Lefamulin

Summary

Lefamulin (CHEMBL3291398) is an approved small molecule (ATC J01XX12); indicated across 3 conditions including bacterial infectious disease and pneumonia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: J01XX12
• Indications: 3 conditions
• Clinical trials: 7
• Chemistry: 507.7 Da · C28H45NO5S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3291398
Name	Lefamulin
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	25185057
ATC	J01XX12
Molecular formula	C28H45NO5S
Molecular weight	507.7
InChIKey	KPVIXBKIJXZQJX-FCEONZPQSA-N

SMILES: C[C@@H]1CC[C@@]23CCC(=O)[C@H]2[C@@]1([C@@H](C[C@@]([C@H]([C@@H]3C)O)(C)C=C)OC(=O)CS[C@@H]4CC[C@H](C[C@H]4O)N)C

IUPAC name: [(1S,2R,3S,4S,6R,7R,8R,14R)-4-ethenyl-3-hydroxy-2,4,7,14-tetramethyl-9-oxo-6-tricyclo[5.4.3.01,8]tetradecanyl] 2-[(1R,2R,4R)-4-amino-2-hydroxycyclohexyl]sulfanylacetate

Also known as: BC-3781, Lefamulin, Lefamulina, Lefamuline, LEFAMULIN

Parent form; salt/anhydrous children: CHEMBL3545309

Patent coverage: 152 distinct patent families (328 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: Voltage-gated inwardly rectifying potassium channel KCNH2, Cytochrome P450 3A4, ATP-dependent translocase ABCB1.

Bioactivity

ChEMBL activities: 5 potent at pChembl ≥ 5 of 7 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CYP3A4	6.12	IC50	750	nM	CHEMBL_ACT_25894878
CYP3A4	6.07	Ki	860	nM	CHEMBL_ACT_25894710
CYP3A4	5.35	IC50	4430	nM	CHEMBL_ACT_14715690
CYP3A4	5.35	IC50	4460	nM	CHEMBL_ACT_25894711
CYP3A4	5.33	IC50	4650	nM	CHEMBL_ACT_14715683

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

3 indications (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
bacterial infectious disease	4	MONDO:0005113	EFO:0000771
pneumonia	3	MONDO:0005249	EFO:0003106
cystic fibrosis	1	MONDO:0009061	MONDO:0009061

Clinical trials

Total trials: 7.

Phase distribution

Phase	Trials
PHASE1	3
PHASE3	2
PHASE2	1
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02559310	PHASE3	COMPLETED	Study to Compare Lefamulin to Moxifloxacin (With or Without Linezolid) for the Treatment of Adults With Pneumonia
NCT02813694	PHASE3	COMPLETED	Study to Compare Lefamulin to Moxifloxacin for the Treatment of Adults With Pneumonia
NCT01119105	PHASE2	COMPLETED	Study Comparing the Safety and Efficacy of Two Doses of BC-3781 vs Vancomycin in Patients With Acute Bacterial Skin and Skin Structure Infection (ABSSSI)
NCT05111002	PHASE1/PHASE2	TERMINATED	Lefamulin for M. Genitalium Treatment Failures
NCT02557789	PHASE1	COMPLETED	Bioavailability and Pharmacokinetics of Lefamulin When Administered to Fed and Fasted Healthy Subjects
NCT03131141	PHASE1	COMPLETED	A Study to Assess Mass Balance Recovery, Metabolite Profile and Identification of IV and Oral 14C-BC-3781
NCT05225805	PHASE1	COMPLETED	Study to Assess the Safety and PK of Oral and IV Xenleta in Adults With Cystic Fibrosis

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).