Benitec Biopharma Limited announced that it has dosed the third patient in its Phase I/IIa clinical trial with TT-034 for hepatitis C, at the Duke Clinical Research Unit (USA). This marks an important step for the study. The collective data regarding the first and the second patients’ outcomes were reviewed by the Data Safety Monitoring Board (DSMB) and the independent institution determined that there were no significant adverse events related with the treatment in the first dosing cohort.
The third patient that recently started the treatment is the first to receive a 1.25 x 10^11 vg/kg concentration of TT-034; this is an increased dose, half log higher than those administered in the first cohort. The TT-034 was designed to be a single-shot cure for hepatitis C, however the current dose that is being administered is still below the dose that might inhibit viral replication. The data regarding this second cohort will be important to study the safety profile regarding this higher dose.
The patient will be studied for 6 weeks and the results will be monitored by the DSMB. If the results from the first patient point to positive safety outcomes, it is expected that the DSMB will recommend that the two remaining patients of the second cohort are dosed as well, with the goal being to dose them both at approximately the same time. Some patients have already been screened at the University of California San Diego and the Duke Clinical Research Unit and identified as patients that passed the initial screening.
TT-034 attacks the hepatitis C viral RNA and targets it in three specific locations. It is a monotherapy, despite the fact it acts as a “triple therapy,” and it reduces the viruses’ capacity to mutate and not become affected by the therapy. As explained in the press release, “Once it reaches the liver cells, it enters the nucleus and produces three separate short hairpin RNAs continuously for the lifetime of the cell.” Furthermore, the TT-034 can treat an existing hepatitis c virus infection and prevent reinfection cases. The safety and efficacy tests showed no adverse effects.