Company Overview of Acetylon Pharmaceuticals, Inc.
Acetylon Pharmaceuticals, Inc. engages in the development of 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 selective compounds for oral administration. It offers ACY-1215 and ACY-241 lead drug candidates that are selective HDAC6 inhibitors currently in Phase 2 and Phase 1b clinical development for the treatment of multiple myeloma. Acetylon Pharmaceuticals, Inc. was founded in 2008 and is based in Boston, Massachusetts. As of December 16, 2016, Acetylon Pharmac...
70 Fargo Street
Boston, MA 02210
Founded in 2008
Key Executives for Acetylon Pharmaceuticals, Inc.
Chief Executive Officer, President and Director
Co-Founder and Chairman of the Board
Scientific Founder, Director and Member of the Scientific Advisory Board
Scientific Founder and Member of the Scientific Advisory Board
Scientific Founder, Director and Member of the Scientific Advisory Board
Compensation as of Fiscal Year 2016.
Acetylon Pharmaceuticals, Inc. Key Developments
Acetylon Pharmaceuticals, Inc. Presents Data on the Use of HDAC6 Inhibitors Ricolinostat (ACY-1215) and ACY-241 in Combination with Pomalidomide and Dexamethasone for the Treatment of Multiple Myeloma
Jun 13 16
Acetylon Pharmaceuticals, Inc. announced that it presented data from multiple clinical trials evaluating the safety and efficacy of two selective HDAC6 inhibitors in combination with pomalidomide (Pom) (Pomalyst®, Celgene) and dexamethasone (Dex) for the treatment of relapsed or relapsed-and-refractory multiple myeloma (RRMM) at the 21st Congress of the European Hematology Association in Copenhagen, Denmark. Positive results from a Phase 2 clinical trial of ricolinostat were presented in an oral presentation by Noopur Raje, M.D. of Massachusetts General Hospital in Boston, Massachusetts. In addition, results of a Phase 1b clinical trial of an alternative liquid formulation of ricolinostat were presented in a poster presentation by Sumit Madan, M.D. of University of Texas Southwestern Medical Center. The ACE-MM-102 clinical trial is a multicenter, single arm, open-label Phase 2 study designed to evaluate the safety and efficacy of ricolinostat administered at an optimal dose and schedule in combination with Pom and Dex in patients with RRMM. Results of this study indicated that treatment with ricolinostat in combination with Pom and Dex was very well tolerated, and toxicities were predominantly low grade. Pharmacokinetic and pharmacodynamic analysis demonstrated selective inhibition of HDAC6 at therapeutic doses. Analysis of 67 efficacy-evaluable patients enrolled at least 6 months prior to the data cut confirmed an overall response rate (ORR) of 46%, a clinical benefit rate (CBR) of 58%, a disease control rate (DCR) of 82%, 9 months duration of response (DOR), and 7 months progression free survival (PFS). These data compare favorably to mature historical data for the MM-002 and MM-003 trials of Pom and Dex alone which demonstrated 31%-33% ORR, 7-8 months DOR, and 4 months median PFS. More mature data on the entire 96 patient study population will be available by the end of the year. The Phase 1b ACE-MM-104 clinical trial is a dose-escalation study of an alternative liquid formulation (ALF) of ricolinostat in combination with Pom and Dex in patients with RRMM. Daily dosing (QD) was better tolerated than twice-daily dosing. One dose-limiting toxicity (DLT), neutropenia, was observed at 180 mg QD, and cohort expansion to 6 patients showed no further DLTs. Pharmacokinetic and pharmacodynamic analysis demonstrated selective inhibition of HDAC6 at therapeutic doses, with no evidence of ricolinostat accumulation or drug-drug interaction with Pom. Early efficacy data indicates a confirmed ORR of 53% and a DCR of 87% after 6 months median follow-up. These results demonstrate that the alternative liquid formulation of ricolinostat is well tolerated in combination with Pom and Dex at doses up to 180 mg QD without major toxicities. This formulation of ricolinostat is also being used in ongoing investigator-sponsored studies in combination