CAMBRIDGE, Mass. — Scholar Rock reported first-quarter financial results and said it is preparing for a potential U.S. launch of apitegromab, its investigational treatment for spinal muscular atrophy, if approved by the U.S. Food and Drug Administration.
The Cambridge biopharmaceutical company said the FDA accepted its Biologics License Application for apitegromab for the treatment of children and adults with spinal muscular atrophy. The agency set a Prescription Drug User Fee Act action date of Sept. 30, 2026.
Apitegromab is an investigational fully human monoclonal antibody designed to inhibit myostatin activation in skeletal muscle. Scholar Rock said it is the first muscle-targeted therapeutic candidate in spinal muscular atrophy to show a statistically significant and clinically meaningful benefit in a pivotal Phase 3 clinical trial.
“With the FDA’s acceptance of our apitegromab BLA, we have achieved another critical milestone as we work with urgency to deliver on our mission to bring the world’s first muscle-targeted treatment to the SMA community,” said David L. Hallal, chairman and chief executive officer of Scholar Rock. “We are grateful for the FDA’s continued high level of engagement, and we are pleased that important progress continues to be made at both of our fill-finish facilities. Our U.S. commercial team stands ready to launch apitegromab on or at any time prior to the September 30th PDUFA date.”
The accepted BLA includes two fill-finish facilities: Catalent Indiana LLC, part of Novo Nordisk, and a second U.S.-based facility. Scholar Rock said the FDA has completed a reinspection of Catalent Indiana, with classification expected within 90 days of the reinspection. The company said the second facility remains on track to provide commercial apitegromab supply early in the third quarter.
Scholar Rock is also planning for a European launch in the second half of 2026, beginning with Germany, pending regulatory progress. The company said a Committee for Medicinal Products for Human Use opinion on its European marketing authorization application is expected near mid-2026.
“Our balance sheet is strong, our clinical-stage pipeline continues to advance, and we are poised, now more than ever, to usher in the next phase of innovation for patients with SMA,” Hallal said.
Scholar Rock said enrollment is progressing in OPAL, a Phase 2 trial evaluating apitegromab in infants and toddlers under age 2 with spinal muscular atrophy who have received an approved SMN1-targeted gene therapy or are receiving an approved SMN2-targeted therapy.
The company is also advancing a subcutaneous formulation of apitegromab designed as a small-volume, self- or caregiver-administered anti-myostatin antibody suitable for an autoinjector. A Phase 1 study in healthy volunteers has been completed.
Beyond spinal muscular atrophy, Scholar Rock expects to initiate FORGE, a Phase 2 trial of apitegromab in facioscapulohumeral muscular dystrophy, in mid-2026. The company is also studying SRK-439, an investigational subcutaneous myostatin inhibitor, in a Phase 1 healthy volunteer study, with topline data expected in the second half of 2026.
For the first quarter, Scholar Rock reported a net loss of $105.5 million, or 83 cents per share, compared with a net loss of $74.7 million, or 67 cents per share, in the first quarter of 2025. The company recorded no revenue in either period.
Research and development expenses rose to $51.8 million from $48.7 million a year earlier. General and administrative expenses rose to $50.2 million from $28.4 million.
As of March 31, Scholar Rock had $479.9 million in cash, cash equivalents and marketable securities, including $100 million from a debt facility drawdown and $98 million in net cash proceeds from its at-the-market program.
