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

A Conversation with Duke’s Dr. Andrew Wang – Creator of the HCM Care App

A few months ago, HCMBeat featured this post about HCM Care, a new educational website and downloadable app for HCM patients and their families, featuring essential information for patients trying to understand their HCM diagnosis, explained in written and video formats.  HCM Care also provides useful information about genetic testing and family screening for their family members.

myokardia_mockup (003)

Dr. Andrew Wang of Duke University’s HCM Clinic in Durham, N.C., developed HCM Care along with 8 other HCM specialists from 6 hospitals, including Mayo Clinic, Cleveland Clinic and Tufts Medical Center.  Funding and support for the project were provided by MyoKardia, a San Francisco biotech company engaged in the development of a precision medicine approach to the treatment of genetic cardiomyopathies. Their HCM medication, MYK-461, is currently in clinical trials in the U.S.

Cynthia Burstein Waldman of HCM Beat had the opportunity via email to talk with Dr. Wang about his HCM practice and his involvement with the development of HCM Care.  What follows is their written correspondence, edited for clarity:

 HCMBeat:  What got you interested in HCM?  Did you first develop an interest during your training at Johns Hopkins, or did it begin later once you arrived at Duke?

Dr. Wang:  During my internal medicine residency, HCM became fascinating to me because of the interesting physical exam findings in patients with outflow tract obstruction and the complexity of the condition causing diverse symptoms and manifestations.  In my early faculty years at Duke (soon after alcohol septal ablation was introduced for the treatment of obstructive HCM), I realized the importance of having a dedicated care approach and experience in treating HCM patients to optimize and individualize treatment, so I started our HCM Clinic at Duke in 2003.  I’ve continued to direct this clinic since then.

HCMBeat:  How many HCM patients do you see in the HCM Clinic at Duke?

Dr. Wang:  We have a dedicated, full day clinic for HCM on 3 Mondays each month.  I see approximately 6 new patient referrals for HCM and 10 returning HCM patients each clinic day.  We treat over 500 HCM patients a year at Duke.

HCMBeat:  Does Duke perform septal myectomy and/or septal alcohol ablation for obstructed HCM? How many of each type of procedure does Duke perform per year?

Dr. Wang:  Our center performs about 30 surgical myectomies which are performed by 2 dedicated cardiac surgeons and 5 alcohol septal ablations each year.

HCMBeat:  Do you have a cardiac genetics counselor at Duke? What about a pediatric HCM specialist?  How many of your diagnosed patients are genetically tested?

Dr. Wang:  Yes, we have a full-time cardiovascular genetics counselor and a pediatric HCM specialist as well.  We perform genetic testing in about 40 HCM patients a year.

HCMBeat:  How did you come up with the idea of the HCM Care website and app?

Dr. Wang:  The idea for the HCM Care website and app originated over 2 years ago and developed from my experiences with treating HCM patients.  We used to send paper copies or hand-outs of helpful educational materials to our HCM patients before or after their clinic visits.  But I realized that these materials were only general overviews, and that patients often had more specific questions about HCM and the answers were either difficult to find or not discussed at all in the handouts.  I thought that a question-based approach would allow a patient or family member to find their specific answer, and perhaps see other questions and answers relevant to their situation.  We’ve been very fortunate to work with an outstanding team of physicians, patients, software and animation designers, and videographers to develop this resource.

HCMBeat:  Are the topics addressed on the website and app the most frequent questions that you encounter from patients?

Dr. Wang:  The questions were written or selected based on several factors: 1) actual questions HCM patients have asked; 2) important topics that most HCM patients should know about the condition; and 3) information that is covered in professional guidelines for treating HCM patients but that is not written for the patient audience.

HCMBeat:  Who are some of the other HCM expert physicians who are featured, and how did you choose them? Do you plan to include other physicians in future editions?

Dr. Wang:  We invited several HCM experts from many different institutions to participate in our videos and to discuss different HCM topics.  (We thought that some patients may prefer to listen or view the information in these videos, rather than read the question and answers as text.)  We selected these physicians based on their expertise in treating HCM and knowledge of recent advancements in the field.  We hope to add more expert interviews, topics and HCM physicians as the app’s content is updated.

HCMBeat:  Currently, the website and app are informational.  Are there plans to make them more interactive?  How might it be used to assist patients with decision making?

