CODY Study

About the CODY Study

CODY is a multi-center Phase 2b clinical study evaluating the safety and effectiveness of a topical gel (experimental topical rapamycin, or PTX-367) for Gorlin syndrome. The CODY study will determine if PTX-367 is safe and effective in preventing the number of new basal cell carcinomas (BCCs) on the face. Your participation in the study is expected to last for six months.

About Gorlin Syndrome

Gorlin syndrome, also known as nevoid basal cell carcinoma syndrome, is a rare genetic disease caused by a mutation in one of several genes (PTCH1, PTCH2, SUFU). These genes are responsible for suppressing the formation of tumors.

People with Gorlin syndrome can develop hundreds of basal cell carcinomas (BCCs) in their lifetimes. Many afflicted individuals undergo repeated surgical removal of these cancerous lesions.

There are no FDA-approved medical therapies for Gorlin syndrome.

About PTX-367 (QTORIN rapamycin)

PTX-367 is a topical gel formulation of a medicine called rapamycin being developed for the potential treatment of Gorlin syndrome. Rapamycin blocks the activation of a naturally occurring protein molecule (mTOR) that has been shown to help tumors form in several studies¹. A recent study also showed that mTOR could be a factor in the formation of BCCs in those with Gorlin syndrome².

^{1. Hua, H., Kong, Q., Zhang, H. et al. Targeting mTOR for cancer therapy. J Hematol Oncol 12, 71 (2019) doi:10.1186/s13045-019-0754-1}

^{2. Back, J. et al. SOX9 Transcriptionally Regulates mTOR-Induced Proliferation of Basal Cell Carcinomas. J Invest Dermatol. 2018 August; 138(8): 1716–1725.}