On May 15, 2020, Pfizer announced positive results from their phase 1B clinical trial on PF-06939926, an investigational gene therapy under development for Duchenne muscular dystrophy.
Preliminary data for 9 boys, still walking, aged 6-12, showed encouraging benefits on a number of parameters with a manageable safety profile. There were a few serious adverse events that fully resolved and has led to the implementation of close monitoring to help mitigate. Click here to read the news release from Pfizer and learn more about this trial.
This data supports their advancement into a pivotal Phase 3 study which if successful will lead to regulatory approval. They plan to open sites in the US and internationally, but it is not clear yet if there will be sites in Canada. To learn more about their phase 3 program and how to get involved speak to your physician and/or visit www.clinicaltrials.gov and enter in (NCT04281485). Note the trial will be limited to boys aged 4-7 years.
Gene therapy is an important therapeutic approach and works by correcting the defected DNA by delivering a synthetic dystrophin gene into the muscle cells that makes a functional protein surrogate. To do this it uses a non-disease carrying virus-like AAV9 in the case of Pfizer’s approach, with a mini-dystrophin gene (DNA) and other components inserted that will replace the defective DNA and start to make functional dystrophin.
Pfizer has 1 of 3 micro or mini dystrophins currently in Clinical trial. Sarepta Therapeutics is working on their SRT-9001 and Solid Biosciences are working on their SGT-001.
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