Q3 2024 Scholar Rock Holding Corp Earnings Call
Yeah.
Speaker Change: Good morning, and welcome to the school of rocks third quarter financial results and business update call. All participants will be in listen only mode. After the company's prepared remarks co participants will have an opportunity to ask questions.
Speaker Change: To ask a question you May Press Star then one one on your Touchtone phone to withdraw your question. Please press Star one one again. Please note. This event is being recorded.
Speaker Change: Before we begin I'd like to point out that we will be making various statements about scholar rock's expectations plans and prospects that constitute forward looking statements for the purposes of the Safe Harbor provisions under private Securities Litigation Reform Act of 1995.
Speaker Change: 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 sections of our website to find our most up to date F E E statements and filings.
Speaker Change: A recording of today's event will also be available on our website should you wanted to rewatch at a later date.
Speaker Change: I'd now like to turn the conference over to Jay Backstrom precedent and C. E O of scholar Rock Jay. Please go ahead.
Jay Backstrom: Thank you Carmen good morning, and welcome everyone. Thank you for joining our third quarter business update it's a very exciting time at scholar rock with the success of Sapphire or phase III registration study in spinal muscular atrophy and a successful financing we have great momentum heading to the end of 2024.
Jay Backstrom: I'm joined on today's call by Ted Myles, Our Chief operating Officer, and Chief Financial Officer.
Jay Backstrom: For our call. This morning, I will start with a company overview, Ted will provide a financial and business update and I'll provide a few closing remarks before opening the call up for questions.
Jay Backstrom: As shown on slide six we had another very successful quarter building on the momentum that we created throughout 2020 for the dedication and commitment across the organization has been remarkable the teams continue to execute and deliver all of our key milestones on time or ahead of schedule for.
Jay Backstrom: Our lead program with the pedigree map in spinal muscular atrophy, a clinical team did an outstanding job delivering the sapphire data with great skill speed and high quality, enabling us to report out the successful results in early October this flawless execution allows us to advance towards the next important milestones.
Submitting the BLA and MAA in Q1 of 2025 and keeps us on track to have our first commercial launch in the U S. In Q4, 2025 with Europe to follow assuming regulatory approvals and.
Jay Backstrom: In addition for our embrace phase II proof of concept with a bitter mab. We completed enrollment ahead of schedule and positioning us to report out top line results earlier than planned now targeting Q2 of 2025.
Jay Backstrom: Similarly, the research team continues to deliver a steady cadence of informative non clinical data with <unk> hundred 39, our novel highly selective anti Myostatin program in obesity and cardio metabolic disorders.
Jay Backstrom: In addition to our focused execution, we expanded our management team with the addition of best Shafer Beth is an industry veteran who joined in September as Chief business Officer to help guide our investment decisions and partnering opportunities to enable us to take full advantage of our validated platform that has produced a robust pipe.
Jay Backstrom: Line of high value potential products.
Jay Backstrom: Turning to slide seven before I provide more detail of our third quarter results I want to start with a reminder, as to why we are here. Our purpose is to create new possibilities for those living with SMA like Liza who made it clear that they want more.
Jay Backstrom: More means gaining muscle strength and function in order to maintain independence for basic daily activities, such as feeding yourself, turning in bed or operating a motorized wheelchair activities that can be maintained or achieved with the 1% to two point improvement on the Hammersmith functional motor scale, a scale designed specifically for SMA.
Jay Backstrom: When asked what she was seeking.
Speaker Change: <unk> made it clear muscle muscle is everything.
Speaker Change: Now to slide eight as we announced in October our pivotal phase III Sapphire study met the primary endpoint with a one eight point improvement of our <unk> plus standard of care compared to placebo plus standard of care as measured by the gold standard SMA specific Hammersmith functional motor scale at week 52, that's clinically meaningful.
Speaker Change: <unk> was statistically significant with a P value of 0.0 192.
Speaker Change: Patients receiving a peterka mab demonstrated early and increasing motor function improvement versus placebo as measured by the Hammersmith scale with the <unk> patients gaining function, while those receiving placebo lost function despite being on standard of care.
Speaker Change: Importantly, <unk> demonstrated consistency of effect across doses and age groups of 2% to 21 and abroad. SMA population further a critical map show transformative clinical activity with 30% of patients who were already receiving standard of care, achieving an additional three point or greater improvement in their hand.
Speaker Change: And scores.
Speaker Change: With respect to safety and Tolerability Sapphire confirmed that <unk> favorable safety profile, which was consistent with the established safety profile seen in over four years of treatment of SMA based on our phase II Topaz study.
Speaker Change: The observed safety profile is consistent with <unk> highly selective approach to blocking milestone.
Speaker Change: We are thrilled with these results and what it means for the SMA community patients their families caregivers and physicians. We believe these data collectively show that <unk> has the potential to become part of a new standard of care in SMA.
Speaker Change: Turning to slide nine to put our results into context. It is helpful to understand the normal trajectory for those receiving standard of care treatment.
Speaker Change: Despite the availability of three approved therapies targeting the SMN protein individuals' lives are still at risk of losing function over time, given the inherent progressive nature of SMA. This was illustrated at the cure SMA meeting in June of this year.
Speaker Change: The graph represents the Hammersmith motor function scores over time for the NUCYNTA arison treated patients from cherish Shine study with the Orange line, representing the trajectory of those randomized to <unk> referred to as early dose group.
Speaker Change: As shown the motor function improved for the first two years after starting treatment with new centers.
Followed by a plateau, where there is no further improvement in motor function. After four years of treatment. However, there is a progressive loss of motor function of approximately one point per year. Despite the continued treatment with this standard of care therapy.
Speaker Change: The purple shaded box represents the time period in terms of duration of this nursing treatment that is similar to the median treatment duration of this and nursing for those enrolled in Sapphire, a time period, where the sapphire patients. We're clearly on the declining phase of their treatment journey, while on standard of care.
Speaker Change: Now to take a look at how the critical map effects motor function over time slide 10 displays the change from baseline in Hammersmith scores by visit these line graphs beautifully articulate the treatment benefit of <unk> over the course of the treatment period.
Speaker Change: As you can see the Hammersmith scores improved in patients on <unk> as early as eight weeks. The first post baseline assessment by contrast, the scores decreased in those on placebo with a change from baseline of minus 1.2 points similar to the long term data on <unk> that indicated a loss of about one point per year.
After four years of treatment.
Speaker Change: Theres early separation between <unk> and placebo the difference widens by the end of the treatment period underscoring the effects of <unk> on the disease course from losing function despite being on standard of care to gaining function by adding a pit of the map.
The strength of the results as illustrated by the Forest plot for all of the pre specified analysis shown on slide 11.
Speaker Change: As shown there is consistency across analyses with all showing improvement favoring a peterka amount, including across doses and across age groups. These analysis speak to the strength and the robustness of our results.
Speaker Change: Moving to slide 12.
Speaker Change: To further underscore our functional improvement, we see 30% of sapphire patients on <unk>, achieving a three point improvement versus 12, 5% for those on placebo. This transformative magnitude of improvement is extraordinary on top of standard of care.
