Biomarker & Intervention Discovery

Chronic Lymphocytic Leukaemia (CLL)

This page lists 11 druggable gene targets, 47 approved or trial-tested drug candidates and 15 recent clinical trials for Chronic Lymphocytic Leukaemia (CLL), with each agent ranked by the strength of its evidence (clinical, mechanistic or correlative). Candidates include existing drugs that could be repurposed. Sources are linked so you can check every claim.

Spontaneous remissionEarly-stage (Rai 0-I): watch-and-wait appropriate in 50-70%; spontaneous remission extremely rare; median time to…
Best-intervention remissionVenetoclax + obinutuzumab (CLL14): 2-year fixed duration, MRD-undetectable remission in 36%, PFS benefit sustained at…
Gap sizeLarge — chlorambucil still used in some elderly patients; BTK inhibitor choice and venetoclax combination timing are…
Primary barrierpractice/access
Full remission profile →

Remission & chronicity

Spontaneous remission
Early-stage (Rai 0-I): watch-and-wait appropriate in 50-70%; spontaneous remission extremely rare; median time to treatment in Rai Stage 0: 10+ years; Richter's transformation (5-10%) extremely poor prognosis
Best-intervention remission
Venetoclax + obinutuzumab (CLL14): 2-year fixed duration, MRD-undetectable remission in 36%, PFS benefit sustained at 5 years vs chlorambucil; ibrutinib continuous: PFS benefit but not curative; BRUIN CLL trial (pirtobrutinib): 73% ORR in ibrutinib-resistant CLL
Gap size
Large — chlorambucil still used in some elderly patients; BTK inhibitor choice and venetoclax combination timing are practice-variable
Primary barrier
practice/access
Chemoimmunotherapy (FCR, BR) largely replaced by BTK inhibitors and venetoclax but chlorambucil still used where cost is a barrier; the 'best drug at first relapse' question is evolving rapidly; CAR-T not yet standard for CLL

Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: Clinical > Mechanistic > Correlative.

ATM 281BCL2 214BTK 254CD19 40CD38 203CD52 4MS4A1 28NOTCH1 122PIK3CD 302TP53 652ZAP70 192
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ETOPOSIDE ? unclear Mechanistic
CP466722 ↓ inhibits Mechanistic
VELIPARIB ? unclear Mechanistic
CISPLATIN ? unclear Mechanistic
OLAPARIB ? unclear Mechanistic
EVEROLIMUS ? unclear Mechanistic
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic
TEMOZOLOMIDE ? unclear Mechanistic
NIRAPARIB ? unclear Mechanistic
AZD1390 ↓ inhibits Mechanistic
SCHISANHENOL ? unclear Mechanistic
INDISULAM ? unclear Mechanistic
PARP/TANKYRASE INHIBITOR 2X-121 ? unclear Mechanistic
XRD-0394 ↓ inhibits Mechanistic
DIPYRIDAMOLE ? unclear Mechanistic
ALNODESERTIB ? unclear Mechanistic
PHLORETIN ? unclear Mechanistic
DURVALUMAB ? unclear Mechanistic
IBRUTINIB ? unclear Mechanistic
LARTESERTIB ↓ inhibits Mechanistic
CERALASERTIB ? unclear Mechanistic
ENPIROLINE ? unclear Mechanistic
ENZALUTAMIDE ? unclear Mechanistic
VALPROIC ACID ? unclear Mechanistic
IRINOTECAN HYDROCHLORIDE ? unclear Mechanistic
FLUOROURACIL ? unclear Mechanistic
A011 ↓ inhibits Mechanistic
VANDETANIB ? unclear Mechanistic
DITHIAZANINE IODIDE ? unclear Mechanistic
CHEMBL:CHEMBL374632 ? unclear Mechanistic
BENDAMUSTINE ? unclear Mechanistic
AZD0156 ↓ inhibits Mechanistic
PJ34 ? unclear Mechanistic
KU-0060648 ? unclear Mechanistic
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic
TOLONIUM CHLORIDE ? unclear Mechanistic
PEGYLATED SN-38 CONJUGATE PLX038 ? unclear Mechanistic
IPILIMUMAB ? unclear Mechanistic
AVELUMAB ? unclear Mechanistic
OXALIPLATIN ? unclear Mechanistic

