Loading...
•
Sep 30, 2024
Scholar Rock Q3 2024 Earnings Report
Scholar Rock reported positive topline data from pivotal Phase 3 SAPPHIRE trial and completed $345 million public offering.
Key Takeaways
Scholar Rock reported positive topline data from the Phase 3 SAPPHIRE trial of apitegromab in SMA patients and completed enrollment in the Phase 2 EMBRAZE trial for apitegromab in obesity. The company also completed an upsized $345 million public offering. Net loss for the quarter was $64.5 million, or $0.66 per share.
Reported positive topline data from pivotal Phase 3 SAPPHIRE trial evaluating apitegromab in patients with Spinal Muscular Atrophy (SMA), achieving primary endpoint.
On track to submit a U.S. Biologics License Application (BLA) and European Union marketing authorisation application (MAA) in 1Q 2025.
Phase 2 EMBRAZE trial enrollment completed for apitegromab in obesity with topline data expected in 2Q 2025.
Successful completion of upsized $345 million public offering to fund planned commercial launch in SMA and continue to advance priority programs.
Scholar Rock
Scholar Rock
Forward Guidance
Scholar Rock is advancing towards its first commercial launch in Q4 2025, aiming to obtain approval for apitegromab and deliver it to patients with SMA, while also progressing its portfolio of myostatin inhibition programs.
Positive Outlook
- Plans to submit a BLA and MAA in the first quarter of 2025 based on positive SAPPHIRE trial results.
- Preparing for the commercial launch of apitegromab to deliver transformative benefits to children and adults living with SMA, if approved.
- Expects to report initial data from the fully enrolled Phase 2 EMBRAZE trial in the second quarter of next year.
- Plans to file an IND for SRK-439 for the treatment of obesity in 2025.
- Advancing mission from a position of strength, supported by a recent public offering on the heels of successful SAPPHIRE data.
Challenges Ahead
- The efficacy and safety of apitegromab, SRK-181, and SRK-439 have not been established.
- Apitegromab, SRK-181, and SRK-439 have not been approved for any use by the FDA or any other regulatory agency.
- Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially.
- Dependence on third parties for development and manufacture of product candidates, including to supply any clinical trials.
- Ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives.