Last June, MyoKardia launched its keenly anticipated pivotal trial for its genetically targeted lead heart drug. Data are expected next year, but partner Sanofi is not waiting around. On Wednesday, the San Francisco-based biotech said its French partner had elected to cease their partnership that was forged in 2014.
The drug, mavacamten, is expected to break new ground in heart disease, a field monopolized by pharma majors largely due to the long, arduous and expensive trials that are commonplace in heart drug development. Unlike other companies focusing on common heart disorders, MyoKardia’s lead experimental treatment is also targeting a so-far untapped condition — obstructive hypertrophic cardiomyopathy — in which a heart protein mutation forces the organ to squeeze more, thickening heart muscles and creating a cascade of consequences that can culminate in death.
In the second tranche of the drug’s Phase II trial in a small group of patients reported in March, the company said its low dose approach was largely successful across a crop of endpoints, except one for peak VO2 (exercise capacity measured by an increase in oxygen consumption).
MyoKardia’s approach to research is to develop drugs for genetically defined patient groups, which is also reflected in its second program, MYK-491, under development for dilated cardiomyopathy. The Sanofi partnership inked in 2014 involved the development of up to three programs through discovery and into clinical development for the treatment of hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM).
On Wednesday, MyoKardia said it had regained the global rights to all the programs (including mavacamten and MYK-491) under the research and collaboration agreement with Sanofi, which will not be extended beyond the initial research term, which ended on December 31, 2018. The biotech’s $MYOK shares were down about 8.5% in early trading.
As part of the deal, MyoKardia received roughly $230 million in funding from Sanofi, and has advanced mavacamten from preclinical development into a late-stage pivotal study for the treatment of HCM, and MYK-491 from discovery to a Phase II proof-of-concept study in patients with DCM.
Meanwhile, Phase II data for mavacamten in non-obstructive HCM are expected in the second half of this year and Phase IIa proof-of-concept data for MYK-491 in DCM are expected before the end of 2019.
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