Annexon, Inc. · NASDAQ:ANNX
Annexon — 40th Annual J.P. Morgan Healthcare Conference 2022
26 slides from Annexon's conference presentation, Jan 10, 2022
Slide 1: 40th Annual J.P. Morgan Healthcare Conference
Exhibit 99.1 40th ANNUAL J.P. MORGAN HEALTHCARE CONFERENCE JANUARY 11, 2022
Slide 2: Forward-looking Statements
Forward-looking Statements This presentation and accompanying oral presentation contain “forward‐looking” statements about Annexon, Inc. and our industr y that involve substantial risks and uncertainties. All statements other than statements of historical facts, including statements regarding our cl
Slide 3: Unlocking a New Generation of Complement Medicines
Unlocking a New Generation of Complement Medicines Disease processes 2 Therapeutic franchises: 3 Autoimmune, Neurodegeneration, Ophthalmology Clinical-stage candidates 3 Phase 2+ clinical trials underway 5 1 MISSION: Clinical data readouts by 2023 7 Bring game-changing therapies to patients sufferin
Slide 4: Rigorous Strategic Approach to Pioneering Complement Platform
Rigorous Strategic Approach to Pioneering Complement Platform Developing potential game-changing therapies for complement-mediated diseases in the body, brain & eye STRONG SELECT HIGH FIT-FOR- DISTINCT C1q BIOLOGICAL PTS PURPOSE DRUG MOAs RATIONALE INDICATIONS CANDIDATES Blocking Aberrant Targeting
Slide 5: C1q Inhibition Stops Classical Complement Activity at the Start
C1q Inhibition Stops Classical Complement Activity at the Start Potential for unique clinical outcomes – improvement shown in two challenging indications Guillain-Barré Syndrome (GBS) Huntington’s Disease (HD) Classical Pathway C1q Patients achieving ≥3 point 56% of patients improved at week improve
Slide 6: Robust Pipeline Diversified by Drug Candidate and Therapeutic Area
Robust Pipeline Diversified by Drug Candidate and Therapeutic Area Catalysts anticipated to readout by 2023 FRANCHISE CANDIDATE PRECLINICAL PHASE 1 PHASE 2 PHASE 2/3 ANX005 (IV) Huntington’s Disease (HD) Neurodegeneration ANX005 (IV) Amyotrophic Lateral Sclerosis (ALS) Neuro ANX105 (IV) Guillain-Bar
Slide 7: Portfolio of Fit-for-Purpose Drug Candidates Designed for Efficacy and Safety
Portfolio of Fit-for-Purpose Drug Candidates Designed for Efficacy and Safety Lead Molecule Antibody Fab: Localized inhibition of C1q in the eye • Intravenous ANX007 • Intravitreal administration • Full systemic/CNS C1q inhibition (IVT) • Geographic atrophy, glaucoma, other eye disease • GBS / acute
Slide 8: Restoring Function in Neurodegenerative Diseases
Restoring Function in Neurodegenerative Diseases ANX005 Huntington’s Disease Amyotrophic Lateral Sclerosis (ALS) 8
Slide 9: Significant Unmet Need for HD Patients – HD Phase 2 Interim Data
Significant Unmet Need for HD Patients – HD Phase 2 Interim Data Inherited, fatal, neurodegenerative disease that affects ~80K people globally with ~300K at risk ANX005 Phase 2 Trial in HD N = 28 adults with/at risk for manifest HD Total CAP score > 400 UHDRS independence score ≥ 80% 6‐ month treatm
Slide 10: Chronic Dosing with ANX005 Generally Well-Tolerated and Achieved Full Target Engagement
Chronic Dosing with ANX005 Generally Well-Tolerated and Achieved Full Target Engagement ANX005 Drug levels in CSF** ANX005 Generally Well-Tolerated* 10000 28 patients enrolled and received > 1 dose 1000 Majority of Adverse Events occurred during initial Day 1 100 infusion (~92.9%); majority low grad
Slide 11: NfL Levels Consistent and Comparable with HD Natural History at Week 24
NfL Levels Consistent and Comparable with HD Natural History at Week 24 Neuronal loss / NfL changes may lag improvement in synaptic function in slower progressing neurodegenerative diseases Plasma NfL Levels CSF NfL Levels 1 2 2 Interpolated data for manifest cohort; Rodriguez, et al., Sci Transl Me
Slide 12: Clinical Function Maintained Over 24-Week Treatment Period
Clinical Function Maintained Over 24-Week Treatment Period • >1600 patient natural data indicates cUHDRS declines by ~1/2 point at week 24 in early HD patients* • 56% of ANX005 treated patients improved on cUHDRS at week 24 Mean cUHDRS Maintained Over 24-Week 56% of Patients Improved Treatment Perio