with paclitaxel and abraxane to treat solid tumors. The Phase 1a/1b ACE-MM-200 clinical trial utilizes a sequential monotherapy/combination trial design to establish the safety, pharmacokinetics, and pharmacodynamics of ACY-241 as both a monotherapy and in combination with Pom and Dex in patients with relapsed or relapsed-and-refractory MM. ACY-241 is an orally available selective HDAC6 inhibitor that is structurally similar to ricolinostat and administered in tablet form. Results of the study demonstrated that ACY-241 is well tolerated as a monotherapy and in combination with Pom and Dex, and toxicities did not differ substantially in frequency or severity from those reported with Pom and Dex alone. Pharmacokinetic and pharmacodynamic analysis indicated dose-linear increases in exposure and dose-dependent, selective increases in acetylated tubulin and histones. ACY-241 achieved exposures several fold higher than ricolinostat without severe toxicity, both alone and in combination with Pom and Dex. Early efficacy data for combination treatment indicates a confirmed ORR of 50% and a DCR of 95% after 3.5 months median follow-up. These results indicate that ACY-241 is well tolerated in combination with Pom and Dex, and early response data for combination treatment even with short follow-up are similar to those of ricolinostat in combination with Pom and Dex. Cohort expansion at biologically relevant combination doses is ongoing.
Acetylon Pharmaceuticals, Inc. Presents at Boston CEO Conference, May-31-2016 11:30 AM
May 26 16
Acetylon Pharmaceuticals, Inc. Presents at Boston CEO Conference, May-31-2016 11:30 AM. Venue: Four Seasons Boston, 200 Boylston Street, Boston, MA 02116, United States. Speakers: Walter C. Ogier, Chief Executive Officer, President and Director.
Acetylon Pharmaceuticals, Inc. Announces the Presentation of Preclinical Data at AACR Supporting the Use of Selective HDAC6 Inhibition to Modulate Chronic Lymphocytic Leukemia Immunobiology
Apr 20 16
Acetylon Pharmaceuticals, Inc. announced the presentation of data demonstrating that selective HDAC6 inhibition results in dose-dependent increases in cell killing as a single treatment and in combination with Bruton’s tyrosine kinase (BTK) inhibitor, ibrutinib (Imbruvica®), in patient-derived cell lines and preclinical models of chronic lymphocytic leukemia (CLL). The studies were completed in collaboration with the laboratory of Javier Pinilla-Ibarz, M.D., Ph.D. at the Moffitt Cancer Center and the data were presented by Moffitt investigator, Eva Sahakian, Ph.D., in a poster presentation at the American Association for Cancer Research (AACR) Annual Meeting 2016 in New Orleans. In CLL, malignant B cells evade immune detection and lead to immune suppression. Recently, histone deacetylases (HDACs) have been shown to play an active role in the regulation of pathogenesis and immune-related pathways in CLL, although their role in B-cell receptor signaling remains unknown. Previously, aberrant overexpression of HDAC6 has been demonstrated in CLL cell lines and patient samples, and the authors sought to understand the mechanistic role of HDAC6 in CLL. In collaboration with Acetylon scientists, the authors demonstrated that selective HDAC6 inhibition in CLL cell lines resulted in dose-dependent reductions in IL-10, a cytokine that regulates cell proliferation in CLL, as well as dose-dependent increases in cell death and a synergistic reduction in cell viability in combination with the BTK inhibitor, ibrutinib. Genetic knockdown of HDAC6 in CLL cells reduced expression of PD-L1 and other immune checkpoint markers, while increasing markers related to antigen presentation, including MHC II. Using an animal model of CLL, the authors then demonstrated with systemic administration of a selective HDAC6 inhibitor, a reduction in disease burden and increased survival in parallel with diminished expression of immune checkpoint markers on T-cells and B-cells, as well as a reduction in the number of immunosuppressive T-cells (Tregs).
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