Remission & chronicity

Spontaneous remission

Dry AMD: slow progressive loss; wet AMD progresses rapidly without treatment (50% of untreated reach legal blindness within 2 years); no spontaneous remission of either form

Best-intervention remission

Anti-VEGF (ranibizumab, aflibercept, bevacizumab): 30-40% vision gain in wet AMD (MARINA, ANCHOR trials); maintain vision in 90%+ vs 62% in placebo; high-dose vitamin supplementation (AREDS2): 25% reduction in progression from intermediate to advanced dry AMD; port delivery system (Susvimo): 6-monthly refillable implant equivalent to monthly injections

Gap size

Large — treatment burden (monthly injections) is the primary barrier to adequate treatment in wet AMD; dry AMD has no proven disease-modifying treatment until 2023

Primary barrier

access/biological

Monthly anti-VEGF injection burden leads to under-treatment in real-world settings; 50% of wet AMD patients in routine care lose vision compared to 5-10% in trials; geographic atrophy (dry AMD) had no approved treatment until pegcetacoplan (Syfovre, 2023)

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

ANGPT2 11 ARMS2 0 C3 44 C5 23 CFB 146 CFD 193 CFH 4 CFI 1 FLT1 277 HTRA1 202 KDR 382 VEGFA 207

ANGPT2

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Source
VANUCIZUMAB	? unclear	Mechanistic	DGIdb
CVX-060	↓ inhibits	Mechanistic	DGIdb
CVX-241	? unclear	Mechanistic	DGIdb
FARICIMAB	↓ inhibits	Mechanistic	DGIdb
AMG-780	↓ inhibits	Mechanistic	DGIdb
NESVACUMAB	? unclear	Mechanistic	DGIdb
RECOMBINANT VASCULAR ENDOTHELIAL GROWTH FACTOR	? unclear	Mechanistic	DGIdb
TREBANANIB	? unclear	Mechanistic	DGIdb
ZANSECIMAB	? unclear	Mechanistic	DGIdb
RIBAVIRIN	? unclear	Mechanistic	DGIdb
MEDI-3617	↓ inhibits	Mechanistic	DGIdb

ARMS2

UniProt Ensembl DGIdb Open Targets

Pipeline results not yet available for this target. Run the pipeline to populate.

C3

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
PEGCETACOPLAN	↓ inhibits	Mechanistic		DGIdb
CLOZAPINE	? unclear	Mechanistic		DGIdb
POT-4	? unclear	Mechanistic		DGIdb
COMPSTATIN	? unclear	Mechanistic	1300.0 nM (Kd)	DGIdb
AL-78898A	↓ inhibits	Mechanistic		DGIdb
AMY-101	↓ inhibits	Mechanistic		DGIdb
CHEMBL265136	↓ inhibits	Mechanistic	53600.0 nM (IC50)	ChEMBL
CHEMBL409335	↓ inhibits	Mechanistic	32000.0 nM (IC50)	ChEMBL
CHEMBL269043	↓ inhibits	Mechanistic	13000.0 nM (IC50)	ChEMBL
CHEMBL414283	↓ inhibits	Mechanistic	6000.0 nM (IC50)	ChEMBL
CHEMBL415619	↓ inhibits	Mechanistic	6000.0 nM (IC50)	ChEMBL
CHEMBL429695	↓ inhibits	Mechanistic	7000.0 nM (IC50)	ChEMBL
CHEMBL265872	↓ inhibits	Mechanistic	6000.0 nM (IC50)	ChEMBL
CHEMBL268084	↓ inhibits	Mechanistic	3000.0 nM (IC50)	ChEMBL
CHEMBL415601	↓ inhibits	Mechanistic	6000.0 nM (IC50)	ChEMBL
CHEMBL411957	↓ inhibits	Mechanistic	7000.0 nM (IC50)	ChEMBL
CHEMBL413626	↓ inhibits	Mechanistic	12000.0 nM (IC50)	ChEMBL
CHEMBL411152	↓ inhibits	Mechanistic	55000.0 nM (IC50)	ChEMBL
CHEMBL438586	↓ inhibits	Mechanistic	47800.0 nM (IC50)	ChEMBL
CHEMBL437340	↓ inhibits	Mechanistic	10200.0 nM (IC50)	ChEMBL
CHEMBL268083	↓ inhibits	Mechanistic	9600.0 nM (IC50)	ChEMBL
CHEMBL2371729	↓ inhibits	Mechanistic	1500.0 nM (IC50)	ChEMBL
CHEMBL445418	↓ inhibits	Mechanistic	1500.0 nM (IC50)	ChEMBL
CHEMBL437270	↓ inhibits	Mechanistic	1400.0 nM (IC50)	ChEMBL
CHEMBL408836	↓ inhibits	Mechanistic	800.0 nM (IC50)	ChEMBL
CHEMBL439706	↓ inhibits	Mechanistic	800.0 nM (IC50)	ChEMBL
CHEMBL2371730	↓ inhibits	Mechanistic	1100.0 nM (IC50)	ChEMBL
CHEMBL430275	↓ inhibits	Mechanistic	600.0 nM (IC50)	ChEMBL
CHEMBL262797	↓ inhibits	Mechanistic	1800.0 nM (IC50)	ChEMBL
CHEMBL437245	↓ inhibits	Mechanistic	1400.0 nM (IC50)	ChEMBL
CHEMBL427664	↓ inhibits	Mechanistic	500.0 nM (IC50)	ChEMBL
CHEMBL412730	↓ inhibits	Mechanistic	23100.0 nM (IC50)	ChEMBL
CHEMBL269247	↓ inhibits	Mechanistic	2700.0 nM (IC50)	ChEMBL
CHEMBL219860	↓ inhibits	Mechanistic	35.0 nM (Kd)	ChEMBL
CHEMBL434556	↓ inhibits	Mechanistic	110.0 nM (Kd)	ChEMBL
CHEMBL1791380	↓ inhibits	Mechanistic	15.0 nM (Kd)	ChEMBL
CHEMBL1791379	↓ inhibits	Mechanistic	120.0 nM (Kd)	ChEMBL
CHEMBL1791382	↓ inhibits	Mechanistic	150.0 nM (Kd)	ChEMBL
CHEMBL427239	↓ inhibits	Mechanistic	17.0 nM (Kd)	ChEMBL
CHEMBL252927	↓ inhibits	Mechanistic	140.0 nM (Kd)	ChEMBL

