Ultragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups

Core Insights - Ultragenyx Pharmaceutical Inc. has initiated the Aurora study to evaluate the efficacy and safety of GTX-102 (apazunersen) for Angelman syndrome, expanding the patient population to include younger and older patients as well as those with non-deletion AS genotypes [1][2] - The Aurora study aims to enroll approximately 60 participants aged 1 to under 65, with a focus on diverse genotypes and age groups, reflecting a commitment to inclusivity in clinical trials [2][3] - GTX-102 has received multiple designations from the FDA and EMA, indicating its potential as a breakthrough therapy for Angelman syndrome, a rare neurogenetic disorder affecting around 60,000 individuals [4][6] Company Overview - Ultragenyx is a biopharmaceutical company dedicated to developing therapies for rare and ultra-rare genetic diseases, with a portfolio aimed at addressing high unmet medical needs [8] - The company emphasizes efficient drug development processes to deliver safe and effective therapies urgently [9] Study Design and Objectives - The Aurora study features an open-label basket design, allowing for the evaluation of GTX-102 across different age groups and genotypes [2] - Participants will be divided into four cohorts, each with specific age ranges and genotypes, focusing on various primary endpoints related to cognitive and response measures [2] - The study includes a 48-week primary efficacy period, with a crossover option for the No Treatment group to receive GTX-102 after 24 weeks [2] Background on Angelman Syndrome - Angelman syndrome is characterized by cognitive and motor impairments, seizures, and requires continuous care, affecting individuals' quality of life [7] - The disorder is caused by the loss of function of the maternally inherited UBE3A gene, with no currently approved therapies available [5][7]