MyoKardia today announced positive topline results for its experimental drug mavacamten from its recent EXPLORER trial for patients with obstructive hypertrophic cardiomyopathy or HCM.
Findings from MyoKardia’s Phase 2 Maverick clinical trial of its experimental drug mavacamten in non-obstructive HCM were announced on Monday at the online 69th Annual Scientific Sessions of the American College of Cardiology.
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.
In consultation with numerous HCM specialists across the U.S., the Hypertrophic Cardiomyopathy Association has compiled a document of guidelines for HCM patients to follow during the current coronavirus crisis.
Some of the specific recommendations are as follows:
- Patient with class 3 or 4 heart failure – specifically those with depressed ejection fractions and those awaiting transplantation – should be advised to stay close to home and minimize exposure in social gatherings.
- All patients should be encouraged to practice good personal hygiene including frequent hand washing and to use of disinfectants on all surfaces, doorknobs and banisters.
- If a HCM patient tests positive for the virus, they should contact their HCM specialist immediately in order to coordinate care with their local physicians and HCM team.
You can find the whole document here.
Additionally, the HCMA has recorded a series of informative webinars with several
HCM experts which you can find here.
A study by researchers from Mayo Clinic published this week in the Journal of the American College of Cardiology found that an artificial intelligence algorithm was able to detect hypertrophic cardiomyopathy, commonly known as HCM, from EKG results with impressive accuracy, particularly among younger patients.
In order to “teach” the computer, the researchers used digital 12-lead ECGs from 2,448 patients with HCM along with 51,153 age- and sex-matched controls. The technology was then tested on 612 HCM patients and 12,788 controls.
The findings showed that the technology was able to identify HCM in a high number of cases, even where the EKG appeared “normal” to the human eye.
The researchers believe that this technology, when refined, may prove to be an efficient tool for HCM screening in the future. The team plans to continue testing the technology in greater subject samples in order to further refine its performance.
In November at the American Heart Association’s Scientific Sessions held in Philadelphia, AHA announced that it would be starting a three-year initiative focused on hypertrophic cardiomyopathy, also known as HCM. The initiative is sponsored by MyoKardia, a San Francisco based company that is currently in clinical trials for mavacamten, the first drug specifically intended to treat HCM.
Last week, Cynthia Waldman of HCMBeat had the opportunity to speak with Amy Schmitz, AHA’s National Corporate Relations Director and Alexson Calahan, a Communication Manager for AHA.
What follows is a summary of their conversation about the forthcoming HCM initiative that has been edited for clarity.
A small study of 29 patients conducted recently in the U.K. found that the use of a biventricular pacing in patients with non-obstructive hypertrophic cardiomyopathy improved symptoms of breathlessness and improved exercise capacity as demonstrated during oxygen consumption testing.
Medications are the only treatments currently available to non-obstructed patients. The authors of this study hypothesized that biventricular pacing could be a viable way to address exercise limitations in non-obstructed patients if medications have been ineffective.
Larger trials may establish biventricular pacing as a viable treatment for non-obstructed patients in the future.
A recent retrospective study conducted by doctors at Yale -New Haven Health System found that patients with hypertrophic cardiomyopathy fare best when treated at a specialty center using a team approach to HCM. The study found that this was especially true for patients coming from disadvantaged backgrounds who often fare worse outside of a HCM specialty setting.
The findings of this study suggest that patients with HCM are best served when referred to HCM specialty care instead of receiving care solely from general cardiologists.
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.
A recent study published in the New England Journal of Medicine found that drinkers suffering from atrial fibrillation who stopped drinking for the period of the study significantly reduced episodes of atrial fibrillation.
According to doctors, alcohol consumption appears to be a significant risk factor and trigger for atrial fibrillation, while teetotaling appears to have a profound impact.
Dr. John Osborne, an American Heart Association spokesperson, said the benefit from giving up drinking was similar to results seen from drugs used to treat atrial fibrillation. Even if patients are not able to completely abstain from alcohol, Osborne advised cutting back significantly. “It costs nothing and led to a substantial reduction in hospital rates. People in the abstinence group also lost an average of 3.8 kilograms [8.4 pounds] in six months,” he said.
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).
A recent study found that African-American patients with HCM tend to be younger and more symptomatic than their white counterparts. Additionally, these patients are less likely to undergo septal reduction therapies or have genetic testing.
The implications, according to Dr. Neal Lakdawala of Brigham and Women’s Hospital who is an author of the study, is that doctors should always consider the possibility of hypertrophic cardiomyopathy in patients with left ventricular hypertrophy. If diagnosed with HCM, these patients should be referred to specialists with experience in treating HCM. This could potentially help these patients avoid the two most devastating complications of HCM: sudden death and stroke.