Scholar Rock’s third quarter marked the start of the U.S. launch of ISEMBYLD, its newly approved treatment for spinal muscular atrophy (SMA). The company also agreed to sell the FDA priority review voucher it received with the drug’s approval to BioNTech for $205 million in cash, subject to closing conditions.
The launch generated $0.6 million in revenue during the quarter, and Scholar Rock ended September with $413.6 million in cash, cash equivalents and marketable securities. The voucher-sale proceeds were not yet included in that quarter-end balance.
Key details
- ISEMBYLD is approved for adults and children aged 2 and older with SMA who are receiving an SMN2-targeted treatment. Scholar Rock describes it as the first and only muscle-targeted therapy for SMA.
- The U.S. launch began after FDA approval on September 11. Vials were shipped to the specialty network before the end of the quarter, generating $0.6 million in third-quarter revenue. The company said the first patients were infused in October and reported early demand from patients, physicians and SMA treatment centers.
- The pivotal SAPPHIRE study showed improved motor function when ISEMBYLD was added to SMN2-targeted treatment. At one year, patients receiving the combination had a clinically meaningful improvement in motor function, while those receiving SMN2-targeted treatment alone experienced a decline.
- Scholar Rock agreed to sell its priority review voucher to BioNTech US for $205 million in cash. The agreement was reached November 9, with payment due when the transaction closes. Closing remains subject to customary conditions, including the end or termination of the applicable U.S. antitrust waiting period.
- Regulatory applications are advancing outside the U.S. Scholar Rock resubmitted its European marketing application, which was validated in October; the company is on track for a European medicines committee opinion in the first half of 2027. Japan’s regulator accepted the company’s new drug application for priority review.
- The company submitted an application for an ISEMBYLD-specific billing code. The application was made to the Centers for Medicare & Medicaid Services in September, seeking a J-code for the therapy.
Plain-English implications
ISEMBYLD has moved from regulatory approval into commercialization, while the agreed voucher sale would provide Scholar Rock with a substantial additional cash inflow once completed. The $413.6 million quarter-end cash figure does not include the $205 million payment, which remains conditional on closing.