Annexon, Inc. · NASDAQ:ANNX

Annexon — Technical Presentation (November 2023)

17 slides from Annexon's technical presentation, November 2023

  1. Slide 1: Efficacy and Safety of Intravitreal Injections of ANX007 in Geographic Atrophy: ARCHER Results

    Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy: Results of the ARCHER Study David Lally, Jeffrey Heier, Charles Wykoff, Glenn Jaffe, Roger A. Goldberg, Allen Hu, Carl Regillo, David Boyer, Karl Csaky, Wendy Murahashi, Harman Hansra, Scott Borland, Lori T

  2. Slide 2: Disclosures

    Disclosures • Financial disclosures:  Affamed (research grants), AGTC (consultant), Aldeyra Therapeutics (research grants), Alexion (research grants), Alimera (consultant), Allergan (speaker), Annexon (consultant; research grants), Apellis (consultant; research grants), Boehringer Ingelheim (consul

  3. Slide 3: Anti-C1q: A Distinct Neuroprotective Mechanism

    Anti-C1q: A Distinct Neuroprotective Mechanism C1q initiates classical complement cascade to drive photoreceptor synapse & cell loss and neuroinflammation • C1q is a key driver of neurodegeneration1 C1q binds stressed photoreceptor synapses and activates the classical pathway • C1q anchors classical

  4. Slide 4: Photoreceptor Cells, Synapses & Function Are Lost Prior to RPE in GA

    Photoreceptor Cells, Synapses & Function Are Lost Prior to RPE in GA Blocking C1q protects photoreceptor cells and function upstream of RPE loss Healthy Human Retina (top) • Uniform layer of photoreceptor synapses (red) and photoreceptor neurons (blue) Healthy Healthy human human retinal retinal tis

  5. Slide 5: ANX007: Differentiated Inhibitor of C1q and Classical Complement to Treat Geographic Atrophy

    ANX007: Differentiated Inhibitor of C1q and Classical Complement to Treat Geographic Atrophy FDA Fast Track status and EMA PRIME Designation granted for ANX007 KEY ATTRIBUTES  Design: Constant region framework modeled after established IVT administered Fab antibodies ANX007 IVT administered antigen

  6. Slide 6: ARCHER: Phase 2 Trial of C1q Inhibitor ANX007 in GA Patients

    ARCHER: Phase 2 Trial of C1q Inhibitor ANX007 in GA Patients​ Randomized, double-masked Included foveal and non-foveal lesions Stratified for lesion location and lesion size 12 months (n=270) Sham ANX007 5mg ANX007 5mg monthly or every other month monthly (EM) every other month (EOM) (n=89) (n=89) (

  7. Slide 7: Patient Demographics and Study Eye Characteristics Generally Well-Balanced Across Groups

    Patient Demographics and Study Eye Characteristics Generally Well-Balanced Across Groups SHAM POOLED ANX007 EM ANX007 EOM CHARACTERISTIC (N=89) (N=89) (N=92) Age, mean (SD) 79.8 (7.49) 79.7 (8.64) 80.5 (8.53) Female, n (%) 59 (66.3%) 47 (52.8%) 60 (65.2%) Caucasian, n (%) 87 (97.8%) 87 (97.8%) 89 (9

  8. Slide 8: Discontinuations Consistent with Previous GA Studies

    Discontinuations Consistent with Previous GA Studies SHAM (N=89) EM (N=89) EOM (N=92) Discontinued treatment 10 (11.2%) 13 (14.6%) 11 (12.0%) Withdrawal by subject Unrelated health issues 2 1 2 Moved / travel / time 1 2 2 Personal reasons / no 2 3 2 reason given Other 1 2 --- Death 2 2 3 Lost to fol

  9. Slide 9: Did Not Significantly Reduce Lesion Area, a Surrogate Biomarker of Functional Change in GA

    ANX007 Did Not Significantly Reduce Lesion Area, a Surrogate Biomarker of Functional Change in GA GA LESION AREA CHANGE FROM BASELINE TO MONTH 12+ 2.5 LS Mean （±SE）Change 2.0 GA Area Change from Baseline from Baseline, mm2 at 12 Months Sham (n=89) 1.5 ANX007 EM (n=89) Arm mm2 % p-value ANX007 EOM (n

