Product Description
recently approved antisense oligonucleotide for spinal muscular atrophy (Sourced from: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5858681/)
Mechanisms of Action: mRNA Agonist
Novel Mechanism: No
Modality: Nucleic Acid
Route of Administration: Injection
FDA Designation: *
Approval Status: Approved
Approved Countries: Argentina | Australia | Austria | Belgium | Bosnia | Brazil | Canada | Chile | Colombia | Croatia | Cyprus | Czech | Denmark | Estonia | European Medicines Agency | Finland | France | Germany | Greece | Hong Kong | Hungary | Iceland | Ireland | Israel | Italy | Japan | Korea | Latvia | Lithuania | Luxembourg | Malaysia | Netherlands | New Zealand | Norway | Poland | Portugal | Romania | Russia | Saudi Arabia | Serbia | Slovakia | Slovenia | Spain | Sweden | Switzerland | Taiwan | Turkey | Ukraine | United Arab Emirates | United Kingdom | United States | Uruguay
Approved Indications: None
Known Adverse Events: None
Company: Biogen
Company Location: CAMBRIDGE MA 02142
Company CEO: Michel Vounatsos
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Argentina, Australia, Belgium, Brazil, Canada, Chile, China, Colombia, Estonia, France, Germany, Greece, Hong Kong, Hungary, Ireland, Israel, Italy, Japan, Korea, Latvia, Lebanon, Mexico, Netherlands, Poland, Qatar, Russia, Saudi Arabia, South Korea, Spain, Sweden, Taiwan, Turkey, United Kingdom, United States
Active Clinical Trial Count: 15
Recent & Upcoming Milestones
- Clinical Outcomes Reported - Biogen presented P3 Muscular Atrophy, Spinal results on 2024-10-08 for Nusinersen
- Clinical Outcomes Reported - Biogen presented P2 Muscular Atrophy, Spinal results on 2024-09-04 for Nusinersen
Highest Development Phases
Phase 3: Muscular Atrophy, Spinal
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT06555419 |
PIERRE-PK | P1 |
Recruiting |
Muscular Atrophy, Spinal |
2026-05-15 |
12% |
2025-06-03 |
Patient Enrollment|Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
2024-514239-21-00 |
295SM101 | P1 |
Recruiting |
Muscular Atrophy, Spinal |
2026-05-05 |
12% |
2025-05-02 |
Treatments |
2014-002098-12 |
NURTURE | P2 |
Completed |
Muscular Atrophy, Spinal |
2024-12-17 |
50% |
2025-05-06 |
Primary Completion Date|Study Completion Date|Treatments|Trial Status |
NCT02386553 |
NURTURE | P2 |
Completed |
Muscular Atrophy, Spinal |
2024-12-17 |
50% |
2025-01-14 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments|Trial Status |
2017-000621-12 |
2017-000621-12 | P2 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2020-12-30 |
2025-07-02 |
Primary Completion Date|Start Date|Study Completion Date|Treatments|Trial Status |
|
jRCT2051230096 |
jRCT2051230096 | P3 |
Not yet recruiting |
Muscular Atrophy, Spinal |
2030-07-31 |
|||
2023-505639-11-00 |
232SM303 | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2027-07-22 |
2025-05-02 |
Treatments |
|
NCT05067790 |
ASCEND | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2027-06-14 |
73% |
2025-05-07 |
Primary Endpoints|Treatments|Trial Status |
jRCT2031220035 |
jRCT2031220035 | P3 |
Recruiting |
Muscular Atrophy, Spinal |
2026-11-30 |
|||
2023-505637-27-00 |
232SM302 | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2026-08-15 |
2025-05-02 |
Treatments |
|
NCT04729907 |
ONWARD | P3 |
Active, not recruiting |
Muscular Atrophy, Spinal |
2026-07-31 |
39% |
2024-09-20 |
Primary Endpoints |
2019-002663-10 |
Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | P3 |
Completed |
Muscular Atrophy, Spinal |
2024-05-30 |
62% |
2025-07-09 |
Treatments |
NCT04089566 |
DEVOTE | P3 |
Completed |
Muscular Atrophy, Spinal |
2024-02-21 |
62% |
2024-07-24 |
|
NCT02594124 |
SHINE | P3 |
Completed |
Muscular Atrophy, Spinal |
2023-08-21 |
60% |
2024-02-07 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
jRCT2061200040 |
jRCT2061200040 | P3 |
Recruiting |
Muscular Atrophy, Spinal |
2023-07-31 |
Recent News Events
Date |
Type |
Title |
|---|---|---|
|
12/04/2025 |
News Article |
Royalty Pharma and Denali Therapeutics Announce $275 Million Royalty Funding Agreement |
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12/04/2025 |
News Article |
Denali Therapeutics and Royalty Pharma Announce $275 Million Royalty Funding Agreement |
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12/02/2025 |
News Article |
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12/01/2025 |
News Article |
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