SMN2: Repurposing drugs for SMA

Repurposing Drugs for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is caused by insufficient levels of the survival motor neuron protein SMN. The SMN locus on chromosome 5q13 contains two inverted copies of SMN called SMN1 and SMN2 which are 99% identical at the amino acid level. SMN1is a fully functional protein and SMN2 skips axon 7 90% of the time. Skipping of exon 7 produces non-functional protein product. 10% of the SMN2 protein includes exon 7 and is fully functional. In the SMA disease state, mutations in the SMN1 locus are the cause of the disease state. Because only 10% of SMN2 is of the fully functional form, it is not sufficient to overcome the deficiency produced by the loss of the SMN1 product. A therapy that either increase the amount of SMN2 product made or to increase the inclusion of exon 7 has been proposed for the treatment of SMA.

Assay description by NIH Chemical Genomics Center [NCGC]. Assay Submitter (PI): Elliot Androphy. Go to ChEMBL assay page for details. 

For this reason, strategies that enhance SMN2 splice variant expression that yields active SMN protein have been considered of therapeutic interest. In this section we will use the SMN2 enhancing experiments stored in the ChEMBL DB to validate a predictive model that allows prediction of SMN2 enhancing activity for the 1.5M unique molecules with 13M activity records in te DB focusing on molecules that are actually prescription drugs.

About 3k among the 1.5M annotated molecules are prescription drugs. For some of them, there is a record of activity on SMN2 expression experiments, but for the most that don’t have any, there is a predicted score. The variable ActualPluspredictedSMN2Score contains the actual score if it exists, but the predicted one if it doesn’t. These scores have a -log(molar potency) scale.

Applying the dashboard based selection methods described in the prediction of SMN2 activity section upon prescription drugs we have created unique drug target pairs with average scores per pair: the already described ActualPluspredictedSMN2Score and the chemblActivityScoreC which shows the effect that the molecule average score for each particular target.

The graph below shows ActualPluspredictedSMN2Score vs chemblActivityScoreC . Marked dots are displayed on the right side showing the targets where the drugs have an activity record  and the value, in bars of their potency at target compared to the potency enhancing SMN2 expression.

It could be interesting checking what they do in animal models…

 

A different view showing the number of records instead of target chemblActivityScoreC for chemblActivityScoreC >5. Here we can estimate the confidence by the number of repeats…

 

And finally a treemap of the repurposed drugs for SMA showing the clinical phase achieved, current indications, published MoAs and colored by their potency enhancing SMN2 expression.

Below, the table with the results.

