in safety or effectiveness of existing treatments.
Speaker Change: I'm excited that our team continues to work collaboratively with regulators and that we remain on track.
Speaker Change: With the successful Sapphire, we're just beginning to tap into the broader potential about truly innovative biotech and platform is.
Speaker Change: There's much more we can do with the promise of competitive map and our platform by delivering advances.
Speaker Change: Vascular disease, including the muscular dystrophy, such as DMD NFS HD.
Speaker Change: Additionally, we are advancing <unk> hundred 39 are highly innovative and potent sub Q anti myostatin antibody to the clinic.
Speaker Change: Based on preclinical data S. RK 49 has the potential to inhibit myostatin and increase muscle mass and creates options for our pipeline.
Speaker Change: <unk> on track to file the IND application, perhaps a faithful to benign to support the first in human study in Q3.
Speaker Change: Now earlier, David mentioned, the potential Rhodes scholar rock 'n play in addressing lean mass and cardio metabolic diseases.
Speaker Change: As we all appreciate whilst GOP on Gulf of quantitative benefits in terms of weight loss much more needs to be done from a qualitative perspective regarding preserving lean mass.
Speaker Change: Notably a quarter to a third of the weight loss with GOP once is due to the loss of muscle looking.
Speaker Change: Looking forward it will be important to preserve muscle from the viewpoint of the associated metabolic benefit and a healthier approach to weight loss.
Speaker Change: Our embrace study is our ongoing phase II trial to investigate preliminarily that potential of further developing our highly selective aframax asset purchase in patients with obesity with the goal of reducing the losses lean mass.
Speaker Change: We look forward to the upcoming readout of our initial data from embrace in June 2025.
Speaker Change: In summary, we remain focused and on track to deliver on our key priorities. This year, we will one drive the U S approval of <unk> in Q3, 2025, and advanced the EU Court approval in 2026.
Speaker Change: To initiate a study of our critical map for infants and toddlers with SMA under the age of two starting in Q3.
<unk>, followed RMB <unk> 49 in the third quarter and finally complete our clinical development plans work with roadmap in additional neuromuscular indications.
Keith: With that I'll turn the key to provide a commercial update Keith.
Keith: Thanks, Akshay and good morning, everyone I'd first like to thank our colleagues from research and development for their focus and commitment over the years to make the September 20, <unk> <unk> date for our pedigree Mab a reality.
Keith: SMA is a disease impacted by both motor neuron degeneration and muscle atrophy.
Keith: Today's therapies only address one piece of the puzzle the motor neurons. Currently there are no approved muscle targeted therapeutics to treat muscle atrophy with a pedigree mab, we have the opportunity to usher in a new era for the treatment of patients with SMA.
Keith: This is a progressive and devastating disease that leads to the loss of mobility limited activities of daily living and a lack of independence.
Keith: Despite the advances made in treating SMA with SMN targeted therapies over the last decade, the contemporaneous natural history data shows that the majority of patients still experience progressive muscle degeneration overtime, the bottomline nearly all patients and families living with SMA.
Keith: Are demanding a transformative new therapy.
Keith: This is supported by a cure SMA survey published last month, where 90% of patients identified that new SMA treatment options improving muscle strength is an important need.
Keith: Our market research and interactions with health care professionals tell us that 80% of treating neurologists agree that preserving muscle should start as early as possible in treating patients living with SMA.
Keith: Today, there are approximately 10000 patients with SMA in the United States and roughly two thirds of them have received an SMN targeted therapy for these patients is akshay shared with our Sapphire data a pit of grab <unk> showed the potential to reverse the progression of SMA.
Keith: From a loss of motor function to a gain of motor function.
Speaker Change: Furthermore, globally. There are approximately 35000 individuals that have already received an SMN targeted therapy neurologists recognized that in the future a treatment approach of dual modalities to target the motor neuron and the muscle will be necessary to treat SMA.
Speaker Change: At scholar rock, we have the opportunity to make a meaningful difference for both children and adults living with SMA first starting in the U S than Europe Asia Pacific and Latin America.
Speaker Change: We are currently building on the momentum that has already been established for a successful U S launch last weekend several of US at scholar rock had the opportunity to meet with patients and their families at the cure SMA walk enroll in Boston.
