Pfizer Says New Data in Duchenne Muscular Dystrophy Support Advancement Into Phase 3 Study

By Michael Dabaie

Pfizer Inc. said new Phase 1b results of a gene therapy in ambulatory boys with Duchenne muscular dystrophy support advancement into a pivotal Phase 3 study.

Pfizer said Friday it updated Phase 1b clinical data on PF-06939926, an investigational gene therapy to treat Duchenne muscular dystrophy. The preliminary data from 9 ambulatory boys with DMD, aged 6 to 12, indicate the intravenous administration of PF-06939926 was well-tolerated during the infusion period, with encouraging efficacy and manageable safety events, even when considering adverse events that were more severe in nature, the company said.

The treatment provided durable and statistically significant improvements across multiple efficacy-related endpoints measured at 12 months post-infusion, Pfizer said.

Three serious adverse events were recorded, two of which reflected likely complement activation. While these two SAEs were severe in nature, all three events fully resolved within 2 weeks, the company said.

"Based on the encouraging preliminary efficacy data and manageable safety events from our Phase 1b study, we believe we may have a potential breakthrough therapy for boys with Duchenne muscular dystrophy, a devastating disease for which there remains a significant medical need, " said Seng Cheng, chief scientific officer of Pfizer Rare Disease Research Unit.

The company said it is advancing its Phase 3 program as quickly as possible and plan to begin dosing patients in the second half of 2020 pending regulatory approval.

Write to Michael Dabaie at [e-mail]

(END) Dow Jones Newswires

May 15, 2020 08:41 ET (12:41 GMT)

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