New HCM drug makes exercise, everyday tasks easier in clinical trials

An international clinical trial has revealed that an investigational drug, aficamten, significantly enhances oxygen utilization during exercise in individuals with a common heart condition, hypertrophic cardiomyopathy (HCM). This pivotal study, published in the New England Journal of Medicine, was also presented at the European Society of Cardiology’s Heart Failure 2024 meeting in Lisbon, Portugal.

Oregon Health & Science University (OHSU) participated in this randomized, double-blind Phase 3 trial, which evaluates aficamten, a drug developed by Cytokinetics to treat the obstructive form of HCM. Out of the 282 adults involved in the trial, 19 were enrolled through OHSU, the highest number for any participating center.

“By having more oxygen available during exercise, patients with obstructive hypertrophic cardiomyopathy can more easily walk, perform household chores, and do other everyday tasks,” said Dr. Ahmad Masri, co-author of the study and director of the OHSU Knight Cardiovascular Institute’s Hypertrophic Cardiomyopathy Center. “Our latest clinical trial results suggest aficamten is a promising treatment for HCM.”

Hypertrophic cardiomyopathy affects about 1 in 500 people and is a leading cause of sudden death among young and otherwise healthy athletes. Often linked to inherited gene mutations, HCM thickens the heart muscles, impairing the heart’s function and reducing exercise capacity due to shortness of breath. In its obstructive form, HCM hinders blood flow out of the heart.

Participants in the trial were divided into two groups: one receiving aficamten and the other a placebo. Scientists measured the participants’ oxygen levels while they exercised on treadmills or bicycles.

Those who took aficamten demonstrated a significant increase in their maximum oxygen use, with an average increase of 1.7 milliliters per kilogram per minute compared to the control group.

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Enhanced peak oxygen uptake can substantially improve a patient's ability to engage in physical activities, whereas reduced oxygen uptake is associated with a higher risk of heart failure, the need for a heart transplant, and mortality.

Currently, non-drug treatment options for obstructive HCM include surgical procedures to remove excess heart muscle. In 2022, the Food and Drug Administration (FDA) approved mavacamten, the first drug designed to target the underlying cause of obstructive HCM.

However, mavacamten presents some challenges, such as an increased risk of heart failure and interactions with commonly used medications, necessitating intense monitoring for patients.

OHSU has been at the forefront of research into new treatments for HCM over the past decade. The university has participated in multiple mavacamten studies and is actively involved in gene therapy research. Currently, OHSU is also part of four other aficamten trials, exploring its potential as a treatment for various forms of HCM and different patient demographics, including children.

“This is an exciting time for treating HCM,” said Dr. Masri. “While we continue to offer traditional surgical and procedural therapies for HCM, we are now also able to offer patients other treatment options: therapies that were recently approved by the FDA and investigational therapies that are available by participating in clinical trials.”

The findings from the aficamten trial offer hope for improved quality of life for patients with obstructive hypertrophic cardiomyopathy. As research continues and more treatment options become available, the management of HCM is poised to undergo significant advancements, benefiting patients worldwide.

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