Table 2.

Emerging biologic therapies for the treatment of adult and juvenile IIM

Biologic	Mechanism	Clinical trial type	Clinical trial number	Patient group	Outcome
rituximab [39]	Monoclonal anti-CD20 antibody that depletes B cells	Randomized, double-blind, placebo-phase trial	NCT00106184	JDM and DM	Higher proportion of JDM (87%) patients treated with rituximab met the definition of improvement more quickly compared to adult DM (78%)
belimumab	Anti-B cell activating factor (BAFF) monoclonal antibody	Multicentre double-blind, placebo-controlled trial	NCT02347891	Refractory IIM	Evaluating the efficacy and safety
abatacept	Modified fully human soluble recombinant protein that consists of cytotoxic T cell lymphocyte antigen-4 (CTLA4) fused with Fc region of human IgG1	Interventional clinical trial	NCT02594735 NCT03215927 NCT02971683	Refractory JDM Myositis-associated ILD IIM	Clinical improvement Evaluate efficacy and safety
bimagrumab [41, 42]	Human recombinant monoclonal anti-ACVR2B activin type 2 receptor antibody	Phase IIb/III double-blind, placebo-controlled multicentre study Phase IIb/III Study	NCT01925209 CBYM338B2203	IBM/IIM	Improvement in muscle volume and strength
spiponimod	Oral selective sphingosine-1-phosphate receptor modulator, acts by preventing the migration of lymphocytes to inflammatory sites and therefore reducing inflammation	Multicentre, phase 2, double-blind, randomized, controlled trial	NCT02029274NCT01148810	IIM	International Myositis Assessment Study (IMACS) definition of improvement
apremilast [44]	Phosphodiesterase-4(PDE-4) inhibitor, reduces the expression of pro-inflammatory cytokines by increasing cyclic adenosine monophosphate	Open-label, single-centre study Phase two, open-label, single group assignment, interventional study	NCT01140503, NCT03529955	DM	30% reduction in the cutaneous disease activity and severity index (CDASI) Safety, efficacy and clinical response
gevokizumab	Humanised IgG2 monoclonal antibody against human IL-1β	Proof-of-concept, randomized, double-blind, placebo-controlled trial	EudraCT number: 2012–005772-34	IIM	Prematurely terminated therefore limited results
eculizumab [46, 47]	Monoclonal humanised antibody against terminal complement components	Randomized, double-blind, placebo-controlled pilot study Phase two, randomized, placebo-controlled, third-party-blind study	NCT00005571	IIM DM	Improvement of global physician score for cutaneous disease Evaluation of safety and efficacy, results pending.
basiliximab [48]	IL-2R chimeric monoclonal antibody; blocks Il-2 receptor on the surface of activated T-cells	Open-label, randomized, parallel assignment without masking, phase-2, single center study	NCT03192657	Amyopathic dermatomyositis (CADM) patients with interstitial pneumonia	Primary outcome measure is survival at 52 weeks
sifalimumab [49]	anti-IFNα monoclonal antibody	Double-blind, phase 1b multicentre randomized control trial	NCT00533091	DM and PM	Neutralisation of IFN gene signature suppression against disease improvement