Scholar Rock Q4 Earnings Call Highlights

Scholar Rock (NASDAQ:SRRK) executives said the company is “poised for a transformative year in 2026,” with its top priority centered on resubmitting the biologics license application (BLA) for apitegromab in spinal muscular atrophy (SMA) after an FDA reinspection of the Catalent Indiana fill-finish facility. Management also outlined continued progress across its anti-myostatin pipeline, commercial launch preparations, and financing strategy during the company’s fourth quarter and full-year 2025 results call.

FDA reinspection remains the gating item for apitegromab resubmission

Chief Executive Officer David Hallal said the company is “prepared to resubmit the apitegromab BLA following a successful FDA reinspection of the Catalent Indiana facility,” reaffirming guidance for BLA resubmission and a U.S. launch following approval in 2026. Executives repeatedly emphasized that the compliance status of the Catalent Indiana site remains the “sole approvability issue” cited in the company’s complete response letter (CRL) received last year.

Hallal described a series of events since a Type A meeting with the FDA in November that included Novo Nordisk and Cure SMA. A week after that meeting, the FDA issued a warning letter to the Catalent Indiana facility; Novo responded to the FDA by mid-December. The FDA then scheduled an early first-quarter meeting with Novo, and according to Hallal, the agency had “no additional requests” to Novo’s remediation plan at that meeting. Hallal also said the FDA sent a field team to Catalent Indiana after the meeting and again did not add requests to the remediation plan, instead stating its intent to conduct a site reinspection following routine manufacturing activities. Management said routine manufacturing activities resumed in late February.

On the resubmission trigger, Hallal told analysts the company would look for “a successful re-inspection” and “some level of confidence that it was a successful re-inspection,” adding Scholar Rock is “at the ready” to submit quickly once that occurs. When asked about review timing, management did not provide a specific turnaround expectation, but noted it had reached an advanced stage with a draft label during the original review cycle and expects alignment on a final label “relatively straightforward” after resubmission, while cautioning that label details are ultimately up to the FDA.

Second fill-finish site advancing, with plans for a supplemental filing

Scholar Rock also discussed progress on a second fill-finish facility intended to add redundancy and support future commercial demand. Hallal said engineering runs are underway and additional manufacturing runs will follow, with expectations to file a supplemental BLA later this year. President of R&D Akshay Vaishnaw similarly said engineering runs are underway with additional manufacturing runs planned for the second quarter, and he reiterated expectations to submit a supplemental filing with the facility later in 2026.

When asked how timelines might change if the second site needed to become the primary facility, Hallal said the company has viewed the effort as important “no matter what,” and does not believe timing would be “altered tremendously,” though he maintained the plan remains a supplemental filing. Hallal added that relying solely on the second facility would “de-risk” U.S. and EU commercial opportunities, based on the company’s confidence in the selected partner.

Europe review continues; decision expected mid-2026

Outside the U.S., management said the apitegromab marketing authorization application (MAA) review continues with the European Medicines Agency (EMA), and the company expects a decision in mid-2026. Executives noted they have kept in close contact with EMA reviewing authorities and said European regulators are aware of developments around Catalent Indiana. Hallal said progress with FDA and Novo “serves us very well” for the ongoing European review, citing mutual recognition between FDA and EMA.

Commercial launch preparations focus on education, access, and infrastructure

Chief Operating Officer Keith Woods said the company has built out critical commercial capabilities ahead of an anticipated launch. Woods characterized the SMA market as continuing to grow and now representing “nearly $5 billion in global annual sales,” while also highlighting what management called persistent unmet need in muscle strength and motor function.

Woods said Scholar Rock’s U.S. customer-facing team is active in the field, focused on disease education emphasizing SMA as a disease of the “motor unit,” consisting of both motor neurons and muscle. The company reported engagement across approximately 140 SMA treatment centers and 2,600 prescribing physicians and multidisciplinary care teams.

