Both Panno and I agree that a possible AMD deal could possibly be made before the end of this year, and that is based on the work that has been developing rather quickly regarding the program. It's my hunch that once efficacy is seen in the TT-034 trial, then proof of shrna's ability to silence genes will be unarguable. This proof of concept could very well be the only thing that truly changes the trajectory of this company in a substantial way, and initiate deals. This is why management and shareholders alike look forward to therapeutic levels. It's where the proof is in the pudding, and ddRNAi is solidified as a major player in the gene therapy realm. Efficacy also is a major launch for the Hepatitis B program as well.
Panno's response
Author’s reply » Often, the FDA requires that new treatments be trialled in at least two different species before it gives approval for an IND. (Sidebar: this could be a problem with the Tribetarna trial because all the pre-clinical work was in mice.) I think that the company has already signed an agreement with a partner to do the testing of the AMD treatment in NHP. This means that, as soon as 4D has a vector that we are satisfied with, testing in a large animal model can begin. This would pave the way for a clinical trial.
There is no doubt that this program is going full steam ahead and I suspect that this schedule is being driven by big pharma. I suspect that one or more of the big pharmas has made it clear to Benitec what needs to be achieved before they buy into the program and the company is doing as much as it can to get to that stage. It would therefore not surprise me to see a deal done for the AMD program before it gets into the clinic. The increased funding that a deal would provide could easily see the start of a trial in 2016.
