Ultragenyx Pharmaceutical (RARE) announced that it has reached alignment with the FDA regarding the phase III study design and endpoints for GTX-102, an antisense oligonucleotide for Angelman syndrome, following the completion of a successful end-of-phase II meeting with the regulatory body. The company expects to initiate the same by the end of 2024. Per RARE, the pivotal phase III sham-controlled study will evaluate the safety and primary efficacy of GTX102 in approximately 120 Angelman syndrome patients, ...