In This Article:
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Cash and Cash Equivalents: Ended the quarter with $364.4 million.
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Debt Facility: Additional $100 million available to draw down this year.
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Anticipated Financial Runway: Into 2027.
Release Date: May 14, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
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Scholar Rock Holding Corp (NASDAQ:SRRK) reported a strong start to 2025 with significant progress in bringing apitegromab to patients with SMA.
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The Phase III SAPPHIRE trial showed statistically significant and clinically meaningful improvement in motor function for SMA patients.
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The FDA granted priority review for the BLA of apitegromab, with a PDUFA date set for September 22, indicating potential for significant impact.
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Scholar Rock is preparing for a US commercial launch of apitegromab in Q3, with plans for global expansion in Europe, Asia Pacific, and Latin America.
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The company is exploring the potential of apitegromab in other neuromuscular disorders and advancing SRK-439, a new anti-myostatin antibody, to the clinic.
Negative Points
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There are concerns about the state of the FDA, although Scholar Rock reports constructive interactions regarding apitegromab's review.
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The company faces challenges in ensuring payer coverage for combination therapies targeting both SMN and muscle in SMA patients.
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The EMBRAZE study in the cardiometabolic space is exploratory, with uncertainties about the role of apitegromab in obesity treatment.
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Launch dynamics for apitegromab may face headwinds due to existing SMA treatments, despite the unmet need for muscle-targeted therapies.
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The potential impact of the President's executive order on drug pricing introduces uncertainty in pricing strategies for apitegromab.
Q & A Highlights
Q: Ahead of apitegromab launch, how are discussions with US payers regarding coverage of combination therapy for SMA patients? Also, what feedback have you received from ex-US payers? A: (David Hallal, CEO) We expect pricing to reflect the rarity and severity of SMA and the value our therapy provides. The budget impact on any single payer should be limited. (R. Woods, Independent Director) Payers recognize that SMN-targeted therapies alone may not suffice, and our early discussions have been positive. In Europe, we plan to start reimbursement discussions, beginning with Germany in 2026.
Q: Can you characterize interactions with the FDA on the apitegromab review? A: (Akshay Vaishnaw, President - R&D) Interactions with the FDA have been collaborative and routine, with no issues specific to apitegromab. We remain on track for the September 22 PDUFA date, and the FDA continues to focus on rare diseases.