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

65 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/PCCGIST
Evidence Score
74
FDA Approved1 trial
Bevacizumab

Avastin

established

Anti-VEGF monoclonal antibody

Vegf SignalingHif Pseudohypoxia
PPGL/PCC
Evidence Score
67
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
Olaparib

Lynparza

clinical trial

PARP inhibitor (PARP1/2)

Sdh Driven HrdOxidative Stress Ros
All SDH tumors
Evidence Score
62
FDA Approved1 trial
Cabozantinib

Cabometyx, Cometriq

established

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

Hif Met Axl SignalingVegf Signaling
PPGL/PCCRCC
Evidence Score
62
FDA Approved3 trials
Telaglenastat

CB-839

clinical trial

Glutaminase inhibitor

Glutamine Dependency
All SDH tumors
Evidence Score
60
2 trials
Regorafenib

Stivarga

established

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

Vegf SignalingMtor Pi3k AktFgfr Signaling
GISTRCC
Evidence Score
60
FDA Approved1 trial
Everolimus

Afinitor

clinical trial

mTOR inhibitor (rapalog)

Mtor Pi3k Akt
All SDH tumors
Evidence Score
58
FDA Approved1 trial
Azacitidine

Vidaza

preclinical

DNA methyltransferase inhibitor (hypomethylating agent)

Epigenetic Dysregulation
All SDH tumors
Evidence Score
58
FDA Approved
Pemigatinib

Pemazyre

clinical trial

Selective FGFR1/2/3 inhibitor

Fgfr Signaling
GIST
Evidence Score
58
FDA Approved1 trial
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 Approved1 trial
Yttrium-90 SIRT

SIR-Spheres, TheraSphere

clinical trial

Liver-directed radioembolization

Sdh Driven Hrd
GIST
Evidence Score
43
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 Approved1 trial
Iobenguane I-131 (Azedra, ¹³¹I-MIBG)

Azedra

established

NET-targeted beta-particle radiopharmaceutical (¹³¹I-labeled guanethidine analog)

Mibg Net Targeted RadiationSdh Driven Hrd
PPGL/PCC
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
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
Capivasertib

Truqap

theoretical

Pan-AKT inhibitor (allosteric PH-domain binding; AKT1/2/3)

Mtor Pi3k Akt
PPGL/PCCGISTRCC
Evidence Score
32
FDA Approved1 trial
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
Sapanisertib

TAK-228, MLN0128

theoretical

Dual mTORC1/2 kinase inhibitor (active-site TOR inhibitor)

Mtor Pi3k Akt
All SDH tumors
Evidence Score
31
1 trial
Panobinostat

Farydak

theoretical

Pan-HDAC inhibitor

Epigenetic Dysregulation
All SDH tumors
Evidence Score
30
FDA Approved1 trial
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
ART558
clinical trial

POLQ inhibitor (DNA polymerase theta inhibitor)

Polq Tmej Backup RepairSdh Driven Hrd
All SDH tumors
Evidence Score
30
2 trials
Palbociclib

Ibrance

theoretical

CDK4/6 inhibitor

Cdkn2a Cdk46 Cell CycleEpigenetic Dysregulation
GIST
Evidence Score
30
FDA Approved
CX-5461
preclinical

G-quadruplex DNA stabilizer

G4 Quadruplex Brcas LethalitySdh Driven Hrd
All SDH tumors
Evidence Score
30
2 trials
Elimusertib

AZD7648

theoretical

DNA-PKcs inhibitor (DNA-dependent protein kinase catalytic subunit inhibitor)

Nhej Dnapk Backup RepairSdh Driven Hrd
All SDH tumors
Evidence Score
29
1 trial
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
Prexasertib

LY2606368

theoretical

CHK1/CHK2 kinase inhibitor

Chk1 Brcas Replication CheckpointSdh Driven Hrd
All SDH tumors
Evidence Score
28
2 trials
Peposertib

M3814

theoretical

DNA-PK (DNA-PKcs/PRKDC) inhibitor

Nhej Dnapk Backup RepairSdh Driven Hrd
All SDH tumors
Evidence Score
28
2 trials
Ganetespib

STA-9090

theoretical

HSP90 inhibitor (non-ansamycin resorcinol-based)

Hsp90 Hif Client ChaperoneHif Pseudohypoxia
All SDH tumors
Evidence Score
28
1 trial
KB-0742
theoretical

CDK9 inhibitor (selective P-TEFb kinase inhibitor)

