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The Acetylon Story

Acetylon Pharmaceuticals is developing next-generation selective small molecule histone deacetylase (HDAC) inhibitors, which allow for epigenetic regulation of gene and protein function for the treatment of patients with critical unmet medical needs. Acetylon has programs in development for the treatment of cancers including multiple myeloma as well as neurodegenerative, inflammatory, metabolic, genetic and infectious parasitic diseases. Selective HDAC inhibitors have the potential to reduce or eliminate the substantial side effects observed with current non-selective approaches while also realizing their extraordinary potential for enhanced therapeutic benefit through higher dosing, extended administration and reduction of combination drug toxicity.

Epigenetics is a way of regulating the biological activity of genes and proteins in cells through chemical modification of associated protein networks. The chemical modification is accomplished by families of catalytic enzymes. Acetylon’s focus is on a class of enzymes called histone deacetylases, or HDACs. Inhibition of different HDACs can alter the expression of genes to form new proteins, alter the proper folding of those new proteins, and alter the degradation of misfolded waste proteins. HDAC inhibitors are thus potentially powerful tools in directing the function and survival of cells, including both normal cells and tumor cells.

Acetylon's selective HDAC inhibitor drug discovery technology was initially developed and licensed to the Company by The Broad Institute of Harvard University and MIT, the Dana-Farber Cancer Institute and Harvard Medical School. Acetylon’s management team and scientific founders have had previous success bringing numerous FDA-regulated drugs and other therapeutic medical products into clinical development and to market in the US and Europe, across most of Acetylon’s targeted disease categories. Since the Company was founded in late 2008, Acetylon has secured $40 million in funding through a combination of private investments by individuals as well as from a major philanthropic organization, The Leukemia & Lymphoma Society, and US Federal government grants. Celgene Corporation also made a $15 million strategic equity investment in Acetylon in February 2012, which was followed in July 2013 by a further non-dilutive payment of $100M in conjunction with a comprehensive collaboration agreement, bringing the total operating funds raised to date by Acetylon to $155 million.

Bringing new therapies to market is the result of creative scientific thinking followed by preparation, trial and error, persistence and dedication. Building on the Founders’ vision, Acetylon has created an open, collaborative and transparent atmosphere that allows our scientists and other staff to make progress every day toward our shared goal of bringing new drugs to patients with cancer and other serious diseases.