SYDNEY and DURHAM, N.C.,
March 21, 2013 /PRNewswire/
-- RNAi-based therapeutics company Benitec Biopharma Limited
(ASX Code: BLT) today announced the selection of the Duke Clinical
Research Unit, the early phase unit of the Duke Clinical Research
Institute (DCRI), Durham, North
Carolina, USA as a site for its upcoming phase I/II
first-in-man trial for TT-034 in Hepatitis C. TT-034 is being
developed as a potential "one-shot-cure" for Hepatitis C (HCV).
"We are very excited to be working with Duke, a world renowned research institution with
significant experience in this area," said Peter French, Ph.D., Chief Executive Officer of
Benitec. "The TT-034 trial marks the transition of Benitec to a
clinical stage company. We expect that positive results from the
trial will provide a value inflection point for the company, and
also be a validation for our ddRNAi technology as an effective
platform for therapeutics."
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 activity 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. The clinical
trial is expected to begin enrolling patients during the second
half of 2013.
Duke's principal investigator for
the study will be Keyur Patel, M.D.
Dr Patel has previous experience with oligonucleotide therapeutics
in HCV, is a recipient of the prestigious American Association for
the Study of Liver Diseases (AASLD) Shelia Sherlock Clinical and
Translational Research award and has over 100 citations in
peer-reviewed publications.
"TT-034 is a potentially transformative new treatment," Dr.
Patel commented. "A therapeutic that could cure an HCV patient with
a single injection would obviously be a big step forward compared
to even the best treatments that are currently on the horizon, as
they all involve comparatively lengthy regimens with a combination
of several drugs."
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 the Duke Clinical Research Unit:
The Duke Clinical Research Unit (DCRU) is a state-of-the-art
research facility located within the Duke Medicine campus that
provides infrastructure support to sponsors and investigators who
are testing new drug candidates and other cutting-edge therapies,
or seeking to identify and validate novel biomarkers. The DCRU has
more than 20 years of early-phase clinical trial experience and has
successfully conducted more than 150 early-phase studies, including
80 phase 1 studies. The experience, support systems and
infrastructure of the DCRU enable provision of the highest level of
program management and services for early-phase clinical trials,
including quality processes, accurate reporting, and regulatory
expertise. The DCRU combines the clinical expertise and scientific
leadership of one of the most prestigious academic medical centres
in the world with the operational capabilities of a full-service
contract research organization. The DCRU is part of the Duke
Clinical Research Institute. The DCRI is a comprehensive academic
research organization and the only one of its kind that can offer
all the services of a commercial contract research organization
with the academic credibility and expertise of an academic medical
center.
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 Duke Clinical Research Unit:
Barry Mangum, PharmD | Director
Clinical Pharmacology
Phone: 919 210 8099 |
Barry.Mangum@duke.edu | www.dcru.org
For Benitec:
Dr Peter
French | Chief Executive Officer
Phone: +61 (02) 9555 6986 | pfrench@benitec.com |
www.benitec.com
SOURCE Benitec Biopharma Limited
