Ann: 2023 Results webinar, page-125

A few things I gleaned from listening to the Q & A in the CureSHANK webinar this morning:

• The dose used in the trial (which is the same dose that will be used going forward) represents, for a 25kg patient, the equivalent of 5cc (1 teaspoon), taken twice daily.
• While an open label trial design was used in the Phase 2 study, it is expected that Phase 3 will be placebo-controlled.
• Safety was the broad reason for the exclusion of 5 patients after screening.
• Participants in the Phase 3 trial will be excluded from the pivotal Phase 3 trial as neither they nor their parents will be treatment- naïve. However, it is hoped that they can be included in the Phase 3 open label extension component of the trial.
• While not yet decided, it is probable that the pivotal trial will be restricted to the same age group as the Phase 2 trial (3-12 years). However, the company remains committed to extending treatment to all age groups and an additional trial in adolescents is a possibility.
• Geography for the trial is still to be determined.
• The last question was whether it was possible that NNZ-2591 might be applicable to syndromes other than PMS. The founder of CureSHANK was thrilled to hear that NNZ-2591 was being tested in other indications as well and might potentially be a therapeutic used across multiple neurodevelopmental rare diseases.