Positive pre-BLA meeting with FDA alignment reached on KYSA-8 single-arm trial in stiff person syndrome (SPS); rolling BLA submission initiated
Provides corporate update including appointment of gene therapy expert Romuald Corbau, Ph.D., as Chief Scientific Officer and an update on the CNS programs
Phase 1 study builds on early signals of anti-tumor activity in patients with refractory T and B cell lymphomas
Ms. Dac brings more than 30 years of experience successfully executing product launches and scaling commercial organizations for a range of therapeutic areas
Single-dose of miv-cel achieved robust and durable improvements in mobility, reversed disability scores, and eliminated the need for chronic immunotherapies – outcomes not previously observed in SPS
VGA039 is a once monthly, subcutaneously self-administered investigational therapy for the treatment of bleeding disorders, initially being evaluated for von Willebrand disease (VWD)
100% of patients achieved rapid, sustained improvements across MG-ADL and QMG at 24 weeks, further increasing confidence in Phase 3 trial
Phase 2a ROCSTAR study is evaluating ROC-101, a potential first-in-class oral pan-ROCK inhibitor, in PAH and ILD-PH patients
Envudeucitinib achieved robust PASI responses by Week 16, with significant continued improvements by Week 24 in PASI 90 (68.0%, 62.1%) and PASI 100 (41.0%, 39.5%)
Advancing first-to-market autoimmune CAR T opportunity in stiff person syndrome (SPS) with key launch preparation activities underway; BLA submission anticipated in 1H 2026
