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Cardiomyopathy Drug project

Micar21 Ltd has a relatively long history in cardiomyopathy research. At the beginning of 2011 we established a project for the discovery of a potential drug for the untreatable heart disease Hypertrophic cardiomyopathy (HCM). In November 2011 we signed an EU funded contract (BG161PO003-1.1.06-0031) for the discovery and lead optimization of HCM cardiac modulators of the mutated MYH7 gene (myosin protein) inhibiting the increased myosin-actin interactions provoked by these mutations. A new class of compounds was selected during this period. However, despite the leading role of Micar21 in the above developments, we participated in this project only as a major partner to another company, which went bankrupt and the project was closed in it initial phase.

Meanwhile, in 2012, the USA-based company MyoKardia Inc. was established with the same purpose. It grew up fast, and, with the partnerships of Sanofi Inc ($200 million contract), MYH7 inhibitor MYK-461 begun to be evaluated in Phase 1 clinical trials on humans. This potential drug will be tested in patients with a specific mutation in the MYH7 gene, but it is still unknown whether and to what degree it would be efficient in patients with different mutations. Considering that only in the above mentioned gene hundreds of mutations have been identified so far, the urgent need for development of several, not a single one, potential drugs remains evident. Moreover, the mutations in the MYH7 linked to the HCM disease are only a part of the many defective genes which have been detected to provoke HCM, and, notably, the occurrence of these mutations varies significantly among the population. For instance, though the MYH7 is the most frequently mutated gene in USA, this is not valid for the patients in most of the EU countries. Thus, specific potential drugs targeting other genes might be required.

Considering the facts above, and having undergone a major company reorganization, with a significant reorganization of the company, Micar21., reopened its HCM project in December 2014. We already have a preliminary agreement with a highly reputable cardiology clinic, and we are looking for partners in the development of this project.

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