Many complex diseases, such as cancer, involve cellular growth abnormalities regulated by the endocrine system. Based upon proprietary insight into the complexities of the endocrine system, Tokai is building a pipeline of therapeutics focusing initially on castration resistant prostate cancer (CRPC).

Tokai's lead drug candidate, TOK-001, is a small molecule, oral drug that disrupts the growth and survival of cancer cells by attacking three specific targets in the prostate cancer tumor, providing a novel and proprietary triple mechanism of action for the treatment of prostate cancer. In preclinical studies, TOK-001 acts as an androgen receptor antagonist, as a CYP17 lyase inhibitor and decreases androgen receptor levels in prostate tumors – the only drug in development that has been shown to exhibit this property. In TOK-001, these three distinct mechanisms of action are combined in one therapy.

In addition, Tokai has an early stage pipeline of other assets focused on semparatide, a synthetic version of parathyroid hormone-related peptide, for improved engraftment in hematopoietic stem cell (HSC) transplantation; and specific progesterone receptor modulators, or SPRMs, for the treatment of uterine fibroids.