At long last, there is a FDA approved drug specifically intended for the treatment of hypertrophic cardiomyopathy. After several years of testing, and based on the results of the groundbreaking EXPLORER-HCM trial, Bristol Myers Squibb’s new drug mavacamten, being marketed under the brand name Camzyos, is now available to HCM patients.
Editor’s note: You have probably noticed a distinct uptick in clinical trials of potential treatments for hypertrophic cardiomyopathy. HCMBeat has been following this trend and has previously published a host of stories about such trials, including this story about the positive results from the REDWOOD-HCM Phase 2 clinical trial, as well as past stories discussing the biopharmaceutical company Cytokinetics, its drug aficamten (previously known as CK-274), and the REDWOOD-HCM trial.
Some of these earlier stories are as follows:
Recently, Cynthia Waldman of HCMBeat had the opportunity to speak over Zoom with Dr. Martin Maron, who recently served as the principal investigator of Cytokinetics’ REDWOOD trial. The conversation focused both on Cytokinetic’s drug aficamten (previously known as CK-274), and the new class of drugs known as “myosin inhibitors.” What follows is a transcript of their conversation (which has been edited for readability).
I was recently asked by the good people at MyoKardia, now a subsidiary of Bristol Myers Squibb, if I would author a plain language summary of an article to be published in the journal Future Cardiology. This article summarizes the results of the EXPLORER-HCM trial, which showed that mavacamten, a new type of drug known as a “myosin modulator,” was successful in reducing HCM symptoms in subset of patients who took the drug during the clinical trial.
I was so fortunate to have Dr. Anjali Owens of the University of Pennsylvania Center for Inherited Cardiac Disease join me as co-author on the article. I also want to thank the teams at Bristol Myers Squibb and Cello Health Communications/SciFluent who supported us on this project. And, I so grateful to the former MyoKardia team and the other members of the EXPLORER-HCM Steering Committee who gave me the opportunity to witness a groundbreaking clinical trial from the front row.
This is the article. When you access the page, click to view the very informative short video, located in the upper right corner of the page, which explains how the drug works in very brief and concise terms.
Check it out!
Cynthia Burstein Waldman, Founder and Editor, HCMBeat
The results of MyoKardia’s Stage 3 EXPLORER study for its experimental drug mavacamten were presented on Saturday at the European Society of Cardiology’s virtual annual meeting and contemporaneously published in The Lancet.
The findings from the study showed that mavacamten improved exercise capacity, reduced left ventricular outflow tract gradient (responsible for the obstruction in HCM) and improved New York Heart Association functional class, correlating with a reduction in symptoms and an improvement in quality of life in patients with obstructive hypertrophic cardiomyopathy.
This was the largest placebo controlled randomized trial ever conducted in HCM. Additionally, mavacamten is the first drug specifically developed for the treatment of hypertrophic cardiomyopathy. All drugs previously used for HCM symptoms were developed to treat other conditions and were used “off label” to treat HCM.
According to Florence, Italy’s Dr. Iacopo Olivotto, who presented the results to ESC’s virtual audience, mavacamten was generally well tolerated and did not appear to have serious side effects.
MyoKardia said the Explorer-HCM data will form the basis of its submission to the FDA which is planned for the first quarter of 2021. Last month, the company announced that the drug was awarded “Breakthrough Drug” designation by the FDA which it hopes will streamline the approval process.
Also, a new trial comparing mavacamten to septal myectomy and septal alcohol ablation entitled VALOR-HCM for patients with symptomatic, obstructive HCM and who are New York Heart Association Class III – IV who meet the criteria for septal reduction therapy and have been referred for such a procedure has just gotten underway with the first patient enrolled earlier this month.