Speaker Change: Again, the strength and consistency of the results demonstrate the ability of the pit or map to alter the course of disease from losing function to gaining function with the potential for profound impact on the lives of those living with SMA.
Speaker Change: We believe that <unk> is suitable for chronic treatment for a broad SMA population based on the efficacy seen with sapphire with improvements seen across all age groups two to 'twenty, one the well tolerated safety profile with safety supported by more than four years of experience in SMA and we are working with urgency to finalize our rare.
Speaker Change: <unk> applications and submit the BLA and MAA in the first quarter of 2025.
Now moving to our cardio metabolic program on slide 14 the.
Speaker Change: The recent approvals and rapid adoption of <unk> and <unk> appetite has had very positive impact on those living with us with obesity.
Speaker Change: A key issue that has emerged as the significant loss of lean muscle mass associated with these highly effective treatments.
Speaker Change: Given the important role muscle plays in energy metabolism in glucose homeostasis, maintaining appropriate levels of lean muscle is essential to healthy living.
Speaker Change: We believe our approach to preserving lean muscle mass with our highly selective novel anti Myostatin MSR K 43, nine when used with the <unk> one receptor agonist can preserve lean muscle mass and promote promote healthy weight management we.
Speaker Change: We see <unk> 439, as part of the next wave of innovation in the treatment of obesity and are working to submit the IND targeted for mid year 2025.
Speaker Change: Now to slide 15.
Speaker Change: We designed a comprehensive non clinical program with <unk> three nine and to date, we've demonstrated strong scientific rationale and promising non clinical evidence, including demonstrating preservation of lean mass during <unk>, one receptor agonist induced weight loss.
Improvement in fasting glucose beyond <unk>, one receptor agonist alone increase in lean mass and attenuation of fat mass regain following <unk> receptor agonists withdrawal greater potency compared to an anti <unk> two antibody and most recently an increase in lean mass and lowered fat mass gain following treatment with metformin.
Speaker Change: <unk> <unk> hundred 39.
Speaker Change: Turning to slide 16, we entered into the area of obesity as we believe we have an elegant solution to preserving lean muscle mass with the potential attractive risk benefit profile for long term healthy weight management, we have the right target.
Speaker Change: Inhibition of Myostatin, a negative regulator of muscle is known to promote muscle growth and function. We are a validation of our approach given the results of sapphire demonstrating improvement in motor function and further preserving lean mass has the potential to improve durability of weight loss.
Speaker Change: And our highly selective approach and targeting the pre in late in form of Myostatin minimize unwanted toxicities and supports the potential for a favorable benefit risk profile.
Speaker Change: I will now only by Ted to provide a financial and business update Ted. Thanks, Jay It's been a very busy few months and I'm excited to provide some additional insight into areas of focus recently and in the months to come.
Ted Myles: Turning to slide 18 based on the positive Sapphire data that we disclosed in October we completed an upsized follow on offering of $345 million on a pro forma basis. This puts our September 30 cash balance at approximately $463 million, which enables us to scale up and focus on driving key priorities fall.
Speaker Change: Namely expanding our anti Myostatin platform and preparing for the commercialization of a pedigree map if approved.
Speaker Change: The success of Sapphire Sapphire trial Derisked, the Pilgrim AD program and provides an opportunity to expand the number of SMA patients that a pedigree mab may help as we have disclosed previously we are looking forward to initiating the <unk> clinical trial in mid 2025 to explore a pedigree mab in combination with standard of care in patients under two.
Speaker Change: Two years of age.
Speaker Change: Another key priority for our anti Myostatin platform is advancing our novel selective anti Myostatin antibody <unk> hundred nine we expect to file our IND for <unk> hundred 39 next year and we look forward to reporting the results of our embrace trial in the second quarter of 2025.
Speaker Change: Embraces a proof of concept study have a pedigree mab in a patient population living with obesity testing the hypothesis of a selective anti myostatin as an important therapeutic approach to healthy weight management.
Speaker Change: And of course, we are fully engaged in commercial launch preparations are a pillar grab in SMA.
Speaker Change: As we've disclosed previously before we had positive data the company was being very thoughtful about planning the work that needed to occur while managing resources carefully.
Speaker Change: Not to invest too much too early.
Speaker Change: With the positive data and the Upsized raise we are now actioning those plans focused on the following key initiatives.
Speaker Change: The first is continued engagement of critical stakeholders, such as <unk> and the patient community.
Speaker Change: The second is ensuring an optimal treatment experience for those with SMA.
Speaker Change: And the third is building a world class commercial team to deliver both the U S and European launch.
Speaker Change: Turning to slide 19, I will provide a bit more insight into our approach to market over the near and intermediate term.
Speaker Change: We have strategically invested in foundational activities over the past several years.
Speaker Change: Among the most important of these investments we've been building strong relationships with HCP patient and advocacy communities and payers in both the U S and Europe.
Speaker Change: We continue to partner listen and learn from these very important stakeholders.
Speaker Change: Our MSL team has been engaging with health care providers at key SMA treatment sites, including cure SMA and MDA centers.
Speaker Change: We also began hiring our payer accounts team to engage and educate national regional and government payers.
Speaker Change: This past June we launched life takes muscle the first muscle muscle focused disease education campaign in SMA, which reflects what we have heard from the community that more is needed beyond current standard of care to treat SMA.
Speaker Change: There has been positive reaction to the campaign from patients caregivers and treaters with engagement on social media channels.
Speaker Change: In 2025, as we I successful launch of a pedigree mab, we're committed to ensuring an excellent and customized treatment experience for patients we aim to serve.
Speaker Change: This also includes working with the channel and hub partners to deliver excellent patient services support and providing the option for monthly home infusion of a pedigree map.
Speaker Change: Finally, we look to complete the build of our field team, we believe that with a total of fewer than 50 full time employees, we can reach and support the U S market in a capital efficient and highly effective manner.
Speaker Change: As we look to 2026, it's with full awareness that SMA is a global disease and our goal is to make <unk> available to as many SMA patients as possible if.
Speaker Change: If approved we plan to commercialize in selected European countries and will partner with distributors and strategic partners to reach patients outside of the U S and Europe.
Speaker Change: Turning to slide 20, we see a potential path to blockbuster status for <unk> in SMA.
A few key market dynamics give us high confidence the scholar Rock's first commercial launch is positioned for success.
Speaker Change: The right market, the right medicine, and the right approach.
Speaker Change: The current treatment landscape for SMA is $4 5 billion and growing.
Speaker Change: While these treatments address motor neuron component of SMA, the muscle component of the disease is yet to be addressed.
Speaker Change: We're committed to developing a paragraph for as many SMA patients as possible who may benefit from this cutting edge therapy.
Speaker Change: Importantly, we looked at our clinical success to demonstrate that our pedigree Rab is safe and effective and is addressing an important unmet medical need for patients with SMA.
Speaker Change: We know where the patients where they SMA patients are and we know many of the physicians who are treating these patients.
Speaker Change: While there is still a great deal of work to be done and we as we continue to educate the broader community. There is a high degree of awareness that muscle targeted therapy is the next important frontier for patients living with SMA.
