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Ionis Pharmaceuticals (ISI) Drug Pipeline

Drug pipeline
Drug NameConditionStageStatusStudy NameTrial Start DateArticle
Obudanersen, Ion582Angelman SyndromePhase IIIRecruitingCHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)Aug 20, 2026―
OlezarsenFamilial Chylomicronemia SyndromePhase IIIRecruitingA Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric ParticipantsJul 10, 2026
Ion775Hypertriglyceridemia, Severe HypertriglyceridemiaPhase IIRecruitingA Study to Assess the Safety, Tolerability and Efficacy of ION775May 18, 2026
Ion337Dravet Syndromephase1AndPhase2RecruitingASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet SyndromeApr 08, 2026
DonidalorsenHereditary Angioedema (Hae)Phase IIIRecruitingDonidalorsen Treatment in Children With Hereditary AngioedemaDec 19, 2025
Ion582Angelman SyndromePhase IIIRecruitingREVEAL: A Phase 3 Study of Obudanersen (ION582) in Angelman SyndromeMar 22, 2025
Ion269Alzheimer Disease, Down SyndromePhase ITerminatedHero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD)Oct 31, 2024
Sham Procedure, Ion440Methyl Cpg Binding Protein 2 (Mecp2) Duplication Syndromephase1AndPhase2RecruitingATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)May 21, 2024
Ion717Prion Diseasephase1AndPhase2Active Not RecruitingPrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717Nov 21, 2023―
Ion356Pelizaeus-Merzbacher DiseasePhase IRecruitingOrbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)Nov 21, 2023

FAQ

What are the FDA phases for drug approval?
The FDA approval process for new drugs is divided into several phases: a. Preclinical testing b. Phase 1 – Safety and dosage c. Phase 2 – Effectiveness and side effects d. Phase 3 – Confirmation of effectiveness, monitoring of adverse reactions, and comparison with existing treatments e. Phase 4 – Post-marketing studies to track long-term safety and effectiveness
What is a clinical trial?
A clinical trial is a carefully designed study in which researchers test a drug, treatment, or medical device in people to evaluate its safety, effectiveness, and potential side effects. These trials are essential for determining whether a new treatment should be approved for widespread use.
What drugs does Ionis Pharmaceuticals Inc. (DE:ISI) have in its pipeline
Ionis Pharmaceuticals DE:ISI is currently developing the following drugs: Obudanersen, Ion582, Olezarsen, Ion775, Ion337, Donidalorsen, Ion582, Ion269, Sham Procedure, Ion440, Ion717, Ion356, Sapablursen, Eplontersen, Ion224, Zilganersen, Ion363, Ionis-Agt-Lrx, Ion251, Ion464, Eplontersen, Inotersen, Ionis-Fb-Lrx, Biib094, Ionis Maptrx, Tofersen. These drug candidates are in various stages of clinical development as the company works toward FDA approval.