As published in The New England Journal of Medicine, findings from the international clinical trial VALOR offers encouraging news for people living with dermatomyositis, a rare autoimmune disease that causes muscle weakness, skin rashes, and inflammation that can affect the lungs, joints, heart, and other organs.
The phase 3 study found that the investigational oral medication brepocitinib significantly improved symptoms and physical function in adults with dermatomyositis whose disease had not responded adequately to previous treatments. Patients receiving the 30 mg dose of brepocitinib experienced meaningful improvements in muscle strength, skin disease activity, and overall quality of life compared with those receiving placebo. Many patients also were able to reduce or stop long-term steroid use, an important goal because chronic steroid therapy can cause serious side effects.
Improvements were seen as early as four weeks after treatment began and were sustained throughout the year-long study. Nearly 70% of patients receiving the higher dose achieved moderate or greater improvement in disease activity, and almost half achieved major improvement.
Senior author Rohit Aggarwal and colleagues noted that dermatomyositis can be difficult to treat and often has a major impact on patients’ mobility, independence, and quality of life. Current therapies may not fully control the disease and can carry significant side effects, highlighting the need for more targeted treatment options.
“Dermatomyositis has long been one of the most challenging diseases to manage, often robbing patients of their mobility, independence, and quality of life,” noted Aggarwal. “The VALOR trial results signal a turning point. Brepocitinib has the potential to transform how we care for these patients, moving us away from broad immunosuppression and steroid dependence toward therapies that precisely target the immune pathways driving the disease.”
The trial included 241 patients treated at 90 sites across 20 countries. Researchers also found that patients taking brepocitinib were more likely to successfully taper off glucocorticoids while maintaining disease control.
While serious infections occurred more frequently in patients receiving the higher dose of brepocitinib, no deaths occurred during the study, and most infections resolved with medical treatment.
Researchers say the findings highlight the potential for targeted therapies that address the immune pathways driving dermatomyositis and could help improve long-term outcomes for patients living with this chronic disease.