Ultragenyx to resume Phase 1/2 trial for GTX-102 in rare genetic disorder

 

• Ultragenyx Pharmaceutical Inc. (RARE), announcing the data from the Phase 1/2 study of GTX-102 in Angelman syndrome, says the additional data, including EEG findings, support prior initial indications of activity with no new adverse events.
• In August 2019, Ultragenyx partnered with GeneTx Biotherapeutics to develop GTX-102, with an exclusive option to acquire GeneTx.
• “We better understand the serious adverse events reported with GTX-102 at higher doses and we see a way forward to redose patients and to enroll new patients into the clinical trial,” commented Scott Stromatt, M.D., Chief Medical Officer of GeneTx.
• The study is currently on hold, and with the receipt of guidance and approval from the FDA, the companies intend to resume the enrollment and dosing as soon as possible.
• Angelman syndrome, a rare neurogenetic disorder due to loss-of-function of the maternally inherited allele of the UBE3A gene, affects 1 in 12,000 to 1 in 20,000 people globally, with no approved therapies.
• GTX-102, an investigational antisense oligonucleotide designed to target and inhibit the expression of UBE3A-AS, has won the Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation by the FDA.
• https://seekingalpha.com/news/3642735-ultragenyx-is-to-resume-phase-1-2-trial-for-gtxminus-102-in-rare-genetic-disorder