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Ionis Pharmaceuticals (IONS)
NASDAQ:IONS
Ion582
Angelman Syndrome
Phase III
Recruiting
REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome
Mar 22, 2025
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Ion269
Alzheimer Disease, Down Syndrome
Phase I
Active Not Recruiting
Hero: 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
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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
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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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Ion356
Pelizaeus-Merzbacher Disease
Phase I
Recruiting
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Nov 21, 2023
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Olezarsen
Severe Hypertriglyceridemia
Phase III
Recruiting
CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
Dec 25, 2022
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Olezarsen
Cardiovascular Diseases, Atherosclerosis, Hypertriglyceridemia
Phase III
Completed
A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia
Nov 02, 2022
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Olezarsen
Severe Hypertriglyceridemia
Phase III
Completed
A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia
Sep 20, 2022
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Donidalorsen
Hereditary Angioedema
Phase III
Active Not Recruiting
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
May 23, 2022
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Olezarsen
Atherosclerotic Cardiovascular Disease, Hypertriglyceridemia, Severe Hypertriglyceridemia
Phase II
Completed
A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia
Apr 26, 2022
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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 (IONS) have in its pipeline
IONS is currently developing the following drugs: Ion582, Ion269, Sham Procedure, Ion440. These drug candidates are in various stages of clinical development as the company works toward FDA approval.