Scholar Rock's Q4 Earnings Call: BLA Resubmission Type, Fill/Finish Timelines, and Europe Strategy Contradict Prior Guidance

Date of Call: Mar 3, 2026

Guidance:

• BLA resubmission and U.S. launch following approvals expected in 2026.
• EMA decision on apitegromab MAA anticipated in mid-2026.
• Second fill/finish facility supplemental BLA submission planned later in 2026.
• New debt facility provides up to $550 million, with $100 million immediately available and additional draws expected.
• Cash and cash equivalents ended 2025 at $368 million.

Business Commentary:

Regulatory and Commercial Readiness:

• Scholar Rock is poised for a transformative year in 2026, with plans to resubmit the apitegromab BLA and achieve a U.S. launch following approvals.
• The company has made significant progress with the FDA, including a Type A meeting, and expects the European Medicines Agency to make a decision on the Marketing Authorization Application (MAA) in mid-2026.
• The positive regulatory momentum is driven by the high unmet need in the SMA community and a shared sense of urgency between Scholar Rock, Novo, and the FDA.

Financial Position and Strategic Investments:

• The company ended 2025 with $368 million in cash and cash equivalents, strengthened by $60.4 million from warrant exercises.
• Scholar Rock secured a new debt facility for up to $550 million to support its transition towards a global commercial space company and to invest in its pipeline.
• These financial measures are aimed at supporting commercial launch readiness, strengthening the supply chain, and advancing clinical programs.

Pipeline Progress and Clinical Development:

• Enrollment and dosing continued in the Phase II OVAL study, evaluating apitegromab in infants and toddlers with SMA.
• A robust, randomized, placebo-controlled Phase II study for apitegromab in FSHD is planned to initiate later in the year.
• The pipeline's advancement is supported by preclinical data and the potential of apitegromab to address significant unmet needs in various neuromuscular diseases.

Commercial Launch Preparations:

• The U.S. commercial team is actively educating prescribers and payers on the unmet need in SMA and the importance of targeting muscle.
• Launch readiness activities in Europe are progressing, with plans to begin in Germany in the second half of 2026.
• The preparation includes building a specialty pharmacy network and establishing a home infusion network to ensure patient access and support.

Innovation and Future Potential:

• Scholar Rock is advancing its anti-myostatin pipeline with a subcutaneous formulation of apitegromab and the SRK-439 myostatin inhibitor.
• The subcutaneous formulation showed promising results in a Phase I study, indicating a favorable pharmacokinetic profile.
• The company is focused on delivering life-transforming therapies for rare and severe neuromuscular diseases globally, with a strong emphasis on muscle-targeted treatments.

Sentiment Analysis:

Overall Tone: Positive

• Multiple statements reflect a positive and confident tone: 'poised for a transformative year in 2026,' 'pleased with FDA's continued level of engagement,' 'we are emboldened by the commitment,' 'we are ready now more than ever,' 'we are encouraged by the progress,' '2026 will be a transformative year for Scholar Rock.'

Q&A:

• Question from Eric Schmidt (Cantor): David, just to put a pin in on is Novo now ready for reinspection, open for reinspection? And then assuming the reinspection does go well, what would trigger your resubmission?
  Response: Management is gratified by the progress and shared urgency with FDA and Novo. The successful reinspection of the Catalent Indiana facility is the gating item; a successful reinspection would trigger immediate BLA resubmission.

• Question from Tazeen Ahmad (Bank of America): Not to belabor the point on timing here, but I know you're confident about the ability of Novo to resolve the issue. But in the event that you do have to revert to your backup facility, you've guided to a supplemental filing in the second half of the year. What would happen to the time lines of that needed to be the primary filing?
  Response: Management is prepared to pivot to a supplemental BLA if needed, but believes timing would not be altered tremendously. They are confident in the current plan for a supplemental BLA.

• Question from Tessa Romero (JPMorgan): So first one is, can you elaborate on what it meant that the FDA sent field team. What was the purpose of that? And is that routine? And then the second one, just to loop back on sort of better understanding the next procedural steps post the reinspection and what the time lines could be there.
  Response: The FDA's field team visit is seen as a sign of high priority and shared urgency to progress the apitegromab file. Management expects the current rapid pace of progress (reinspection, resubmission, review) to continue.

