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.
When Stanford biochemist Jim Spudich settled down in bed with a book recommended by his wife, he had no idea that the book would inspire one of the biggest discoveries of his career. Spudich drifted off to sleep while reading The Haunted Mesa, a science fiction novel by Louis L’Amour. His scientific discovery was based on an image he saw in his dreams when the image of a mesa morphed into a myosin molecule.
According to this press release, MyoKardia expects to dose the first patient in the EXPLORER-HCM trial of mavacamten (formerly known as MYK-461) for obstructive HCM in the second quarter of 2018.
MyoKardia says that it expects 220 patients to enroll in the 30 week long trial. These patients will be randomly assigned to receive either mavacamten or a placebo. Participants will also be able to continue on their normal beta blockers or calcium channel blockers.
MyoKardia, a San Francisco biotech company currently in clinical trials on a HCM drug called Mavacamten, has come up with a way to spot HOCM simply by using a wristband fitness monitor. The wristband works through the use of optical biosensors which monitor arterial pulse waves.
During MyoKardia’s trials, the bracelet biosensors were used on HOCM patients and non-affected controls. The algorithm was able to distinguish HOCM patients from unaffected individuals more than 95% of the time, suggesting that a non-invasive way to screen for HOCM may not be too far in the future.
The linked article at Medgadget contains an interview with MyoKardia’s Dr. Robert McDowell, MyoKardia’s Chief Scientific Officer, and Dr. Eric Green, MyoKardia’s Senior Director of Translational Research with more on the happenings at MyoKardia.
Editor’s Note: This is the 4th of 4 blog entries which summarizes the presentations given at the recent International HCM Summit VI in Boston. The presenter and their hospital affiliation are noted below, along with the topic of their presentation. When possible, you may access the presenters’ slides via hyperlink by clicking on the name. (Note that not all presenters made their slides available).
MyoKardia’s stock prices jumped today after their recent Stage II trial of the experimental drug mavacamten (formally known as MYK-461) demonstrated a statistically significant reduction to left ventricular outflow tract gradients as well as improvement to aerobic capacity in patients with obstructive hypertrophic cardiomyopathy.
Of the 10 patients who completed the study, 8 saw their gradient reduced to normal levels after 12 weeks on the drug. The study also showed improvements in both peak oxygen consumption (peak VO2) and New York Heart Association classifications: 7 patients moved up one NYHA class while 2 patients improved by two classes.
The drug seemed to have mild to moderate side effects, though one patient was forced to drop out of the trial due to a recurrence of atrial fibrillation which necessitated discontinuation of mavacamten and a return to anti-arrythmic drugs which had been discontinued due to participation in the trial.
MyoKardia hopes to enroll between 200 and 250 patients in its next phase trial (Explorer HCM) which it plans to begin before the end of 2017.
For more information on MyoKardia and recent drugs being developed for HCM read these past blog entries: