We are developing selective inhibitors for HDAC6, a key cancer drug target overexpressed in several solid tumors.

Target: HDAC6

Histone deacetylase 6 (HDAC6) has emerged as an important drug target for cancer therapy. Despite its name, it is principally a non-histone protein deacetylase, regulating the acetylation status of several substrates implicated in cancer, notably α-tubulin, and it is structurally-, expressionally-, and functionally-distinctive within the HDAC superfamily.

Its attractiveness as a cancer drug target stems from its overexpression in several solid tumors, its regulation of cell motility, and its role in apoptosis, stress response and aggresomal protein degradation. Moreover, HDAC6 has recently been implicated in cell signal transduction, in oncogenic processes such as dysregulation of ciliation, and a role for the target in tumor immunotherapy has emerged.

Inhibitor: KA2507

Drawing on considerable experience in metalloenzyme inhibitor therapeutics, we have designed and developed a novel class of HDAC6 inhibitors.

KA2507 is a highly-potent, selective, orally-administered small molecule inhibitor of HDAC6. Based on its potency and specificity and the role of HDAC6 in cancer, KA2507 has significant potential in the treatment of solid and hematological cancers, as a single agent or in combination therapy.

Phase I clinical trial

The safety and efficacy profiles of KA2507 are currently under investigation in a Phase I clinical trial in patients with solid tumors at The University of Texas MD Anderson Cancer Center.

For more information please visit ClinicalTrials.gov: NCT03008018.

Next-generation HDAC6 inhibitors

Our R&D program is focused on a new generation of brain-penetrant, non-genotoxic molecules targeting glioblastoma and other indications.