Diffusion Pharmaceuticals LLC announced that Trans Sodium Crocetinate (TSC), the company’s lead drug candidate, has been granted an Orphan Drug Designation by the US Food and Drug Administration (FDA) for the treatment of primary brain cancer, also known as glioblastoma multiforme or GBM. GBM is one of the most deadly forms of cancer, with over half of its victims dying within 18 months of diagnosis.

“This is an important development for Diffusion and TSC’s clinical program, and potentially for patients suffering from GBM,” said David Kalergis, the company’s chief executive officer. “We are currently initiating a phase I/II clinical trial of TSC to test the drug’s safety and effectiveness in patients newly diagnosed with GBM.”

The standard treatment for GBM includes surgical removal of the tumour bulk, followed by radiation and chemotherapy. Factors within the tumour micro-environment of GBM tissue are thought to account for tumour recurrence, often within months. TSC is believed to enhance oxygen diffusion into this micro-environment, making treatment-resistant tumour cells up to three times more susceptible to standard radiation and chemotherapy treatments.

The United States Orphan Drug Act of 1983 was created to promote and support the development of new drug therapies for diseases that affect fewer than 200,000 people in the United States. Orphan Drug Designation provides a sponsor seven years of market exclusivity for the designated therapeutic indication in the United States, from the point at which the therapy is granted marketing approval thereby offering competitive protection. Orphan Drug Designation also provides access to regulatory support from the FDA, potential FDA fee reductions and tax credits related to development expenses.

TSC is a novel small molecule that treats oxygen deficiency (hypoxia) at the cellular level by enhancing the diffusion of oxygen selectively to oxygen-deprived tissues. TSC creates more hydrogen bonds among the water molecules which make up the bulk of blood plasma, reducing resistance and enabling a faster rate of oxygen diffusion through the plasma. TSC’s ability to safely promote the movement of oxygen to hypoxic tissue has been consistently demonstrated in animal models, including cancer, stroke and heart attack. In addition, TSC has been tested in safety and dose-ranging clinical trials in 70 human subjects. These studies, which showed that TSC was well-tolerated with minimal side effects, also provided valuable pharmacokinetic and dosing information for the next phase of clinical development.

Diffusion Pharmaceuticals is a clinical-stage company developing first-in-class drugs which target the numerous unmet medical needs characterized by hypoxia at the cellular level.