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Acetylon Pharmaceuticals Initiates Phase 2 Study of Ricolinostat (ACY-1215) in Combination with Pomalyst


Acetylon Pharmaceuticals Initiates Phase 2 Study of Ricolinostat (ACY-1215) in Combination with Pomalyst®

Boston – December 4, 2014 – Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase (HDAC) inhibitors for enhanced therapeutic outcomes, today announced the initiation of a Phase 2 study of ricolinostat (ACY-1215), a selective HDAC6 inhibitor, in combination with Pomalyst® (pomalidomide, Celgene) for the treatment of relapsed-and-refractory multiple myeloma. The Phase 2 segment of the trial follows successful completion of a Phase 1b segment earlier this year, involving dose escalation of ricolinostat. The study, ACE-MM-102, is the second trial of ricolinostat in combination with one of Celgene’s IMiD® (immunomodulatory drug) compounds. Ricolinostat is also being evaluated in an ongoing Phase 1b/2 study in combination with Revlimid® (lenalidomide, Celgene).

“Our collaboration with Celgene has permitted our clinical investigators and their patients to access ricolinostat in combination with best-in-class IMiD drugs for multiple myeloma. Based on early signs of clinical activity with ricolinostat in combination with Revlimid, and supported by our preclinical results presented at ASH in December 2013 (Quayle and Jones), we believe that ricolinostat has the potential to demonstrate synergistic effects with Pomalyst as well,” said Walter C. Ogier, President and Chief Executive Officer and co-founder of Acetylon. “We are additionally encouraged by the excellent tolerability which ricolinostat has demonstrated thus far in combination with the IMiD class of drugs. In other clinical trials of non-selective HDAC inhibitors, their combination with other anti-cancer drugs has been associated with dose-limiting levels of gastrointestinal, hematologic and cardiac toxicity and fatigue.” 

The Phase 2 study of ricolinostat in combination with Pomalyst and low-dose dexamethasone is a multicenter, open-label trial to evaluate the safety and efficacy of the drug combination in patients with relapsed-and-refractory multiple myeloma. The primary endpoint of the Phase 2 study is overall response rate. Ricolinostat is being administered orally in the Phase 2 portion of the trial per an optimized schedule determined during the Phase 1b portion, involving daily dosing for the first 21 days of each 28 day treatment cycle. The Phase 2 trial is being conducted at 20 leading cancer treatment centers for multiple myeloma in the United States and Canada, with expansion to sites in Europe planned for early 2015. Additional information about the clinical trial including participating medical centers is available at https://clinicaltrials.gov/ct2/show?term=ricolinostat&rank=4.

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 2 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

Revlimid®, IMiD® and Pomalyst® are registered trademarks of Celgene Corporation.


Walter C. Ogier
President and Chief Executive Officer
(617) 435-2664

MacDougall Biomedical Communications

Kari Watson
(781) 235-3060

Michelle Avery
(781) 235-3060

Doug MacDougall
(781) 235-3060