Still, gene-based therapy continues to be very important and we’re continuing to learn a lot from these trials. And really big news is with tominersen, which is an antisense oligonucleotide, an ASO, and some results have been released and shared in the recent weeks. And tominersen, as many people know the drug dosing was stopped in March of last year due to unfavorable risk-to-benefit ratio in the drug versus the placebo group...
Still, gene-based therapy continues to be very important and we’re continuing to learn a lot from these trials. And really big news is with tominersen, which is an antisense oligonucleotide, an ASO, and some results have been released and shared in the recent weeks. And tominersen, as many people know the drug dosing was stopped in March of last year due to unfavorable risk-to-benefit ratio in the drug versus the placebo group. And Roche, the company running this trial released some data there was… while it did lower the mutant huntingtin levels effectively, there was no clinical efficacy. And in fact, not only was there no clinical efficacy, but those on the participants on the drug, the more frequently dosed group, they did worse than those who were on the placebo.
And they also performed a post hoc analysis. And in doing that, they divided the participants into four subgroups based on age and the CAP score. And CAP score is it takes into account age and the CAG repeat expansion and it’s an estimate of the length and severity of the individual’s exposure to the effects of the expanded CAG repeat. So they perform the median split. And so based on the age and the CAP score, so they had four different groups with low agent, low CAP score and different combinations of which one’s high and which one’s low. But the bottom line is when they did the post hoc analysis in the low age and the low CAP score group that being age of less than 48 years old and CAP score of under 500, there was a trend towards the less frequently dosed group being numerically superior to the placebo group when they looked at this composite score that takes into account a clinical performance or clinical exam scores, cognitive scores and functional scores.
So again, this was statistically not significant and as a post hoc analysis, but based on that analysis, there’s some considerations for a potential Phase II study again still using the tominersen. It’s not a certain thing that it’s going to go into Phase II, but if that were to be designed, then there would be considerations for younger patients, those with lower CAP score and probably lower dosing, or maybe less frequent dosing. But the goal would be to have a lower goal of mutant huntingtin lowering and less exposure to the drug. So that was the big news for tominersen.
And the other ASO drug which is allele-specific and that means a drug is targeted at a specific SNP, and that’s a drug called WVE-003, and that’s also in development and will go into a trial. And the main difference for that drug is compared to its predecessors which were targeted at two other SNPs, it has a different backbone chemistry. So that different backbone chemistry is expected to lead to better distribution, greater potency, greater half-life, and there’s some positive animal data. So that’s something to look forward to as well.
