Results indicated the trial is unlikely to meet its primary endpoint, the change from baseline in the six-minute walk test at 24 weeks.
Pfizer has discontinued development of a therapy to treat dilated cardiomyopathy, a disease in which the heart doesn’t pump efficiently. The phase 3 trial, REALM-DCM, was evaluating PF-07265803 in patients with symptomatic dilated cardiomyopathy (DCM) as the result of a mutation of the gene encoding the lamin A/C protein (LMNA). Results indicated the trial is unlikely to meet its primary endpoint, the change from baseline in the six-minute walk test at 24 weeks.
“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,” Chris Boshoff, M.D., Ph.D., chief development officer, oncology and rare disease, Pfizer Global Product Development, said in a press release. “Although this outcome is disappointing, Pfizer remains committed to continuing our work to evolve the treatment paradigm for patients with rare cardiovascular diseases.”
The therapy (formerly ARRY-371797) was obtained by Pfizer as part of its 2019 acquisition of Array Biopharma. It is a small-molecule inhibitor of the p38α mitogen activated protein kinase pathway, which demonstrated improvement in functional capacity in a phase 2 study.
Dilated cardiomyopathy is a disease that often starts in the heart’s left ventricle, and the heart muscle dilates, stretches and becomes thinner. As the heart becomes weaker, heart failure can occur. Dilated cardiomyopathy is the third leading cause of heart failure in the United States. There is no specific treatment for DCM as a result of this gene mutation.