TABLE 2.

Recent and ongoing trials investigating new molecules interfering with intracellular pathways in IIM.

Molecule	Mechanism of action	Type of trial	Patients	Primary outcome measure	Results
Brepocitinib (NCT05437263)	anti-JAK1/TYK2	Phase III, randomized, double-blind, placebo-controlled	DM	Total improvement score (TIS) at week 52	Ongoing
Enpatoran (NCT05650567)	TLR-7/8 antagonist	Phase IIa, randomized, parallel, double-blind, placebo controlled	DM, PM, ASS	- TIS at week 24; - Adverse events; - Clinically significant changes in laboratory, vital signs and ECG	Ongoing
IMO-8400 (83)	TLR7/8/9 antagonist	Phase II, randomized, double-blind, placebo-controlled	DM	Modified CDASIv2 activity score	Did not meet primary endpoint
Apremilast (84)	PDE4 inhibitor	Phase IIa, open-label, non-randomized controlled	DM	Overall response rate based on a 4 point decrease in CDASI at 3 months	Met primary endpoint
KZR-616 (NCT04033926)	Immunoproteasome inhibitor	Phase II, randomized, double-blind, placebo-controlled, crossover	DM, PM	Mean change in TIS	Ongoing
GLPG3667 (NCT05695950)	TYK2 inhibitor	Phase II, randomized, double-blind, placebo controlled	DM	Efficacy, safety, tolerability, pharmacokinetics	Not yet recruiting