Biogen (BIIB) Piper Sandler Virtual Novel Targets in Immunology Symposium summary

Strategic focus and pipeline overview

• Lupus, particularly SLE and CLE, is prioritized due to high unmet need and disease heterogeneity, with two late-stage assets: dapirolizumab and litifilimab.

• Decades of research and collaborations have informed target selection and clinical execution, aiming for better efficacy and patient-tailored options.

• The pipeline includes both biologics and plans for oral therapies, reflecting a multi-modal, multi-indication strategy.

• Early-stage programs target neuroinflammation (LRRK2) and B-cell biology (CD38), with potential applications beyond initial indications.

• Ongoing expansion into BTK and IRAK4 degrader programs supports a portfolio approach for autoimmune and inflammatory diseases.

Clinical development and trial design

• Litifilimab targets BDCA2 on plasmacytoid dendritic cells, disrupting interferon-driven inflammation and showing efficacy in both SLE and CLE, including skin and joint improvements.

• Dapirolizumab's phase III success includes steroid-sparing benefits, with up to a fifth of patients reducing steroid use to safer levels.

• Trials use different primary endpoints (SRI-4 for litifilimab, BICLA for dapirolizumab), both accepted by regulators and measured as primary/secondary in respective studies.

• CLE trials focus on rapid symptom resolution (24-week primary endpoint), while SLE studies emphasize both onset and durability of effect over 52 weeks.

• Safety considerations include dapirolizumab's lack of placental transfer, potentially enabling use in pregnancy and breastfeeding.

Industry landscape and future outlook

• The SLE treatment landscape is expected to diversify with upcoming phase III readouts for oral agents, supporting a non-exclusive, multi-therapy approach.

• Lifelong, relapsing-remitting disease nature and organ-specific manifestations necessitate a broad range of treatment options.

• BTK inhibitor and degrader programs in MS and other autoimmune diseases are being evaluated for differentiation and multi-indication potential.

• CD38-targeted therapies, including next-generation assets, are being developed for renal and broader autoimmune indications.

• Multiple data milestones are anticipated in the coming years, with ongoing efforts to align clinical strategy with evolving patient and regulatory needs.