SRRK - NASDAQ

Good morning and welcome to Scholar Rock's Third Quarter 2024 Financial Results and Business Update Call. All participants will be in listen-only mode. After the company’s prepared remarks all participants will have an opportunity to ask questions. [Operator Instructions] Please note this event is being recorded. 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 on 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 sections of our website to find our most up to date SEC statements and filings. A recording of today's event will also be available on our website should you want to rewatch at a later date. I would now like to turn the conference over to Jay Backstrom, President and CEO of Scholar Rock. Jay, please go ahead.

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, our Phase 3 registration study in spinal muscular atrophy and a successful financing, we have great momentum heading to the end of 2024. I'm joined on today's call by Ted Myles, our Chief Operating Officer and Chief Financial Officer. For our call this morning, I will start with the 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. As shown on Slide 6, we had another very successful quarter building on the momentum that we created throughout 2024. 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 our lead program with apitegromab 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 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. In addition for our EMBRA

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. 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, namely expanding our anti-myostatin platform and preparing for the commercialization of apitegromab if approved. The success of the SAPPHIRE trial de-risked the apitegromab program and provides an opportunity to expand the number of SMA patients that apitegromab may help. As we have disclosed previously, we are looking forward to initiating the OPAL clinical trial in mid-2025 to explore apitegromab in combination with standard of care in patients under two years of age. Another key priority for our anti-myostatin platform is advancing our novel selective anti-myostatin antibody SRK-439. We expect to file our IND for SRK-439 next year and we look forward to reporting the results of our EMBRA

Thank you, Ted. 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. 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. In closing, we believe apitegromab has the potential to transform the standard of care in SMA. SAPPHIRE met its primary endpoint with a 1.8 point improvement for apitegromab plus standard of care compared to placebo plus standard of care is measured by the gold standard SMA specific Hammersmith Functional Motor Scale. This improvement is both clinically meaningful and statistically significant with apitegromab patients showing a gain in their functional scores compared to a decline in scores for those randomized placebo despite receiving standard of care. We believe apitegromab is suitable for chronic treatment for a broad SMA population based on the efficacy data from SAPPHIRE with motor function improvements seen across all age groups 2 to 21 and the well tolerated safety profile with the safety data from SAPPHIRE further supported by more than four years of experience in SMA. As Ted highlighted, we are working urgently to bring apitegromab as soon as possible to those living with SMA and are finalizing our regulatory applications and preparing for commercial launch assuming approval. The success in SMA will serve as a catalyst for us to become a fully integrated commercial biotech company, as well as becoming the foundation for a neuromuscular franchise with multibillion dollar potential. Clearly, a very exciting time at Scholar Rock. That concludes our prepared remarks. We'll now open the call up for questions.

[Operator Instructions] It comes from the line of Jenna Lee with Jefferies.

This is Jenna Lee on the line for Michael Yee. So we know that Lilly recently started a new Phase 2. Could you just talk about the differences in trial design, dosing, methods of administration and patient population, et cetera? And how do you put that into context against your apitegromab? And then secondly, could you also walk us through the different scenarios of how your obesity data may read out next year and what are the key parameters that you're looking for that could define good data?

So this is Jay. So with respect to the Lilly trial and our EMBRA

One moment for our next question, please. And it's from the line of Etzer Darout with BMO Capital Markets.

A couple ones, first on the OPAL study, just was wondering, given sort of what you've learned with apitegromab from SAPPHIRE, how you're thinking about sort of those for that, in combination in the OPAL study? And then also maybe on obesity, just how you're thinking about the differences in some of the combination approaches, the profiles of myostatin inhibition with respect to something like anilines or other sort of new muscle preservation approaches that are being explored? And how you're --anything that you've kind of seen in terms of sort of differences or similarities in these different approaches and muscle preservation just broadly?

So let's start with OPAL. It's a study under two and you of course we're authorized to study two and above. I'm very keen to get that trial open. With respect to dosing, we're going to take full advantage of all of the PK/PD data that we have, not only from the TOPA

Our next question is from Gary Nachman with Raymond James.

So as you're preparing the filing in SMA will you be seeking approval for both doses 10 mgs/kg and 20 mgs/kg or just the lower dose? And do you think FDA and EU will approve for the entire age range 2 to 12 and also the exploratory 13 to 21? It sounds like you're pretty confident in that. And I guess are you having a pre NDA meeting with the FDA specifically to ensure that you're aligned on that front? And then just one more. How have -- I know you just set up the payer team, but how have initial payer discussions been going for pembrolizumab and SMA? How are they viewing the SAPPHIRE data? And any updates on, I guess, how you're thinking about pricing, some range maybe you could give us that's factored into that greater than a $1 billion opportunity?

So maybe kind of start and see if I got your questions in kind of rank order. So first of all, with respect to dose, as we mentioned when we released the data, 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's thinking around lowest dose is 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 and relationship to age, again, as we anticipated the data coming from SAPPHIRE before we saw it, our thinking was that if the point estimate of effect size was similar between the 13 year 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 SAPPHIRE data, it was spot on the exact point estimate. So FDA views this as a single disease. There's no reason to believe that it will work in someone 12 and not 13. So we're going into this feeling confident that we have the full age range in there, given the consistency of data. So feel good about that. And, of course, more to come, but frankly from FDA's labeling approach, they really don't define it by age short of gene therapy, which is approved under 2. 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 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 FDA in helping understand how they'd like to see our data presented. So feel very good about that. And yes, it's good corporate practice to have a pre-BLA meeting with the FDA, just to be sure there's nothing else that they consider but we feel very good about where we are. And so we're off and running on that. With respect to the payer interaction, maybe I'll just make a comment, I'll 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 and interaction with the payers. What I can tell you as I think about our data, just look at the data that we've presented so far, gaining function instead of losing function, helping preserve and maintain function, the value on top of the additional therapy. I think we 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.

Thanks Jay and Gary, as you may expect, it's a bit early for us to unveil our pricing assumptions and provide guidance in that regard. What I can comment on further to Jay's comments is the payer discussions have been going on, payer work's been going on since even before we had SAPPHIRE data. SAPPHIRE data exceeded our expectations, so that only helps our case. What I can point to is 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 ultimately end up on a SMN corrector. And so reimbursement for $2 million gene therapy followed by an expensive SMN corrector. And in some cases patients will switch from one SMN corrector 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 help patients 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 wind is at our back and, and we're going to continue to do our work and we'll continue to update the market as we learn more.

Just one quick follow up, Ted, 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.

So I think it's safe to assume that we'll 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, 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, we're kind of 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.

Thank you. Our next question is from the line of Srikripa Devarakonda with Truist Securities.

Hi, this is Alex Nugent for Srikripa. And building on Gary's question, labeling a little bit, given that apitegromab shows benefit across all the patient populations and how SMA 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 view SMA that would alter their interpretation and ultimately labeling decisions?

So if you take a look at the current FDA approach to labeling, if you look at the nusinersen and risdiplam labels. FDA tends to see SMA as a monogenic disease, a single disease, a kind of a continuum. And so their labeling approach is broad. It's for the treatment of pediatric and adult patients with SMA, not confined by type, by ambulatory status but 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 with respect to that. Europe tends to follow a similar approach. They may, depending there is a little bit of different thinking around sometimes what does get in or doesn't get in the label, but I can tell you from our pre-submission interaction so far, we feel like they're seeing this similarly. They see it as a single disease. We have prime designation which underscores they recognize the value and potential of this going forward. So, again, more to come as we get closer down the road. 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.

Thank you. And as 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.