Showing top 40 of 44 agents ranked by evidence tier.

C5

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
ZILUCOPLAN	↓ inhibits	Mechanistic		DGIdb
ECULIZUMAB	↓ inhibits	Mechanistic		DGIdb
TESIDOLUMAB	~ modulates	Mechanistic		DGIdb
OLENDALIZUMAB	↓ inhibits	Mechanistic		DGIdb
POZELIMAB	↓ inhibits	Mechanistic		DGIdb
CROVALIMAB	↓ inhibits	Mechanistic		DGIdb
AVACINCAPTAD PEGOL	↓ inhibits	Mechanistic		DGIdb
RAVULIZUMAB	↓ inhibits	Mechanistic		DGIdb
ZILUCOPLAN SODIUM	~ modulates	Mechanistic		DGIdb
VILOBELIMAB	↓ inhibits	Mechanistic		DGIdb
PEXELIZUMAB	↓ inhibits	Mechanistic		DGIdb
NOMACOPAN	↓ inhibits	Mechanistic		DGIdb
AVACINCAPTAD PEGOL SODIUM	? unclear	Mechanistic		Open Targets
CARPROFEN	↓ inhibits	Mechanistic	580.0 nM (Kd)	ChEMBL
OXAPROZIN	↓ inhibits	Mechanistic	640.0 nM (Kd)	ChEMBL
SULINDAC	↓ inhibits	Mechanistic	570.0 nM (Kd)	ChEMBL
RALOXIFENE	↓ inhibits	Mechanistic	710.0 nM (Kd)	ChEMBL
CHEMBL2204196	↓ inhibits	Mechanistic	440.0 nM (IC50)	ChEMBL
CHEMBL2203296	↓ inhibits	Mechanistic	210.0 nM (IC50)	ChEMBL
CHEMBL4756677	↓ inhibits	Mechanistic	620.0 nM (IC50)	ChEMBL
CHEMBL5594087	↓ inhibits	Mechanistic	940.0 nM (Kd)	ChEMBL
CHEMBL5594178	↓ inhibits	Mechanistic	700.0 nM (Kd)	ChEMBL
CEMDISIRAN	? unclear	Clinical		Open Targets

