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.