Acetylon Announces Upcoming Scientific Presentations On The Use Of Selective HDAC Inhibitors In Treating Genetic Blood Disorders And Cancers

-- Results of Multiple Clinical and Preclinical Studies to be Presented at the 58th Annual Meeting of the American Society of Hematology --

BOSTON – December 2, 2016 – Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase (HDAC) inhibitors for enhanced therapeutic outcomes, today announced that multiple posters and oral presentations on its preclinical and clinical programs will be presented at the American Society of Hematology Annual Meeting (ASH), held from December 3-6 in San Diego, California.

“The lineup of data presentations for Acetylon assets at ASH includes exciting advancements in our selective HDAC1,2 inhibitor preclinical program in sickle cell disease and beta-thalassemia and selective HDAC6 inhibitor preclinical programs in multiple myeloma, lymphoma, and leukemia. We will also present an update on the Phase 1a/1b ACE-MM-200 study of the selective HDAC6 inhibitor, citarinostat (ACY-241), in combination with Pomalyst (pomalidomide) and dexamethasone in patients with relapsed or relapsed-and-refractory multiple myeloma,” said Walter C. Ogier, President and Chief Executive Officer of Acetylon. “We are encouraged by the results of our preclinical and clinical programs, which provide extensive support for the continued development of selective HDAC inhibitors for the treatment of hematologic cancers and genetic blood disorders, and we look forward to productive discussions around all of these presentations at ASH.”

Details of the presentations are as follows:
HDAC1/2 Inhibitor ACY-957
Title: The Histone Deacetylase 1 and 2 (HDAC1/2) Inhibitor ACY-957: Impact of Dosing Schedule on Pharmacokinetics (PK), Pharmacodynamics (PD), Hematopoietic Toxicity, and Gamma Globin (HBG, ?) Expression in Monkey
Date: Sunday, December 4, 2016
Time: 10:30 AM PST
Location: Room 7AB (San Diego Convention Center)
Session: 112. Thalassemia and Globin Gene Regulation: Fetal Hemoglobin Regulation
Abstract Number: 323
In an oral presentation, Jeffrey R. Shearstone, Ph.D., Associate Director of Biology at Acetylon, will present data comparing the effects of multiple dosing schedules for the selective HDAC1/2 inhibitor, ACY-957, on gamma globin (HBG) protein expression in primates. Results suggest that dose level and schedule may be further refined to optimize the therapeutic window of HDAC1/2-selective inhibitors for substantial induction of fetal hemoglobin to treat hemoglobinopathies, which include sickle cell disease and beta-thalassemia, two blood disorders with the highest prevalence worldwide of any genetic disease.

HDAC6 Inhibitor ACY-241
Title: Selective HDAC6 Inhibitor ACY-241, an Oral Tablet, Combined with Pomalidomide and Dexamethasone: Safety and Efficacy of Escalation and Expansion Cohorts in Patients with Relapsed or Relapsed-and-Refractory Multiple Myeloma (ACE-MM-200 Study)
Date: Sunday, December 4, 2016
Time: 6:00pm – 8:00pm PST
Location: Hall GH (San Diego Convention Center)
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster II
Abstract Number: 3307
Acetylon will present early results of the Phase 1a/1b ACE-MM-200 clinical trial evaluating the safety and preliminary anti-cancer activity of the selective HDAC6 inhibitor, citarinostat (ACY-241), in combination with pomalidomide (Pom) (Pomalyst®, Celgene) and dexamethasone (Dex) for the treatment of relapsed or relapsed-and-refractory multiple myeloma (RRMM).

Title: Anti-Tumor Activities of XBP1 Antigen-Specific Cytotoxic T Lymphocytes Are Enhanced By HDAC6 Inhibitor ACY-241
Date: Saturday, December 3, 2016
Time: 5:30 PM-7:30 PM PST
Location: Hall GH (San Diego Convention Center)
Session: 653. Myeloma: Therapy, excluding Transplantation: Poster I
Abstract Number: 2143
Investigators from Dana-Farber Cancer Institute will give a poster presentation on preclinical data demonstrating potent immunomodulatory effects of the selective HDAC6 inhibitor, citarinostat (ACY-241), which supports a role for selective HDAC6 inhibition in the improvement of tumor-specific cytotoxic T-cell function and tumor cell recognition in combination with an antigen-specific cancer vaccine.

Title: Combination of a Novel HDAC 6 Inhibitor ACY-241 with Anti-PD-L1 Antibody Enhances Anti-Tumor Immunity and Cytotoxicity in Multiple Myeloma
Date: Sunday, December 4, 2016
Time: 12:45 PM PST
Location: Grand Hall B (Manchester Grand Hyatt San Diego)
Session: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Novel Immune Approaches for Myeloma Therapy
Abstract Number: 382
In an oral presentation, an investigator from Dana-Farber Cancer Institute will present preclinical data on the use of the selective HDAC6 inhibitor, citarinostat (ACY-241), in combination with an anti-PD-L1 antibody to enhance cytolytic activity of immune cells derived from multiple myeloma (MM) patients against tumor cells in vitro. The results being presented provide the basis for combining citarinostat and an anti-PD-L1 antibody to restore immune function, enhance MM cytotoxicity, and potentially improve patient outcomes.

HDAC6 Inhibitors Ricolinostat and ACY-738
Title: Ricolinostat (ACY-1215), a Selective HDAC6 Inhibitor, Alone and in Combination with Bendamustine Is Effective in Preclinical Studies in Lymphoma Cell Lines
Date: Sunday, December 4, 2016
Time: 6:00 PM-8:00 PM PST
Location: Hall GH (San Diego Convention Center)
Session: 605. Molecular Pharmacology, Drug Resistance—Lymphoid and Other Diseases: Poster II
Abstract Number: 2772
An investigator from the University of Modena and Reggio Emilia will present preclinical data demonstrating that treatment of lymphoma cell lines with the combination of ricolinostat (ACY-1215) and bendamustine synergistically induces anti-proliferative and pro-apoptotic effects, suggesting that bendamustine in combination with an epigenetic therapy, such as ricolinostat, may be a promising treatment regime for managing lymphomas.

Title: Combinatorial Effect of HDAC6i and Ibrutinib Therapy in CLL Murine Model
Date: Saturday, December 3, 2016
Time: 5:30 PM-7:30 PM PST
Location: Hall GH (San Diego Convention Center)
Session: 642. CLL: Therapy, excluding Transplantation: Poster I
Abstract Number: 2035
An investigator from the H. Lee Moffitt Cancer Center & Research Institute will present data on the use of a selective HDAC6 inhibitor, ACY-738, in a preclinical model of chronic lymphocytic leukemia, which demonstrates reduced tumor burden after treatment with ACY-738 as a single agent and a further decrease in tumor burden with the combination of ACY-738 and the BTK inhibitor Ibrutinib.

About Acetylon

Acetylon Pharmaceuticals, Inc., based in Boston, Massachusetts, is the leader in the development of novel, selective 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 Class I and Class II histone deacetylase (HDAC) selective compounds for oral administration. Alteration of HDAC regulation through selective HDAC inhibition is thought to be applicable to a broad range of diseases including cancer, autoimmune and neurodegenerative diseases, neuropathy, and hemoglobinopathies including sickle cell disease and beta-thalassemia. www.acetylon.com

CONTACTS:
Acetylon:
Walter C. Ogier
President and Chief Executive Officer
(617) 245-1300

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