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Pfizer Inc. (NYSE:PFE) shares are ticking lower after the company disclosed disappointing results from its Phase 3 CIFFREO study, evaluating the investigational gene therapy for Duchenne muscular dystrophy (DMD) in young boys.
The study did not achieve its primary endpoint of improving motor function in patients aged 4 to 7 years.
The multinational, placebo-controlled trial assessed the efficacy of the gene therapy using the North Star Ambulatory Assessment (NSAA) over one year.
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Despite high hopes, both the primary and key secondary outcomes, such as 10-meter run/walk velocity, showed no significant improvement over placebo.
The trial aimed to introduce a shortened version of the human dystrophin gene, which is absent in DMD patients, into their cells, reported Reuters.
Duchenne muscular dystrophy is a devastating genetic condition causing progressive muscle degeneration and weakness, primarily affecting boys. It manifests in early childhood and leads to severe physical impairments and life expectancy reduction.
Pfizer remains committed to the ongoing monitoring of all participants and is currently reassessing the next steps for its DMD program.
This setback follows a recent serious adverse event that paused dosing in another related trial, underscoring the challenges in developing treatments for this severe genetic disorder.
This trial outcome adds to Pfizer’s challenges, as the company seeks new revenue streams following the decline in sales from its COVID-19 products.
The failure of this therapy, intended as a one-time treatment, may impact Pfizer’s financial forecasts and ongoing research priorities.
Price Action: PFE shares are trading lower by 0.43% at $27.54 in premarket at the last check Thursday.
Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editors.
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