With Hopeful Interim Data, Ultragenyx Bets On GeneTx, Angelman Syndrome Candidate

It remains early days but Ultragenyx Pharmaceutical Inc. likes its chances to succeed in the rare neuro-genetic disorder Angelman syndrome where Ovid Therapeutics, Inc. notably flamed out in 2020. Ultragenyx unveiled interim Phase I/II data showing safety and indications of clinical activity on 18 July for GTX-102, a candidate it is developing with GeneTx Biotherapeutics LLC, and now the company has exercised an option to acquire its collaborator.

Data from a handful of patients in UK/Canada and US cohorts of the Phase I/II study – called early but promising by multiple analysts – follow earlier findings of potential efficacy but also serious safety concerns with higher doses of GTX-102, an antisense oligonucleotide (ASO) designed to target and inhibit expression of UBE3A-AS. Ultragenyx and GeneTx agreed to partner on development of GTX-102 in August 2019, in deal that gave the Novato, CA-based firm an option to purchase its partner