SYDNEY, April 15, 2013 /PRNewswire/ -- RNAi-based
therapeutics company Benitec Biopharma Limited (ASX Code: BLT)
today announced the selection of the University of California, San Diego (UCSD), Health
Sciences as the second site for its upcoming phase I/II
first-in-man trial for TT-034 in Hepatitis C infections (HCV).
Benitec previously announced the selection of Duke Clinical
Research Unit as the other site. TT-034 is being developed as a
potential "one-shot-cure" for HCV.
A consultant and sub-principal investigator for the study from
UCSD Health Sciences will be Robert
Gish, M.D., clinical professor of Medicine and medical
director of Hepatology. Dr. Gish is a renowned hepatitis researcher
with previous experience using RNAi based therapeutics for HCV. He
has over 500 publications in the field and is a fellow of the
American College of Physicians and the American Association for the
Study of Liver Disease.
"I look forward to working with Benitec and Duke University on this important program," Dr.
Gish commented. "This is the first time that this therapeutic
modality is being tested in humans, and if it is successful I
believe it can be a significant step forward, not only for HCV
treatment but potentially also as a treatment modality for
Hepatitis B."
The principal investigator for the study at UC San Diego is
David Wyles, M.D., associate
professor of Medicine at the UC San Diego AntiViral Research
Center, the clinical research site that will be conducting the
trial. His research interests include the laboratory evaluation of
new antiviral therapies for HCV, drug resistance to HCV antivirals,
and HCV viral fitness.
Peter French, Ph.D., chief
executive officer of Benitec said, "We are elated that UC San Diego
and Dr. Gish will participate in this study. We now have two
top clinical research teams working with Benitec on this
trial. This constellation of expertise will greatly benefit
our HCV program and can help demonstrate the power of our ddRNAi
technology in the clinic. Having our two clinical centers in
place moves us a step closer to initiating the Phase I/II clinical
trial, which we expect to occur during the second half of
2013."
The phase I/II clinical trial is an open-label dose escalation
study to evaluate the safety and activity of single doses of TT-034
in patients with chronic HCV genotype 1 infection who have failed
previous treatments. The trial is expected to involve 14 patients
in 5 sequential dose cohorts. Additional consolidation cohorts may
be added during the study to confirm the results of the trial. The
primary safety endpoints are dose limiting adverse events. The
primary end points are serum viral load reduction and degree of
hepatocyte transduction (measured through liver biopsies). There is
a pre-specified interim read on safety and activity within months
of trial commencement.
About TT-034
TT-034 is a potentially transformative therapeutic that is
intended to provide a "one-shot-cure" for Hepatitis C with a single
injection. TT-034 works through RNA interference (RNAi), which is a
naturally occurring regulatory process in cells that acts to
"silence" genes after they have been transcribed from DNA into
messenger RNA. Benitec's proprietary ddRNAi approach involves the
introduction of a DNA vector that produces short hairpin RNAs
(shRNAs) that are processed by the cell into siRNAs. This approach
emulates the cell's own gene silencing mechanism and provides long
term activity (months). Moreover, the virus vector used to deliver
the TT-034 construct, an engineered non-replicating
adeno-associated virus (AAV8), targets almost exclusively liver
cells (where HCV replicates). TT-034 is further designed to prevent
viral escape through mutations (a major problem for most HCV drugs)
by using three different shRNAs to simultaneously target three
separate highly conserved regions in the HCV genome. In mice and
monkeys, TT0-034 has been shown to transduce 100% of hepatocytes in
the liver and provide high shRNA activity for 180 days (the
duration of the studies), without adverse effects.
About UC San Diego Health Sciences
University of California, San Diego
Health Sciences comprises clinical and academic entities – UC San
Diego Health System, the region's only academic health system; UC
San Diego School of Medicine, one of the top US research-intensive
schools of medicine; and Skaggs
School of Pharmacy and Pharmaceutical Sciences. The US
National Institutes of Health (NIH) ranks UC San Diego Health
Sciences as one of the top institutions in research funding per
faculty member, and the School of Medicine is in the top 10 in
total NIH research funding. Part of the University of California system, UC San Diego –
founded in 1960 – is renowned for collaborative and
cross-disciplinary research that transcends traditional boundaries
in science, engineering and the humanities.
About Benitec Biopharma Limited:
Benitec Biopharma Limited (ASX Code: BLT), based in Sydney, Australia, has a pipeline of in-house
and partnered therapeutic programs based on its patented
gene-silencing technology, ddRNAi. Benitec is developing treatments
for chronic and life-threatening human conditions. Its most
advanced program is TT-034 for the treatment of chronic HCV
infection. Benitec has licensed ddRNAi technology to other
biopharmaceutical companies who are advancing their programs toward
the clinic for applications including HIV/AIDS, retinitis
pigmentosa and Huntington's disease. For more information on
Benitec refer to the Company's website at www.benitec.com.
For more information please contact:
For UCSD:
Ms. Jackie
Carr, Communications Director, UCSD Health
System
Phone: +1-619-543-6163. jcarr@ucsd.edu.
http://som.ucsd.edu
For Benitec:
Dr. Peter
French, Chief Executive Officer
Phone:
+61 (02) 9555 6986. pfrench@benitec.com. www.benitec.com
SOURCE Benitec Biopharma Limited
