RNAi-based therapeutics company Benitec Biopharma has selected the Duke Clinical Research Unit, the early phase unit of the Duke Clinical Research Institute (DCRI) of Durham, N.C., as a site for its upcoming phase I/II first-in-man trial for TT-034 in hepatitis C (HCV). TT-034 is being developed as a potential "one-shot-cure" for 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., CEO 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 five 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. 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," said Patel. "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."