Product Description
Pevonedistat is a NEDD8-activating enzyme (NAE) inhibitor that leads to cancer cell death by disrupting protein homeostasis. The Phase 3 PANTHER study (Pevonedistat-3001) did not achieve pre-defined statistical significance for the primary endpoint of event-free survival (EFS) and the data results are currently being evaluated. The safety profile was consistent with previously reported data for this combination. Pevonedistat is an investigational drug for which safety and efficacy have not been established. (Sourced from: https://www.takeda.com/newsroom/newsreleases/2021/takeda-provides-update-on-phase-3-panther-pevonedistat-3001-trial/)
Mechanisms of Action: NEDD8 Inhibitor
Novel Mechanism: Yes
Modality: Small Molecule
Route of Administration: Intravenous
FDA Designation: Breakthrough Therapy - Myelodysplastic Syndrome *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Takeda
Company Location: TOKYO M0 103-8668
Company CEO:
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Canada, France, Germany, Italy, Poland, Spain, United States
Active Clinical Trial Count: 6
Recent & Upcoming Milestones
Highest Development Phases
Phase 3: Acute Myeloid Leukemia
Phase 2: Myelodysplastic Syndrome
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|
| Pevonedistat-2002 | P2 |
Active, not recruiting |
Acute Myeloid Leukemia |
2025-06-27 |
2025-05-02 |
Treatments |
|
| SHAPE | P2 |
Completed |
Acute Myeloid Leukemia|Myelodysplastic Syndrome |
2023-01-31 |
28% |
2023-02-16 |
Primary Endpoints |
| SHAPE | P2 |
Completed |
Myelodysplastic Syndrome|Acute Myeloid Leukemia |
2023-01-31 |
2025-06-14 |
Treatments |
|
| PEVENAZA | P2 |
Active, not recruiting |
Acute Myeloid Leukemia |
2022-09-06 |
12% |
2024-06-06 |
Primary Endpoints|Treatments |
| 2019-001323-12 | P3 |
Completed |
Acute Myeloid Leukemia |
2024-11-05 |
2025-06-17 |
Treatments |
|
| PEVOLAM | P3 |
Active, not recruiting |
Acute Myeloid Leukemia |
2023-06-30 |
17% |
2025-03-18 |