TABLE 1.
List of antisense oligonucleotides (ASOs) in clinical trials
| Development product name | Target | Application | Stage | Development company/University | NCT# |
|---|---|---|---|---|---|
| Ongoing clinical trials | |||||
| AZD4785/IONIS‐KRAS‐2.5Rx | KRAS | Advanced solid tumors | Phase I | AstraZeneca | NCT03101839 |
| AZD5312/IONIS‐AR‐2.5RX/ARRx | Androgen receptor (AR) | Cancer – prostatic | Phase I/II | University of Michigan Rogel Cancer Center | NCT03300505 NCT02144051 |
| BP1001/Prexigebersen | GRB‐2 | Cancer | Phase I/II | Bio‐Path Holdings | NCT04196257 |
| NCT02923986 | |||||
| NCT02781883 | |||||
| NCT01159028 | |||||
| BP1002 | Bcl‐2 | Advanced lymphoid malignancy | Phase I | Bio‐Path Holdings | NCT04072458 |
| EZN‐2968/Anti‐HIF‐1a/LNA AS ODN | HIF‐1a | Solid tumor | Phase I | National Cancer Institute (NCI) | NCT01120288 |
| NCT00466583 | |||||
| NCT02564614 | |||||
| EZN‐4176 | AR exon 4 | Prostatic neoplasm | Phase I | Enzon Pharmaceuticals | NCT01337518 |
| G4460/C‐MYB asODN | C‐Myb | Hematological malignancy | Phase II | Abramson Cancer Center of the University of Pennsylvania | NCT00002592 |
| GRN163L/Imetelstat® | RNA component of telomerase | Cancer | Phase II/III | Geron Corporation | NCT02598661 |
| GTI‐2040 | R2 component of R2 | Cancer | Phase II | Aptose Biosciences | NCT00565058 |
| NCT00068588 | |||||
| NCT00087165 | |||||
| IGV‐001/IGF‐1R/AS ODN | IGF‐1R | Glioblastoma | Phase I Phase II | David Andrews, Thomas Jefferson University | NCT02507583 |
| NCT01550523 NCT04485949 | |||||
| IONIS‐STAT3RX/AZD9150/Danvatirsen | STAT3 | Cancer | Phase I/II | Ionis Pharmaceuticals | NCT01563302 NCT02417753 |
| ISIS 5132/CGP69846A | C‐Raf‐1 | Ovarian/breast cancer | Phase II | Eastern Cooperative Oncology Group | NCT00003236 |
| NCT00003892 | |||||
| LErafAON‐ETU | C‐Raf‐1 | Cancer | Phase I | INSYS Therapeutics | NCT00100672 |
| LY900003/ISIS 3521 | PKC‐α | Cancer | Phase III | Ionis Pharmaceuticals | NCT00017407 |
| NCT00034268 | |||||
| MRG‐106/Cobomarsen | miR‐155 | Cutaneous T‐cell lymphoma/mycosis fungoides | Phase I/II | miRagen Therapeutics | NCT02580552 |
| NCT03713320 | |||||
| NCT03837457 | |||||
| OGX‐011/Custirsen | Clusterin | Cancer | Phase III | Achieve Life Sciences | NCT01188187 |
| NCT01578655 | |||||
| OGX‐427/Apatorsen | HSP27 | Cancer | Phase II | British Columbia Cancer Agency | NCT01120470 |
| NCT02423590 | |||||
| NCT01829113 NCT01780545 | |||||
| NCT01454089 | |||||
| RO7070179 | HIF‐1a | Hepatocellular carcinoma | Phase I | Hoffmann‐La Roche | NCT02564614 |
| SPC2996 | Bcl‐2 | Chronic lymphocytic leukemia | Phase I/II | Santaris Pharma A/S | NCT00285103 |
| Discontinued and terminated clinical trials | |||||
| AEG35156 | XIAP | Cancer | Phase II | Aegera Therapeutics | NCT00363974 |
| Multiple studies terminated | NCT00882869 NCT00558545 NCT00557596 | ||||
| EL625/Cenersen/Aezeaa | P53 | Myelodysplastic syndromes/Leukemia/Lymphoma | Phase II, Terminated | Eleos | NCT00074737 |
| G3139/Oblimersen/Genasenseb | Bcl‐2 | Cancer/Waldenstroms Macroglobulinemia | Phase II/III, Terminated | M.D. Anderson Cancer Center | NCT00030641 NCT00017602 NCT00016263 NCT00024440 NCT01200342 |
| NCT00543205 | |||||
| VEGF‐AS/Veglinb | VEGF | Mesothelioma | Phase I/II, Terminated | University of Southern California | NCT00668499 |
Note: Termination reasons are (a) lack of funding and (b) withdrawn from business.