Acetylon Pharmaceuticals

About Acetylon Pharmaceuticals

Acetylon Pharmaceuticals, a biopharmaceutical company established in 2008, focused on the development of selective histone deacetylase (HDAC) inhibitors. The company was founded by a distinguished group of scientists from the Dana-Farber Cancer Institute and Harvard University, including Dr. James Bradner, Dr. Kenneth Anderson, Dr. Ralph Mazitschek, and Dr. Marc Cohen. This founding team brought together deep expertise in chemistry and cancer biology, particularly in the area of chromatin-modifying enzymes, which formed the scientific bedrock of the company.

Operating in the oncology and rare diseases therapeutic markets, Acetylon's primary business was the discovery and clinical development of novel drug candidates. Its business model centered on advancing these candidates through the clinical trial process to demonstrate safety and efficacy, with the ultimate goal of commercialization or strategic partnership. The company's core technology, licensed from Harvard and the Dana-Farber Cancer Institute, involved creating highly selective HDAC inhibitors, specifically targeting HDAC6. This selectivity was a key differentiator, as it promised to minimize the side effects associated with earlier, less-selective HDAC inhibitors by targeting specific disease pathways.

The company's lead product candidate was ricolinostat (ACY-1215), an oral, selective HDAC6 inhibitor. Ricolinostat was investigated in multiple clinical trials for the treatment of various cancers, most notably multiple myeloma, where it was often tested in combination with other therapies. The proposed benefit of ricolinostat was its ability to enhance the effectiveness of other cancer treatments while potentially offering a more favorable safety profile compared to non-selective inhibitors. Beyond oncology, Acetylon explored the potential of its HDAC inhibitors for other conditions, including sickle cell disease, beta-thalassemia, and inflammatory diseases.

Acetylon's journey reached a significant milestone in 2016 when it was acquired by Celgene Corporation in a deal valued at a minimum of $100 million, with the potential for up to $1.6 billion based on regulatory and sales milestones. Celgene had been a strategic partner and investor since 2013, and the acquisition represented the culmination of Acetylon's successful development program, particularly the progress of ricolinostat.