Pfizer treats first patient in trial for investigational mini-dystrophin gene therapy for the treatment of Duchenne muscular dystrophy
Pfizer has stated that they have begun their Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in patients with Duchenne muscular dystrophy (DMD). The first patient received an infusion of the mini-dystrophin gene, administered under the supervision of principal investigator, Edward Smith, MD, Associate Professor of Pediatrics and Neurology at Duke University Medical Center. Screening and enrollment of patients will continue at up to four clinical research sites in the United States. Early data from this trial are expected in the first half of 2019, once all patients have been evaluated for one full year post-treatment.
“On behalf of the community of individuals and families living with Duchenne muscular dystrophy, we applaud the important step Pfizer has taken to advance a potentially transformational treatment option for boys stricken with this terrible disease,” said Debra Miller, CEO and Founder of Cure Duchenne. “The momentum we are seeing in the field of gene therapy emphasizes the maturing opportunity to advance the science. Today, there are very limited treatment options for our boys. Through collaboration and ongoing dialogue with companies like Pfizer, we hope to succeed in finding therapies that could dramatically change the outcomes for those with DMD.”
The multi-centre, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 will enrol approximately 12 ambulatory male patients aged 5 to 12 years with DMD. In addition to evaluating safety and tolerability, the study will assess measurements of dystrophin expression and distribution, as well as assessments of muscle strength, quality and function. As part of the screening process, potential candidates for treatment will be tested to confirm a negative result for antibodies against the adeno-associated virus serotype 9 capsid and for a T-cell response to dystrophin.
Source: World Pharma News