With preclinical proof of concept that its lead candidate mavacamten acts via two distinct mechanisms to treat cardiomyopathy, MyoKardia Inc. is ready to accelerate a pipeline of preclinical programs that exploit the mechanisms separately for different genetic or phenotypically defined diseases.
At the November American Heart Association meeting, MyoKardia presented data showing that in addition to reducing excessive contractile strength in hypertrophic cardiomyopathy (HCM), mavacamten improves the heart’s ability to relax afterwards.
That provided justification for two of MyoKardia’s recently announced follow-on programs and additional discovery programs addressing contraction and relaxation separately.
Mavacamten is a small molecule allosteric modulator of cardiac myosin in Phase III testing to treat obstructive HCM and Phase II testing to treat non-obstructive HCM.
On Nov. 11, MyoKardia presented preclinical data showing an analog of mavacamten helped hearts recover better after contraction in a genetic minipig model of non-obstructed HCM. Specifically, the compound significantly increased ventricular volume as measured by