News about trials with antisense oligonucleotides (ASO) in people with Angelman syndrome
We would like to provide a brief update on the three Phase 3 trials involving an ASO from the pharmaceutical industry.
Ultragenyx investigated its drug GTX-102 (apazunersen) in the Aspire study. An analysis of the primary outcome measures was conducted very recently; these showed no significant improvement. Consequently, the study yielded negative results and is being discontinued.
At ENCORE, we had decided at an earlier stage not to participate in this study.
Ionis is currently conducting the Reveal and Champion studies on ION582 (obudanersen); all participants have now started, and we eagerly await the results once they have completed their follow-up assessments.
ENCORE would have liked to participate in this study, but the manufacturer's submission to the ethics committee (CCMO) in the Netherlands was rejected. Approval was granted in other countries.
Oak Hill Bio is in the start-up phase of the Beacon study on Rugonersen; this study has been submitted for ethical approval. We expect to be able to participate in it through ENCORE.