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Dec 31, 2023
Scholar Rock Q4 2023 Earnings Report
Scholar Rock reported its full year financial results and highlighted business progress.
Key Takeaways
Scholar Rock reported a net loss of $165.8 million for the full year ended December 31, 2023. The company is on track to release topline data for its Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy in the fourth quarter of 2024 and to initiate a Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 RA in mid-2024. The company ended the year with approximately $280 million in cash, cash equivalents, and marketable securities.
Pivotal Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy is on track to report topline data in 4Q 2024.
FDA clearance of IND application was announced to initiate Phase 2 proof-of-concept trial with apitegromab to treat obesity; expected to commence in mid-2024.
Preclinical data was presented supporting potential benefit of SRK-439 to preserve lean muscle mass as part of healthier weight management at Keystone Symposia.
Ended 2023 with approximately $280 million in cash, cash equivalents, and marketable securities, which is expected to fund operations into 2H 2025
Scholar Rock
Scholar Rock
Forward Guidance
Scholar Rock is focused on advancing its pipeline, with key milestones expected in the near term. The company anticipates releasing topline data from the Phase 3 SAPPHIRE trial in Q4 2024 and initiating a Phase 2 trial for apitegromab in combination with a GLP-1 RA in mid-2024. SRK-439, a novel preclinical myostatin inhibitor for obesity, is moving toward IND.
Positive Outlook
- On track to release topline data for Phase 3 SAPPHIRE trial in SMA in Q4 2024.
- Phase 2 proof-of-concept trial with apitegromab in combination with a GLP-1 RA expected to commence in mid-2024.
- SRK-439, a novel preclinical myostatin inhibitor for obesity, moving toward IND.
- Apitegromab is the only muscle-targeted therapy candidate to show clinical proof-of-concept in SMA.
- Capital raise in the fourth quarter extended cash runway.
Challenges Ahead
- Apitegromab has not been approved for any use by the US FDA or any other health authority, and its safety and efficacy have not been established.
- The efficacy and safety of SRK-439 have not been established and SRK-439 has not been approved for any use by the FDA or any other regulatory agency.
- Clinical data from earlier trials may not be predictive of results from future trials.
- Dependence on third parties for development and manufacture of product candidates.
- Competition from third parties developing products for similar uses.