Table 3.

Small Molecule and Biologic Clinical Trials for IRDs and AMD

Disease Target	Product	Modality	Phase	Sponsor	NCT Number
Retinitis pigmentosa	N-acetyl cysteine (NAC)	Small molecule	3	Johns Hopkins University	NCT05537220
Stargardt disease	ALK001 C20-D3-retinyl acetate	Small molecule	2	Alkeus Pharma	NCT02402660
	Tinlarebant	Small molecule	3	Belite Bio	NCT05244304
	Metformin	Small molecule	1/2	National Eye Institute	NCT04545736
	STG-001	Small molecule	2a	Stargazer Pharma	NCT04489511
	Emixustat hydrochloride	Small molecule	3	Kubota Vision	NCT03772665
Usher syndrome	N-acetyl cysteine amide (NACA)	Small molecule	1/2	Nacuity Pharma	NCT04355689
Non-neovascular AMD	NGM621	Biologic	1	NGM Biopharma	NCT04014777
	NGM621	Biologic	2	NGM Biopharma	NCT04465955
	Iptacopan	Small molecule	2	Novartis	NCT05230537
	ALK-001	Small molecule	3	Alkeus	NCT03845582
	LBS-008/Tinlarabent	Small molecule	3	Belite Bio	NCT05949593
	CT1812	Small molecule	2	Cognition Therapeutics	NCT05893537
Neovascular AMD	AXT107	Biologic	1/2	AsclepiX Therapeutics	NCT05859776

Note: Inclusion in this table does not necessarily indicate that a trial is active.