This is the most important statement in the announcement:
While the actual numbers are good, they are not outstanding. Having said that, this is data from a mouse model and needs to be seen as a good start and definite grounds for continuing the in vivo work. Furthermore, if the numbers continue to drop, as stated, then the final level data, had it been collected, could have been outstanding.
What I would also like to see is the data gathered from the TT-034 trial relating to the vector. For BB-HB-331 to be a cure, the vector will have to reach 100% of hepatocytes (or very close to this number). The advantage we have is knowing whether or not such a level of transfection was reached and at what dose. This knowledge will determine if the current vector up to the job, as the ddRNAi component seems to have promise.
BB-HB-331 versus Tenofovir. We really cannot make a comparison at this stage. However, we do know that tenofovir, like the rest of the HAART combinations, does not cure HBV, it only represses it. Tenofovir has to be taken for life but BB-HB-331 is designed to be a single infusion treatment. Even if BB-HB-331 ends up not curing HBV but only repressing, the drug still has a future because a single infusion is better than a lifetime of treatment.
For those interested, this is what Arrowhead had to say about ARC-520,
Alnylam still believes that HBV is worth pursuing but they have deeper cash reserves than we do. Let’s hope that we can get a partner when the next round of in vivo data is available so that we too can start throwing some serious cash at the research.
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