SYDNEY, Jan. 7, 2015 /PRNewswire/ — Benitec
Biopharma Limited (ASX: BLT, OTC: BTEBY) is pleased to advise that the
third patient in its Phase I/IIa clinical trial of TT-034 for hepatitis C
was dosed earlier today at the Duke Clinical Research Unit (USA).
This is a significant step for this “first in man” study, and follows
review of the collective data from the first two patients by the
independent Data Safety Monitoring Board (DSMB). The DSMB determined
that the patients from the first dosing cohort were clear of any
significant treatment-related adverse events.
newly dosed patient is the first to receive the increased dose of
TT-034 (1.25 x 10^11 vg/kg, a concentration that is a half log higher
than the doses administered in the first cohort). While TT-034 is
designed as a potential “one-shot” cure for hepatitis C, the current
dose is still below that expected to inhibit viral replication and data
from the second dosing cohort are therefore expected to serve primarily
as a further safety assessment.
with previous patients, the newly dosed patient will be monitored for
six weeks and results will be reviewed by the DSMB. Should the results
indicate appropriate safety outcomes, the DSMB is expected to recommend
that the remaining two patients in the second cohort be dosed. It is
aimed to dose both at approximately the same time. The trial sites at
Duke Clinical Research Unit and University of California San Diego have identified a number of patients who have passed initial screening who can be prepared in anticipation of this outcome.
is a ddRNAi-based therapeutic, designed to treat and potentially cure
hepatitis C (HCV) with a single administration. TT-034 targets the
hepatitis C viral RNA at three separate, highly conserved sites. As such
it acts as a “triple therapy” even though it is a monotherapy, and
minimizes the ability of the virus to mutate and escape the therapy.
Once it reaches the liver cells, it enters the nucleus and produces
three separate short hairpin RNAs continuously for the lifetime of the
cell. Thus TT-034 has the potential to not only treat the existing HCV
infection, but also to guard against reinfection for months to years
without the need to re-treat. TT-034 safety and efficacy has been tested
extensively in pre-clinical in vivo studies with no adverse effects
observed at therapeutic doses.
About Benitec Biopharma Limited
Biopharma Limited is an ASX-listed biotechnology company (ASX: BLT;
OTC: BTEBY), which has developed a patented gene silencing technology
called DNA-directed RNA interference (ddRNAi). ddRNAi has the potential
to produce ‘single-shot’ treatments and even cures for a range of
chronic and life- threatening human conditions. Based in Sydney, Australia, with labs in Hayward CA
(USA) and collaborators and licensees around the world, the company is
developing ddRNAi-based therapeutics for diseases including hepatitis C
and B, drug resistant lung cancer and wet age-related macular
degeneration. Benitec has licensed ddRNAi to other biopharmaceutical
companies for human therapeutic applications including HIV/AIDS,
Huntington’s Disease, cancer, chronic neuropathic pain and retinitis
pigmentosa. For more information visit www.benitec.com
For further information regarding Benitec and its activities, please contact the persons below, or visit the Benitec website at www.benitec.com
SOURCE Benitec Biopharma Limited