smn2RepurposingTable

drugNameshortNameGene nameschemblActivityScoreCsmn2ScorepredictionRFRegressioncountOfScoresdrugIndicationsMoAsconcatenatedActionTypemaxPhaseActualPlusPredictedSMN2ScorekindOfValue
PHENYTOINGEMIGMNN6.50003813440386.7617275032521EPILEPSY, INJURY, SEIZURESSodium channel alpha subunit blockerBLOCKER46.761727503252predicted
PHENYTOINFEN1FEN1 RAD26.19997064075596.7617275032521EPILEPSY, INJURY, SEIZURESSodium channel alpha subunit blockerBLOCKER46.761727503252predicted
KETOPROFENPGH2PTGS2 COX25.9452672944917.37.02291666666676ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENS22A6SLC22A6 OAT1 PAHT, Slc22a6 Oat15.77478883508467.37.1218754ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENAMPCampC ampA b4150 JW41115.457.35.8751ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENPGH1PTGS1 COX16.31403015887997.36.97150974025977ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENEHMT2EHMT2 BAT8 C6orf30 G9A KMT1C NG367.64975198166587.35.8751ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENS22A7Slc22a7 Nlt Oat25.747.36.50833333333331ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENTAUMAPT MAPTL MTBT1 TAU5.47.36.20751ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENSMNSMN1 SMN SMNT; SMN2 SMNC7.37.35.8751ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENS22ABSLC22A11 OAT45.62973141792587.37.31ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENPFKApfk5.0709849003767.37.31ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENGEMIGMNN6.53625627875297.36.50833333333331ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENLMNALMNA LMN16.57.35.79583333333331ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
KETOPROFENFEN1FEN1 RAD25.22382202741787.37.22083333333331ARTHRALGIA, MIGRAINE DISORDER, MYALGIA, OSTEOARTHRITIS, OSTEOARTHRITIS, KNEE, PAIN, RHEUMATIC DISEASE, SCIATICACyclooxygenase inhibitorINHIBITOR47.3actual
LOXOPROFENPFKApfk6.12096149476287.04251PAIN37.0425predicted
LOXOPROFENTRXR1Txnrd1 Trxr15.66589326272717.04253PAIN37.0425predicted
LOXOPROFENHSPB1HSPB1 HSP27 HSP285.82509743782177.04251PAIN37.0425predicted
SODIUM LAURYL SULFATELMNALMNA LMN19.04575749056076.86751PAIN, SKIN DISEASE16.8675predicted
OXEDRINERORGRORC NR1F3 RORG RZRG5.57506938555016.156.151CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
OXEDRINEPPARDPPARD NR1C2 PPARB5.96070488191576.156.151CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
OXEDRINETPOTHPO MGDF56.156.152CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
OXEDRINEGLP1RGLP1R5.57499594244276.156.152CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
OXEDRINEACM1Chrm1 Chrm-15.856.156.151CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
OXEDRINEEHMT2EHMT2 BAT8 C6orf30 G9A KMT1C NG365.89987592469746.156.152CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
OXEDRINESMNSMN1 SMN SMNT; SMN2 SMNC6.156.156.151CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
OXEDRINEBLMBLM RECQ2 RECQL38.556.156.152CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
OXEDRINENMUR2NMUR2 NMU2R TGR15.186.156.151CARDIOVASCULAR DISEASE, EYE ALLERGY06.15actual
BIOTINKDM4AKDM4A JHDM3A JMJD2 JMJD2A KIAA06778.45593195564976.74753IRON DEFICIENCY ANEMIA, MULTIPLE SCLEROSIS, TYPE II DIABETES MELLITUS46.7475predicted
BIOTINIDEIDE5.616.74752IRON DEFICIENCY ANEMIA, MULTIPLE SCLEROSIS, TYPE II DIABETES MELLITUS46.7475predicted
BIOTINSAV56.74751IRON DEFICIENCY ANEMIA, MULTIPLE SCLEROSIS, TYPE II DIABETES MELLITUS46.7475predicted
