HIGHLIGHTS FROM THE INTERNATIONAL HCM SUMMIT, PART IV

Editor’s Note:  This is the 4th of 4 blog entries which summarizes the presentations given at the recent International HCM Summit VI in Boston.  The presenter and their hospital affiliation are noted below, along with the topic of their presentation.  When possible, you may access the presenters’ slides via hyperlink by clicking on the name. (Note that not all presenters made their slides available).

To see Part I of this series of highlights from the HCM Summit VI, click here, to see Part II of this series click here, and to see part III of this series. click here.

Dr. Martin Maron, Tufts Medical Center, Boston, MA – A New Drug For Obstructive HCM

  • Mavacamten, formerly known as MYK-461, is being tested as a treatment for heart failure symptoms in HCM. It works by decreasing the power of the heart’s contraction. On average, both ejection fraction and left ventricular outflow tract gradient decreased by 15% in the recently completed Stage II trial.
  • Not all patients who took the drug showed improvement in symptoms and functional capacity.  The percentage of patients showing clinical improvement on mavacamten was similar to that seen with disopyramide.
  • Currently it is unclear what the long term effects of the drug will be.  This may become clearer after the upcoming Stage III trials.

Dr. Iacopo Olivotto, Careggi University Hospital, Florence, Italy spoke about the sudden termination of the clinical trial of the Gilead drug eleclazine.  A link to the HCMBeat story about this can be found here. 

Dr. Steven Heitner, Oregon Health & Science University, Portland, OR spoke about gene editing in HCM and described the recent work done with CRISPR at his institution.  A link to the HCMBeat story about this research can be found here.

Dr. Benjamin Levine, Southwestern Medical Center, Dallas, TX spoke about exercise in HCM. While the risk of sudden death in young athletes may have suggested that a more sedentary lifestyle was appropriate for HCM patients, the recent RESET-HCM trial has challenged this, finding that moderate exercise is safe for patients with HCM.  (Click here for a HCMBeat interview with the principal investigators of this study, Drs. Sara Saberi and Sharlene Day).

Dr. Levine also mentioned that high-intensity interval training (HIIT) in animal models of HCM show that HIIT may be protective against myocardial disarray.

Dr. Euan Ashley, Stanford University Medical Center, Palo Alto, CA spoke about the potential ways that personalized medicine might impact HCM care in the future.  Genetic mutations may influence the tendency toward arrhythmia, age of diagnosis, and exercise capacity.  New technologies like photoplethysmography which detects blood volume changes may provide data which can be helpful in personalizing treatments.

THIS CONCLUDES THE SUMMARIES OF THE PRESENTATIONS OF THE 2017 HCM SUMMIT.  To see Part I of this series of highlights from the HCM Summit VI, click here, to see Part II of this series click here, and to see part III of this series. click here.

 

Encouraging Results for MyoKardia HCM Drug

MyoKardia’s stock prices jumped today after their recent Stage II trial of the experimental drug mavacamten (formally known as MYK-461)  demonstrated a statistically significant reduction to left ventricular outflow tract gradients as well as improvement to aerobic capacity in patients with obstructive hypertrophic cardiomyopathy.  

Of the 10 patients who completed the study, 8 saw their gradient reduced to normal levels after 12 weeks on the drug.  The study also showed improvements in both peak oxygen consumption (peak VO2) and New York Heart Association classifications:  7 patients moved up one NYHA class while 2 patients improved by two classes.

The drug seemed to have mild to moderate side effects, though one patient was forced to drop out of the trial due to a recurrence of atrial fibrillation which necessitated discontinuation of mavacamten and a return to anti-arrythmic drugs which had been discontinued due to participation in the trial.

MyoKardia hopes to enroll between 200 and 250 patients in its next phase trial (Explorer HCM) which it plans to begin before the end of 2017.

MyoKardia also plans a clinical trial of mavacamten in non-obstructive HCM patients in the second half of 2017.

For more information on MyoKardia and  recent drugs being developed for HCM read these past blog entries:

MyoKardia HCM Drug Has Success in Cats

End of the Road for Eleclazine and Liberty HCM Study

HCM Drug Trial Advances to Next Round

Drug for Non-Obstructive HCM Moves Along