GTX-102 Phase 3 Results: What We Know, What We’re Still Learning, and Next Steps
FAST’s CSO, Allyson Berent, addresses the Aspire trial results in a candid letter, exploring what the data mean, what questions remain, and how the community will move forward together.
Ultragenyx Announces Phase 3 Aspire results in Angelman Syndrome
The Phase 3 ASPIRE study for GTX-102 (apazunersen) did not meet its primary or key secondary endpoints. FAST expresses gratitude to participating families and awaits further details from Ultragenyx.
Ionis completes enrollment in pivotal cohort of the Phase 3 REVEAL study evaluating obudanersen in Angelman syndrome
Ionis completes enrollment for Cohort 1 of the pivotal Phase 3 REVEAL study evaluating obudanersen for Angelman syndrome. Cohort 2 screening continues for ages 18–50.
Update on Rugonersen Development and Planned Phase 3 Study (BEACON)
Oak Hill Bio shared an update on rugonersen’s development, preparing to launch its Phase 3 study in mid-2026. Eligibility details are now available on clinicaltrials.gov.
An Update on the ION582 clinical development program including HALOS, REVEAL, and CHAMPION Trials
Ionis shares an update on ION582 development, covering progress across the HALOS, REVEAL, and newly announced CHAMPION trials expanding research into additional AS genotypes.
Ultragenyx Completes Enrollment of Phase 3 Aspire Study Evaluating GTX-102 for the Treatment of Angelman Syndrome
Ultragenyx announces the Phase 3 ASPIRE study for apazunersen (GTX-102) is fully enrolled with 129 participants, reaching this milestone in just seven months.
First patient dosed in pivotal Phase 3 REVEAL clinical study of ION582 in Angelman syndrome
Ionis Pharmaceuticals has dosed the first participant in its global Phase 3 REVEAL trial, evaluating the safety and efficacy of ION582 as a potential treatment for Angelman syndrome.
Oak Hill Bio to continue the development of rugonersen, an investigational ASO previously owned by Roche.
Oak Hill Bio has entered into an exclusive global licensing agreement with Roche to acquire and advance rugonersen, a Phase 3-ready investigational ASO treatment for Angelman syndrome.
IONIS REVEAL Phase 3 Clinical Trial Update
The next chapter for ION582 is here: Ionis’ Phase 3 REVEAL trial is now listed, marking an important step toward a potential treatment for Angelman syndrome. Join FAST, ASF, and Ionis to learn what comes next.
Ultragenyx Announces First Patient(s) Dosed in Pivotal Phase 3 Aspire Study Evaluating GTX-102 in Angelman Syndrome
Ultragenyx announces the first patients dosed in its pivotal Phase 3 ASPIRE study evaluating GTX-102 for Angelman syndrome, a major step toward potential treatment.