The first patient dosed in the trial has to date experienced no treatment related adverse effects. Liver biopsy results from the first patient were encouraging and in line with expectations. TT-034 DNA and short hairpin RNA (shRNA) were both detected in the biopsy at sub therapeutic levels, indicating that liver transduction occurred.
As the first dose was intended to prove safety only, this is as good a piece of news as could be expected (although no results from a single patient can be seen as representative of the broader HCV community).
This safety outcome enabled the trial's Data Safety Monitoring Review Board to approve the dosing of the next patient (in accordance with the trial protocols).
The first two cohorts in the trial are being dosed at sub therapeutic levels and so no data on the efficacy of TT-034 was expected at this stage and none was released. However, if we examine the last sentence in the statement above and compare this to what we do know we can get some idea of what may be occurring. The statement tells us that TT-034 has be transduced into the patient's genome and that shrna is being produced. We also know that TT-034's shrna molecules are extremely efficient at knocking down HCV RNA (refs: 1, 2). The combination of these two pieces of information tells us that, even at these sub therapeutic dosing levels, viral RNA must be being knocked down and therefore there must be some reduction in the viral load of this first patient.
The questions we now need to ask are: is this viral load reduction measurable and for how long will the reduction be sustained?
The answers to these questions cannot be determined at this point in time and so we will have to wait until more data is release by the company. What we can say is that all the indications so far are very positive and hopefully the next cohorts will prove the case for TT-034 being an effective treatment for HCV.
Disclosure: The author is long BNIKF.
Additional disclosure: The article is not intended as investment advice. Readers should do their own research.