Pfizer halts Duchenne trials

Aug. 30, 2018

Pfizer is ending two ongoing clinical studies for a Duchenne muscular dystrophy drug after determining that the evidence "did not support a significant treatment effect.”

The two studies — a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004) — were evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy.

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 (with the primary analysis after one year); all subjects used background corticosteroid therapy. The open-label extension study was designed to evaluate long-term safety and efficacy of domagrozumab.

Pfizer says it will continue its research in DMD and rare neuromuscular diseases, with the goal of bringing therapies to patients with unmet needs.

Read the press release