Table 1 Drugs that target amino acid metabolism in clinical trials

From: Amino acid metabolism in health and disease

Name

Research and development code

Target

Disease

Phase

Trial registration number

AXA1125

AXA-1125

 

NAFLD

 

NCT04073368

   

NASH

Phase 2

NCT04880187

   

COVID-19

Phase 2

NCT05152849

BCAT 1 Inhibitor

ERG-24;

ERG-245

Basigin (BSG);

Branched Chain Amino Acid Transaminase 1 (BCAT1);

Matrix Metallopeptidase 2 (MMP2); Matrix Metallopeptidase 9 (MMP9)

cancer/rheumatoid arthritis

Pre-clinical

 

BCAT2 modulator

 

Branched Chain Amino Acid Transaminase 2 (BCAT2)

organic acidemia/diabetes mellitus

Pre-clinical

 

BCKDK inhibitor

 

Branched Chain Keto Acid Dehydrogenase Kinase (BCKDK)

Insulin resistance; Maple glycosuria; Metabolic disorder; Type 2 diabetes

Pre-clinical

 

Nanvuranlat

JPH-203

L-type amino acid transporter 1 (LAT1)

Biliary tract carcinoma; Skin allergy; Solid tumor

Phase 2

 

4-L-[131I]iodo-phenylalanine

131I-ACD-101

L-type amino acid transporter 1 (LAT2)

glioblastoma

Phase 2

NCT03849105

QBS-10072S

QBS-10072S

L-type amino acid transporter 1 (LAT3)

Advanced solid tumor; Astrocytoma; Cholangiocarcinoma; Glioblastoma; Mesothelioma; Metastatic bladder cancer; Metastatic brain tumor; Metastatic breast cancer; Metastatic colorectal cancer; Metastatic esophageal carcinoma; Metastatic head and neck cancer; Metastatic liver cancer; Metastatic lung cancer; Metastatic ovarian cancer; Metastatic pancreatic cancer; Metastatic prostate cancer; Metastatic renal cell carcinoma; Metastatic gastric cancer; Metastatic urinary tract carcinoma; Sarcoma; Stage IV melanoma; Thymus tumor; Tongue disease; Cervical cancer

Phase 2

NCT04430842

O-(2-[18F] fluoroethyl)-L-tyrosine

TLX101-CDx

L-type amino acid transporter 1 (LAT4)

Glioblastoma; glioma

Clinical

NCT05632562; NCT03451123; NCT03216148;

NCT02286531; NCT01579253; NCT01443676

4-L-[124I] iodo-L-phenylalanine

124I-ACD-101

L-type amino acid transporter 1 (LAT5)

Brain tumor

Phase 1

 

astatinated IPA

211At-TLX-102

L-type amino acid transporter 1 (LAT6)

Multiple myeloma

Pre-clinical

 

R-OKY-034F

OKY-034

L-type amino acid transporter 1 (LAT7)

Pancreatic tumor

Phase 2

 

[18F] AA-7

[18 F] NKO-028

L-type amino acid transporter 1 (LAT8)

Cancer; glioma

Clinical

 

Crisantaspase

JZP-341

Asparaginase

Acute lymphoblastic leukemia; Adenocarcinoma; Advanced solid tumor; Hematologic tumor; Metastatic colorectal cancer

Pre-clinical

 

L-asparaginase

ERY-001

Asparaginase

Metastatic breast cancer; Metastatic pancreatic cancer; Ductal adenocarcinoma of pancreas; Solid tumor

Phase 3

NCT05660473; NCT05631327; NCT05581030;

NCT05326984; NCT05326516; NCT04956666;

NCT03618238

Pegaspargase biosimilar

PF-690

Asparaginase

Acute lymphoblastic leukemia; hematoma

Pre-clinical

 

PJ-017

PJ-017

Asparaginase

Advanced solid tumor

Pre-clinical

 

Pegargiminase

ADI-PEG-20

Arginine deiminase (ADI)

Acute myelogenous leukemia; Advanced solid tumor; Glioblastoma; Glioma; Hepatocellular carcinoma; Melanoma; Mesothelioma; Metastatic pancreatic cancer; Non-small cell lung cancer; Prostate tumor; Soft tissue sarcoma; Solid tumor; Uveal melanoma

Phase 3

NCT05616624; NCT05001828; NCT04587830;

