Clinical trial results of ACY-1215 in multiple myeloma published in scientific journal of ASH

Acetylon Pharmaceuticals today announced the publication of favorable results of preclinical safety and efficacy testing of its oral selective HDAC6 inhibitor drug candidate, ACY-1215, in multiple myeloma. In studies conducted at the Massachusetts General Hospital and at the Dana-Farber Cancer Institute, ACY-1215, when administered either as a single agent or in synergistic combination with the first-in-class proteasome inhibitor drug bortezomib (Velcade^®, Takeda Millennium Pharmaceuticals), demonstrated effectiveness in two in vivo disease models of multiple myeloma as well as against drug-resistant multiple myeloma patient cells. Inhibition of HDAC6 results in inhibition of a critical intracellular mechanism for degradation of misfolded proteins called the "aggresome" pathway, which was demonstrated to result in potent killing of myeloma cells via apoptosis (programmed cell death) while being well tolerated and non-toxic to normal cells. The results have been published in a special on-line edition of the scientific journal of the American Society of Hematology, Blood, (116(21), 2997, Nov. 19, 2010) and were presented yesterday at the Society's 52^nd Annual Meeting in Orlando, Florida.

“Based on these new data, we are encouraged that the selective HDAC6 inhibitor, ACY-1215, in addition to overcoming drug resistance in multiple myeloma, should also overcome the substantial side effect profile that is associated with current first-generation, non-selective HDAC inhibitors”

"These preclinical efficacy and safety results provide the rationale for the initiation of human clinical trials of ACY-1215 in multiple myeloma," stated Noopur Raje, M.D., Associate Professor of Medicine at Harvard Medical School and Director of the Center for Multiple Myeloma at the Massachusetts General Hospital Cancer Center. "We are particularly encouraged by the effectiveness we have observed in several different disease models of this challenging cancer, including overcoming bortezomib resistance in multiple myeloma patient cells by synergistic combination of this selective HDAC6 inhibitor."

Acetylon is currently focused on the development of potential drug candidates based on next-generation Class II-selective HDAC inhibitors. The Class IIB enzyme, HDAC6, has emerged as an important target in inflammatory disease, neurologic disease and broadly in cancer. Acetylon Pharmaceuticals believes that its next-generation, selective HDAC inhibitor compounds may accomplish enhanced clinical utility by reducing or eliminating the debilitating and sometimes life-threatening side effects associated with the current first-generation of non-selective HDAC inhibitors.

"Based on these new data, we are encouraged that the selective HDAC6 inhibitor, ACY-1215, in addition to overcoming drug resistance in multiple myeloma, should also overcome the substantial side effect profile that is associated with current first-generation, non-selective HDAC inhibitors," commented Walter Ogier, President and Chief Executive Officer of Acetylon. "We are currently focusing our efforts on advancing ACY-1215 and other promising selective HDAC inhibitors into human clinical trials."