CFB

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
COMPOUND 51 [PMID: 19743866]	↓ inhibits	Mechanistic		DGIdb
IPTACOPAN HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
IPTACOPAN	↓ inhibits	Mechanistic	7.9 nM (Kd)	DGIdb
COMPSTATIN	↓ inhibits	Mechanistic	3400.0 nM (IC50)	ChEMBL
CHEMBL504763	↓ inhibits	Mechanistic	19000.0 nM (IC50)	ChEMBL
CHEMBL1089597	↓ inhibits	Mechanistic	10000.0 nM (IC50)	ChEMBL
CHEMBL1089595	↓ inhibits	Mechanistic	50000.0 nM (IC50)	ChEMBL
CHEMBL1089596	↓ inhibits	Mechanistic	7000.0 nM (IC50)	ChEMBL
CHEMBL1093412	↓ inhibits	Mechanistic	12000.0 nM (IC50)	ChEMBL
CHEMBL1089593	↓ inhibits	Mechanistic	3000.0 nM (IC50)	ChEMBL
CHEMBL1089594	↓ inhibits	Mechanistic	3000.0 nM (IC50)	ChEMBL
CHEMBL1089577	↓ inhibits	Mechanistic	23000.0 nM (IC50)	ChEMBL
CHEMBL1089578	↓ inhibits	Mechanistic	13000.0 nM (IC50)	ChEMBL
CHEMBL1089585	↓ inhibits	Mechanistic	5000.0 nM (IC50)	ChEMBL
CHEMBL1089592	↓ inhibits	Mechanistic	5000.0 nM (IC50)	ChEMBL
CHEMBL1089584	↓ inhibits	Mechanistic	4000.0 nM (IC50)	ChEMBL
CHEMBL1089574	↓ inhibits	Mechanistic	19000.0 nM (IC50)	ChEMBL
CHEMBL455818	↓ inhibits	Mechanistic	19000.0 nM (IC50)	ChEMBL
CHEMBL3949381	↓ inhibits	Mechanistic	290.0 nM (IC50)	ChEMBL
CHEMBL3945639	↓ inhibits	Mechanistic	90.0 nM (IC50)	ChEMBL
CHEMBL3908792	↓ inhibits	Mechanistic	50.0 nM (IC50)	ChEMBL
CHEMBL3931270	↓ inhibits	Mechanistic	720.0 nM (IC50)	ChEMBL
CHEMBL3894205	↓ inhibits	Mechanistic	1525.0 nM (IC50)	ChEMBL
CHEMBL3954424	↓ inhibits	Mechanistic	40.0 nM (IC50)	ChEMBL
CHEMBL3904754	↓ inhibits	Mechanistic	295.0 nM (IC50)	ChEMBL
CHEMBL3927308	↓ inhibits	Mechanistic	40.0 nM (IC50)	ChEMBL
CHEMBL3899277	↓ inhibits	Mechanistic	2350.0 nM (IC50)	ChEMBL
CHEMBL3926271	↓ inhibits	Mechanistic	540.0 nM (IC50)	ChEMBL
CHEMBL3910541	↓ inhibits	Mechanistic	460.0 nM (IC50)	ChEMBL
CHEMBL3913748	↓ inhibits	Mechanistic	60.0 nM (IC50)	ChEMBL
CHEMBL3958120	↓ inhibits	Mechanistic	90.0 nM (IC50)	ChEMBL
CHEMBL4114217	↓ inhibits	Mechanistic	1.0 nM (IC50)	ChEMBL
CHEMBL4247942	↓ inhibits	Mechanistic	23.0 nM (IC50)	ChEMBL
CHEMBL4244153	↓ inhibits	Mechanistic	250.0 nM (IC50)	ChEMBL
CHEMBL4636550	↓ inhibits	Mechanistic	6600.0 nM (IC50)	ChEMBL
CHEMBL4643155	↓ inhibits	Mechanistic	8800.0 nM (IC50)	ChEMBL
CHEMBL4636547	↓ inhibits	Mechanistic	33000.0 nM (IC50)	ChEMBL
CHEMBL4648708	↓ inhibits	Mechanistic	65000.0 nM (IC50)	ChEMBL
CHEMBL4637156	↓ inhibits	Mechanistic	8000.0 nM (IC50)	ChEMBL
CHEMBL4633534	↓ inhibits	Mechanistic	11000.0 nM (IC50)	ChEMBL

Showing top 40 of 146 agents ranked by evidence tier.

