Neuren Pharmaceuticals announced it has completed its Phase 2 clinical trial of trofinetide in pediatric Rett syndrome. Neuren remains on schedule to receive top-line results from the trial in the second half of March 2017 and soon thereafter intends to engage potential commercial partners regarding the remaining development and commercialization of trofinetide in the major markets. The randomized, double-blind, placebo-controlled Phase 2 clinical trial for girls aged 5 to 15 years with Rett syndrome has been conducted at 12 sites in the United States, led by clinicians experienced in the diagnosis and treatment of Rett syndrome. Neuren received grant funding from Rettsyndrome.org, which contributed towards the cost of the trial. The trial comprised a demanding schedule of visits and procedures during 11 weeks from screening to follow-up. 82 subjects commenced the trial and only one subject withdrew prior to completion. 62 subjects were randomized into one of four treatment groups: 50mg/kg, 100mg/kg, 200mg/kg, or placebo. A further 20 subjects were randomized into one of two treatment groups: 200mg/kg, or placebo. The primary endpoint for the trial in this younger population is the safety and tolerability of trofinetide compared with placebo. In addition, a number of outcome measures have been included in the study design in order to provide meaningful insights into the efficacy of trofinetide in younger subjects. The efficacy analysis will prioritize 5 syndrome-specific measures, the first 3 of which were used in Neuren's prior Phase 2 trial in subjects aged 16 to 45 years with Rett syndrome: The Motor Behavior Assessment, in which the clinician rates the subject's current level of function; The Caregiver Top 3 Concerns visual analog scale, in which the subject's caregiver assesses the severity of concerns identified for each subject on an individual basis; The Clinical Global Impression of Improvement (CGI-I), in which the clinician rates how much the subject's overall illness has improved or worsened, relative to baseline; The Domain Specific Concerns Visual Analog Scale, in which the clinician assesses the severity of concerns identified for each subject on an individual basis. The analysis will examine the mean changes for each treatment group, as well as the proportion of subjects in each treatment group that showed improvements. The clinical importance of the observed changes will be evaluated. Results from two non-clinical chronic toxicity studies of trofinetide will be required prior to initiating extended dosing in a Phase 3 trial and submitting New Drug Applications. The first toxicity study is nearing completion and the second toxicity study is scheduled to commence in mid-2017, concluding in the first half of 2018. Plans are well advanced that would enable manufacturing to commence in the second half of 2017 in preparation for supplying a pivotal Phase 3 trial in Rett syndrome.