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.
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.
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.
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