Scholar Rock Sells Rare Disease Voucher for $205 Million, Reports Q3 2026 Results

Scholar Rock Holding Corporation reported its financial results for the third quarter ended September 30, 2026, and announced a definitive agreement to sell a Priority Review Voucher (PRV) for $205 million. The company’s wholly owned subsidiary, Scholar Rock Inc., entered into an asset purchase agreement with BioNTech US Inc. to sell the voucher, which was awarded by the U.S. Food and Drug Administration (FDA) on September 11, 2026, following the approval of ISEMBYLD (apitegromab-mstn). The transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

Regarding its commercialization efforts, Scholar Rock reported net product revenue of $0.6 million from sales of ISEMBYLD in the U.S. during the third quarter of 2026. The drug, which is the first and only muscle-targeted therapy for spinal muscular atrophy (SMA), was approved by the FDA in September 2026 and is indicated for adults and children two years of age and older who are currently receiving an SMN2-targeted treatment. The company noted that ISEMBYLD was shipped to the specialty network prior to the quarter-end and that initial patients were infused in October 2026.

On the regulatory front, Scholar Rock resubmitted its Marketing Authorisation Application (MAA) for apitegromab to the European Medicines Agency (EMA) in October 2026. The submission was validated, and the company expects a Committee for Medicinal Products for Human Use (CHMP) opinion in the first half of 2027. Additionally, the company submitted a Japanese New Drug Application (JNDA) for apitegromab to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), which was accepted under priority review.

Financially, Scholar Rock reported a net loss of $107.2 million for the quarter ended September 30, 2026, compared to a net loss of $102.2 million in the prior year period. Research and development expenses totaled $54.1 million for the quarter, while selling, general, and administrative expenses were $52.5 million. As of September 30, 2026, the company held cash, cash equivalents, and marketable securities totaling $413.6 million.