Therapeutic Programs

Acetylon has developed a pipeline of optimized, orally administered, HDAC6 selective compounds representing several, chemically distinct and separately patentable chemical “scaffolds” or families. Acetylon’s first clinical candidate, the selective HDAC6 inhibitor “ACY-1215,” is targeted for the treatment of hematologic and solid tumor cancers and is currently being entered into US Phase I clinical testing for the treatment of multiple myeloma. Acetylon is also exploring in preclinical studies several additional potential cancer indications, including certain lymphomas, leukemias, and solid tumors, where selective inhibition of HDAC6 is anticipated by Acetylon to potentially provide a favorable anti-cancer effect as a single agent and/or in combination with existing anti-cancer agents.

Non-oncology indications

Acetylon is currently advancing other orally bioavailable, highly selective, potent inhibitors of HDAC6 into late stage preclinical development. Such compounds have demonstrated biological activity in the areas of autoimmunity, neurodegeneration, cardiac disorders, stroke, diabetes and depression in preclinical studies.

Autoimmune diseases are a major and growing public health problem. In the United States, systemic lupus erythematosus, inflammatory bowel disease and severe psoriasis affect millions of patients , and there are an estimated 400,000 cases of multiple sclerosis. Most currently available treatments are biologic drugs, which are typically 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.

Additional areas of exploration

Acetylon is also pursuing development of selective HDAC inhibitors for other disease indications including parasitic diseases such as malaria and major genetic diseases such as sickle cell disease and thalassemia major (beta-thalassemia). Acetylon has successfully demonstrated the potent inhibition of the life cycle of the most common severe form of malaria (plasmodium falciparum) in human blood cells without appreciable toxicity to white blood cells (unpublished data).