Diffusion Pharmaceuticals LLC, a biotechnology company developing innovative cancer treatments, today announced that it has successfully completed a Phase I clinical trial of its lead compound trans sodium crocetinate (TSC) in nine patients with newly diagnosed primary brain cancer (glioblastoma or GBM). A Safety Monitoring Committee comprised of expert, independent clinicians and researchers met on November 14, 2012, reviewed the Phase I patient safety data and authorized immediate open enrollment for the follow-on Phase II study.
The Phase II trial will explore TSC’s effects on tumor regrowth, patient quality-of-life and overall survival in newly diagnosed GBM patients when dosed in conjunction with conventional radiation therapy. The study will enroll a total of fifty patients at twenty cancer institutions throughout the U.S., with a target date of complete enrollment by early 2013. Patients in the Phase I brain cancer study were treated at six cancer institutions around the country. Fourteen additional centers will be open for the Phase II study, which has already begun enrolling patients. Further information about the study may be viewed online at Glioblastoma Clinical Trial Phase II.
“This confirmation of TSC’s safety and dosing regimen has prompted the immediate expansion of treatment to a larger number of newly diagnosed GBM patients,” said David G. Kalergis, MBA/JD, the CEO of Diffusion Pharmaceuticals. “The timely opening of Phase II means that statistically-powered efficacy data should be available on schedule later in 2013.”
The Phase II study builds on several previous Phase I/II studies establishing TSC’s human safety profile, pharmacokinetics and dosing regimen in eighty human subjects, including normal healthy volunteers, peripheral artery disease, and GBM patients. The current study will focus on the efficacy and safety of TSC as an addition to the standard-of-care in newly diagnosed GBM patients. Study endpoints include TSC’s effects on tumor regrowth as determined by MRI, the patient’s quality-of-life during and after their treatment, and their overall survival at one and two years after treatment.
TSC was granted an FDA Orphan Drug designation for its use in GBM in 2011, and an Orphan Drug designation for its use in brain metastases is pending. Orphan Drug designation confers substantial benefits, including enhanced patent protection and marketing rights, waiver of certain FDA filing fees, and favorable tax treatment.
Dr. James Larner and Dr. Jason Sheehan of the University of Virginia Health Sciences Center are Study Co-Chairs for this multi-institutional trial. Other cancer institutions participating in the trial may be found at Glioblastoma Trial Locations for Phase II.
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 a very well tolerated 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 and approved chemotherapy.
About Diffusion Pharmaceuticals
Diffusion Pharmaceuticals is a clinical-stage company developing first-in-class drugs that 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 currently being planned. Diffusion Pharmaceuticals, which is privately held, is located in Charlottesville, Virginia.