Scholar Rock Holding ((SRRK)) has held its Q4 earnings call. Read on for the main highlights of the call.
Scholar Rock’s recent earnings call exuded a strong positive sentiment, underscoring significant advancements in its Spinal Muscular Atrophy (SMA) program and the commercial preparations for apitegromab. Despite these optimistic developments, the company acknowledged challenges and uncertainties in its obesity program, particularly concerning regulatory expectations and the competitive landscape.
Successful BLA Submission for Apitegromab
Scholar Rock has successfully submitted the Biologics License Application (BLA) for apitegromab in the U.S. as of January and is on track to submit the Marketing Authorization Application (MAA) in the EU by March. This milestone marks a crucial step in bringing apitegromab to market, reflecting the company’s commitment to advancing its SMA program.
Positive Results for Apitegromab in SMA
The earnings call highlighted the positive results of apitegromab combined with standard-of-care, which delivered a clinically meaningful and statistically significant improvement of 1.8 points on the Hammersmith Functional Motor scale compared to placebo. Notably, 30% of patients achieved an additional 3-point or greater improvement, showcasing the potential of apitegromab in enhancing patient outcomes.
Commercial Preparation for Apitegromab Launch
Scholar Rock is actively preparing for the potential U.S. launch of apitegromab in the fourth quarter of 2025. The company plans to scale its customer-facing team and engage with U.S. commercial and federal payers, laying the groundwork for a successful market entry.
Strong Engagement with SMA Community
In its efforts to raise awareness, Scholar Rock has introduced the ‘Life Takes Muscle’ campaign and has been actively engaging with leading CureSMA centers and neurologists. This initiative underscores the company’s dedication to supporting the SMA community and enhancing its outreach efforts.
Challenges in Obesity Drug Development
The development of SRK-439 and the EMBRAZE study faces challenges in demonstrating clinically meaningful preservation of lean muscle mass. This is compounded by a highly competitive and evolving regulatory landscape, posing significant hurdles for the obesity program.
Regulatory Uncertainty in Obesity Trials
Recent FDA guidance on obesity trials, emphasizing BMI and overall weight loss, has introduced uncertainty for programs focusing on lean muscle preservation and body composition changes. This regulatory shift presents a challenge for Scholar Rock’s obesity initiatives.
Forward-Looking Guidance
Looking ahead, Scholar Rock has provided substantial guidance for 2025, with plans to launch apitegromab for SMA in the U.S. in the fourth quarter and in the EU in 2026. The company anticipates top-line data for the EMBRAZE study in Q2 2025 and plans to file the IND for SRK-439 in Q3. These strategic developments are part of Scholar Rock’s broader ambition to establish a multibillion-dollar neuromuscular franchise.
In summary, Scholar Rock’s earnings call reflects a positive outlook, driven by significant progress in its SMA program and strategic preparations for apitegromab’s commercial launch. While challenges persist in the obesity program, the company’s forward-looking guidance and strategic initiatives underscore its commitment to advancing its pipeline and achieving long-term growth.