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Scholar Rock (NASDAQ:SRRK) shares continued to tumble for the second straight session on Thursday in a move attributed to the decision by the FDA to delay its decision on SRP-9001, a gene therapy developed by Sarepta Therapeutics (SRPT) for Duchenne muscular dystrophy (DMD).
After Sarepta (SRPT) disclosed that the agency is considering a potential accelerated approval for SRP-9001 targeting a small subset of patients with DMD, Truist analyst Srikripa Devarakonda opined that similar concerns might have weighed on SRRK shares on Wednesday.
Cambridge, Massachusetts-based Scholar Rock (SRRK) is aiming for a potential commercial rollout for its lead candidate, apitegromab, for the motor neuron disorder spinal muscular atrophy (SMA) in 2025.
Devarakonda recalls results from a ≤5-year-old cohort in an earlier SRRK study that "appeared to drive the majority of the benefit," which, she says, have fueled concerns over an age-restricted label.
The analyst also highlights the differences between the SRPT's one-time gene therapy and apitegromab, a monoclonal antibody that can be discontinued or combined with other treatments if required.
Apitegromab is currently undergoing a pivotal Phase 3 clinical trial in certain patients with SMA; a topline readout from the trial is expected in 2024.