Scholar Rock Secures $205 Million Voucher Deal To Fund Its Move Into Drug Sales

Scholar Rock, which markets the muscle-targeted spinal muscular atrophy drug ISEMBYLD, agreed to sell an FDA review voucher for $205 million in cash without issuing shares. The agreement with BioNTech US was signed Monday and reported in Tuesday’s SEC filing; payment is due when the sale closes.

Cash Beyond The First Shipments

The pending sale adds a financing source as product revenue begins. Scholar Rock ended September with $413.6 million in cash and investments, against $367.6 million at the end of December 2025, according to its Tuesday earnings release.

Another $150 million is available to borrow through its existing debt agreement, the company said in its earnings presentation. That capacity is separate from money already held and the voucher proceeds still to come.

ISEMBYLD’s initial U.S. inventory shipments produced $0.6 million in third-quarter revenue, compared with none a year earlier. The quarterly net loss widened to $107.2 million from $102.2 million, including stock-based pay of $17.9 million and $18.3 million, respectively. Loss per share narrowed to $0.81 from $0.90.

Research spending reached $54.1 million, versus $50.5 million a year earlier, while selling and administrative costs fell to $52.5 million from $53.1 million.

Prescriptions Are Still Becoming Infusions

ISEMBYLD received FDA approval on September 11, 2026, for patients aged two and older already receiving an SMN2-targeted treatment. It targets muscle rather than replacing that existing therapy. The approval also earned Scholar Rock the voucher it now plans to sell.

The first patients received infusions in October. By October 31, more than one-third of the top 140 U.S. SMA treatment centers had enrolled initial patients. Scholar Rock said moving from prescription to infusion can take more than 60 to 90 days on average at this early launch stage, with insurance authorization and hospital approvals among the steps involved.

Scholar Rock Chairman and CEO David L. Hallal pointed to the “breadth of early demand across SMA treatment centers, physicians, and patients.”

The company applied in September for a drug-specific insurance billing code, expected to become available April 1, 2027.

Europe, Japan And More Muscle Drugs

Europe’s regulators validated the resubmitted apitegromab approval application in October. Scholar Rock replaced the Catalent Indiana fill-finish facility in that application with the alternate facility used for U.S. approval, and expects a European regulatory committee opinion in the first half of 2027.

Japan accepted its application for priority review and granted orphan-drug designation. Scholar Rock noted that Japan’s Pharmaceuticals and Medical Devices Agency has typically targeted approximately nine months for priority reviews.

The company is also discussing a self- or caregiver-administered injection formulation of apitegromab with regulators. Enrollment and dosing continue in a mid-stage study for SMA patients under two and in a separate study for facioscapulohumeral muscular dystrophy, a muscle-wasting disease.

The latter trial is expected to enroll approximately 60 patients, comparing apitegromab with placebo over 52 weeks. Its main measure is the change in lean muscle volume at 12 months.

Scholar Rock expects initial results from its early-stage SRK-439 study in healthy volunteers by year-end 2026, with public disclosure planned for early 2027.

Sources

• 8-K Filing — Scholar Rock Holding Corp (SRRK) — SEC EDGAR
• Exhibit srrk-ex99_1.htm — Scholar Rock Holding Corp 8-K exhibit — SEC EDGAR
• Exhibit srrk-ex99_2.htm — Scholar Rock Holding Corp 8-K exhibit — SEC EDGAR
• Scholar Rock Reports Third Quarter 2026 Financial Results and Recent Business Highlights — businesswire.com
• 8-K filed 2026-09-14 — Scholar Rock Holding Corp (earlier disclosure) — SEC EDGAR