Speaker Change: We continue our stakeholder engagement and SMA disease education life takes muscle is the first muscle focused SMA disease awareness initiative.
Speaker Change: Our fully staffed U S market access team is currently meeting with key U S commercial and federal payers, our process of hiring and Onboarding, our customer facing team of roughly 50 sales reimbursement and patient support personnel as well underway, we expect to be fully staffed by mid <unk>.
Speaker Change: 2025, well ahead of our potential launch in late September finally, we.
Speaker Change: We believe that a pedigree mab has the potential to be a first in class.
Speaker Change: First in class therapeutic to establish a new standard of care in SMA now I will turn the call over to Vikas, because thank you Keith and good morning, everyone. I am pleased to provide a business update and provide insights into how we are thinking about resource allocation in the future.
Vikas: The opportunity with a particular mop in SMA alone offers the potential for many years of sustainable growth and will enable strategic thoughtful investment in our pipeline to develop new indications and new therapies.
Speaker Change: The number of patients.
Speaker Change: These pipeline investments will be aligned to our commercial success.
Speaker Change: We ended the quarter with cleared and $64 $4 million during the quarter, we continued to increase our investments in commercial readiness.
Speaker Change: Inventory build out.
Speaker Change: As we look ahead, we are prioritizing the commercial launch and ongoing clinical programs, we have an additional $100 million under our debt facility that we can draw down this year to support the upcoming launch, bringing our anticipated run rate into 2027.
Speaker Change: We are working on building a tighter financial plan and we'll share more details over the next few quarters as we move forward I will focus first on driving strong performance with financial discipline next investing in capital efficient commercial buildout and heartfelt cap.
Speaker Change: Little allocation to advance our pipeline with that I will turn it back to David.
Speaker Change: Thanks, because in closing we are acutely focused on the key priorities that will enable us to build and scale scholar rock into the next global biotech powerhouse first regulatory approvals and the upcoming U S launch of <unk> for patients with SMA.
Speaker Change: Followed by a series of country launches in the coming years next develop a pedigree mab for additional rare severe and debilitating neuromuscular diseases, and finally phase our capital allocation and investments thoughtfully to support our Hy Vee.
Speaker Change: <unk> commercial and development initiatives.
Speaker Change: Behalf of every member of the scholar rock team, we are deeply aware of our responsibility to patients and their families and will work with urgency to ensure that no patient with SMA is left behind with that we'll now open the line for questions operator.
Speaker Change: Thank you as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced.
Speaker Change: Your question. Please press star one again.
Allison: First question comes from Allison, Brett <unk> with Piper Sandler Your line is now open.
Allison: Hey, good morning team and thanks for taking the question.
Allison: Just two from me.
Allison: How does that map launched could you just frame for us how your discussions with U S payers are gone.
Allison: A receptive are they at a conference or a combination therapy for pellets and SMN targeting and a muscle targeted therapy in SMA patients.
Allison: And can you can you also describe that feedback for ex U S payers and governments as well.
Allison: And then second could you just characterize interactions with FDA on the pick a mab reveal obviously theres a lot of concern about the state of the agency right. Now. So just just wanted to know if here.
Allison: If you have anything to report on there. Thank you.
Allison: Thanks Sally.
Allison: So why don't I just touch on pricing then Keith will comment more specifically and then Akshay will take on the regulatory update question.
Allison: One of the things that Keith and I have spent a long time sort of thinking about it.
Doing as it relates to in general.
Allison: Really what we're talking about is access for a potentially transformative therapy for a small population of patients.
Allison: And we would sort of expect.
Allison: As we think about.
Allison: Establishing access plans that.
Allison: That we consider sort of pricing for our therapies that would be reflective of.
Allison: The rarity of the disease, the severity of the disease and the value that our that our therapy would provide the patients in this specific case patients with SMA.
Allison: And I think one of the other things that Keith and I think a lot about.
Allison: It's also given the rarity of disease, we would expect that the budget impact.
Allison: To any one single payer in the U S.
Allison: Or even globally would be very limited for the high value proposition as it relates to sort of the engagement with payers largely in the U S. I'll turn it over to Keith for some more commentary.
Keith: Thanks, David So Ali what we know in speaking with payers is that with SMN targeted therapies alone patients can still have progressive motor function loss.
