By selectively modulating both T-cell and macrophage/monocyte inflammatory disease mediators,  Acetylon believes that highly selective inhibitors of HDAC6 may be successfully developed to become first-in-class members of the next generation of small molecule disease modulating anti-rheumatic drugs (“DMARD”s), with enhanced efficacy and safety versus current, costly biologic drug alternatives.  Acetylon is currently advancing orally bioavailable, highly selective, potent inhibitors of HDAC6 into late stage preclinical development. Acetylon and its scientific advisors are also exploring the potential for selective inhibition of other Class II HDACs, for the treatment of inflammatory diseases.  In particular, HDACs 7 and 9 have been implicated in inflammatory disorders such as rheumatoid arthritis and inflammatory bowel disease.

Autoimmune diseases are a major and growing public health problem.  In the United States, rheumatoid arthritis and inflammatory bowel disease and severe psoriasis affect millions of patients each, and there are an estimated 400,000 cases of multiple sclerosis.  Most currently available treatments are biologic drugs, which are typically very costly, must be administered by injection, and have been associated with significant risks including FDA “black box warnings” for opportunistic infections and secondary cancers.  Despite these limitations, biologic drugs for these indications currently generate more than $20 billion in annual sales worldwide.  There is a clear need for new, targeted oral drugs that demonstrate effectiveness in slowing or reversing the progression of autoimmune disease and exhibit improved safety and administration profiles.