Headquartered in Melbourne, Australia, Neurizon's lead candidate, NUZ-001, is an oral therapy targeting amyotrophic lateral sclerosis (ALS) by inhibiting TDP-43 protein aggregation, a key pathological feature of the disease. Preclinical studies have demonstrated that NUZ-001 and its active metabolite significantly reduce TDP-43 aggregation and improve motor neuron function. ​

In November 2024, the European Medicines Agency issued a positive opinion for Orphan Medicinal Product Designation for NUZ-001, offering regulatory incentives and 10 years of market exclusivity upon approval. Neurizon has also submitted an Investigational New Drug application to the U.S. FDA to support the inclusion of NUZ-001 in the HEALEY ALS Platform Trial, with patient enrollment anticipated in the first half of 2025. ​