Pfizer provides update on investigational gene therapy phase 3 study for ambulatory boys with Duchenne muscular dystrophy

NEW YORK, June 12, 2024–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) announced today that CIFFREO, a global Phase 3, multicenter, randomized, double-blind, placebo-controlled study evaluating the investigational mini-dystrophin gene therapy, fordadystrogen movaparvovec, in outpatients with dystrophy Duchenne muscular dystrophy (DMD) did not meet its primary endpoint of improvement in motor function among 4- to 7-year-old boys treated with gene therapy compared with placebo. The primary endpoint in the final analysis was assessed by the change in the North Star Ambulatory Assessment (NSAA) one year after treatment. Secondary endpoints, including 10-meter run/walk speed and time to rise from floor speed, also showed no significant difference between participants treated with 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 serious treatment-related 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 results from the study at future medical and patient advocacy meetings, with the goal of ensuring that learning from this trial can help inform future clinical research and develop treatment options that can improve care for boys living with Duchenne muscular dystrophy,” said Dan Levy, MD, PhD, Development Lead for Duchenne muscular dystrophy, Pfizer . “We are grateful to the boys, their families, advocates and investigators who participated in this research and ongoing efforts to advance treatment options for this debilitating disease.”

Pfizer will continue to closely monitor all participants enrolled in the study and is evaluating appropriate next steps for 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 fordadistrogen movaparvovec in ambulatory male participants, aged 4 to 7 years, with a diagnosis genetic DMD who are on a stable daily regimen of glucocorticoids. 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 a dosing pause due to a fatal serious adverse event in the Phase 2 DAYLIGHT trial (NCT05429372). DAYLIGHT is a study evaluating the safety and tolerability of fordadystrogen movaparvovec in participants aged 2 to 3 years with DMD. Pfizer is actively working to gather additional information on the event to understand the possible cause.

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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 the ages of 3 and 5. The disease mainly affects boys. Muscle weakness can begin as early as age 3, first affecting the muscles of the hips, pelvic area, thighs, and shoulders, and later the skeletal (voluntary) muscles of the arms, legs, and trunk. In early adolescence, patients usually lose their ability to walk and the heart and respiratory muscles are also affected, resulting in premature death. DMD is the most common form of muscular dystrophy worldwide with an incidence of 1 in every 5,000 live male births.¹

About Pfizer: Advances that change patients’ lives

At Pfizer, we apply our science and global resources to bring therapies to people that significantly extend and improve their lives. We strive to set the standards for quality, safety and value in the discovery, development and manufacture of healthcare products, including innovative medicines and vaccines. Every day, Pfizer colleagues work in developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. In line with our responsibility as one of the world’s leading innovative biopharmaceutical companies, we partner with healthcare providers, governments and local communities to support and expand access to reliable and affordable healthcare worldwide. For 175 years, we’ve worked to make a difference for everyone who relies on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. Additionally, to learn more, please visit us at www.Pfizer.com and follow us at X at @Pfizer AND @Pfizer NewsLinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure notice

The information contained in this release is as of June 12, 2024. Pfizer undertakes no obligation to update the forward-looking statements contained in this release as a result of new information or future events or developments.

This release contains forward-looking information about fordadystrogene movaparvovec, an investigational mini-dystrophy gene therapy for Duchenne muscular dystrophy, key results from the Phase 3 CIFFREO study, and plans to share more detailed results from the study at future medical defense meetings and patients that involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by these statements. Risks and uncertainties include, among others, uncertainties inherent in research and development; uncertainties related to further analysis of data from the Phase 3 CIFFREO study, including sharing more detailed results from the study at future medical 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 further 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, and in its subsequent reports on Form 10-Q, including in sections entitled “Risk Factors”. and “Information and Factors That May Affect the Future,” as well as in its subsequent reports on Form 8-K, all of which have been filed with the U.S. Securities and Exchange Commission and are 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.

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