Pfizer to Discontinue Development Program for PF-07265803 for LMNA-Related Dilated Cardiomyopathy
Decision follows results of interim futility analysis which indicate Phase 3 REALM-DCM trial unlikely to meet primary endpoint
NEW YORK, AUGUST 3, 2022 – 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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The information contained in this release is as of August 3, 2022. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about Pfizer’s rare disease portfolio, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of any of Pfizer’s rare disease products or product candidates; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when any new or supplemental drug applications may be filed in any jurisdictions for any of Pfizer’s rare disease products or product candidates; whether and when regulatory authorities in any jurisdictions may approve any applications for any of Pfizer’s rare disease products or product candidates that may be pending or filed for any potential indication, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy, and, if approved, whether any of Pfizer’s rare disease products or product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of any of Pfizer’s rare disease products or product candidates; the impact of COVID-19 on our business, operations and financial results; and competitive developments.
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