I was so fortunate to have Dr. Anjali Owens of the University of Pennsylvania Center for Inherited Cardiac Disease join me as co-author on the article. I also want to thank the teams at Bristol Myers Squibb and Cello Health Communications/SciFluent who supported us on this project. And, I so grateful to the former MyoKardia team and the other members of the EXPLORER-HCM Steering Committee who gave me the opportunity to witness a groundbreaking clinical trial from the front row.
This is the article. When you access the page, click to view the very informative short video, located in the upper right corner of the page, which explains how the drug works in very brief and concise terms.
Check it out!
Cynthia Burstein Waldman, Founder and Editor, HCMBeat
More positive data about the Bristol Myers Squibb experimental drug mavacamten was revealed at last weekend’s American College of Cardiology meeting and simultaneously published in The Lancet.
The data showed improvement in how patients felt taking the drug, as reported and quantified by the patients themselves. The clinical trial participants filled out a questionnaire called the KCCQ, or Kansas City Cardiomyopathy Questionnaire, 6 different times over the 38 weeks that the trial was ongoing.
Analysis of these responses showed that just over a third of patients who took mavacamten reported a substantial improvement in symptoms, though not all participants responded to the drug. And, it is important to note that the improvements seen by patients returned to baseline once mavacamten was discontinued.
DISCLOSURES: CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL AND IS CREDITED AS AN AUTHOR OF THE EXPLORER STUDY. until recently CYNTHIA also SERVED ON MYOKARDIA’S PATIENT ADVISORY BOARD.
This week, Bristol Myers Squibb submitted its New Drug Application for mavacamten to the FDA. Mavacamten is the first drug specifically designed to treat obstructive hypertrophic cardiomyopathy. The application was based on the recent positive results of the Phase 3 EXPLORER-HCM trial.
The FDA has assigned a Prescription Drug User Fee Act goal date of January 28, 2022 to the drug, which means that the FDA is expected to complete its review of mavacamten by January 28, 2022.
DISCLOSURES: CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL AND IS AN AUTHOR OF THE STUDY AS PUBLISHED IN THE LANCET. CYNTHIA ALSO SERVES ON MYOKARDIA’S PATIENT ADVISORY BOARD.
The results from the Myokardia EXPLORER- MRI sub-study are in, and there is more positive news for mavacamten (formerly known as MYK-461). According to the results of this small 35 patient study, patients who took mavacamten showed reduction of left ventricular size and wall thickness on MRI. These patients also had a reduction in their left atrial volume. All three of these measurements are predictors of poor outcome for HCM patients Additionally, this study found a reduction in certain biomarkers such as NT-proBNP, which indicate heart stress and injury.
This morning MyoKardia announced that it was being acquired by drug company Bristol Myers Squibb (BMS) for $13.1 billion. BMS plans to continue MyoKardia’s development of mavacamten as the first drug specifically intended to treat hypertrophic cardiomyopathy. The deal is expected to close before the end of 2020.
You can read a press release about the merger from MyoKardia here or a press release from BMS here. You can also read about it in the Wall Street Journalhere.
DISCLOSURES: HCMBEAT HAS RECEIVED UNRESTRICTED EDUCATIONAL GRANTS FROM MYOKARDIA. ADDITIONALLY, CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL AND IS CREDITED AS AN AUTHOR OF THE STUDY AS PUBLISHED IN THE LANCET. CYNTHIA ALSO SERVES ON MYOKARDIA’S PATIENT ADVISORY BOARD.
The results of MyoKardia’s Stage 3 EXPLORER study for its experimental drug mavacamten were presented on Saturday at the European Society of Cardiology’s virtual annual meeting and contemporaneously published in The Lancet.
The findings from the study showed that mavacamten improved exercise capacity, reduced left ventricular outflow tract gradient (responsible for the obstruction in HCM) and improved New York Heart Association functional class, correlating with a reduction in symptoms and an improvement in quality of life in patients with obstructive hypertrophic cardiomyopathy.
This was the largest placebo controlled randomized trial ever conducted in HCM. Additionally, mavacamten is the first drug specifically developed for the treatment of hypertrophic cardiomyopathy. All drugs previously used for HCM symptoms were developed to treat other conditions and were used “off label” to treat HCM.
According to Florence, Italy’s Dr. Iacopo Olivotto, who presented the results to ESC’s virtual audience, mavacamten was generally well tolerated and did not appear to have serious side effects.
Also, a new trial comparing mavacamten to septal myectomy and septal alcohol ablation entitled VALOR-HCM for patients with symptomatic, obstructive HCM and who are New York Heart Association Class III – IV who meet the criteria for septal reduction therapy and have been referred for such a procedure has just gotten underway with the first patient enrolled earlier this month.
DISCLOSURES: HCMBEAT HAS RECEIVED UNRESTRICTED EDUCATIONAL GRANTS FROM MYOKARDIA. ADDITIONALLY, CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVED AS A PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL and is credited as an author of the study as published in the lancet. cynthia also serves on myokardia’s patient advisory board.
MyoKardia today announced positive topline results for its experimental drug mavacamten from its recent Stage 3 EXPLORER trial for patients with obstructive hypertrophic cardiomyopathy or HCM.
According to the press release, the drug met all primary and secondary endpoints, while relieving outflow tract obstruction, improving symptoms and enhancing quality of life for the patients enrolled in the trial. Furthermore, the drug was well tolerated and appeared to be free of significant side effects.
MyoKardia plans to seek FDA approval for mavacamten, the first drug specifically targeting the underlying biological mechanism of HCM, in the first quarter of 2021.
Full results of the EXPLORER study will be presented at a scientific meeting later this year.
DISCLOSURES: CYNTHIA BURSTEIN WALDMAN OF HCMBEAT SERVES AS PATIENT ADVISOR ON THE STEERING COMMITTEE OF MYOKARDIA’S EXPLORER TRIAL AND AS A MEMBER OF MYOKARDIA’S PATIENT ADVISORY BOARD.
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.