- Neuren Pharmaceuticals (NEU) opens enrolment for phase two neurological clinical trials of its drug candidate NNZ-2591
- The study is for children with Phelan-McDermid syndrome (PMS) and Pitt Hopkins syndrome (PTHS), with the first trials scheduled to take place at Rush University Medical Centre in Chicago
- The open label, phase two trials will each enrol a single group of up to 20 children to examine safety, tolerability and efficacy of the treatment over 13 weeks
- Top line results from each trial are expected in the first half of 2023
- Neuren Pharmaceuticals are down 0.18 per cent on the market, to trade at $5.44 per share as of 10:49 pm AEST
Neuren Pharmaceuticals (NEU) has opened enrolment for phase two neurological clinical trials of its drug candidate NNZ-2591.
The studies are for children with Phelan-McDermid syndrome (PMS) and Pitt Hopkins syndrome (PTHS), with first trials scheduled to take place at Rush University Medical Centre in Chicago.
PMS and PTHS are neurological disorders that emerge in early childhood and impact nearly every aspect of life, with no approved medicines to treat the symptoms.
The open label, phase two trials will each enrol a single group of up to 20 children to examine safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment.
Each patient will receive NNZ-2591 as an oral liquid dose twice a day, subject to safety and tolerability. The treatment period will be preceded by four weeks of observation to examine the baseline characteristics prior to treatment, followed by a final assessment two weeks after end of treatment.
“We are very excited to be working with the Phelan-McDermid and Pitt Hopkins communities in the United States to undertake these ground breaking clinical trials,” Neuren CEO Jon Pilcher said.
“These seriously debilitating conditions have no approved medicines and we are eager to assess the potential impact of NNZ-2591, having observed highly encouraging effects in mouse models of each syndrome.”
Top line results from each trial are expected in the first half of 2023.
Meanwhile, Neuren also recently launched a phase two trial of NNZ-2591 in Angelman syndrome (AS). In parallel with the trial, Neuren is executing foundational work to prepare for phase three development across multiple indications.
Neuren Pharmaceuticals were down 0.18 per cent on the market, to trade at $5.44 per share as of 10:49 pm AEST.