Scholar Rock’s experimental drug, apitegromab, has shown promising results in the treatment of spinal muscular atrophy (SMA), a rare disease characterized by progressive muscle weakness. The drug, which targets myostatin to inhibit muscle growth, demonstrated significant improvement in muscle function in patients with SMA during a Phase 3 study. Scholar Rock plans to seek regulatory approval for apitegromab in the US and Europe, with a potential commercial launch in 2025. The company raised $300 million through a stock offering following positive study results, allowing for further development and commercialization of the drug. Scholar Rock aims to address the unmet needs in SMA treatment with its unique approach.
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Scholar Rock Scores $300M for Regulatory Filings & Launch Plans for Neuromuscular Disease Drug