BIOTINSO1B1SLCO1B1 LST1 OATP1B1 OATP2 OATPC SLC21A66.12709467695216.74751IRON DEFICIENCY ANEMIA, MULTIPLE SCLEROSIS, TYPE II DIABETES MELLITUS46.7475predicted
BIOTINSO1B3SLCO1B3 LST2 OATP1B3 OATP8 SLC21A86.46748603914926.74751IRON DEFICIENCY ANEMIA, MULTIPLE SCLEROSIS, TYPE II DIABETES MELLITUS46.7475predicted
MYCOPHENOLIC ACIDPPARGPPARG NR1C35.15141945725346.36.33ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDIMDH1IMPDH1 IMPD17.04692307692316.36.313ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDSMNSMN1 SMN SMNT; SMN2 SMNC6.62142857142866.36.37ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDIMDH2IMPDH2 IMPD27.47833333333336.36.324ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDLEFlef pXO1-107 BXA0172 GBAA_pXO1_01725.96.36.31ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDGEMIGMNN6.43616308133556.36.31ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDNF2L2NFE2L2 NRF25.63621884741916.36.31ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDPMP22Pmp22 Cd25 Pmp-225.61611154804216.36.32ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDLMNALMNA LMN16.5256.36.32ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDGLP1RGLP1R6.04997609347676.36.31ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDEHMT2EHMT2 BAT8 C6orf30 G9A KMT1C NG3656.36.31ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
MYCOPHENOLIC ACIDSYUASNCA NACP PARK15.14999741390756.36.31ACUTE GRAFT VS. HOST DISEASE, CHRONIC KIDNEY DISEASE, IMMUNE SYSTEM DISEASE, INFECTION, INJURYInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorINHIBITOR46.3actual
DIFLUNISALRORGRorc Nr1f3 Rorg Thor7.26.62251FEVER, HIV INFECTION, PAINCyclooxygenase inhibitorINHIBITOR46.6225predicted
TEZOSENTANEDNRAEDNRA ETA ETRA7.646.21HEART FAILURE, PULMONARY HYPERTENSIONEndothelin receptor ET-A antagonist, Endothelin receptor ET-B antagonistANTAGONIST36.2predicted
NIFLUMIC ACIDHIF1AHIF1A BHLHE78 MOP1 PASD856.04754ARTHRALGIA, MYALGIA, RHEUMATIC DISEASE06.0475predicted
NIFLUMIC ACIDPFKApfk5.12098444659046.04751ARTHRALGIA, MYALGIA, RHEUMATIC DISEASE06.0475predicted
NIFLUMIC ACIDPGH1PTGS1 COX15.976.3851ARTHRALGIA, MYALGIA, RHEUMATIC DISEASE06.385predicted
NIFLUMIC ACIDAPEX1APEX1 APE APE1 APEX APX HAP1 REF15.95000714307996.3351ARTHRALGIA, MYALGIA, RHEUMATIC DISEASE06.335predicted
FELBAMATENPSR1NPSR1 GPR154 GPRA PGR146.17.47.41EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
FELBAMATECP2D6CYP2D6 CYP2DL15.73333333333337.47.43EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
FELBAMATEKPYMPKM OIP3 PK2 PK3 PKM257.46.7252EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
FELBAMATEACM1Chrm1 Chrm-16.57.47.41EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
FELBAMATEEHMT2EHMT2 BAT8 C6orf30 G9A KMT1C NG366.03678712134487.47.42EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
FELBAMATESMNSMN1 SMN SMNT; SMN2 SMNC7.47.47.41EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
FELBAMATEKCNH2KCNH2 ERG ERG1 HERG5.25000102344167.47.41EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
FELBAMATENMDE2GRIN2B NMDAR2B5.32735893438637.47.41EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
FELBAMATEGEMIGMNN6.96861991119527.47.42EPILEPSYGlutamate [NMDA] receptor antagonistANTAGONIST47.4actual
CLAZOSENTANEDNRAEDNRA ETA ETRA9.896.6751SUBARACHNOID HEMORRHAGEEndothelin receptor ET-A antagonistANTAGONIST36.675predicted
CLAZOSENTANEDNRBEDNRB ETRB6.766.6751SUBARACHNOID HEMORRHAGEEndothelin receptor ET-A antagonistANTAGONIST36.675predicted
ICLAPRIMDYRDHFR, folA7.6052878745286.068333333333310INFECTION, PNEUMONIA36.0683333333333predicted
