A small, groundbreaking cardio study pushes MyoKardia to a pivotal showdown — shares soar

MyoKardia $MYOK has come up with the proof-of-concept efficacy data that the South San Francisco-based biotech was looking for in its small but potentially groundbreaking cardio study. And now it will explore bypassing one element in its development plan and see whether the FDA will sign up on a straight shot at a pivotal study that could point the company down a straight path to the marketplace.

Tassos Gianakakos

MyoKardia’s shares rocketed up 83% as investors took a look at the numbers and its plans for its drug mavacamten.

The field is currently dominated by a few major drugs looking to influence the course of heart disease for large numbers of patients. While raising the bar on safety and efficacy in heart disease is an obstacle course rarely navigated by anyone outside of giant pharma, this biotech is following a game-changing molecular strategy with a rifle-shot approach at disease.

MyoKardia’s initial focus on heart disease centers on an ailment called obstructive hypertrophic cardiomyopathy, where a mutation in heart proteins forces the heart to squeeze more, thickening heart muscles and creating a cascade of effects and symptoms that can lead to afibrillation and death.

“By targeting the motor protein in the heart, offsetting the mutation, you’re bringing the force of the squeeze back to normal,” MyoKardia CEO Tassos Gianakakos tells me. “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.”

Outcomes in the field take a fortune to nail down, but MyoKardia’s concentrated on post-exercise peak left ventricular outflow tract, or LVOT, gradient from baseline to week 12, in 11 patients who were recruited for the study. One of those patients was forced out due to episodes of atrial fibrillation possibly linked to the requirement that the patients in the first cohort wash out beta blockers that are used to keep the condition

But for the 10 who completed the dosing, the baseline measure of 124.9 plunged to 18.9 — below the diagnostic threshold for the disease in 8 of 10 patients — while peak VO2  dropped more than the 10% to 15% level than the biotech had told analysts to watch out for.

“While it is 10 patients,” notes the CEO, “the treatment is massive.”

As a result of the data, and the efficacy on peak VO2, which is the planned pivotal endpoint, Gianakakos is now planning to meet with the FDA and see if he can bypass a Phase IIb and go straight to a registration study, cutting as much as 18 months out of the development timeline.

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.

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