CFD

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
LAMPALIZUMAB	? unclear	Mechanistic		DGIdb
COMPOUND 2 [PMID: 28621538]	↓ inhibits	Mechanistic		DGIdb
EXAMPLE 373 [WO2012093101]	↓ inhibits	Mechanistic		DGIdb
DANICOPAN	↓ inhibits	Mechanistic		DGIdb
CHEMBL2180764	↓ inhibits	Mechanistic	2.0 nM (IC50)	ChEMBL
CHEMBL2180765	↓ inhibits	Mechanistic	1.0 nM (IC50)	ChEMBL
CHEMBL2180766	↓ inhibits	Mechanistic	3.0 nM (IC50)	ChEMBL
CHEMBL2181048	↓ inhibits	Mechanistic	24750.0 nM (IC50)	ChEMBL
CHEMBL3109653	↓ inhibits	Mechanistic	6.0 nM (IC50)	ChEMBL
CHEMBL3109654	↓ inhibits	Mechanistic	3.0 nM (IC50)	ChEMBL
CHEMBL3109655	↓ inhibits	Mechanistic	2.0 nM (IC50)	ChEMBL
CHEMBL3890777	↓ inhibits	Mechanistic	18.85 nM (Kd)	ChEMBL
CHEMBL3951127	↓ inhibits	Mechanistic	12000.0 nM (Kd)	ChEMBL
CHEMBL3941259	↓ inhibits	Mechanistic	16.98 nM (Kd)	ChEMBL
CHEMBL3924715	↓ inhibits	Mechanistic	2180.0 nM (IC50)	ChEMBL
CHEMBL2220483	↓ inhibits	Mechanistic	98.0 nM (IC50)	ChEMBL
CHEMBL3901198	↓ inhibits	Mechanistic	3700.0 nM (IC50)	ChEMBL
CHEMBL346059	↓ inhibits	Mechanistic	2900.0 nM (IC50)	ChEMBL
CHEMBL3673894	↓ inhibits	Mechanistic	25.0 nM (IC50)	ChEMBL
CHEMBL3942410	↓ inhibits	Mechanistic	1250.0 nM (IC50)	ChEMBL
CHEMBL3908849	↓ inhibits	Mechanistic	500.0 nM (IC50)	ChEMBL
CHEMBL4112048	↓ inhibits	Mechanistic	24.0 nM (IC50)	ChEMBL
CHEMBL3663968	↓ inhibits	Mechanistic	7.0 nM (IC50)	ChEMBL
CHEMBL3663969	↓ inhibits	Mechanistic	25.0 nM (IC50)	ChEMBL
CHEMBL3663970	↓ inhibits	Mechanistic	450.0 nM (IC50)	ChEMBL
CHEMBL3663971	↓ inhibits	Mechanistic	3980.0 nM (IC50)	ChEMBL
CHEMBL3663972	↓ inhibits	Mechanistic	30.0 nM (IC50)	ChEMBL
CHEMBL3663973	↓ inhibits	Mechanistic	2100.0 nM (IC50)	ChEMBL
CHEMBL3663974	↓ inhibits	Mechanistic	32.0 nM (IC50)	ChEMBL
CHEMBL3663975	↓ inhibits	Mechanistic	9.0 nM (IC50)	ChEMBL
CHEMBL3663976	↓ inhibits	Mechanistic	250.0 nM (IC50)	ChEMBL
CHEMBL3663977	↓ inhibits	Mechanistic	604.0 nM (IC50)	ChEMBL
CHEMBL3663978	↓ inhibits	Mechanistic	7.0 nM (IC50)	ChEMBL
CHEMBL3663979	↓ inhibits	Mechanistic	11135.0 nM (IC50)	ChEMBL
CHEMBL3663980	↓ inhibits	Mechanistic	139.0 nM (IC50)	ChEMBL
CHEMBL3663981	↓ inhibits	Mechanistic	55.0 nM (IC50)	ChEMBL
CHEMBL3663982	↓ inhibits	Mechanistic	1100.0 nM (IC50)	ChEMBL
CHEMBL3663983	↓ inhibits	Mechanistic	94.0 nM (IC50)	ChEMBL
CHEMBL3663984	↓ inhibits	Mechanistic	4048.0 nM (IC50)	ChEMBL
CHEMBL3663985	↓ inhibits	Mechanistic	815.0 nM (IC50)	ChEMBL

Showing top 40 of 193 agents ranked by evidence tier.

CFH

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
ECULIZUMAB	? unclear	Mechanistic		DGIdb
CR2-FH FUSION PROTEIN TT30	? unclear	Mechanistic		DGIdb
MENINGOCOCCAL GROUP B VACCINE	? unclear	Mechanistic		DGIdb
CHEMBL559015	↓ inhibits	Mechanistic	44000.0 nM (IC50)	ChEMBL

CFI

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
RECOMBINANT TRANSFORMING GROWTH FACTOR-BETA 1	? unclear	Mechanistic		DGIdb

FLT1

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
TIVOZANIB HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
RABEPRAZOLE	↓ inhibits	Mechanistic		DGIdb
ZM-306416	↓ inhibits	Mechanistic		DGIdb
LINIFANIB	↓ inhibits	Mechanistic		DGIdb
PAZOPANIB HYDROCHLORIDE	↓ inhibits	Mechanistic		DGIdb
FAMITINIB	↓ inhibits	Mechanistic		DGIdb
LUCITANIB	↓ inhibits	Mechanistic		DGIdb
PEGPLERANIB SODIUM	? unclear	Mechanistic		DGIdb
MOTESANIB	↓ inhibits	Mechanistic		DGIdb
LENVATINIB	↓ inhibits	Mechanistic		DGIdb
CEDIRANIB	↓ inhibits	Mechanistic		DGIdb
SORAFENIB	↓ inhibits	Mechanistic		DGIdb
SEMAXANIB	↓ inhibits	Mechanistic	8.0 nM (IC50)	DGIdb
VEGF RECEPTOR TYROSINE KINASE INHIBITOR III	↓ inhibits	Mechanistic		DGIdb
CEP-7055	↓ inhibits	Mechanistic		DGIdb
X-82	↓ inhibits	Mechanistic		DGIdb
PAZOPANIB	↓ inhibits	Mechanistic		DGIdb
VANDETANIB	↓ inhibits	Mechanistic	900.0 nM (IC50)	DGIdb
DOVITINIB	↓ inhibits	Mechanistic		DGIdb
FRUQUINTINIB	↓ inhibits	Mechanistic		DGIdb
CEP-11981	↓ inhibits	Mechanistic		DGIdb
REGORAFENIB	↓ inhibits	Mechanistic		DGIdb
BARASERTIB-HQPA	? unclear	Mechanistic		DGIdb
CP-547632	? unclear	Mechanistic		DGIdb
L-21649	↓ inhibits	Mechanistic		DGIdb
BRIVANIB ALANINATE	↓ inhibits	Mechanistic		DGIdb
CP-459632	↓ inhibits	Mechanistic		DGIdb
SUNITINIB	↓ inhibits	Mechanistic		DGIdb
GLESATINIB	↓ inhibits	Mechanistic		DGIdb
ILORASERTIB	↓ inhibits	Mechanistic		DGIdb
PF-562271	? unclear	Mechanistic		DGIdb
SU-14813	↓ inhibits	Mechanistic		DGIdb
PEXMETINIB	↓ inhibits	Mechanistic		DGIdb
TELBERMIN	↑ activates	Mechanistic		DGIdb
NINTEDANIB ESYLATE	↓ inhibits	Mechanistic		DGIdb
TAMATINIB	? unclear	Mechanistic		DGIdb
VEGFA	? unclear	Mechanistic		DGIdb
PEXIDARTINIB	↓ inhibits	Mechanistic		DGIdb
OSI-930	↓ inhibits	Mechanistic		DGIdb
GW843682X	? unclear	Mechanistic		DGIdb

