Pfizer plans to restart Phase 3 clinical trial of DMD gene therapy!

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In December, Pfizer announced that a patient died in a bedridden cohort of a Phase Ib clinical trial evaluating the experimental gene therapy PF-06939926 in Duchenne muscular dystrophy (DMD)

According to a statement released at the time, Pfizer was working with an independent external data monitoring committee to review the data to understand exactly what happened and has notified the FDA

However, Pfizer expects to reopen patient enrollment in Phase 3 clinical trials soon

In a conference call Tuesday, Pfizer Chief Scientific Officer Mikael Dolsten said the patient was a 16-year-old boy with advanced DMD who experienced hypovolemia and cardiogenic shock (the inability of the heart to pump enough blood)

Mikael Dolsten said, "We are investigating what caused this

However, sirolimus was not used in a phase 3 clinical study that Pfizer hopes to restart soon, which enrolled ambulatory patients with DMD

However, Pfizer has previously requested that patients with certain genetic mutations that could put patients at a higher risk of heart inflammation are excluded from the trial

PF-06939926 is an experimental recombinant adeno-associated virus serotype 9 (rAAV9) capsid that delivers a shortened version of the human dystrophin gene to patients

The safety of gene therapy has come under scrutiny over the past year because of unexpected or more severe side effects in clinical trials for multiple different diseases

In addition to Pfizer's gene therapy, Sarepta is advancing a gene therapy for DMD and is enrolling patients in a Phase 3 study that could provide results by 2023

Reference article: Pfizer aims to restart late-stage trial of Duchenne gene therapy following safety setback