Australian biotechnology company Neuren Pharmaceuticals (ASX:NEU) has reported a phase one trial of its twice-daily oral investigative therapy NNZ-2591 found it was safe and well-tolerated for seven days.
The company said the data will form part of its planned Investigational New Drug (IND) application to the US FDA in preparation for phase two trials in Phelan McDermid, Angelman and Pitt Hopkins syndromes.
Neuren CEO Jon Pilcher said, “This trial was the first human dosing for NNZ-2591 and we are very pleased with the outcome. Twice daily oral dosing for seven days was safe and well-tolerated at doses we expect to be within the effective therapeutic range, which gives us confidence for dosing patients in our planned Phase 2 trials.”
The trial was conducted at commercial clinical trial facilities in Perth and Sydney.
The company said the primary objective was to evaluate safety and tolerability with a secondary objective to evaluate pharmacokinetic (PK) parameters.
“Two double-blind placebo-controlled cohorts of eight healthy adult volunteers were dosed orally twice per day for seven days,” said the company.
“In each cohort, six subjects received NNZ-2591 and two subjects received placebo. Each cohort was titrated up to the target dose, with the target dose in the second cohort double the target dose in the first cohort. These two cohorts were preceded by preliminary testing of single oral doses of NNZ2591, which enabled modelling of potential multiple dosing regimens.
“Adverse Events (AEs) were recorded and blood samples were analysed for safety parameters. Cardiac tests and neurological examinations were also carried out. Blood samples were collected for the PK analyses, which are currently ongoing.
“No Serious Adverse Events (SAEs) were reported. All reported AEs were mild or moderate and resolved during the trial. There were no clinically significant findings from safety laboratory tests, vital signs, or cardiac tests.”