What Should Mavacamten Cost?

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 benefitsICER 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

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

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.

More Positive Data on Mavacamten Presented at ACC Meeting

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.

Continue reading “More Positive Data on Mavacamten Presented at ACC Meeting”

Cytokinetics Announces its Phase 2 Clinical Trial – “Redwood-HCM”

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).

There are currently two companies in clinical trials for HCM:  Cytokinetics and MyoKardia. You can read more about their efforts here and here.

 

2 Companies Testing Drugs for HCM

Two San Francisco based companies are now conducting clinical trials for three drugs specifically targeting HCM.

Continue reading “2 Companies Testing Drugs for HCM”

Scientists Get $10 Million Grant to Develop HCM Treatments

A group of scientists led by Stanford University’s Dr. James Spudich, working together with researchers from the University of California-Santa Barbara, the University of Washington and the Institut Curie in Paris, has recently been awarded a $10 million grant by the National Institute of General Medical Sciences to develop novel treatments for hypertrophic cardiomyopathy (HCM). 

The researchers hope that the added resources from this grant will help them find ways to correct pathological heart protein changes they believe to be at the root of HCM. The team then plans to partner with pharmaceutical companies to develop more personalized approaches to HCM treatment.

Dr. Spudich has long been involved in HCM research and has been a founder of two separate companies which are currently engaged in drug trials for potential HCM treatments:  MyoKardia and Cytokinetics.

A story about Dr. Spudich and the inspiration for his work was featured in this recent post on HCMBeat.

Positive Results for MyoKardia Drug Mavacamten

This week in the journal Annals of Internal Medicine, MyoKardia reported positive results from its open label phase 2 clinical trial of its drug mavacamten (formerly known as MYK-461) for obstructive hypertrophic cardiomyopathy. The study was conducted at 5 HCM centers and enrolled 21 subjects with  obstructive HCM. All subjects saw some degree of improvement to their condition after taking mavacamten.

Continue reading “Positive Results for MyoKardia Drug Mavacamten”

The High Hanging Fruit: Treatment for Non-Obstructive HCM – Commentary by Dr. Sharlene Day

A recent study conducted in the U.K. evaluated whether the anti-anginal drug trimetazidine would improve symptoms and exercise capacity for those patients with non-obstructive hypertrophic cardiomyopathy. 

Unfortunately, this study which was conducted by Dr. Perry Elliott and his colleagues at University College London, found that trimetatazidine did not improve exercise capacity in these patients. Following the results of this study, trimetazidine will now join ranolazine and spironolactone in the compost heap of drugs which tried and failed to improve HCM symptoms.  While a third drug, perhexiline, was found to improve symptoms for non-obstructive HCM, its limitations, including potentially serious side effects, stand in the way of its common usage.

In a companion editorial to this study entitled “Non-Obstructive Hypertrophic Cardiomyopathy-the High Hanging Fruit,” Dr. Sharlene Day of the University of Michigan’s HCM Center discusses the difficulties seen in drug trials related to non-obstructive HCM.

Continue reading “The High Hanging Fruit: Treatment for Non-Obstructive HCM – Commentary by Dr. Sharlene Day”

Irish Researchers Investigate Drug for HCM

EpiCor Therapeutics, a Irish biotech start-up, is investigating whether 5-azacytidine, a drug previously used to treat bone marrow disorders, may reverse the hypertrophy of HCM.  According to the researchers, the drug targets the mechanisms that drive the abnormal thickening of the heart muscle by inhibiting DNA methylation, thereby reducing the growth of cardiac cells.

EpiCor’s work with 5-azacytidine was declared the overall winner of the 2016 Venture Launch Accelerator Programme at Ireland’s University College Dublin where it won the 2016 Start-Up of the Year Award.

So far, EpiCor has gotten approximately €550,000 ($690,000 U.S.) in capitalization from Enterprise Ireland’s Commercialization Fund. The next step for the drug would be to obtain regulatory approval and then, clinical trials.