Showing top 40 of 281 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
NONSTEROIDAL ANTIINFLAMMATORY DRUG ? unclear Mechanistic
ENDOTHELIN RECEPTOR ANTAGONIST ? unclear Mechanistic
BORTEZOMIB ? unclear Mechanistic
DOXORUBICIN HYDROCHLORIDE ? unclear Mechanistic
MITOXANTRONE HYDROCHLORIDE ? unclear Mechanistic
VENETOCLAX ↓ inhibits Mechanistic
NAVITOCLAX ↓ inhibits Mechanistic
ISOTRETINOIN ? unclear Mechanistic
BRENTUXIMAB VEDOTIN ? unclear Mechanistic
OXALIPLATIN ? unclear Mechanistic
TAZAROTENE ? unclear Mechanistic
LISAFTOCLAX ↓ inhibits Mechanistic
GOSSYPOL ? unclear Mechanistic 320.0 nM (Ki)
EDARAVONE ? unclear Mechanistic
TENIPOSIDE ? unclear Mechanistic
DITIOCARB ? unclear Mechanistic
CARBOPLATIN ? unclear Mechanistic
CILENGITIDE ? unclear Mechanistic
ESTRAMUSTINE ? unclear Mechanistic
RECOMBINANT INTERFERON ? unclear Mechanistic
CISPLATIN ? unclear Mechanistic
AZD4320 ↓ inhibits Mechanistic
PELCITOCLAX ? unclear Mechanistic
ORAL CONTRACEPTIVE ? unclear Mechanistic
ANG1005 ? unclear Mechanistic
OBATOCLAX ↓ inhibits Mechanistic
ANTI-CD19 CAR T CELLS AT101 ↓ inhibits Mechanistic
FOSBRETABULIN DISODIUM ? unclear Mechanistic
APG-1252-M1 ↓ inhibits Mechanistic
SONROTOCLAX ↓ inhibits Mechanistic
DULANERMIN ? unclear Mechanistic
RALTITREXED ? unclear Mechanistic
EPIRUBICIN HYDROCHLORIDE ? unclear Mechanistic
ETOPOSIDE ? unclear Mechanistic
MULTIDRUG RESISTANCE MODULATOR ? unclear Mechanistic
BCL-2 INHIBITOR BCL201 ? unclear Mechanistic
PROTUBOXEPIN A ? unclear Mechanistic
RIBAVIRIN ? unclear Mechanistic
DIETHYLSTILBESTROL ? unclear Mechanistic
APRINOCARSEN ? unclear Mechanistic

Showing top 40 of 214 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PRN694 ↓ inhibits Mechanistic
RN486 ↓ inhibits Mechanistic
PD166285 ? unclear Mechanistic
PTD10 ↓ inhibits Mechanistic
BRANEBRUTINIB ↓ inhibits Mechanistic
ACALABRUTINIB ↓ inhibits Mechanistic
COMPOUND 71 [WO2018191577A1] ↓ inhibits Mechanistic
COMPOUND 25 [PMID: 31260299] ↓ inhibits Mechanistic
COMPOUND 38 [PMID: 24915291] ↓ inhibits Mechanistic
IBRUTINIB ↓ inhibits Mechanistic 0.5 nM (IC50)
ZM447439 ? unclear Mechanistic
BTK INHIBITOR TL-895 ? unclear Mechanistic
ZYBT1 ↓ inhibits Mechanistic
EVOBRUTINIB ↓ inhibits Mechanistic
FENEBRUTINIB ↓ inhibits Mechanistic
TOLEBRUTINIB ↓ inhibits Mechanistic
RILZABRUTINIB ? unclear Mechanistic
CGI1746 ↓ inhibits Mechanistic
TAMATINIB ? unclear Mechanistic
ZANUBRUTINIB ↓ inhibits Mechanistic
ENTRECTINIB ? unclear Mechanistic 158.49 nM (Ki)
LUXEPTINIB ↓ inhibits Mechanistic
BTK INHIBITOR 16 [PMID: 30122225] ↓ inhibits Mechanistic
ZELEBRUDOMIDE ? unclear Mechanistic
BIIB068 ↓ inhibits Mechanistic
PROTAC 23 [PMID: 38321747] ↓ inhibits Mechanistic
NVP-TAE684 ? unclear Mechanistic
COMPOUND 36 [PMID: 21958547] ↓ inhibits Mechanistic
CINSEBRUTINIB ↓ inhibits Mechanistic
COMPOUND 9 [PMID: 26006010] ↓ inhibits Mechanistic
DASATINIB ANHYDROUS ? unclear Mechanistic
NEMTABRUTINIB ? unclear Mechanistic
JNJ-64264681 ↓ inhibits Mechanistic
SPEBRUTINIB ↓ inhibits Mechanistic
CHEMBL:CHEMBL1997335 ? unclear Mechanistic
RITLECITINIB ↓ inhibits Mechanistic
BIRELENTINIB ↓ inhibits Mechanistic
PF-562271 ? unclear Mechanistic
ORELABRUTINIB ↓ inhibits Mechanistic
MIDOBRUTINIB ↓ inhibits Mechanistic

