Inadequate disease control remains a persistent challenge in the management of idiopathic inflammatory myopathies (IIM), particularly for dermatomyositis (DM) and polymyositis (PM), according to new research from Spherix Global Insights. Fewer than half of DM and PM patients achieve adequate disease control with current therapies, highlighting the urgent need for more effective, steroid-sparing options.
While glucocorticoids remain the first-line standard of care across IIM subtypes, their long-term use carries significant toxicity risks. This reliance on steroids underscores the limited efficacy of current second-line agents, including methotrexate, which is frequently added to glucocorticoid regimens with only modest success. The research suggests that the heterogeneity of IIM, coupled with the lack of targeted therapies, contributes to the high rate of inadequate disease control. Clinically amyopathic dermatomyositis (CADM) presents an added layer of complexity due to its primarily cutaneous manifestations and inconsistent response to immunosuppressants.
Rarer IIM subtypes like inclusion body myositis (IBM), anti-synthetase syndrome (ASyS), and necrotizing myopathy (NM) pose even greater therapeutic challenges. These conditions are often underdiagnosed and highly refractory to current treatments, leading to calls for earlier and more aggressive intervention. While intravenous immunoglobulin (IVIg), rituximab, and combination disease-modifying antirheumatic drug (DMARD) strategies are increasingly used, significant barriers remain, including diagnostic delays, insurance coverage limitations, and the absence of disease-modifying therapies. The unmet need is particularly acute for patients with interstitial lung disease (ILD) or severe muscle involvement.
This therapeutic gap has created significant anticipation for emerging pipeline agents, especially those offering the potential for rapid, steroid-sparing disease control. Anifrolumab (Saphnelo) is currently favored by rheumatologists due to its established use in lupus and targeted mechanism of action. Brepocitinib, a dual TYK2/JAK1 inhibitor, also holds promise due to its oral administration and broad anti-inflammatory properties. Other candidates generating interest include efgartigimod, dazukibart, and CD19-directed CAR T-cell therapies.
The focus for future IIM drug development centers on faster onset of action, broader disease targeting across affected organs (muscle, skin, lung), and improved patient convenience, such as once-daily oral dosing. With a significant portion of IIM patients eligible for advanced systemic therapies, the market is ripe for disruption. The greatest opportunity lies in developing agents with robust safety profiles, durable efficacy, and the potential to reduce or eliminate the need for long-term corticosteroid use. This will require not only novel mechanisms of action but also improved diagnostic tools and strategies to ensure timely intervention and appropriate patient selection for advanced therapies. The long-term success of these emerging therapies will depend on their ability to address the multifaceted challenges posed by the diverse spectrum of IIM subtypes.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
