Dr Jay Edelberg Discusses PIONEER-OLE and Design of EXPLORER-HCM in Patients With Obstructive HCM

Results of the PIONEER-OLE study has shown promising results of mavacamten for patients with obstructive HCM that will be further evaluated in our pivotal phase 3 EXPLORER-HCM trial, said Jay Edelberg, MD, PhD, senior vice president of Clinical Development at MyoKardia.

Transcript

What are the most important takeaways from PIONEER-OLE from a health system perspective?

I'm really excited about what PIONEER-OLE is telling us about mavacamten, and more importantly about the promise for patients with obstructive hypertrophic cardiomyopathy or HCM. What we've seen, first of all, is that our patients are able to get their symptoms improved quickly. They go from a New York Heart Association class 2 or class 3, often down to class 1 relatively quickly and stay there for the duration of the trial that we've seen up to 1 year. We've seen that the gradient which is a manifestation of how much that obstruction is, comes down very, very quickly. More than that we're seeing the patients are continuing to do well with their activities of daily living, as measured by the New York Heart Association, and we're seeing some really promising signs. We're seeing that the septum, which is thickened in these patients, is actually getting smaller at progressive points that we're measuring—really exciting what we're seeing.

Can you discuss the design and timeline of EXPLORER-HCM?

EXPLORER-HCM is our pivotal phase 3 program for mavacamten in obstructive hypertrophic cardiomyopathy. In this study, patients are randomized evenly between being treated with mavacamten and placebo. Patients are symptomatic with obstructive HCM, which means that they have an elevated gradient, either at rest or with exercise. They're treated with mavacamten or placebo for 30 weeks and our primary endpoint is to look at improvements in their symptoms and their function, as measured by New York Heart Association class, as well as peak VO2.