Speaker Change: Based on these dynamics, we believe a pedigree mab has a total revenue potential of greater than $1 billion.
And that assumes a competitive muscle targeted therapies market in SMA.
Speaker Change: Turning to slide 21, there is no doubt the scholar rock is firing on all cylinders. We have a very exciting 2025 ahead of us and we're running with a sense of urgency and excitement as we strive for continued operational excellence.
Speaker Change: There are multiple upcoming milestones and value inflection points for scholar rock our team remains on track to complete the regulatory submissions in the first quarter of 2025.
Speaker Change: We're expanding our myostatin clinical efforts as we look to the under two SMA population as well as those living with obesity and we are driving forward to transitioning scholar rock into a commercial company with a planned launch of a pedigree mab in SMA.
Jay Backstrom: Now I'll pass back to Jay for some concluding comments before we open the line for questions Jay Thank you Ted.
Jay Backstrom: Moving on to slide 22, with the success of Sapphire, we are getting closer to fulfilling our mission of creating new possibilities for people living with serious diseases, starting with SMA.
Jay Backstrom: Sapphire is a validation of our industry, leading scientific platform and of our selective myostatin inhibition programs and further reinforces our confidence in our approach to healthy weight management and obesity.
Jay Backstrom: In closing, we believe <unk> has the potential to transform the standard of care in SMA.
Sapphire met its primary endpoint with a one eight point improvement for our <unk> plus standard of care compared to placebo plus standard of care as measured by the gold standard SMA specific Hammersmith functional motor scale. This improvement is both clinically meaningful and statistically significant with a picker map patients showing a gain in their functional scores.
Jay Backstrom: Compared to a decline in scores for those randomized to placebo. Despite receiving standard of care. We believe that <unk> is suitable for chronic treatment for a broad SMA population based on the efficacy data from Sapphire with motor function improvement seen across all age groups <unk> through 'twenty, one and the well tolerated safety profile with the safety data.
Jay Backstrom: From Sapphire further supported by more than four years of experience in SMA as Ted highlighted we are working urgently to bring a picker, Matt as soon as possible to those living with SMA and are finalizing our regulatory applications and preparing for commercial launch assuming approval the.
Jay Backstrom: With success and set in the SMA will serve as a catalyst for us to become a fully integrated commercial biotech company as well as becoming the foundation for our neuromuscular franchise with multibillion dollar potential clearly a very exciting time at scholar rock that.
Speaker Change: That concludes our prepared remarks, we will now open the call up for questions.
Speaker Change: Thank you so much and as a reminder to ask a question. Please press star one one on your telephone and wait for your name to be announced.
Speaker Change: To withdraw yourself press Star one again, one moment for our first question.
Speaker Change: He comes from the line of generally with Jefferies. Please proceed.
Speaker Change: Hi, Good morning, Thanks for taking our question. This is generally on the line from Michael E.
Speaker Change: So we know that Lilly recently started a new phase II could you just talk about the differences in trial design does the method of administration in patient population and question on how do you put that into context against your <unk>.
Speaker Change: And then secondly could you also walk us through the different scenario of how your obesity data readout next year and what are the key parameters that you are looking for that could define good data. Thank you.
Jay Backstrom: Yes. Thank you. Good morning. So this is Jay so with respect to the Lilly trial and are embraced study and we'll start with our embrace trial are embraced study as designed.
Jay Backstrom: To assess our ability to target selectively anti myostatin with the pit or mab with the primary endpoint of lean muscle mass to validate our thinking around our approach to preserving lean muscle and has a 24 week end point with lean mass as its primary endpoint and we have additional.
Jay Backstrom: Safety as well as exploratory endpoints. So it's really a proof of concept to guide our thinking around <unk> 39.
Jay Backstrom: We are using a peterka, Matt because we can <unk> four through nine will be a sub Q presentation. That's the reason, where we're focused on it going into clinic.
Jay Backstrom: Lilly trial is looking at are still a number of doses exploring their program against that there's appetite. Our program is looking at there is appetite as well as we included that in our study. So we're kind of moving forward I think we're in a very good place with S. R. K fourth run 43 nine to open up our IND.
Jay Backstrom: Mid mid year next year and that will position us to be able to continue to run forward and assess this more formally in clinic.
Jay Backstrom: With respect to what we want to see from our embraced trial, we're looking to assess our ability to maintain and preserve lean muscle mass as we've shared throughout all of our non clinical experiments that preservation is really clinically meaningful it enhances the ability to.
Jay Backstrom: To manage weight loss Durably. It has an effect on reducing fat mass. It has an effect on glucose metabolism. So a lot of a lot of interest a lot of interesting ideas on the exploratory endpoints that will carry into the clinic with <unk> 439.
Jay Backstrom: Okay.
Speaker Change: Got it thank you so much.
Jay Backstrom: Okay Yeah.
One moment for our next question please.
Jay Backstrom: And it is from the line of et cetera.
Speaker Change: BMO capital markets. Please proceed.
Speaker Change: Great. Thanks for taking the questions a couple ones first.
Speaker Change: First on the <unk> study just was wondering given sort of what you've learned with the political mab.
Speaker Change: From Sapphire, how are you thinking about sort of dos.
Speaker Change: That.
Speaker Change: In combination.
Speaker Change: And Nielsen study and then also maybe on the obesity, just how you're thinking about the differences in some of the combination approaches.
Speaker Change: The profiles of miles that inhibition rich with respect to something like the <unk> or are there sort of muscle preservation approaches that are being explored in a year.
Speaker Change: Anything that you've kind of seen in terms of sort of differences or similarities in these different approaches and muscle preservation.
Speaker Change: Just broadly thank you.
Speaker Change: Okay. Thank you for the questions so let's start with Opal.
Speaker Change: It's a study under two and of course, we're authorized to study two and above I'm very keen to get that trial open with respect to dosing, where it's going to take full advantage of all of the PK PD data that we have not only from the Topaz study, but also now with the Sapphire data and really do modeling to kind of assess the doses. Our team is working on that now.
Speaker Change: I've mentioned previously we've been authorized to proceed with that study, we've socialized that both with the FDA and the European authorities.
Speaker Change: Waiting to get the final clinical data at the PK PD data, which will help us understand because its milligram per kilogram dosing it'll really be based on our <unk>.
Speaker Change: Assessment that 10 milligram per kilogram is going to be the effective dose, but it'll be a combination of assessing all of the data together to guide the dosing and that under two patient population.
Speaker Change: And then with respect to the emerging therapies in the weight loss field.
Speaker Change: What we have today are the leaders with <unk> appetite and some magnetite and obviously theres a lot of innovation following.
Speaker Change: As we mentioned at our New York event, and you heard from Dr. <unk>.
Speaker Change: <unk> at Yale lean muscle loss happens almost regardless of the means of weight loss, including bariatric surgery. For example, so as we think about these other therapies going forward, it's still going to be a component of lean mass loss still critical to maintain that lean mass loss and frankly, our strategy with targeting <unk> hundred 39 will be.
