Ultragenyx announces end-of-Phase 2 meeting with FDA for GTX-102

Ultragenyx Pharmaceutical announced the successful completion of an end-of-Phase 2 EoP2 meeting with the U.S. Food and Drug Administration FDA , supporting its Phase 3 study plans for GTX-102, an antisense oligonucleotide for Angelman syndrome.”FDA alignment on our Phase 3 study design for GTX-102 allows for rapid initiation of a global double-blind sham-controlled pivotal study by the end of this year,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “In addition to this pivotal study in patients with a full UBE3A gene deletion, we are working to initiate a study to evaluate GTX-102 in patients with other mutations. This will allow for the potential treatment of more children and adults impacted by this devastating disease.” Phase 3 design and next steps ..The EoP2 meeting focused on discussion of the Company’s interim Phase 1/2 data and resulted in alignment with the FDA on the Phase 3 study design and endpoints. The pivotal Phase 3, will be a global, randomized, double-blind, sham-controlled trial and will include a 48-week primary efficacy analysis period enrolling approximately 120 patients with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. The primary endpoint will be improvement in cognition assessed by Bayley-4 cognitive raw score. Control patients completing the study will be eligible to roll over onto treatment after the double-blind period is over.