Showing top 40 of 254 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
AUTOLOGOUS ANTI-CD19 CAR T-CELLS IM19 ? unclear Mechanistic
BLINATUMOMAB ? unclear Mechanistic
LONCASTUXIMAB TESIRINE ~ modulates Mechanistic
AUTOLOGOUS CD19CAR-CD28-CD137/CD27/CD3ZETA-ICASP9-EXPRESSING T-LYMPHOCYTES ? unclear Mechanistic
TISAGENLECLEUCEL ? unclear Mechanistic
AUTOLOGOUS ANTI-CD19 CHIMERIC ANTIGEN RECEPTOR T-CELLS C-CAR011 ? unclear Mechanistic
VADACABTAGENE LERALEUCEL ? unclear Mechanistic
LISOCABTAGENE MARALEUCEL ? unclear Mechanistic
AUTOLOGOUS ANTI-CD19CAR-4-1BB-CD3ZETA-EGFRT-EXPRESSING T LYMPHOCYTES ? unclear Mechanistic
MDX-1342 ~ modulates Mechanistic
AXICABTAGENE CILOLEUCEL ? unclear Mechanistic
ANTI-CD19 CAR T CELLS PREPARATION ? unclear Mechanistic
INEBILIZUMAB ? unclear Mechanistic
ALLOGENEIC ANTI-CD19-CAR-CD28-IL-15-EXPRESSING NATURAL KILLER T-CELLS ? unclear Mechanistic
AZERCABTAGENE ZAPRELEUCEL ? unclear Mechanistic
TAFASITAMAB ? unclear Mechanistic
DENINTUZUMAB MAFODOTIN ~ modulates Mechanistic
COLTUXIMAB RAVTANSINE ? unclear Mechanistic
OBEXELIMAB ↓ inhibits Mechanistic
AMG-729 ~ modulates Mechanistic
CD19CAR-CD3ZETA-4-1BB-EXPRESSING ALLOGENEIC T-LYMPHOCYTE CELLS ? unclear Mechanistic
AUTOLOGOUS ANTI-CD19 CAR-T CELLS TBI-1501 ? unclear Mechanistic
AUTOLOGOUS CD19 CAR-EXPRESSING CD4+/CD8+ T-CELLS MB-CART19.1 ? unclear Mechanistic
AFM-11 ? unclear Mechanistic
BREXUCABTAGENE AUTOLEUCEL ~ modulates Mechanistic
TAPLITUMOMAB PAPTOX ~ modulates Mechanistic
ANTI-CD19 CAR T-CELLS XLCART001 ? unclear Mechanistic
AUTOLOGOUS ANTI-CD19 CAR-EXPRESSING T-LYMPHOCYTES CLIC-1901 ? unclear Mechanistic
DENINTUZUMAB ~ modulates Mechanistic
AUTO3 ? unclear Mechanistic
DEGLYCOSYLATED RICIN A CHAIN-CONJUGATED ANTI-CD19/ANTI-CD22 IMMUNOTOXINS ? unclear Mechanistic
DUVORTUXIZUMAB ? unclear Mechanistic
ANTI-CD19 CAR-T CELLS ? unclear Mechanistic
AUTOLOGOUS ANTI-CD19 CHIMERIC ANTIGEN RECEPTOR T-CELLS SJCAR19 ? unclear Mechanistic
ONCOLYSIN B ~ modulates Mechanistic
ANTI-CD20 CAR T CELLS PREPARATION ? unclear Mechanistic
CD19-TARGETED CAR T2 CELLS ? unclear Mechanistic
OBECABTAGENE AUTOLEUCEL ~ modulates Mechanistic
JCAR014 ? unclear Mechanistic
RELMACABTAGENE AUTOLEUCEL ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ANTI-CD38 CAR T CELLS PREPARATION ? unclear Mechanistic
MEZAGITAMAB ~ modulates Mechanistic
THROMBIN ALFA ? unclear Mechanistic
ISATUXIMAB ? unclear Mechanistic
DARATUMUMAB ↓ inhibits Mechanistic
HUMAX-IL15 ↓ inhibits Mechanistic
MK-0159 ↓ inhibits Mechanistic
FELZARTAMAB ↓ inhibits Mechanistic
KUROMANIN ↓ inhibits Mechanistic 6300.0 nM (IC50)
DELPHINIDIN ↓ inhibits Mechanistic 14600.0 nM (IC50)
PELARGONIDIN ↓ inhibits Mechanistic 16300.0 nM (IC50)
MALVIDIN ↓ inhibits Mechanistic 17000.0 nM (IC50)
QUERCETAGETINIDIN ↓ inhibits Mechanistic 14200.0 nM (IC50)
PEONIDIN ↓ inhibits Mechanistic 20900.0 nM (IC50)
CYANIDIN ↓ inhibits Mechanistic 21800.0 nM (IC50)
DIOSMETINIDIN ↓ inhibits Mechanistic 32100.0 nM (IC50)
PETUNIDIN ↓ inhibits Mechanistic 39200.0 nM (IC50)
FISETINIDIN ↓ inhibits Mechanistic 70300.0 nM (IC50)
LUTEOLIN ↓ inhibits Mechanistic 8200.0 nM (IC50)
MYRICETIN ↓ inhibits Mechanistic 24800.0 nM (IC50)
QUERCETIN ↓ inhibits Mechanistic 37900.0 nM (IC50)
ROBINETIN ↓ inhibits Mechanistic 37900.0 nM (IC50)
QUERCETAGETIN ↓ inhibits Mechanistic 48600.0 nM (IC50)
LUTEOLINIDIN ↓ inhibits Mechanistic 6000.0 nM (IC50)
CHEMBL3342444 ↓ inhibits Mechanistic 3300.0 nM (IC50)
CHEMBL3342440 ↓ inhibits Mechanistic 6400.0 nM (IC50)
CHEMBL3342442 ↓ inhibits Mechanistic 33000.0 nM (IC50)
CHEMBL3342443 ↓ inhibits Mechanistic 27000.0 nM (IC50)
CHEMBL3422364 ↓ inhibits Mechanistic 14000.0 nM (IC50)
CHEMBL3422366 ↓ inhibits Mechanistic 26000.0 nM (IC50)
CHEMBL3426029 ↓ inhibits Mechanistic 55.0 nM (IC50)
CHEMBL3426030 ↓ inhibits Mechanistic 89.0 nM (IC50)
CHEMBL3426031 ↓ inhibits Mechanistic 52.0 nM (IC50)
CHEMBL3426032 ↓ inhibits Mechanistic 6.0 nM (IC50)
CHEMBL3426033 ↓ inhibits Mechanistic 3.9 nM (IC50)
CHEMBL3426034 ↓ inhibits Mechanistic 7.3 nM (IC50)
CHEMBL3426035 ↓ inhibits Mechanistic 10.0 nM (IC50)
CHEMBL3426036 ↓ inhibits Mechanistic 54.0 nM (IC50)
CHEMBL3426037 ↓ inhibits Mechanistic 3.8 nM (IC50)
CHEMBL3426038 ↓ inhibits Mechanistic 13.0 nM (IC50)

