Slide 1: ALS Phase 2a Clinical Trial Update
ALS Phase 2a Clinical Trial Update May 31, 2022 Exhibit 99.2
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation contains forward‐looking statements that involves substantial risks and uncertainties. Any statements about the company’s future expectations, plans and prospects, including statements about its strategy, future operations, development of its product cand
Slide 3: Tegoprubart: Pipeline in a Product Opportunity
Tegoprubart: Pipeline in a Product Opportunity Note: Development plans and timelines may change, including based on US and global regulatory interactions. Product Candidate Indication Development Stage Anticipated Milestones Pre-clinical Phase 1 Phase 2 Phase 3 Tegoprubart (AT-1501) Amyotrophic Late
Slide 4: ALS Overview & Market Opportunity
Characterized by gradual, progressive muscle weakness Affects ~30,000 Americans ~5,000 new cases diagnosed annually in the US and ~600,000 cases globally Average age of 55 at time of diagnosis Only 10% of ALS cases are hereditary ALS Overview & Market Opportunity People with ALS ultimately lose the
Slide 5: Pathophysiology of Amyotrophic Lateral Sclerosis
Pathophysiology in the Periphery Protein misfolding and cytoskeletal changes decrease axon transport Deficits in axon transport result in loss of neuromuscular junctions and muscle atrophy Macrophages phagocytose Schwann cells and neurons resulting in demyelination and presenting antigens to infiltr
Slide 6: Mechanism Overview of CD40L Inflammatory Signaling
tegoprubart T cell dependent antigen tegoprubart B CELL T CELL TCR CD40L CD40L Mechanism Overview of CD40L Inflammatory Signaling Interaction of CD40 with CD40L on immune cells mediates activation of the co-stimulatory immune pathway, controlling "cross talk” between the adaptive and innate immune s
Slide 7: Blocking CD40L Ameliorates Disease in SOD1 Mice
Blocking CD40L Ameliorates Disease in SOD1 Mice Source: Lincecum, 2010. D100 Blocking CD40L improves body-weight maintenance, delays disease onset and extends survival in SOD1 mice Kaplan-Meier time-to-event analysis for time required to attain peak body weight. Time to peak was not significantly (p
Slide 8: Inflammatory Pathways are Activated in the Periphery in ALS Animal Models
Inflammatory Pathways are Activated in the Periphery in ALS Animal Models Source: Lincecum, 2010. Onset of paralysis Day 70 Activation of Inflammatory Pathway in ALS Rodent Skeletal Model D30 D60 D100 D100 Macrophages accumulate on peripheral nerves in skeletal muscle Staining shows Myelin (Red), Ma
Slide 9: Blocking CD40L Reduces Neuroinflammation and Improves Motor Neuron Survival in SOD1 Mice
Blocking CD40L Reduces Neuroinflammation and Improves Motor Neuron Survival in SOD1 Mice Source: Lincecum, 2010. Anti-CD40L treatment reduces neuroinflammation in the spinal cord resulting in improved motor neuron survival Anti-CD40L (MR1) treatment reduces macrophage infiltrate and “attack” of dene
Slide 10: Blocking CD40L Improves Neuromuscular Junction Occupancy and Demyelination in SOD1 Mice
Blocking CD40L Improves Neuromuscular Junction Occupancy and Demyelination in SOD1 Mice Source: Lincecum, 2010; ALS Therapy Development Institute, unpublished data. Anti-CD40L treatment reduces macrophage infiltrate resulting in increased neuromuscular junction occupancy Staining: Myelin (green), al
Slide 11: Inflammatory Biomarkers & CD40L Levels Elevated in Serum of Patients with ALS
Inflammatory Biomarkers & CD40L Levels are Elevated in Serum of Patients with ALS Source: Brodovitch, 2021; Ots, 2022. Levels of inflammatory biomarkers have been found to be elevated in ALS patients and correlated with disease progression CD40-CD40L signaling between antigen presenting cells and T-
Slide 12: Phase 2a ALS: Study Design
Phase 2a ALS: Study Design Day: 1 29 15 101 43 57 71 -28 to -4 12-week, open label, multiple ascending dose level study Four sequential dose cohorts of 9 patients (1 and 2 mg/Kg) and 18 patients (4 and 8 mg/Kg) each Each subject serves as own control by comparing changes from baseline assessment
Slide 13: Phase 2a ALS: Demographics
Phase 2a ALS: Demographics 54 subjects recruited Average age - 59 years old 72% male 96% Caucasian Average baseline ALSFRS-R of 39.5 7 subjects had ALSFRS–R < 35 or ALS Bulbar domain scores ≤ 4 at first infusion Demographics and stage of disease confirm overall recruitment of target population Recru
Slide 14: Phase 2a ALS: Planned Data Generation
Phase 2 ALS: Planned Data Generation Pro-inflammatory Biomarkers Exploratory Endpoints Biomarkers of CD40L target engagement Safety & Tolerability Key sub-analyses: Compare subjects who did or not achieve target engagement as defined by a change in CXCL-13 Compare subjects who had target engagement
Slide 15: Phase 2a ALS: Safety & Tolerability
Phase 2a ALS: Safety & Tolerability All adverse events were reviewed by an independent data safety monitoring board that recommended continued dosing 35.2% of patients had 1 or more drug-related adverse events (AEs) No drug-related serious or severe AEs Occurrence of drug-related adverse events was
Slide 16: Phase 2a ALS: Key Observed Biomarker Decreases
Phase 2a ALS: Key Observed Biomarker Decreases at Week 12 Pro-Inflammatory CD40L Target Engagement Up to 23 of 32 inflammatory biomarkers detected were significantly reduced at 4 and 8 mg/Kg dose levels, including: IgA, IgE, IgM, C3, CXCL9 & CXCL10 IL-1 was not significantly detected in the 54 subje
Slide 17: Phase 2a ALS: Monthly Change in ALSFRS-R by Cohort
Monthly Change in ALSFRS-R (% Improvement vs. PRO-ACT) Group All Subjects Baseline Criteria Positive Target Engagement High Responders PRO-ACT (Comparator) -0.83 -0.83 -0.83 -0.83 All -1.02 22.9% n=54 -0.75 (9.6%) n=47 -0.67 (19.3%) N=40 -0.60 (27.7%) n=37 Low Dose (1/2 mgs) -0.89 7.2% n=18 -0.89 7.
Slide 18: Phase 2a ALS: Data Summary
Phase 2 ALS: Data Summary Pro-inflammatory Biomarkers Exploratory Endpoints Biomarkers of CD40L target engagement Safety & Tolerability 35.2% of patients had 1 or more drug-related adverse events (AEs) No drug-related serious or severe AEs Occurrence of drug-related adverse events was balanced acros
Slide 19: 2022 Execution Priorities
2022 Execution Priorities Note: Development plans and timelines subject to change based on several factors, including US and global regulatory agency interactions. Complete ALS Phase 2 study and release data Begin Kidney Transplantation clinical trial enrollment Advance tegoprubart subcutaneous form
Slide 20: Q&A
Q&A
Slide 21: Eledon Pharmaceuticals contact details
Eledon Pharmaceuticals 19900 MacArthur Blvd., Suite 550 Irvine, California 92612 USA info@eledon.com +1 949-238-8090