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Pfizer: DMD Gene Therapy Continues to Show Promise

May 15, 2020

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 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.

About Pfizer

Pfizer Canada is the Canadian operation of Pfizer Inc., one of the world’s leading biopharmaceutical companies.

We strive to profoundly impact the health of Canadians through the discovery, development and delivery of medicines and vaccines. Our diverse portfolio offers a broad range of benefits and solutions to meet the needs of Canadians.

Pfizer’s innovative portfolio focuses on the discovery and development of new medicines and vaccines. By focusing on the best science and patient experience, Pfizer’s leadership and significant investments support faster delivery of breakthrough medicines that can fulfill unmet needs.

Pfizer also offers a comprehensive portfolio of legacy and multisource medicines that deliver quality care and sustainable solutions to the Canadian healthcare system.

At Pfizer, we operate with integrity and abide by the most stringent code of ethics and compliance. We support the Canadian economy and are committed to giving back to the communities where we work and live.

Ultimately, our goal is to ensure that all Canadians have timely access to best-in-class treatments and quality health care.