MyoKardia, Inc. (NASDAQ:MYOK) Q3 2019 Earnings Conference Call - Final Transcript
Nov 11, 2019 • 08:30 am ET
seen in the OLE study across diverse parameters are nothing short of remarkable. Tassos?
Thank you Mark and Jay for the data review. So before we open the line up to your questions let me summarize what these data mean for the big picture at MyoKardia and how it sets up our company over the next six to nine months. We're moving mavacamten forward in 2 patient segments in addition to obstructive HCM. As Jay mentioned MAVERICK gives us confidence to advance in nonobstructive HCM and in a subset of of HFpEF. Much like PIONEER guided our design of EXPLORER MAVERICK has generated the data we needed for nonobstructive patients including identifying the analogous biomarkers to LVOT gradient. We're ready to finalize our nonobstructive HCM plans and aim to discuss them with the FDA in the first half of next year. In parallel we'll be taking our first thoughtful step into an adjacent subgroup of HFpEF patients roughly estimated to be about 10% to 20% of all HFpEF bringing our precision medicine approach to one of the largest remaining areas of medical need. Our confidence in the successful EXPLORER trial has also gone up a lot with today's data. Our safety database just grew substantially and mavacamten continues to be consistently well tolerated.
We've looked at the placebo arm in MAVERICK and found that our estimates for placebo response in EXPLORER is spot on. So we're feeling really good about EXPLORER's design and powering assumptions. And our confidence in Mavacamten's efficacy in EXPLORER is supported by both studies today. The OLE of course in the same patient population and the subgroup data from MAVERICK tells us mavacamten is improving left ventricular compliance enabling the heart to fill at lower pressures which should help obstructive HCM patients as well. Finally while early the evidence is mounting that mavacamten may be enabling structural changes in the heart that could ultimately be disease modifying with multiple and diverse markers all improving and moving in concert towards normal. So let's take a look at where MyoKardia will be in 6 to nine months. For mavacamten we'll be through EXPLORER and moving towards our first NDA filing in obstructive HCM. The SRT study will be underway looking at obstructive HCM patients who have been referred for surgery.
The MAVERICK insights you heard about today will have positioned us for registration path and nonobstructive HCM and we'll have started a Phase II study in a targeted HFpEF our first non-HCM indication with mavacamten. For 224 we expect to be reading out data from our Phase I program and we'll be moving into Phase II study in HCM and/or targeted HFpEF. And for MYK-491 we'll be underway in a Phase II genetically defined DCM population and gearing up to drive a precision registration program in patients with diastolic dysfunction. And all the while we'll be mining the proprietary clinical insights from our studies including MAVERICK and EXPLORER which will enable important patient profiling and novel research programs.