Showing top 40 of 203 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
ALEMTUZUMAB ↓ inhibits Mechanistic
ALEMTUZUMAB BETA ~ modulates Mechanistic
GATRALIMAB ? unclear Mechanistic
THERAPEUTIC ANDROGEN ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
RITUXIMAB ~ modulates Mechanistic
UBLITUXIMAB ~ modulates Mechanistic
VELTUZUMAB ? unclear Mechanistic
OFATUMUMAB ~ modulates Mechanistic
OBINUTUZUMAB ~ modulates Mechanistic
OCARATUZUMAB ~ modulates Mechanistic
TOSITUMOMAB ~ modulates Mechanistic
TISAGENLECLEUCEL ? unclear Mechanistic
IODINE-131-TOSITUMOMAB ? unclear Mechanistic
EPCORITAMAB ? unclear Mechanistic
GLOFITAMAB ~ modulates Mechanistic
TRU-015 ~ modulates Mechanistic
ODRONEXTAMAB ? unclear Mechanistic
OCRELIZUMAB ~ modulates Mechanistic
ANTI-CD20 CAR T CELLS PREPARATION ? unclear Mechanistic
DI-LEU16-IL2 ? unclear Mechanistic
MONOCLONAL ANTIBODY 1F5 ? unclear Mechanistic
MT-3724 ~ modulates Mechanistic
MOSUNETUZUMAB ? unclear Mechanistic
PLAMOTAMAB ? unclear Mechanistic
AUTOLOGOUS ANTI-CD20 CAR TRANSDUCED CD4/CD8 ENRICHED T-CELLS MB-CART20.1 ? unclear Mechanistic
FBT-A05 ~ modulates Mechanistic
YTTRIUM Y 90 IBRITUMOMAB TIUXETAN ~ modulates Mechanistic
BSAB (CD89-CD20) ? unclear Mechanistic
LFB-R603 ~ modulates Mechanistic
DIVOZILIMAB ? unclear Mechanistic
IBRITUMOMAB TIUXETAN ? unclear Mechanistic
TOSITUMOMAB 131I ~ modulates Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
CRENIGACESTAT ? unclear Mechanistic
NIROGACESTAT ? unclear Mechanistic
BRONTICTUZUMAB ↓ inhibits Mechanistic
IMR-1 ↓ inhibits Mechanistic
MK-0752 ? unclear Mechanistic
ENOTICUMAB ? unclear Mechanistic
HYDROCORTISONE BUTYRATE ? unclear Mechanistic
NOTCH SIGNALING PATHWAY INHIBITOR MK0752 ? unclear Mechanistic
RO4929097 ↓ inhibits Mechanistic
JAGGED2 ↑ activates Mechanistic
CHEMBL3410170 ↓ inhibits Mechanistic 2.0 nM (IC50)
CHEMBL3410169 ↓ inhibits Mechanistic 1.0 nM (IC50)
CHEMBL3410168 ↓ inhibits Mechanistic 2.0 nM (IC50)
CHEMBL3410167 ↓ inhibits Mechanistic 2.0 nM (IC50)
CHEMBL3410166 ↓ inhibits Mechanistic 9.0 nM (IC50)
CHEMBL3410165 ↓ inhibits Mechanistic 2060.0 nM (IC50)
CHEMBL3410164 ↓ inhibits Mechanistic 3.0 nM (IC50)
CHEMBL3410163 ↓ inhibits Mechanistic 1.0 nM (IC50)
CHEMBL3410162 ↓ inhibits Mechanistic 3.0 nM (IC50)
CHEMBL3410161 ↓ inhibits Mechanistic 4.0 nM (IC50)
CHEMBL3410160 ↓ inhibits Mechanistic 3.0 nM (IC50)
CHEMBL3410159 ↓ inhibits Mechanistic 54.0 nM (IC50)
CHEMBL3410158 ↓ inhibits Mechanistic 169.0 nM (IC50)
CHEMBL3410157 ↓ inhibits Mechanistic 32.0 nM (IC50)
CHEMBL3410156 ↓ inhibits Mechanistic 231.0 nM (IC50)
CHEMBL3410155 ↓ inhibits Mechanistic 1190.0 nM (IC50)
CHEMBL3410154 ↓ inhibits Mechanistic 13.0 nM (IC50)
CHEMBL3410153 ↓ inhibits Mechanistic 1.0 nM (IC50)
CHEMBL3410152 ↓ inhibits Mechanistic 3.0 nM (IC50)
CHEMBL3410151 ↓ inhibits Mechanistic 2.0 nM (IC50)
VAREGACESTAT ↓ inhibits Mechanistic 7.8 nM (IC50)
CHEMBL3984405 ↓ inhibits Mechanistic 4.9 nM (IC50)
CHEMBL3896661 ↓ inhibits Mechanistic 1.8 nM (IC50)
CHEMBL3893305 ↓ inhibits Mechanistic 8.5 nM (IC50)
CHEMBL3915833 ↓ inhibits Mechanistic 2.3 nM (IC50)
CHEMBL3985584 ↓ inhibits Mechanistic 7.2 nM (IC50)
CHEMBL3891599 ↓ inhibits Mechanistic 4.4 nM (IC50)
CHEMBL3977486 ↓ inhibits Mechanistic 6.0 nM (IC50)
CHEMBL3913200 ↓ inhibits Mechanistic 3.0 nM (IC50)
CHEMBL3944645 ↓ inhibits Mechanistic 2.5 nM (IC50)

