Table 3.
Therapeutic name
|
Target gene/protein
|
Route of administration
|
Phase/status
|
Clinical trial identifier
|
Outcome
|
siRNA targeted therapeutics | |||||
ALN-VSP02 | VEGF, KSP | Systemic | Phase I (2011)/terminated | NCT00882180 | It was well-tolerated and had antitumor activity |
IV infusion | Phase I (2012)/completed | NCT01158079 | |||
Atu027 | PKN3 | Systemic | Phase I (2012) | NCT00938574 | It was safe in patients with advanced solid tumors |
IV infusion | Phase I/II (2016)/completed | NCT01808638 | |||
CALAA-01 | RRM 2 | Systemic; IV infusion | Phase I (2013)/terminated | NCT00689065 | It was well tolerated during the initial dose escalation portion of the phase Ia study |
siRNA-EphA2-DOPC | EphA2 | Systemic; IV infusion | Phase I 2015/active | NCT01591356 | It was well tolerated at all doses tested in preclinical studies |
TKM-PLK1 (TKM-080301) | PLK-1 | Systemic; IV infusion | Phase I/II (2016)/completed | NCT02191878 | It was tolerated and showed preliminary antitumor efficacy |
DCR-MYC (DCRM1711) | MYC | Systemic | Phase I (2017) | NCT02110563 | It was well tolerated and showed promising initial clinical and metabolic responses across various dose levels |
IV infusion | Phase Ib/2 (2016)/terminated | NCT02314052 | |||
NBF-006 | GSTP | Systemic; IV infusion | Phase1 (2019)/active | NCT03819387 | Significant tumor growth inhibition and overall survival benefit was observed |
miRNA targeted therapeutics | |||||
MRX34 | miR-34a mimic | IV infusion | Phase I/terminated-2016. Phase I-II/withdrawn-2016 | NCT01829971 | Unexpected severe immune-mediated toxicities observed |
NCT02862145 |
DCR-MYC: Lipid nanoparticle-formulated small inhibitory RNA; EphA2: Ephrin type-A receptor 2; GSTP: Glutathione S-transferase pi gene; IV: Intravenous; KSP: Kinesin spindle protein; miRNA: MicroRNA; PKN3: Protein kinase N3; PLK-1: Polo-like kinase 1; RRM2: Ribonucleotide reductase regulatory subunit M2; siRNA: Small interfering RNA; VEGF: Vascular endothelial growth factor.