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Expert Interview

Slingshot members are talking to an expert! The topic is:

Discussing the treatment landscape for hypertrophic cardiomyopathy and MyoKardia's Mavacamten data in obstructive HCM

Ticker(s): MYOK

Who's the expert?

Institution: Stanford

  • Cardiologist and physician Scientist at Stanford Amyloid Center with a focus on Advanced Heart Failure and Transplant Cardiology.
  • Currently manages 200 patients with ATTR amyloidosis and has published numerous papers on this topic.
  • Research focuses on amyloidosis, transplant immunology, mechanical circulatory support, and non-ischemic cardiomyopathies.

Interview Questions

The goal of this call is to better understand the current treatment landscape of hypertrophic cardiomyopathy, get your view on the recent Myokardia data in obstructive HCM, and hear how you might use mavacamten if approved.

Added By: joe_mccann

Can you please start by giving us a background on your clinical practice? How many patients do you treat? How many have HCM? What % is obstructive vs non-obstructive?

  • Can you describe the typical obstructive HCM patient, how do they present, how symptomatic are these patients, and how are they currently treated?
  • What % of patients are on beta blockers and/or calcium channel blockers? How effective are beta blockers and calcium channel blockers?
  • Where is the biggest need for improvement on top of current treatments?

Added By: joe_mccann

Can we get your high level view of the data from the EXPLORER study? How did the result compare to your expectations?

  • How does the baseline characteristics on slides 5 and 6 compare to your typical obstructive patient?
  • How meaningful is the 37% response for mavacamten on the primary endpoint vs. 17% for the placebo patients?
  • The primary endpoint of the study was a composite endpoint requiring either a 1.5 ml improvement on peak VO2 and a 1 class improvement on NYHA or a 3 ml improvement in peak VO2 and no worsening in NYHA class. How clinically meaningful is this endpoint to patients and physicians?

Added By: joe_mccann

Can we go through the secondary endpoints on slide 8? Which here are the most meaningful to you as a prescriber or as a patient?

  • The drug arm saw improvements across the board on LVOT gradient, peak VO2, NYHA class, KC CM questionnaire, and shortness of breath.
On slide 10 the company goes the in more detail the results for LVOT obstruction – how meaningful is getting 74% of patients below 50 and 57% below 30 on post-exercise LVOT?The company points to 27% “complete response” vs. 1% for placebo, defined as NYHA class 1 and obstruction reduced below 30. How impressive is getting these type of patient in to that sort of response?Did anything in the safety on slide 13 stand out to you? How important was no signal of reduced ejection fraction for the drug?How would you sum up the data? On a scale from 1-10 how excited are you for mavacamten in obstructive HCM?

Added By: joe_mccann

Commercial Opportunity:

  • How would you expect to use mavacamten if approved? What % of your diagnosed obstructive patients would be eligible for treatment and then how many would you recommend the therapy for?
  • What is your expectation for how quickly the commercial uptake would ramp? Is this something you would call patients in for to get them started on or would you wait for whenever your next checkup to recommend? How frequently do you see your patients currently?
  • The company estimates there are around 500,000 HCM patients in the US with about 100,000 diagnosed. Further with 2/3s being obstructive and 1/3 being non-obstructive. Do those estimates seem reasonable? Do you believe it is as underdiagnosed as Myokardia suggests? Could a new approved treatment drive higher diagnosis rates?

Added By: joe_mccann

Other questions:

  • The company is also developing mavacamten for non-obstructive HCM with a focus on the subgroup of patients with elevated filling pressure and elevated troponin. They reported data from the phase II MAVERICK study recently.
  • What did you think of the MAVERICK data? Is there a role for this drug in non-obstructive in the subgroup of patients they identified?
  • What would you want the phase III endpoint to be for non-obstructive? Is change in pro-BNP sufficient or what other endpoints would you want to see?
  • Heart failure with persevered ejection fraction patients is another indication the company is exploring for mavacamten. Similar to non-obstructive, they are focusing on a subgroup of patients with elevated filling pressure and elevated troponin. Do you see a role for this drug in the right heart failure patient?
  • Is there anything else being developed in for cardiac disease that you are excited about?

Added By: joe_mccann

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