Neuren Pharmaceuticals (ASX: NEU) is developing new therapies for highly debilitating neurodevelopmental disorders that emerge in early childhood and are characterised by impaired connections and signalling between brain cells. Neuren's deep expertise in neurodevelopmental disorder therapy has been accumulated over years of clinical development.
Neuren's lead product trofinetide is licensed to Acadia Pharmaceuticals for North America, with Neuren retaining all rights outside North America and full access to all data generated by Acadia. On 10 March 2023, the US Food and Drug Administration (FDA) approved DAYBUE™ (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. DAYBUE is the first and only drug approved for the treatment of Rett syndrome. Neuren previously conducted double-blind placebo-controlled Phase 2 trials in Rett syndrome and Fragile X syndrome, which demonstrated clinical benefit from treatment with trofinetide.
Neuren's second product NNZ-2591 has shown positive effects in mouse models of Phelan-McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome. Neuren has completed a Phase 1 clinical trial and is conducting Phase 2 trials in Phelan-McDermid syndrome, Angelman syndrome, and Pitt Hopkins syndrome. Neuren is also planning a Phase 2 trial in Prader-Willi syndrome. The target number of patients across these four disorders is estimated as five times the number of Rett syndrome patients.
Currently there are no drugs approved for these conditions, apart from growth hormone to treat some aspects of Prader-Willi syndrome. Some drugs that are approved for other indications are also used to treat selected symptoms, but none are more than modestly effective and none are disease-modifying.
Because these are serious medical conditions with unmet need, drugs being developed to treat them may qualify for favourable regulatory pathways intended to expedite the development and approval of therapeutically important drugs. The FDA and the European Medicines Agency (EMA) have both granted orphan drug designation for trofinetide in Rett and Fragile X syndromes and for NNZ-2591 in each of Phelan-McDermid, Angelman and Pitt Hopkins syndromes. The FDA has also granted orphan drug designation for Prader-Willi syndrome. Orphan drug designation is a special status that the regulatory authorities may grant to a drug to treat a rare disease or condition. Orphan drug designation qualifies the sponsor of the drug for seven years of marketing exclusivity in the United States and ten years in the Europe Union following approval, as well as other development incentives.
Trofinetide and NNZ-2591 are covered by issued patents, with additional patent applications pending. All patents are owned by Neuren and no royalties are payable to third parties.