MyoKardia raises $46 million to develop drugs for genetic heart diseases

MyoKardia has completed a $46 million Series B financing. The Series B financing included investments from Casdin Capital, Cormorant Asset Management, Perceptive Life Sciences, and BridgeBio. Proceeds will be used to develop drugs for genetic heart diseases.

In September 2012, Third Rock Ventures launched MyoKardia with $38 million. The company’s initial focus is on hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM). In September 2014, Sanofi cut a $245 million deal with MyoKardia to co-develop two programs for HCM and one for DCM.

HCM is the most common genetic heart disease, affecting one in 500 people. It is characterized by an abnormal thickening of the heart muscle, making it difficult for the heart to relax and refill with fresh blood between beats. DCM is a condition in which the heart becomes enlarged and weakened, and is unable to pump blood efficiently. It is estimated to occur in one in 2,500 people.

HCM and DCM are commonly caused by mutations in the sarcomeric genes. More than 20 years ago, Christine Seidman and Jonathan Seidman at Harvard Medical School, founders of MyoKardia, showed that a mutation (R403Q) in human β-cardiac myosin leads to HCM[1]. MyoKardia intends to develop mutation-specific dugs for patients.

MYK-461, MyoKardia’s lead product candidate, is a first-in-class, small molecule allosteric modulator of cardiac myosin. In HCM patients, mutant myosin leads to over-contraction of the heart muscle cells. MYK-461 was designed to correct the over-contraction driving HCM. In March 2015, MyoKardia initiated the Phase I trial of MYK-461.

MyoKardia’s approach is similar to what Vertex Pharmaceuticals (NASDAQ: VRTX) has done with ivacaftor, an approved drug for the treatment of cystic fibrosis caused by certain mutations in the CFTR protein.

[1] Cell. 1990, 62(5), 999-1006.

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