Acetylon Pharmaceuticals Celebrates Headquarters Expansion in Boston's Innovation District

March 20, 2014

FOR IMMEDIATE RELEASE

Boston – March 20, 2014 – Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase (HDAC) inhibitors for enhanced therapeutic outcomes, today announced the expansion of its headquarters facility located in Boston’s Innovation District. Martin J. Walsh, Mayor of Boston, and Robert K. Coughlin, President and Chief Executive Officer of MassBio, are scheduled to join Acetylon employees and other invited guests this morning in a ribbon cutting ceremony at the Company.

“Our newly expanded Boston headquarters facility will accommodate our continued organizational growth in support of additional clinical trials of our lead drug candidate, ricolinostat, in two important blood cancers, multiple myeloma and lymphoma,” commented Walter C. Ogier, President and Chief Executive Officer of Acetylon. “We anticipate initiating multiple Phase 2 clinical trials of ricolinostat during 2014, building on the favorable Phase 1 clinical trial results which were reported at the American Society of Hematology Annual Conference and announced by the Company this past December.”

“Acetylon is well-positioned to make a positive impact on the treatment of cancer in the coming years and further the standing of Boston as a global leader in biotech and the life sciences,” commented Mayor Marty Walsh. “We are pleased that Acetylon has chosen to further expand in the Boston Innovation District to support the rapid growth that this community has seen over the past few years.”

Robert Coughlin added, “Companies like Acetylon, which are developing innovative therapies for patients in need, are a major reason why Massachusetts draws such high-caliber scientific and medical talent and expertise, and we look forward to Acetylon’s continuing growth to help advance our Commonwealth’s extensive life sciences community.”

In July of 2013, Acetylon signed a strategic collaboration with Celgene Corporation that included an exclusive option for the future acquisition of Acetylon by Celgene and $100 million in upfront cash which, in part, is supporting the expansion of clinical trials of Acetylon’s HDAC inhibitor program in hematology in 2014. In 2011, the Leukemia & Lymphoma Society pledged $5 million to support Acetylon’s first Phase 1 clinical trial of ricolinostat, and the support is expected to continue as the drug enters Phase 2 clinical testing. Acetylon has also raised $35 million from private investors and a $15 million earlier investment from Celgene.

About Ricolinostat

Blood cancers such as multiple myeloma and lymphoma are characterized by successive genetic mutations resulting in uncontrolled cell proliferation and dysfunctional production of intracellular proteins. Ricolinostat (ACY-1215) selectively inhibits the intracellular enzyme HDAC6, which leads to an accumulation of excess protein and in addition may disrupt critical proliferative signals in malignant cells. Disruption of these molecular processes in cancer cells triggers programmed cell death, called "apoptosis," with little or no effect on normal cells. Currently available HDAC drugs affect the expression of numerous genes in normal cells as well as cancer cells, which can result in side effects such as gastrointestinal dysfunction, lowered blood platelet levels and risk of hemorrhage and profound fatigue as well as potential for significant cardiac toxicity. Selective inhibition of HDAC6 is expected to reduce or eliminate these often-severe side effects associated with non-selective HDAC inhibition and may enable the development of optimized treatment regimens, including maximally effective combination drug therapies.

About Acetylon

Acetylon Pharmaceuticals, Inc., based in Boston, Massachusetts, is a leader in the development of novel small molecule drugs targeting epigenetic mechanisms for the enhancement of therapeutic outcomes in cancer and other critical human diseases. The Company’s epigenetic drug discovery platform has yielded a proprietary portfolio of optimized, orally-administered Class I and Class II histone deacetylase (HDAC) selective compounds. Alteration of HDAC regulation through selective HDAC inhibition is thought to be applicable to a broad range of diseases including cancer, sickle cell disease and beta-thalassemia, and autoimmune and neurodegenerative diseases. Acetylon’s lead drug candidate, ricolinostat (ACY-1215), is a selective HDAC6 inhibitor currently in Phase 1b clinical development for the treatment of multiple myeloma. The Company recently announced a strategic collaboration agreement with Celgene Corporation, which includes an exclusive option for the future acquisition of Acetylon by Celgene. Acetylon’s scientific founders are affiliated with Harvard University, the Dana-Farber Cancer Institute, the Massachusetts General Hospital, and Harvard Medical School. www.acetylon.com

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