Keith: And at the MBA in Dallas in March we heard that a proportion of patients are already receiving more than one SMN therapy. So I assume <unk> targeted therapy. So right now it's not uncommon that youll see a payer pay for more than one of these therapies as you know with our data when we go in addition to an SMN <unk>.
Keith: <unk> therapy, we see improvement consistent across all age groups of 2% to 21 and so.
Keith: With that in mind, these patients need of better therapeutic and our early discussions with payers.
Keith: <unk>.
Keith: Proven to be positive and we will continue those discussions as for Europe. We plan to as you know we filed and we're going through the regulatory process right. Now we will be preparing to go into reimbursement discussions, let's remember that we are going to sequence through Europe, and we will start with Germany.
Keith: 2026.
Keith: And then we will rollout over time, how we will sequence through the remainder of the countries in Europe. Thanks for the question.
Keith: Hey, maybe a closer look at the.
Speaker Change: Regulatory sure. Thanks, David So thanks, Alex for the person on FDA interactions look it's a time of evolution and change at the FDA, we will recognize that but what I'm delighted by the pace and the collaborative conversations we've had with the FDA are all getting asper routine and we continue to guide that we're focused.
Speaker Change: And on working with the FDA and delivering on that set September 22nd producer date. So really we're seeing no issues specific to a critical mass in the SMA approval.
Speaker Change: And we're also delighted this FDA commissioner Martin macro rate actually made a point that with all the changes one of the main areas of focus continues to be rare disease.
Speaker Change: Four patients need for rare disease.
Speaker Change: Paul.
Speaker Change: Systems go and we continue to work productively with regulators here and in Europe.
Speaker Change: Okay.
Michael Yee: And our next question comes from Michael Yee with Jefferies. Your line is open.
Speaker Change: Hey, guys. Good morning, maybe just two questions on obesity coming up.
Michael Yee: <unk>.
Michael Yee: Given that it's towards the end of the second quarter, but also that theres two different endpoints I think 24 and 32 weeks are you thinking about providing more data.
Michael Yee: A more complete package of information to help people out in terms of longer term follow up data on some of the metabolic parameters that could come out in June have you thought about that and then just holistically the prior.
Michael Yee: C J I just mentioned.
Michael Yee: 20% to 40% reduction of muscle loss can be great, but I think people are still sort of trying to grasp. What do you think are getting resolved and what is exciting can you give us some color on that thanks.
Yes, great questions Mike.
Michael Yee: Yes.
And I noted this is our first effort at really understanding what role we may be able to play in this space. We know that there is a need that the medical community is trying to address.
Michael Yee: I'll turn it over to Akshay for sort of a closer look at what one might expect in terms of.
As you were saying the 24 week end point, but then the eight week.
Michael Yee: Sort of withdrawal of therapies as well Akshay, yes, thanks, Mike.
Speaker Change: <unk> will be providing the 24 week data. That's the main study we will certainly provide any necessary follow up information that we have that helps us understand that helps everybody else understand the potential of the 19 Myostatin approach.
<unk> space.
Speaker Change: We're excited to test this hypothesis preliminarily, it's an important one I think we all appreciate that loss of muscle is almost certainly not so good thing as people lose weight, especially when it's a quarter to a third of the weight losses due to muscle and so the primary focus will be the 24 week data change in lean mass the safe.
Speaker Change: And helping us understand the path forward, so I'll leave it at that but it's not so long to wait now to get the data.
Speaker Change: And the next question comes from cash from marrow with J P. Morgan Your line is open.
Speaker Change: Hey, David and team. Thanks, so much for taking our questions. This morning. So.
<unk> noted collaboration with regulators and that you remain on track here can you confirm or not if you have completed your mid cycle meeting and if so can you comment on any high level discussions you have had around labeling and at this time what has the agency side about the need or lack thereof.
Speaker Change: For an ad com.
Speaker Change: Thank you.
Speaker Change: Thanks tests.
Speaker Change: As Akshay and I, both noted in our call today, we remain on track and maybe for a closer look on just kind of where we are akshay, Kevin can provide a little bit little bit more detail. Yeah. I mean, there's not much more to provide other than the conversations have been extremely constructive and right on track I would say.
