September 16, 2014 12:20 PM ET


Company Overview of Acetylon Pharmaceuticals, Inc.

Company Overview

Acetylon Pharmaceuticals, Inc. discovers and develops selective small molecule histone deacetylase inhibitors and other targeted drugs for the treatment of cancers, inflammatory, neurodegenerative, genetic, and infectious diseases. It has a strategic partnership with Celgene Corporation. The company was founded in 2008 and is based in Boston, Massachusetts.

70 Fargo Street

Suite 205

Boston, MA 02210

United States

Founded in 2008





Key Executives for Acetylon Pharmaceuticals, Inc.

Chief Executive Officer
Age: 57
Co-Founder and Chairman of the Board
Age: 51
Scientific Founder
Scientific Founder and Member of the Advisory Board
Scientific Founder
Age: 63
Compensation as of Fiscal Year 2014.

Acetylon Pharmaceuticals, Inc. Key Developments

Acetylon Pharmaceuticals Reports Ricolinostat (ACY-1215) is Active and Well-tolerated in Ongoing Phase 1b Studies in Patients with Refractory Multiple Myeloma at the 19(th) Congress of the EHA

Acetylon Pharmaceuticals, Inc. has continued to demonstrate promising activity and tolerability in combination with Revlimid(R) (lenalidomide) or Velcade(R) (bortezomib) for the treatment of relapsed or refractory multiple myeloma. Data from the studies were presented at the 19(th) Congress of the European Hematology Association (EHA) in Milan, Italy. All 22 evaluable patients receiving ricolinostat in combination with Revlimid demonstrated stable disease or better, with 64% having partial response or better, including 1 complete response, 5 very good partial responses, and 8 partial responses (including 1 PRu) as evaluated by the International Myeloma Working Group (IMWG) standards. 3 patients also achieved minor responses. 10 of the evaluable patients were previously refractory to either full dose or maintenance Revlimid. Ricolinostat in combination with Velcade demonstrated an overall response rate (ORR), defined as partial response or better, of 53% in 19 evaluable patients, or 36% of the intention-to-treat population. 10 patients were previously refractory to Velcade. Ricolinostat has been well-tolerated in both studies to-date. In combination with Revlimid, treatment emergent adverse events (AEs) have been mostly low grade and considered not related to ricolinostat and included diarrhea, fatigue, headache, muscle spasms, neutropenia, rash and upper respiratory infection. Two grade 3 neutropenia events and one grade 3 syncope event were considered possibly related to ricolinostat. In combination with Velcade, treatment emergent AEs have been mostly grade 1-2 and not attributed to study drug. AEs greater than or equal to grade 3 have included hematologic abnormalities (11), elevated amylase (2), other asymptomatic laboratory abnormalities (3), and one patient with stomach cramps, diarrhea, and fatigue. One patient with cardiac history had a fatal pulmonary embolism after 3 cycles of treatment. No maximum tolerated dose has been established in either trial. Pharmacodynamic assessments in peripheral blood cells showed preferential increases in acetylated tubulin, a marker of HDAC6 inhibition, compared to acetylated histone, a Class 1 HDAC marker, in all patients receiving doses of 80 mg or greater.

Acetylon Pharmaceuticals, Inc. Announces Headquarters Expansion in Boston's Innovation District

Acetylon Pharmaceuticals, Inc. announced the expansion of its headquarters facility located in Boston's Innovation District.

Acetylon Pharmaceuticals, Inc. Presents Data on Potent Activity of Ricolinostat (ACY-1215) in Preclinical Models of Lymphoma

Acetylon Pharmaceuticals, Inc. announced that positive preclinical data of selective HDAC6 inhibitor, ricolinostat (ACY-1215), in models of lymphoma were presented on December 09, 2013 in an oral presentation by Dr. Jennifer Amengual, Assistant Professor of Medicine & Experimental Therapeutics, Columbia University Medical Center, and in a poster session on December 8 at the 55(th) Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA. The data highlight the potent activity of ricolinostat in preclinical models of lymphoma, both alone and in combination with proteasome inhibitor, bortezomib (Velcade(R)), PI3K inhibitors, GS-101 and GDC-0941, and Bruton's tyrosine kinase inhibitor, ibrutinib (Imbruvica(TM)), recently granted accelerated approval for the treatment of patients with mantle cell lymphoma following prior therapy. Highlights of the Presentations at ASH: Dual Targeting With the Selective Histone Deacetylase (HDAC) 6 Inhibitor, ACY-1215, and Bortezomib (BOR) Leads to Marked Disruption of Protein Degradation Pathways and Apoptosis in Preclinical Models of Lymphoma (oral presentation, Abstract # 648): In six lymphoma cell lines, ricolinostat in combination with bortezomib (Velcade(R)) led to a synergistic increase in apoptosis; In an in vivo model of diffuse large B-cell lymphoma (DLBCL), a single treatment of the combination of ricolinostat and bortezomib (Velcade(R)) demonstrated statistically significant delay in tumor growth and extended overall survival. Inhibition of HDAC6 in Combination with Targeted Agents Results in Broad Synergistic Decreases in Viability in Non-Hodgkin's Lymphoma (NHL) Cells (poster presentation, Abstract # 3071): Ricolinostat in combination with Bruton's tyrosine kinase inhibitor, ibrutinib (Imbruvica(TM)), or PI3K inhibitors, GS-1101 and GDC-0941, resulted in synergistic decreases in non-Hodgkin's lymphoma (NHL) cell viability. Ricolinostat also demonstrated significant decrease in NHL cell viability as a single agent.

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