Speaker Change: Similar to what we thought about in SMA, we will be agnostic to the treatment therapy, because we believe that our safety profile and target lends itself to combination strategies. So as those things get closer to clinic will have an opportunity to further assess it but for now I think our program is still built around combination with <unk>.
Speaker Change: Yeah.
Speaker Change: Great. Thank you.
Speaker Change: Okay.
Speaker Change: Okay.
Speaker Change: Thank you and as a reminder that is star one line. If you do have a question.
Speaker Change: Our next question is from Gary Nachman with Raymond James. Please proceed.
Speaker Change: Good morning, and congrats on all the progress so as you're preparing the filing in SMA will you be seeking approval for both doses 10, 20, Meg per kg or just a lower dose and do you think FDA and EU will accrue for the entire age range, 2% to 12 and also the exploratory <unk> to 'twenty.
Speaker Change: And it sounds like you're pretty confident in that.
Speaker Change: I guess are you, having a pre NDA meeting with the FDA specifically to ensure that you are aligned on that front.
Speaker Change: And then just one more.
Speaker Change: How have I know you just set up the payer team, but how have initial payer discussions been going for a pedigree of SMA how are they viewing the sapphire data and any updates on I guess, how you're thinking about pricing.
Speaker Change: Some range, maybe you could give us that's factored into that greater than $1 billion opportunity.
Yeah, Hey, Gary Good morning, So maybe kind of starting to see if I got your questions and kind of rank order.
So first of all with respect to dose as we mentioned when we released the data.
Speaker Change: 10, and 20 performed interchangeably and so as a result of that we're going to go forward with the 10 milligram dose do not expect that we will have two doses in the label given the consistency of the results and FDA is thinking around lowest doses best dose. So we feel very very good about that and so we're going to go forward with the 10 milligram dose with respect to the label.
Speaker Change: In relationship to age.
Speaker Change: As we anticipated the data coming from Sapphire before we saw it in our thinking was it if the point estimate of the effect size was similar between the 13 and 21 year old group. We're in very very good shape to demonstrate that this should be used independent of age and as you saw with our staff. Our data was spot on exact point estimate so FDA views this as a single dose.
There's no reason to believe that it will work and some on 12 not 13. So we're going into this feeling confident that we have deployed age range in there given the consistency of data so feel good about that.
Speaker Change: Of course more to come but frankly from FDA labeling approach they really don't define it by age short of gene therapy, which is approved under two so feel very good about the age and the breadth of that in our label.
Speaker Change: <unk> had really good interactions already from the regulatory front, we've already engaged with.
Speaker Change: We've already engaged with a pre submission meeting with the European agency given the fact, we have prime designation. So we're all teed up for the regulatory applications, we've already engaged FTA and helping understand how they'd like to see our data presented so feel very good about that and yes. It is good corporate practice to have a pre BLA meeting.
Speaker Change: With the FDA just to be sure. There is nothing else that they consider but we feel very good about where we are and so we're off and running on that.
Speaker Change: With respect to the payer interaction maybe I'll just make a comment on kind of hand over to Ted to add some additional color as you can imagine it's early days with the Sapphire data we've already done some preliminary look in interaction with the Payors what I can tell you as I think about our data to just look at the data that we presented so far gaining function instead of losing function, helping preserve in Maine.
Ted Myles: Pain function the value on top of the additional therapy I think they have a really very good value proposition and the potential to really make a significant impact on those living with SMA and so feel that that'll be recognized by the payers as we go forward, but more to come on the specifics.
Speaker Change: Thanks, Jay and <unk>.
Speaker Change: Gary as you May expect it's a bit early for us to.
Speaker Change: Unveil our pricing assumptions and provide guidance in that regard.
Speaker Change: What I can comment on further to Jay's comments.
Speaker Change: The payer discussions have been going on payer work has been going on since even before we had sapphire data sapphire data exceeded our expectations. So that only helps helps our case.
What I can point to as current market dynamics and what we're seeing in the market right. Now is that patients for example would get on gene therapy and in some cases.
Speaker Change: Ultimately end up on the SMN character and so.
Speaker Change: <unk> for $2 million gene therapy, followed by an expensive SMN collector and in some cases patients will switch from one SMN correct or to another so what we're seeing in the market is that payers are supporting these patients.
Speaker Change: The patient advocacy group is very well organized and very impactful.
To help patients in.
Speaker Change: In their treatment paradigm, they're very motivated as we talked about earlier to not only maintain function that they have but gain more and so we think the wins at our back and we're going to continue to do our work and we'll continue to update the market as we learn more.
Speaker Change: Okay, Great just one quick follow up.
Speaker Change: Are you going to hire the 50 or so reps prior to approval and have them ready to go or are you going to wait till you actually get the approval just in terms of how we're modeling that thanks.
Speaker Change: So I think it's safe to assume that will continue to be very financially prudent and capital efficient to make sure that we're running through the start line, but we're also not going to disadvantage ourselves when we launch so as we think about the way we balanced bill.
Speaker Change: Building, a scaffolding and being ready to get ready in advance of data that we released last month and now we're executing on the plans that we have thoughtfully developed prior to having data.
Speaker Change: Pulling the same playbook as we get ready for commercial launch so that fine balance of making sure that we're deploying the capital the right amount of capital at the right time.
Okay, great. Thank you.
Speaker Change: Thank you our.
Speaker Change: Our next question is from the line of <unk> <unk> with <unk> Securities. Please proceed.
Speaker Change: Hi, This is Alex on August one for quite a while.
Speaker Change: And building on Gary's question labeling a little bit given that our clinical map shows benefit of course, all the patient populations.
Speaker Change: And how acclimate is viewed with the FDA what are the chances that you might get a broader label that also includes irrespective of ambulatory status and are there any differences in how the FDA or the ECS you estimate that would.
Speaker Change: Alternative interpretation and ultimately labeling decisions.
Speaker Change: Yes. Good question. So if you take a look at his current FDA approach to labeling if you look at the new <unk> and rest of the plan labels FDA tends to see SMA as a monogenic disease, a single disease, a kind of a continuum and so theyre labeling approach is broad it's for the treatment of pediatric and adult.
Speaker Change: Patients with SMA not confined by type by ambulatory status could rather for the treatment of SMA as a continuum and we anticipate that they will follow the same labeling convention for our program and of course more to come on that but no reason to believe that they would do differently. So that's what we're anticipating.
Speaker Change: With respect to that.
Speaker Change: Europe tends to follow a similar approach they may depending there is a little bit of a different thinking around sometimes what does get entered it doesn't get in the label, but I can tell you from our pre submission interaction. So far we feel like they are seeing the similarly, they see it as a single disease, we have prime designation, which underscores they recognize the value of it.
Speaker Change: Potential of this going forward, so again more to come as we get closer down the road labeling negotiation tends to be towards the end of the approval process, but going into it given the strength of our data we feel very good about our position going forward and the potential for a broad label.
Speaker Change: Thanks, and congrats on the progress.
Speaker Change: Thank you.
Speaker Change: Thank you and I see no further questions in the queue I will conclude our Q&A session and program for today. Thank you all who participated and you may now disconnect.
Speaker Change: Yes, thank you for joining bye.
