When Boston-based biotech startup Acetylon Pharmaceuticals began operations in 2008, it grabbed attention for having attracted a big-named backer: the Kraft Group, the Foxborough, MA, holding company founded by New England Patriots owner Robert Kraft. Acetylon ultimately raised nearly $40 million from Kraft, a group of unnamed individuals, and, most recently, the Leukemia & Lymphoma Society. It’s enough to take Acetylon through the first two phases of human testing for its lead compound—a multiple myeloma drug that the company started dosing patients with last week.
Acetylon is one of many companies working on a class of drugs that inhibit histone deacetylases (HDACs), which are enzymes that regulate gene expression and that play a role in many cancers. The HDAC inhibitors that are on the market today are known as “pan-HDAC inhibitors,” because they target all 11 HDAC enzymes. Acetylon’s drug, called ACY-1215, only targets one: HDAC6.
Actylon CEO Walter Ogier believes that selectivity may give ACY-1215 an important edge. “HDAC inhibitors have a lot of activity,” he said during a mid-September interview at Acetylon’s brand-new Seaport Center office. That activity includes shutting down some normal cellular activities, which can result in side effects ranging from fatigue to nausea to anorexia. “Patients have a really lousy quality of life and find it hard to stay on drug regimens,” he says. “The opportunity we have is to maintain or improve on the anti-cancer effectiveness by being more selective.”
HDAC6 is involved in a process by which diseased cells destroy and dispose of damaged proteins. This enzyme is over-produced in some cancer cells, particularly in patients who become resistant to bortezomib (Velcade), a blockbuster