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 | 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 | 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 | Refractory JDM Myositis-associated ILD IIM | Clinical improvement Evaluate efficacy and safety | |
Human recombinant monoclonal anti-ACVR2B activin type 2 receptor antibody | Phase IIb/III double-blind, placebo-controlled multicentre study Phase IIb/III Study | 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 | 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 | 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 |
Monoclonal humanised antibody against terminal complement components | Randomized, double-blind, placebo-controlled pilot study Phase two, randomized, placebo-controlled, third-party-blind study | 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 | 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 | DM and PM | Neutralisation of IFN gene signature suppression against disease improvement |