Invex Therapeutics Ltd. announces the Paediatric Committee (PDCO) of the European Medicines Agency (EMA) has accepted the Company's PIP for the development of PresendinTM for Idiopathic Intracranial Hypertension (IIH) in pubescent boys and girls aged less than 18 years. In the UK, overall paediatric IIH incidence is approximately 0.7 per 100,000 of population with increasing incidence linked to age and weight to 4.18 and 10.7 per 100,000 in obese boys and girls aged 12­15 years. In the European Union, sponsors must possess a compliant PIP prior to applying for marketing approval of new drugs.

A successful clinical trial in adolescents will allow Invex to file for an additional market authorisation (MAA) in Europe, thereby expanding market access in an adolescent population where the incidence of IIH continues to climb and associated healthcare costs are high. The proposed pediatric trial will be a randomised, placebo-controlled, double-blind, multi-centre trial recruiting at least 40 post-pubertal males and females with IIH who are <18 years of age (as described above) and an additional number of adult IIH patients for treatment with either PresendinTM or placebo over 24 weeks. The primary endpoint will be determined by the change in papilloedema (swelling of the optic disc) assessed by optical coherence tomography over 24 weeks.

Intracranial pressure (ICP) will not need to be measured in the agreed pediatric trial given that the positive effect of PresendinTM on ICP will have already been established in the IIH EVOLVE trial. Key secondary endpoints for the trial will include perimetric mean deviation, headache evaluation and visual acuity at 24 weeks, along with safety assessments and concomitant medication use throughout the study. The final trial design will be guided by clinical outcomes from the IIH EVOLVE Phase III clinical trial.