TT-034 is Benitec's (BNIKF, BTEBY) one-shot treatment for HCV.
Today, the company released two ASX announcements regarding the trial of TT-034. This news had been long awaited by shareholders who are becoming used to delays in the trial.
The first release confirmed that cohort two in the trial has now been fully subscribed and that one new trial site, the Texas Liver Institute in San Antonio, is now actively recruiting patients for the trial. The new site is a well respected clinical site and so it is very good news that the team at Benitec has been able to bring this site onboard, even if it has taken them much longer to do so than the team initially indicated. It is still a concern that the UC, San Diego does not appear to have recruited any patients to date and hopefully this situation can be rectified shortly.
The announcement also made it clear that the issuing of news on a patient by patient basis will not continue. This may be frustrating for shareholders but no other trial is reported in that way and so it should not be expected of the TT-034 trial.
The second announcement was a copy of the presentation given by Dr Suhy to the Australian Gene Therapy Conference. The presentation, by and large, consisted of information that had already been made available through other presentations and announcements. However, slide 26 was of particular interest as it quoted some average hepatocyte transduction rates for the first three patients and concluded that these were "roughly consistent with pre-clinical studies". As the data provided did not quote the number of days after dosing that these rates were measured, and as they were not recorded as individual shrna's (as is the case with the pre-clinical studies), a base from which to compare these data cannot be established.
On the same slide it says, "If consistent with pre-clinical studies, cohort 3 may have a significantly higher percentage of DNA transduction". One would hope that this would be the case. However, in addition to the good news on the TT-034 safety profile, sometime very soon, Benitec is going to have to change its description of the trial and start talking about viral load reduction and not rates of transduction. My take on this comment on cohort 3 is that the team is only expecting a modest reduction in viral load in cohort 3 patients. However, with the escalated doses in cohorts 4&5 to follow, this could still mean that TT-034 is in with shot of being a successful treatment for HCV.
The Quarterly Report showed that the company is in a sound financial position with $AUD26.7 in cash at the end of March 2015.
Additional disclosure: The author is long on Benitec. This article is not intended to be investment advice. Readers should do their own research.