Showing top 40 of 277 agents ranked by evidence tier.

HTRA1

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
GALEGENIMAB	? unclear	Mechanistic		DGIdb
HYDROGEN PEROXIDE	? unclear	Mechanistic		DGIdb
CHEMBL4453669	↓ inhibits	Mechanistic	2.3 nM (IC50)	ChEMBL
CHEMBL4550102	↓ inhibits	Mechanistic	8.3 nM (IC50)	ChEMBL
CHEMBL4540825	↓ inhibits	Mechanistic	2.6 nM (IC50)	ChEMBL
CHEMBL4467136	↓ inhibits	Mechanistic	1.5 nM (IC50)	ChEMBL
CHEMBL4435648	↓ inhibits	Mechanistic	2.0 nM (IC50)	ChEMBL
CHEMBL4540590	↓ inhibits	Mechanistic	0.4 nM (IC50)	ChEMBL
CHEMBL4530656	↓ inhibits	Mechanistic	0.6 nM (IC50)	ChEMBL
CHEMBL4446982	↓ inhibits	Mechanistic	0.5 nM (IC50)	ChEMBL
CHEMBL4459683	↓ inhibits	Mechanistic	0.6 nM (IC50)	ChEMBL
CHEMBL4473497	↓ inhibits	Mechanistic	1.7 nM (IC50)	ChEMBL
CHEMBL4468950	↓ inhibits	Mechanistic	0.6 nM (IC50)	ChEMBL
CHEMBL4461666	↓ inhibits	Mechanistic	0.4 nM (IC50)	ChEMBL
CHEMBL4565155	↓ inhibits	Mechanistic	0.8 nM (IC50)	ChEMBL
CHEMBL4462243	↓ inhibits	Mechanistic	0.9 nM (IC50)	ChEMBL
CHEMBL4438347	↓ inhibits	Mechanistic	1.6 nM (IC50)	ChEMBL
CHEMBL4542635	↓ inhibits	Mechanistic	0.3 nM (IC50)	ChEMBL
CHEMBL4444635	↓ inhibits	Mechanistic	0.7 nM (IC50)	ChEMBL
CHEMBL4568952	↓ inhibits	Mechanistic	0.9 nM (IC50)	ChEMBL
CHEMBL4585025	↓ inhibits	Mechanistic	0.4 nM (IC50)	ChEMBL
CHEMBL4588306	↓ inhibits	Mechanistic	0.4 nM (IC50)	ChEMBL
CHEMBL4453892	↓ inhibits	Mechanistic	0.5 nM (IC50)	ChEMBL
CHEMBL4471645	↓ inhibits	Mechanistic	0.3 nM (IC50)	ChEMBL
CHEMBL4533961	↓ inhibits	Mechanistic	0.6 nM (IC50)	ChEMBL
CHEMBL4473901	↓ inhibits	Mechanistic	79.5 nM (IC50)	ChEMBL
CHEMBL4471168	↓ inhibits	Mechanistic	68.0 nM (IC50)	ChEMBL
CHEMBL4538055	↓ inhibits	Mechanistic	77.3 nM (IC50)	ChEMBL
CHEMBL4591831	↓ inhibits	Mechanistic	0.9 nM (IC50)	ChEMBL
CHEMBL4462378	↓ inhibits	Mechanistic	0.5 nM (IC50)	ChEMBL
CHEMBL4557186	↓ inhibits	Mechanistic	0.5 nM (IC50)	ChEMBL
CHEMBL4441695	↓ inhibits	Mechanistic	0.5 nM (IC50)	ChEMBL
CHEMBL4533165	↓ inhibits	Mechanistic	0.3 nM (IC50)	ChEMBL
CHEMBL4542403	↓ inhibits	Mechanistic	0.5 nM (IC50)	ChEMBL
CHEMBL4561913	↓ inhibits	Mechanistic	9.0 nM (IC50)	ChEMBL
CHEMBL4475600	↓ inhibits	Mechanistic	4.3 nM (IC50)	ChEMBL
CHEMBL4514775	↓ inhibits	Mechanistic	2.3 nM (IC50)	ChEMBL
CHEMBL4558074	↓ inhibits	Mechanistic	20.2 nM (IC50)	ChEMBL
CHEMBL4456940	↓ inhibits	Mechanistic	36.3 nM (IC50)	ChEMBL
CHEMBL4447678	↓ inhibits	Mechanistic	6.4 nM (IC50)	ChEMBL

