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.

MyoKardia HCM Drug Has Success in Cats

MyoKardia’s experimental drug MYK-461, currently in Stage 2 trials for humans, has now been shown to eliminate left ventricular obstruction in five cats with HCM. It has already been shown to inhibit traits of HCM in mice.

Addressing these findings, Associate Professor Joshua Stern, chief of the Cardiology Service at the University of California, Davis, veterinary hospital, stated:

“There has been little to no progress in advancing the treatment of HCM in humans or animals for many years,” Stern said. “This study brings new hope for cats and people.

Based on these positive results, U.C.Davis is hoping to conduct a clinical trial of MYK-461 to determine whether it could become the standard of care for cats with HCM.

The full text of the article published in Plos One can be found here.

End of the Road for Eleclazine and Liberty HCM Study

 Eleclazine:  The Liberty HCM Trial

It appears to be the end of the road for the Gilead drug eleclazine, a late sodium channel inhibitor previously known as GS-6615.  Eleclazine, with properties similar to the anti-angina drug ranolazine (which was approved by the FDA in 2006), was the subject of a recently terminated HCM clinical trial known as Liberty-HCM.  The HCM eleclazine study focused on whether the drug would improve symptoms and exercise capacity in patients with HCM by increasing their peak oxygen uptake, resulting in improved VO2 max readings on exercise testing.  The HCM study began enrolling patients in February 2015. Data collection had been scheduled to continue through June 2017. Continue reading “End of the Road for Eleclazine and Liberty HCM Study”

HCM Drug Trial Advances to Next Round

 

MyoKardia, a San Francisco based bio-phamaceutical company developing drugs specifically for HCM and other genetic cardiomyopathies, announced data from their Stage 1 trials showing that the drug,  MYK-461, benefits patients with HCM.

Specifically they found that the drug reduced ejection fractions and left ventricular outflow tract gradients in certain of the 101 individuals who participated in their Phase 1 trials.

The next step for the drug is to try to duplicate these findings in Phase 2 trials which will commence later this year.