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Pfizer's Phase 1b Results On DMD Support Advancement Into Pivotal Phase 3 Study

Pfizer Inc. (PFE) said that the preliminary data from 9 ambulatory boys with Duchenne muscular dystrophy or DMD, aged 6 to 12 indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period, with encouraging efficacy and manageable safety events, even when considering those adverse events that were more severe in nature.

In an updated on Phase 1b clinical data on PF-06939926, the company said the treatment provided durable and statistically significant improvements across multiple efficacy-related endpoints measured at 12 months post-infusion, including sustained levels of mini-dystrophin expression and improvements on the North Star Ambulatory Assessment (NSAA) rating scale, which is a validated measure of muscle function.

PF-06939926 is an investigational gene therapy being developed to treat Duchenne muscular dystrophy or DMD.

The company noted that three serious adverse events were recorded, two of which reflected likely complement activation.

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

DMD is a devastating and life-threatening X-linked disease that is caused by mutations in the gene encoding dystrophin, which is needed for proper muscle membrane stability and function.

Patients present with muscle degeneration that progressively worsens with age to the extent that they require wheelchair assistance when they are in their early teens, and unfortunately, usually succumb to their disease by the time they are in their late twenties.

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