Ionis finishes enrollment for Angelman syndrome study
- Ionis Pharmaceuticals (NASDAQ:IONS) completed patient enrollment in the pivotal pediatric cohort of its Phase 3 REVEAL study evaluating obudanersen.
- The finalization of this primary testing group allows the drug developer to remain on track to deliver topline trial data.
- Ionis plans to complete adult cohort enrollment by late 2026 and initiate a separate Phase 3 CHAMPION trial for alternative genotypes.
Ionis Pharmaceuticals (NASDAQ:IONS) announced it completed patient enrollment in the pivotal pediatric cohort of its global Phase 3 REVEAL clinical study evaluating its investigational RNA-targeted medicine, obudanersen.
The trial segment successfully randomized 136 participants between the ages of 2 and 18 who have a genetically confirmed diagnosis of Angelman syndrome.
"Completion of enrollment in the pivotal cohort of REVEAL marks an important step towards a potential disease modifying treatment for people living with this serious and complex neurological condition, for which there are no approved medicines," said Ionis Chief Development Officer Holly Kordasiewicz.
The clinical protocols utilize the clinician-administered Bayley Scales for Infant and Toddler Development-4 assessment to evaluate the primary endpoint of structural improvement in expressive communication.
Following the announcement, Ionis Pharmaceuticals' share price was up at $81.80.
The corporation expects to complete enrollment for its companion adult cohort during the third quarter of 2026 before releasing overarching topline study data in the second half of 2027.
The California-based biotechnology firm also intends to initiate its separate Phase 3 CHAMPION study before the end of 2026 to evaluate the drug candidate against rarer genetic variations of the disorder.
