Priovant Therapeutics Completes Enrollment in Global Phase 3 Study of Brepocitinib for Dermatomyositis

Priovant Therapeutics has successfully completed the enrollment phase of its Phase 3 VALOR study, which aims to evaluate the efficacy of brepocitinib in treating dermatomyositis (DM). This trial is significant as it is the largest interventional study ever conducted for this condition, having enrolled 241 subjects from 90 different sites across four continents.

Dermatomyositis is an idiopathic inflammatory disease affecting approximately 40,000 adults in the United States. It manifests through severe muscle weakness and skin lesions, leading to significant morbidity. Many patients with DM experience frequent falls, and a substantial number require mobility aids such as canes or wheelchairs. The disease can also cause disfiguring rashes and, in some cases, affects other organs like the lungs through interstitial lung disease. Currently, the only approved treatments for DM are steroids and intravenous immunoglobulin (IVIg).

Brepocitinib is an orally administered, dual selective inhibitor of TYK2 and JAK1. By inhibiting these enzymes, brepocitinib aims to suppress the signaling of multiple pathogenic cytokines that drive the disease activity in DM. The drug has already been tested in over 1,400 subjects and has shown positive outcomes in seven Phase 2 studies covering various autoimmune diseases.

Dr. Ruth Ann Vleugels, a distinguished chair in dermatology and director of several autoimmune skin disease programs, commented on the significance of the VALOR study. She noted that DM has limited treatment options and highlighted brepocitinib as a promising investigational therapy. The rapid enrollment in the VALOR study, she added, underscores the medical community's commitment to finding better treatment solutions for this debilitating disease.

The VALOR study is a double-blind, placebo-controlled trial where subjects have been randomized in a 1:1:1 ratio to receive either brepocitinib 30 mg, brepocitinib 15 mg, or a placebo. The primary endpoint of the study is the Total Improvement Score (TIS) at 52 weeks. This score is a composite measure of six different aspects of DM disease activity. Secondary endpoints include further assessments of skin and muscle disease, the impact on other organs, steroid-sparing benefits, and patient-reported quality of life outcomes. Results from this study are expected in the second half of 2025, potentially leading to a New Drug Application (NDA) submission.

Lynn Wilson, President of Myositis Support and Understanding (MSU), expressed optimism about the potential impact of the VALOR study on the dermatomyositis patient community. Paula Eichenbrenner, Executive Director of The Myositis Association (TMA), added that the successful enrollment of the study brings much-needed hope and excitement to patients and families affected by this challenging disease.

Apart from focusing on dermatomyositis, Priovant is also advancing the development of brepocitinib for treating non-infectious uveitis (NIU), a severe autoimmune condition and a leading cause of blindness among the working-age population. Preliminary data from the Phase 2 NEPTUNE study have shown promising results. Additional data on vascular leakage and macular edema will be presented at upcoming ophthalmology conferences. Priovant plans to share top-line results from the NEPTUNE study and initiate a Phase 3 program by the end of 2024.

Ben Zimmer, CEO of Priovant, emphasized the company's commitment to developing brepocitinib as a transformational therapy for highly morbid autoimmune diseases. He expressed gratitude to the patients, investigators, and research staff involved in these studies and looked forward to achieving significant milestones in the coming years.

Priovant Therapeutics is a biotechnology company specializing in developing innovative therapies for autoimmune diseases with high morbidity and limited treatment options. Brepocitinib, their leading therapeutic candidate, targets key cytokines linked to autoimmunity and is being evaluated for its efficacy in treating dermatomyositis and non-infectious uveitis.