Dec. 3 at 4:56 PM
$ANNX — Jefferies Conference Review
Overall Takeaway:
Annexon delivered a very constructive update across all core programs. Management expressed strong confidence in the regulatory path for ANX005, highlighted meaningful EMA progress, clarified FDA timelines, and emphasized the importance of the FORWARD study in bridging U.S. regulatory expectations. The company also reinforced confidence in its GA Phase 3 program and its first-in-class complement small molecule. Tone throughout the presentation was notably positive.
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1. ANX005 (GBS) — EMA Filing Accepted; FDA Update Coming Early Q1
Management provided clear guidance on the progress of their lead neuroimmunology program:
• ANX005 is the company’s GBS therapy.
• The European Medicines Agency (EMA) has accepted the marketing authorization application for GBS.
• Annexon expects formal EMA movement and guidance in early Q1.
FDA Status
• The U.S. FDA BLA submission remains delayed, driven largely by shifting FDA expectations for neuroimmunology programs.
• To address this, Annexon is conducting the FORWARD Study, an open-label, global bridging study enrolling both U.S. and European GBS patients.
• Management expressed strong confidence that FORWARD will provide the clinical data needed for constructive FDA alignment.
Investor Impact:
EMA momentum + FORWARD bridging creates a clear and credible regulatory path despite FDA slowdowns.
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2. FDA Alignment — Update Expected Early New Year
Management guided that:
• A meaningful FDA regulatory update will occur in early Q1.
• The FORWARD Study is expected to bridge clinical expectations, provide fresh patient data, and restore momentum to the BLA pathway.
The tone was optimistic and focused on regulatory progress.
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3. Geographic Atrophy (GA) — Phase 3 Readout Expected Mid-2026
Annexon highlighted continued enthusiasm about their GA program:
• Phase 3 GA readout remains on track for mid-2026.
• Their upstream complement inhibition approach is designed to:
• preserve key retinal cell function,
• maintain photoreceptor integrity, and
• reduce functional vision loss, not just slow lesion expansion.
• Management emphasized that the company is educating the broader ophthalmology industry on why upstream inhibition could outperform downstream C5/C3 approaches.
Investor Impact:
If validated, ANNX could deliver differentiated clinical benefit versus current GA competitors.
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4. Complement Small Molecule (Autoimmune) — Only Small Molecule in the Complement Space
Management sounded highly confident about their complement-inhibition small molecule:
• Program update expected early Q1.
• This remains the only small molecule in the complement inhibition category.
• Annexon has invested years of design, optimization, and mechanism work into this molecule.
• Management expressed very strong confidence that this program represents a substantial catalyst.
Investor Impact:
A first-in-class small molecule provides commercial, dosing, and cost advantages versus biologics—and expands the autoimmune market opportunity.
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5. Tone and Positioning — Very Positive Across All Programs
Across the full pipeline, management’s tone was strong:
• ANX005 (GBS): EMA accepted; FORWARD progressing well; FDA update imminent.
• GA Phase 3: High mechanistic confidence; major mid-2026 catalyst.
• Complement small molecule: Expected early-Q1 update; only one of its kind in the space.
ANNX enters 2025 with a dense and meaningful catalyst path.
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Bottom Line
The Jefferies presentation reinforced a fundamentally strong outlook for Annexon:
• ANX005 (GBS):
• EMA filing accepted
• FDA alignment update early Q1
• FORWARD open-label bridging study producing the needed patient data
• GA Phase 3:
• Readout mid-2026
• Upstream inhibition offers mechanistic advantage
• Complement Small Molecule (Autoimmune):
• Early Q1 update
• Only small molecule in complement inhibition
Management was confident, constructive, and clear on all timelines, establishing ANNX as a catalyst-rich story heading into 2025.
