Tag: hypertrophic cardiomyopathy

Is a Drug for Non-Obstructive HCM on the Horizon?

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Ninerafaxstat, an investigational drug being studied by Imbria Pharmaceuticals,  may be the first drug specifically for non-obstructive hypertrophic cardiomyopathy.

The results of the Improve-HCM Phase 2 study were published last week in the Journal of the American College of Cardiology (ACC) and simultaneously presented at last week’s  meeting of the ACC in Atlanta.

The Improve study found that the drug is safe, and that patients demonstrated improvements to both exercise performance and symptoms.

The findings support advancement to a Phase 3 study to focus on efficacy of the drug.

You can read more about the IMPROVE trial here.

Is Aficamten the Blockbuster Drug Cytokinetics is Hoping For?

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Cytokinetics today released positive results from Sequoia-HCM, its Phase 3 clinical trial of the experimental drug aficamten in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).  Patients treated with aficamten showed significantly increased exercise capacity as demonstrated by a 1.74 point increase in peak oxygen uptake (pVO2) measured during cardiopulmonary exercise testing.  Improvements were also seen in provoked outflow tract gradients, and most importantly, patients felt better.  

The study had enrollment of approximately 300 patients who were followed for 24 weeks. 

Aficamten was well-tolerated, adverse events were comparable to those taking a placebo, and importantly, there were no instances of worsening heart failure or treatment interruptions due to low left ventricular ejection fraction (LVEF).

According to an article in Med City News, the drug may have an advantage over the Bristol Myers Squibb drug mavacamten (sold under the brand name Camzyos) because there were no reports of worsening heart failure. In the Phase 3 EXPLORER trial of mavacamten, seven patients in the treatment group experienced reductions to their LVEF.  

Based on the projected success of aficamten, Cytokinetics stock price jumped by 83% and the company is said to be the subject of takeover interest from AstraZeneca and Novartis.

Cytokinetics plans to apply for FDA approval for the drug in the second half of 2024.

Complete results from SEQUOIA-HCM will be presented at an upcoming medical conference.

You can read more about the drug in these previous articles on HCMBeat:

Cytokinetics Teases Upcoming Phase 3 Aficamten Trial in nHCM

Promising Data about Aficamten Presented at Meetings

Aficamten Updates from Cytokinetics

More on Aficamten & Mavacamten from ACC 2022

Aficamten Gets “Breakthrough Drug” Status from FDA

Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron

Results Published from REDWOOD-HCM Phase 2 Trial

The Future of HCM Treatment

 

 

 

 

 

 

First Patient Undergoes Gene Therapy for HCM

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Tenaya Therapeutics and the Cleveland Clinic today announced that the first patient in Tenaya’s groundbreaking gene therapy clinical trial for hypertrophic cardiomyopathy has been dosed. This trial is testing a groundbreaking therapy intended to treat HCM caused by the MYBPC3 gene which is one of the most common HCM genes.

The Phase 1b trial is looking at the investigational drug TN-201.  Preliminary results are expected in 2024.

You can find much more information about the clinical trial and Tenaya’s plans in this HCMBeat interview with Tenaya’s Executive Medical Director Laura Robertson M.D.

Viz.ai Receives FDA Approval for its HCM Detection Algorithm

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Viz.ai has received FDA approval for an artificial intelligence algorithm that can spot HCM from a standard electrocardiogram or EKG.

The algorithm automatically reviews EKGs as they are performed, and alerts the cardiology care team if it identifies a patient with suspected HCM.  Then, the care team is able to follow up with the patient and conduct further testing, if deemed appropriate.

The technology was developed through a collaboration between Viz.ai and Bristol Myers Squibb that was announced earlier this year.

New Imaging Techniques Spot HCM Before it Develops

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Research funded by the British Heart Foundation using novel cardiac imaging techniques was described in a recent article in Circulation. These techniques, such as quantitative perfusion cardiac magnetic resonance imaging and cardiac diffusion tensor imaging, can detect pre-clinical hypertrophic cardiomyopathy (HCM) before the development of any thickening in the heart.

With novel treatments like myosin modulators now available to patients, and with  experimental treatments such as gene therapy now on the horizon, these new imaging techniques could allow doctors to intervene at a much earlier stage in order to potentially prevent disease.

You can read more about these imaging techniques here.

Gene Therapy for HCM: Interview with Tenaya Therapeutics Executive Medical Director Dr. Laura Robertson M.D.

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Editor’s note:  Recently, Cynthia Waldman of HCMBeat had the chance to interview Dr. Laura Robertson of Tenaya Therapeutics . Dr. Robertson is charged with leading the clinical development program for Tenaya’s experimental gene therapy treatment for HCM.  

You have probably read about gene therapy and wondered whether it could ever be used to treat hypertrophic cardiomyopathy.  HCMBeat has written several stories about the growing field of gene therapy, shining a light on the researchers and companies focused on this effort.   

