Product Description
ION363 is an investigational antisense medicine designed to reduce the production of the Fused in Sarcoma (FUS) protein.
Mechanisms of Action: FUS Reducer
Novel Mechanism: Yes
Modality: Nucleic Acid
Route of Administration: Injection
FDA Designation: *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Otsuka
Company Location:
Company CEO:
Additional Commercial Interests: Ionis
Clinical Description
Countries in Clinic: Belgium, Brazil, Canada, Germany, Ireland, Italy, Japan, Korea, Netherlands, Poland, South Korea, Spain, Sweden, Switzerland, Taiwan, United Kingdom, United States, Unknown Location
Active Clinical Trial Count: 3
Recent & Upcoming Milestones
Highest Development Phases
Phase 3: Amyotrophic Lateral Sclerosis|Sarcoma
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT04768972 |
FUS-ALS | P3 |
Active, not recruiting |
Amyotrophic Lateral Sclerosis|Sarcoma |
2026-06-01 |
38% |
2025-08-13 |
|
2024-512163-31-00 |
ION363-CS1 | P3 |
Active, not recruiting |
Amyotrophic Lateral Sclerosis|Sarcoma |
2028-10-02 |
2025-05-02 |
Treatments |
|
jRCT2031240101 |
jRCT2031240101 | P3 |
Not yet recruiting |
Amyotrophic Lateral Sclerosis |
2028-03-31 |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
01/12/2026 |
News Article |
Ionis well-positioned for continued momentum and substantial value creation in 2026 with two new independent launches and several pivotal data readouts |
|
10/29/2025 |
News Article |
Ionis reports third quarter 2025 financial results and highlights progress on key programs |
|
04/30/2025 |
News Article |
Ionis reports first quarter 2025 financial results |
|
02/19/2025 |
News Article |
Ionis reports fourth quarter and full year 2024 financial results |