Scholar Rock Expects Most Early ISEMBYLD Patients To Wait Until 2027 For Infusions
Insurance reviews and hospital approvals are slowing treatment starts, while management expects steady additions rather than a sales burst.
Scholar Rock Holding, which develops antibody treatments for rare neuromuscular diseases, expects most ISEMBYLD patients enrolled through October to begin infusions in 2027, Chairman and Chief Executive Officer David Hallal said on the company's earnings call Tuesday. The treatment won FDA approval on September 11, 2026, but insurance and hospital access processes are slowing the path from prescription to treatment.
Prescriptions Face A Long Wait
Chief Operating Officer Keith Woods said prescription-to-infusion waits can exceed 60 to 90 days. Insurers must review authorization requests and appeals, hospitals must approve the drug for use, and billing currently requires a code that is not specific to ISEMBYLD.
Hospital approval alone does not complete the process. Some centers have approved the medicine but still need to add it to their ordering systems; others will not consider it until next year. Woods called the waiting period "not outside of industry norm" and expects it to shorten as coverage policies develop.
Anthem and Centene's initial policies cover 47 million people and align with the approved label, Woods said. He expects more label-aligned policies over the next six months. Individual patients have also secured approvals from commercial and government plans that have yet to publish policies.
Through October, 98% of patients with prescriptions were using Scholar Rock Supports, the company's program for helping patients obtain coverage and begin treatment. Hallal described the obstacles as "typical speed bumps, no stop signs" and said management remains confident eligible patients will ultimately obtain coverage.
Steady Enrollments, Not A Sales Burst
A Bank of America analyst asked whether delayed starts would produce a concentrated sales surge in the first two quarters of 2027. Hallal instead forecast steady patient additions, pointing to consistent weekly enrollments.
When a UBS analyst requested enrollment totals for forecasting, Hallal replied, "we're not quantifying that." He said patients who had never received ISEMBYLD outnumbered those beginning to transition from clinical trials and early access.
More than a third of the top 140 U.S. spinal muscular atrophy treatment centers had enrolled initial patients through October, with additional enrollments outside that group. Children were the largest group, but adults also represented a substantial proportion. Enrollees included people whose motor function was improving, plateauing or declining.
ISEMBYLD targets muscle rather than motor-neuron health. Its approval covers adults and children aged two or older already receiving an SMN2-targeted therapy, which addresses motor neurons. In the pivotal trial, patients receiving the combination gained motor function at one year, while those receiving the background treatment alone lost function.
The first commercial infusions began in October. Third-quarter revenue of $0.6 million came from initial inventory shipments to the specialty network, rather than those treatment starts; the company recorded no revenue a year earlier.
A $205 Million Funding Bridge
Scholar Rock agreed to sell its priority review voucher for $205 million, with payment due when the transaction closes. Hallal said the proceeds would support commercial and research expansion without an equity offering at this time.
The company ended September with $413.6 million in cash, cash equivalents and marketable securities. Chief Financial Officer Vikas Sinha said approval also unlocked access to an additional $150 million under its existing debt facility. The third-quarter net loss was $107.2 million, compared with $102.2 million a year earlier.
Beyond the U.S., Scholar Rock expects a European regulatory committee opinion in the first half of 2027 and is preparing for a German launch later that year, pending approval. Japan accepted its application for priority review; management anticipates a potential launch near year-end 2027.
Woods expects an ISEMBYLD-specific insurance billing code on April 1, 2027. He cautioned that insurer authorization requirements will still be part of getting patients onto treatment.
Sources
- Scholar Rock Holding results release (Form 8-K, EX-99.1) — SEC EDGAR
- BioNTech to buy Scholar Rock PRV for $205M (BNTX:NASDAQ) | Seeking Alpha — seekingalpha.com
- Scholar Rock Holding Corporation (SRRK) Reports Q3 Loss, Beats Revenue Estimates — Yahoo Finance
- Scholar Rock Reports Third Quarter 2026 Financial Results and Recent Business Highlights — businesswire.com