Dr. Wang:  We are very interested in having HCM patients discuss their thoughts about specific HCM topics in future video interviews.  This patient perspective is absent in the current content, and I think patients would value hearing other HCM patients talk about their treatment and what factored into their decisions.  Although the app is not intended to provide medical advice to the individual patient, we welcome ideas for new questions or content to be added in future updates.

HCMBeat:  What has been the feedback on HCM Care from your patients? 

Dr. Wang:  Many patients have said that they previously “Googled” for information about HCM, but found it challenging to find answers to their questions.  Some have come away from their searches even more uncertain or frightened.

My patients have generally been very excited to have this educational resource.  Several have found the animation of the HCM heart very helpful by allowing them visualize obstruction.  Hopefully after having seen the app or website, they have a better, more reassured understanding of this treatable condition.  Since the launch of HCM Care in March of this year, over 1,000 users have benefited from the information provided.

HCMBeat:  On behalf of HCMBeat and the HCM Community, I would like to thank you for your efforts to help patients understand what is often an overwhelming and confusing diagnosis. I would also like to thank you for taking the time to answer these questions.

HCM Care is downloadable as an app for Android and Apple mobile devices.  The version in the Apple App store can be found here:  https://itunes.apple.com/us/app/hcm-care/id1202279004?mt=8

There is also a web-based version found here:  www.hcmcare.com



Email the HCM Care team with any suggestions for new questions or with any comments at: HCMCareapp@gmail.com.


New Educational Resource for HCM Patients

Have you heard that there is a new online educational resource about HCM? Check out HCM Care.com, an informational website about HCM developed by MyoKardia in partnership with Duke Clinical Research Institute.

This website features general information about HCM including diagnosis, testing, treatment, lifestyle, genetics and family screening.  It is also available as a FREE downloadable app for both Apple and Android operating systems.

Click here to find on iTunes and Google Play.
HCMCare features video clips from the following physicians:

  • Dr. James Daubert from Duke University Medical Center
  • Dr. Milind Desai from Cleveland Clinic
  • Dr. Carolyn Ho from Brigham and Women’s Hospital
  • Dr. Martin Maron from Tufts Medical Center
  • Dr. Andrew Wang from Duke University Medical Center

Be sure to check out HCMCare, as well as many other helpful resources which are listed on HCMBeat’s Resources page.

MyoKardia HCM Drug Has Success in Cats

MyoKardia’s experimental drug MYK-461, currently in Stage 2 trials for humans, has now been shown to eliminate left ventricular obstruction in five cats with HCM. It has already been shown to inhibit traits of HCM in mice.

Addressing these findings, Associate Professor Joshua Stern, chief of the Cardiology Service at the University of California, Davis, veterinary hospital, stated:

“There has been little to no progress in advancing the treatment of HCM in humans or animals for many years,” Stern said. “This study brings new hope for cats and people.

Based on these positive results, U.C.Davis is hoping to conduct a clinical trial of MYK-461 to determine whether it could become the standard of care for cats with HCM.

The full text of the article published in Plos One can be found here.

End of the Road for Eleclazine and Liberty HCM Study

 Eleclazine:  The Liberty HCM Trial

It appears to be the end of the road for the Gilead drug eleclazine, a late sodium channel inhibitor previously known as GS-6615.  Eleclazine, with properties similar to the anti-angina drug ranolazine (which was approved by the FDA in 2006), was the subject of a recently terminated HCM clinical trial known as Liberty-HCM.  The HCM eleclazine study focused on whether the drug would improve symptoms and exercise capacity in patients with HCM by increasing their peak oxygen uptake, resulting in improved VO2 max readings on exercise testing.  The HCM study began enrolling patients in February 2015. Data collection had been scheduled to continue through June 2017. Continue reading “End of the Road for Eleclazine and Liberty HCM Study”

HCM Drug Trial Advances to Next Round


MyoKardia, a San Francisco based bio-phamaceutical company developing drugs specifically for HCM and other genetic cardiomyopathies, announced data from their Stage 1 trials showing that the drug,  MYK-461, benefits patients with HCM.

Specifically they found that the drug reduced ejection fractions and left ventricular outflow tract gradients in certain of the 101 individuals who participated in their Phase 1 trials.

The next step for the drug is to try to duplicate these findings in Phase 2 trials which will commence later this year.