MyoKardia (MYOK) Will Update on MYK-461, MYK-491, Precision Pipeline at R&D Day

September 21, 2016 7:04 AM EDT
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MyoKardia, Inc. (Nasdaq: MYOK) will highlight the expected path to registration for its lead product candidate MYK-461 in the initial indication of symptomatic oHCM and its plans to progress MYK-491 into the clinic for DCM. The Company’s scientific leadership will also provide further insights into MyoKardia’s precision medicine and pipeline strategy enabled by its product engine. The updates will be presented at MyoKardia’s inaugural R&D Day today in New York.

“We believe our precision medicine strategy and strong scientific foundation will continue to drive novel therapies aimed at addressing important treatment needs for patients with serious cardiovascular diseases,” said Tassos Gianakakos, chief executive officer. “The encouraging early results from our two most advanced programs have supported our enthusiastic advancement of both into the next stage of development.”

“Our recent discussions with the U.S. Food and Drug Administration (FDA), including confirmation that mortality-based efficacy endpoints will not be required for registration, represent an important milestone for MyoKardia, allowing us to confirm MyoKardia’s plans for registration of MYK-461 in oHCM.”

Mr. Gianakakos continued, “We are also excited to share more detail around our MYK-491 clinical program for the treatment of DCM. MYK-491 is the second clinical candidate generated by MyoKardia’s product engine and has shown promising preclinical data that supplies us with a compelling rationale to support advancement into Phase 1 in the first half of next year in a disease area desperate for more therapeutic options.”

MYK-461 in oHCM

Conclusions from Recent Regulatory Interactions

Additionally, MyoKardia will report key conclusions from recent regulatory interactions, which clarify the potential clinical development pathway in oHCM:

  • Mortality-based efficacy endpoints will not be required for registration.
  • Improvement in functional capacity and/or clinical symptoms are suitable endpoints for registration.
  • A single Phase 3 pivotal study demonstrating significant improvement in functional capacity or symptoms may be adequate for approval.

Further Detail on PIONEER-HCM

MyoKardia has initiated a Phase 2 study, PIONEER-HCM, which is an open-label, single-arm pilot study of MYK-461 in patients with symptomatic oHCM, an indication for which the FDA granted MYK-461 Orphan Drug Designation in 2016. Other details include:

  • First outpatient study with MYK-461 in target indicated population for anticipated treatment duration.
  • Primary endpoint is to assess level of reduction in left ventricular outflow tract (LVOT) gradient over 12 weeks of drug exposure.
    • Based on published studies and MyoKardia research, LVOT gradient reduction is expected to improve symptoms and functional capacity.
    • PIONEER-HCM will begin to characterize relationships among reductions in contractility, LVOT gradient and endpoints including functional capacity and clinical symptoms.
  • Topline data is expected to be released in the second half of 2017.
  • Study is designed to enable progression to larger Phase 2 study to finalize dosing and endpoints before subsequent single pivotal study.

Phase 1 Data

Duke University School of Medicine Professor Andrew Wang, M.D., a participant in the MYK-461 investigational program, will review Phase 1 data in detail, including additional information around the two patients with LVOT obstruction dosed in the Company’s Phase 1 single ascending dose (SAD) study in HCM patients and additional pharmacokinetic, or PK, and pharmacodynamic, or PD, data from the SAD and multiple ascending dose (MAD) studies of MYK-461.

MYK-491 in DCM

MYK-491 is the Company’s candidate for several well-defined sub-groups of DCM, including genetic DCM, designed to return the diseased DCM heart to normal contractility by increasing the overall force of the heart’s contraction. Similar to MYK-461, MYK-491 targets the underlying biomechanical defect caused by sarcomere mutations and has been designed to be potentially disease modifying.

Other updates on the MYK-491 program include:

  • Preclinical research demonstrates across multiple animal model systems that MYK-491 is able to achieve significant, dose-dependent increases in stroke volume with minimal effect on diastolic function.
  • MyoKardia intends to initiate a Phase 1 SAD study in the first half of 2017, with topline results expected in the third quarter of 2017.

Precision Medicine Platform and Vision 2020

During the R&D Day presentations, MyoKardia leadership will also discuss key elements of the Company’s strategy, including its precision medicine approach and product engine and future objectives for its research activities.

“We are working hard at MyoKardia to build a company that represents much more than two product candidates. The efficiency with which these drugs have moved into and through clinical trials and their promising early results are a testament to our team and to our precision approach. Through the dedication of our employees, the commitment of our founders and the cardiomyopathy community’s support, in just four years we have built a durable competitive advantage in our field, which forms the basis for our Vision 2020 strategy,” said Mr. Gianakakos.

“We look forward to sharing more details on how we hope to fulfill our mission to change the world for patients with serious cardiovascular disease through bold and innovative science.”

In addition to Mr. Gianakakos and Dr. Wang, R&D Day presenters include:

  • Jonathan Fox, M.D., Ph.D., chief medical officer; Michael Graziano, Ph.D., vice president, research biology; Robert McDowell, Ph.D., senior vice president, drug discovery
  • Christine Seidman, M.D., a MyoKardia co-founder and professor of genetics and the Thomas W. Smith professor of medicine at Harvard Medical School and Brigham and Women's Hospital
  • Srihari Naidu, M.D., director, Cardiac Catheterization Laboratory, Interventional Cardiology Fellowship Program and HCM Treatment Center at Winthrop University Hospital
  • Daniel Judge, M.D., associate professor of medicine, Johns Hopkins University School of Medicine; Medical Director, Center for Inherited Heart Disease

R&D Day Webcast

To access the live webcast of MyoKardia’s R&D Day presentation, please visit the “Events & Presentations” page within the Investors & Media section of the MyoKardia website at A replay of the webcast will be available on the MyoKardia website for 30 days following the event.

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