Neuren Pharmaceuticals (ASX: NEU) has received approval from the US Food and Drug Administration (FDA) to proceed with the Phase 2 trial of NNZ-2591 in Pitt Hopkins syndrome (PTHS). The Investigational New Drug application (IND) for the PTHS program is now active.
We are excited to proceed with this ground-breaking trial in Pitt Hopkins syndrome and look forward to working with the community in the US. Neuren now has three INDs active for NNZ-2591 and we are advancing our plan to address multiple neurodevelopmental disorders that have such high unmet need,” Neuren CEO, Jon Pilcher, said.
The Phase 2 trial will be conducted at four hospitals in the United States, enrolling up to 20 children aged 3 to 17 years with PTHS to examine safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591. Commencement is subject to ethics approval from the Institutional Review Boards (IRBs).
Top-line results from the trial are anticipated in H1 2023.
Neuren also recently received FDA approval to proceed with Phase 2 trials of NNZ-2591 in Angelman syndrome and Phelan-McDermid syndrome.
In parallel with the three Phase 2 trials, Neuren is executing the foundational work to prepare for Phase 3 development of NNZ-2591 across multiple indications.
Neuren is advancing plans for an IND and Phase 2 trial in a fourth neurodevelopmental disorder, Prader-Willi syndrome, targeting commencement in H2 2022 with results anticipated in H2 2023.
About Pitt Hopkins syndrome
Pitt Hopkins syndrome (PTHS) is a neurodevelopmental condition affecting both males and females, caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18. The incidence of PTHS has been estimated at between 1 in 34,000 and 1 in 41,000 people.
Characteristics of PTHS are developmental delay with moderate-to-severe intellectual disability and behavioural differences, hyperventilation and/or breath-holding while awake, seizures, gastrointestinal issues, lack of speech, sleep disturbance, stereotypic hand movements and distinctive facial features.
Some individuals with PTHS are diagnosed with autism. There is currently no treatment approved for PTHS.
About Neuren
Neuren is developing two new drug therapies to treat multiple serious neurological disorders that emerge in early childhood, none of which have any approved medicines.
The lead compound, trofinetide, achieved positive results in a Phase 3 clinical trial for Rett syndrome and has also completed a Phase 2 clinical trial in Fragile X syndrome.
Both programmes have Fast Track designation from the US Food and Drug Administration (FDA). Neuren has granted an exclusive licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of trofinetide in North America, while retaining all rights outside North America.
Neuren is preparing to initiate Phase 2 trials of its second drug candidate, NNZ-2591, for each of PhelanMcDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome. Recognising the urgent unmet need, all six programs have been granted “orphan drug” designation in the United States.
Orphan drug designation provides incentives to encourage development of therapies for rare and serious diseases.
