MyoKardia ($MYOK) is working on making good on its IPO last fall. It’s up by more than one-third on early data for its lead candidate in hypertrophic cardiomyopathy (HCM). MyoKardia plans to head into Phase II testing for the candidate, MYK-461, during this half of 2016.
That trial will be in symptomatic obstructive HCM (oHCM), an indication for which the FDA has already granted the candidate its Orphan Drug Designation. MYK-461 is partnered with Sanofi ($SNY) under a deal worth up to $200 million that dates back to 2014. Investors are likely betting that this latest news improves MyoKardia’s chances to cash in under that partnership.
The biotech has already received $45 million in an upfront payment and an investment by Sanofi, but the remaining cash, or $85 million, awaits a decision by Sanofi to continue the collaboration that’s due before the end of this year.
In addition, MyoKardia starts to tap into $45 million in R&D funding once it moves beyond early human efficacy studies. The biotech is slated to lead worldwide development for MYK-461 under the Sanofi deal, with the pharma holding ex-U.S. commercialization rights for it.
The two Phase I trials that MyoKardia has just released data for were mostly concerned with safety, but they offered a hint at efficacy as well. The data offer a “dose-dependent reduction in contractility consistent with the magnitude of reduction” that the company expects will be sufficient to offer “meaningful clinical benefit,” although the results were not further detailed.
MYK-461 has been tested in three Phase I trials in 86 healthy volunteers and 15 HCM patients.
“The data suggest a dose-dependent reduction in excess cardiac contractility, the hallmark of HCM, that we believe will be clinically relevant,” said MyoKardia CMO Dr. Jonathan Fox in a statement. “Based on the scientific literature and our own research, we believe an agent that produces a modest reduction in contractility, for example, a relative reduction in left ventricular ejection fraction of 5% to 10%, has potential as a treatment for HCM patients.”
Excess left ventricular contractility underlies HCM and leads to symptoms including shortness of breath, reduced effort tolerance and chest pain. HCM increases the risk of atrial fibrillation that can lead to stroke or arrhythmias that can result in sudden cardiac death. oHCM, the indication for the upcoming Phase II trial for MYK-461, includes the further complication of an abnormally thickened heart muscle that obstructs the left ventricular outflow tract.
MYK-461 is an oral small molecule that allosterically modulates the function of cardiac myosin, the protein driving heart muscle contraction. MyoKardia was founded in 2012 by Third Rock Ventures to create novel therapeutics for heritable cardiomyopathies.
“The growing body of research continues to support MYK-461’s potential to change the course of this devastating disease,” said MyoKardia CEO Tassos Gianakakos. “We look forward to initiation of the PIONEER-HCM study later this year and to discussing the path to registration with the FDA in the coming months.”
The recent advance kicked MyoKardia’s share price up to a bit over $475 million.