SYDNEY, Australia I April 7, 2015 I Benitec Biopharma (ASX:BLT; OTCPK:BTEBY), a biopharmaceutical company focused on providing potentially curative therapies with its proprietary gene-silencing technology called ddRNAi or “expressed RNAi,” is pleased to advise that laboratory results from liver biopsies in the company’s ‘first in man’, Phase I/IIa clinical trial of TT-034 for hepatitis C confirmed that the trial is proceeding according to expectations.
TT-034 works by producing (in the liver) three silencing short hairpin RNAs (shRNAs), each responsible for targeting a different part of the hepatitis C virus’ genome. The expression of the three shRNAs in patients’ liver cells is thus an essential requirement for TT-034 to exert a clinical reduction of hepatitis C viral load. The most recent assay of the biopsies confirmed this expression occurred in all three patients dosed to date. These results were obtained from the biopsies of the first two patients in cohort 1 and the first patient in cohort 2. The second patient in cohort 2 has not yet been biopsied, and the third patient in cohort 2 is yet to be dosed due to a personal issue.
In cohorts 1 and 2, the dose of TT-034 is sub-therapeutic and, therefore, the amount of shRNA produced will not result in reduction of hepatitis C viral load.
Furthermore, to date there have been no treatment-related serious adverse effects (SAEs) in any of the four patients dosed.
More detail on the TT-034 trial
TT-034 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 minimises 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 life time of the cell. Thus it has the potential to not only treat the existing HCV infection but to guard against reinfection for months to years without the need to re-treat. It has been extensively tested in pre-clinical in vivo studies and no adverse effects were seen at any therapeutic dose. However, as it is regulated as a gene therapy, the trial design is to primarily ensure that treatment with TT-034 is safe, hence the gradual dose escalation.
About Benitec Biopharma Limited
Benitec Biopharma Limited is an ASX-listed biotechnology company (ASX:BLT; OTC:BTEBY) which has developed a patented gene-silencing technology called ddRNAi or ‘expressed RNAi’. 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 chronic and life-threatening human conditions including Hepatitis C and B, drug resistant lung cancer and wet age-related macular degeneration. Benitec has also licensed ddRNAi to other biopharmaceutical companies for applications including HIV/AIDS, Huntington’s disease, chronic neuropathic pain and retinitis pigmentosa.
SOURCE: Benitec
