Pfizer Says New Data in Duchenne Muscular Dystrophy Support Advancement Into Phase 3 Study
May 15 2020 - 8:56AM
Dow Jones News
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 michael.dabaie@wsj.com
(END) Dow Jones Newswires
May 15, 2020 08:41 ET (12:41 GMT)
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