Obudanersen, Ion582
Angelman Syndrome
Phase III
Recruiting
CHAMPION: A Study to Evaluate the Efficacy and Safety of Obudanersen (ION582) in Children and Adults With Angelman Syndrome (AS)
Aug 20, 2026
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Sham Procedure, Ion440
Methyl Cpg Binding Protein 2 (Mecp2) Duplication Syndrome
Phase I/II
Recruiting
ATTUNE: 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
Ion717
Prion Disease
Phase I/II
Active Not Recruiting
PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Nov 21, 2023
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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
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 (ISI) have in its pipeline
ISI is currently developing the following drugs: Obudanersen, Ion582, Olezarsen, Ion775. These drug candidates are in various stages of clinical development as the company works toward FDA approval.