Scholar Rock (SRRK) Q2 FY26 earnings call: guidance and Q&A

How confident is Scholar Rock that the second fill/finish facility alone can support approval by the PDUFA date, and what FDA interactions support this progress?

Management is highly confident, noting the second facility has more vials ready than Catalent Indiana had at the last PDUFA, has passed recent FDA/EMA inspections, and the FDA review of its data package is progressing well. They confirmed that removing Catalent Indiana from the application would not impact the timeline.

“That second facility has had successful recent site inspections by FDA and EMA. None of the approvals from this facility in the last 12 months have required a PAI or a PLI.”

asked by Eric Schmidt · answered by David Hallal

If Catalent Indiana remains OAI, is there a deadline for administratively withdrawing that site to avoid PDUFA delays?

Management is in ongoing correspondence with the FDA. They stated that if the inspection classification remains OAI, it would be a simple step to notify the agency of the site's removal, and they expect no impact on the ongoing timeline, based on prior discussions with the FDA.

“We've discussed with the agency, it would be a relatively simple step to notify them that on their signal that we are removing that site and the review would progress with that second facility.”

asked by Srikripa Devarakonda · answered by David Hallal

What specific procedural items need to be completed by September 30 for approval, and is there any reason to believe the FDA won't complete the review by then?

Akshay Vaishnaw confirmed that all necessary data for the second fill/finish site have been submitted and the review is progressing well. He stated there is no reason to believe the FDA cannot complete its work by the September 30 PDUFA date, highlighting the agency's guidance and partnership.

“Their review of the second fill/finish site is progressing well. And as David said, we see no reason why we can't get to the PDUFA date with this drug approved before or at that time.”

asked by Tessa Romero · answered by Akshay Vaishnaw

How are payers framing apitegromab in terms of step edits, time limits, or re-review of benefit, and what are the expectations for initial authorization?

Keith Woods stated that the goal is broad reimbursement aligned with the potential label, not just study inclusion criteria. He expects prior authorizations and likely initial denials due to various reasons (e.g., J-code, policy not created), leading to an appeal process. He anticipates the average time from prescription to infusion to be greater than 60 days during the first 6 months post-launch.

“The majority of them, you are most likely going to receive a denial for various reasons, whether it's a J-code or a policy not created or for some other reason. That will then send us into an appeal process, which can sometimes take some time.”

asked by Cory Kasimov · answered by Robert Keith Woods

What was included in the data package for the second fill/finish facility, are stability runs complete, and is the facility fully launch-ready?

David Hallal confirmed the data package included engineering and PPQ runs, and the FDA has the full package. He stated that no more testing is required for the product vialed at that facility, and it is currently at the third-party labeling and packaging facility awaiting approval for launch.

“Everything is straightforward and perfunctory. And there is, I guess -- just to underscore the question, there is no more testing that is required for the product that has been vialed at that facility at all.”

asked by Amy Li · answered by David Hallal

What remaining commercial initiatives are needed before approval, and what is the low-hanging fruit for SMA patients, including the timeline to get them on board?

Keith Woods stated the team is ready to launch immediately. Key remaining work involves preparing treatment centers for patient enrollment into the Scholar Rock Supports program post-approval. Initial focus will be on converting Early Access Program and ONYX patients, but the process will take time due to prior authorizations and appeals, with an expected >60 days from prescription to infusion initially.

“The team is kind of chomping at the bit to really get out there and launch this product. With that being said, there's always additional work that we can do. One of the main things that's taking place that I mentioned is really working with the various centers, so that we can be prepared with these treatment centers on a case-by-case basis to work through the process of enrolling patients into our Scholar Rock Supports program.”

asked by Gary Nachman · answered by Robert Keith Woods

Can management speak to labeling discussions and whether they expect largely identical labels between the U.S. and Europe?

Akshay Vaishnaw noted that the dialogue through the medical review has been very constructive. While they cannot comment on the final label, they are comfortable with the conversations and look forward to getting the drug approved for the right patients. David Hallal added that the goal is to serve a meaningful number of patients globally.

“I would just say we are very happy and comfortable with the way the dialogue has gone through the medical review. And obviously, it's not for us to comment on the final label, but we are certainly grateful for the engagement and very constructive conversations.”

asked by Marc Frahm · answered by Akshay Vaishnaw

If the FDA maintains OAI for Catalent, what is the potential timeline to get the second fill/finish facility into the MAA and the earliest projected European approval timeline?

Akshay Vaishnaw stated that dialogue with the EMA is ongoing regarding the mechanism to incorporate the second fill/finish facility. He noted the EMA's flexibility and engagement, similar to the FDA, and that they will be guided by the EMA's advice, with updates to follow in due course.

“And as that dialogue is ongoing, I don't want to second guess what the final advice will be vis-a-vis necessity or the mechanism by which we incorporate the second fill/finish facility. But suffice it to say that the engagement and flexibility that both FDA and CHMP have shown us is very gratifying to us.”

asked by Kalpit Patel · answered by Akshay Vaishnaw