|[November 19, 2012]
Diffusion Pharmaceuticals Successfully Completes Phase I Brain Cancer Trial
CHARLOTTESVILLE, Va. --(Business Wire)--
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
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.
For more information about Diffusion Pharmaceuticals LLC, visit
Pharmaceuticals LLC web site.
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