Ultragenyx (RARE) Aligns With FDA on Phase III Neuro Study Plans

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, ...