Key elements of launch readiness described on the call included:

• Specialty pharmacy network expansion: Woods said patients receiving an SMN-targeted therapy through a specialty pharmacy will be able to access apitegromab through the same specialty pharmacy.
• Home infusion network: The company has established a home infusion network of more than 10,000 affiliated nurses nationwide through patient access partners.
• Patient services program: Management is preparing to launch “Scholar Rock Supports,” designed to provide individualized support to patients, caregivers, and providers.
• Disease awareness campaign: The company launched the next phase of its “Life Takes Muscle” campaign in January.
• Payer engagement: Woods said the company is advancing discussions with national and regional payers as well as Medicare and Medicaid and has been able to review clinical data with payers.

Hallal also cautioned that while management expects demand, there may be initial “access speed bumps,” citing the need for a miscellaneous J-code at launch and potentially slower reimbursement processes in some channels, including Medicaid.

In Europe, Woods said Germany preparations include establishing local leadership, initiating a compassionate use program, and progressing reimbursement planning. The company is also advancing reimbursement dossiers in multiple countries, strengthening distributor relationships, and building EMEA infrastructure, with plans to begin a European launch in the second half of the year starting with Germany, contingent on approval.

Pipeline updates include OPAL, FORGE in FSHD, subcutaneous apitegromab, and SRK-439

Vaishnaw said enrollment and dosing continue in the Phase II OPAL study evaluating apitegromab in infants and toddlers under age two, including participants treated with SMN1-targeted gene therapy or receiving SMN2-targeted therapy. He described the trial as a step toward evaluating apitegromab across the spectrum of treated patients and emphasized the company’s view that early intervention could support muscle during critical development.

In facioscapulohumeral muscular dystrophy (FSHD), Vaishnaw said the investigational new drug (IND) application is cleared and the company plans to initiate a randomized, double-blind, placebo-controlled Phase II study called FORGE in mid-2026 that is expected to enroll 60 patients. He said the primary endpoint will focus on lean muscle volume measured by sensitive imaging techniques, with functional assessment using “validated” on-site biomonitoring methods in FSHD.

On subcutaneous apitegromab, Vaishnaw highlighted Phase I data in healthy volunteers showing that 800 mg subcutaneous administration produced an “overlapping pharmacodynamic profile” compared with 800 mg IV, suggesting favorable bioavailability and comparable pharmacodynamics. He said the company plans to engage U.S. and European regulators later in the year to align on development strategy, but did not provide a timeline to market.

For SRK-439, a subcutaneously administered myostatin inhibitor, Vaishnaw said Phase I dosing in healthy volunteers is underway with top-line data expected in the second half of 2026. He also cited non-human primate data suggesting SRK-439 is “10 times more potent” than apitegromab based on observed lean mass changes at low doses. In Q&A, management said SRK-439 is currently viewed as a proprietary asset with no plans to partner, and the company expects to share further development plans after Phase I data are available.

Financially, Chief Financial Officer Vikas Sinha reported $368 million in cash and cash equivalents at the end of 2025, including $60.4 million from warrant exercises during the fourth quarter. The company also announced a new debt facility of up to $550 million with Blue Owl Capital, including $100 million available at closing (used to repay a prior $100 million Oxford Finance facility), another $100 million expected to be drawn by March 31, an option to draw up to $150 million following FDA approval of apitegromab, and an additional option for up to $200 million subject to mutual consent. Sinha also said the company intends to monetize a Priority Review Voucher after approval to further strengthen the balance sheet.

About Scholar Rock (NASDAQ:SRRK)

Scholar Rock is a clinical-stage biotechnology company focused on the discovery and development of protein therapeutics that selectively target growth factors involved in disease processes. The company’s research platform is designed to modulate endogenous growth factor activation and signaling with high specificity, aiming to restore normal biological function across a range of disorders. Scholar Rock’s approach is distinguished by its emphasis on engineering antibodies and biologics that interact with growth factor precursors or latent complexes rather than the active form, potentially offering improved safety and efficacy profiles.

The company’s lead program, SRK-015 (appercept), is an investigational monoclonal antibody targeting the activation of latent myostatin proproteins and is being evaluated for the treatment of spinal muscular atrophy (SMA).