Ionis amends REVEAL Phase 3 for obudanersen in Angelman syndrome to two-arm design

Ionis Pharmaceuticals had two clinical trial updates posted during the week.

• The "REVEAL" study (NCT06914609), a Phase 3, randomized, double-blind, placebo-controlled trial of obudanersen in children and adults with Angelman syndrome caused by a UBE3A deletion or mutation, is listed as active not recruiting. The study initiated in 2024 with three dosing groups (40 mg obudanersen, 80 mg obudanersen, and placebo), but following additional review of data from the ongoing Phase 1/2 trial of obudanersen (HALOS), REVEAL was amended in December 2025 to a two-arm study of 80 mg obudanersen and placebo, with participants previously randomized to 40 mg transitioned to 80 mg.¹ The trial has an actual enrollment of 156 participants, and estimated primary completion is listed for August 2027¹.
• The "CHAMPION" study (NCT07782827) is a Phase 3, open-label trial testing obudanersen in participants with Angelman syndrome due to paternal uniparental disomy or imprinting defects. The primary purpose of the study is to evaluate efficacy of obudanersen in participants with AS due to uniparental disomy or imprinting defects (UPD/ID) as measured through expressive communication.² The study remains listed as recruiting, with an estimated enrollment of 30 participants across two age-based cohorts² and an estimated primary completion of May 2028.²