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Speaker Change: Good morning, and welcome to scholar Rock's third quarter financial results and business update call.
Participants will be in listen only mode. After the company's prepared remarks co participants will have an opportunity to ask questions.
Speaker Change: To ask a question you May Press Star then one one on your Touchtone phone to withdraw your question. Please press Star one again. Please note. This event is being recorded.
Speaker Change: Before we begin I'd like to point out that we will be making various statements about scholar rock's expectations plans and prospects that constitute forward looking statements for the purposes of the Safe Harbor provisions under private Securities Litigation Reform Act of 1995.
Speaker Change: Any forward looking statements represent our views only as of today and should.
Speaker Change: Not be relied upon as representing our views as of any future date.
Speaker Change: I encourage you to go to the investors and media sections of our website to find our most up to date FCC statements and filings.
Speaker Change: According of today's event will also be available on our website. She you wanted to Rewatch at a later date.
Speaker Change: I'd now like to turn the conference over to Jay Backstrom, President and CEO of scholar Rock Jay. Please go ahead.
Jay Backstrom: Thank you Carmen good morning, and welcome everyone. Thank you for joining our third quarter business update it's a very exciting time at scholar rock with the success of Sapphire or phase III registration study in spinal muscular atrophy and a successful financing we have great momentum heading to the end of 2024.
Jay Backstrom: I'm joined on today's call by Ted Myles, Our Chief operating Officer, and Chief Financial Officer.
Jay Backstrom: For our call. This morning, I will start with a company overview, Ted will provide a financial and business update and I'll provide a few closing remarks before opening the call up for questions.
Jay Backstrom: As shown on slide six we had another very successful quarter building on the momentum that we created throughout 2020 for the dedication and commitment across the organization has been remarkable the teams continued to execute and deliver all of our key milestones on time or ahead of schedule.
Jay Backstrom: For our lead program with <unk> in spinal muscular atrophy, the clinical team did an outstanding job delivering the sapphire data with great skill speed and high quality, enabling us to report out the successful results in early October this flawless execution allows us to advance toward the next important milestones.
Jay Backstrom: Of submitting the BLA and MAA in Q1 of 2025 and keeps us on track to have our first commercial launch in the U S. In Q4, 2025 with Europe to follow assuming regulatory approvals.
Jay Backstrom: In addition for our embrace phase II proof of concept with the Peterka Mab, we completed enrollment ahead of schedule and positioning us to report out top line results earlier than planned now targeting Q2 of 2025.
Jay Backstrom: Similarly, the research team continues to deliver a steady cadence of informative non clinical data with SRP 4009, our novel highly selective anti Myostatin program in obesity and cardio metabolic disorders.
Jay Backstrom: In addition to our focused execution, we expanded our management team with the addition of best Shaper. Beth is an industry veteran who joined in September as Chief business Officer to help guide our investment decisions and partnering opportunities to enable us to take full advantage of our validated platform that has produced a robust pipe.
Jay Backstrom: Line of high value potential products.
Jay Backstrom: Turning to slide seven before I provide more detail of our third quarter results I want to start with a reminder, as to why we are here. Our purpose is to create new possibilities for those living with SMA like Liza who made it clear that they want more more.
Jay Backstrom: More means gaining muscle strength and function in order to maintain independence for basic daily activities, such as feeding yourself, turning in bed or operating a motorized wheelchair activities that can be maintained or achieved with a 1% to two point improvement on the Hammersmith functional motor scale, a scale designed specifically for SMA.
Speaker Change: When asked what she was seeking <unk> clear muscle muscle is everything.
Speaker Change: Now to slide eight as we announced in October our pivotal phase III Sapphire study met the primary endpoint with a one eight point improvement of our <unk> plus standard of care compared to placebo plus standard of care as measured by the gold standard SMA specific Hammersmith functional motor scale at week 52, this clinically mean.
Speaker Change: The full benefit was statistically significant with a P value of 0.0 192 patients receiving a peterka mab demonstrated early and increasing motor function improvement versus placebo as measured by the Hammersmith scale with the <unk> patients gaining function, while those receiving placebo lost function despite being on.
Standard of care.
Speaker Change: Importantly, <unk> demonstrated consistency of effect across doses and age groups of 2% to 21 and abroad. SMA population further <unk> show transformative clinical activity with 30% of patients who were already receiving standard of care, achieving an additional three point or greater improvement in their hand.
Speaker Change: <unk> scores.
Speaker Change: With respect to safety and Tolerability Sapphire confirmed that <unk> favorable safety profile, which was consistent with the established safety profile seen in over four years of treatment of SMA based on our phase II Topaz study.
Speaker Change: The observed safety profile is consistent with the <unk> highly selective approach to blocking milestone.
Speaker Change: We are thrilled with these results and what it means for the SMA community patients their families caregivers and physicians. We believe these data collectively show that <unk> has the potential to become part of a new standard of care in SMA.
Speaker Change: Turning to slide nine to put our results into context. It is helpful to understand the normal trajectory for those receiving standard of care treatment.
Speaker Change: Despite the availability of three approved therapies targeting the SMN protein individuals' like lives are still at risk of losing function over time, given the inherent progressive nature of SMA. This was illustrated at the cure SMA meeting in June of this year.
Speaker Change: The graph represents the Hammersmith motor function scores over time for the NUCYNTA arison treated patients from cherish Shine study with the Orange line, representing the trajectory of those randomized to NUCYNTA arson referred to as early dose group.
Speaker Change: As shown the motor function improved for the first two years after starting treatment with new centers and followed by a plateau, where theres no further improvement in motor function. After four years of treatment. However, there is a progressive loss of motor function of approximately one point per year. Despite the continued treatment with this standard of care therapy.
Speaker Change: The purple shaded box represents the time period in terms of duration of this nurse and treatment that is similar to the median treatment duration of this and nursing for those enrolled in Sapphire, a time period, where the sapphire patients. We're clearly on the declining phase of their treatment journey, while on standard of care.
Speaker Change: Now to take a look at how the critical map effects motor function over time slide 10 displays the change from baseline in Hammersmith scores by visit.
Speaker Change: These line graphs beautifully articulate the treatment benefit of <unk> over the course of the treatment period.
Speaker Change: As you can see the Hammersmith scores improved in patients on <unk> as early as eight weeks. The first post baseline assessment by contrast, the scores decreased in those on placebo with a change from baseline of minus 1.2 points similar to the long term data on NUCYNTA person that indicated a loss of about one point per year.
After four years of treatment.
Speaker Change: Theres early separation between <unk> and placebo the difference widens by the end of the treatment period underscoring the effect of <unk> on the disease course from losing function despite being on standard of care to gaining function by adding a pit of amount.
Speaker Change: The strength of the results as illustrated by the Forest plot for all of the pre specified analysis shown on slide 11.
Speaker Change: As shown there is consistency across analyses with all showing improvement favoring a peterka amount, including across doses and across age groups. These analysis speak to the strength and the robustness of our results.
Speaker Change: Moving to slide 12.
