Dawn of a New Era: Scholar Rock Poised for Breakthrough with Apitegromab

Scholar Rock is on the cusp of becoming a commercial-stage entity, with its lead drug apitegromab for Spinal Muscular Atrophy (SMA) under Priority Review by the FDA.
The company has strengthened its leadership team in preparation for a potential U.S. launch of apitegromab in Q3 2025 and expansion into Europe.
Apitegromab, a novel muscle-targeted therapy, demonstrated significant clinical benefits in its pivotal Phase 3 SAPPHIRE trial for SMA patients.

CAMBRIDGE, Mass. – A wave of transformation is sweeping through Scholar Rock (NASDAQ: SRRK) as it stands at a pivotal "inflection point," gearing up to potentially launch its groundbreaking therapy, apitegromab, for patients battling Spinal Muscular Atrophy (SMA)⁶. The air is thick with anticipation as the company focuses on a U.S. debut in Q3 2025, a milestone that could redefine treatment for this debilitating neuromuscular disease.

The U.S. Food and Drug Administration (FDA) has recognized the urgency, granting apitegromab Priority Review for its Biologics License Application (BLA), setting a PDUFA target action date of September 22, 2025^{5, 6}. Simultaneously, European regulators are assessing its Marketing Authorisation Application (MAA)⁶. This dual regulatory momentum underscores the promise of apitegromab, an investigational monoclonal antibody that uniquely targets myostatin to build muscle. It stands as the only muscle-targeted candidate for SMA to achieve statistically significant and clinically meaningful improvements in a pivotal Phase 3 trial (SAPPHIRE)^{3, 4}.

"Scholar Rock is at an inflection point as we evolve to a commercial-stage biopharmaceutical company," declared David L. Hallal, Chief Executive Officer. To spearhead this evolution, the company has welcomed Akshay Vaishnaw, Keith Woods, and Vikas Sinha to its leadership, fortifying its operational might to serve patients globally⁶.

Beyond SMA, Scholar Rock's innovative science extends to other high-need areas. Apitegromab is also being explored in a Phase II trial for obesity, aiming to preserve muscle mass during weight loss, with topline results eagerly awaited mid-2025^{6, 7}. Further fueling the pipeline is SRK-439, a preclinical candidate with potential to potently inhibit myostatin [].

While navigating this transformative period, Scholar Rock reported a net loss for Q1 2025, reflecting its investment in these pioneering efforts. However, with $364.4 million in cash, cash equivalents, and marketable securities, the company is well-funded into 2027 to pursue its mission⁶.

Scholar Rock, a leader in TGFβ superfamily biology, continues its decade-long commitment to developing life-changing therapies. As they harness cutting-edge science, the future for patients with severe diseases looks brighter. Learn more about their approach on the Scholar Rock website and follow @ScholarRock on X and LinkedIn.

Dawn of a New Era: Scholar Rock Poised for Breakthrough with Apitegromab

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