A Phase 3 clinical trial aimed at the non-obstructed HCM population (nHCM) appears to be planned for Cytokinetics’ next-generation myosin inhibitor, aficamten.
Based on positive data from Cohort 4 of the Phase 2 REDWOOD-HCM trial, Cytokinetics is planning to soon launch a Phase 3 clinical trial for non-obstructed hypertrophic cardiomyopathy. Non-obstructed HCM patients are a group whose only current treatment options are diuretics, beta-blockers, and other drugs used in the traditional heart failure setting. In extreme cases, heart transplantation may be the only path available to these patients.
According to data presented on Sunday at the American College of Cardiology 72nd Annual Scientific Sessions (held in my beloved hometown of New Orleans, the city with the best and least heart-healthy food in the world), 41 patients with nHCM in the cohort showed improvement to both their NT-proBNP and troponin levels (both are biomarkers of heart wall stress seen in blood tests). And, the drug was generally well tolerated.
Separately, data was presented showing that obstructed HCM (HOCM) patients who took aficamten for a 48 week period showed significant improvement to their left ventricular outflow tract gradients (LVOT) while 88% of patients experienced improvement of at least one NYHA Functional Class.
All 19 HOCM patients enrolled in the trial who fit the eligibility criteria for septal reduction therapy (septal myectomy or septal alcohol ablation) at the start of the trial improved so much that they were no longer eligible after 48 weeks on aficamten.
It would be wonderful to have better options for the treatment HCM, so here’s hoping that aficamten continues down the road to FDA approval.
For more about aficamten, read these prior posts from HCMBeat:
2 Companies Testing Drugs for HCM
HCM Clinical Trials – the Latest News
Positive Signs from REDWOOD-HCM
Promising Data about Aficamten Presented at Meetings
More on Aficamten & Mavacamten from ACC 2022
Aficamten Updates from Cytokinetics
Aficamten Gets “Breakthrough Drug” Status from FDA
Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron
HCM Beat 
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