CBL0102 is a clinical stage oncology drug. It is an orally administered small molecule being developed for orphan indications in developing markets by our Incuron, LLC subsidiary.
CBL0102 is a Quinacrine, a compound with a long history of use in humans as a treatment for malaria, osteoarthritis and autoimmune disorders. Quinacrine was not, however, previously used as an oncology agent. The interaction of CBL0102 with the FACT (Facilitates Chromatin Transcription) complex results in simultaneous NF-kB suppression, Heat Shock Factor 1 suppression and p53 activation. This modulation of three key cellular pathways causes suppression of cancer cell growth.
In 2008, we concluded a Phase II study in the United States in 31 patients with late stage, hormone refractory (androgen-independent) prostate cancer that had not responded to or relapsed following previous hormonal therapy and/or chemotherapy. The study results showed that one patient had a partial response, while 50% of the patients exhibited a decrease or stabilization in the rate of prostate cancer progression. CBL0102 was well-tolerated and there were no serious adverse events attributed to the drug.
Due to particularly high accumulation of CBL0102 in the liver, a multi-center Phase I trial of CBL0102 was opened in the Russian Federation in 2010 in patients with liver metastases of solid tumors of epithelial origin, or primary advanced hepatic carcinoma for which standard therapy has failed or does not exist. This trial is ongoing.