August 7, 2017
By Mark Terry, BioSpace.com Breaking News Staff
South San Francisco – MyoKardia simultaneously released positive data from a Phase II clinical trial and its second-quarter financials.
On the financial side, it announced collaboration and license revenue for the quarter of $5.6 million compared to $3.5 million in the same period last year. For the first half of the year, collaboration and license revenue was $11.3 million compared to $7.1 from 2016. R&D expenses for the quarter were $13.7 million up from $9.3 million in the same period in 2016. They were primarily driven by clinical trials, including the Phase III PIONEER-HCM trial of mavacamten and the Phase I trial for mYK-491 and related expansion of R&D staff and contract research expenses.
As of June 30, MyoKardia reported cash and cash equivalents of $117.3 million. The company believes that with its cash and expected payments from Sanofi under a collaboration agreement, it has enough money to fund operations into at least 2019.
Otherwise, the company reported positive top-line data from the first patient cohort of PIONEER-HCM, its phase II clinical trial of mavacamten in patients with symptomatic, obstructive hypertrophic cardiomyopathy (oHCM). Patients showed a statistically significant improvement in the primary endpoint, change in post-exercise peak left ventricular outflow tract (LVOT) gradient from baseline to week 12. They also showed positive secondary endpoints, including peak oxygen consumption and New York Heart Association class.
The company also announced that its second, low-dose cohort had completed enrollment. Based on the data to-date, and discussions with the U.S. Food and Drug Administration (FDA), it is planning its next study, EXPLORER-HCM, to be a pivotal study, which it hopes to launch by the end of the year. By bypassing Phase IIb, it could cut up to 18 months from the developmental timeline.
Symptomatic, obstructive hypertrophic cardiomyopathy occurs when the heart muscle cells enlarge, blocking blood flow. It is a common cause of sudden cardiac arrest.
“We are positioned for a successful second half of 2017 with several key clinical and regulatory developments expected on the heels of the positive topline data announced today from PIONEER-HCM,” said Tassos Gianakakos, MyoKardia’s chief executive officer, in a statement. “We have made great progress across our entire pipeline, both clinical-stage and research-stage programs, that strengthens our confidence in MyoKardia’s unique approach to treat heritable cardiomyopathies. We look forward to sharing important details about these programs throughout the remainder of this year.”
Gianakakos told John Carroll of Endpoints News, “By targeting the motor protein in the heart, offsetting the mutation, you’re bring the force of the squeeze back to normal. The heart relaxes, that obstruction moves out of the way…. By dampening the mutational effect, the heart remodels. We’d love to see that heart look like a normal heart.”
Company shares rocketed on the news, jumping 31.8 percent in premarket trading this morning. are currently trading at $31.55. On Friday, August 4, shares traded at $17.15.
Carroll writes, “I’ve covered MyoKardia right from the Series A with an idea of starting a biotech that would break things down in Cardio R&D on a genetic basis. Gianakakos estimates that there are some 400,000 patients with this particular condition, about 65,000 of whom are symptomatic of the disease. That’s the kind of target that a small, focused biotech company can hit with a small sales force. And he believes that if the FDA signs off on his pivotal plans, he can get started later this year on a 2-year effort.”