Speaker Change: To further underscore our functional improvement, we see 30% of sapphire patients on <unk>, achieving a three point improvement versus 12, 5% for those on placebo. This transformative magnitude of improvement is extraordinary on top of standard of care.
Speaker Change: Again, the strength and consistency of the results demonstrate the ability of the pit or map to alter the course of disease from losing function to gaining function with a potential for profound impact on the lives of those living with SMA.
Speaker Change: We believe that <unk> is suitable for chronic treatment for a broad SMA population based on the efficacy seen with sapphire with improvements seen across all age groups, 2% to 21, the well tolerated safety profile with safety supported by more than four years of experience in SMA and we are working with urgency to finalize our rare.
Speaker Change: <unk> applications and submit the BLA and MAA in the first quarter of 2025.
Speaker Change: Now moving to our cardio metabolic program on slide 14.
The recent approvals and rapid adoption of <unk> appetite has had very positive impact on those living with vessels with obesity. However, a key issue that has emerged as the significant loss of lean muscle mass associated with these highly effective treatments.
Speaker Change: Given the important role muscle plays in energy metabolism in glucose homeostasis, maintaining appropriate levels of lean muscle is essential to healthy living.
Speaker Change: We believe our approach to preserving lean muscle mass with our highly selective novel anti Myostatin SRP 439, when used with the <unk> receptor agonist can preserve lean muscle mass and promote promote healthy weight management we.
Speaker Change: We see <unk> as part of the next wave of innovation in the treatment of obesity and are working to submit the IND targeted for mid year 2025.
Speaker Change: Now to slide 15.
Speaker Change: We designed a comprehensive non clinical program with S. R. K 43, nine and to date, we've demonstrated strong scientific rationale and promising non clinical evidence, including demonstrating preservation of lean mass during GOP, one receptor agonist induced weight loss improvement in fasting glucose beyond <unk>, one receptor agonist alone increase.
Speaker Change: And lean mass and attenuation of fat mass regain following <unk> receptor agonists withdrawal greater potency compared to an anti <unk> two antibody and most recently an increase in lean mass and lowered fat mass gain following treatment with metformin and SRP 439.
Speaker Change: Turning to slide 16, we entered into the area of obesity as we believe we have an elegant solution to preserving lean muscle mass with the potential attractive risk benefit profile for long term healthy weight management, we have the right target and a bit <unk>.
Speaker Change: Inhibition of Myostatin, a negative regulator of muscle is known to promote muscle growth and function. We are a validation of our approach given the results of sapphire demonstrating improvement in motor function and further preserving lean mass has the potential to improve durability of weight loss <unk>.
Speaker Change: And our highly selective approach and targeting the Korean late in form of Myostatin minimize unwanted toxicities and supports the potential for a favorable benefit risk profile.
Ted Myles: I will now buy tad to provide a financial and business update Ted Thanks, Jay it's been a very busy few months and I'm excited to provide some additional insight into areas of focus recently and in the months to come.
Ted Myles: Turning to slide 18 based on the positive Sapphire data that we disclosed in October we completed an upsized follow on offering of $345 million on a pro forma basis. This puts our September 30 cash balance at approximately $463 million, which enables us to scale up and focus on driving key priorities forward.
Ted Myles: Namely expanding our anti Myostatin platform and preparing for the commercialization of a pedigree map if approved.
Ted Myles: The success of Sapphire Sapphire trial Derisked, the Pilgrim AD program and provides an opportunity to expand the number of SMA patients that a pedigree mab may help as we have disclosed previously we are looking forward to initiating the <unk> clinical trial in mid 2025 to explore a particular mab in combination with standard of care in patients under two <unk>.
Ted Myles: Years of age.
Ted Myles: Another key priority for our anti Myostatin platform is advancing our novel selective anti Myostatin antibody <unk> hundred nine we expect to file our IND for <unk> hundred 39 next year and we look forward to reporting the results of our embrace trial in the second quarter of 2025.
Ted Myles: Embraces a proof of concept study have a pedigree mab in a patient population living with obesity.
Ted Myles: Testing the hypothesis of a selective anti myostatin as an important therapeutic approach to healthy weight management.
Ted Myles: And of course, we are fully engaged in commercial launch preparations are a pedigree mab in SMA.
Ted Myles: As we've disclosed previously before we had positive data the company was being very thoughtful about planning the work that needed to occur while managing resources carefully.
Ted Myles: Not to invest too much too early.
Ted Myles: With the positive data and the Upsized raise we are now actioning those plans focused on the following key initiatives.
Ted Myles: The first is continued engagement of critical stakeholders, such as <unk> and the patient community.
The second is ensuring an optimal treatment experience for those with SMA.
Ted Myles: And the third is building a world class commercial team to deliver both the U S and European launch.
Ted Myles: Turning to slide 19, I will provide a bit more insight into our approach to market over the near and intermediate term.
Ted Myles: We have strategically invested in foundational activities over the past several years.
Ted Myles: Among the most important of these investments we've been building strong relationships with HCP patient and advocacy communities and payers in both the U S and Europe.
Ted Myles: We continue to partner listen and learn from these very important stakeholders.
Ted Myles: Our MSL team has been engaging with health care providers at key SMA treatment sites, including cure SMA and MDA centers. We also began hiring our payer accounts team to engage and educate national regional and government payers.
Ted Myles: This past June we launched life takes muscle the first muscle muscle focused disease education campaign in SMA, which reflects what we have heard from the community that more is needed beyond current standard of care to treat SMA.
Ted Myles: There has been positive reaction to the campaign from patients caregivers and treaters with engagement on social media channels.
Ted Myles: In 2025, as we I successful launch of a pedigree mab, we're committed to ensuring an excellent and customized treatment experience for patients we aim to serve.
Ted Myles: This also includes working with channel and hub partners to deliver excellent patient services support and providing the option for monthly home infusion of a pedigree map.
Ted Myles: Finally, we look to complete the build of our field team, we believe that with a total of fewer than 50 full time employees, we can reach and support the U S market in a capital efficient and highly effective manner.
Ted Myles: As we look to 2026, it's worth full awareness that SMA is a global disease and our goal is to make a pedigree <unk> available to as many SMA patients as possible.
Ted Myles: If approved we plan to commercialize in selected European countries and will partner with distributors and strategic partners to reach patients outside of the U S and Europe.
Turning to slide 20, we see a potential path to blockbuster status for our pedigree mab in SMA.
Ted Myles: A few key market dynamics give us high confidence that scholar Rock's first commercial launch is positioned for success.
Ted Myles: The right market, the right medicine, and the right approach.
Ted Myles: The current treatment landscape for SMA is $4 5 billion and growing.
Ted Myles: While these treatments address motor neuron component of SMA, the muscle component of the disease is yet to be addressed.
Ted Myles: We're committed to developing a paragraph for as many SMA patients as possible who may benefit from this cutting edge therapy.
Ted Myles: Importantly, we look to our clinical success to demonstrate that <unk> is safe and effective and is addressing an important unmet medical need for patients with SMA.
Ted Myles: We know where the patients where they SMA patients are and we know many of the physicians who are treating these patients.
