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Pfizer Doses First Patient Using Investigational Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

PPMD is thrilled to learn that Pfizer Inc. has initiated a Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in Duchenne, with the first boy receiving an infusion of the mini-dystrophin gene on March 22, 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 is expected to 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. Read the full release and Pfizer’s letter to the community below.


Read Pfizer's Press Release:

Pfizer Inc. has initiated a Phase 1b clinical trial for its mini-dystrophin gene therapy candidate, PF-06939926, in boys with Duchenne muscular dystrophy (DMD). The first boy received an infusion of the mini-dystrophin gene on March 22nd, 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 is expected to 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-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 will enroll approximately 12 ambulatory boys aged 5 to 12 years with DMD. In addition to evaluating safety and tolerability, the study will evaluate 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 (AAV9) capsid and for a T-cell (immune) response to dystrophin.

“Investment in this trial represents the culmination of years of research on behalf of patients by scientists at Pfizer and academic medical centers, along with the support of the DMD patient advocacy community, in the important quest to advance a program that could potentially change the trajectory of this debilitating disease,” said Greg LaRosa, PhD, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Disease Research Unit. “We’ve listened to the patient community and we know there is a dire need for treatment options; with this in mind, we have built on important scientific advances to design a therapy with the potential to deliver the mini-dystrophin gene to the body and address the underlying cause of DMD, regardless of mutation. This trial will assess the safety of this approach to gene therapy and could provide valuable data demonstrating its potential impact to slow down or stop the progression of DMD.”

Click here to view the full press release.

Read Pfizer's Letter to the Community:

Pfizer is pleased to provide the following update regarding the Phase 1b, Multicenter, Open-label, Single Ascending Dose Study to Evaluate the Safety and Tolerability of PF-06939926 in Ambulatory Boys with Duchenne Muscular Dystrophy (DMD).  The first boy received an infusion of the mini-dystrophin gene on March 22nd, administered under the supervision of the principal investigator at the study site.  This first participant continues to be monitored.

 

The study will enroll approximately 12 ambulatory boys aged 5 to 12 years with DMD.  In addition to evaluating safety and tolerability, the study will evaluate measurements of dystrophin expression and distribution, as well as assessments of muscle strength, quality, and function. As part of the screening process, potential candidates invited by the study principal investigator will be tested to confirm a negative result for antibodies against the adeno-associated virus, serotype 9 (AAV9) capsid and for a T-cell (immune) response to dystrophin. Screening and enrollment of patients is expected to 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 the first patient completes one full year post-treatment. More information about the trial and participating sites may be found at www.clinicaltrials.gov (NCT03362502). 

 

We recognize the commitment that the DMD community has shown for this study and for Pfizer’s research and development efforts related thereto.   We further recognize the potential impact innovative therapies, like gene therapy, may have in transforming the lives of individuals and families affected by DMD and we share with you that hope and vision.  It is with heartfelt gratitude that we extend our sincerest ‘thank you’ to all the boys and families who continue to express interest in and who participate in clinical research.  We are furthermore indebted to the advocacy associations and advocates who provide the tools and support needed for families to engage in clinical research, who continue to care for the Duchenne community and who lend their expertise in the research and development process. 

 

Additional public-facing information about this milestone achievement can be found at: https://www.pfizer.com/news/press-release/press-release-detail/pfiz....

Kind regards,

 

Katherine Beaverson

Patient Advocacy Lead

 

Cc:       Tara Moorehead

Michael Binks, MD

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