Mavacamten vs. Septal Reduction – VALOR-HCM Trial Results Published

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The VALOR-HCM trial results have just been published in the Journal of the American College of Cardiology. 

This study enrolled 112 obstructive HCM patients for a 16 week double blind trial of the drug mavacamten (brand name Camzyos). All patients in the trial had been referred for septal reduction therapy – either septal myectomy or septal alcohol ablation – to treat their highly symptomatic obstructive hypertrophic cardiomyopathy.   The researchers looked at whether the addition of mavacamten to their other drugs would improve their symptoms enough so that they no longer met the criteria for septal reduction therapy (SRT) under the 2011 ACC/AHA Guidelines.

You can read many more details about the VALOR-HCM study here in this recent blog post on HCMBeat.

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Recommendations for HCM Depart from 2020 ACC/AHA Guidelines

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A recent article by several noted HCM experts shares their recommendations for the screening and evaluation of patients with HCM using several scenarios commonly encountered in clinical practice. Notably, this paper makes several recommendations which differ from the 2020 ACC/AHA Guidelines for the Diagnosis and Treatment of Patients with Hypertrophic Cardiomyopathy (2020 Guidelines).

In particular, the article recommends:

  • Cardiac MRI with contrast upon initial evaluation, and every 3 – 5 years thereafter.  (The 2020 Guidelines recommend Cardiac MRI in certain situations, but not for all patients)
  • Genetic testing only in cases where a pathogenic or likely pathogenic mutation has been identified in a family member and in limited other circumstances (the 2020 Guidelines recommends genetic testing for all as part of the initial assessment)
  • Screening of family members 12 or older only (the 2020 Guidelines recommend screening of children of any age when genetic HCM is diagnosed in a family member and no later than puberty, in any event)
  • That HCM patients be disqualified from competitive sports. (The 2020 guidelines instead recommend a shared decision making approach).

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Aficamten Updates from Cytokinetics

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At two recent meetings, South San Francisco biotech company Cytokinetics presented encouraging data on its experimental drug for hypertrophic cardiomyopathy, aficamten (formerly known as CYK-274). 

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Increased COVID Risk for HCM Patients

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A recent retrospective study of HCM patients with COVID-19 concluded that HCM patients are at increased risk of hospitalization and death from a COVID-19 infection. 

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FDA Approves Mavacamten under Brand Name Camzyos

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At long last, there is a FDA approved drug specifically intended for the treatment of hypertrophic cardiomyopathy.  After several years of testing, and based on the results of the groundbreaking EXPLORER-HCM trial, Bristol Myers Squibb’s new drug mavacamten, being marketed under the brand name Camzyos, is now available to HCM patients.

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Sports and HCM – Moving Toward Shared Decision Making

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While competitive sports used to be frowned upon in the HCM literature, there is now some evidence that a patient’s risk from exercise is low when they have been implanted with an implantable cardioverter defibrillator (ICD). This feature from Medpage Today gives an overview of contemporary thinking about exercise in HCM patients.

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More on Aficamten & Mavacamten from ACC 2022

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In addition to the presentation of the results of the VALOR-HCM study which compared mavacamten to septal reduction techniques, covered here in its own HCMBeat blog entry, ACC 2022 featured two other presentations about the new class of drugs known as myosin inhibitors, such as Bristol Myers Squibb’s mavacamten and Cytokinetics’ aficamten.

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VALOR-HCM Trial – Mavacamten vs. Septal Reduction Therapy – RESULTS ARE IN!

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The Phase 3 VALOR-HCM trial results were presented this morning at the American College of Cardiology annual meeting in Washington, DC by the principal investigator, Dr. Milind Desai of the Cleveland Clinic, and the results are good!

What was the VALOR-HCM study?

15 – 20  million people worldwide are estimated to have HCM, with 2/3 of this group having the obstructive form which can cause severe symptoms. Historically, these patients have been treated with medications approved for other conditions, and if those don’t relieve symptoms, they are referred on for septal reduction therapies (SRT) like alcohol septal ablation (a catheter based procedure) or septal myectomy (open heart surgery), which are invasive therapies requiring specialized care and which are not widely available.

The VALOR study was designed to compare mavacamten head to head with SRT to see if mavacamten could be a non-invasive treatment alternative for obstructive HCM.

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Traditional Cardiovascular Risk Factors Worsen HCM

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According to this recent article in the European Heart Journal, traditional cardiovascular risk factors such as obesity, hypertension and diabetes are associated with more severe forms of HCM. Therefore, the authors suggest that these additional risk factors should be aggressively managed so as to limit their impact on HCM.  

Gene Therapy for HCM

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A recent article on Biospace.com describes a method of targeted gene therapy for HCM patients currently being developed by Tenaya Therapeutics. This therapy is intended for patients whose HCM is caused by a mutation in the MYBPC3 gene.

According to Tenaya CEO Faraz Ali, animal models show that this therapy can reverse declining heart function.

You can find more information on about Tenaya’s approach here.