Ted Myles: While there is still a great deal of work to be done and we as we continue to educate the broader community. There is a high degree of awareness that muscle targeted therapy is the next important frontier for patients living with SMA.
Ted Myles: Based on these dynamics, we believe a pedigree mab has a total revenue potential of greater than $1 billion and that assumes a competitive muscle targeted therapies market in SMA.
Ted Myles: Turning to slide 21, there is no doubt the scholar rock is firing on all cylinders. We have a very exciting 2025 ahead of us and we're running with a sense of urgency and excitement as we strive for continued operational excellence there.
Ted Myles: There are multiple upcoming milestones and value inflection points for scholar rock.
Ted Myles: Our team remains on track to complete the regulatory submissions in the first quarter of 2025.
Ted Myles: We're expanding our myostatin clinical efforts as we look to the under two SMA population as well as those living with obesity and we are driving forward to transitioning scholar rock into a commercial company with a planned launch of our pedigree mab in SMA.
Jay Backstrom: Now I will pass back to Jay for some concluding comments before we open the line for questions Jay Thank you Ted.
Jay Backstrom: Moving on to slide 22, with the success of Sapphire, we are getting closer to fulfilling our mission of creating new possibilities for people living with serious diseases, starting with SMA.
Jay Backstrom: Sapphire is a validation of our industry, leading scientific platform and of our selective myostatin inhibition programs and further reinforces our confidence in our approach to healthy weight management and obesity.
Jay Backstrom: In closing, we believe <unk> has the potential to transform the standard of care in SMA.
Jay Backstrom: Sapphire met its primary endpoint with a one eight point improvement for <unk> plus standard of care compared to placebo plus standard of care as measured by the gold standard SMA specific Hammersmith functional motor scale. This improvement is both clinically meaningful and statistically significant with a pedigree mab patients showing a gain in their functional scores.
Jay Backstrom: Compared to a decline in scores for those randomized to placebo. Despite receiving standard of care. We believe a peterka mab is suitable for chronic treatment for a broad SMA population based on the efficacy data from Sapphire with motor function improvement seen across all age groups two through 'twenty, one and a well tolerated safety profile with the safety data.
Jay Backstrom: From Sapphire further supported by more than four years of experience in SMA as Ted highlighted we are working urgently to bring a peterka amount as soon as possible to those living with SMA and are finalizing our regulatory applications and preparing for commercial launch assuming approval with success and set in the <unk>.
Jay Backstrom: <unk> will serve as a catalyst for us to become a fully integrated commercial biotech company as well as becoming the foundation for our neuromuscular franchise with multibillion dollar potential clearly a very exciting time at scholar rock.
Speaker Change: That concludes our prepared remarks, we'll now open the call up for questions.
Speaker Change: Thank you so much and as a reminder to ask a question. Please press star one one on your telephone and wait for your name to be announced to withdraw yourself Press Star One again, one moment for our first question.
Speaker Change: And it comes from the line of generally with Jefferies. Please proceed.
Speaker Change: Hi, Good morning, Thanks for taking our question. This is generally on the line from Michael E.
Speaker Change: So we know that Lilly recently started a new phase <unk> could you just talk about the differences in trial design.
Speaker Change: I thought of administration in patient population and a question on how do you put that into context against your political map.
Speaker Change: And then secondly could you also walk us through the different scenario of how your obesity data readout next year and what are the key parameters that youre looking for that could define good data. Thank you.
Jay Backstrom: Yes. Thank you. Good morning. So this is Jay so with respect to the Lilly trial and are embraced study and let's start with our embraced trial are embraced study as designed.
Jay Backstrom: To assess our ability to target selectively anti myostatin with the Pitter mab with the primary endpoint of lean muscle mass to validate our thinking around our approach to preserving lean muscle and has a 24 week end point with lean mass as its primary endpoint and we have additional.
Jay Backstrom: Safety as well as exploratory end points. So it's really a proof of concept to guide our thinking around <unk> 39.
Jay Backstrom: We are using a <unk> because we can <unk> four through nine will be a sub Q presentation and Thats. The reason why we're focused on it going into clinic.
Jay Backstrom: There Lilly trial is looking at is still a number of doses exploring their program against that there's appetite. Our program is looking at there's appetite as well as we included that in our study. So we're kind of moving forward I think we're in a very good place with S. R. K fourth Ryan for three nine to open up our IND.
Jay Backstrom: Mid year next year and that will position us to be able to continue to run forward and assess this more formally in clinic.
Jay Backstrom: With respect to what we want to see from our embraced trial, we're looking to assess our ability to maintain and preserve the muscle mass as we've shared throughout all of our non clinical experiments that preservation is really clinically meaningful it enhances the ability to.
Jay Backstrom: To manage weight loss durably it Hasnt November reducing fat mass. It has an effect on glucose metabolism. So a lot of a lot of interest a lot of interesting ideas on the exploratory endpoints that will carry into clinic with <unk> 439.
Jay Backstrom: Yes.
Speaker Change: Got it thank you so much.
Speaker Change: Okay. Thank you one moment for our next question. Please.
Speaker Change: And is from the line of Sarah <unk> with BMO capital markets. Please proceed.
Speaker Change: Great. Thanks for taking the questions a couple ones.
Speaker Change: First on the <unk> study just was wondering given sort of what you've learned with the <unk>.
Speaker Change: From Sapphire, how are you thinking about sort of dos.
That in combination.
And Neil to study and then also maybe an obesity just how you're thinking about the differences in some of the combination approaches.
Speaker Change: The profiles of miles that inhibition rich with respect to something like the <unk> or are there sort of new muscle preservation approaches that are being explored in a year.
Speaker Change: Anything that you've kind of seen in terms of sort of differences or similarities in these different approaches and muscle preservation.
Just broadly thank you.
Speaker Change: Okay. Thank you for the question so let's start with Opal.
Speaker Change: It's a study under two and of course, we're authorized to study two and above I'm very keen to get that trial open with respect to dosing, where it's going to take full advantage of all of the PK PD data that we have not only from the Topaz study, but also now with the Sapphire data and really do modeling to kind of assess the doses. Our team is working on that now.
Speaker Change: I've mentioned previously we've been authorized to proceed with that study, we've socialized that both the FDA and the European authorities.
Really waiting to get the final clinical data at the PK PD data, which will help us understand because its milligram per kilogram dosing it will really be based on our <unk>.
Speaker Change: Assessment that 10 milligram per kilogram is going to be the effective dose there will be a combination of assessing all of the data together to guide the dosing and that under two patient population.
Speaker Change: And then with respect to the emerging therapies in the weight loss field.
Speaker Change: Clearly what we have today are the leaders with <unk> appetite and some magnetite and obviously theres a lot of innovation following as we as we mentioned at our New York event and you heard from Dr. <unk>.
Speaker Change: <unk> at Yale lean muscle loss happens almost regardless of the means of weight loss, including bariatric surgery. For example, so as we think about these other therapies going forward still going to be a component of lean mass loss still critical to maintain that lean mass loss and frankly, our strategy with targeting <unk> three nine will be.