NCT03449901

Pegzilarginase

AEB-1102

Arginase 1 (ARG1)

Acute myelogenous leukemia; Amino acid and protein metabolism disorders; Melanoma; Myelodysplastic syndrome; Small cell lung cancer; Uveal melanoma

New drug marketing application

 

Eryminase

 

Arginine deiminase (ADI)

Protein metabolism disorder

Pre-clinical

 

PFI-102

PFI-102

Peptidyl Arginine Deiminase 4 (PADI4)

Rheumatoid arthritis; Systemic lupus erythematosus

Pre-clinical

 

JBI-1044

JBI-1044

Peptidyl Arginine Deiminase 4 (PADI4)

Autoimmune disease; Cancer; Novel coronavirus pneumonia infection (COVID-19); Hidradenitis suppurativa; Inflammatory disease; Metastatic liver cancer; Rheumatoid arthritis; vasculitis

Pre-clinical

 

Arginine-depleting enzym

NEI-01

 

Acute myelogenous leukemia; Advanced solid tumor

Phase 1

NCT05226468

PEGylated arginine degrading enzymes

PJ-016

Arginase (ARG)

Metastatic carcinoma

Drug discovery

 

TNG-462

TNG-462

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor; Cholangiocarcinoma; Mesothelioma; Metastatic non-small cell lung cancer; Neuro-tumor; Solid tumor

Pre-clinical

 

AMG-193

AMG-193

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor; Non-small cell lung cancer

Phase 2

NCT05094336

MRTX-9768

MRTX-9768

Protein Arginine Methyltransferase 5 (PRMT5)

cancer

Pre-clinical

 

TNG-908

TNG-908

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor; Bladder cancer; Cholangiocarcinoma; Glioblastoma; Mesothelioma; Metastatic non-small cell lung cancer; Neuro-tumor; Squamous cell carcinoma

Phase 2

NCT05275478

PRT-543

PRT-543

Protein Arginine Methyltransferase 5 (PRMT5)

Acute myelogenous leukemia; Adenomatoid tumor; Advanced solid tumor; Breast tumor; Chronic granular monocytic leukemia; Diffuse large B-cell lymphoma; Hematologic tumor; Mantle cell lymphoma; Myelodysplastic syndrome; Myelofibrosis; Non-small cell lung cancer; Ovarian tumor; Spina bifida; Uveal melanoma

Phase 1

NCT03886831

PRT-811

PRT-811

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor; Brain tumor; Glioblastoma; Glioma; myelofibrosis

Phase 1

NCT04089449

MRTX-1719

MRTX-1719

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor

Phase 2

NCT05245500

Onametostat

JNJ-64619178

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor; Myelodysplastic syndrome; Non-hodgkin’s lymphoma

Phase 1

NCT03573310

PRMT-5 inhibitors

CTx-0262135

Protein Arginine Methyltransferase 5 (PRMT5)

Cancer; hematopathy

Drug discovery

 

SKL-27969

SKL-27969

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor

Phase 2

NCT05388435

SYHX-2001

SYHX-2001

Protein Arginine Methyltransferase 5 (PRMT5)

Acute myelogenous leukemia; Adenomatoid tumor; Advanced solid tumor; Hematologic tumor; Melanoma; Pancreatic tumor

Phase 1

NCT05407909

GSK-3226593

GSK-3226593

Protein Arginine Methyltransferase 5 (PRMT5)

Soft tissue sarcoma

Pre-clinical

 

SH-3765

SH-3765

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor; Non-hodgkin’s lymphoma

Phase 1

NCT05015309

AM-9747

AM-9747

Protein Arginine Methyltransferase 5 (PRMT5)

Cancer

Pre-clinical

 

PF-06939999

PF-06939999

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor; Endometrial carcinoma; Metastatic bladder cancer; Metastatic esophageal carcinoma; Metastatic head and neck cancer; Metastatic non-small cell lung cancer; Squamous cell carcinoma; Cervical cancer

Phase 1

NCT03854227

AGX-323

AGX-323

Protein Arginine Methyltransferase 5 (PRMT5)

Cancer

Pre-clinical

 

ALG-070043

ALG-070043

Protein Arginine Methyltransferase 5 (PRMT5)

Hepatocellular carcinoma; Non-small cell lung cancer

Pre-clinical

 

PRT-220

PRT-220

Protein Arginine Methyltransferase 5 (PRMT5)

