Table III.

Complement inhibitors in clinical development for paroxysmal nocturnal haemoglobinuria.

Class	Agent	Target	Clinical trial ID	Design	Patient population*	Study treatment	Results
Terminal inhibitors	Ravulizumab	C5	NCT02598583	Phase I/II, open‐label	Untreated PNH	Intra‐patient DE by IV infusions	Yes 95
			NCT02605993	Phase I/II, open‐label	Untreated PNH	MAD; IV infusions
			NCT02946463	Phase III, randomized versus Ecu	Untreated PNH	IV infusions (every eight weeks)	Yes 47 , 48 , 96
			NCT03056040	Phase III, randomized versus Ecu	Stable responders PNH	IV infusions (every eight weeks)	Yes 47 , 48 , 79
	Crovalimab	C5	NCT03157635	Phase I/II, multi‐part study	Untreated PNH and stable responders PNH	Intra‐patient DE by IV infusions, followed by SC injections	Yes 83
	LFG316 (tesidolumab)	C5	NCT02534909	Phase II, open‐label	Untreated PNH	IV infusions	Pending
	REGN3918 (pozelimab)	C5	NCT03946748	Phase II, open‐label, POC	Untreated PNH	IV and SC infusions	Pending
			NCT04162470	Phase II, open‐label, extension	Pozelimab‐treated PNH	IV and SC infusions	Ongoing
	ABP959	C5	NCT03818607	Phase III, randomized versus Ecu	Stable responders PNH	IV infusions	Ongoing
	Elizaria	C5	NCT04463056	Phase III, randomized versus Ecu	Untreated and eculizumab‐treated PNH	IV infusions	Pending
Proximal inhibitors	Pegcetacoplan	C3	NCT02264639	Phase Ib, open label, MAD, POC	Poor responders PNH	Daily, SC infusions	Yes 86
			NCT02588833	Phase Ib, open label, MAD, POC	Untreated PNH	Daily, SC infusions
			NCT03531255	Phase III, open label, extension	PNH exposed to APL‐2	Daily, SC infusions
			NCT03500549	Phase III, randomized versus Ecu	Poor responders PNH	SC infusions, BIH	Yes 87
	Danicopan	FD	NCT03053102	Phase Ib, open label, MD, POC	Untreated PNH	Orally, TID	Yes 89
			NCT03181633	Phase II, open‐label, extension	PNH exposed to ACH‐4471	Orally, TID	Ongoing
			NCT03472885	Phase II, open label, MD, POC	Poor responders PNH	Orally, TID	Yes 90
			NCT04469465	Phase III, randomized versus Ecu	Phase III, randomized vs Ecu	Orally, TID	Ongoing
	ACH5020	FD	NCT04170023	Phase II, open label, POC	Danicopan‐treated PNH, poor‐responders to anti‐C5 and untreated PNH	Orally, BID	Ongoing
	BCX9930	FD	NCT04330534	Phase I–II	PNH untreated	Orally, BID	Pending
			NCT04702568	Phase II, open label, extension	PNH, BCX9930‐treated	Orally, BID	Ongoing
	Iptacopan	FB	NCT03439839	Phase II, open label, POC	Poor responders PNH	Orally, BID	Yes 93
			NCT03896152	Phase II, open label, POC	Untreated PNH	Orally, BID	Pending
			NCT04558918	Phase III, randomized versus Ecu	Poor responders PNH	Orally, BID	Ongoing

BID, bis in die (twice a day); BIH, bis in hebdomade (twice a week); DE, dose escalation; Ecu, eculizumab; IV, intravenous; LDH, lactate dehydrogenase; MAD, multiple ascending doses; MD, multiple doses; PNH, paroxysmal nocturnal haemoglobinuria; POC, proof‐of‐concept; SC, subcutaneous; TID, ter in die (thrice a day).

^*Stable or poor response is intended to standard eculizumab treatment.