Acetylon Pharmaceuticals is unveiling its technology today after closing a $7.25 million Series A round of financing to develop novel drugs to treat multiple myeloma and rheumatoid arthritis, according to the company. Many of the startup’s backers have close ties to the Dana-Farber Cancer Institute in Boston—including the company that owns the NFL’s New England Patriots, Acetylon president and CEO Walter Ogier tells Xconomy.
The biotech startup is researching drugs designed to block a specific enzyme involved in cancer and many other diseases, based on discoveries by scientists affiliated with Dana-Farber and Harvard University. The firm has drummed up investments from an interesting mix, including Acetylon co-founder and chairman Mark Cohen, a technology entrepreneur who serves on the board of Dana-Farber, and the Kraft Group, the Foxborough, MA, holding company founded by Patriots owner Robert Kraft, who is also a major supporter of Dana-Farber, according to Ogier.
Acetylon was formed last year to develop drugs that target one among a class of enzymes known as HDACs (short for histone deacetylases), which are involved in regulating the expression of certain genes in most types of cells. Interfering with these enzymes can interfere with gene expression, making them popular targets among pharma and biotech companies developing drugs to treat cancer and other diseases. In fact, the leading HDAC inhibitor drug on the market is Whitehouse Station, NJ-based drug giant Merck’s (NYSE:MRK) vorinostat (Zolinza) for treating the immune system cancer called lymphoma.
The problem with many HDAC inhibitors is that they can disrupt the function of healthy cells, causing side effects such as vomiting, fatigue, and even heart attacks, said Acetylon’s Ogier, who has joined the company as its first and only employee. Acetylon aims to avoid such side effects by developing drugs that home in on one specific enzyme known as HDAC6, as opposed to earlier drugs in this class that inhibit the function of many of the 18 known enzymes of this type.
“This is an interesting company because we have some new molecules that are well on their way to being in clinical trials,” Ogier said, “but we also have a very powerful discovery platform.”
For you science junkies, here’s why blocking the HDAC6 enzyme could be useful in treating diseases: in studies, the enzyme has shown to play a key role in functions in diseased cells that destroy and dispose of damaged proteins. But when the enzyme is inhibited, these so-called misfolding proteins pile up in diseased cells to a point where the cells self-destruct. This could be useful in treating certain illnesses.
The way Ogier tells it, the company grew out of a research collaboration between Dana-Farber physician and researcher Kenneth Anderson, an expert on multiple myeloma, and Harvard chemist Stuart Schreiber, who has co-founded such well-known Massachusetts biotechs as Vertex Pharmaceuticals (NASDAQ:VRTX), Ariad Pharmaceuticals (NASDAQ:ARIA), and Infinity Pharmaceuticals (NASDAQ:INFI), as well as biotech startup Forma Therapeutics. Anderson, a scientific founder of Acetylon, and Schreiber were among the lead researchers in a study that showed that a novel compound that inhibited HDAC6 could play a role in killing blood cancer cells. Other scientific co-founders of the biotech are Dana-Farber and Harvard Medical School researcher James Bradner and Harvard Medical School scientist Ralph Mazitschek, who is based at Massachusetts General Hospital in Boston. Bradner and Mazitschek developed the startup’s technology for identifying which HDAC enzymes are blocked by certain compounds. (Editor’s note: A previous version of this paragraph described Schreiber as a scientific founder of Acetylon, based on a press release from the company that erroneously described him as such. He is not a scientific co-founder of the startup.)
Acetylon, which is initially focused on multiple myeloma and rheumatoid arthritis, expects to zero in this year on its first compound to advance into human clinical trials, Ogier said. The firm, which now operates virtually, plans to soon move into lab and office space most likely in Cambridge, MA, and grow to about 10 employees over the next year and a half. He believes that the company’s technology could also lead to the discovery of drugs for psoriasis, Parkinson’s disease, and sickle cell anemia, to name a few.
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