Speaker Change: As to the details of when and how the various interactions with Carter I really think this is the time, we'll space, but I do want to reassure everybody that our ability today to guide on the call that we're heading that September 22 producer date is based on a series of very constructive conversations so I'll leave it at that and more news as we go.
Speaker Change: Got it.
Speaker Change: And the next question will come from David Nearing Garden with Wedbush. Your line is open.
Hey, Thanks for taking the question I just had one on the additional potential indications for particular mab.
Speaker Change: As I recall, there was a lot of exploration in preclinical models at least.
Speaker Change: DMD and related disorders is there any.
Speaker Change: Particular.
Speaker Change: New development or.
Speaker Change: New treatments that you think are particularly attractive to.
Speaker Change: Youre breaking up pretty dramatic forward into one of the other muscle.
Speaker Change: Neuromuscular indications.
Speaker Change: Just kind of help us out with thinking about the expansion plans. Thanks.
David Akshay: Thanks, very much David Akshay, yes.
Speaker Change: Thanks, David So look I think with a predator mab in a really unique and potent anti myostatin platform revenue incredible opportunity to not just help an SMA across the spectrum of disease that but well beyond back into other neuromuscular disorders, which many of which are very severe and life threatening.
Speaker Change: As you said, we've certainly spoken about and done a lot of good work on at DMT and Sshd modules, two very important muscular dystrophy.
Speaker Change: We've shared those data people are very excited about them and the fact that we validated our pedigree map as.
Having the ability to put on the degree of muscle that it can in such a serious disease SMA, obviously bodes well in these other neuromuscular disorders.
Speaker Change: There are other indications beyond DMD and FSA AOS.
Speaker Change: <unk> is one that we can talk about as well and we've got some thinking there and there'll be more beyond that but I think at the current time, we're very encouraged by the multiple data both from BMD and Sshd, we're working through details of exactly how to.
Begin to study and those disorders that move share more details around that later in the year, but theres no question that in Asia that is.
Speaker Change: As strong as a pet owner map clearly has to be investigating additional indications.
Speaker Change: Thanks.
Gary Nachman: And our next question comes from Gary Nachman with Raymond James Your line is open.
Gary Nachman: Thanks, and good morning.
Speaker Change: So if you get the SMA approval on the <unk>, how quickly will you be able to launch will everything be completely in place.
Speaker Change: Already on our sales and marketing side.
Speaker Change: <unk> with access and patient support programs and how much commercial supply will you have that launch just in terms of meeting the demand.
Speaker Change: And then say if the proof of concept data.
Speaker Change: B City are positive just talk about the likely next steps with 439 after you file the IND.
Speaker Change: What type of Phase one study will you run and how long before it can potentially move into a phase III.
Speaker Change: Yes, Thank you and as Keith noted on the call. He is working with urgency here by mid year to have the team in place for more commentary on like readiness.
Speaker Change: In order to serve patients at approval Keith Yes, Gary. Thanks for the question I mean, I think the first thing that I want to call out is that the team here has already been at work and has already been building preparing for this launch for some time.
Speaker Change: Our fully staffed when it comes to our marketing team and as I mentioned, our market access team.
Speaker Change: We have also.
Speaker Change: We're fully staffed in our leadership team for our patient support programs.
Speaker Change: We will as I mentioned in the prepared remarks, we will have the entire commercial team fully staffed by mid 2025. So we should have a couple of months prior to launch.
Speaker Change: In regard to how soon will we launch after September 22nd date, we're going to be prepared to be out there. The next day I know that the supply chain team is working so that we can have product available to patients as soon as possible and then I think the last thing that you had asked was in regard to supply and I want to assure you that we have ample supply.
Speaker Change: To be able to have a very successful launch.
Speaker Change: Thanks, Keith and regarding like sort of the.
Speaker Change: The Embraer study the proof of concept and I think Gary as you noted.
Speaker Change: How do we think about 439 as Akshay mentioned, we're moving forward.
Speaker Change: With an anticipated IMD for Src <unk> $4 39 in Q3, no matter, what we see this as <unk>.
Speaker Change: Just another indication for us that we are the world leaders in Myostatin biology, and we're very excited to move this into first in human studies.
Speaker Change: Beautiful thing about <unk> 439 is optionality.
Speaker Change: And support our ambition and SMA and other additional rare severe.
Speaker Change: And debilitating neuromuscular disorders or depending upon the.
