The Future of HCM Care

Dr. Stephen Heitner, together with his colleagues at Oregon Health & Sciences University, published an article last week in the European Journal of Heart Failure which gives a glimpse into the treatment of hypertrophic cardiomyopathy (HCM) in the future.  Although recent publications have stated that the majority of HCM patients today have a favorable prognosis when receiving appropriate treatment, a heavy disease burden continues to be placed upon patients.  Hence, better and more effective treatments for HCM are still needed in order to lessen this burden.

The article provides a very thorough overview of both current and future HCM therapies and divides them into a few different categories listed below.

Drugs:

  • Potential for New Drugs:  No drugs have been specifically approved for the treatment of HCM by the Federal Drug Administration. Up until now, all drugs used to treat HCM patients (with the limited exception of propanolol) have been off-label uses.  However, this is likely to soon change. Current trials of myosin modulators like MyoKardia’s mavacamten and Cytokinetic’s CK-274 have so far yielded promising results and have the potential to change the treatment paradigm for HCM.

 

Arrhythmia Management:

 

  • Subcutaneous ICDs (S-ICDs) are sometimes a reasonable choice for preventing sudden cardiac death in HCM patients when pacing is not required. This allows the patient to avoid risks associated with lead malfunction.

Septal Reduction Therapy:

 

Gene Editing and Silencing:

  • Pre-implantation Genetic Diagnosis (PGD):  Current medical technology allows patients with known HCM gene(s) to use pre-implantation genetic diagnosis of embryos together with in-vitro fertilization in order to avoid passing along the HCM gene to the embryo.  The future may soon see these methods being used in tandem with gene repair, using CRISPR/Cas9 in order to edit out the errant gene and replace it with a normal one.

 

  • Gene Silencing:  Allele specific gene silencing may also prove to be a technique used in the future for preventing HCM. This technique involves the introduction of an adenovirus containing a short RNA segment designed to turn off the HCM gene.

 

In conclusion, this paper highlights the many things that HCM patients have to be optimistic about going forward.  Perhaps one day soon one of these methods will lead to the ultimate goal – a cure!

Cytokinetics Announces its Phase 2 Clinical Trial – “Redwood-HCM”

Cytokinetics today announced that its Phase 2 double-blind study of its experimental drug CK-274 entitled “REDWOOD-HCM” (Randomized Evaluation of Dosing With CK-274 in Obstructive Outflow Disease in HCM (hypertrophic cardiomyopathy) has begun enrollment.  The trial will enroll patients with symptomatic, obstructive HCM.

CK-274 is a next-generation cardiac myosin inhibitor which the company hopes will prove to be beneficial for the treatment of hypertrophic cardiomyopathy (HCM).

There are currently two companies in clinical trials for HCM:  Cytokinetics and MyoKardia. You can read more about their efforts here and here.

 

HCM Clinical Trials – the Latest News

NEWS FROM MYOKARDIA

MyoKardia recently announced a new clinical trial of its drug, mavacamten (formerly known as MYK-461) which will compare the clinical results of mavacamten with septal reduction therapies currently used in clinical practice, i.e. the open heart surgical procedure known as septal myectomy and the catheter based procedure known as alcohol septal ablation.

The study will be run by the Cleveland Clinic with Dr. Milind Desai serving as principal investigator.  MyoKardia expect to begin enrolling patients in early 2020.

Read the press release here.

 

NEWS FROM CYTOKINETICS:

Cytokinetics released positive data from its Phase 1 study of the drug currently known as CK-274 in a poster session at the HFSA 23rd Annual Scientific Meeting in Philadelphia.  The study found that CK-274 was safe and well tolerated, while no serious adverse events or negative changes to vital signs, ECGs or laboratory tests were observed.

The company will now be moving into a Phase 2 clinical trial of CK-274 in patients with obstructive hypertrophic cardiomyopathy, expected to begin in late 2019.

Read their press release here.

 

 

DISCLOSURES:  HCMBEAT HAS RECEIVED PAST UNRESTRICTED EDUCATIONAL GRANTS FROM MYOKARDIA.  ADDITIONALLY, CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVES AS A PATIENT ADVISOR ON THE STEERING COMMITTEE FOR MYOKARDIA’S EXPLORER TRIAL.

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”