Speaker Change: Similar to what we thought about in SMA, we will be agnostic to the treatment therapy, because we believe that our safety profile and target lends itself to combination strategies. So as those things get closer to clinic will have an opportunity to further assess it but for now I think our program is still built around combination with <unk>.
Speaker Change: Yeah.
Speaker Change: Great. Thank you.
Speaker Change: Okay.
Speaker Change: Okay.
Speaker Change: Thank you and as a reminder that is star one line. If you do have a question.
Speaker Change: Our next question is from Gary Nachman with Raymond James. Please proceed.
Speaker Change: Good morning, Thanks, Congrats on all the progress so as you're preparing the filing in SMA will you be seeking approval for both doses 10, 20, Meg per kg or just the lower dose and do you think FDA and EU will accrue for the entire age range two to 12 and also the exploratory <unk> to 'twenty.
Speaker Change: And it sounds like you're pretty confident in that.
Speaker Change: And I guess are you, having a pre NDA meeting with the FDA specifically to ensure that you are aligned on that front.
Speaker Change: And then just one more.
Speaker Change: How have.
Speaker Change: Maybe just set up the payer team, but how have initial payer discussions been going for the SMA how are they viewing the sapphire data and any updates on I guess, how are you thinking about pricing.
Speaker Change: <unk> range, maybe you could give us that's factored into that greater than $1 billion opportunity. Thanks.
Speaker Change: Yeah, Hey, Gary Good morning, So maybe kind of starting to see if I got your questions and kind of rank order.
Speaker Change: So first of all with respect to dose and as we mentioned when we released the data.
Speaker Change: 10, and 20 performed interchangeably and so as a result of that we're going to go forward with the 10 milligram dose do not expect that we will have two doses in the label given the consistency of the results and FDA thinking around lowest doses best dose. So we feel very very good about that and so we're going to go forward with the 10 milligram dose with respect to the label.
Speaker Change: In relationship to age.
Speaker Change: As we anticipated the data coming from SaaS or before we saw it in our thinking was it if that point estimate of effect size was similar between the 13 and 21 year old group. We are in very very good shape to demonstrate that this should be used independent of age and as you saw with our staff. Our data was spot on the exact point estimate so FDA views this as a single dose.
Speaker Change: There is no reason to believe that it will work and someone 12 not 13, so were going into this feeling confident that we have deployed age range in there given the consistency of data so feel good about that.
Speaker Change: Yes of course more to come but frankly from FDA labeling approach. They really don't define it by age short of gene therapy, which is approved under two so feel very good about the age and the breadth of that in our label.
Had really good interactions already from the regulatory front, we've already engaged with.
Speaker Change: Yeah.
Speaker Change: We've already engaged with a pre submission meeting with the European agency given the fact, we have prime designation. So we're all teed up for the regulatory applications, we've already engaged FTA and helping understand how they'd like to see our data presented so feel very good about that and yet it's good corporate practice to have a pre BLA meeting with the FDA just.
Speaker Change: To be sure there is nothing else that they consider but we feel very good about where we are and so we're off and running on that.
Speaker Change: With respect to the payer interaction maybe I'll just make a comment on kind of hand over to Ted to add some additional color as you can imagine it's early days with the Sapphire data we've already done some preliminary look in interaction with the Payors what I can tell you as I think about our data to just look at the data that we presented so far gaining function instead of losing function, helping preserve in Maine.
Speaker Change: <unk> function the value on top of the additional therapy. Thank you have a really very good value proposition and the potential to really make a significant impact on those living with SMA and so feel that that will be recognized by the payers as we go forward, but more to come on the specifics.
Speaker Change: Thanks, Jay and <unk>.
Speaker Change: Gary as you May expect it's a bit early for us to.
Speaker Change: Unveil our pricing assumptions and provide guidance in that regard.
Speaker Change: What I can comment on further to Jay's comments.
Speaker Change: The payer discussions have been going on payer work has been going on since even before we had sapphire data sapphire data exceeded our expectations. So that only helps helps our case.
Speaker Change: What I can point to as current market dynamics and what we're seeing in the market right. Now is that patients for example will get on gene therapy and in some cases.
Speaker Change: Ultimately end up on the SMN collector, and so reimbursement for $2 million gene therapy, followed by an expensive SMN collector and in some cases patients will switch from one SMN correct or to another so what we're seeing in the market is that payers are supporting these patients.
The patient advocacy group is very well organized and very impactful to.
Speaker Change: To help patients.
Speaker Change: In their treatment paradigm. They are very motivated as we talked about earlier to not only <unk>.
Maintain function that they have but gain more and so we think the wins at our back and we're going to continue to do our work and we'll continue to update the market as we learn more.
Speaker Change: Okay, Great just one quick follow up.
Speaker Change: Are you going to hire the 50 or so reps prior to approval and have them ready to go or are you going to wait till you actually get the approval just in terms of how we're modeling that thanks.
Speaker Change: So I think it's safe to assume that will continue to be very financially prudent and capital efficient to make sure that we're running through the start line, but we're also not going to disadvantage ourselves when we launch so as we think about the way we balanced.
Speaker Change: Building, a scaffolding and being ready to get ready in advance of data that we released last month and now we're executing on the plans that we had thoughtfully developed prior to having data.
Speaker Change: Pulling the same playbook as we get ready for commercial launch so that fine balance of making sure that we're deploying the capital the right amount of capital at the right time.
Speaker Change: Okay, great. Thank you.
Speaker Change: Thank you.
Speaker Change: Our next question is from the line of Pat <unk> with <unk> Securities. Please proceed.
Speaker Change: Hi, This is Alex xenakis on for quite a while.
Speaker Change: Building on Gary's question labeling a little bit given that a political map shows benefit across all the patient populations.
And how acclimate is viewed with the FDA what are the chances that you might get a broader label that also includes irrespective of ambulatory status and are there any differences in how the FDA or the <unk> SMA that would alter that get their interpretation and ultimately labeling decision.
Speaker Change: Yes. Good question. So if you take a look at the current FDA approach to labeling if you look at the new <unk> and rest of the plan labels FDA tends to see SMA as a monogenic disease, a single disease, a kind of a continuum.
Speaker Change: So theyre labeling approach is broad it's for the treatment of pediatric and adult patients with SMA not confined by type by ambulatory status could rather for the treatment of SMA as a continuum and we anticipate that they will follow the same labeling convention for our program.
Speaker Change: More to come on that but no reason to believe that they would do differently. So that's what we're anticipating with respect to that.
Europe tends to follow a similar approach they may depending there is a little bit of a different thinking around sometimes what does get entered doesn't get in the label, but I can tell you from our pre submission interaction. So far we feel like theyre seeing the similarly, they see it as a single disease, we have prime designation, which underscores they recognize the value in <unk>.
Speaker Change: <unk> of this going forward, so again more to come as we get closer down the road in our labeling negotiation tends to be toward the end of the approval process, but going into it given the strength of our data we feel very good about our position going forward and the potential for a broad label.
Speaker Change: Thanks, and congrats on the progress.
Speaker Change: Thank you.
Speaker Change: And I see no further questions in the queue I will conclude our Q&A session and program for today.
Speaker Change: You, all who participated and you may now disconnect.