Scholar Rock (NASDAQ:SRRK) reported $0.6 million in third-quarter revenue from initial shipments of its newly approved spinal muscular atrophy treatment ISEMBYLD, while executives said insurance and reimbursement hurdles are likely to push most enrolled patients’ first infusions into 2027.
The company began its U.S. launch after the Food and Drug Administration approved ISEMBYLD in September for patients ages 2 and older with spinal muscular atrophy, or SMA, who are receiving an SMN2-targeted treatment. Scholar Rock estimates that about 6,600 people in the U.S. are eligible based on their current therapy.
Chairman and CEO David Hallal said demand has been steady across a range of patients and prescribers during the first six weeks of the launch. However, the company did not disclose the number of patients who had enrolled in its support program.
Launch demand meets access delays
Through the end of October, more than one-third of the top 140 U.S. SMA treatment centers had enrolled initial patients, and a significant number of enrollments came from outside those centers, Hallal said. Patients new to ISEMBYLD outnumbered those beginning to transition from clinical trials and the expanded access program.
Children made up the largest group of enrollees, although a substantial number of adults also enrolled, according to Hallal. Chief Operating Officer Keith Woods said the group included patients with improving, stable and declining motor function.
Executives cautioned that the time from prescription to infusion can average more than 60 to 90 days early in the launch. Woods cited factors including prior authorizations, appeals of denials, hospital formulary approvals and the need to use an unspecified billing code until an ISEMBYLD-specific code becomes available April 1, 2027.
Hallal said the absence of established medical policies and the drug’s inclusion in payer systems were among the reasons for early denials. Woods expects the timeline to shorten as more policies are established, although prior authorizations will remain part of the process.
Through October, Scholar Rock had reached payers responsible for more than 90% of covered lives in the U.S., Woods said. He highlighted published policies from Anthem and Centene, which together represent 47 million covered lives, as aligned with the drug’s label. The company also reported patient-by-patient approvals from commercial and government plans that had not yet issued policies.
Asked whether a bone fracture warning had affected demand, Hallal said it had not affected demand or prescribing patterns.
Europe and Japan preparations
Scholar Rock resubmitted its European marketing application for apitegromab, the drug marketed as ISEMBYLD in the U.S., in early October. President of R&D Akshay Vaishnaw said the application includes an alternate fill-finish facility in place of Catalent Indiana, and that the review is ongoing. The company expects an opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use in the first half of 2027.
Woods said Scholar Rock is preparing for a potential launch in Germany later in 2027, followed by other European countries over time, subject to approval and reimbursement.
In Japan, the company submitted its application in early November and received priority review. Vaishnaw said Japan’s regulator typically targets an approximately nine-month review cycle for such applications. Pending approval and a subsequent pricing and reimbursement process, Scholar Rock anticipates a launch near the end of 2027.
Pipeline and finances
Scholar Rock is developing a high-concentration subcutaneous version of apitegromab and plans to update investors on its development path after discussions with U.S. and European regulators. Vaishnaw said pharmacokinetic and pharmacodynamic bridging could be a key part of the company’s regulatory case, but the final approach remains subject to those discussions.
The company is also enrolling infants and toddlers under age 2 in its Phase 2 OPAL study in SMA. Separately, dosing has begun in the Phase 2 FORGE trial testing apitegromab in facioscapulohumeral muscular dystrophy, a study expected to enroll 60 patients. For its separate myostatin inhibitor SRK-439, Scholar Rock expects Phase 1 top-line data by year-end and intends to share results in early 2027.
Third-quarter operating expenses were $106.6 million, including $17.9 million in noncash stock-based compensation, Chief Financial Officer Vikas Sinha said. The company ended the quarter with $413.6 million in cash, equivalents and marketable securities.
Scholar Rock also entered a definitive agreement in November to sell its priority review voucher for $205 million, with cash expected upon closing. Sinha said the ISEMBYLD approval also gave the company access to an additional $150 million under its existing debt facility. Management said it does not currently need to access capital markets to fund its priorities.
About Scholar Rock (NASDAQ:SRRK)
Scholar Rock is a biopharmaceutical company based in Cambridge, Massachusetts, that develops medicines targeting growth factors and signaling proteins involved in disease. Its research has focused on the transforming growth factor beta (TGF-β) superfamily, which plays roles in processes including muscle development and immune regulation. The company’s lead program has been apitegromab, a selective inhibitor of myostatin developed for spinal muscular atrophy (SMA). Scholar Rock has studied the medicine as a potential treatment alongside existing SMA therapies, with the goal of improving patients’ motor function.