Showing top 40 of 202 agents ranked by evidence tier.

KDR

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Potency	Source
CEDIRANIB	↓ inhibits	Mechanistic		DGIdb
XL820	↓ inhibits	Mechanistic		DGIdb
EUPATORIN	↓ inhibits	Mechanistic		DGIdb
CABOZANTINIB S-MALATE	↓ inhibits	Mechanistic		DGIdb
TOCERANIB	↓ inhibits	Mechanistic		DGIdb
LAZERTINIB	↓ inhibits	Mechanistic		DGIdb
VADIMEZAN	? unclear	Mechanistic		DGIdb
VANDETANIB	↓ inhibits	Mechanistic	70.0 nM (IC50)	DGIdb
SURUFATINIB	↓ inhibits	Mechanistic		DGIdb
CANLITINIB	↓ inhibits	Mechanistic		DGIdb
FORETINIB	↓ inhibits	Mechanistic		DGIdb
PEGPLERANIB SODIUM	? unclear	Mechanistic		DGIdb
REGORAFENIB	↓ inhibits	Mechanistic		DGIdb
LINIFANIB	↓ inhibits	Mechanistic		DGIdb
MOTESANIB	↓ inhibits	Mechanistic		DGIdb
ILORASERTIB	↓ inhibits	Mechanistic		DGIdb
SUNITINIB	↓ inhibits	Mechanistic		DGIdb
COMPOUND 13A [PMID: 23639540]	↓ inhibits	Mechanistic		DGIdb
PONATINIB	↓ inhibits	Mechanistic		DGIdb
XL-820	↓ inhibits	Mechanistic		DGIdb
BX-795	↓ inhibits	Mechanistic		DGIdb
TELATINIB	↓ inhibits	Mechanistic		DGIdb
SORAFENIB	↓ inhibits	Mechanistic		DGIdb
LUCITANIB	↓ inhibits	Mechanistic		DGIdb
PP-121	↓ inhibits	Mechanistic		DGIdb
NINGETINIB	↓ inhibits	Mechanistic		DGIdb
TINENGOTINIB	↓ inhibits	Mechanistic		DGIdb
BJ-2412	↓ inhibits	Mechanistic		DGIdb
VEGF RECEPTOR TYROSINE KINASE INHIBITOR III	↓ inhibits	Mechanistic		DGIdb
PF-00337210	↓ inhibits	Mechanistic		DGIdb
AST-487	↓ inhibits	Mechanistic		DGIdb
RAMUCIRUMAB	↓ inhibits	Mechanistic		DGIdb
AG-13958	↓ inhibits	Mechanistic		DGIdb
SU-14813	↓ inhibits	Mechanistic		DGIdb
X-82	↓ inhibits	Mechanistic		DGIdb
ENMD-2076	↓ inhibits	Mechanistic		DGIdb
GOLVATINIB	↓ inhibits	Mechanistic		DGIdb
CENISERTIB	? unclear	Mechanistic		DGIdb
LENVATINIB	↓ inhibits	Mechanistic		DGIdb
SULFATINIB	↓ inhibits	Mechanistic		DGIdb

Showing top 40 of 382 agents ranked by evidence tier.

