MyoKardia, Inc. (NASDAQ:MYOK) Q3 2019 Earnings Conference Call - Final Transcript

Nov 11, 2019 • 08:30 am ET

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MyoKardia, Inc. (NASDAQ:MYOK) Q3 2019 Earnings Conference Call - Final Transcript

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Q & A
Operator
Operator

[Operator Instructions] Our first question comes from Anupam Rama with JP Morgan, your line is open.

Analyst
Anupam Rama

Hey guys, thanks for taking the question. Just on the EF reduction observed here in MAVERICK I believe in the PIONEER study you saw EF dip below 50% at drug concentrations that were 1000 nanograms per mL. And here in MAVERICK you chose drug concentrations that are lower but it still appears that 5 patients kind of dip below 50%. How are you thinking about EF here heading into the 2Q EXPLORER results? Was there anything particular about these 5 patients' baseline characteristics worth noting when it come to EF.

Executive
Jay Edelberg

So in this study it was a dose-ranging study. So we specifically pushed the dose based solely on concentration without regard to any clinical response. And so therefore by definition we expected some patients to be at upper levels and that their ejection fraction may come down. And that's exactly what we learned. Though what this tells us now coming out of MAVERICK is that we have physiologic we have biomarkers that we can follow to guide our dosing in our future studies just like we're doing in EXPLORER.

Executive
Tassos Gianakakos

And Anupam just to add what we're seeing here really lines up with all of our modeling our preclinical modeling our earlier studies around PK and effect on EF. And the thing to keep in mind too because your question was specifically around thinking about EXPLORER. Look at that OLE data the EF is really hasn't moved at all for these patients. So dosing in the obstructed patients we feel really good about and MAVERICK is supporting our strategy to dose in EXPLORER. And separately as we think about moving forward in the nonobstructed patient population we feel really good that this data has given us the ability to identify a well-tolerated dose that's going to keep nonobstructed patients in the normal range.

Analyst
Anupam Rama

Great, thanks for taking our question.

Executive
Tassos Gianakakos

Thanks a lot.

Operator
Operator

Our next question comes from Marty Auster with Credit Suisse, your line is open.

Analyst
Marty Auster

Hey, Ron, thanks for the question. I had a couple. I was wondering if you could provide more detail on the placebo group performance in the ITP versus baseline on some of the functional end points you looked at NYHA and VO2 max? And then also I was wondering if you could maybe provide a little more information on the subgroup with greater diastolic dysfunction where some efficacy measures were observed. What's the size of that subgroup? And how was that defined?

Executive
Tassos Gianakakos

Marty so let me hit the placebo response rate. Yes this was a key takeaway that increased even further our confidence in EXPLORER. So we had modeled an estimated 25% placebo response. We're looking at the responder definition in EXPLORER and we saw 21%. And so this supports the assumptions in EXPLORER validating the power assumptions that we've got in that study. And we're feeling even better about it after MAVERICK. In terms of the subpopulations this I