Neuren Pharmaceuticals (ASX: NEU) is a biopharmaceutical company developing new therapies for debilitating neurodevelopmental disorders that are characterised by impaired connections and signalling between brain cells.
Neuren’s strategy emphasises product development opportunities with five key attributes: solid scientific rationale, significant unmet medical need, compelling market opportunity, favourable regulatory treatment with a clear path to approval, and potential for development for additional conditions.
Neuren's lead product trofinetide (also known as NNZ-2566) is licensed to ACADIA Pharmaceuticals for North America, with Neuren retaining all rights outside North America. Trofinetide has completed Phase 2 development for Rett syndrome, and is in Phase 2 development for Fragile X syndrome. Neuren has conducted double-blind placebo-controlled Phase 2 trials in each disorder, which demonstrated clinical benefit from treatment with trofinetide. Neuren's second drug candidate NNZ-2591 has shown positive effects in models of a number of disorders, including Phelan-McDermid syndrome. Neuren is progressing the non-clinical studies required prior to commencing clinical trials.
Currently there are no drugs approved for these conditions and there are few drugs in clinical development. 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. Trofinetide provides an opportunity potentially to be the first approved therapy for one or more of these important indications. Rett syndrome and Fragile X syndrome patients are cared for in specialty clinics. As a consequence, from a commercial point of view they are attractive, because a small number of readily identifiable physicians will represent the large majority of prescribers in each of these conditions.
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 US Food and Drug Administration (FDA) has granted Fast Track designation for trofinetide in Rett syndrome, Fragile X syndrome and moderate to severe TBI. Fast Track designation provides for early and frequent communication with the FDA, ensuring that questions and issues are resolved quickly to minimise any potential impact on the progress of development. The FDA and the European Medicines Agency (EMA) have both granted orphan drug designation for trofinetide in Fragile X syndrome and in Rett 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.
Triofinetide 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.