VEGFA

UniProt Ensembl DGIdb Open Targets

Therapeutic Agent	Effect Direction	Evidence Tier	Source
BEVASIRANIB	? unclear	Mechanistic	DGIdb
RANIBIZUMAB	↓ inhibits	Mechanistic	DGIdb
CP-547632	? unclear	Mechanistic	DGIdb
EW-A-401	? unclear	Mechanistic	DGIdb
BEVACIZUMAB 111IN	~ modulates	Mechanistic	DGIdb
FENOFIBRATE MICRONIZED	? unclear	Mechanistic	DGIdb
PEGAPTANIB	↓ inhibits	Mechanistic	DGIdb
DEXAMETHASONE	? unclear	Mechanistic	DGIdb
SQUALAMINE	? unclear	Mechanistic	DGIdb
AFLIBERCEPT	↓ inhibits	Mechanistic	DGIdb
PAZOPANIB	? unclear	Mechanistic	DGIdb
BEVACIZUMAB GAMMA	~ modulates	Mechanistic	DGIdb
CILOSTAZOL	? unclear	Mechanistic	DGIdb
ADALIMUMAB	? unclear	Mechanistic	DGIdb
MUPARFOSTAT	↓ inhibits	Mechanistic	DGIdb
SILDENAFIL	? unclear	Mechanistic	DGIdb
PENTOSAN POLYSULFATE	? unclear	Mechanistic	DGIdb
THALIDOMIDE	? unclear	Mechanistic	DGIdb
RISUTEGANIB	? unclear	Mechanistic	DGIdb
SELECTIVE CYTOKINE INHIBITORY DRUG CC-1088	? unclear	Mechanistic	DGIdb
PHENETHYL ISOTHIOCYANATE	? unclear	Mechanistic	DGIdb
EMVODODSTAT	? unclear	Mechanistic	DGIdb
CARFILZOMIB	? unclear	Mechanistic	DGIdb
FARICIMAB	↓ inhibits	Mechanistic	DGIdb
ALBUTEROL SULFATE	? unclear	Mechanistic	DGIdb
LENALIDOMIDE	↓ inhibits	Mechanistic	DGIdb
SUNITINIB	? unclear	Mechanistic	DGIdb
SHARK CARTILAGE EXTRACT AE-941	? unclear	Mechanistic	DGIdb
NM-3	? unclear	Mechanistic	DGIdb
DILPACIMAB	? unclear	Mechanistic	DGIdb
PEMETREXED DISODIUM	? unclear	Mechanistic	DGIdb
BI-836880	? unclear	Mechanistic	DGIdb
BROLUCIZUMAB	↓ inhibits	Mechanistic	DGIdb
DOMATINOSTAT	? unclear	Mechanistic	DGIdb
ANTHRACYCLINE ANTINEOPLASTIC ANTIBIOTIC	? unclear	Mechanistic	DGIdb
VEGF/HGF-TARGETING DARPIN MP0250	? unclear	Mechanistic	DGIdb
WARFARIN	? unclear	Mechanistic	DGIdb
CELECOXIB	? unclear	Mechanistic	DGIdb
NAVICIXIZUMAB	? unclear	Mechanistic	DGIdb
VEGLIN	? unclear	Mechanistic	DGIdb

Showing top 40 of 207 agents ranked by evidence tier.

Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Age-Related Macular Degeneration (AMD): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

An Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan (APL-2) in Subjects With Geographic Atrophy Secondary to AMD Completed

NCT04770545 Phase: 3 Started: 2021-03-04

This is a phase 3, open-label, multicenter, extension study to evaluate the long-term safety and efficacy of pegcetacoplan (APL-2) in subjects with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) who participated in Study APL2-103 (NCT03777332) or completed the treatment ...

Safety and Efficacy of Oral HX9428 Tablets in Subjects With Wet Age-related Macular Degeneration (wAMD) Recruiting

NCT07185841 Phase: 1/2 Started: 2025-07-01

This study will evaluate the safety, tolerability, pharmacokinetics and preliminary efficacy of Oral HX9428 tablets in patients with wet age-related macular degeneration(wAMD)

Extension Study for the Port Delivery System With Ranibizumab (Portal) Recruiting

NCT03683251 Phase: 3 Started: 2018-09-20

This study will evaluate the long-term safety and tolerability of the Port Delivery System with ranibizumab (PDS) (100 mg/mL) in participants with neovascular age-related macular degeneration (nAMD) who have either completed Phase II Study GX28228 (Ladder), Phase III Study GR40548 (Archway), Phase I...

Study to Assess the Injection Burden, Adverse Events, Change in Disease Activity, and Long-Term Preservation of Visual Acuity of Surabgene Lomparvovec in Adult Participants With Neovascular Age-Related Macular Degeneration (nAMD) Recruiting

NCT07007065 Phase: 3 Started: 2025-11-05

Neovascular age-related macular degeneration (nAMD), also known as "wet" AMD, is the abnormal growth of new blood vessels in the light-sensitive tissue at the back of the eye called the retina. The purpose of this study is to assess how safe and effective Surabgene Lomparvovec is in treating partici...

A Study to Determine the Efficacy, Safety, and Durability of Faricimab in Participants With Neovascular Age-Related Macular Degeneration Active Not Recruiting

NCT06795048 Phase: 4 Started: 2025-03-14

This study is a Phase IIIb/IV, multicenter, randomized, two-arm, open-label 100-week study to investigate the efficacy, safety, and durability of intravitreal 6-mg faricimab administered at up to 24-week intervals in patients with neovascular age-related macular degeneration (nAMD) that are treatmen...

