Ionis Pharmaceuticals’ Orbit Study: A Promising Step for Rare Disease Treatment

Ionis Pharmaceuticals Inc. ((IONS)) announced an update on their ongoing clinical study.

Study Overview: The Orbit Study, officially titled ‘A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease,’ aims to assess the safety and tolerability of the drug ION356. This study is significant as it targets Pelizaeus-Merzbacher Disease (PMD), a rare genetic disorder affecting the central nervous system.

Intervention/Treatment: The intervention being tested is ION356, a drug administered via intrathecal injection. The purpose of ION356 is to evaluate its safety and pharmacological effects in treating PMD.

Study Design: This is an interventional study with a randomized allocation and a sequential intervention model. There is no masking involved, meaning all participants and researchers know which treatment is being administered. The primary purpose of the study is treatment-focused.

Study Timeline: The study began on November 21, 2023, and the most recent update was submitted on April 29, 2025. These dates are crucial for tracking the study’s progress and ensuring transparency in its development.

Market Implications: The ongoing recruitment and updates in this study could positively impact Ionis Pharmaceuticals Inc.’s stock performance, as successful results may enhance investor confidence. The study’s focus on a rare disease also positions Ionis as a leader in niche pharmaceutical markets, potentially setting it apart from competitors.

The study is currently ongoing, with further details available on the ClinicalTrials portal.