• Question from Ryan (Leerink, on behalf of Mani Foroohar): Maybe just 1 sticking with the review, kind of based off your latest conversations with the FDA, I'm curious what your expectations are for a turnaround time following BLA submission to eventual approval. Are there any details that still need to be worked out, label, et cetera, with regulators? And then maybe just as a second one on the pipeline. Can you talk about the strategy for 439.
  Response: Turnaround time specifics not provided, but the sole approvability issue is the Catalent facility status. For SRK-439, it is a high-potency, potentially low-volume drug; management has no current intention to partner it and will share further plans after Phase I data.

• Question from Srikripa Devarakonda (Truist): Timelines wise, not to be to the point, you expect -- you continue to expect inspection, BLA submission, U.S. launch, everything to happen in 2026. For the launch to be in 2020, say, can it still happen with a Class II physician? Or do you diligence suggests that this is most likely going to be a class of submission and in any of your recent conversations with the FDA, was there any hint or indication for a potential CPV for apitegromab?
  Response: Management reaffirms 2026 guidance, pointing to steady FDA prioritization. They have considered Class I vs. Class II and note that even Class II resubmissions can be decided before the full 6-month timeline. They are in close communication with FDA on all initiatives.

• Question from Michael Ye (UBS): Can you talk a little bit about the expectations for the label as it relates to either ambulatory, non ambulatory and with no issues regarding age subgrouping... And then a follow-up, assuming approval, maybe for Vikas, can you just remind us, given that your drug is a weight-based drug, how to think about the comparable pricing relative to other drugs.
  Response: Label discussions with FDA have been straightforward; final details depend on FDA. Pricing specifics cannot be shared yet, but will be detailed at launch. The drug is weight-based, leading to a range, but pricing will consider SMA's rarity, severity, and progressive nature, along with compelling clinical data.

• Question from Amy Li (Jefferies): So looking ahead to launch, what commercial analogs would you point us to as we think about the initial uptake and launch trajectory? And then maybe another one on subcu epi you think approval will require a full clinical study in SMA, a smaller bridging study or primarily human factor studies?
  Response: Launch uptake is expected to be robust due to high interest, but may face initial access speed bumps. Subcutaneous apitegromab has promising Phase I data; discussions with FDA are needed to determine the regulatory path forward, but it aims to leverage existing PKPD knowledge.

• Question from Unknown Analyst (Citi Group, on behalf of Geoff Meacham): Maybe just thinking about the second fill/finish facility. If you guys were to switch over to that one, would it completely derisk the supply chain from the U.S. and launch perspective? And then on the launch, what specific leading indicators of payer and physician readiness are you guys tracking?
  Response: Progress on the second fill/finish facility is rapid; it would derisk the supply chain if relied upon solely. Payer readiness is indicated by access discussions with national, regional, Medicare, and Medicaid, with physicians believing in multiple modalities for SMA treatment.

• Question from Unknown Analyst (TD Cowen, on behalf of Mark): Just one quick question kind of cross ones and does the status of the MMA review, will that market also be served by the Novocain Indiana facility? And if so, has the taken any action in response to the FDA inspection findings.
  Response: Progress with FDA on the BLA serves the EMA MAA review. Continued remediation and a successful reinspection will support the EMA decision near midyear. Any supplemental BLA using the second facility would also be important.

• Question from Etzer Darout (Barclays): Has the FDA requested or could they request additional safety data that could extend review of apitegromab. And then on HSD, just wondered would you be looking at any functional endpoints in the Phase II study that you're planning?
  Response: FDA has requested an update to the safety database, which is agreed upon and will be included in the BLA resubmission. For the FSHD Phase II study, the primary endpoint is muscle volume increase, with a secondary endpoint to assess functional impact.

• Question from Unknown Analyst (BMO Capital Markets, on behalf of Evan Seigerman): I just wanted to how are you thinking about expectations for time to profitability and whether you anticipate any additional need for financing ahead of that kind of profitability hinge point?
  Response: Management follows a typical rare disease revenue trajectory, expecting profitability in 2-3 years post-launch, balancing investment in the pipeline for long-term shareholder value.

• Question from Allison Bratzel (Piper Sandler): Just drilling down on some of the prior discussion around review timing. I know you talked a lot about FDA sense of urgency on apitegromab, I guess, is there a good precedent for FDA spending less than 6 months to review a Class II resubmission? And can you just clarify, does your guidance for commercial launch in '26 assume a Class II resubmission and the full 6-month review?
  Response: A precedent exists (e.g., Regeneron's 2023 resubmission) where a Class II resubmission was approved within less than 6 months. The 2026 launch guidance assumes a rapid process but does not specify Class I vs. Class II.

