Gefurulimab Clinical Bispecific

Antibody Information

Entry ID	274
INN	Gefurulimab
Status	Clinical
Drug code(s)	ALXN1720
Brand name	None
mAb sequence source	mAb humanized
General Molecular Category	Bispecific
Format, general category	Fragment
Format details	sdAb, VHH-VHH'
Isotype (Fc)	None
Light chain isotype	None
Linker	None
Ave. DAR	None
Conjugated/fused moiety	None
Discovery method/technology	Camelid-derived

Therapeutic information

Target(s)	Complement C5, Albumin
Indications of clinical studies	Generalized Myasthenia Gravis, Proteinuria, Phase 1 in healthy volunteers
Primary therapeutic area	Immune-mediated / inflammatory disorders

Development stage information

Phase lengths*

*The graph represents early-stage clinical development phase lengths. For molecules approved or under evaluation for marketing authorization in the US is provided a complete overview of all clinical development phase lengths. Phase lengths are calculated from the start of the first in human (FIH) study (Start of clinical phase). “Start of Phase 2” bar represents Phase 1 length (Start of clinical phase to start of Phase 2); “Start of Phase 3” bar represents Phase 1+2 length (Start of clinical phase to start of Phase 3); “Date BLA/NDA submitted” bar represents Phase 1+2+3 length (Start of clinical phase to Date BLA/NDA submitted); and “Date of first US approval” bar represents Phase 1 to first US approval length (Start of clinical phase to Date of first US approval).

Most advanced stage of development (global)	Phase 3
Status	Active
Start of clinical phase (IND filing or first Phase 1)	September 04, 2019
Start of Phase 2
Start of Phase 3	October 15, 2022
Date BLA/NDA submitted to FDA
Year of first approval (global)	None
Date of first US approval
INN, US product name	None
US or EU approved indications	None

Company information

Company	AstraZeneca
Licensee/Partner	None
Comments about company or candidate	NCT06607627 Phase 3 study in Generalized Myasthenia Gravis due to start in Mar 2025. Gefurulimab was granted orphan drug designation by the FDA for the treatment of patients with gMG. (September 2023) Phase 3 study for Generalized myasthenia gravis (NCT05556096) started in Nov 2022 is recruiting as of June 7, 2023 According to company website, 7 of nine cohorts are complete in a Phase 1 healthy volunteer study of ALXN1720. Due to COVID-19, the study was temporarily paused but is planned to restart in the third quarter of 2020. AstraZeneca acquired Alexion (formerly Syntimmune) NCT04920370 Phase 1 started in Sep 2019.
Full address of company	Cambridge, United Kingdom Europe United Kingdom https://www.astrazeneca.com/our-company/contact-us.html

Description/comment

ALXN1720 is a novel anti-C5 albumin-binding bi-specific mini-body optimized for sub-cutaneous delivery that binds and prevents activation of human C5. MW = 25 kDa; VH - VH'
Heavy-chain variable region antigen-binding fragment (VHH) antibodies targeting C5 and human serum albumin (HSA) were isolated from llama immune-based libraries and humanized. Gefurulimab comprises an N-terminal albumin-binding VHH connected to a C-terminal C5-binding VHH via a flexible linker. https://doi.org/10.1016/j.molimm.2023.12.004

Additional information

Anticipated events	None
Factor(s) contributing to discontinuation	None