Scholar Rock’s Optimistic Earnings Call Highlights Progress

Scholar Rock Holding ((SRRK)) has held its Q2 earnings call. Read on for the main highlights of the call.

The recent earnings call for Scholar Rock Holding conveyed a sentiment of cautious optimism. The company highlighted successful clinical trial results and strong regulatory progress, particularly for its lead product, apitegromab. The readiness for a commercial launch further bolstered the positive outlook. However, some challenges were noted, specifically observations at CDMO sites by the FDA, which require attention.

Regulatory Progress for Apitegromab

Scholar Rock announced that the Biologics License Application (BLA) for apitegromab has been accepted under priority review by the FDA, with a target action date set for September 22, 2025. Additionally, the company is working towards potential approval in Europe by mid-2026, marking significant regulatory milestones for the company.

Positive Clinical Trial Results

The company shared encouraging results from its clinical trials, notably the SAPPHIRE trial, which demonstrated statistically significant improvements in motor function for SMA patients. The Phase II EMBRAZE study also met its primary endpoint, showing a 54% greater lean mass preservation, alongside a favorable safety profile.

Commercial Launch Preparations

Scholar Rock is gearing up for the commercial launch of apitegromab in the U.S., with a fully trained customer-facing team ready to go post-FDA approval. Preparations for a European launch are also in progress, targeting 2026 as the launch year.

Financial Position and Runway

The company reported a strong financial position, ending the quarter with $295 million in cash, which is expected to sustain operations into 2027. Scholar Rock also has additional funding options, including a $50 million debt facility and an expected $16 million from warrant exercises.

CDMO Observations by FDA

During site inspections, the FDA noted observations at two Contract Development and Manufacturing Organizations (CDMOs), which could potentially impact the approval timeline for apitegromab. Scholar Rock is actively addressing these observations by submitting responses to the FDA.

Forward-Looking Guidance

Scholar Rock’s forward-looking guidance emphasizes three core priorities: achieving regulatory approvals for apitegromab, expanding its application into additional rare neuromuscular diseases, and maintaining disciplined capital allocation. The company is on track for the U.S. commercial launch, with a priority review and target action date of September 22, 2025, and plans for a European launch starting in Germany in 2026. The company is also preparing to initiate the Phase II OPAL Trial in young children and explore new indications for apitegromab by the end of 2025.

In conclusion, Scholar Rock’s earnings call highlighted a cautiously optimistic outlook, driven by positive clinical and regulatory developments for apitegromab. The company’s robust financial position and strategic preparations for commercial launch underscore its readiness to capitalize on these advancements, despite facing some regulatory challenges.