Ann: Further Significant Improvement in Rett Syndrome Patients, page-11

Not sure what the source of confusion is htae, by my read:

• For Primary end-point CGI-I (4 anchors) base line was 4.0, plus 12 weeks was 3.1 and after 20 weeks was 2.7, both periods statistically significant against baseline
• For Primary end-point (9 anchors) at 12 weeks a reduction of 0.4 was also statistically significant.
• If only 2 anchors are required for primary end-point registration trial (as stated above) then I can't see the issue, however if the regulator wants access to the 9 anchors I'm sure that will be part of the data pack.
• For the Secondary end-points, RSBQ was highly significant whilst CGI-S still significant but less dramatically so.

My expectation is that these early phase clinical trails are (along with safety) very much about finding which indications are positive impacted for the specific drug, with each drug having it's own configuration of indications to treat. Trofinetide presumably has a different suite of indications for which it is successful.