January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.
Priovant Therapeutics' brepocitinib demonstrated significant efficacy in the Phase 3 VALOR trial for dermatomyositis, meeting its primary endpoint with a 15.3-point greater improvement in Total Improvement Score versus placebo at Week 52. The treatment showed meaningful corticosteroid tapering and improvements across all nine key secondary endpoints, with benefits sustained through one year. Additional analyses revealed rapid itch reduction and improved skin-related quality of life in patients.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07775612 |
Pain-Sleep in Rheumatic Disease |
NOT_YET_RECRUITING | NA |
| NCT07486869 |
Emapalumab MDA5 Rapidly Progressive Interstitial Lung Disease (RP-ILD) Study |
NOT_YET_RECRUITING | PHASE2 |
| NCT06599411 |
Identification of Cutaneous and Blood Biomarkers Predictive of Response to Systemic Treatments During Chronic Inflammatory Skin Diseases |
RECRUITING | |
| NCT06154252 |
RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy |
RECRUITING | PHASE2/PHASE3 |
| NCT05979441 |
A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Active Idiopathic Inflammatory Myopathy |
ENROLLING_BY_INVITATION | PHASE3 |
| NCT05523167 |
A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy. |
COMPLETED | PHASE2/PHASE3 |
| NCT05437263 |
A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT04972760 |
Baricitinib in Patients With Relapsing or naïve Dermatomyositis |
RECRUITING | PHASE3 |
| NCT03816345 |
Testing an Immunotherapy Anti-cancer Drug, Nivolumab, for Advanced Cancers in Patients With Autoimmune Disorders, AIM-NIVO |
RECRUITING | PHASE1 |
| NCT03582800 |
Subcutaneous Injection of Sodium Thiosulfate for Ectopic Calcifications or Ossifications. A Pilot Study |
RECRUITING | PHASE2 |
