Latozinemab

Summary

Latozinemab (CHEMBL4650439) is a phase-3 clinical-stage antibody targeting SORT1; indicated across 4 conditions including neurodegenerative disease and amyotrophic lateral sclerosis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Antibody
• Targets: 1 (SORT1)
• Indications: 4 conditions
• Clinical trials: 10

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4650439
Name	Latozinemab
Type	Antibody
Max phase	3

Also known as: AL-001, Al001, AL001, Latozinemab, LATOZINEMAB

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
SORT1	sortilin 1	Antagonist		1.4%	Q99523

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

Aggregated over 1 target gene(s): SORT1.

Top Reactome pathways

4 total, by targets touching each:

Pathway	Targets	Genes
Membrane Trafficking	1	SORT1
trans-Golgi Network Vesicle Budding	1	SORT1
Golgi Associated Vesicle Biogenesis	1	SORT1
Vesicle-mediated transport	1	SORT1

Dominant GO biological processes

GO term	Targets
ossification	1
protein targeting to lysosome	1
Golgi to endosome transport	1
endocytosis	1
G protein-coupled receptor signaling pathway	1
neuropeptide signaling pathway	1
endosome to lysosome transport	1
extrinsic apoptotic signaling pathway via death domain receptors	1
regulation of gene expression	1
myotube differentiation	1
vesicle organization	1
endosome transport via multivesicular body sorting pathway	1
response to insulin	1
negative regulation of fat cell differentiation	1
obsolete D-glucose import	1

Indications & clinical

Indications

4 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
neurodegenerative disease	3	MONDO:0005559	EFO:0005772
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976
frontotemporal dementia	2	MONDO:0017276	MONDO:0017276
dementia	1	MONDO:0001627	HP:0000726

Clinical trials

Total trials: 10.

Phase distribution

Phase	Trials
PHASE2	3
PHASE1/PHASE2	3
PHASE3	2
PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04374136	PHASE3	TERMINATED	A Phase 3 Study to Evaluate Efficacy and Safety of AL001 in Frontotemporal Dementia (INFRONT-3)
NCT06111014	PHASE3	TERMINATED	Continuation Study for Latozinemab
NCT06707753	PHASE1/PHASE2	ACTIVE_NOT_RECRUITING	Safety and Tolerability of AL-001 Ophthalmic Injection in Subjects with WAMD
NCT06839339	PHASE2	NOT_YET_RECRUITING	Efficacy and Safety of AL-001 Ophthalmic Injection in Subjects with WAMD
NCT07540338	PHASE1/PHASE2	RECRUITING	A Study to Investigate Lithium Brain/Plasma Pharmacokinetics and Safety of an AL001 Oral Capsule Compared to a Marketed Immediate-release Lithium Carbonate Capsule in Subjects With Bipolar I Disorder
NCT03987295	PHASE2	COMPLETED	A Phase 2 Study to Evaluate Safety of Long-term AL001 Dosing in Frontotemporal Dementia (FTD) Patients (INFRONT-2)
NCT05053035	PHASE2	TERMINATED	A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS
NCT05363293	PHASE1/PHASE2	COMPLETED	Multiple Ascending Dose Safety, Tolerability, PK Study of AL001 in Alzheimer’s Disease Patients & Healthy Adult Subjects
NCT06921590	PHASE1	ACTIVE_NOT_RECRUITING	A Study to Investigate Lithium Brain/Plasma Pharmacokinetics and Safety of an AL001 Oral Capsule Compared to a Marketed Immediate-release Lithium Carbonate Capsule in Healthy Adult Subjects
NCT03636204	PHASE1	COMPLETED	A First in Human Study in Healthy Volunteers and in Participants With Frontotemporal Dementia With Granulin (GRN) Mutation

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

1 molecules share ≥1 primary target. Top 1 by shared-target count:

Molecule	Source	Status	Shared targets
REMINERTANT	ChEMBL	Phase 3	SORT1