Table 2.
Oligonucleotide therapeutics for NASH in clinical trials
| Name (company) | Targeted gene | Targeting agent | Disease | Latest status | ClinicalTrials.gov identifier | Reference |
|---|---|---|---|---|---|---|
| AZD2693 (Ionis Pharmaceuticals) | PNPLA3 | ASO/ASO-GalNAc conjugate | NASH | phase 2 | NCT04483947 | Ionis Pharmaceuticals306 |
| ION224 (Ionis Pharmaceuticals) | DGAT2 | ASO/ASO-GalNAc conjugate | NASH | phase 2 | NCT04932512 | Ionis Pharmaceuticals311 |
| ALN-HSD (Alnylam Pharmaceuticals) | HSD17B13 | siRNA/(ESC+)-GalNAc conjugate | NASH | phase 2 | NCT05519475 | Regeneron Pharmaceuticals352 |
| ARO-HSD (Arrowhead Pharmaceuticals) | HSD17B13 | siRNA/siRNA-GalNAc conjugate | NASH | phase 1 | NCT04202354 | Mak et al.353 |
| ION455 (Ionis Pharmaceuticals) | HSD17B13 | ASO/ASO-GalNAc conjugate | NASH | phase 1 | NCT05143905NCT05560607 | Ionis Pharmaceuticals354 |
| BMS-986263 (Bristol Myers Squibb) | HSP47 | retinoid-conjugated LNP containing siRNA | NASH | phase 2 | NCT04267393 | Lawitz et al.288 |
| AMG 609 (Amgen) | PNPLA3 | siRNA-GalNAc conjugate | NAFLD | phase 1 | NCT04857606 | N/A |
| JNJ-75220795 (Arrowhead Pharmaceuticals) | PNPLA3 | TRIM platform419 | fatty liver disease | phase 1 | NCT04844450NCT05039710 | Arrowhead Pharmaceuticals363 |
| ALN-PNP (Alnylam Pharmaceuticals) | PNPLA3 | siRNA (in ESC+/GalNAc platform) | NASH | phase 1 | NCT05648214 | N/A |
| AZD4076 (Regulus Therapeutics) | miR-103/107 | GalNAc-conjugated anti-miRNA | NAFLD/T2D | phase 1/2a | NCT02826525 | Regulus Therapeutics401 |
| NASH | phase 1 | NCT02612662 |
PNPLA3, patatin-like phospholipase domain-containing 3; DGAT2, diacylglycerol acyltransferase 2; HSD17B13, 17β-hydroxysteroid dehydrogenase 13; HSP47, 47-kDa heat-shock protein.