Ultragenyx Pharmaceutical (RARE.US) announced the latest data obtained in a phase 1/2 clinical trial of GTX-102, an investigational therapy used to treat Angelman syndrome (Angelman syndrome).
The Zhitong Finance App learned that Ultragenyx Pharmaceutical (RARE.US) announced the latest data obtained in a phase 1/2 clinical trial of GTX-102, its ongoing treatment for Angelman syndrome (Angelman syndrome). Data analysis showed that patients in extended cohort A and B receiving GTX-102 treatment showed rapid and clinically significant functional improvements in multiple fields on day 170. Furthermore, some patients in the dose escalation cohort showed long-term and ongoing clinical benefits at day 758, far exceeding natural historical data. Based on this data, the company plans to launch a critical phase 3 clinical trial.
According to reports, Angel syndrome is a neurological developmental disorder caused by loss or mutation of the UBE3A gene. The main characteristics of the disease include developmental delay, intellectual disability, and speech and balance disorders. In 2019, UltraGenyx and GeneTX reached a partnership to jointly conduct research on GTX-102, an antisense oligonucleotide therapy for this disease. GTX-102 can promote the expression of the parent UBE3A allele in neuronal cells and produce key protein products missing in patients. In 2022, UltraGenyx announced the completion of the acquisition of GeneTX with an advance payment amount of $75 million, and GeneTX is also eligible for additional milestone payments.