11 Dec Diffusion Pharmaceuticals Receives FDA Orphan Designation for TSC, a First-in-Class Drug for the Treatment of Metastatic Brain Cancer
Charlottesville, VA – December 11, 2012 (Business Wire) – Diffusion Pharmaceuticals LLC announced today that trans sodium crocetinate (TSC), the company’s lead drug candidate, has been granted an Orphan Drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of metastatic brain cancer. Metastatic brain cancer is one of the most deadly forms of cancer, with most of its victims dying within a year of diagnosis. The FDA has previously granted Diffusion Pharmaceuticals an Orphan Drug designation for the use of TSC in the treatment of the less-frequently occurring brain cancer known as glioblastoma multiforme or GBM. A Phase II clinical trial testing TSC in newly diagnosed GBM patients is now enrolling patients at twenty major cancer centers around the US. A Phase II clinical study testing TSC in brain metastases is currently being planned in cooperation with these centers.
“This Orphan Drug designation for TSC is an important step for Diffusion Pharmaceuticals,” said David Kalergis, the company’s Chief Executive Officer. “There is currently no approved drug for the treatment of this condition, and TSC, if successful, would provide a much-needed treatment option. When combined with TSC’s progress in the on-going Phase II trial in GBM patients, we are hopeful that this new Orphan Drug designation will help us to initiate a trial of TSC in brain metastases as soon as possible.”
Brain metastases, or cancer cells which have migrated to the brain from other locations in the body, affect about 170,000 patients in the US every year, and better treatment is considered a pressing unmet medical need. Currently, treatment may include surgical removal of the tumor bulk and limited radiation therapy. There are no drugs approved by the FDA for the treatment of brain metastases, although doctors may sometimes prescribe certain chemotherapy drugs “off label.” Factors within the micro-environment of metastatic tumor tissue, especially oxygen deprivation (hypoxia), are thought to play a role in treatment resistance and tumor recurrence. TSC is believed to enhance the diffusion of oxygen into this hypoxic micro-environment, making treatment-resistant tumor cells up to three times more susceptible to standard radiation treatment.
About Orphan Drug Designation
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.
Trans sodium crocetinate (TSC) is a proprietary, first-in-class small molecule belonging to the novel “oxygen diffusion-enhancing compound” family of drugs. Its mechanism of action is unlike that of any previous drug, combining potent efficacy-enhancing properties with an excellent human safety profile. Many cancers, including GBM and brain metastases, contain cells that are resistant to treatment because of diminished oxygen levels (hypoxia) within the tumor. TSC causes more oxygen to diffuse into hypoxic tumor tissue without affecting normal tissue, thereby enhancing the tumor killing power of conventional treatments. Published animal studies in GBM show that TSC controls tumor growth and triples survival without observed side-effects when used in conjunction with standard-of-care radiation.
About Diffusion Pharmaceuticals
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. These proprietary small molecules work by a novel mechanism of action that enhances the diffusion of oxygen selectively to hypoxic tissue. Potential breakthrough clinical applications include oncology, cardiovascular diseases, stroke and respiratory disorders. Diffusion’s lead program is the use of TSC in the treatment of solid cancerous tumors. A Phase II clinical study of TSC dosed concurrently with radiation in newly diagnosed GBM is currently enrolling patients at twenty cancer centers throughout the US. A Phase II clinical trial of TSC in the treatment of brain metastases is being planned. Diffusion Pharmaceuticals, which is privately held, is located in Charlottesville, Virginia.