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Acetylon Announces Issuance of a United States Patent with Composition of Matter Claims for Its Lead HDAC6 Inhibitor Drug Candidate Rocilinostat (ACY-1215)

Acetylon to Present at the 19th Annual Future Leaders in the Biotech Industry Conference

BOSTON, Mass.-April 20, 2012 -- Acetylon Pharmaceuticals Inc. today announced that it has been granted U.S. Patent 8,148,526 by the United States Patent and Trademark Office (USPTO). This patent contains fundamental composition of matter claims covering the Company’s selective HDAC6 inhibitor, ACY-1215TM, which is currently being investigated in human clinical trials for the treatment of multiple myeloma. Acetylon also announced that Chief Executive Officer, Walter Ogier, will present an overview of the Company at 1:30 PM today at the 19th Annual Future Leaders in the Biotech Industry Conference, hosted by BioCentury and Thomson Reuters at the Millennium Broadway Hotel & Conference Center in New York City.

Acetylon’s patent application was granted "prioritized examination" status under the provisions of the new “America Invents Act,” signed into law by President Obama in September 2011. The American Invents Act provides for prioritized “fast track” examination of patent applications that satisfy specific limitations regarding the number and type of examined claims.

“Given the widespread interest in the development of selective histone deacetylase (HDAC) inhibitors, it is important for Acetylon to demonstrate that we have unique, patentable drug candidates within a competitive composition-of-matter field of intellectual property.” commented Walter Ogier, Chief Executive Officer of Acetylon. “The rapid issuance of this important US patent and our presentation this afternoon at Future Leaders reinforces Acetylon’s position as a leader in the development of selective HDAC inhibitors.”

“Acquiring U.S. patent protection for ACY-1215 is a major step for Acetylon as the company continues to explore the use of this drug in the treatment of multiple myeloma,” stated John H. van Duzer, Ph.D., Vice President of Chemistry and Manufacturing for Acetylon. “We are also continuing to move forward with additional patent applications which claim further compounds similar to ACY-1215 as well as other unique series of molecules.”

About HDAC6 Inhibition

The Class IIB histone deacetylase enzyme, HDAC6, has emerged as an important target in inflammatory disease, neurologic disease and broadly in cancer. Acetylon’s next-generation HDAC6 inhibitor program is focused on enhancing drug potency and reducing or eliminating side effects common to HDAC inhibition through highly selective targeting of the HDAC6 enzyme. Inhibition of HDAC6 versus other isoforms uniquely preserves normal gene expression in cells, thereby minimizing patient toxicity. At the same time, HDAC6 inhibition severely disrupts diseased cells’ ability to produce normal proteins through disruption of the HSP-90 protein chaperone system, and to dispose of damaged misfolded proteins through modification of microtubules and disruption of the aggresome protein disposal pathway. Metabolically active cancer and autoimmune cells produce large amounts of misfolded proteins and inhibition of HDAC6 further increases the generation and accumulation of protein “trash”, triggering self-destruction of diseased cells via programmed cell death and leading to regression of disease.

About Acetylon Pharmaceuticals, Inc.

Acetylon Pharmaceuticals, Inc. is applying its unique capabilities to discover and develop next-generation, highly selective small molecule drugs to realize the therapeutic potential of HDAC inhibition to treat cancer, autoimmune and other diseases, while reducing the side effects common to this class of drugs. The Company is located in Boston and is based on technology initially developed at the Dana-Farber Cancer Institute and at Harvard University. www.acetylon.com


CONTACT:
Acetylon

Walter C. Ogier
President and Chief Executive Officer
(617) 435-2664
wogier@acetylon.com

MEDIA:
MacDougall Biomedical Communications

Kari Watson
(781) 235-3060
kwatson@macbiocom.com

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