Date: 2016-04-07
Type of information: Initiation of the trial
phase: 2
Announcement: initiation of the trial
Company: Neuren Pharmaceuticals (Australia)
Product: trofinetide - NNZ-2566 - glycyl-L-2-methylprolyl-L-glutamic acid
Action
mechanism: peptide hormone. NNZ-2566/trofinetide is an analog of glypromate (GPE) which is derived from IGF-1 and occurs naturally in the brain. GPE affects glial cells (astrocytes and microglia) while IGF-1 itself mostly affects neurons. IGF-1 is a growth factor stimulated by growth hormone. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical both for normal development and for responding to injury and disease. Glypromate comprises the last three peptides of IGF-1 and trofinetide is Neuren’s chemically modified form of GPE that can mimic GPE’s natural function in the brain. The small modification results in the drug having an increased half-life in the circulation, better stability for easier storage and shipping, and suitability for use as an oral medication, whereas GPE itself and IGF-1 can only be administered by injection.
Disease: Rett syndrome
Therapeutic area: Rare diseases
Country: USA
Trial details:
Latest
news: * On April 7, 2016, Neuren Pharmaceuticals announced that the first subject has commenced its Phase 2 clinical trial of trofinetide in children and adolescents with Rett syndrome. Neuren’s program is receiving valuable support from rettsyndrome.org, including grant funding towards the cost of this pediatric trial. The randomized, double-blind, placebo-controlled, dose-ranging study in subjects aged 5 to 15 years is being conducted at up to 12 sites across the United States, led by clinicians experienced in Rett syndrome including the experts who led Neuren’s first trial in adolescents and adults. Neuren plans to enroll approximately 64 subjects and to complete the trial by the end of 2016.
The trial will evaluate the safety, efficacy and pharmacokinetics of three dose levels of trofinetide, 50mg/kg, 100mg/kg and 200mg/kg each taken orally twice daily. Efficacy outcome measures include the Motor Behavior Assessment, a measure that captures key clinical symptoms of Rett syndrome. The trial duration for each subject including follow-up is approximately 10 weeks.
Neuren’s previous Phase 2 clinical trial demonstrated clinical benefit from treatment with trofinetide in subjects aged 16 to 45 years at a dose level of 70mg/kg twice daily. The current trial will evaluate the safety and efficacy of trofinetide in a younger age group, at higher doses and for a longer duration of treatment, as well as confirming the optimum dose levels for the subsequent Phase 3 trial in children, adults and adolescents.
