Completed animal studies pave way for clinical trial of brain cancer therapeutic

CHARLOTTESVILLE, Va.—Sept. 19 saw Diffusion Pharmaceuticals Inc., a clinical-stage biotechnology company focused on the development of novel small-molecule therapeutics for cancer and other hypoxia-related diseases, report the successful completion of dosing of two animal toxicology studies required by the U.S. Food and Drug Administration (FDA) to support Diffusion’s planned Phase 3 clinical trial testing its lead molecule trans sodium crocetinate (TSC) in newly diagnosed glioblastoma (GBM) patients.

As outlined at the end of Phase 2 FDA meeting, the number of TSC dosing exposures per patient will double in the planned Phase 3 trial as compared to the Phase 2 trial. As part of the agreement reached on a trial design for the Phase 3 study, the FDA requested that Diffusion augment its existing TSC toxicology studies with additional three-month TSC toxicology data in both rats and dogs. The animal dosing for these three-month studies has now been successfully completed.

David G. Kalergis, Diffusion’s Chairman and CEO, said, “The successful completion of dosing for these three month animal toxicology studies is an important milestone in support of Diffusion’s ability to conduct a Phase 3 pivotal trial of TSC in newly diagnosed GBM patients.”

Diffusion’s technology is, it says “a paradigm shift in the approach to the treatment of cancer and other diseases involving hypoxia, or oxygen deprivation, in that it facilitates the diffusion of oxygen to the hypoxic tissues.” Diffusion is developing its lead drug, TSC, for use in the many cancer types in which tumor hypoxia is known to diminish the effectiveness of current treatments. TSC targets the cancer’s hypoxic microenvironment, reoxygenating treatment-resistant tissue and making the cancer cells more vulnerable to the therapeutic effects of treatments such as radiation therapy and chemotherapy, without the apparent addition of any serious side effects, according to the company.

A Phase 2 clinical program, completed in the second quarter of 2015, evaluated 59 patients with newly diagnosed GBM. This open-label, historically controlled study demonstrated a favorable safety and efficacy profile for TSC combined with standard of care. The FDA has agreed upon the design of a Phase 3 trial in newly diagnosed GBM. Additional planned studies, subject to receipt of necessary financing, include the Phase 2 trial in pancreatic cancer and a study in brain metastases.