Slide 1: Phase 3 Study Evaluating Efficacy and Safety of ANX005 in Patients with Guillain-Barré Syndrome
Phase 3 Study Evaluating Efficacy and Safety of ANX005 in Patients with Guillain-Barré Syndrome Dr. Quazi Deen Mohammad, MBBS, MD, FCPS National Institute of Neurosciences and Hospital (NINS), Dhaka, Bangladesh ANX005 is investigational and has not been approved for any indication in any jurisdictio
Slide 2: Disclosures
Disclosures Consultancy/advisory role with Annexon Biosciences 2
Slide 3: GBS is a Neurological Emergency with Long-Term Disability
GBS is a Neurological Emergency with Long-Term Disability Requires a Targeted and Effective Intervention to Immediately Block the Classical Complement Pathway POST-INFECTIOUS • Following infection, complement-activating COMPLEMENT- autoantibodies attack nerves leading to MEDIATED DISEASE nerve damag
Slide 4: Well Designed and Executed Pivotal Phase 3 Trial
Well Designed and Executed Pivotal Phase 3 Trial Randomized, Double-Blind, Placebo-Controlled Study (Best Supportive Care, no IVIg or PE) PATIENT SELECTION • Baseline GBS-DS score 3-5 2 DOSES SELECTED TO DETERMINE MOST • <10 days from onset of weakness EFFECTIVE DURATION OF INHIBITION • Stratified b
Slide 5: Baseline Characteristics Similar and Well Balanced Across Treatment Groups
Baseline Characteristics Similar and Well Balanced Across Treatment Groups Stratified by Key Prognostic Factors: MRC Sumscore and Time Since Onset of Weakness Placebo ANX005 30mg/kg ANX005 75mg/kg Baseline Characteristic (N=81) (N=79) (N=81) Age at Screening (years); mean (SD) 34.2 (12.59) 36.9 (14.
Slide 6: Primary Endpoint: ANX005 30 mg/kg Showed a Highly Significant and Clinically Meaningful Treatment Effect
Primary Endpoint: ANX005 30 mg/kg Showed a Highly Significant and Clinically Meaningful Treatment Effect on GBS-DS at Week 8 2.41-fold higher likelihood of being in a better state of health relative to placebo Grotta Bar of GBS-DS at Week 8 OR 2.41, p=0.0058 ANX005 30mg/kg N=78 Approach: Collapse 7-
Slide 7: GBS-DS Week 1 and Week 4: Early and Robust Treatment Effect with ANX005 30 mg/kg
GBS-DS Week 1 and Week 4: Early and Robust Treatment Effect with ANX005 30 mg/kg Week 1 GBS DS: OR 7.22, p<0.00011 Week 4 GBS DS: OR 2.49, p=0.00731 15% 44% 41% 3% 46% 51% • 0-1: Good State of Health • 2-3: Disabled • 4-6: Severely Disabled/Death 1Nominal 7 Topline Results Subject to Change
Slide 8: Week 8 Sensitivity Analyses: Robustness of the Primary Endpoint with ANX005 30 mg/kg
Week 8 Sensitivity Analyses: Robustness of the Primary Endpoint with ANX005 30 mg/kg Responder Analysis: ≥3-point at week 8 Traditional Dichotomy: (0-1, 2-6) at week 8 2x More Patients 2.5x More Patients Improved 3 Points or More Were Able to Run or Better (OR 3.34) p=0.0065 100% 30% 12% 29% 29% 80%
Slide 9: Accelerated Path to Recovery
Accelerated Path to Recovery Significantly more patients reached ‘normal’ by Week 26 in prespecified analysis OR 4.14 p=0.00921 21.5% % of Patients at GBS DS 2.5 times more treated patients fully recover at Week 26 (GBS-DS = 0) 8.6% Effect begins early and grows throughout study 0 0 1OR 4.14 1Nomina
Slide 10: Getting Better Sooner: Consistently Showed Faster Recovery Across Clinically Important Measures
Getting Better Sooner: ANX005 30 mg/kg Consistently Showed Faster Recovery Across Clinically Important Measures Helping patients achieve their independence sooner ANX005 56 Days WALKING EARLIER 30 mg/kg: N=79 31 days earlier, p=0.0211 PLACEBO 87 Days N=81 ANX005 20 Days OFF VENTILATION EARLIER 30 mg
Slide 11: GBS Phase 3 Results are Highly Relevant to US and European Populations
GBS Phase 3 Results are Highly Relevant to US and European Populations Significant treatment effect in western-world type patients US and European GBS Demographics ANX005 Phase 3 Study Results 80% have US/Europe baseline MRC of >20 Severe Disease GBS-DS 3.03x more likely to be better (MRC 0-20) 20%
Slide 12: ANX005 Generally Safe and Well-Tolerated
ANX005 Generally Safe and Well-Tolerated ANX005 ANX005 Placebo 30mg/kg 75mg/kg N=81 Majority of AEs were mild (Grade 1) to moderate N=79 N=81 (Grade 2) All Grades All Grades All Grades • Most common related events were infusion related reactions Number of Subjects Reporting TEAEs, n 79 80 Majority
Slide 13: ANX005 GBS Phase 3: A Transformational Advancement for the GBS Community
ANX005 GBS Phase 3: A Transformational Advancement for the GBS Community 1 Patients treated with ANX005 30 mg/kg were 2.4x more likely to be in a better health compared to placebo Phase 3 Met Primary Endpoint (p=0.0058) 2 Treatment during active disease phase was effective Defined Effective Treatmen
Slide 14: Special Thank You! to Patients and their Families & Care-partners
GBS-02 Principal Bangladesh: Khan Abul Kalam Azad, Zhahirul Islam, Prof Badrul Alam Investigators and Study Team Philippines: Jose Navarro, Fasl Goldanne Buenaflor, Lovelyn Matienzo, Bernadette Chua-Macrohon, Anuta Meldi, Margaret Modequillo, Mary Warren Ilaga, John Harold Hiyadan, Martha Bolanos Sc