Neuren Pharmaceuticals (ASX: NEU) has successfully completed manufacturing of the drug substance for Phase 2 trials of NNZ-2591 on schedule.
CEO, Jon Pilcher, said Neuren is preparing to submit Investigational New Drug (IND) applications to the US Food and Drug Administration for trials in children with Phelan- McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome.
We have successfully developed a proprietary process for large scale manufacturing with exceptional purity and high yield,” Mr Pilcher said.
“This is a key part of the strong foundations we have built for NNZ-2591, which can now be leveraged across multiple valuable indications.
“As well as supplying the upcoming trials in Phelan-McDermid, Angelman and Pitt Hopkins, the campaign has produced enough drug substance at no extra cost to supply a Phase 2 trial in Prader-Willi syndrome.”
Mr Pilcher said four of the company’s milestones for 2021 have already been achieved. Five important milestones remain, leading up to the Rett syndrome Phase 3 trial topline results expected in Q4 2021.
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 drug compound, trofinetide, is currently in a Phase 3 clinical trial for Rett syndrome and has completed a Phase 2 clinical trial in Fragile X syndrome. Both programs have been granted Fast Track designation by 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 plans to initiate Phase 2 trials of its second drug candidate, NNZ-2591, for each of Phelan- McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome in 2021. Neuren is also preparing for a Phase 2 trial in Prader-Willi syndrome.
Because of the urgent unmet need, five programmes have been granted “orphan drug” designation in both the United States and the European Union, a designation that provides incentives to encourage therapies for rare and serious diseases.