JAK Inhibitors in Juvenile Myositis: From Mechanism to the Clinic
JAK inhibitors have moved from investigational promise to frontline clinical consideration in juvenile myositis faster than almost any other therapeutic class in the field’s history. The summit convened an international faculty of basic scientists, clinical trialists, and industry leaders to examine that trajectory in full — from the laboratory evidence establishing why JAK inhibition worked in dermatomyositis, through the existing clinical trial data, to the regulatory horizon and what approval of the first indication‑specific therapy could have meant for patients, families, and the clinicians who cared for them.
Sessions covered the mechanistic rationale for JAK/IFN pathway targeting in juvenile myositis, published clinical trial data on baricitinib in pediatric IIM, the Phase 3 adult trial results for brepocitinib (the first trial of its kind to meet its primary endpoint in dermatomyositis), and a regulatory and pipeline update from Priovant Therapeutics. The summit closed with an expert case panel featuring Cure JM Medical Advisory Board members discussing real‑world management of JDM patients treated with JAK inhibitors.
View the Full Presentation (or watch individual presenters below):
“Inhibition of the Type I Interferon Receptor Pathway Protects Against Muscle Weakness Induced by Dermatomyositis Serum”
 Daniel C. Andersson, MD, PhD, Associate Professor, Department of Physiology and Pharmacology, Karolinska Institute; Specialist Physician, Karolinska University Hospital, Stockholm, Sweden |
“Baricitinib in Juvenile Idiopathic Inflammatory Myopathies: Clinical Trial Evidence”
 Charalampia Papadopoulou, MD, PhD, Consultant and Honorary Associate Professor in Paediatric Rheumatology, Great Ormond Street Hospital and UCL Great Ormond Street Institute of Child Health, London, UK |
“Brepocitinib Phase 3 Adult Trial: What the Data Show”
 Ruth Ann Vleugels, MD, MPH, MBA, Chair, Penn Medicine Dermatology |
“Regulatory Update and Pediatric Indication: Where Brepocitinib Stands”
 Katharina Shaw, MD, Senior Vice President, Dermatology Franchise, Priovant Therapeutics |
