Table Info

Table 1.

Non-exhaustive list of disclosed C5 therapeutics

Drug name	Company	Modality	Highest clinical stage achieved	Target indications
Eculizumab	Alexion/AZ	Humanized mAb	Marketed	aHUS (market), gMG (market), PNH (market), NMOSD (market), gMG pediatric (filed), NMOSD pediatric: phase 3
Ravulizumab	Alexion/AZ	Humanized long acting mAb	Marketed	PNH (market), aHUS (market), PNH pediatrics (market), aHUS pediatrics (market), gMG (market), NMOSD (filed), HSCT-TMA (phase 3), CSA-AKI (phase 3), Renal Basket (phase 2), Dermatomyositis (phase 2)
Avacopan	ChemoCentryx/Amgen	C5aR antagonist small molecule	Marketed	AAV: approved (United States, Japan, and European Union) C3G: phase 2 HS: phase 2
Zimura (Avacincaptad pegol)	Iveric Bio	Pegylated RNA aptamer	Marketed	GA: marketed (in the United States) Stargardt disease: phase 2
Zilucoplan	UCB	15-amino acid macrocyclic peptide	Marketed	gMG (market)
Vilobelimab	InflaRx	Anti-C5a mAb	Marketed	COVID-19: emergency authorization in the United States, Pyoderma Gangrenosum: phase 3
Pozelimab	Regeneron	mAb	Approved (CHAPLE)	PNH: phase 3 gMG: phase 3 GA: phase 3 (planned)
Eculizumab biosimilars	Multiple	mAb	Multiple phases including marketed	PNH (marketed)
Nomacopan (Coversin, AK576)	Akari	Small protein	Phase 3	HSCT-TMA: phase 3 GA: preclinical
Cemdisiran	Alnylam	GalNAc conjugated siRNA	Phase 3	PNH: phase 3 gMG: Phase 3 GA: phase 3 (planned)
Crovalimab	Chigai/Roche	Humanized long acting mAb	Phase 3	PNH: filed (Japan, China, United States, and European Union) aHUS: phase 3 LN: phase 1 GBS: phase 3 Scleroderma: phase 2
CAN106	CanBridge	Humanized long acting mAb	Phase1/2	PNH: phase 1b/2 (China)
RLYB-114, RLYB, 116	Rallybio	Antibody mimetic fusion protein + HSA binder, pegylated mAb	Phase 1	Rare disease, ophthalmology
KP-104	Kira Bio	Bi-functional mAb (include FH_1-5 tail)	Phase 1	IgAN, C3G, SLE-TMA, PNH: all indications phase 2
CB-301, CB-401	Cascade Biotech	Not disclosed	Preclinical	Not disclosed
PBP1603	Prestige	Eculizumab biosimilar	Preclinical	Not disclosed
HMI-104	Homology Medicine	rAAV gene therapy	Preclinical	Not disclosed
STP145G	Sirnaomics	siRNA	Preclinical	Not disclosed
IAB-101	ImmunAbs	mAb	Preclinical	Not disclosed

GalNAc: N-acetylgalactosamine; aHUS: atypical haemolytic uremic syndrome; AAV: antineutrophilic cytoplasmic antibody-associated vasculitis; C3G: C3 glomerulopathy; CSA-AKI: cardiac surgery-associated acute kidney injury; COVID-19: coronavirus disease 2019; FH: factor H; gMG: generalized myasthenia gravis; GA: geographic atrophy; GBS: Guillain-Barre syndrome; HSCT-TMA: hematopoietic stem cell transplantation-associated thrombotic microangiopathy; HS: hidradenitis suppurativa; HAS: human serum albumin; IgAN: IgA nephropathy; mAb: monoclonal antibody; NMO: Neuromyelitis Optica; PNH: paroxysmal nocturnal haemoglobinuria; rAAV: recombinant adeno-associated virus; siRNA: silencing ribonucleic acid; SLE-TMA: systemic lupus erythematosus-associated thrombotic microangiopathy; LN: lupus nephritis

Declarations

Acknowledgments

Authors want to thank Matt Ferenc for his suggestions in improving the manuscript.

Author contributions

MK: Conceptualization. MK and KNR: Writing—original draft. MY, AP, and PD: Writing—review & editing. All authors agree with the contents of the manuscript.

Conflict of interest

KNR, MY, AP, PD and MK are employees and shareholders at Apellis Pharmaceuticals Inc. Apellis researches, produces, and markets products that inhibit the complement system.

Ethical approval

Not applicable.

Consent to participate

Not applicable.

Consent to publication

Not applicable.

Availability of data and materials

Not applicable.

Funding

Not applicable.

Copyright

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