Showing top 40 of 122 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PF-04691502 ↓ inhibits Mechanistic
COMPOUND 27 [PMID: 35834807] ↓ inhibits Mechanistic
AMDIZALISIB ↓ inhibits Mechanistic
SAMOTOLISIB ↓ inhibits Mechanistic
GEDATOLISIB ↓ inhibits Mechanistic 6.0 nM (IC50)
FIMEPINOSTAT ↓ inhibits Mechanistic
PICTILISIB ↓ inhibits Mechanistic 3.0 nM (IC50)
DEZAPELISIB ↓ inhibits Mechanistic
BGT-226 ↓ inhibits Mechanistic
OMIPALISIB ↓ inhibits Mechanistic
DACTOLISIB ↓ inhibits Mechanistic
DUVELISIB ↓ inhibits Mechanistic
LENIOLISIB ↓ inhibits Mechanistic
RECILISIB ↓ inhibits Mechanistic
STK16-IN-1 ↓ inhibits Mechanistic
LENIOLISIB PHOSPHATE ↓ inhibits Mechanistic
AMG-319 ↓ inhibits Mechanistic
TGX-221 ↓ inhibits Mechanistic
SF1126 ↓ inhibits Mechanistic
PANULISIB ↓ inhibits Mechanistic
GSK2636771 ↓ inhibits Mechanistic
IDELALISIB ↓ inhibits Mechanistic
NVP-CLR457 ↓ inhibits Mechanistic
VOXTALISIB ↓ inhibits Mechanistic
UMBRALISIB TOSYLATE ↓ inhibits Mechanistic
WORTMANNIN ↓ inhibits Mechanistic 9.0 nM (IC50)
IZORLISIB ↓ inhibits Mechanistic
PWT-33587 ↓ inhibits Mechanistic
CH-5132799 ? unclear Mechanistic
AZD8186 ↓ inhibits Mechanistic
PAXALISIB ↓ inhibits Mechanistic
APITOLISIB ↓ inhibits Mechanistic
COMPOUND 15A [PMID: 32069401] ↓ inhibits Mechanistic
5-QUINOXALIN-6-YLMETHYLENE-THIAZOLIDINE-2,4-DIONE ↓ inhibits Mechanistic
BUPARLISIB ↓ inhibits Mechanistic
NEMIRALISIB ↓ inhibits Mechanistic
PUQUITINIB MESYLATE DIHYDRATE ↓ inhibits Mechanistic
AMG319 ↓ inhibits Mechanistic
TASELISIB ↓ inhibits Mechanistic
PARSACLISIB ↓ inhibits Mechanistic