Slide 13: Clinical Improvement Statistically Better in High Complement Patients
Clinical Improvement Statistically Better in High Complement Patients Significant Improvement in cUHDRS in Patients with Excess Complement Activity 75% of patients with excess complement activity (9/12) improved on cUHDRS at week 24 vs. 36% with low activity (4/11) p=0.03* All patients Baseline comp
Slide 14: Leveraging Recent HD Data to Inform Future Development
Leveraging Recent HD Data to Inform Future Development Potential for rapid clinical improvement ü Patients can be stratified by level of complement KEY LEARNINGS activity for potentially better response ü Potential to mitigate drug-related adverse events with enhanced screening, monitoring and next
Slide 15: ANX005 for ALS in Phase 2 Trial
ANX005 for ALS in Phase 2 Trial Amyotrophic Lateral Sclerosis (ALS): aberrant C1q activity involved in both CNS & PNS, impacting synapse loss & disability • Rapid, progressive loss of muscle function needed to move, speak, eat and breathe • ~19K U.S. patients High baseline NfL levels – potentially v
Slide 16: Improving Patient Outcomes in Autoimmune Diseases
Improving Patient Outcomes in Autoimmune Diseases ANX005 Guillain‐ Barré Syndrome Warm Autoimmune Hemolytic Anemia ANX009 Lupus Nephritis 16
Slide 17: ANX005 for GBS in Phase 2/3 Trial
ANX005 for GBS in Phase 2/3 Trial Well-tolerated, achieved full target engagement, reduced NfL and prevented disability in POC trial ANX005 Phase 1b Data in GBS Guillain-Barré Syndrome (GBS) Autoantibody attack on peripheral nerves Statistically Significant Patients achieving ≥3 point causing C1q ac
Slide 18: ANX005 for wAIHA in Phase 2 Trial
ANX005 for wAIHA in Phase 0/2 Trial Targeting patients with demonstrated excess complement activity at baseline ANX005 Preclinical Data – Presented at ASH 2021 Warm Autoimmune Hemolytic Anemia (wAIHA): Dysregulated destruction of healthy red blood cells causing anemia that affects daily Ex Vivo Bloc
Slide 19: ANX009 Entering Phase 1b for Lupus Nephritis
ANX009 Entering Phase 1 for Lupus Nephritis Targeting patients with demonstrated excess complement activity at baseline Serum Complement Activation vs LN Severity Lupus Nephritis (LN): Severe, life-threatening kidney disease Copy of C4d/C4 v/s UPCR 100 • Autoantibody-driven activation of C1q / class
Slide 20: Restoring Function in Neurodegenerative Retinal Diseases
Restoring Function in Neurodegenerative Retinal Diseases ANX007 Geographic Atrophy 20
Slide 21: ANX007 for GA in Phase 2 Trial
ANX007 for GA in Phase 2 Trial Intravitreal ANX007 Protects Photoreceptors and Retinal Geographic Atrophy (GA): Advanced form of Function in Mouse Model of Photoreceptors Damage age-related macular degeneration (AMD) involving dysregulated complement activity Anti-C1q Protects Photoreceptor Protects
Slide 22: Annexon Biosciences
ANNEXON biosciences
Slide 23: Broad Portfolio Provides Vast Opportunity to Help Patients
Broad Portfolio Provides Vast Opportunity to Help Patients Significant market opportunity in rare and prevalent diseases with high unmet need NEURO AUTOIMMUNE OPHTHALMOLOGY DEGENERATION 240K 4M 6M+ U.S. Patients U.S. Patients U.S. Patients MG 29K CIDP 29K ITP 30K ALS 19K AD 6M MMN 6K CAD 6K GLA 3M F
Slide 24: Multiple Value-Creating Catalysts Anticipated Through 2023
Multiple Value-Creating Catalysts Anticipated Through 2023 NEURODEGENERATION 1H 2022 2H 2022 2023 HD ANX005 (IV) Full Phase 2 Data ALS ANX005 (IV) Full Phase 2 Data ANX105 ANX005 (IV) First in Human Initiation ANX105 ANX005 (IV) First in Human Data AUTOIMMUNE 1H 2022 2H 2022 2023 GBS ANX005 (IV) Pha
Slide 25: Positioned For Explosive Growth with Potential Game-Changing Treatments
Positioned For Explosive Growth with Potential Game-Changing Treatments for Complement-mediated Disease Anti-C1q platform with broad and deep applicability to address destructive immune activity across autoimmune, neurodegenerative & ophthalmic diseases Demonstrated improvement in clinical measures
Slide 26: Thank You
ANNEXON Ne AP biosciences THANK YOU
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