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- Neuren Pharmaceuticals (NEU) has received guidance from the U.S. Federal Drug Administration for phase-two clinical trials of its NNZ-2591 candidate
- The company will test the drug for the treatment of three serious neurological disorders that emerge in childhood and, at present, have no approved medicine
- The FDA’s guidance allows Neuren to prepare investigational new drug (IND) applications to obtain clearance for the trials
- The treatment will be tested in 10 to 20 paediatric patients across 13 weeks to confirm the safety and tolerability of dosing
- One trial in Angelman syndrome will take place in Australia, with two others in Phelan-McDermid and Pit Hopkins syndromes to be conducted in the U.S.
- Subject to IND clearance, the studies are set to commence later this year
- Neuren Pharmaceuticals shares are down 3.08 per cent at $1.26 each
Neuren Pharmaceuticals (NEU) has received guidance from the U.S. Federal Drug Administration (FDA) Office of Neuroscience for phase-two clinical trials of its NNZ-2591 candidate in three serious neurological disorders.
The Melbourne-based company is developing drug therapies to treat Phelan-McDermid, Angelman and Pitt Hopkins syndromes — debilitating neurodevelopment disorders that emerge in childhood and have no approved medicines.
NEU says the guidance from the FDA is an important milestone, enabling the company to proceed with preparing investigational new drug (IND) applications for clearance to start the trials.
Notably, the company’s Angelman syndrome trial is set to take place in Australia, with the remaining two trials to be conducted in the U.S.
Each trial will test the treatment in 10 to 20 paediatric patients over 13 weeks, with the primary objective of confirming the safety and tolerability of dosing during this timeframe.
As a secondary objective, the studies will aim to assay efficacy by observing the extent of change in each patient from a well-characterised baseline, using a range of measures.
Overall, the Neuren is aiming to generate information that will enable the design of pivotal registration trials with optimum primary efficacy endpoints.
“Our positive dialogue with the FDA was very helpful and, with only modifications, confirmed our plans for three disorders that have such urgent, unmet need,” Neuren CEO Jon Pilcher said of the upcoming research.
“NNZ-2591 has ideal attributes — a unique mechanism of action, consistent and compelling results in the model of each disorder, a clearly identified optimum dose and proprietary manufacturing with high-purity and yield.
“The final piece of the jigsaw is to demonstrate its impact in patients, so we are eager to expedite that data from these three phase-two trials.”
Subject to IND clearance, the trials are set to commence as planned in the second half of the year, with top-line results targeted in the second half of 2022.
Neuren Pharmaceuticals shares are down 3.08 per cent at $1.26 each at 1:40 pm AEST.