• Question from Unknown Analyst (Wolfe Research, on behalf of Kalpit Patel): Previous milestone inhibitors in FSHD increased muscle mass that meaningful functional improvement. Can you give some color on how apitegromab aims to address this historical hurdle and what a clinically meaningful functional improvement might be in the planned Phase II?
  Response: Apitegromab aims to address past hurdles with its selectivity and potency. The Phase II study includes a functional endpoint (muscle function testing) alongside the primary muscle volume metric to evaluate global functional improvement.

Contradiction Point 1

BLA Resubmission Type and Timeline

Guidance on BLA resubmission type shifts from being unspecified to being a Class II, creating a contradiction.

Srikripa Devarakonda (Truist) - Srikripa Devarakonda (Truist)

20260303-2025 Q4: The guidance remains 2026 for BLA resubmission and U.S. launch. The team has analyzed that even with a Class II resubmission, a decision is often rendered before the full 6-month timeline. - David Hallal(CEO)

Do you still expect a Class II resubmission leading to a Commissioned Prior Voucher (CPV) versus a Class I resubmission being more likely? - Eric Schmidt (Cantor Fitzgerald & Co.)

2025Q3: It is not for them [the FDA] to specify Class I or Class II; the agency has shown commitment to acting urgently to get the drug to patients. - Akshay Vaishnaw(President of R&D)

Contradiction Point 2

Second Fill/Finish Facility Timing

The start date for commercial capacity at the backup facility is stated as Q1 2026 in 2025Q3, but 20260303-2025 Q4 indicates readiness for a pivot to it in 2026.

Tazeen Ahmad (Bank of America) - Tazeen Ahmad (Bank of America)

20260303-2025 Q4: The company is confident in the progress with the primary facility but has prepared for a supplemental BLA as the backup plan. Whether it’s an SBLA or a resubmission, the timing is not expected to change significantly. - David Hallal(CEO)

How would reverting to the backup facility affect the supplemental filing timelines in the second half of the year? - Mani Foroohar (Leerink Partners LLC)

2025Q3: Commercial capacity there starts in Q1 2026. - David Hallal(CEO)

Contradiction Point 3

Commercialization Strategy in Europe

Contradiction on the strategy for European market commercialization.

Salvator Carusso (TD Cowen) - Salvator Carusso (TD Cowen)

20260303-2025 Q4: There is a mutual recognition agreement between the FDA and EMA. The progress on resolving the FDA issues supports the EMA’s mid-2026 decision timeline. Novo is engaged in discussions with EMA reviewers. - David Hallal(CFO), Akshay Vaishnaw(CEO)

Does the status of the MAA review in Europe depend on the Catalent Indiana facility, and has Novo taken any action in response to FDA inspection findings there? - Andres Maldonado (H.C. Wainwright)

2025Q1: Direct global commercialization is the priority, leveraging the team's experience. Partnering outside the US is not a focus. - David Hallal(CFO), Akshay Vaishnaw(CEO)

Contradiction Point 4

Supply Chain Robustness and Backup Facility Readiness

Contradiction on whether the supply chain is currently robust or if a backup plan is still being prepared.

Tazeen Ahmad (Bank of America) - Tazeen Ahmad (Bank of America)

20260303-2025 Q4: The company is confident in the progress with the primary facility but has prepared for a supplemental BLA as the backup plan. Whether it’s an SBLA or a resubmission, the timing is not expected to change significantly. - David Hallal(CEO)

If you revert to your backup facility, how would the timelines for the supplemental filing in the second half of the year be affected? - Eric Schmidt (Cantor)

2025Q2: The apitegromab supply chain is robust, with launch supply already manufactured, vialled, and ready. - David Hallal(CEO)

Contradiction Point 5

Nature of FDA Interaction and BLA Review Pathway

Contradiction on the level of FDA engagement and the regulatory pathway from the perspective of the company.

Tessa Romero (JPMorgan) - Tessa Romero (JPMorgan)

20260303-2025 Q4: The FDA’s action of sending a field team to the site demonstrates an extraordinary level of engagement and prioritization. - David Hallal(CEO)

What does the FDA field team's visit signify, is it routine, and what are the next steps and timelines post-reinspection? - Tessa Romero (JPMorgan)

2025Q2: The late-cycle meeting with the FDA was constructively collaborative... The tone is encouraging, and they are confident the FDA is on track for the PDUFA date. - David Hallal(CEO), Akshay Vaishnaw(President of R&D)