Ionis Pharmaceuticals Inc. ((IONS)) announced an update on their ongoing clinical study.
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Ionis Pharmaceuticals Inc. is conducting a Phase 3 study titled ‘REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome’ to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome. This genetic disorder is caused by a deletion or mutation of the UBE3A gene, and the study aims to provide a potential treatment option for affected individuals.
The intervention being tested is ION582, a drug administered via intrathecal injection. It is designed to target the genetic cause of Angelman syndrome and is being tested in various dosages alongside a placebo for comparison.
The study follows a randomized, parallel assignment model with quadruple masking, meaning that participants, care providers, investigators, and outcomes assessors are unaware of the treatment allocations. The primary purpose of the study is treatment-focused.
The study began on March 22, 2025, with the latest update submitted on October 16, 2025. These dates are crucial as they mark the progression and current status of the study, which is still recruiting participants.
The successful development of ION582 could significantly impact Ionis Pharmaceuticals’ stock performance by enhancing its portfolio and attracting investor interest. Considering the competitive landscape, advancements in treatment for rare genetic disorders like Angelman syndrome could position Ionis favorably against competitors.
The study is ongoing, with further details available on the ClinicalTrials portal.