@ScoonieThe results looked encouraging to me at first read. I wasn't able to attend the webinar and have only given this a quick scan, so my comments could be way out of line.

The lower patient numbers and that fact this wasn't a placebo controlled, double-blind trial means that its not really a fair comparison to put the results alongside Lavender. That objection aside, the NTI 164 RSBQ scores have a 95% CI of -20.3 to -6.5, which compares with a 95% CI in the Lavender trial of -5.1 with a SE of 0.99, so you could estimate the 95% CI as -7.0 to -3.2.

Its not clear to me what the comparison of the CGI-I scores is, or if one is even possible. In addition to the small patient sample and lack of placebo control, it is not immediately clear to me how many or which anchors were used in the trofinetide trials. So while NTI164 seems to have a positive effect, I don't think any comparison is possible. Because for both, the effect is modest, lack of placebo control and sample size for NTI164 are going to be important. Which is maybe why no comparison was offered ... the interval would just be too wide. In the publications on trofinedite clinical trial results, there is a bit of detail set out in how the clinicians are trained to a "gold standard" so that they apply the 7-point scale consistently. So I guess we'll have to wait until the peer-reviewed paper comes out for NTI164 to see if the methods are truly comparable. But even then, the lack of a placebo control will throw a question over any effect, as the clinicians will be a different group and it is a subjective test. Again, I am not an expert and so these are more questions than statements.

It is also not clear what the next step for NTI164 for Rett's is. Presumably, they are going to see what gets through the peer review process, and what feedback they get at the Rett's Conference in October this year. They'd want to have a journal publication by then, and that should be OK if everything is good in their study design and analysis.

I am not an expert in this area by any means, but I think they've have to figure out whether they are going to do a full Phase 2, to help design the end points for a Phase 3 study. With the smaller sample size at Phase 1/2, the end point design might be trickier in going for a combined Phase 2/3. Being the second-to-market probably raises the bar for the approval. Second-to -market has to show some kind of benefit (whether in elements of efficacy, side effects, or benefits for some sub-population) relative to first. NTI164 does look favourable on side effects, if I am reading things properly.

So until there's more information on the way forward, it is hard to judge the timeline for Retts.

$NEU got to Phase 2 readout in 2017 on 82 patients and it took another 6 years to get to market. They didn't have a straightforward passage through Phase 3 - I haven't understood all the details, but recruitment got halted at some point.

So, with NTI164 at Phase 1/2, I think there are several years ahead for the Retts indication. I'm guessing DAYBUE has a clear run for at least 3 years and maybe more than 4.

I haven't read any commentary on the presentation, but I do note that the SP initially ran up 8-9% then fell back 17% to be down about 10% on the day. Did something come up in the investor call that put a wet blanket on what looks to be promising, at first glance?

Anyway, one to keep on the radar screen.

Disc Not held