A recent one-liner in a presentation by one of the TT-034 trial sites has thrown up a serious issue with regard to the criteria for recruiting patients. In that presentation the University of California, San Diego stated that a high AAV8 neutralizing antibody (nAb) count was the main reason why it has so far failed to recruit a single patient for the trial. This reason is not one previously alluded to in company announcements concerning the delays in the TT-034 trial.
If the number of patients presenting for the trial having nAb is much higher than expected and if this criteria will be extended to the commercial application of TT-034 (should it get that far), then there are some serious ramifications for market-share, return on investment and, indeed, for the HBV program. These matters are serious, material matters affecting the company and as such they should be addressed by Benitec's ($BNIKF, $BTEBY)management, if there is any substance to them.
I am quite sure that Benitec's Board and management are well aware of their obligations on such matters and so I believe that they are following one of two courses of action: Either they are waiting for confirmation of the percentage of the patient populations with nAb at both trial sites, with a view to releasing this information; or they intending no release of information because they are convinced that the nAb situation will have no material impact on the trial (other than what was intended by having this exclusion criteria in the first place).
It may be that shareholders have implied more meaning in the UCSD statement than they should have and that there is no new decrease in patient viability but I still think that the matter should be addressed by management. I say this because UCSD is a trial site and so it is a credible source of information and because the statement is inconsistent with the information coming out of Duke, the other clinical site.
I would point out that the inclusion/exclusion criteria may be relaxed in any new trial. If the current trial proves to be both safe and efficacious, the FDA may agree to criteria that are more inclusive for the Pllb trial. This may mean that AAV8 nAb patients are included so that the actual degree of immune response can be assessed. It is possible that the presence of nAb could still be a manageable situation. This would be the best outcome for the program.
I am looking forward to reading more information from management before this matter gets raised at the AGM.
Disclosure: The author is long BNIKF.
Additional disclosure: This article is not intended as investment advice. Readers should do their own research.