Ann: Trading Halt, page-80

I'd be careful with the assumption that "the data is better" due to minimal side effects. There's a lot more to this.

First of all, you're comparing a trial with 14 participants compared to a Phase 3 with 187 participants. Sample size matters a lot with these trials. NEU's Phase 2 trial had 82 patients and that reported back in 2019 so that might give you a more realistic timeframe on how these things work. The OD designation and the fact there was no approved treatment helped "speed things along" also.

Regarding the GI issues (diarrhoea):

- It should be noted the majority of Rett girls have constipation and the majority of them are on laxatives to help this.
- Within the clinical trial, there was no protocol for managing GI issues (such as stopping the laxatives they were on). After the P3 trial, protocols were developed, and persistence improved as a result.
- It became standard to titrate doses and increase over time to give the patient time for their body to use to the compound. This has made a large positive impact.
- It is believed the GI issues stem (at least partially) due to the formulation of the oral liquid (not the compound itself) - a reformulation could help address this.


I don't want to dampen NTI's results, but it would be important to understand the real story of where NTI is in relation to where NEU is.