ADX-038 / ADARx - LARVOL DELTA

Interim Phase 1 Study Results of ADX-038: A Novel siRNA Against Complement Factor B, and Next Steps in IgAN and C3 Glomerulopathy (C3G) (KIDNEY WEEK 2025) - "The durable on-target PD effects support its potential as an effective therapeutic agent for kidney diseases with a convenient (Q3M) subcutaneous dosing schedule. Based on these results, a Phase 2 study has been initiated to assess the safety and efficacy of ADX-038 in patients with complement mediated kidney diseases (eg. IgAN and C3G)."

P1 data • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Infectious Disease • Nephrology • Renal Disease • Respiratory Diseases • CFB

Phase 2 Study of ADX-038 in Participants With Geographic Atrophy (clinicaltrials.gov) - P2 | N=240 | Not yet recruiting | Sponsor: ADARx Pharmaceuticals, Inc. | Initiation date: Aug 2025 ➔ Nov 2025

Trial initiation date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Phase 2 Study of ADX-038 in Complement-Mediated Kidney Disease (clinicaltrials.gov) - P2 | N=30 | Not yet recruiting | Sponsor: ADARx Pharmaceuticals, Inc.

New P2 trial • Glomerulonephritis • IgA Nephropathy • Nephrology • Renal Disease

Phase 2 Study of ADX-038 in Participants With Geographic Atrophy (clinicaltrials.gov) - P2 | N=240 | Not yet recruiting | Sponsor: ADARx Pharmaceuticals, Inc.

New P2 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients (clinicaltrials.gov) - P1/2 | N=50 | Recruiting | Sponsor: ADARx Pharmaceuticals, Inc. | Phase classification: P1 ➔ P1/2 | Trial completion date: Jun 2025 ➔ Sep 2026 | Trial primary completion date: Nov 2024 ➔ Mar 2026

Phase classification • Trial completion date • Trial primary completion date • Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Safety, Tolerability, PK and PD of ADX-038 in HV and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients (clinicaltrials.gov) - P1 | N=53 | Recruiting | Sponsor: ADARx Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CFB

Safety, Tolerability, PK and PD of ADX-038 in HV and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients (clinicaltrials.gov) - P1 | N=53 | Not yet recruiting | Sponsor: ADARx Pharmaceuticals, Inc.

New P1 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CFB