MyoKardia, Inc. (NASDAQ:MYOK) Q3 2019 Earnings Conference Call - Final Transcript
Nov 11, 2019 • 08:30 am ET
tolerability we've achieved. In addition to demonstrating safety we identified a patient profile with diastolic dysfunction that we believe most likely to achieve benefit from mavacamten. This is a big deal. It's what MyoKardia was formed to do. To give some perspective 3 million people in the U.S. have diseases of diastolic dysfunction referred to as HFpEF who historically have been addressed as a single group and managed in an undifferentiated way with no approved therapies. With data emerging from MAVERICK we believe we can now subtype these patients both those with HCM and those with HFpEF. As a result we'll be advancing Mavacamten's development both in nonobstructive HCM as well as in our first expansion beyond HCM into a well-defined subgroup of patients with HFpEF in a precision-efficient fashion.
Turning to the PIONEER-OLE study. At one year of doses mavacamten safety and tolerability profile remains solid and the treatment benefits are sustained gradient is reduced EF remains above normal with little change from baseline. And patients are feeling better and we're seeing a wide range of biomarkers moving towards normal levels supportive of important improvements in their hearts. Beyond our decisions to advance in nonobstructive HCM and expand into HFpEF the data and insights from both studies have also increased our confidence in EXPLORER in a number of ways. First the safety and tolerability in MAVERICK was very consistent with what we've seen to date. MAVERICK also gave us an opportunity to confirm our expectations for anticipated placebo response and further validate our powering assumptions in EXPLORER. The evidence of long-term benefit and potential for mavacamten to slow or reverse HCM's progression in our obstructive patient population continues to grow. And the diastolic disease benefit observed in MAVERICK adds to our belief that we will see significant clinical benefit in EXPLORER. I know we're excited to get into it.
So let me hand the call over to Jay who will take us through the top line data from our MAVERICK Phase II trial in nonobstructive patients. Jay?
Thank you Tassos. And good morning to everyone. I'm excited to be here today to talk about the top line results from MAVERICK. Hypertrophic cardiomyopathy or HCM is a chronic progressive disease that changes over time. While some people with HCM might not experience much in the way of symptoms others struggle with ordinary activities and far too many develop atrial fibrillation stroke heart failure and sudden cardiac death. Both the obstructive and nonobstructive forms of the disease genetic mutations that lead to excessive contractions are the same but the ways that the hypertrophy or thickening of the heart muscle manifest can vary. In obstructive patients we typically see significant thickening of the septum near the mitral valve that results in an obstruction to blood flow in the left ventricular outflow track. In nonobstructive HCM the thickening of the heart muscle tends to be more symmetric and the disease manifests are driven by the inability of the left ventricle to expand and