Benitec (BNTC, OTCPK:BNIKF) is to present early findings of its TT-034 clinical trial for HCV at the American Association for the Study of Liver Diseases in San Francisco next month. The abstract outlines the progress to date.
Based on the information in the abstract, I think that the company is quietly confident that TT-034 will prove to be successful. This view is supported by the following:
- There have been no safety issues reported to date.
- TT-034 has been detected in the liver biopsies at levels similar to those seen in the non-human primates used in the pre-clinical studies.
- The individual therapeutic components (three shrna's) at the third level of dosing have been reported at levels that pre-clinical tests showed to be therapeutic.
This is all very positive news! However, there are also some things that are not mentioned which need clarification before shareholders start counting their profits.
Firstly, the pre-clinical rate of liver cells transduced by the AAV vector is still being reported. While the indication is that this transduction rate is "similar" to those in patients in the trial (at the various dosing levels), there is no firm statement of what transduction levels have been observed. It is said that in the pre-clinical stage nearly 100% of hepatocytes were transduced at therapeutic doses but no definitive figure has been reported for the patients in the trial.
Secondly, there is no mention of sustained viral load reduction. No matter what other measures are used, this one is the key measure of whether or not the treatment is working. Given the relatively small number of patients dosed at a therapeutic level, I can fully understand why the company would be reluctant to publish any data on this measure at this point in time. This is, therefore, nothing to be alarmed about. That said, there is no guarantee that the trial is progressing in the same way as that of the pre-clinical replicon model of the disease. So, while reference to shrna's levels is encouraging, there is still no data to show that these shrna's are achieving the same level of success as that reported in the pre-clinical, replicon studies. When this is confirmed, investors can start thinking about how much the treatment could be worth. Until then, there is still a degree of uncertainty.
It has taken a while for the company to get to the stage of having data available and what is available (to the public) is still incomplete, but I think there is every reason for the company and its shareholders to be optimistic about the performance of TT-034. Furthermore, if the AVV vector is proven to transduce human liver cells at the same rate as the pre-clinical model, then this will be a big boost for Benitec's HBV treatment and that could be worth a whole lot more than TT-034.
Disclosure: I am/we are long BNTC.
Additional disclosure: This article is not intended to be investment advice. Readers should do their own research.