Showing top 40 of 302 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PIRARUBICIN ? unclear Mechanistic
OXALIPLATIN ? unclear Mechanistic
SIREMADLIN ↓ inhibits Mechanistic
CARBOPLATIN ? unclear Mechanistic
DAUNORUBICIN HYDROCHLORIDE ? unclear Mechanistic
CHEMBL:CHEMBL260560 ? unclear Mechanistic
MELPHALAN ? unclear Mechanistic
LAPACHONE ? unclear Mechanistic
VITAMIN K3 ? unclear Mechanistic
CHEMBL:CHEMBL260451 ? unclear Mechanistic
DINOSEB ? unclear Mechanistic
RIMCAZOLE ? unclear Mechanistic
TRACAZOLATE ? unclear Mechanistic
CAPIVASERTIB ? unclear Mechanistic
AZD7648 ? unclear Mechanistic
QUINALIZARIN ? unclear Mechanistic
INOSITOL ? unclear Mechanistic
ECONAZOLE NITRATE ? unclear Mechanistic
L-733,060 ? unclear Mechanistic
U50488 ? unclear Mechanistic
CHEMBL:CHEMBL260028 ? unclear Mechanistic
CHEMBL:CHEMBL601140 ? unclear Mechanistic
CHEMBL:CHEMBL408563 ? unclear Mechanistic
ENALAPRIL MALEATE ? unclear Mechanistic
HOMIDIUM BROMIDE ? unclear Mechanistic
IOXYNIL ? unclear Mechanistic
ACALABRUTINIB ? unclear Mechanistic
INDOPROFEN ? unclear Mechanistic
MERCAPTOPURINE ? unclear Mechanistic
AUTOLOGOUS DENDRITIC CELLS TRANSDUCED WITH WILD-TYPE P53 ADENOVIRUS VACCINE ? unclear Mechanistic
ZIRAM ? unclear Mechanistic
IFOSFAMIDE ? unclear Mechanistic
DOCETAXEL ANHYDROUS ? unclear Mechanistic
SOTULETINIB ? unclear Mechanistic
SERTRALINE HYDROCHLORIDE ? unclear Mechanistic
PD166285 ? unclear Mechanistic
SAPANISERTIB ? unclear Mechanistic
ENZALUTAMIDE ? unclear Mechanistic
PYRITHIONE ZINC ? unclear Mechanistic
CHEMBL:CHEMBL1169388 ? unclear Mechanistic

Showing top 40 of 652 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
SYK INHIBITOR II ↓ inhibits Mechanistic
TRIDOLGOSIR ? unclear Mechanistic
MIVAVOTINIB ↓ inhibits Mechanistic
HYDROGEN PEROXIDE ? unclear Mechanistic
ALOISINE ↓ inhibits Mechanistic
CHEMBL443592 ↓ inhibits Mechanistic 250.0 nM (IC50)
CHEMBL386787 ↓ inhibits Mechanistic 320.0 nM (IC50)
CHEMBL439120 ↓ inhibits Mechanistic 30.0 nM (IC50)
CHEMBL99125 ↓ inhibits Mechanistic 1900.0 nM (IC50)
CHEMBL319210 ↓ inhibits Mechanistic 3200.0 nM (IC50)
CHEMBL113185 ↓ inhibits Mechanistic 125.0 nM (IC50)
CHEMBL419757 ↓ inhibits Mechanistic 188.0 nM (IC50)
CHEMBL420672 ↓ inhibits Mechanistic 46.0 nM (IC50)
CHEMBL112172 ↓ inhibits Mechanistic 31.0 nM (IC50)
CHEMBL112346 ↓ inhibits Mechanistic 8.0 nM (IC50)
CHEMBL322066 ↓ inhibits Mechanistic 54.0 nM (IC50)
CHEMBL112518 ↓ inhibits Mechanistic 26.0 nM (IC50)
CHEMBL324576 ↓ inhibits Mechanistic 424.0 nM (IC50)
CHEMBL325912 ↓ inhibits Mechanistic 11.0 nM (IC50)
CHEMBL112561 ↓ inhibits Mechanistic 332.0 nM (IC50)
CHEMBL112263 ↓ inhibits Mechanistic 1900.0 nM (IC50)
CHEMBL323755 ↓ inhibits Mechanistic 396.0 nM (IC50)
CHEMBL112623 ↓ inhibits Mechanistic 4300.0 nM (IC50)
CHEMBL102765 ↓ inhibits Mechanistic 18000.0 nM (IC50)
CHEMBL3706656 ↓ inhibits Mechanistic 30000.0 nM (IC50)
CHEMBL319179 ↓ inhibits Mechanistic 40000.0 nM (IC50)
CHEMBL318300 ↓ inhibits Mechanistic 7000.0 nM (IC50)
CHEMBL102766 ↓ inhibits Mechanistic 7000.0 nM (IC50)
CHEMBL103838 ↓ inhibits Mechanistic 6000.0 nM (IC50)
CHEMBL419539 ↓ inhibits Mechanistic 22000.0 nM (IC50)
CHEMBL320372 ↓ inhibits Mechanistic 19000.0 nM (IC50)
CHEMBL316967 ↓ inhibits Mechanistic 4000.0 nM (IC50)
CHEMBL423330 ↓ inhibits Mechanistic 94000.0 nM (IC50)
CHEMBL132006 ↓ inhibits Mechanistic 4000.0 nM (IC50)
CHEMBL411163 ↓ inhibits Mechanistic 38.0 nM (IC50)
CHEMBL130106 ↓ inhibits Mechanistic 96000.0 nM (IC50)
CHEMBL133511 ↓ inhibits Mechanistic 72000.0 nM (IC50)
CHEMBL129842 ↓ inhibits Mechanistic 61000.0 nM (IC50)
CHEMBL134030 ↓ inhibits Mechanistic 31000.0 nM (IC50)
CHEMBL130931 ↓ inhibits Mechanistic 3000.0 nM (IC50)

