PER 0.00% 8.0¢ percheron therapeutics limited

Ann: Trial Met Primary & Exceeded Secondary Endpoint Expectations, page-205

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    (RTTNews) - 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.


    How can Pfizer can go from a Phase B1 to a Phase 3

    Does not impede on ATL1102 re DMD inflammation..
 
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