Pfizer’s phase 3 Duchenne muscular dystrophy (DMD) gene therapy failure has blown a $230 million hole in the New York pharma's second quarter financials (PDF). The drugmaker disclosed the discontinuation of the candidate—and the financial fallout—alongside the axing of a respiratory syncytial virus (RSV) combination vaccine.
The Big Pharma reported the failure of a phase 3 clinical trial for the DMD gene therapy fordadistrogene movaparvovec in June. At that time, Pfizer was still evaluating the next steps for the program. Six weeks later, the company has confirmed there will be no significant next steps, jettisoning the candidate from its pipeline and taking a $230 million charge in the process.
Pfizer’s decision brings a troubled program to an end. One month before the phase 3 fail, Pfizer paused dosing in the crossover portion of the phase 3 trial after a young boy in another study of the candidate died. The company also laid off 150 workers in North Carolina due to the gene therapy's failure.
Sarepta Therapeutics’ DMD gene therapy Elevidys has also suffered setbacks, notably when it failed to hit the primary goal of a pivotal study, but the biotech has continued to grow sales and push to reach more patients. The FDA expanded Elevidys’ label in June.
Pfizer disclosed the discontinuation of the DMD gene therapy alongside news that it is stopping work on a phase 2 respiratory vaccine combination. The candidate, PF-07960613, combined vaccines designed to protect against RSV and COVID-19.
No candidate by that name is listed on ClinicalTrials.gov but the database does include a Pfizer phase 2 trial of a combined vaccine for RSV and COVID-19. The study, which ran from June 2023 to the start of 2024, enrolled more than 1,000 people aged 65 years and older to receive vaccine regimens including a combination of RSVpreF and bivalent BNT162b2 on top of a quadrivalent influenza shot.
RSVpreF is the RSV protein subunit vaccine that Pfizer sells as Abrysvo. Bivalent BNT162b2 is an updated version of Pfizer’s COVID-19 vaccine Comirnaty. The study assessed the rates of local reactions, systemic events and adverse events in people who received various combinations of the vaccines and placebo. Pfizer also looked at immune responses.