Cytokinetics drug Myqorzo meets twin efficacy goals in study of genetic heart disease

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.

Cytokinetics said Tuesday that its drug Myqorzo significantly improved heart failure symptoms and cardiovascular fitness in patients with non-obstructive hypertrophic cardiomyopathy, an inherited heart disorder. 

The results, reported in a company press release, achieved the dual efficacy goals of a Phase 3 clinical trial, called ACACIA, with statistical significance.

Cytokinetics is in the early days of Myqorzo’s commercial launch as a treatment for the more severe “obstructive” form of hypertrophic cardiomyopathy, or HCM. The successful outcome of the ACACIA study, if also cleared by regulators, could greatly expand the number of HCM patients eligible for treatment — and boost the drug’s peak sales to $5 billion annually, according to analyst forecasts. 

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