. 2020 Jul;72(3):639–667. doi: 10.1124/pr.119.019026

TABLE 2.

Current ongoing or recently completed clinical trials for therapeutic targeting of miRNAs

Drug (Company)	Delivery Strategy	Target miR	Therapeutic Purpose	Target Disease	Status	Clinical Trial Gov Identifier	References
Miravirsen (Santaris Pharma)	Anti-miR LNA	miR-122	Inhibition	HCC	Phase 2a	NCT01200420	Janssen et al., 2013
Miravirsen (Santaris Pharma)	Anti-miR LNA	miR-122	Inhibition	HCC	Completed (2010–2012)	NCT01200420	Janssen et al., 2013
MRX34 (Mirna Therapeutics)	Mimic miR in liposome	miR-34a	Restoration	Primary liver cancer	Phase 1	NCT01829971	Beg et al., 2017
				SCLC	Terminated (2013–2016)
				Lymphoma
				Melanoma
				MM
				RCC
				NSCLC
TargomiRs (EnGeneIC)	Mimic miR in EDV with EGFR antibody	miR-16	Restoration	MPM	Phase 1	NCT02369198	van Zandwijk et al., 2017
TargomiRs (EnGeneIC)	Mimic miR in EDV with EGFR antibody	miR-16	Restoration	NSCLC	Completed (2015–2017)	NCT02369198	van Zandwijk et al., 2017
Cobomarsen/MRG106 (MiRagen)	Anti-miR LNA	miR-155	Inhibition	MF	Phase 1	NCT02580552	NA
				CLL	Phase 1
				DLBCL	Ongoing (2015–)
				ATLL	Ongoing (2015–)
Cobomarsen/MRG106 (MiRagen)	Anti-miR LNA	miR-155	Inhibition	Cutaneous T-Cell lymphoma/MF	Phase 2	NCT03713320	NA
Cobomarsen/MRG106 (MiRagen)	Anti-miR LNA	miR-155	Inhibition	Cutaneous T-Cell lymphoma/MF	Ongoing (2018–)	NCT03713320	NA
Cobomarsen/MRG106 (MiRagen)	Anti-miR LNA	miR-155	Inhibition	Cutaneous T-Cell lymphoma/MF	Phase 2	NCT03837457	NA
Cobomarsen/MRG106 (MiRagen)	Anti-miR LNA	miR-155	Inhibition	Cutaneous T-Cell lymphoma/MF	Ongoing (2019–)	NCT03837457	NA
Remlarsen/MRG201 (MiRagen)	Mimic miR	miR-29	Restoration	Healthy volunteers	Phase 1	NCT02603224	NA
Remlarsen/MRG201 (MiRagen)	Mimic miR	miR-29	Restoration	Healthy volunteers	Completed (2015–2017)	NCT02603224	NA
Remlarsen/MRG201 (MiRagen)	Mimic miR	miR-29	Restoration	Keloid	Phase 2	NCT03601052	NA
Remlarsen/MRG201 (MiRagen)	Mimic miR	miR-29	Restoration	Keloid	Ongoing (2018–)	NCT03601052	NA
MRG-110 (MiRagen)	Anti-miR LNA	miR-92a	Inhibition	Healthy volunteers	Phase 1	NCT03603431	NA
MRG-110 (MiRagen)	Anti-miR LNA	miR-92a	Inhibition	Healthy volunteers	Completed (2018–2019)	NCT03603431	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Healthy volunteers	Phase 1	NCT02731885	Scherrer et al., 2016
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Healthy volunteers	Completed (2014–2015)	NCT02731885	Scherrer et al., 2016
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	HIV infection	Phase 2	NCT02735863	Rutsaert et al., 2019
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	HIV infection	Completed (2016–2017)	NCT02735863	Rutsaert et al., 2019
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	HIV infection; healthy volunteers	Phase 1/2	NCT02990325	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	HIV infection; healthy volunteers	Completed (2016–2017)	NCT02990325	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Ulcerative colitis	Phase 2	NCT03093259	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Ulcerative colitis	Completed (2017–2018)	NCT03093259	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Ulcerative colitis	Phase 2b	NCT03760003	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Ulcerative colitis	Ongoing (2018–)	NCT03760003	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Rheumatoid arthritis	Phase 2	NCT03813199	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Rheumatoid arthritis	Ongoing (2019–)	NCT03813199	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Crohn disease	Phase 2	NCT03905109	NA
ABX464 (Abivax S.A.)	Small molecule	miR-124	Overexpression	Crohn disease	Ongoing (2019–)	NCT03905109	NA

ATLL, adult T-cell leukemia/lymphoma; EDV, nonliving bacterial minicell nanoparticles; MF, mycosis fungoides; MM, multiple myeloma; MPM, malignant pleural mesothelioma; NA, not available; RCC, renal cell carcinoma; SCLC, small-cell lung cancer.