Scholar Rock Holding Corporation Common Stock Citigroup’s Biopharma Back to School Summit 2026

It's great to be here with you.

So quickly, we're in an exciting moment in time for the company. 15 years since we founded the organization and now really 3 weeks away from our PDUFA date with the FDA for our monoclonal antibody apitegromab, which is the first and only myostatin inhibitor that's ever been proven to deliver statistically significant and clinically meaningful benefits in a phase III trial, despite the fact that nearly all major pharma and biotech companies have tried to drug myostatin since it was discovered at Johns Hopkins in 1997 as the body's natural negative regulator for growing muscle. Obviously, if one could harness the potential of safely inhibiting myostatin and effectively inhibiting myostatin, you could restore and deliver muscle growth, muscle strength for a whole host of diseases. And we, of course, are focused on a set of muscular atrophies and muscular dystrophies.

The first, of course, is spinal muscular atrophy, where 35,000 patients globally are receiving at least one SMN targeted therapy. And in our 7-year development program, where we took no shortcuts, our phase III trial was 188 patients, and we delivered a statistically meaningful benefit in the highest bar, the Hammersmith Functional Motor Scale, for returning motor function improvement in those patients. So on file with the FDA and ready to launch, Geoff, we'll talk about that with our time together this morning. The action date for the FDA is September 30th. Looking to resubmit our MAA in Europe because we've recently withdrawn, all tied to the compliance issues at our third-party fill-finish manufacturing facility.

We were really excited to announce that we have an agreement with PMDA to file our JNDA by the end of this year, needing no additional clinical trials in a local Japanese population. This really advances our ambition to reach these 35,000 patients and upwards to 50 countries around the world, as Vikas and I and the team have done at other companies. We are super excited about that and look forward to digging in with Geoff this morning.

Absolutely. Look, Geoff is appropriately so asking probably the question on everyone's mind, which is if we had a PDUFA date last year of September 22, 2025, all signs read positive for an approval. We were discussing the label essentially a day or two before our PDUFA date, and then we received a CRL for the sole approvability issue being a general site inspection at our third-party fill-finish facility, Catalent, Indiana, owned by Novo Nordisk. That same facility now has just had another poor inspection in April of 2026. That was a re-inspection that was supposed to clear the facility. Why are we confident that being back on file with the FDA with an action date in just 20 days, why are we confident that we are heading toward an approval? It really dates back to the following.

At the same time that we were working with Novo Nordisk and the FDA for them to remediate their facility and improve their GMP practices, we were also standing up an alternate fill-finish facility that was in good standing with both U.S. and European regulators. We actually announced on November 14, which was our third quarter earnings call of 2025, that we were under a contract with an alternate fill-finish facility and that we had secured commercial manufacturing at that facility in the first half of 2026. So we did that very quickly. We rolled out tech transfer in breakneck speed. On early December, first week in December, we actually requested a Type C meeting with the FDA to talk about the progress at our alternate or second fill-finish facility.

That led to a March 3 Type C meeting where really what was on the table was the progress that we had made at this second fill-finish facility. And Geoff, it was during that Type C meeting with all the leaders of the FDA, OMQ and OPQ, who oversee facilities and product quality, that they said, "Look, this is an important drug for an important patient population. Our suggestion is that we actually have a horse race. Resubmit your BLA with both Catalent, Indiana, and your alternate fill-finish facility. Whichever one enables approval fastest stays in the BLA. Whichever one would not, you remove from the BLA with no timeline hit, no major amendment, and away we go." That was March 3 of this year.

On March 31 of this year, we announced that we had resubmitted our BLA just the day before, and that we had resubmitted it in complete alignment with the FDA with both fill-finish facilities. Now, look when we heard inspectors showed up at Catalent, Indiana on April 13, just like a couple of weeks after we resubmitted our BLA, you might imagine with all the attention that Novo Nordisk was putting on remediating this plant, we thought that would have obviously been the fastest path to an approval. We would have dropped our alternate site, and then we would have resubmitted that alternate site as an sBLA after approval. We now all know that that inspection from April 13 to April 24 didn't go so well. The Form 483 had multiple observations that all read like the poor inspection in 2025.

There was definitely improvements at the plant, but not enough to get the FDA through it downgrading that facility. It kind of started to read for us like the most expeditious path was going to be this alternate facility. Importantly, we have now viled more commercial apitegromab at this second facility than we ever did at Catalent Indiana, and vials from this second facility are now awaiting labeling and packaging in our third-party provider on the day of approval. We are in a very good position from a launch perspective. Now, specifically to Geoff's question, was it a poor inspection and we removed Catalent Indiana and we're kind of flying blind on the second facility? The answer to that question is no. In fact, what gives us great confidence is that we're in ongoing dialogue with the FDA.

Based upon the OAI classification of the April re-inspection, we actually said to the FDA, "We think it's time to remove Catalent Indiana." The FDA said, "Wait a minute. Let us just complete our review of your alternate facility, and then we'll actually walk you through the process to remove Catalent Indiana." And of course, in early August, we did announce, by press release, that we had removed Catalent Indiana from the BLA under FDA guidance, and that was following their review. I don't want to say that the FDA's ever completely done, but they definitely had a checkpoint in the way. So things are reading positive from both the CMC review and then the clinical review. There just isn't very much to do since the label was essentially finalized during the last cycle. And we remain very confident that we're going to be receiving an approval here in September.

It is an important point. This is not a boutique or opaque fill finish facility. It is a substantial facility with a track record globally. They have had European positive inspections as well as U.S. regulator positive inspections over the long run and recently. One of the things, Geoff, that we thought was important when we identified this facility was that they would have multiple lines that were actually ideal, where they had lines that were qualified for our vial configuration. Now, why was that important? That was important for speed, because if you change anything about your packaging, that would definitely not enable us to add a second vialer as quickly as we did. So we validated multiple lines with our engineering and PPQ runs. We have scaled up manufacturing there, which of course, again, it is fill finish, so they are taking our drug substance and they are vialing it.