MyoKardia, 23andMe to Create HCM Digital Community

October 25, 2018
MyoKardia, 23andMe to Create HCM Digital Community
Source: Tomekbudujedomek/Getty Images

Precision medicine developer MyoKardia said it will partner with 23andMe to create a digital community designed to enable patients to access information and research opportunities in hypertrophic cardiomyopathy (HCM).

Through their collaboration, whose value was not disclosed, the companies plan to launch next year a community designed to improve understanding by patients, and deepen their engagement and advocacy, in HCM. The community will allow 23andMe customers to access information and research opportunities in HCM.

More than 6,000 HCM patients are customers of 23andMe, according to MyoKardia.

The companies also plan to partner in deploying a survey intended to collect baseline and follow-up longitudinal data from people with HCM who have consented to participate in further research. MyoKardia said it intends with 23andMe to study the resulting set of de-identified, population-scale phenotypic and genotypic data, with the goal of gaining additional into HCM.

Summary results will be made available to patients through 23andMe’s platform, which serves the direct-to-consumer (DTC) genetic testing pioneer’s more than 5 million customers.

“By collaborating with 23andMe, we strive to share new insights into how HCM manifests across diverse patient groups through a unique combination of genotypic and phenotypic data. This important resource can help support patients as they navigate the challenges of daily life with HCM,” said Richey Neuman, M.D., MPH, FACP, VP of medical affairs at MyoKardia.

A form of HCM is the indication of MyoKardia’s lead therapeutic candidate: Mavacamten (formerly MYK-461) is under study in patients with symptomatic, obstructive HCM in the pivotal Phase III EXPLORER-HCM trial (NCT03470545).

Mavacamten is a novel, oral, allosteric modulator of cardiac myosin intended to reduce hypercontractility. The treatment is also being assessed in non-obstructive HCM in the Phase II MAVERICK-HCM trial (NCT03442764).

Launched in 2012 by Third Rock Ventures, MyoKardia holds U.S. rights to mavacamten, and has licensed ex-U.S rights to Sanofi as part of a broader licensing and collaboration agreement signed in 2014, and originally estimated to generate up to $200 million.

As of the end of the second quarter, MyoKardia disclosed in its latest Form 10-Q quarterly filing, it had received form Sanofi $129 million, consisting of:

  • $35 million in upfront cash.
  • $24 million in equity investments ($10 million upfront, $14 million subsequently).
  • A $25 million milestone payment for submitting an IND application to the FDA.
  • A $45 million milestone payment after Snofi extended the collaboration two years, to December 31, 2018.

The Sanofi agreement also covers MYK-491—for which MyoKardia retains a co-commercialization option in the U.S. while Sanofi holds ex-U.S. rights to the treatment candidate—and three preclinical programs: A second HCM candidate, being developed to lower cardiac muscle contractility in HCM patients; a second DCM candidate; and and LUS-1, designed to counteract muscle disruption resulting in impaired relaxation of the heart.

MYK-491 is designed to increase the overall extent of the heart’s contraction in DCM patients by increasing cardiac contractility. MYK-491 is being studied in a Phase Ib trial (NCT03447990) aimed at evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of MYK-491 in patients with DCM and stable heart failure.