Table 2.
Clinical trials targeting cancer and ribosome biogenesis
| Drugs | Diseases | Mechanism | Phase | Trial identifier |
|---|---|---|---|---|
| BMH-21 | Cancer | BMH-21, as a first-in-class small-molecule, directly inhibits transcription elongation and DNA occupancy of RNA Pol I. | N/A | N/A |
| CX-5461 | Cancer | CX-5461 (Pidnarulex), a synthetically-derived small molecule that selectively kills cancer cells through the binding and stabilization of G4 DNA structure | Phase 1 | NCT02719977 |
| CX-5461 | Advanced solid cancer | Through the binding and stabilization of G4 DNA structure | Phase 1 | NCT04890613 |
| CX-3543 (Quarfloxin) |
Advanced solid tumors, lymphoma neuroendocrine tumors, carcinoid cancer advanced solid tumors, lymphoma B-cell chronic lymphocytic leukemia |
Quarfloxin is a first-in-class small-molecule targeted cancer therapeutic derived from the validated fluoroquinolone class of drugs. Quarfloxin was rationally designed to target a G-quadruplex (QPLX) DNA structure and disrupt protein-DNA interactions essential to cancer cells. The QPLX targeted by quarfloxin forms within ribosomal DNA and the QPLX is bound by the nucleolin protein |
Phase 1 | NCT00955786 |
| Phase 2 | NCT00780663 | |||
| Phase 1 | NCT00955292 | |||
| Phase 2 | NCT00485966 | |||
| Camptothecin (Topotecan and Irinotecan) | Sarcoma | Inhibits topoisomerase I and regulates early rRNA processing | Phase 3 | NCT00354744 |
| Ellipticines | Cancer | Affects the combination of SL1 with rDNA promoters, inhibits topoisomerase II and Pol I | N/A | N/A |