Racemetyrosine

Summary

Racemetyrosine (CHEMBL1330596) is a phase-3 clinical-stage small molecule targeting TH; indicated across 6 conditions including exocrine pancreatic carcinoma and breast neoplasm.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 1 (TH)
• Indications: 6 conditions
• Clinical trials: 4
• Chemistry: 195.21 Da · C10H13NO3

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1330596
Name	Racemetyrosine
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	3125
Molecular formula	C10H13NO3
Molecular weight	195.21
InChIKey	NHTGHBARYWONDQ-UHFFFAOYSA-N

SMILES: CC(CC1=CC=C(C=C1)O)(C(=O)O)N

IUPAC name: 2-amino-3-(4-hydroxyphenyl)-2-methylpropanoic acid

Also known as: .alpha.-methyltyrosine, D, l-metyrosine, Dl-metyrosine, DNP-01, Racemetirosina, Racemetirosine, Racemetyrosine, RACEMETYROSINE COMPONENT OF SM-88, SM-88, Tyrosine, SID50105713

Patent coverage: 685 distinct patent families (2,124 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

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
TH	L-Tyrosine hydroxylase	Inhibition		0.2%	P07101

Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: ATP-dependent DNA helicase Q1, Cruzipain, Neuropeptide S receptor.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 3 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
P25779	6.9	Potency	125.9	nM	CHEMBL_ACT_3979858

Target pathways

Aggregated over 1 target gene(s): TH.

Top Reactome pathways

1 total, by targets touching each:

Pathway	Targets	Genes
Catecholamine biosynthesis	1	TH

Dominant GO biological processes

GO term	Targets
response to hypoxia	1
synaptic transmission, dopaminergic	1
heart morphogenesis	1
dopamine biosynthetic process from tyrosine	1
heart development	1
visual perception	1
learning	1
memory	1
mating behavior	1
locomotory behavior	1
regulation of heart contraction	1
anatomical structure morphogenesis	1
animal organ morphogenesis	1
dopamine biosynthetic process	1
epinephrine biosynthetic process	1

Indications & clinical

Indications

6 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
exocrine pancreatic carcinoma	3	MONDO:0005192	EFO:0002618
breast neoplasm	2	MONDO:0021100	MONDO:0007254
breast carcinoma	2	MONDO:0004989	EFO:0000305
autism	2	MONDO:0005260	EFO:0003758
autism spectrum disorder	2	MONDO:0005258	EFO:0003756
neoplasm	1	MONDO:0005070	EFO:0000616

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
PHASE2	3
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02562612	PHASE1/PHASE2	WITHDRAWN	Study of SM-88 in Advanced Cancers
NCT02947048	PHASE2	COMPLETED	Safety of L1-79 in Adolescent and Adult Males With Autism
NCT04720664	PHASE2	TERMINATED	Oral SM-88 in Patients With Metastatic HR+/HER2- Breast Cancer
NCT05067582	PHASE2	COMPLETED	A 12-Week Crossover Study to Assess the Efficacy, Safety and Tolerability of L1-79 in Subjects Aged 12-21 Years With Autism Spectrum Disorder

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).