Showing top 40 of 192 agents ranked by evidence tier.


Clinical Trials

Drug, biologic and supplement trials (treatment or prevention) from ClinicalTrials.gov whose main condition is Chronic Lymphocytic Leukaemia (CLL): recruiting, active or completed, most recently updated first. Data retrieved 2026-10-28.

NCT06564038 Phase: 1/2 Started: 2025-01-30

The purpose of this study is to assess the safety and efficacy of surovatamig (formerly AZD0486) administered as monotherapy or in combination with other anticancer agents in participants with hematological malignancies

NCT03534323 Phase: 1/2 Started: 2018-07-12

This research study is assessing a new drug, duvelisib, in combination with a drug that is already FDA approved, venetoclax, as a possible treatment for participants with CLL or those with Richter's Syndrome

NCT04269902 Phase: 3 Started: 2021-03-02

This phase III trial compares early treatment with venetoclax and obinutuzumab versus delayed treatment with venetoclax and obinutuzumab in patients with newly diagnosed high-risk chronic lymphocytic leukemia or small lymphocytic lymphoma. Venetoclax is in a class of medications called B-cell lympho...

NCT06697184 Phase: 1/2 Started: 2025-01-23

The purpose of this study is to establish the safety of novel dosing and ramp-up schedules for sonrotoclax in participants with hematological malignancies.

NCT03737981 Phase: 3 Started: 2019-01-31

This phase III trial compares adding a new anti-cancer drug (venetoclax) to the usual treatment (ibrutinib plus obinutuzumab) in older patients with chronic lymphocytic leukemia who have not received previous treatment. The addition of venetoclax to the usual treatment might prevent chronic lymphocy...

NCT03516617 Phase: 2 Started: 2018-09-10

This phase II trials studies how well acalabrutinib with or without obinutuzumab works in treating patients with early-stage chronic lymphocytic leukemia or small lymphocytic lymphoma. Acalabrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Immunothe...

NCT03701282 Phase: 3 Started: 2019-02-12

This phase III trial studies how well ibrutinib and obinutuzumab with or without venetoclax work in treating patients with chronic lymphocytic leukemia. Ibrutinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Obinutuzumab is a monoclonal antibody. It bin...

NCT06943872 Phase: 3 Started: 2025-06-11

The goal of this study is to compare how well sonrotoclax plus obinutuzumab works versus venetoclax plus rituximab in treating adults with relapsed and/or refractory (R/R) chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). The study will also compare how well sonrotoclax plus rituxim...

NCT07024706 Phase: 2 Started: 2026-06-04

This study will evaluate the efficacy and safety of finite-duration acalabrutinib plus venetoclax therapy in patients with relapsed CLL or SLL, who have previously responded to first line (1L) cBTKi + BCL2i therapy (± obinutuzumab) and maintained a response for at least two years post-treatment.

NCT04941716 Phase: 2 Started: 2023-05-31

This phase II trial is to evaluate the effects of acalabrutinib in combination with venetoclax in treating patients with chronic lymphocytic leukemia or small lymphocytic lymphoma that does not respond to treatment (refractory) or that has come back (recurrent). Acalabrutinib may stop the growth of ...

NCT02970318 Phase: 3 Started: 2017-02-02

This study is designed to evaluate the efficacy of acalabrutinib compared with rituximab in combination with idelalisib or bendamustine in previously treated subjects with chronic lymphocytic leukemia (CLL).

NCT05105841 Phase: 2 Started: 2021-11-08

Chronic lymphocytic leukemia (CLL) is the most common leukemia in Western countries, representing approximately 30% of all adult leukemias. There is a large difference in proportion of malignant lymphoma between the United States (US) and Japan was seen in CLL/small lymphocytic lymphoma (SLL) (Japan...

NCT06043674 Phase: 2 Started: 2024-01-22

This research is being done to evaluate Glofitamab by itself or in combination with Polatuzumab Vedotin, Pirtobrutinib, or Atezolizumab as possible treatments for Chronic Lymphocytic Leukemia (CLL) that has transformed into Richter's Transformation (RT). The names of the study drugs involved in this...

