Interview with Dr. Lou Vaickus, Tolerx, Question 5

LV: We are bullish about our chances for a good outcome with otelixizumab. In nonhuman animal models of Type 1 diabetes the standard published dose of an anti-CD3 antibody was a "high dose." So we have drastically lowered the dose there (break) too and have seen results that are just as good as with much higher doses. This mimics what we are doing in people with Type 1diabetes to a "T" and gives us even more encouragement about the optimized regimen that we are using in our Phase 3 studies.  

Speaking of "T", another interesting fact is that when we look at the effects of otelixizumab in the laboratory using human cells we see that differential effect (inhibit the T killer/effector cells and stimulate the good ones, the T regulatory cells) and it is most prominent at low dose levels, similar to the levels we would expect to see in the clinic after patients get otelixizumab. 

Our preliminary results in our Phase 2 studies, as I mentioned, are also promising. But, the proof is in the two large pivotal Phase 3 studies and the primary endpoint in both remains blinded until 12 months after the last patient gets dosed. We are allowed to see other results in an unblinded fashion in these studies so we know that some people are increasing their insulin use while others have decreased their insulin or stopped it altogether. What we don't know is what everyone is waiting for: did the folks who reduced/stopped their insulin injections receive otelixizumab? And, how long will this effect last? And, did those who reduced their insulin do so because they made higher amounts of their own (endogenous) insulin (i.e., was a reduction in insulin injections accompanied by an increase in C-peptide, our surrogate marker of beta cell function?). Lots of questions to be answered but we will need to await the final results.