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!

More Positive Results for MyoKardia Drug

Findings from MyoKardia’s Phase 2 Maverick clinical trial of its experimental drug mavacamten in non-obstructive HCM were announced on Monday at the online 69th Annual Scientific Sessions of the American College of Cardiology.

Dr. Carolyn Ho of Harvard’s Brigham and Women’s Hospital reported to online viewers that mavacamten was generally well tolerated.  In addition, the drug showed promising results in this Phase 2 trial intended to demonstrate the safety of the drug. In particular, some relevant additional observations were reductions in blood levels of two biomarkers of cardiac stress and injury – namely  NT-proBNP which indicates hemodynamic stress, and cardiac troponin I, which is a biomarker of myocardial injury.

These findings look promising for non-obstructive HCM patients who currently have very limited treatment options.

2 Companies Testing Drugs for HCM

Two San Francisco based companies are now conducting clinical trials for three drugs specifically targeting HCM.

Continue reading “2 Companies Testing Drugs for HCM”