Speaker Change: Sort of results that we see with embrace.
Depending upon.
Speaker Change: Our thoughts about further study there and how we might approach it $4 39 as I think was previously noted by our team could have some optionality for us in a different space that being cardio metabolic disorders and obesity. So we'll be guided by the data.
Speaker Change: We absolutely will sort of focus on what we see.
Speaker Change: In this first exploratory a readout in the embrace study and then we will provide you all here in the coming month or two further plans for SRP $4 39.
Speaker Change: Alright, great. Thank you.
Speaker Change: And our next question comes from Chris <unk>.
Speaker Change: Deborah Khanda with true with your line is open.
Speaker Change: Good morning team. Thank you so much for taking our question today.
Speaker Change: Given the financial close getting closer to participate just a follow up question from one of the prior analyst would you be able to provide any kind of metrics or guidance. Once the drug is approved.
Speaker Change: Any color on how you see the early demand.
Speaker Change: And not sure how much you expect the recent executive order on Jeff how are you going to have an impact on orphan disease area, but in light of that executive order any comments on how are you.
Speaker Change: Think about pricing in the U S versus ex U S. I know, it's early to give us exact number yet.
Speaker Change: Thank you Justin and good day.
Speaker Change: Two great questions why don't I take sort of the President's Executive order first and then Keith can comment a little bit about launch dynamics.
Speaker Change: And kinetics as you know and I think you've just noted it in your question.
Speaker Change: Aerie early to <unk>.
Speaker Change: Comment on the President's executive order.
Speaker Change: MFN pricing.
Speaker Change: However, it really does not change in any way our plans to commercialize the Edgar Mab not only in the U S, but in Europe Asia Pacific and Latin America.
Speaker Change: We do believe should any elements of the executive order be implemented the implemented bowl.
Speaker Change: It would obviously be better to have not yet established pricing.
Speaker Change: For a therapy versus let's just say products that are already out there with established pricing.
In all countries, we would expect that the price of a political or mab will be reflective of the rarity of SMA the severity of SMA. Despite the use of all.
Speaker Change: Sort of SMN targeted therapies as we noted still a loss of motor function. Despite the use of those overtime and then of course the.
Speaker Change: The strong value proposition that <unk> may be able to provide to patients and families with SMA.
Speaker Change: And then as I also noted a bit earlier.
Speaker Change: Given the rarity of the disease.
Speaker Change: Pilgrim App.
Speaker Change: Would not really impact.
Speaker Change: Tremendously any real budget.
Speaker Change: The magnitude of any single payer or let's just say in the U S or in any country around the world. So we think no matter what happens with the President's executive order for MFN, We think we're going to be in a position of strength.
Speaker Change: We think about the launch of our <unk> globally.
Speaker Change: For the 35000 patients in the world that have received an SMN targeted therapy for a closer look at sort of your first part of your question on launch dynamics and kinetics.
Speaker Change: I'll ask Keith to comment yes, Chris Thank you for the question.
Speaker Change: First of all in regard to guidance, we are not going to be providing guidance. At this time I can give you some aspects that from working in rare disease for now two decades, but one thing that im very excited about the SMA marketplaces.
Speaker Change: 100% of newborn screening in the U S and as we noted in the prepared statements that we know of the two thirds of SMA patients of the 10000 in the U S that have already had an SMA targeted therapy.
Speaker Change: These are good aspects to began in a launch also the fact that we have the centers of excellence with the it's highly concentrated because of the cure SMA model. All of these things give me optimism now thinking about this the centers of excellence are also going to be many at your academic hospitals. So we will be going.
Speaker Change: Through formulary processes, and such and some of them are quite timely theyre not just set based on our launch date, but when the academic centers and other centers are going through it. So we expect that.
Speaker Change: Between that as well as applying for a J code that we would receive some time six plus months later that we will prepare for a consistent and steady launch.
Speaker Change: Great. Thank you so much.
Carla: Thanks Carla.
Speaker Change: The next question comes from Marc Frahm with TV Cowen Your line is open.
Marc Frahm: Hi, Thanks for taking my question, maybe just following up a little bit on <unk>.
Marc Frahm: To the question before launch trajectory.
Speaker Change: As he mentioned the SMA population now has 100% newborn screening in the U S.