Graft versus host disease

Pre-clinical

 

OATD-02

OATD-02

Arginase 1 (ARG1); Arginase 2 (ARG2)

Advanced solid tumor; Metastatic colorectal cancer; Metastatic ovarian cancer; Metastatic pancreatic cancer; Metastatic renal cell carcinoma

Phase 1

NCT05759923

IO-112

IO-112

Arginase 1 (ARG1)

Solid tumor

Phase 1

NCT03689192

Resminostat

YHI-1001

Arginase 1 (ARG1);

Histone deacetylase (HDAC);

Histone Deacetylase 1 (HDAC1);

Histone Deacetylase 2 (HDAC2);

Histone Deacetylase 3 (HDAC3);

Histone Deacetylase 6 (HDAC6)

Biliary tract tumor; Cholangiocarcinoma; Colorectal cancer; Cutaneous T-lymphoblastoma; Gallbladder tumor; Hepatocellular carcinoma; Hodgkin,s lymphoma; Mycosis fungoides;

Non-small cell lung cancer; Pancreatic neoplasm; Sezary syndrome;

Solid tumor

Phase 2

NCT02400788

CB-280

CB-280

Arginase (ARG)

Cystic fibrosis

Phase 1

NCT04279769

Arginase inhibitor

AZD-0011

Arginase (ARG)

Cancer

Pre-clinical

 

Numidargistat

INCB-01158

Arginase (ARG);

T cell receptor gene (TCR)

Advanced solid tumor; Biliary tract carcinoma; Bladder cancer; Cancer; Colorectal cancer; Endometrial carcinoma; Esophageal tumor; Head and neck tumors; Lung tumor; Melanoma; Mesothelioma; Multiple myeloma; Non-small cell lung cancer; Ovarian tumor; Renal cell carcinoma; Squamous cell carcinoma; Gastric tumor; Transitional cell carcinoma

Phase 2

NCT03314935; NCT02903914

OATD-05

OATD-05

Arginase (ARG)

Cancer

Drug discovery

 

Pegylated, cobalt-replaced human arginase

PT-01

Arginase (ARG)

Cancer; Metastatic carcinoma; Metastatic liver cancer; Stage III melanoma; Stage IV melanoma

Phase 1

NCT04136834

Bicyclic arginase inhibitors

 

Arginase 1 (ARG1)

Cancer

Pre-clinical

 

C-0021158

C-0021158

Arginase 2 (ARG2)

Cancer

Drug discovery

 

Pegylated human arginase

PEG-BCT-100

Arginase (ARG)

Acute lymphoblastic leukemia; Acute myelogenous leukemia; Advanced solid tumor; Cancer; Glioma; Hepatocellular carcinoma; Hormone-resistant prostate cancer; Neuroblastoma; Renal cell carcinoma; Retinopathy; Sarcoma; Stage IV melanoma

Phase 2

NCT03455140; NCT02899286; NCT02285101;

NCT02089633; NCT02089763; NCT01092091;

NCT00988195

AB-474

AB-474

Arginase 1 (ARG1)

Cancer

Pre-clinical

 

ZB-49-0010

ZB-49-0010

Arginase 2 (ARG2)

Atherosclerosis; Cardiovascular diseases; hypertension

Pre-clinical

 

SCR-6920

SCR-6920

Methylthioadenosine Phosphorylase (MTAP);

protein arginine N-methyltransferase (PRMT);

Protein Arginine Methyltransferase 5 (PRMT5)

Advanced solid tumor; Hematologic tumor; Metastatic non-small cell lung cancer; Non-hodgkin,s lymphoma

Phase 1

NCT05528055

Telaglenastat hydrochloride

CB-839

Glutaminase (GLS)

Cervical cancer

Pre-clinical

 

Sirpiglenastat

DRP-104

Glutaminase (GLS)

Advanced solid tumor; Autoimmune disease; Cancer; AIDS related dementia syndrome; Inflammatory disease; Laryngeal tumor; Lung tumor; Metastatic non-small cell lung cancer; Oral tumor; Throat tumor; Squamous cell carcinoma; Urinary tract tumor

Phase 2

NCT04471415

IPN-60090

IPN-60090

Glutaminase (GLS)

Ovarian tumor

Pre-clinical

 

Macrocyclic glutaminase 1

 

Glutaminase (GLS)

Advanced solid tumor

Pre-clinical

 

