Scholar Rock Holding Corporation ($SRRK) is a Cambridge-founded, clinical-stage biopharma built by top academic scientists around a platform that targets protein growth factors. In this episode we track their long, painstaking journey to the brink of commercialization: a pivotal Phase 3 win for their lead drug apitegromab in spinal muscular atrophy (an add-on designed to boost muscle function) that could transform them from lab-to-commercial-stage. The upside is clear—Phase 3 data de-risked the program, the therapy complements existing blockbuster SMA treatments, and the company is exploring broader muscle-preservation uses—but the path is perilous. SRRK is heavily dependent on a single asset, faces regulatory hurdles (including a prior FDA setback) and recent manufacturing problems at a third-party plant, and will need to win physician adoption and payer coverage for an add-on therapy. Is Scholar Rock a niche innovator poised to scale alongside big SMA players, or a high-stakes, single-drug gamble?

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