NCT05971251 Phase: 1 Started: 2023-12-18

Study is a phase I study to determine the maximum tolerated dose of adding Loncastuximab Tesirine to Aclabrutinib in the treatment of chronic lymphocytic leukemia.

NCT05797233 Phase: 1 Started: 2023-08-28

Background: About 23,000 people die from B-cell cancers in the US each year. These cancers, often called leukemia or lymphoma, affect a type of white blood cell called B cells. These cancers are difficult to treat, and the therapies used can have bad side effects. Researchers want to try a new type ...


Agents Found By Disease-Level Search

Searched directly from “Chronic Lymphocytic Leukaemia (CLL)” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 11-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

47 agents shown (approved or with trial evidence) — 29 not found by the gene-target search above.

rituximab

Approved · 16 trials · source: ClinicalTrials.gov, Open Targets
NCT01998880 NCT00004112 NCT00669318

cyclophosphamideNOT in gene-target list

Phase 3 · 12 trials · source: ClinicalTrials.gov, Open Targets
NCT00621452 NCT00003116 NCT00005854

cyclosporineNOT in gene-target list

Phase 2 · 9 trials · source: ClinicalTrials.gov, Open Targets
NCT00003116 NCT00005854 NCT00112593

fludarabine phosphateNOT in gene-target list

Approved · 8 trials · source: ClinicalTrials.gov, Open Targets
NCT01658319 NCT00856388 NCT00112593

Ibrutinib

Approved · 7 trials · source: ClinicalTrials.gov, Open Targets
NCT02759016 NCT02048813 NCT02264574

Fludarabine

Phase 3 · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT00276848 NCT05797233 NCT02251548

Mycophenolate mofetilNOT in gene-target list

Phase 2 · 6 trials · source: ClinicalTrials.gov, Open Targets
NCT04473911 NCT00112593 NCT02593123

Melphalan

Phase 2 · 5 trials · source: ClinicalTrials.gov, Open Targets
NCT04473911 NCT00244946 NCT00856388

filgrastimNOT in gene-target list

Phase 3 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00003116 NCT03147612 NCT00002832

cytarabine

Phase 2 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00244946 NCT00080886 NCT03147612

methotrexate

Phase 2 · 4 trials · source: ClinicalTrials.gov, Open Targets
NCT00005854 NCT00521430 NCT00317785

busulfanNOT in gene-target list

Phase 2 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT00003116 NCT00003335 NCT00521430

Sirolimus

Phase 3 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT04473911 NCT01251575 NCT00317785

obinutuzumab

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT01998880 NCT02264574 NCT04434196

Ofatumumab

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT01244451 NCT02135133 NCT01455051

Bendamustine

Phase 3 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT01244451 NCT02970318 NCT01400685

anti-thymocyte globulinNOT in gene-target list

· 3 trials · source: ClinicalTrials.gov
NCT00856388 NCT00003335 NCT00521430

Lenalidomide

Phase 3 · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT00774345 NCT01400685 NCT00096044

Idelalisib

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT02970318 NCT02135133 NCT02787369

BI 836826NOT in gene-target list

Phase 1 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT02759016 NCT01296932

therapeutic autologous lymphocytesNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00621452 NCT00244946

polymerase chain reactionNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00621452 NCT01005368

bone marrow aspirationNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT00621452 NCT06757647

western blottingNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT01658319 NCT01005368

Venetoclax

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT02980731 NCT02966756

Zilovertamab vedotinNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT03833180 NCT05458297

carmustineNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00244946 NCT00080886

etoposide

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00244946 NCT00080886

paclitaxelNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00004105 NCT00891072

chlorambucil

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01998880 NCT02264574

PegfilgrastimNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT03147612 NCT00472849

doxorubicin hydrochloride

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00004112 NCT00036790

Computed TomographyNOT in gene-target list

· 2 trials · source: ClinicalTrials.gov
NCT06757647 NCT06839053

SargramostimNOT in gene-target list

Phase 2 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT02593123 NCT00669318

alemtuzumab

Approved · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00669318 NCT01392079

methylprednisolone

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT00003335 NCT00521430

aldesleukinNOT in gene-target list

Phase 2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00621452

gene rearrangement analysisNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00621452

genetically engineered lymphocyte therapyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00621452

ixabepiloneNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00006221

Routine Blood Draw - Phase 1 onlyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06224309

Fludarabine plus CyclophosphamideNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00276848

becatecarinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00087204

methoxyamineNOT in gene-target list

Phase 1 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01658319

DNA analysisNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01658319

TBINOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04473911

RGI-2001NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04473911

Generated by the Open Source Medicine Foundation Biomarker Pipeline — 2026-10-08. Data sources: DGIdb, Open Targets, ChEMBL, PubMed, ClinicalTrials.gov. Not medical advice.