BPTES

D-JHU-29

Glutaminase (GLS)

Breast tumor; Hematologic tumor; Pancreatic neoplasm; Rett syndrome

Pre-clinical

 

RP-10107

RP-10107

Glutaminase (GLS)

Solid tumor

Pre-clinical

 

DRP-367

DRP-367

Glutaminase (GLS)

Autoimmune disease; Cancer; Inflammatory disease

Drug discovery

 

Tiptuximab

 

Glutaminase (GLS)

Cancer; Non-small cell lung cancer

Pre-clinical

 

Kidney mitochondrial glutaminase inhibitors

 

Glutaminase (GLS)

Cancer

Pre-clinical

 

Sirpiglenastat

JHU-083

Glutaminase (GLS)

Advanced solid tumor; Autoimmune disease; Cancer; AIDS related dementia syndrome; Inflammatory disease; Laryngeal tumor; Lung tumor; Metastatic non-small cell lung cancer; Oral tumor; Throat tumor; Squamous cell carcinoma; Urinary tract tumor

Phase 2

 

xCT inhibitor

 

Solute Carrier Family 7 Member 11 (SLC7A11)

Ovarian tumor

Pre-clinical

 

Florilglutamic acid (18F)

BAY-94–9392

Solute Carrier Family 7 Member 11 (SLC7A11)

Cancer; Hepatocellular carcinoma

Phase 1

 

DC-10

DC-10

Solute Carrier Family 7 Member 11 (SLC7A11)

Cancer

Pre-clinical

 

AX-09

AX-09

Solute Carrier Family 7 Member 11 (SLC7A11)

Colorectal cancer; Metastatic breast cancer; Non-small cell lung cancer

Pre-clinical

 

xCT-mAb

AbX-09

Solute Carrier Family 7 Member 11 (SLC7A11)

Colorectal cancer; Metastatic breast cancer; Non-small cell lung cancer

Pre-clinical

 

Cysteine/cystine

PR0–071

Solute Carrier Family 7 Member 11 (SLC7A11)

Central nervous system diseases; Impulse control disorder; Obsessive compulsive disorder; Schizophrenia; trichotillomania

Phase 2

 

MEDI-7247

MEDI-7247

Solute Carrier Family 1 Member 5 (SLC1A5)

Hematologic malignancy

Phase 1

NCT03811652; NCT03106428

IDE-397

IDE-397

Methionine Adenosyltransferase 2A (MAT2A)

Advanced solid tumor; Metastatic bladder cancer; Metastatic esophageal carcinoma; Metastatic head and neck cancer; Metastatic non-small cell lung cancer; Metastatic pancreatic cancer; Metastatic gastric cancer

Phase 2

NCT04794699

S-95035

S-95035

Methionine Adenosyltransferase 2A (MAT2A)

Solid tumor

Pre-clinical

 

AG-270

AG-270

Methionine Adenosyltransferase 2A (MAT2A)

Advanced solid tumor; Lymphoma; Metastatic non-small cell lung cancer; Ductal adenocarcinoma of pancreas

Phase 1

NCT03435250

SCR-7952

SCR-7952

Methionine Adenosyltransferase 2A (MAT2A)

Cancer; Solid tumor

Pre-clinical

 

S-095033

S-095033

Methionine Adenosyltransferase 2A (MAT2A)

Metastatic esophageal carcinoma; Squamous cell carcinoma

Pre-clinical

 

Evexomostat

SDX-7320

Methionyl Aminopeptidase 2 (METAP2)

Cancer; Hepatocellular carcinoma; Idiopathic pulmonary fibrosis; Metastatic breast cancer; Metastatic colorectal cancer; Metastatic non-small cell lung cancer; Metastatic prostate cancer; Type 2 diabetes mellitus; Prostatic tumor

Phase 2

NCT05570253; NCT02743637

APL-1202

APL-1202

Methionyl Aminopeptidase 2 (METAP2)

Bladder cancer

Phase 3

NCT04813107; NCT04736394; NCT04601766;

NCT04498702; NCT04490993; NCT03672240

M-8891

M-8891

Methionyl Aminopeptidase 2 (METAP2)

Advanced solid tumor; cancer

Phase 1

 

SDX-7195

SDX-7195

Methionyl Aminopeptidase 2 (METAP2)

Metabolic disorder

Pre-clinical

NCT04073368

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