Sanofi, MyoKardia Partner on Genetic Heart Disorders

(NASA.gov)

17 September 2014. The pharmaceutical company Sanofi and biopharmaceutical developer MyoKardia are collaborating on development and commercialization of three MyoKardia therapies for inherited heart diseases. The deal could earn MyoKardia as much as $200 million in milestone payments and equity investments from Sanofi.

MyoKardia, in South San Francisco, California, designs small-molecule therapies for two types of genetic heart disorders: hypertrophic cardiomyopathy and dilated cardiomyopathy that result from mutations in protein genes of muscles used in heart contractions. With hypertrophic cardiomyopathy, heart muscles become abnormally thick, making it more difficult for the heart to pump blood. In dilated cardiomyopathy, the heart’s left ventricle — the main pumping chamber — becomes enlarged, resulting in less pumping force than a healthy heart.

The company was started in 2012 by four academic researchers in heart muscle biology and cardiovascular genetics from Harvard Medical School, Brigham and Women’s Hospital, and University of Colorado, and received its initial financing of $38 million from Third Rock Ventures, a life sciences venture capital firm. In May, MyoKardia unveiled its Sarcomeric Human Cardiomyopathy Registry, or SHaRe, repository of de-identified clinical data on genetic heart disease, with data on some 5,800 patients, developed with researchers from 7 medical centers.

The agreement covers three MyoKardia therapies, two for hypertrophic cardiomyopathy and one addressing dilated cardiomyopathy. MyoKardia will be responsible for continued development of the three therapies through initial demonstrations of efficacy in patients, after which the two companies will share development costs of the hypertrophic cardiomyopathy drugs, while Sanofi covers further development costs of the dilated cardiomyopathy program.

MyoKardia will retain product product rights for the two hypertrophic cardiomyopathy therapies, while Sanofi has worldwide rights to develop and commercialize the dilated cardiomyopathy drug. The companies divide up geographic commercialization activities, with MyoKardia responsible for commercialization of the hypertrophic cardiomyopathy drugs in the U.S., and Sanofi responsible for areas outside the U.S. where it now operates. If further uses of the therapies emerge, Sanofi will have the option to co-promote either of the hypertrophic cardiomyopathy therapies in the U.S., while MyoKardia will have the option to co-promote the dilated cardiomyopathy drug in the U.S.

Also under the deal, Sanofi, based in Paris, paid MyoKardia an upfront payment of $45 million in licensing fees and equity investment. MyoKardia can qualify as well for up to $155 million in further payments for achieving milestones, research and development services, and further equity investments from Sanofi.

The agreement is part of a Sanofi initiative it calls Sunrise that seeks out partnerships with early-stage drug development projects that the company believes can benefit from investment and its commercialization expertise. In addition to MyoKardia, the Sunrise program has a collaboration with WarpDrive Bio, a developer of natural therapeutic products based on genomics.

Read more:

• Heart Assn Challenge Seeks Heart Disease Prevention Tools
• Spin-Off Company Developing Cardiac Drug Tests
• Biopharm Starts Genetic Heart Disease Clinical Database
• Heart Disease in Lab Recreated with Stem Cells, Chip Device
• Injectable Hydrogel Developed to Prevent Heart Attack Damage

Hat tip: FirstWord Pharma

*     *     *