Here is STAT’s biotech scorecard, our regular ledger of stock-moving biotech events, for the fourth quarter:
The Food and Drug Administration’s decision in June to grant conditional approval to a gene therapy for Duchenne muscular dystrophy was a watershed moment for the treatment of the neuromuscular disease, and a big win for its maker, Sarepta Therapeutics. Confirming the gene therapy’s benefit — and potentially broadening its use to include older boys — will be determined by a Phase 3 clinical trial that will read out later this quarter.
Pfizer is developing its own gene therapy for Duchenne in a fully enrolled Phase 3 study with final results expected in 2024. But before the verdict is revealed, Pfizer intends to conduct an interim analysis later this quarter. The most likely outcome of this early look will be a determination to continue the study to its completion. But surprises — including an early stop for overwhelming efficacy — can’t be ruled out.
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