Speaker Change: They've gotten very concentrated into these centers of excellence to receive estimate therapy like this.
Speaker Change: Those would seem to be a tailwind to the launch relative to prior SMA launches, but then.
Speaker Change: While there is certainly unmet need maybe it's not quite as dramatic as it was before the original estimate corrector launched.
Speaker Change: Which would maybe be a headwind.
Speaker Change: I think theres no doubt and this we should look at prior SMA launches as a good proxy for the trajectory of that a bit agreement might have and I guess to the extent that you don't think those are good proxies is there another loss of their launches out there in the rare disease space that you think maybe or more analogous to what we should expect with the pit agreement.
Mark: Well, it's a great question Mark I think first of all one of the things that.
Speaker Change: Our team is always focused on as opposed to looking at other sort of proxies or other launches, we just kind of look.
Speaker Change: Look in the mirror and try to compete with ourselves and think about the best way that we can serve patients immediately and then overtime.
Speaker Change: And so youre right Theres, probably some headwinds there is probably some tailwind, but I think a couple.
Speaker Change: Couple of things that I would just underscore.
Speaker Change: <unk> targeted therapies in and of themselves with longer term data. That's been presented absent of scholar rock have shown that there can be a return to the progressive motor.
Speaker Change: <unk> function loss of the disease, Despite what happens earlier when treatment is initiated.
Speaker Change: And then we ourselves showed in the Sapphire study.
Speaker Change: Obviously at the time in which we were.
Speaker Change: Capturing patients and enrolling them in the study there was overall loss of motor function, rather than what we demonstrated with our primary endpoint. The gold standard Hammersmith scale again of motor function. So we do think that Theres a lot of urgency.
Speaker Change: <unk> for patients and I think Keith even commented on how that was underscored at the last months presentation by cure SMA.
Speaker Change: Patients and families were looking for at the same time, we really want to play the long game.
Speaker Change: Independent of sort of launch trajectory, we want to build a team and a model to serve patients immediately and then over time for sort of a steady.
Speaker Change: Assistant growth.
Speaker Change: <unk> of our business by serving patients in a very meaningful way I don't know Keith we'd like to add a little bit more onto that as well Keith.
Speaker Change: You bring up the point of the launch compared to other estimate and targeted therapies. When there was nothing else available. Okay. So they truly had no other treatment. So I think this since the urgency to be able to get to that product was great. As we take a look at the information we find that patients that are one SMN targeted therapies.
Speaker Change: Most of them see their doctor on average two times per year.
Speaker Change: So it's not like September 22nd happens and there have an appointment the next week. So I just want to guide you on that I agree with your statement that the fact that we know where these patients are as it is an advantage. However, there are going to be some headwinds as David mentioned.
Speaker Change: Okay.
Speaker Change: And our next question will come from Evan <unk> with BMO capital. Your line is open.
Speaker Change: Hi, Michael Hoffman on for Abbott and thank you for taking our question.
Speaker Change: Currently we have noted some stronger rebound in <unk> sales to start 2025.
Speaker Change: We're hoping to get your updated thoughts on a long term changes that may occur in the SMA market, specifically with <unk>.
Speaker Change: <unk> being used in conjunction with Sunrise. There are you internally expecting any longer term growth range, but rather that may benefit.
Speaker Change: Our political upon launch.
Speaker Change: Sure.
Speaker Change: No. It's a great question I think.
Speaker Change: One of the things that we would note is that.
Speaker Change: As Akshay shared earlier in the.
Speaker Change: Sapphire study really in a prespecified way, depending upon which SMN targeted therapy.
Speaker Change: Patients were received.
Speaker Change: Recede.
Speaker Change: We feel like we can help all patients independent of whether or not they receive spin rozzer or risk of plant.
Speaker Change: We will continue to sort of assess the nearly $5 billion annual market across the three different SMN targeted therapies, whether or not it's the highly innovative gene therapies xeljanz.
Speaker Change: Or the other highly innovative.
Speaker Change: <unk> targeted therapies like spin rather than risk to plan and we would just expect to follow those dynamics, but recognized with the Sapphire data and then as Akshay noted earlier on today's call.
Speaker Change: Upcoming.
Under two study the <unk> study, where we would expect more experience for our pedigree or mab for patients who have received Soc. Gen. Smart, we think we're going to be in a position of strength to really help and provide potential transformational benefits for all patients that have received SMN targeted therapies over time.
