Pipeline

Program/Technology	Target	Indication	Discovery	IND-enabling	Phase 1	Phase 2	Phase 3	Ownership
Hepatic
Agazisiran	CFB	Renal (IgAN, C3G, IC-MPGN)
Agazisiran, Renal (IgAN, C3G, IC-MPGN) Close
Agazisiran	CFB	PNH
Agazisiran, PNH Close
Agazisiran	CFB	GA
Agazisiran, GA Close
Onvuzosiran	PKK	HAE
Onvuzosiran, HAE Close
ADX-626	FXI	Secondary Stroke Prevention & Stroke Prevention in AFib
ADX-626, Secondary Stroke Prevention & Stroke Prevention in AFib Close
Extrahepatic
ADX-077	Undisclosed1	Obesity
ADX-077, Obesity Close
ADX-199	APP	Neurodegeneration
ADX-199, Neurodegeneration Close

Hepatic
Discovery	IND-enabling	Phase 1	Phase 2	Phase 3
Agazisiran  Target: CFB | Indication: Renal (IgAN, C3G, IC-MPGN) Ownership:
Agazisiran  Target: CFB | Indication: PNH Ownership:
Agazisiran  Target: CFB | Indication: GA Ownership:
Onvuzosiran  Target: PKK | Indication: HAE Ownership:
ADX-626  Target: FXI | Indication: Secondary Stroke Prevention & Stroke Prevention in AFib Ownership:
Extrahepatic
Discovery	IND-enabling	Phase 1	Phase 2	Phase 3
ADX-077  Target: Undisclosed1 | Indication: Obesity Ownership:
ADX-199  Target: APP | Indication: Neurodegeneration Ownership:

1) A novel, undisclosed target in adipose tissue, which we refer to as “OBE1” whose reduction in expression has been shown to reduce adiposity, with the objective to reduce body weight and fat while preserving lean mass.
AFib = Atrial Fibrillation; APP = Amyloid-β Precursor Protein; C3G = Complement 3 Glomerulopathy; CFB = Complement Factor B; FXI = Factor XI; GA = Geographic Atrophy; HAE = Hereditary Angioedema; IC-MPGN = Immune Complex-Mediated Membranoproliferative Glomerulonephritis; IgAN = IgA Nephropathy; PKK = Prekallikrein; PNH = Paroxysmal Nocturnal Hemoglobinuria; SSP =  Secondary Stroke Prevention

1) A novel, undisclosed target in adipose tissue, which we refer to as “OBE1” whose reduction in expression has been shown to reduce adiposity, with the objective to reduce body weight and fat while preserving lean mass.
AFib = Atrial Fibrillation; APP = Amyloid-β Precursor Protein; C3G = Complement 3 Glomerulopathy; CFB = Complement Factor B; eGFR = Estimated Glomerular Filtration Rate; FXI = Factor XI; GA = Geographic Atrophy; HAE = Hereditary Angioedema; Hb = Hemoglobin; IC-MPGN = Immune Complex-Mediated Membranoproliferative Glomerulonephritis; IgAN = IgA Nephropathy; PKK = Prekallikrein; PNH = Paroxysmal Nocturnal Hemoglobinuria; SSP =  Secondary Stroke Prevention; UPCR = Urine Protein-Creatinine Ratio