The Future of HCM Treatment

Doctors from the University of Pennsylvania this week published an overview of current therapies available for the treatment of HCM.  While this article mentions all of the old standards – beta blockers, calcium channel blockers, myectomy and alcohol ablation, the real focus is on the future of HCM treatment. In particular, the article describes several new and/or experimental therapies which look promising for the future. These include:

Myosin inhibitors:

like mavacamten and aficamten.  Two more myosin inhibitors with similar properties are currently under investigation.

Valsartan: 

The angiotensin II receptor blocker valsartan has shown promise in reducing left ventricular mass in early HCM

Septal Reduction:

Novel methods of septal reduction have been used experimentally with promising results.  These include:

    • Percutaneous septal radiofrequency ablation which uses the same radiofrequency ablation techniques previously used to treat arrhythmias to treat outflow tract obstruction.

    • Transcatheter myotomy which has shown promising early results in pigs and so far on one person. In this procedure, catheters and guidewires are inserted into the basal septum and electrosurgery is used to cut the heart muscle.

Gene Therapy:

Cardiac gene therapy utilizes adeno-associated viruses (AAV) to introduce the therapy.  This therapy is in early stages, but could be a future option for a subset of patients with genetic HCM.

More Research Needed for Patients with Non-Obstructive HCM:

A phase 2 trial called IMPROVE-HCM by a company called Imbria Pharmaceuticals is looking at the drug IMB-101 in patients with nonobstructive hypertrophic cardiomyopathy while Maverick-HCM showed that mavacamten was safe in non-obstructive HCM and improved cardiac biomarkers. However, mavacamten has not yet been tested in a Phase 3 trial for patients with non-obstructive HCM.  A cohort of the REDWOOD-HCM phase 2 trial for Cytokinetics’ drug aficamten in non-obstructive patients is currently in progress.

Non-obstructed patients need more options!

Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron

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: 

2 Companies Testing Drugs for HCM

HCM Clinical Trials – the Latest News

Positive Signs from REDWOOD-HCM

Cytokinetics Moves Forward with HCM Drug Trial

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). 

Continue reading “Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron”

Cytokinetics Announces Positive Results from REDWOOD-HCM Phase 2 Clinical Trial

Cytokinetics today announced positive topline results for its experimental drug CK-274 from its recent Phase 2 REDWOOD-HCM trial for patients who have obstructive hypertrophic cardiomyopathy (HOCM).

According to the press release, this drug, a next-in-class cardiac myosin inhibitor, showed consistent and meaningful reduction in left ventricular outflow tract gradients in HOCM with its effects evident within 2 weeks of starting the drug. The benefits continued until the end of treatment 10 weeks later. No patients had to  discontinue the drug or take a break from treatment due to reduced left ventricular ejection fraction. And, the drug was well tolerated and appeared to be free from significant side effects.

Cytokinetics plans to commence a Stage 3 for CK-274 trial by the end of 2021. Full results of the Stage 2 REDWOOD study will be presented at an upcoming scientific meeting.