COTI-2 is a clinical stage, orally available small molecule targeting p53, a tumor suppressor gene that is mutated in over 50% of all cancers.
COTI‐2 is being evaluated for the treatment of gynecologic cancers and head and neck squamous cell carcinoma (HNSCC) in two dose-escalation Phase 1 clinical trials at MD Anderson Cancer Center and Northwestern University. In August 2017, we announced top-line safety and tolerability data from the gynecological malignancies trial, and further released pharmacokinetic data in November 2017 and pharmacodynamic data in December 2017. The second dose-escalation trial was initiated in HNSCC in August 2017 with patient dosing beginning in October 2017. We have further plans to expand the scope of these initial trials into other p53-mediated cancers, as a single agent and potentially in combination with approved cancer therapies. COTI-2 holds orphan drug status from the FDA for the treatment of ovarian cancer.
Extensive preclinical studies demonstrated COTI-2’s ability to restore mutant p53 function and induce cancer cell death in a tumor-agnostic manner, with specific and non-toxic properties. COTI-2’s effect on the p53 pathway, low toxicity, combination effectiveness with standard agents, and potential for longer term outpatient therapy as an oral agent, support a potentially dramatic change in the treatment of susceptible cancers.