Third Rock’s MyoKardia Gets $38M for Genetic Forms of Heart Disease

9/20/12Follow @xconomy

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a close connection in Boston to Christine Seidman, a former postdoc in his lab at UCSF, who he says later went on to “break open this field,” and is now a Howard Hughes Medical Institute investigator collaborating with her husband Jonathan at Harvard Medical School.

Treatment of heart disease has made strides over the decades with now-generic medicines that lower high blood pressure and reduce LDL cholesterol. While those drugs have been prescribed to millions of people, cardiologists haven’t been able to capitalize on deep understanding of the genetic roots of the disease in the same way cancer physicians or other specialists do, Homcy says.

That’s beginning to change, as researchers have been able to identify changes as subtle as a single amino acid that can have an impact on how the sarcomere, the basic unit of heart muscle, functions, Homcy says. The founding team of advisors will help take this work to another level, by enabling MyoKardia to develop new assays and protein expression tests which should provide a good understanding of the biology of disease, which chemists can then use to make specific drugs.

There are always a lot of unknowns in a project at this early stage of research. For starters, MyoKardia will want to know just how many different genetic variations are really the major culprits leading to these forms of heart disease. Scientists currently believe there are “hundreds,” Homcy says.

Rather than make a hundred different drugs for 100 genetically distinct diseases, MyoKardia hopes to categorize the genetic diseases into about five to seven different “buckets,” Homcy says, which would each have their own specific drug. Like Cambridge, MA-based Vertex Pharmaceuticals (NASDAQ: VRTX) has done with ivacaftor (Kalydeco), a drug for a rare form of cystic fibrosis, MyoKardia plans to start in a well-defined genetic population, and eventually develop more drugs and more combinations of drugs to help more patients, Homcy says.

Homcy said he doesn’t believe there are any competitors, even in Big Pharma, taking a similar approach to the genetic forms of heart disease. One other company, South San Francisco-based Cytokinetics (NASDAQ: CYTK), which Homcy used to serve as a board member, is working on drugs to improve muscle contractility, but is focused more on the muscle tissue than the genetics, Homcy says. Spudich is a co-founder of both MyoKardia and Cytokinetics.

MyoKardia is currently based out of Third Rock’s San Francisco office, and will be looking for space to grow in South San Francisco this fall, a spokeswoman says. Homcy, a co-founder of Portola Pharmaceuticals and a former head of R&D at COR Therapeutics, says he’s only doing the CEO gig part-time. As MyoKardia looks to grow, one of his important early tasks is to find a permanent CEO to replace him. “I’ll stay as long as necessary,” Homcy says. “But this company really needs a full time management team. There’s so much to do.”

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