Phase 2, page-11

Two further trains of thought I’d like to add ….


Recent discussion here has centred on the likelihood of success in the Phase 2 Phelan McDermid study due to report out before the end of this year. We are asking - What is the chance it will succeed? What is the likely impact on share price if the study succeeds or fails? We are viewing this study as having a binary outcome.

My question is… how will “success” be judged in a small trial with a primary outcome of safety and tolerability and 14 secondary exploratory efficacy outcomes?



All six indications in which both trofinetide and NNZ-2591 are currently being developed have overlap with autism.

All are monogenic neurodevelopmental disorders. Consequently, genetic therapies aiming to “cure” or dramatically improve these disorders are an attractive therapeutic approach. There are currently multiple genetic therapies in development (preclinical and clinical) for all the indications in which trofinetide and NNZ-2591 are currently being developed. In time, I am sure there will be a genetic therapy (eg. gene therapy, antisense therapy) successfully developed and approved in all of these conditions.

However, Autism Spectrum Disorder is different. Highly heterogeneous, with approximately 100 different genes strongly identified with the disorder, it cannot be successfully tackled by a therapy targeting just one gene. Hence, a drug like NNZ-2591, which addresses symptoms irrespective of the causative gene, has a major advantage.