Dalzanemdor

Summary

Dalzanemdor (CHEMBL5095095) is a phase-3 clinical-stage small molecule; indicated across 3 conditions including huntington disease and neurodegenerative disease.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 3 conditions
• Clinical trials: 11
• Chemistry: 456.6 Da · C27H43F3O2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL5095095
Name	Dalzanemdor
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	86305217
Molecular formula	C27H43F3O2
Molecular weight	456.6
InChIKey	BVBRUQYHUXKZMQ-JNVAYQLDSA-N

SMILES: C[C@H](CC[C@@](C)(C(F)(F)F)O)[C@H]1CC[C@@H]2[C@@]1(CC[C@H]3[C@H]2CC=C4[C@@]3(CC[C@](C4)(C)O)C)C

IUPAC name: (3S,8S,9S,10R,13R,14S,17R)-3,10,13-trimethyl-17-[(2R,5S)-6,6,6-trifluoro-5-hydroxy-5-methylhexan-2-yl]-1,2,4,7,8,9,11,12,14,15,16,17-dodecahydrocyclopenta[a]phenanthren-3-ol

Also known as: Dalzanemdor, Sage-718, SAGE-718, SAGE718, DALZANEMDOR

Patent coverage: 10 distinct patent families (20 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: 8 (assay-derived). Sample: Glutamate receptor ionotropic, NMDA 2B, Glutamate NMDA receptor; GRIN1/GRIN2B, Glutamate NMDA receptor; GRIN1/GRIN2A, Glutamate receptor ionotropic, NMDA 2A, Glutamate receptor ionotropic, NMDA 2D, Ionotropic glutamate receptor NMDA 1/2C, Ionotropic glutamate receptor NMDA 1/2D, Glutamate receptor ionotropic, NMDA 2C.

Bioactivity

ChEMBL activities: 8 potent at pChembl ≥ 5 of 8 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
GRIN1	7.1	EC50	79	nM	CHEMBL_ACT_24810687
GRIN2A	7.1	EC50	79	nM	CHEMBL_ACT_29263964
GRIN2B	7.1	EC50	79	nM	CHEMBL_ACT_29263966
GRIN2C	7.1	EC50	79	nM	CHEMBL_ACT_29263967
GRIN2D	7.1	EC50	79	nM	CHEMBL_ACT_29263968
GRIN1	7.07	EC50	86	nM	CHEMBL_ACT_24810650
GRIN1	6.82	EC50	150	nM	CHEMBL_ACT_24810808
GRIN2D	6.37	EC50	430	nM	CHEMBL_ACT_24810809

Target pathways

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

Indications & clinical

Indications

3 indications (0 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
Huntington disease	3	MONDO:0007739	MONDO:0007739
neurodegenerative disease	1	MONDO:0005559	EFO:0005772

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 11.

Phase distribution

Phase	Trials
PHASE2	6
PHASE1	4
PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05655520	PHASE3	TERMINATED	A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington’s Disease
NCT04476017	PHASE2	COMPLETED	A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Parkinson’s Disease Mild Cognitive Impairment (PD-MCI)
NCT04602624	PHASE2	COMPLETED	A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Mild Cognitive Impairment or Mild Dementia Due to Alzheimer’s Disease (AD)
NCT05107128	PHASE2	COMPLETED	A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington’s Disease (HD)
NCT05318937	PHASE2	COMPLETED	A Study to Evaluate the Effects of SAGE-718 in Participants With Parkinson’s Disease Cognitive Impairment
NCT05358821	PHASE2	COMPLETED	28-Day Study of SAGE-718 on Functioning Capacity in Participants With Huntington’s Disease
NCT05619692	PHASE2	COMPLETED	A Study to Evaluate the Effects of SAGE-718 in Participants With Mild Cognitive Impairment or Mild Dementia Due to Alzheimer’s Disease (AD)
NCT03770780	PHASE1	COMPLETED	A Study to Assess the Electrophysiology, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Response Using Magnetic Resonance Imaging of SAGE-718 Using a Ketamine Challenge in Healthy Subjects
NCT03771586	PHASE1	COMPLETED	A Study to Assess the Electrophysiology, Safety, Tolerability, and Pharmacokinetics of SAGE-718 Using a Ketamine Challenge in Healthy Subjects
NCT03787758	PHASE1	COMPLETED	A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of SAGE-718 Oral Solution in Patients With Huntington’s Disease - Part B
NCT03844906	PHASE1	COMPLETED	A Study to Assess Electrophysiology, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of SAGE-718 Using Ketamine Challenge in Healthy Subjects

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

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