  10. Slide 10: Effect on Lesion Growth Improves with Longer Treatment

    ANX007 Effect on Lesion Growth Improves with Longer Treatment CHANGE IN LESION GROWTH OVER TIME+ 5 +4.1% Growth Compared to Sham % Difference in Lesion 0 -1.0% EM (n=89) EOM (n=92) -5 -5.6% -10 -10.5% -15 0 to 6 Months 6 to 12 Months +The least-square (LS) mean and its standard error (SE) are based

  11. Slide 11: Statistically Significant Protection From Vision Loss as Measured by BCVA ≥15-Letter Loss

    ANX007 Demonstrated Statistically Significant Protection From Vision Loss as Measured by BCVA ≥15-Letter Loss PATIENTS WITH PERSISTENT BCVA ≥15-LETTER LOSS THROUGH MONTH 12+ 25 21.3% 5.6% 10.9% 8.3% 20 • First known significant preservation of % Patients 15 vision in GA 10 * • Dose-dependent respons

  12. Slide 12: Significant, Time-Dependent Protection From BCVA ≥15-Letter Vision Loss with Monthly Treatment

    Significant, Time-Dependent Protection From BCVA ≥15-Letter Vision Loss with ANX007 Monthly Treatment BCVA ≥15-LETTER LOSS AT 2 CONSECUTIVE VISITS THROUGH MONTH 12 OR LAST VISIT 100% % Patients without ≥15 Letter BCVA Loss 95% EM (n=89) 72% Risk Reduction ANX007 EM HR (CI) = 0.28 (0.11 to 0.76) 90%

  13. Slide 13: BCVA Subgroup Analysis: Protection from Vision Loss in Foveal and Non-Foveal Patients

    ANX007 BCVA Subgroup Analysis: Protection from Vision Loss in Foveal and Non-Foveal Patients PATIENTS WITH PERSISTENT BCVA ≥15-LETTER LOSS THROUGH MONTH 12+ 30 25.0% 5.9% 18.4% 17.8% 5.3% 2.3% Sham 20 EM % Patients EOM Nominal summary 10 p-value vs. sham^ EM 0.0019 11/44 3/51 9/49 8/45 2/38 1/43 EOM

  14. Slide 14: Protection from Vision Loss Consistent Across Baseline Characteristics

    ANX007 Protection from Vision Loss Consistent Across Baseline Characteristics HAZARD RATIO (ANX007 EM) HAZARD RATIO (ANX007 EOM) OVERALL adj. for baseline BCVA BCVA Baseline BCVA < 55 letters Baseline BCVA ≥ 55 letters adj. for baseline LLVD LLVD Baseline LLVD < 30 letters Baseline LLVD ≥ 30 letters

  15. Slide 15: BCVA ≥15-Letter Loss Accelerates After Cessation of Treatment

    BCVA ≥15-Letter Loss Accelerates After Cessation of Treatment Visual Function Loss Parallels Sham in Off-Treatment Period % PATIENTS WITH ANY BCVA ≥15-LETTER LOSS FROM BASELINE 30 Off Treatment • Low frequency (<10% per Sham Pooled (n=89) timepoint) of single BCVA 25 ANX007 EOM (n=92) ANX007 EM (n=8

  16. Slide 16: ANX007 Generally Well-Tolerated

    ANX007 Generally Well-Tolerated ADVERSE EVENTS OF SPECIAL INTEREST SHAM ANX007 EM ANX007 EOM n (%) (N=89) (N=89) (N=92) 3 4 4 Choroidal Neovascularization (3.4%) (4.5%) (4.3%) 1 2 Endophthalmitis 0 (1.1%) (2.2%) INTRAOCULAR INFLAMMATION DETAILS* n 1^ Iritis – 1 Retinal Vascular Occlusion 0 0 (1.1%)

  17. Slide 17: ANX007: A Novel Neuroprotective Agent Demonstrating Benefit in Vision in the ARCHER Trial

    ANX007: A Novel Neuroprotective Agent Demonstrating Benefit in Vision in the ARCHER Trial  C1q inhibition: distinct neuroprotective MOA  ANX007 treatment demonstrated:  Consistent visual function benefits  Highly statistically significant effect on visual acuity endpoints  Dose- and time-depend

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