This tool is for research exploration only. It is not a medical device and does not provide medical advice. Drug candidates, evidence scores, and AI-generated content are not clinical recommendations. Always consult your medical team before making any treatment decisions.

Drug Candidates

38 candidates mapped to SDH-deficient disease pathways

Belzutifan

Welireg

established

HIF-2α inhibitor

Hif Pseudohypoxia
All SDH tumors
Evidence Score
88
FDA Approved1 trial
Sunitinib

Sutent

established

Multi-kinase inhibitor (VEGFR/PDGFR/KIT)

Vegf SignalingMtor Pi3k Akt
GISTPPGL/PCCRCC
Evidence Score
78
FDA Approved1 trial
Rogaratinib
clinical trial

Pan-FGFR inhibitor (FGFR1/2/3/4)

Fgfr SignalingEpigenetic Dysregulation
GIST
Evidence Score
76
1 trial
Temozolomide

Temodar, Temodal

clinical trial

Alkylating agent

Epigenetic Dysregulation
PPGL/PCC
Evidence Score
68
FDA Approved
Bevacizumab

Avastin

established

Anti-VEGF monoclonal antibody

Vegf Signaling
PPGL/PCC
Evidence Score
65
FDA Approved
Tazemetostat

Tazverik

clinical trial

EZH2 inhibitor (PRC2 inhibitor)

Epigenetic Dysregulation
All SDH tumors
Evidence Score
65
FDA Approved1 trial
Decitabine

Dacogen

preclinical

DNA methyltransferase inhibitor (hypomethylating agent)

Epigenetic Dysregulation
All SDH tumors
Evidence Score
62
FDA Approved
Cabozantinib

Cabometyx, Cometriq

established

Multi-kinase inhibitor (VEGFR2/MET/AXL/RET/KIT)

Hif Met Axl SignalingVegf Signaling
PPGL/PCCRCC
Evidence Score
62
FDA Approved2 trials
Telaglenastat

CB-839

clinical trial

Glutaminase inhibitor

Glutamine Dependency
All SDH tumors
Evidence Score
60
2 trials
Azacitidine

Vidaza

preclinical

DNA methyltransferase inhibitor (hypomethylating agent)

Epigenetic Dysregulation
All SDH tumors
Evidence Score
58
FDA Approved
Olaparib

Lynparza

preclinical

PARP inhibitor (PARP1/2)

Sdh Driven HrdOxidative Stress Ros
All SDH tumors
Evidence Score
58
FDA Approved
Regorafenib

Stivarga

established

Multi-kinase inhibitor (VEGFR/KIT/PDGFR/FGFR/RAF)

Vegf SignalingMtor Pi3k Akt
GISTRCC
Evidence Score
58
FDA Approved
Everolimus

Afinitor

preclinical

mTOR inhibitor (rapalog)

Mtor Pi3k Akt
All SDH tumors
Evidence Score
55
FDA Approved
Daporinad (FK866)

APO866

preclinical

NAMPT inhibitor (NAD⁺ biosynthesis inhibitor)

Nad MetabolismOxidative Stress Ros
All SDH tumors
Evidence Score
48
1 trial
Niraparib

Zejula

theoretical

PARP inhibitor (PARP1/2)

Sdh Driven Hrd
All SDH tumors
Evidence Score
45
FDA Approved
Hydroxychloroquine

Plaquenil

preclinical

Autophagy inhibitor (lysosomal alkalinizer)

Autophagy Survival
All SDH tumors
Evidence Score
42
FDA Approved2 trials
Erdafitinib

Balversa

theoretical

Pan-FGFR inhibitor (FGFR1/2/3/4), FDA-approved

Fgfr Signaling
GIST
Evidence Score
42
FDA Approved
Metformin

Glucophage

preclinical

Biguanide / AMPK activator

Mtor Pi3k AktOxidative Stress Ros
All SDH tumors
Evidence Score
40
FDA Approved
DENSPM (N1,N11-Diethylnorspermine)
preclinical

Polyamine analogue / SSAT inducer

Polyamine MetabolismOxidative Stress Ros
PPGL/PCC
Evidence Score
38
Dichloroacetate (DCA)
preclinical

