CAMBRIDGE, Mass. — Scholar Rock reported a second-quarter net loss of $109.9 million as the biopharmaceutical company prepared for a potential U.S. launch of apitegromab, its experimental treatment for spinal muscular atrophy.
The loss was nearly unchanged from $110 million in the same quarter last year. Loss per share narrowed to 84 cents from 98 cents.
Scholar Rock recorded no revenue in either period.
Research and development expenses declined to $58.2 million from $62.4 million, while general and administrative expenses increased to $50.7 million from $49.7 million.
The company ended the quarter with $492.1 million in cash, cash equivalents and marketable securities. That total included $62.8 million in net proceeds raised through its at-the-market stock program.
Scholar Rock said the U.S. Food and Drug Administration continues to review its biologics license application for apitegromab, with a target decision date of Sept. 30.
Apitegromab is a monoclonal antibody designed to inhibit activation of myostatin in skeletal muscle. The company is seeking approval for the treatment of spinal muscular atrophy, a rare neuromuscular disease.
Scholar Rock said the regulatory review now includes two fill-finish manufacturing facilities, providing separate potential paths to an FDA approval decision. The company has submitted the required data package for the second facility and said it has sufficient supply available for commercialization if the drug is approved.
The FDA’s inspection classification for the Catalent Indiana manufacturing facility remains pending.
“We are on the threshold of securing the world’s first ever regulatory approval of a myostatin inhibitor,” Chairman and CEO David L. Hallal said.
Scholar Rock said its U.S. commercial team is prepared to launch apitegromab immediately following a potential approval and has been working with spinal muscular atrophy treatment centers and prescribers.
The company is also discussing the next steps for apitegromab’s marketing application with the European Medicines Agency. The European review includes the Catalent facility, and Scholar Rock is considering adding the second fill-finish site to the application.
Enrollment and dosing are continuing in the Phase 2 OPAL trial, which is evaluating apitegromab in infants and toddlers younger than 2 who have received gene therapy or are taking another approved spinal muscular atrophy treatment.
Scholar Rock also initiated the Phase 2 FORGE trial of apitegromab in patients with facioscapulohumeral muscular dystrophy.
The study is expected to enroll approximately 60 patients who will receive either intravenous apitegromab or a placebo every four weeks for one year. Its primary goal is to measure changes in lean muscle volume.
The company is also conducting a Phase 1 study of SRK-439, a subcutaneous myostatin inhibitor designed to increase muscle mass. Initial data are expected in late 2026.