Speaker Change: Thank you for your question.
Speaker Change: I appreciate it thanks.
Speaker Change: Our next question comes from Andres Mounter Nando with H C. Wainwright Your line is open.
Speaker Change: Hi, everyone. Thanks for taking my question just a quick one from me.
Speaker Change: Progress towards U S launch in your ongoing readiness in Europe, I guess, how long the Sally meeting with potential to secure a commercial partner for Europe, and then as context, obviously, you want to fire on all cylinders across the pipeline, but how would you prioritize the additional indications for <unk>.
Speaker Change: In this situation.
Speaker Change: Yes. Thank you I think we noted this just a few weeks ago on our April 28 call.
Speaker Change: Given the experience that.
Speaker Change: This collective team has had between Alexia on our myeloma genetics.
Speaker Change: We feel like there is nobody better suited to serve patients globally than us directly.
Speaker Change: So it is not a priority of ours to think about a partner outside of the U S. We feel like this is something that we know well and in fact do well and we will continue to run our playbook that not only do we know the playbook, we feel like we wrote that playbook.
Speaker Change: As it relates to other indications as Akshay noted will be data driven the team's been doing some wonderful work.
Speaker Change: And looking pre clinically at additional rare.
Speaker Change: Severe and debilitating neuromuscular diseases, and Akshay will be providing further guidance on that.
Speaker Change: Towards the end of this year and into next thank you.
Dennis Kennedy: And the next question comes from Dennis Kennedy Lifesize Capital Your line is open.
Dennis Kennedy: Hey, Thanks for taking the question.
Speaker Change: As we look towards the <unk> date in June could you just help frame expectations for the <unk> monotherapy arm.
Speaker Change: Specifically, how much of the lean mass loss that we expected one year with <unk> typically occurs by week 24.
Speaker Change: Weighted to that do you think eight weeks of follow up post treatment is sufficient time to demonstrate a weight regain inpatient and the tears appetite monotherapy arm.
Speaker Change: Yes, Dennis these are great questions and in fact, I think within your questions you.
Speaker Change: So to highlight some of the limitations for us.
Speaker Change: And sort of <unk>.
24 week endpoint and then eight weeks following but nonetheless, as Akshay and I have noted.
Speaker Change: We feel like it's important to understand for a first time, if there is a role that we can play akshay.
Speaker Change: So Dennis I mean.
Speaker Change: <unk> consistently insured loss of lean mass.
Speaker Change: We won that massive symmetrical to the overall loss in body masks and so you can expect that at week 24.
Speaker Change: About 25 to.
Speaker Change: Steady ascent of the weight loss that will have occurred in that zip tie that loan off will be due to loss of muscle.
Speaker Change: We're projecting that based on historical basis, there's no reason to believe that it would be any different in the context of this study.
Speaker Change: Now will that be the maximum amount of lean mass loss at week 24, I don't think so because in.
Speaker Change: The pivotal studies <unk> studies, so much long debt.
Speaker Change: And overall the impact from muscle loss of muscle mass is even greater obviously.
Speaker Change: <unk> patients lose weight.
Speaker Change: But within the context of embrace.
Speaker Change: Out of weight loss at week 20.
Speaker Change: Four we lean mass loss, we expect your other point about eight weeks of follow up sufficient well look at this stuff.
Speaker Change: <unk> was an exploratory study to understand the potential of competitive mab in a pure clean safe potent anti myostatin approach in the context of obesity that doesn't think follow up answer the whole question, but you can anticipate that there'll be some way regain during that period and the trajectory of that would be quite important to monitor.
Speaker Change: <unk> the two arms.
Speaker Change: Great. Thank you.
Speaker Change: Thanks, Jeff.
Speaker Change: At this time I am showing no further questions in the queue.
Speaker Change: Thank you operator.
Speaker Change: Okay.
Speaker Change: Thanks for everybody joining the call today, and we'll look forward to.
Speaker Change: Continuing to update you and keeping you apprised of our progress as an organization. Thank you.
Speaker Change: This concludes today's conference call. Thank you for participating you may now disconnect.
Speaker Change: Okay.
Speaker Change: [music].
Speaker Change: Yes.
Speaker Change: [music].