MyoKardia, Inc. recently announced that Sanofi has notified the company it has elected to continue the global cardiomyopathy research collaboration formed in August 2014.
The terms of the research agreement provided Sanofi the option of either concluding the collaboration at year-end 2016 or extending the agreement. Per Sanofi’s decision to advance the collaboration, MyoKardia is now eligible for a $45-million milestone payment payable by January 31, 2017.
“MyoKardia and Sanofi share a passion for science and a commitment to patients. The continuation of this research collaboration provides valuable support for further innovation and development of critically important therapies for patients with serious cardiovascular diseases,” said Tassos Gianakakos, Chief Executive Officer.
The research and development agreement is one of the most significant commitments to heritable cardiomyopathies, and encompasses three MyoKardia programs. Two of these programs are focused on hypertrophic cardiomyopathy (HCM), and one targets dilated cardiomyopathy (DCM).
In September, MyoKardia outlined its expected path to registration for its lead product candidate MYK-461 in the initial indication of symptomatic, obstructive HCM, for which the company was granted Orphan Drug Designation by the US FDA. MyoKardia is studying MYK-461 in the Phase II PIONEER-HCM trial.
MyoKardia received a separate $25-million milestone payment from Sanofi in November 2016 for the filing of an Investigational New Drug (IND) application with the FDA for its MYK-491 program in DCM. MyoKardia intends to initiate a Phase I study of MYK-491 in healthy volunteers in the first half of 2017. In the United States, MyoKardia maintains commercial rights to MYK-461 and HCM-2 as well as co-promotion rights for MYK-491.
MyoKardia is a clinical-stage biopharmaceutical company pioneering a precision medicine approach to discover, develop, and commercialize targeted therapies for the treatment of serious and rare cardiovascular diseases. MyoKardia’s initial focus is on the treatment of heritable cardiomyopathies, a group of rare, genetically driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. MyoKardia has used its precision medicine platform to generate a pipeline of therapeutic programs for the chronic treatment of the two most prevalent forms of heritable cardiomyopathy — hypertrophic cardiomyopathy, or HCM, and dilated cardiomyopathy, or DCM. MyoKardia’s most advanced product candidate, MYK-461, is an orally administered small molecule designed to reduce excessive cardiac muscle contractility leading to HCM and has been evaluated in three Phase I clinical trials. MyoKardia is now studying MYK-461 in the Phase II PIONEER-HCM trial in symptomatic, obstructive HCM (oHCM), a subset of HCM. In April 2016, the US FDA granted MYK-461 Orphan Drug Designation for the treatment of symptomatic oHCM. MYK-491, the second clinical candidate generated by MyoKardia’s product engine, is designed to increase the overall force of the heart’s contraction in DCM patients by increasing cardiac contractility. MyoKardia intends to initiate a Phase I study of MYK-491 in healthy volunteers in the first half of 2017. A cornerstone of the MyoKardia platform is the Sarcomeric Human Cardiomyopathy Registry, or SHaRe, a multi-center, international repository of clinical and laboratory data on individuals and families with genetic heart disease, which MyoKardia helped form in 2014. MyoKardia believes that SHaRe, currently consisting of data from approximately 10,000 individuals, is the world’s largest registry of patients with heritable cardiomyopathies. MyoKardia’s mission is to change the world for patients with serious cardiovascular disease through bold and innovative science. For more information, visit www.myokardia.com.