Cdk9 Super Enhancer ElongationEpigenetic Dysregulation
GIST
Evidence Score
27
1 trial
CCS1477

Inobrodib

clinical trial

CBP/p300 bromodomain inhibitor

Cbp P300 Hat CoactivatorEpigenetic Dysregulation
All SDH tumors
Evidence Score
27
2 trials
[²¹¹At]MABG (Alpha-Particle MIBG)
preclinical

NET-targeted alpha-particle radiopharmaceutical (²¹¹At-labeled guanethidine analog)

Mibg Net Targeted RadiationSdh Driven Hrd
PPGL/PCC
Evidence Score
26
RBS2418 (Uzaribat)
theoretical

ENPP1 inhibitor

Cgas Sting Innate ImmuneSdh Driven Hrd
All SDH tumors
Evidence Score
26
1 trial
Alisertib

MLN8237

theoretical

Aurora A kinase inhibitor (selective, oral)

Aurora Kinase Hif FeedbackHif Pseudohypoxia
All SDH tumors
Evidence Score
26
1 trial
Imetelstat

Rytelo

theoretical

Telomerase inhibitor (competitive TERT template antagonist)

Tert Telomerase Reactivation
PPGL/PCC
Evidence Score
25
FDA Approved1 trial
Ulevostinag

MK-1454

theoretical

Cyclic dinucleotide STING agonist (intratumoral)

Cgas Sting Innate ImmuneSdh Driven Hrd
All SDH tumors
Evidence Score
25
1 trial
Eflornithine (DFMO)

Iwilfin, Ornidyl

theoretical

ODC1 inhibitor (ornithine decarboxylase inhibitor / polyamine synthesis inhibitor)

Polyamine Metabolism
PPGL/PCC
Evidence Score
23
FDA Approved
Bempedoic Acid

Nexletol, Nilemdo

theoretical

ACLY inhibitor (ATP-citrate lyase inhibitor, prodrug)

Reductive CarboxylationGlutamine Dependency
All SDH tumors
Evidence Score
23
FDA Approved
Oleclumab

MEDI9447

theoretical

Anti-CD73 monoclonal antibody (immune checkpoint, adenosine pathway)

Hif Cd73 Adenosine ImmunosuppressionHif Pseudohypoxia
All SDH tumors
Evidence Score
23
2 trials
Dichloroacetate (DCA)
theoretical

Pyruvate dehydrogenase kinase inhibitor

Hif PseudohypoxiaOxidative Stress Ros
All SDH tumors
Evidence Score
22
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
Elesclomol

STA-4783

theoretical

Copper ionophore

Cuproptosis Lipoylation MtfasDe Novo Lipogenesis
All SDH tumors
Evidence Score
22
1 trial
Linsitinib

OSI-906

theoretical

IGF1R/IR dual kinase inhibitor

Hif Igf2 Igf1r Growth SignalingHif Pseudohypoxia
PPGL/PCC
Evidence Score
22
2 trials
Etrumadenant

AB928

clinical trial

Adenosine A2A/A2B receptor antagonist (dual; oral small molecule)

Hif Cd73 Adenosine ImmunosuppressionHif Pseudohypoxia
All SDH tumors
Evidence Score
22
2 trials
Magrolimab

Hu5F9-G4

clinical trial

Anti-CD47 monoclonal antibody (macrophage phagocytosis-checkpoint inhibitor)

Hif Cd47 Phagocytosis EvasionHif Pseudohypoxia
All SDH tumors
Evidence Score
22
1 trial
Plerixafor

AMD3100, Mozobil

theoretical

CXCR4 chemokine receptor antagonist

Hif Cxcr4 Chemokine MetastasisHif Pseudohypoxia
PPGL/PCC
Evidence Score
20
FDA Approved1 trial
SLC-0111

WBI-5111

theoretical

Carbonic anhydrase IX/XII inhibitor (CA9/CA12 selective)

Hif Ca9 Ph RegulationHif Pseudohypoxia
All SDH tumors
Evidence Score
20
2 trials
Relatlimab

Opdualag

theoretical

LAG-3 checkpoint inhibitor (anti-LAG-3 monoclonal antibody)

Succinate Lag3 ExhaustionSuccinate Immune Evasion
All SDH tumors
Evidence Score
20
FDA Approved1 trial
Ipilimumab

Yervoy

theoretical

CTLA-4 checkpoint inhibitor (anti-CTLA-4 IgG1κ monoclonal antibody)

Ido1 Kynurenine Treg Ctla4Succinate Immune Evasion
All SDH tumors
Evidence Score
20
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