Scholar Rock Holding Corporation Common Stock 12th Annual Cantor Fitzgerald Global Healthcare Conference

Thanks, Eric. It is great to be here with you and Cantor Fitzgerald. It is a really exciting time for the company. This is Scholar Rock's 15th year in existence. Vikas can join us up here whenever he would like. We have a series of regulatory, commercial, and clinical milestones in front of us in just the next 12 months. First and foremost, as Eric is noting, we are on file with the FDA with our Biologics License Application for apitegromab, which is a monoclonal antibody that we have developed for spinal muscular atrophy. We have taken that program through 7 years of clinical development, a phase II TOPAZ study, a phase III SAPPHIRE study, and delivered the first-ever myostatin inhibitor that has ever demonstrated clinically meaningful and statistically significant improvement in motor function in a phase III trial.

Of course, that has led us to this moment in time where we are now on file with the FDA. We expect a decision in the next 21 days, with the action date of September 30th. Eric and I will get into that a bit further. We also expect to be refiling our MAA with the European Medicines Agency in the coming weeks and months. We will get into that, of course. Just like in the U.S., in Europe, we were delayed by an issue with our third-party fill finish manufacturing facility or drug product facility, Catalent Indiana. But we have successfully stood up an alternate fill finish facility in rapid time. We also noted just recently that we are anticipating also submitting our application for apitegromab in children and adults living with SMA in Japan.

So we will be submitting to PMDA by the end of this year and are super excited to enter that market of 120 million population country in a faster amount of time than we had otherwise thought. That is because we did gain agreement with the PMDA to be able to file without a local clinical trial for apitegromab. In addition to those regulatory milestones, we are super excited to commercialize apitegromab in the U.S., Europe, Japan, and up to 50 countries around the world. I am sure we will get into that this morning. Finally, we do have a series of important ongoing clinical trials that are happening at the company.

The first that I would note is the phase II OPAL study, which is a study with apitegromab in the youngest of patients suffering with SMA under 2 years of age that have received ZOLGENSMA, the gene therapy. That trial continues to progress and enroll nicely. We have also just recently announced for apitegromab and FSHD that we have started to dose our initial study participants in the phase II FORGE study. This is a 60-patient, double-blind, randomized, placebo-controlled trial for patients with the moderate phenotype of FSHD receiving apitegromab, and we look forward to robustly enrolling that study. Then finally, by the end of this year, we should have our initial data on SRK-439, which is a novel high potency, high affinity myostatin antibody. We are the world leaders in myostatin inhibition.

This is a standalone asset, and we will have a readout in healthy volunteers toward the end of this year, and certainly no later than J.P. Morgan, just into 2027. So busy time at the company, Eric. A lot of important milestones, all in the advancement of sort of extending our leadership position in myostatin biology as the world leaders that have been able to unlock the benefits of inhibiting myostatin safely and effectively. That really sets up for a whole host of things for us to do over these next 12 months, and more importantly, what we are going to discuss over these next 20, 25 minutes together.

Thanks for being here. Let's- I would note that Vikas has enabled us to have the capital to do all of these things, as in our last earnings call, we indicated that we have a cash balance of nearly a half a billion dollars.

Certainly, we are well supported by our balance sheet to drive all of our commercial, regulatory, manufacturing, and clinical development initiatives.

Oh, no. He's going to get into it in much more detail.

Yeah. Eric raises the most important point of all. Obviously, we were surprised and disappointed on September 22nd of 2025 that we did receive a complete response letter for our initial BLA for apitegromab. Notably, the FDA indicated in that complete response letter that the sole approvability issue at that time was the state of compliance, or lack thereof, at our drug product manufacturing facility, our fill/finish facility, which was Catalent Indiana in Bloomington. And of course, following the complete response letter, we worked very closely with Catalent Indiana, the owners, Novo Nordisk, to work aggressively to remediate the challenges in that facility. But at the same time, as Eric and I have discussed, while not a lot of folks were paying attention, we were very focused on standing up an alternate fill/finish facility in record time.

And so if you recall, Eric, we had our Type A meeting following the complete response letter on November 12th of 2025. That was a collaborative and constructive meeting with the FDA. We were joined at that Type A meeting by Novo Nordisk to discuss the progress at the facility. We were also joined by Cure SMA to discuss the high unmet need for patients living with SMA. So that was on November 12th. One thing that I am not sure a lot of folks paid a lot of attention to on November 14th, which was our issued press release for our Q3 earnings call, is that we indicated that we were now under contract with an alternate fill/finish facility, and we had secured commercial manufacturing in the first half of 2026.