Product Description
Elamipretide, is a peptide compound that readily penetrates cell membranes, and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin. (Sourced from: https://www.stealthbt.com/programs-pipeline/)
Mechanisms of Action: MPTP Inhibitor,Apoptosis Inhibitor
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: Intravenous,Subcutaneous
FDA Designation: Orphan Drug - Barth SyndromeOrphan Drug - Friedreich Ataxia|AtaxiaOrphan Drug - Mitochondrial MyopathiesOrphan Drug - Muscular Dystrophies|Muscular Dystrophy, DuchennePriority Review - Barth Syndrome *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Stealth Biotherapeutics
Company Location: GRAND CAYMAN E9 KY1-9005
Company CEO:
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Czech Republic, Germany, Hungary, Italy, Netherlands, New Zealand, Norway, Spain, United Kingdom, United States
Active Clinical Trial Count: 5
Recent & Upcoming Milestones
Highest Development Phases
Phase 3: Deficiency Diseases|Macular Degeneration|Mitochondrial Diseases|Mitochondrial Myopathies
Phase 2: Cerebellar Ataxia|Friedreich Ataxia
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|
| ELViS-FA | P2 |
Completed |
Cerebellar Ataxia|Friedreich Ataxia |
2024-06-25 |
61% |
2024-08-16 |
Primary Endpoints |
| ReNEW | P3 |
Recruiting |
Macular Degeneration |
2026-08-01 |
34% |
2024-08-17 |
Primary Endpoints|Treatments |
| NuPower | P3 |
Active, not recruiting |
Deficiency Diseases|Mitochondrial Diseases|Mitochondrial Myopathies |
2024-09-01 |
13% |
2023-10-15 |
Patient Enrollment|Primary Endpoints|Treatments|Trial Status |
| SPIAM-301 | P3 |
Recruiting |
Macular Degeneration |
2027-12-28 |
2025-05-02 |
Treatments |
|
| 2021-003907-16 | P3 |
Completed |
Unknown |
2024-09-30 |
2025-06-09 |
Treatments |