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AZD0120
AZD0120 is a selective inhibitor of fibroblast growth factor receptor 1 (FGFR1) that blocks aberrant signaling in cancer cells dependent on FGFR1 activation.
AZD0120 is a selective inhibitor of fibroblast growth factor receptor 1 (FGFR1) that blocks aberrant signaling in cancer cells dependent on FGFR1 activation. Used for FGFR1-amplified or FGFR1-altered solid tumors.
At a glance
| Generic name | AZD0120 |
|---|---|
| Also known as | GC012F |
| Sponsor | AstraZeneca |
| Drug class | FGFR1 inhibitor |
| Target | FGFR1 (Fibroblast Growth Factor Receptor 1) |
| Modality | Biologic |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
FGFR1 is frequently dysregulated in certain cancers through gene amplification or translocation, driving proliferation and survival. AZD0120 selectively targets FGFR1 to inhibit downstream signaling pathways, inducing cell cycle arrest and apoptosis in FGFR1-dependent tumors. This approach is designed to provide clinical benefit in cancers with FGFR1 alterations while minimizing off-target effects.
Approved indications
- FGFR1-amplified or FGFR1-altered solid tumors
Common side effects
- Hyperphosphatemia
- Diarrhea
- Nausea
- Fatigue
- Retinal toxicity
Key clinical trials
- Study of GC012F, CAR-T Therapy Targeting CD19 and BCMA in Chinese Participants With Relapsed or Refractory AL Amyloidosis (PHASE1)
- A Phase 1b/2 Study of CAR T Cell Therapy Targeting CD19 and BCMA in Participants With Relapsed or Refractory AL Amyloidosis. (PHASE1, PHASE2)
- A Study Comparing AZD0120, a Dual-targeted CAR-T Against B-cell Maturation Antigen (BCMA) and CD19, Versus Standard Regimens in Participants With Relapsed Refractory Multiple Myeloma (DURGA-4) (PHASE3)
- A Phase I, Open-label, Multicenter Study to Evaluate the Safety, Tolerability, Cellular Kinetics, Immunogenicity, Pharmacodynamics, and Preliminary Efficacy of AZD0120 in Participants With Multiple Myeloma (DURGA-2) (PHASE1)
- A Study to Investigate the Safety, Tolerability, and Efficacy of AZD0120 in Adults With Refractory SLE (PHASE1, PHASE2)
- A Study of AZD0120 in Autoimmune Diseases (PHASE1)
- An Open-label Study of AZD0120 in Adults With Multiple Sclerosis (PHASE1)
- CAR-T Therapy Targeting CD19 and BCMA in Highly Sensitized Kidney Transplant Participants (EARLY_PHASE1)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |