Scholar Rock NASDAQ: SRRK said the U.S. Food and Drug Administration has accepted for review its resubmitted Biologics License Application for apitegromab, the company’s investigational treatment for children and adults with spinal muscular atrophy, and set a PDUFA action date of Sept. 30, 2026.
Chairman and Chief Executive Officer David Hallal said the accepted filing includes two fill-finish facilities: Catalent’s Indiana site and a second U.S.-based facility. He said that gives the company “two independent paths to apitegromab approval.”
The update follows a complete response letter issued last September, which Scholar Rock said was tied solely to observations from a routine general site inspection of the Catalent, Indiana fill-finish facility, owned and operated by Novo Nordisk. Hallal said the FDA has since completed an unannounced re-inspection of that facility, and that under FDA guidelines, the agency has up to 90 days to classify the site.
Hallal said Scholar Rock resubmitted the BLA in late March “in complete alignment with the FDA” to include both manufacturing sites. The company said the apitegromab drug product required for FDA review and potential approval from the second facility has been filed, and it expects to have commercial supply from that facility available in early third quarter, ahead of the Sept. 30 action date.
Company Says Launch Could Come Before PDUFA Date
Scholar Rock executives repeatedly said the company is preparing to launch apitegromab immediately if approved, including ahead of the formal PDUFA date. In response to an analyst question, Hallal said Class II resubmissions related to manufacturing issues can sometimes receive action before the deadline.
“We just know that we need to be prepared because many times Class 2 resubmissions and action can be taken by the agency well ahead of that PDUFA date,” Hallal said.
Akshay Vaishnaw, president of R&D, said the company anticipates approval of apitegromab for children and adults with SMA “by the end of the Q3,” potentially supported by either or both fill-finish facilities.
Vaishnaw also discussed the European regulatory process, saying the company’s Marketing Authorization Application is progressing through EMA review. He said Scholar Rock and the EMA had been scheduled for an oral explanation meeting, but the meeting was no longer needed after the two sides aligned in advance. The company continues to expect a CHMP opinion near mid-year.
Vaishnaw noted that approval in Europe also requires FDA clearance of the Catalent Indiana facility. Hallal said European regulators have been kept informed about the FDA process and have been “very flexible” on timing.
Commercial Preparation Focuses on SMA Treatment Centers and Payers
Chief Operating Officer Keith Woods said Scholar Rock’s U.S. commercial team is preparing for launch “immediately upon approval,” which could occur any time through Sept. 30. He said muscle strength and motor function remain major unmet needs nearly a decade after the introduction of SMN-targeted therapies, citing company-referenced data that 95% of patients continue to experience persistent and progressive muscle atrophy.
Woods said data shared by Cure SMA indicated that about one-third of people living with SMA in the U.S. have received two or more SMN-targeted treatments, either sequentially or in combination. He said the company views that as evidence of remaining unmet need and an opportunity for apitegromab as a muscle-targeted therapy.
The company said its U.S. field team has reached approximately 140 SMA treatment centers, 2,600 prescribing physicians and multidisciplinary care teams. Woods said Scholar Rock is preparing its patient services program, Scholar Rock Supports, to provide individualized support to patients, caregivers and providers.
During the question-and-answer session, Woods said the company has expanded site-of-care planning, including access through partners to more than 10,000 home infusion nurses across the U.S. He also said Scholar Rock has expanded its specialty pharmacy network so patients do not have to use multiple specialty pharmacies for different SMA treatments.
In Europe, Woods said Scholar Rock has established its regional headquarters in Switzerland and has local leadership in place in Germany, where it expects to launch following EMA approval. He said the company has hired medical and commercial field teams in Germany and is enrolling patients in a compassionate use program.
Pipeline Includes SMA Infants, FSHD and Subcutaneous Programs
Beyond the pending SMA application, Vaishnaw said Scholar Rock is continuing to enroll and dose patients in the Phase 2 OPAL trial, which is evaluating apitegromab in infants and toddlers under age 2. The study includes participants treated with SMN1-targeted gene therapy or receiving ongoing SMN2-targeted therapy.
The company is also preparing to initiate a randomized, double-blind, placebo-controlled Phase 2 trial called FORGE in facioscapulohumeral muscular dystrophy, or FSHD. Vaishnaw said the study is expected to begin enrollment soon and will include 60 patients. He described FSHD as a rare neuromuscular disease with more than 30,000 diagnosed patients in the U.S. and Europe and no approved therapies.
Vaishnaw said Scholar Rock prioritized FSHD based on unmet need, preclinical data from the FLExDUX4 mouse model and prior randomized studies suggesting that increases in muscle mass may be associated with functional benefit in FSHD.
Scholar Rock is also advancing a subcutaneous formulation of apitegromab. Vaishnaw said Phase 1 data presented in January showed favorable bioavailability and a pharmacodynamic profile comparable to intravenous administration. The company plans to engage U.S. and European regulators later this year following apitegromab approval.
The company also discussed SRK-439, described in prepared remarks as a novel high-potency anti-myostatin antibody currently in Phase 1. Vaishnaw said dosing in a healthy volunteer study is progressing and that top-line data are expected later this year.
Cash Position Strengthened in First Quarter
Chief Financial Officer Vikas Sinha said Scholar Rock ended the first quarter of 2026 with $480 million in cash equivalents and marketable securities. The total includes a $100 million drawdown from the company’s existing debt facility in March and $98 million in net cash proceeds from its at-the-market equity program during the quarter.
Sinha said first-quarter operating expenses were $102 million, and that operating expenses excluding stock-based compensation were $84 million. He said Scholar Rock’s 2026 financial priorities remain focused on supporting commercial launch readiness, funding R&D programs and evaluating opportunities to strengthen the balance sheet.
Upon FDA approval of apitegromab, Sinha said Scholar Rock will have the option to draw an additional $150 million from its existing debt facility. The company also plans to monetize a priority review voucher to further strengthen its balance sheet.
Hallal closed the call by saying Scholar Rock is focused on bringing what it describes as the first muscle-targeted therapy to children and adults living with SMA while building a broader neuromuscular disease pipeline.
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).
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