Pfizer (PFE - Free Report) announced updated efficacy and safety data from an early-stage study on its investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD), a rare muscular degenerative disease. Pfizer said that the data supports the advancement of the candidate, PF-06939926, into a pivotal phase III study.
Duchenne muscular dystrophy is a rare genetic disease and a severe form of muscular dystrophy, primarily affecting males. It progressively weakens and degenerates skeletal and heart muscles. DMD is caused by the absence of a particular gene, dystrophin, which the body uses to keep the muscle cells intact.
Preliminary safety data from the phase Ib study on nine ambulatory boys (aged 6 to 12) with DMD showed that treatment with PF-06939926 led to durable and statistically significant improvements across multiple efficacy-related endpoints measured at 12 months post-infusion. This included sustained levels of mini-dystrophin expression and improvements in muscle function measured by the North Star Ambulatory Assessment (NSAA) rating scale. The three patients who received PF-06939926 at the low dose had an average normal dystrophin level of 24% in muscle biopsies taken 12 months after treatment with PF-06939926. However, the three patients administered the higher dose demonstrated a much higher average of 51.6% normal dystrophin. Meanwhile, Pfizer reported a median NSAA 3.5 point improvement from baseline.
While three severe serious adverse events (SAEs) were recorded in the study, Pfizer said that all of them were fully resolved within two weeks. However, the safety issues seen with PF-06939926 raised some investor concern.
The data was presented at the American Society of Cell and Gene Therapy virtual 2020 annual meeting
Pfizer plans to begin dosing patients in the phase III study in the second half of 2020 if it gets the necessary FDA approval.
Pfizer’s shares have declined 3.6% this year so far compared with a decrease of 1% for the industry.
Sarepta Therapeutics, Inc.'s (SRPT - Free Report) Exondys 51 and Vyondys 53 and PTC Therapeutics, Inc.’s (PTCT - Free Report) Emflaza are among the approved treatments of DMD in the United States. However, the focus for DMD treatment is currently shifting to development of gene therapies. Gene therapies deliver a functional copy of the dystrophin to muscle cells to restore its production.
Sarepta is also conducting studies to evaluate gene therapies for DMD. Its lead gene therapy candidate, SRP-9001, an AAV-mediated micro-dystrophin gene therapy candidate, is in a phase I/II study for treating DMD. In fact, Sarepta’s stock rose more than 11% on Friday, as some analysts believed that benefits of PF-06939926 appear lower than those seen in case of Sarepta’s SRP-9001.
Solid Biosciences Inc. (SLDB - Free Report) also has an early-stage gene therapy candidate, SGT-001, which is being developed for DMD. However, the FDA has placed a clinical hold on the IGNITE phase I/II study on SGT-001.
Pfizer currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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