Pfizer terminates domagrozumab clinical studies for the treatment of Duchenne

Pfizer announced that it is terminating two ongoing clinical studies evaluating domagrozumab for the treatment of Duchenne muscular dystrophy: a Phase 2 safety and efficacy study and an open-label extension study. The Phase 2 study, did not meet its primary efficacy endpoint, which was to demonstrate a difference in the mean change from baseline in 4 Stair Climb following one year of treatment with domagrozumab as compared to placebo in patients with DMD. Further evaluation of the totality of evidence including secondary endpoints did not support a significant treatment effect. The decision comes after a thorough review of data available at the time of the primary analysis, which evaluated all study participants after one year of treatment, as well as those participants who were in the trial beyond one year. The studies were not terminated for safety reasons. Pfizer will continue to review the data to better understand any insights they may provide, and will share results with the scientific and patient community. Pfizer is continuing research in DMD and rare neuromuscular diseases, with the goal of bringing therapies to patients with unmet needs. The company’s continued partnership with advocacy associations and the community is critical to finding innovative therapies for these diseases. Pfizer has one ongoing clinical trial in DMD with a gene therapy, PF-06939926, which is an investigational, recombinant AAV9 capsid carrying a truncated or shortened version of the human dystrophin gene under the control of a human muscle specific promotor. The Phase 2 double-blind, placebo-controlled, multicenter clinical trial investigated the efficacy and safety of domagrozumab, administered in monthly IV doses, in 121 boys aged 6 to 15 with DMD, regardless of underlying mutation. It was designed as a two-year, placebo-controlled study; all subjects used background corticosteroid therapy. The open-label extension study was designed to evaluate long-term safety and efficacy of domagrozumab.
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