NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE: PFE) today announced that CIFFREO, a global Phase 3, multicenter, randomized, double-blind, placebo-controlled study evaluating the investigational mini-dystrophin gene therapy fordadistrogen movaparvovec, in outpatients with Duchenne muscular dystrophy (DMD) did not meet its primary endpoint of improvement in motor function in boys aged 4 to 7 years treated with gene therapy compared to placebo. The primary endpoint of the final analysis was assessed by the change in the North Star Ambulatory Assessment (NSAA) one year after treatment. Key secondary endpoints, including 10-meter run/walk speed and time to rise from ground speed, also did not show a significant difference between participants treated with the fordadistrogen movaparvovec and placebo.
The overall safety profile of fordadistrogene movaparvovec in the CIFFREO trial was manageable, with mostly mild to moderate adverse events, and treatment-related serious adverse events generally responding to clinical management.
“We are extremely disappointed that these results did not demonstrate the relative improvement in motor function that we had hoped for. We plan to share more detailed study results at upcoming medical and patient advocacy meetings, with the goal of ensuring that learnings from this trial can help improve future clinical research and development of treatment options likely to improve the care provided to boys living with Duchenne muscular dystrophy. said Dan Levy, MD, PhD, head of Duchenne muscular dystrophy development at Pfizer. “We are grateful to the boys, their families, advocates and investigators who participated in this research and the continued efforts to advance treatment options for this debilitating disease. »
Pfizer will continue to closely monitor all participants enrolled in the study and is currently evaluating appropriate next steps in the program.
About the Fordadistrogene Movaparvovec clinical program
CIFFREO is a global, phase 3, multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of the investigational gene therapy fordadistrogene movaparvovec in ambulatory male participants, aged 4 to 7 years, with a genetic diagnosis of DMD. who are on a stable daily glucocorticoid regimen. The primary endpoint of the study is a change from baseline to one year in the North Star Ambulatory Assessment (NSAA) total score. For more information, visit ciffreoduchennetrial.com or clinicaltrials.gov.
The CIFFREO study is currently on treatment pause due to a serious and fatal adverse event in the phase 2 DAYLIGHT trial (NCT05429372). DAYLIGHT is a study evaluating the safety and tolerability of the fordadistrogen movaparvovec in participants aged 2 to 3 years with DMD. Pfizer is actively working to gather additional information about the event to understand the potential cause.
About Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a serious genetic disease characterized by progressive muscle degeneration and weakness. Symptoms usually appear in early childhood, between 3 and 5 years of age. The disease mainly affects boys. Muscle weakness can begin as early as age 3, first affecting the muscles of the hips, pelvic region, thighs and shoulders, then the skeletal (voluntary) muscles of the arms, legs and trunk. By early adolescence, patients usually lose their ability to walk and the heart and respiratory muscles are also affected, ultimately leading to premature death. DMD is the most common form of muscular dystrophy worldwide, with an incidence of 1 in 5,000 live births in men.1
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Disclosure Notice
The information contained in this release is as of June 12, 2024. Pfizer undertakes no obligation to update any forward-looking statements contained in this release as a result of new information or future events or developments.
This release contains forward-looking information on fordadistrogene movaparvovec, an investigational mini-dystrophin gene therapy for Duchenne muscular dystrophy, topline results from the CIFFREO Phase 3 study and plans to share more detailed results from the study at upcoming medical and patient advocacy meetings that involve significant risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among others, uncertainties inherent in research and development; uncertainties related to further analysis of data from the CIFFREO phase 3 study, including sharing more detailed study resultsduring upcoming doctor and patient advocacy meetings; uncertainties regarding the commercial success of Pfizer’s gene therapy portfolio; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.
A more detailed description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023, as well as its subsequent reports on Form 10-Q, including in sections entitled “Risk Factors”. and “Forward-Looking Information and Factors That May Affect Future Results,” as well as in its subsequent reports on Form 8-K, all of which are filed with the United States Securities and Exchange Commission and available at www.sec.gov And www.pfizer.com.
1 Crisafulli S, Sultana J, Fontana A, Salvo F, Messina S, Trifirò G. Global epidemiology of Duchenne muscular dystrophy: an updated systematic review and meta-analysis. Orphanet J Rare Dis. 2020;15(1):141.
Source: Pfizer Inc.