Tag: drug

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

 

 

 

 

 

 

Mavacamten (Brand Name Camzyos) Approved in Europe

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Bristol Myers Squibb’s first-in-class myosin modulator mavacamten (brand name Camzyos) has been approved by the European Commission for the treatment of obstructive hypertrophic cardiomyopathy in all countries which are part of the European Union.

See the press release here.

Ranolazine Improves Chest Pain in HCM

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A recent paper by Italian HCM specialists found that chest pain completely resolved for 73% of patients who took the drug ranolazine. The researchers also found that the drug was safe and well tolerated.  In addition, it reduced arrhythmias for those whose arrhythmias were triggered by ischemia and it improved biomarkers in HCM patients.

This study is a follow up to the 2018 RESTYLE-HCM study, which found that while ranolazine did not improve heart failure symptoms in HCM, it was useful for treating chest pain.

Promising Data about Aficamten Presented at Meetings

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Encouraging data about the Cytokinetics investigational drug aficamten was presented over the weekend at 2 scientific meetings held in Washington D.C.

Continue reading “Promising Data about Aficamten Presented at Meetings” →

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.

Continue reading “FDA Approves Mavacamten under Brand Name Camzyos” →

Guest Blogger Gwen Mayes, JD, MMSc – Cautiously Awaiting the Release of Mavacamten

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            Fifteen years ago, I referred myself to Mayo Clinic in Rochester, Minnesota, to be evaluated for a septal myectomy by what was at the time, one of the country’s few expert centers for the treatment of hypertrophic cardiomyopathy.   I traveled to Mayo from my native state, Kentucky, on a brief hiatus from a well-established career in health policy in Washington, DC.

Continue reading “Guest Blogger Gwen Mayes, JD, MMSc – Cautiously Awaiting the Release of Mavacamten” →

Aficamten Gets “Breakthrough Drug” Status from FDA

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Cytokinetics has announced that its experimental drug aficamten, currently in trials as a potential treatment for obstructive hypertrophic cardiomyopathy, has received breakthrough therapy designation from the FDA.  This designation is awarded by the FDA to certain drugs which may offer substantial improvement to patients over available therapies. The designation could shorten the FDA approval process for the drug by about 4 months.

You can read more about Cytokinetics and aficamten in these older posts from HCMBeat: 

Interview with Dr. Martin Maron about Cytokinetic’s Drug Aficamten 

Cytokinetics Announces Positive Results from REDWOOD-HCM Phase 2 Clinical Trial

Cytokinetics Moves Forward with HCM Drug Trial

Cytokinetics Announces its Phase 2 Clinical Trial

Positive Signs from REDWOOD-HCM

The Future of HCM Care

HCM Clinical Trials – the Latest News

2 Companies Testing Drugs for HCM

Scientists Get $10 Million Grant to Develop HCM Treatments

 

 

What Should Mavacamten Cost?

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On October 22, patients, physicians, and other interested parties will have the opportunity to provide input on the value and cost of mavacamten – the first drug specifically designed to treat hypertrophic cardiomyopathy. Last year, Bristol Myers Squibb paid $13.1 billion to acquire MyoKardia, the San Francisco biotech company that developed the drug and brought it through clinical trials.

At a virtual public meeting, The Institute for Clinical and Economic Review or ICER will listen to further testimony in order to evaluate mavacamten’s value and potential benefits. ICER is a non-profit organization that evaluates the cost effectiveness of drugs and medical procedures. Many insurance companies rely on ICER’s findings when deciding how much to pay for a certain treatment or test. 

In an Effectiveness Report which was published today, ICER valued the benefit that mavacamten would bring to a patient at between $12,000 to $15,000 a year. By contrast, some analysts have suggested that mavacamten could carry a price tag as high as $75,000 per patient per year.

 If you would like to share your thoughts at the online public meeting click here to sign up.  

You can find a press release from ICER about their review of mavacamten here.

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

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Editor’s note:  You have probably noticed a distinct uptick in clinical trials of potential treatments for hypertrophic cardiomyopathy.  HCMBeat has been following this trend and has previously published a host of stories about such trials, including this story about the positive results from the REDWOOD-HCM Phase 2 clinical trial, as well as past stories discussing the biopharmaceutical company Cytokinetics, its  drug aficamten (previously known as CK-274), and the REDWOOD-HCM trial.

Some of these earlier stories are as follows: 

2 Companies Testing Drugs for HCM

HCM Clinical Trials – the Latest News

Positive Signs from REDWOOD-HCM

Cytokinetics Moves Forward with HCM Drug Trial

Recently, Cynthia Waldman of HCMBeat had the opportunity to speak over Zoom with Dr. Martin Maron, who recently served as the principal investigator of Cytokinetics’ REDWOOD trial.  The conversation focused both on Cytokinetic’s drug aficamten (previously known as CK-274), and the new class of drugs known as “myosin inhibitors.”  What follows is a transcript of their conversation (which has been edited for readability). 

Continue reading “Cytokinetic’s Drug Aficamten & Upcoming HCM Summit – Interview with Dr. Martin Maron” →

Drug Trial for Non-Obstructive HCM

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Yet another company is developing a new drug for hypertrophic cardiomyopathy, and this time, the drug is intended for non-obstructive HCM patients.  The company is Imbria Pharmaceuticals, and this week they announced the randomizing of the first patient in their Phase 2 study of the drug IMB-101 in patients with non-obstructive HCM.  The study, called IMPROVE-HCM, is a Phase 2 study that will look at the safety and tolerability of this drug in non-obstructed HCM patients. IMB-101 is designed to increase the efficiency of the heart’s use of energy which will be measured through cardiopulmonary exercise testing over a 12 week period.

You can read this press release and you can read more about the trial on ClinicalTrials.gov here.