Pyruvate dehydrogenase kinase inhibitor

Hif PseudohypoxiaOxidative Stress Ros
All SDH tumors
Evidence Score
35
Denifanstat

TVB-2640

preclinical

FASN inhibitor (fatty acid synthase inhibitor)

De Novo LipogenesisGlutamine Dependency
All SDH tumors
Evidence Score
35
2 trials
Lutetium-177 DOTATATE

Lutathera

established

Peptide receptor radionuclide therapy (PRRT) / SSTR2-targeted radioligand therapy

Sstr2 Somatostatin VulnerabilitySdh Driven Hrd
PPGL/PCC
Evidence Score
35
FDA Approved3 trials
Birabresib

OTX015, MK-8628

theoretical

BET bromodomain inhibitor (pan-BRD2/3/4)

Epigenetic Dysregulation
GIST
Evidence Score
33
1 trial
Ceralasertib

AZD6738

preclinical

ATR kinase inhibitor

Atrx Alt Replication Stress
PPGL (ATRX+)
Evidence Score
33
1 trial
Pembrolizumab

Keytruda

theoretical

Anti-PD-1 monoclonal antibody (checkpoint inhibitor)

Hif Pdl1 Checkpoint EvasionSuccinate Immune Evasion
All SDH tumors
Evidence Score
32
FDA Approved2 trials
Ym155 (Sepantronium Bromide)

YM155, Sepantronium

preclinical

Survivin (BIRC5) transcription inhibitor / IAP inhibitor

Hif Driven Survivin ApoptosisSdh Driven Hrd
All SDH tumors
Evidence Score
31
1 trial
Panobinostat

Farydak

theoretical

Pan-HDAC inhibitor

Epigenetic Dysregulation
All SDH tumors
Evidence Score
30
FDA Approved
AZD3965
preclinical

MCT1 inhibitor (monocarboxylate transporter 1 inhibitor, SLC16A1)

Succinate Immune Evasion
PPGL/PCC
Evidence Score
30
1 trial
Pevonedistat

TAK-924

preclinical

NAE inhibitor (neddylation inhibitor, NEDD8-activating enzyme inhibitor)

Neddylation
PPGL/PCC
Evidence Score
30
Palbociclib

Ibrance

theoretical

CDK4/6 inhibitor

Cdkn2a Cdk46 Cell CycleEpigenetic Dysregulation
GIST
Evidence Score
30
FDA Approved
Enasidenib

Idhifa

theoretical

IDH2 inhibitor

Epigenetic Dysregulation
All SDH tumors
Evidence Score
28
FDA Approved
Brequinar

DUP-785, NSC 368390

theoretical

DHODH inhibitor (dihydroorotate dehydrogenase inhibitor)

Pyrimidine Synthesis Vulnerability
All SDH tumors
Evidence Score
28
1 trial
[212Pb]VMT-α-NET
clinical trial

Alpha-particle SSTR2-targeted radioligand therapy (RLT)

Sstr2 Somatostatin VulnerabilitySdh Driven Hrd
PPGL/PCC
Evidence Score
28
2 trials
ART558
theoretical

POLQ inhibitor (DNA polymerase theta inhibitor)

Polq Tmej Backup RepairSdh Driven Hrd
All SDH tumors
Evidence Score
27
Eflornithine (DFMO)

Iwilfin, Ornidyl

theoretical

ODC1 inhibitor (ornithine decarboxylase inhibitor / polyamine synthesis inhibitor)

Polyamine Metabolism
PPGL/PCC
Evidence Score
23
FDA Approved
Epacadostat

INCB024360

theoretical

IDO1 inhibitor (indoleamine 2,3-dioxygenase 1 inhibitor)

Succinate Immune Evasion
PPGL/PCC
Evidence Score
22
1 trial
Teriflunomide

Aubagio

theoretical

DHODH inhibitor (active metabolite of leflunomide; FDA-approved)

Pyrimidine Synthesis Vulnerability
All SDH tumors
Evidence Score
22
FDA Approved1 trial
Ascorbic Acid (High-dose IV)
theoretical

TET enzyme cofactor / α-KG-dependent dioxygenase activator

Epigenetic DysregulationOxidative Stress Ros
PPGL/PCC
Evidence Score
18