Pfizer Inc. (NYSE: PFE) today announced that an interim futility analysis of the global Phase 3 trial, REALM-DCM, designed to evaluate the efficacy and safety of PF-07265803 in patients with symptomatic dilated cardiomyopathy (DCM) due to a mutation of the gene encoding the lamin A/C protein (LMNA), indicated the trial is unlikely to meet its primary endpoint upon completion. Based on these results, the Phase 3 trial and further development of PF-07265803 will be discontinued. This decision is not based on safety concerns.
“This development confirms the complexity of advancing new treatments for rare cardiovascular diseases and the need to further increase knowledge in this space. We thank the patients, families, investigators and members of the advocacy community who contributed to this research,” said Chris Boshoff, M.D., Ph.D., Chief Development Officer, Oncology and Rare Disease, Pfizer Global Product Development. “Although this outcome is disappointing, Pfizer remains committed to continuing our work to evolve the treatment paradigm for patients with rare cardiovascular diseases.”
Pfizer is communicating with worldwide regulatory authorities, investigators and community groups regarding the discontinuation. Under their investigator’s guidance, patients enrolled in the Phase 3 trial will stop study medication and complete any necessary follow-up evaluations. Detailed data from the REALM-DCM study will be presented at future medical meetings to help inform ongoing research.
About REALM-DCM
REALM-DCM (NCT03439514) is a multinational, Phase 3, placebo-controlled study evaluating the efficacy and safety of PF-07265803 in patients with symptomatic LMNA-related DCM. The primary outcome measure is change from baseline in the six-minute walk test at 24 weeks, which is a measure of participant exercise tolerance as an estimate of functional capacity.
About PF-07265803
PF-07265803 (formerly ARRY-371797) is an investigational compound being studied for the treatment of symptomatic DCM due to an LMNA gene mutation. PF-07265803 is a potent and selective, oral, small-molecule inhibitor of the p38α mitogen activated protein kinase pathway, which demonstrated improvement in functional capacity (6-minute walk test; 6MWT) in a 48-week, open-label, Phase 2 study among patients with symptomatic LMNA-related DCM. Pfizer obtained PF-07265803 from Array Biopharma upon acquisition in July 2019.
About Pfizer Rare Disease
Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac, renal and inherited metabolic disorders.
Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.
About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us.
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