Continue reading “Gene Therapy for HCM: Interview with Tenaya Therapeutics Executive Medical Director Dr. Laura Robertson M.D.” →

Vigorous Exercise Safe for HCM Patients

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The long awaited results of the LIVE-HCM study have now been published.

HCM previously blogged about the study in March when they were first announced at the American College of Cardiology meetings.

This study followed 1534 HCM patients between the ages of 8 and 60,. Most participated in various forms of exercise over the three year period of the study, such as running, swimming and basketball.  The study also included 126 participants who carried a gene associated with HCM, but did not show overt signs of the disease. 42% of study participants exercised vigorously, 43% exercised moderately, and 16% were not active.

Contrary to an older school of thought which cautioned against vigorous exercise in HCM patients, the results of the study showed NO increase in cardiac arrests, ventricular arrhythmia or fainting among the vigorous exercisers, disproving the long held assumption that vigorous exercise was dangerous for patients with hypertrophic cardiomyopathy.  Also, the study found no cardiac events occurring among the group who only carried a gene for HCM but did not have the disease.

 

Delays Common in HCM Diagnosis

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As many as 60% of patients with hypertrophic cardiomyopathy don’t receive an appropriate diagnosis when they first come to medical attention.

In fact, HCM patients receive on average of 4 misdiagnoses before finally receiving an accurate HCM diagnosis and appropriate treatment.  A recent paper highlights the this issue which is largely due to a lack of knowledge of HCM in the medical community.  Even cardiologists often fail to accurately diagnose and treat HCM, emphasizing something that those of us with HCM already know – not all cardiologists are equal when it comes to recognizing and treating HCM.  Experts are key! 

Medical professional and patient education and awareness initiatives are critical to address these issues. Hopefully, over time we will start to see improvements to HCM diagnosis and treatment now that there are increasingly new professional and educational initiatives focused on and around HCM, such as the HCM Society.

Weight Loss Improves HCM

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HCM Beat in half circle 3.75

Researchers at New York University’s Langone Health published a recent paper reporting on 6 patients who lost a significant amount of weight (at least 18 lbs.) and who had undergone cardiac MRI or CT scans before and after the weight loss. 

The researchers found that following weight loss, the scans showed a decrease to the dimensions of the left ventricle, and the patients experienced improvement to their symptoms.

The researchers suggest that while more study is needed, weight loss can benefit overweight patients and may be especially helpful to those who have limited treatment options. 

 

Cynthia Burstein Waldman © 2023   

 

Exercise and HCM: Not Enemies Anymore

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Lots of good news for active HCM patients was presented at last weekend’s 23rd Annual American College of Cardiology Scientific Sessions.

The long-awaited results of the LIVE-HCM study were presented, and the results were quite reassuring for HCM patients who are vigorous exercisers.

This study followed 1534 HCM patients between the ages of 8 and 60,. Most participated in various forms of exercise over the three year period of the study, such as running, swimming and basketball.  The study also included 126 participants who carried a gene associated with HCM, but did not show overt signs of the disease. 42% of study participants exercised vigorously, 43% exercised moderately, and 16% were not active.

Contrary to an older school of thought which cautioned against vigorous exercise in HCM patients, the results of the study showed NO increase in cardiac arrests, ventricular arrhythmia or fainting among the vigorous exercisers, disproving the long held assumption that vigorous exercise was dangerous for patients with hypertrophic cardiomyopathy.  Also, the study found no cardiac events occurring among the group who only carried a gene for HCM but did not have the disease.

According to Dr. Rachel Lampert of the Yale School of Medicine who was the lead investigator of the study, the data from this study “does not support restriction of vigorous exercise for individuals with HCM.”  Dr. Lampert also spoke about the benefits to health of exercise in general, and the need for shared decision-making between HCM patients and experienced HCM physicians when making decisions around exercise.

You can see this video of Dr. Lampert talking about the LIVE-HCM study.

And in other good news for HCM athletes, another paper supportive of competitive sports in patients with genetic heart disease was presented at ACC23.  This presentation showed that 95% of competitive athletes who played at the Division 1 or professional level who elected to return to play after being diagnosed with a genetic heart disease did not experience cardiac events such as fainting, seizures, ICD shocks, sudden cardiac arrests or sudden cardiac death.  Again, focusing on the need for shared decision-making and risk stratification with an expert, the researchers believe that with a comprehensive return-to-play protocol including a personal AED, annual follow-ups and continued risk evaluation, together with coordination with team and/or organization medical staff, a return to competitive sports may be possible.

Comprehensive risk assessment, combined with shared decision making around the decision to participate in sports, seems to be the way of the future.  It is an important concept that was included and discussed in the 2020 ACC/AHA Guidelines for the Diagnosis and Treatment of HCM and represents an important advance for patients.