GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

SARASOTA, Fla. and NOVATO, Calif., Jan. 15, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that GeneTx’s Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020.

"The FDA’s clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community,” said Paula Evans, Chief Executive Officer at GeneTx.  “Assessing an investigational therapy that aims to address the underlying cause of this devastating disorder has the potential to meaningfully alter the clinical course of patients’ lives.”

“Based on preclinical data, we believe that GTX-102 offers tremendous promise and may one day provide patients with a potentially transformative therapeutic option,” said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. “We look forward to continuing our partnership with GeneTx to address this devastating disorder for which there are currently no approved treatments.”

“The FDA cleared the IND which allows GTX-102 to enter the clinic,” said Scott Stromatt, MD, Chief Medical Officer, GeneTx.  “GeneTx’s Phase 1/2 study, named KIK-AS (Knockdown of UBE3A-antisense in Kids with AS, is expected to enroll 20 patients at multiple trial sites, beginning in the first half of 2020. The goal of this multiple dose, dose escalating, open-label study is to examine the safety, tolerability, and pharmacokinetics of GTX-102 in pediatric patients with Angelman syndrome. On February 15, 2020 we will open a website with more information on the KIK-AS study and provide a toll free number for parents and caregivers to obtain information on enrolling into the clinical study.”

About Angelman Syndrome

Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.