2 Companies Testing Drugs for HCM

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

MyoKardia, which was founded in 2012 by a group of HCM researchers (including Stanford’s James Spudich, one of the founders of Cytokinetics – the second company conducting a HCM drug trial – see below), was the first entrant into the HCM area with the development of its drug, mavacamten (formerly known as MYK-461).

Mavacamten is currently the subject of the Phase 3 EXPLORER-HCM clinical trial for obstructive HCM, now fully enrolled with results expected in 2020, as well as the Phase 2 MAVERICK-HCM trial for non-obstructive HCM, with results are expected later this year.

And, MyoKardia announced this week that it is will begin testing a second drug for HCM.  The new drug, currently known as MYK-224, is the subject of a new Phase 1 clinical trial.  This drug targets the sarcomeric proteins of the heart muscle like MyoKardia’s first drug, mavacamten. According to the press release, MYK-224 may provide dosing advantages for some patients over other drugs.

Cytokinetics, a company founded in 1998 which was previously focused on other muscle related conditions like ALS, has decided to set its sights on HCM.  Cytokinetics is currently conducting a Phase 1 clinical trial assessing the safety and tolerability of its drug CK-274, a cardiac myosin inhibitor intended to reduce cardiac contractility.

At a recent cardiology meeting in Boston, Cytokinetics presented data showing that CK-274 decreased cardiac contractility in healthy animals.

Stay tuned to HCMBeat for the latest details and updates about these drugs.

UPDATE:  Data Presented at August 31, 2019 European Society of Cardiology Congress in Paris

At ESC, MyoKardia announced results from the PIONEER-Open Label Extension study from 12 patients who had been enrolled in the Phase 2 PIONEER-HCM study of mavacamten. These patients were evaluated after a total of 36 weeks on the drug.  The study results showed reduction in both resting and provoked left ventricular outflow tract gradients, while left ventricular ejection fraction remained normal at all times.  Further, certain biomarkers of heart disease showed improvement with mavacamten treatment.  Most strikingly, NT-proBNP, a blood indicator of cardiac wall stress, decreased almost to normal.   

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.