A Masked, Placebo-controlled Study to Assess Iptacopan in Age-related Macular Degeneration Completed

NCT05230537 Phase: 2 Started: 2022-02-17

The purpose of this study is to assess the effect of Iptacopan to prevent conversion of early or intermediate age-related macular degeneration (AMD) eyes to new incomplete retinal pigment epithelium and outer retinal atrophy (iRORA) or late AMD.

4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration Active Not Recruiting

NCT06864988 Phase: 3 Started: 2025-03-03

A Phase 3, Randomized, Double-Masked, Active-Controlled Trial in Adults with Macular Neovascularization Secondary to Age-Related Macular Degeneration

A Study to Assess the Efficacy and Safety of RO7965032 in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration Recruiting

NCT07851259 Phase: 2 Started: 2027-01-08

This study will assess the safety and efficacy of RO7965032 to address a significant unmet medical need in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD)

A Dose-masked Study of Intravitreal Remigromig (EYE103) in Participants With NVAMD or Macular Edema Following BRVO Recruiting

NCT07205887 Phase: 2 Started: 2025-09-12

EYE-RES-104 is a randomized, dose-masked study of intravitreal remigromig (EYE103) in participants with neovascular age-related macular degeneration (NVAMD) or macular edema following branch retinal vein occlusion (BRVO). The study will consist of 4 patient cohorts, with participants in each cohort ...

Single Intravitreal (IVT) Injection of 4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration Recruiting

NCT07064759 Phase: 3 Started: 2025-07-22

This Phase 3 study is evaluating the safety and efficacy of 4D-150 versus aflibercept in participants with neovascular age-related macular degeneration (nAMD). Enrollment in the primary global cohort has been completed; Study centers in Japan remain open for enrollment for the Japan cohort.

Study in Human iPSCs-derived RPE Cells Transplantation for Advanced (Late-stage) AMD Recruiting

NCT07418177 Phase: 1/2 Started: 2025-10-14

Phase I Study of the Safety and Preliminary Efficacy of Human induced pluripotent stem cells-derived Retinal Pigment Epithelial Cells (hiPSC-RPEs) Subretinal Transplantation in Adanced (Late-Stage) Age-Related Macular Degeneration(AMD) Patients.

A Study of 36-Week Refill Exchanges of Port Delivery System (PDS) With Ranibizumab in nAMD Recruiting

NCT06847542 Phase: 3 Started: 2025-11-27

The purpose of this study is to evaluate the effectiveness, safety, and PROs of the port delivery system with ranibizumab 100 milligrams/milliliters (mg/mL) refilled every 36 weeks (Q36W) in participants with nAMD.

A Study to Evaluate Efficacy, Safety, and Immunogenicity With ABP 938 8 mg Versus EYLEA® HD (Aflibercept) in Participants With Neovascular Age-related Macular Degeneration Recruiting

NCT07614776 Phase: 3 Started: 2026-05-27

The aim of this trial is to demonstrate similarity in efficacy between ABP 938 8 mg and aflibercept (US) 8 mg by evaluating the change in best corrected visual acuity (BCVA) in participants with neovascular age-related macular degeneration (nAMD)

Study to Assess the Efficacy & Safety of KHK4951 in Patients With Neovascular Age-Related Macular Degeneration Completed

NCT06116890 Phase: 2 Started: 2024-01-31

The purpose of this study is to evaluate efficacy and safety of KHK4951 eye drops in patients with nAMD.

A Study to Investigate Efficacy and Safety of FWY003 Compared With Placebo in Participants With Geographic Atrophy Secondary to Age-related Macular Degeneration Recruiting

NCT07441642 Phase: 2 Started: 2026-03-09

To characterize the dose response relationship of FWY003 in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).

Agents Found By Disease-Level Search

Searched directly from “Age-Related Macular Degeneration (AMD)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 12-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

56 agents shown (approved or with trial evidence) — 49 not found by the gene-target search above.

Ranibizumab

Phase 3 · 7 trials · source: ClinicalTrials.gov, Open Targets
NCT01972763 NCT01277302 NCT04514653

AGN211745

Phase 2 · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT00363714 NCT00363714 NCT00363714

Bevacizumab

Phase 3 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00376701 NCT00376701 NCT00376701

SCT520FFNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT06672536 NCT06672536 NCT06672536

Aflibercept

Phase 3 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT07705555 NCT01796964 NCT04592419

Ranibizumab 0.5 mgNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01976338 NCT02130024

Lutein/ Zeaxanthin + Omega-3-FANOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00763659 NCT00763659

bevasiranib

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00722384 NCT00722384

MA09-hRPENOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01469832

TG100801

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00414999

K8NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06164587

VitrectomyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03244007

Spironolactone 50 MGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03744767

Avastin (Bevacizumab)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00376701

ASKG712NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05456828

MinocyclineNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02140164

0.5mg RanibizumabNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01710839

Targeted Pan Retinal PhotocoagulationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01710839

ABBV-RGX-314 Dose 1NOT in gene-target list