Bristol Myers Squibb’s (BMS) Phase III trial investigating Camzyos (mavacamten) in patients with non-obstructive hypertrophic cardiomyopathy (nHCM) has failed to meet its dual primary endpoints.
In the ODYSSEY-HCM (NCT05582395) trial, the cardiac myosin inhibitor did not show a benefit compared to placebo in either the Kansas City Cardiomyopathy Questionnaire – Clinical Summary Score (KCCQ-23 CSS) or peak oxygen consumption (pVO2) after 48 weeks. The study was a Phase III randomised, double-blind, placebo-controlled study that enrolled 580 adult patients with symptomatic (NYHA class II or III) nHCM.
The company has not mentioned the status of secondary endpoints, which evaluated a change from baseline in ventilatory efficiency (VE/VCO2), NYHA functional class, N-terminal pro B-type natriuretic peptide (NT-proBNP) levels, and the Hypertrophic Cardiomyopathy Symptom Questionnaire-Shortness of Breath (HCMSQ-SoB) after 48 weeks.
Dr. Roland Chen, senior vice president, drug development, immunology and cardiovascular medicines at BMS, said: “These findings represent the first Phase III clinical trial data for a cardiac myosin inhibitor in non-obstructive HCM. Importantly, these results do not change the favourable benefit-risk profile that has been consistently demonstrated across our Camzyos clinical trials in obstructive HCM and the robust body of real-world effectiveness and safety evidence showing its benefit for people living with obstructive HCM around the world.”
This Phase III failure comes as a blow to BMS, which was hoping to gain label expansion for the drug’s use in nHCM. Camzyos gained approval from the US Food and Drug Administration (FDA) in 2022 as a treatment for obstructive forms of HCM and remains the only FDA-approved treatment for that condition. European approval followed in June 2023. The therapy was the main asset that inspired BMS’ acquisition of MyoKardia for $13bn in 2020.
Despite having held its spot as the only HCM therapy for nearly three years, BMS is gearing up for its first potential competition in the space as the FDA has accepted a marketing application by Cytokinetics for its therapy aficamten for treating obstructive HCM.
Investigators said the Phase III trial results make them believe HCM and nHCM should be classified as different diseases.
Dr. Milind Desai, vice chair of the Heart, Vascular & Thoracic Institute at the Cleveland Clinic said: “The findings of this trial help us understand that obstructive HCM and non-obstructive HCM are two unique diseases. ODYSSEY-HCM indicates that we must consider new ways of thinking about potential treatment approaches for nHCM.”
GlobalData predicts Camzyos will reach blockbuster status next year with a global sales forecast of $2.39bn in 2030.
GlobalData is the parent company of Clinical Trials Arena.
