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Phase 2 trial of drug to treat hypertrophic cardiomyopathy to be launched
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MyoKardia Inc. announced it successfully completed phase 1 trials and will launch a phase 2 trial of its novel drug to treat hypertrophic cardiomyopathy.
The drug (MYK-461) has been granted an orphan drug designation by the FDA.
“These data indicate that MYK-461 has the potential to treat patients with hypertrophic cardiomyopathy, a disease for which there are limited treatment options,” Euan A. Ashley, FRCP, DPhil, an associate professor of CV medicine, genetics and pathology at Stanford University Medical Center, and an investigator in the MYK-461 development program, said in a press release. “MYK-461, if approved, may be the first drug to target the underlying biomechanical cause of this debilitating disease.”
MyoKardia stated in the release that it will initiate a phase 2 study focusing on obstructive hypertrophic cardiomyopathy, in which an abnormally thickened heart muscle obstructs the left ventricular outflow tract.
Data from two phase 1 trials showed favorable tolerability to single doses of MYK-461 (up to 144 mg) and to repeat doses (up to 25 mg for 28 days) in both healthy volunteers (n = 86) and patients with hypertrophic cardiomyopathy (n = 15), according to the release.
In a subgroup of a phase 1 study, two patients with hypertrophic cardiomyopathy and a history of LV outflow tract obstruction had provocable gradients before being given a single 96-mg dose of MYK-461. According to the release, in one patient, the gradient fell from 28 mm Hg to 5 mm Hg, and in the other, it fell from 42 mm Hg to 9 mm Hg.
One adverse event was reported in one patient with hypertrophic cardiomyopathy, who had a vasovagal reaction that resolved spontaneously, the company stated in the release.
Disclosure: Ashley reports ownership interest with Personalis.