Biogen (BIIB) Stifel 2025 Virtual CNS Forum summary

Pipeline progress and strategic focus

• Significant pipeline progress in 2024, prioritizing key internal programs in Alzheimer's, lupus, and rare diseases, with accelerated timelines for proof-of-concept and phase III trials in 2025–2026.

• External innovation advanced through M&A (HI-Bio, felzartamab) and collaborations (Stoke Therapeutics, zorevunersen), with multiple phase III starts planned.

• Focused development on high-conviction, mid-to-late stage assets, aiming to establish franchises in core areas of expertise.

• Diversification strategy balances neuroscience with immunology and rare disease, leveraging deep expertise and aiming for higher probability of technical and regulatory success.

• Calculated risk-taking emphasized, with de-risked late-stage programs and regulatory alignment before advancing.

Key program updates

• Alzheimer's: Leqembi IV approved, subcutaneous maintenance under review with PDUFA in August 2024, subcutaneous initiation filing expected in 2024 and FDA outcome by mid-2026.

• Tau program (BIIB080): Robust preclinical and early clinical data show target engagement and durable tau reduction; phase II Celia study enrollment completed ahead of schedule, readout expected in 2026.

• Lupus: Dapirolizumab pegol achieved positive phase III results with significant reduction in severe flares and steroid use; litifilimab also in phase III, both targeting different mechanisms.

• Felzartamab: Compelling phase II data in AMR, IgAN, and PMN, with durable responses and unique mechanism; phase III starts planned for 2025.

• Spinraza High Dose: Regulatory submissions in US and Europe, PDUFA date in September 2024, supported by strong neurofilament and post-gene therapy data.

Market and innovation catalysts

• Uptake of Leqembi influenced by healthcare infrastructure, diagnostic hurdles, and the potential for blood-based biomarkers, with high probability of an FDA-approved IVD in the next 1–2 years.

• Blood-based biomarkers (e.g., Fujirebio) under FDA review, expected to improve diagnostic accuracy and access, with reimbursement and scaling as key factors.

• Brain shuttle delivery approaches for oligonucleotides are a strategic priority, with ongoing internal progress and partnerships.

• Patient engagement and heterogeneity in lupus inform trial design and asset positioning, aiming to address unmet needs with multiple modalities.

• Continued collaboration and momentum in launches, such as Zurzuvae for PPD, highlight focus on patient and prescriber needs.