Lonafarnib
Hutchinson-Gilford Progeria Syndrome
Explore 2 assets in Hutchinson-Gilford Progeria Syndrome →Floatz Rating
B58/100
Confidence
Indicative
Sponsor
PRG Science & Technology Co., Ltd.
Modality
Small molecule
Development Phase
Phase 2
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
43High confidence
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT06775041CT.gov Study to Determine Optimal Dose and Evaluate Safety, Tolerability, and Pharmacokinetics of Progerinin in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) | — | Active Not Recruiting | — | — | — |
NCT00916747CT.gov Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria | — | Unknown | — | — | — |
NCT00879034CT.gov A Study of Zoledronic Acid, Pravastatin, and Lonafarnib for Patients With Progeria | — | Completed | — | — | — |
NCT00425607CT.gov Phase II Trial of Lonafarnib (a Farnesyltransferase Inhibitor) for Progeria | — | Completed | — | — | — |
NCT03895528CT.gov Lonafarnib for Patients With Hutchinson-Gilford Progeria Syndrome or Progeroid Laminopathy | — | Approved For Marketing | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95High confidence
Detailed axis rationale is planned and will be published soon.
Same indication · Hutchinson-Gilford Progeria Syndrome
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Lonafarnib (this asset) | PRG Science & Technology Co., Ltd. | P2 | B · 58 |
| Pravastatin | Boston Children's Hospital | P2 | B |
| Zoledronate | Boston Children's Hospital | P2 | B |
| Progerinin | PRG Science & Technology Co., Ltd. | P2 | B |
Other indications for Lonafarnib
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Metastatic Neoplasm | — | P3 | B · 57 |
| Renal Pelvis/Ureter Urothelial Carcinoma | — | P2 | CCC · 55 |
| Carcinoma Of Urethra | — | P2 | CCC · 55 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Lonafarnib in Hutchinson-Gilford Progeria Syndrome. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/lonafarnib-hutchinson-gilford-progeria-syndrome
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