SYDNEY, May 29, 2014 /PRNewswire/ — RNAi-based therapeutics company,
Benitec Biopharma Limited (asx:BLT) is pleased to announce that it has
dosed the first patient in its ‘first in man’, Phase I/IIa clinical
trial for TT-034, a ddRNAi-based therapeutic, designed to treat and
potentially cure hepatitis C (HCV) with a single injection.
commencement of this clinical trial of TT-034 represents a landmark in
the Company’s history. The trial is the first time Benitec’s gene
silencing technology, ddRNAi, has been used systemically in patients.
can be used safely in patients with HCV. Preclinical work in non-human
primates demonstrated very low toxicity results at therapeutically
relevant doses, and we’re hopeful that we will see the same favourable
tolerability in humans. In addition, we will be able to assess the
impact of TT-034 treatment on HCV viral load in these patients, and this
important efficacy marker constitutes one of the secondary endpoints of
study in a total of 14 patients chronically infected with HCV genotype
1. Initial patient cohorts will be treated with a sub-therapeutic dose
of TT-034 to ensure that there are no unexpected safety concerns, before
proceeding to higher, potentially therapeutic doses.
is independent of Benitec, will carefully assess the data from each
patient, in particular the safety data. The DSMB assessment will occur
after the first patient in each cohort and between cohorts, and will
determine the timing of each subsequent dosing.