Science

Acetylon Pharmaceuticals, Inc. is pioneering innovative approaches to the discovery and development of next-generation, selective deacetylase (HDAC) enzyme inhibitors, which have potential to be first-in-class, oral administered drugs with enhanced effectiveness and safety versus current alternatives.  Acetylon’s proprietary “Iterative Biasing Chemistry” drug development platform technology, sourced from Harvard University, provides rapid, efficient methodology for discerning small molecule drug structure-activity relationships for selective HDAC inhibition.   Acetylon’s small molecule drug candidates are designed to selectively inhibit specific enzymes of the HDAC family in order to achieve the powerful therapeutic potential of isoform-selective HDAC inhibition with reduced side effects – thereby changing the treatment paradigms for patients with cancers as well as severe inflammatory, neurodegenerative, infectious and genetic diseases.  Acetylon’s first clinical candidate, the selective HDAC6 inhibitor “ACY-1215,” is targeted for the treatment of hematologic and solid tumor cancers and is entering Phase 1-2 clinical trials in leading hospitals.

Opportunity

Current non-selective HDAC inhibitor drugs and drug candidates, while achieving substantial therapeutic benefit, have limited utility due to toxicities arising from insufficient target specificity.  Based on preclinical studies, Acetylon’s emerging orally dosed drug candidates have potential for a breakthrough in enhanced safety and therapeutic potential compared to the current standard-of-care HDAC inhibitors in hematologic and solid tumor cancers, and compared to standard-of-care anti-proliferative and immunosuppressive biologic drugs in autoimmune diseases.  Non-selective HDAC inhibition has been shown to alter the expression levels of more than 1,000 genes in human cells, which Acetylon believes is responsible for the dose-limiting side effects of existing HDAC inhibitor drugs and drug candidates in cancer patients and makes them unsuitable for use in the treatment of chronic diseases.  Acetylon is targeting realization of the potential for therapeutic HDAC inhibition through selective targeting of individual isoforms of the 18-member HDAC enzyme family, including in particular Class II HDACs which have little or no epigenetic impact on cellular gene expression.

Corporate Background

Acetylon is based in Boston, Massachusetts and, since being founded in late 2008, has been funded through a combination of private investment by individuals as well as funding from a major philanthropic organization, The Leukemia & Lymphoma Society, and by a US Federal government grant.  Acetylon’s selective HDAC inhibitor drug discovery technology was initially developed and licensed to Acetylon by The Broad Institute of Harvard University and MIT, the Dana-Farber Cancer Institute and Harvard Medical School.  Acetylon’s management team and scientific founders have had previous success bringing numerous FDA-regulated drugs and other therapeutic products into clinical development and to market in the US and Europe, across each of Acetylon’s targeted disease categories.

Acetylon operates under a cost-efficient, high quality, focused business model.  The Company employs a small, experienced management and scientific team together with expert advisors from academia and industry plus leading contract research, development and manufacturing organizations in the US, Europe and Asia to rapidly and productively advance promising lead compounds through lead optimization, drug candidate selection, preclinical development and into human clinical trials.  Since initiating drug development operations in early 2009, Acetylon has successfully advanced its lead oral HDAC6 inhibitor drug candidate, ACY-1215, from early stage lead identification to FDA allowance of a Phase 1-2 clinical trial in multiple myeloma.  Acetylon will also soon be selecting a second oral HDAC6 inhibitor drug candidate for clinical development in inflammatory autoimmune disease indications.