PRULIFLOXACINANDRAR DHTR NR3C45.64050791798176.68753CHRONIC BRONCHITIS, CHRONIC OBSTRUCTIVE PULMONARY DISEASE, URINARY TRACT INFECTION36.6875predicted
MODAFINILSC6A3SLC6A3 DAT1, Slc6a35.46454545454556.048295454545411ALZHEIMERS DISEASE, ATTENTION DEFICIT HYPERACTIVITY DISORDER, BIPOLAR DISORDER, BRAIN NEOPLASM, COCAINE DEPENDENCE, EWING SARCOMA, FATIGUE, METAMPHETAMINE DEPENDENCE, NARCOLEPSY WITH CATAPLEXY, NON-SMALL CELL LUNG CARCINOMA, OBSTRUCTIVE SLEEP APNEA, ORTHOSTATIC HYPOTENSION, OSTEOARTHRITIS, OVARIAN CARCINOMA, POST-TRAUMATIC STRESS DISORDER, SCHIZOPHRENIA, SLEEP APNEA, STROKE, TINNITUS, UNIPOLAR DEPRESSIONDopamine transporter inhibitorINHIBITOR46.0482954545454predicted
MODAFINILQ9WTR4Slc6a256.41ALZHEIMERS DISEASE, ATTENTION DEFICIT HYPERACTIVITY DISORDER, BIPOLAR DISORDER, BRAIN NEOPLASM, COCAINE DEPENDENCE, EWING SARCOMA, FATIGUE, METAMPHETAMINE DEPENDENCE, NARCOLEPSY WITH CATAPLEXY, NON-SMALL CELL LUNG CARCINOMA, OBSTRUCTIVE SLEEP APNEA, ORTHOSTATIC HYPOTENSION, OSTEOARTHRITIS, OVARIAN CARCINOMA, POST-TRAUMATIC STRESS DISORDER, SCHIZOPHRENIA, SLEEP APNEA, STROKE, TINNITUS, UNIPOLAR DEPRESSIONDopamine transporter inhibitorINHIBITOR46.4predicted
MODAFINILLMNALMNA LMN18.746.41ALZHEIMERS DISEASE, ATTENTION DEFICIT HYPERACTIVITY DISORDER, BIPOLAR DISORDER, BRAIN NEOPLASM, COCAINE DEPENDENCE, EWING SARCOMA, FATIGUE, METAMPHETAMINE DEPENDENCE, NARCOLEPSY WITH CATAPLEXY, NON-SMALL CELL LUNG CARCINOMA, OBSTRUCTIVE SLEEP APNEA, ORTHOSTATIC HYPOTENSION, OSTEOARTHRITIS, OVARIAN CARCINOMA, POST-TRAUMATIC STRESS DISORDER, SCHIZOPHRENIA, SLEEP APNEA, STROKE, TINNITUS, UNIPOLAR DEPRESSIONDopamine transporter inhibitorINHIBITOR46.4predicted
MODAFINILEHMT2EHMT2 BAT8 C6orf30 G9A KMT1C NG365.45000414846946.41ALZHEIMERS DISEASE, ATTENTION DEFICIT HYPERACTIVITY DISORDER, BIPOLAR DISORDER, BRAIN NEOPLASM, COCAINE DEPENDENCE, EWING SARCOMA, FATIGUE, METAMPHETAMINE DEPENDENCE, NARCOLEPSY WITH CATAPLEXY, NON-SMALL CELL LUNG CARCINOMA, OBSTRUCTIVE SLEEP APNEA, ORTHOSTATIC HYPOTENSION, OSTEOARTHRITIS, OVARIAN CARCINOMA, POST-TRAUMATIC STRESS DISORDER, SCHIZOPHRENIA, SLEEP APNEA, STROKE, TINNITUS, UNIPOLAR DEPRESSIONDopamine transporter inhibitorINHIBITOR46.4predicted
PHENYLEPHRINE HYDROCHLORIDELMNALMNA LMN15.5256.152CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDEHIF1AHIF1A BHLHE78 MOP1 PASD876.152CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDETRXR1Txnrd1 Trxr15.87383611119426.151CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDELYAGGAA6.556.152CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDEGLP1RGLP1R5.19999767457356.152CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDEADRB2ADRB2 ADRB2R B2AR5.81118766754766.152CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDEGEMIGMNN5.93621643940266.151CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDEPMP22Pmp22 Cd25 Pmp-225.94218180556796.151CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDEIDHCIDH1 PICD6.53625627875296.151CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDEP53TP53 P536.84985783815146.151CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
PHENYLEPHRINE HYDROCHLORIDESO1B1SLCO1B1 LST1 OATP1B1 OATP2 OATPC SLC21A66.05489343428656.151CARDIAC ARRHYTHMIA, CATARACT, HYPOTENSION, OVARIAN CYST, PREECLAMPSIAAdrenergic receptor alpha-1 agonistAGONIST46.15predicted
TIVOZANIBCDK3CDK3 CDKN36.09337546121986.118756COLORECTAL ADENOCARCINOMA, GLIOBLASTOMA MULTIFORME, HEPATOCELLULAR CARCINOMA, NEOPLASM, NON-SMALL CELL LUNG CARCINOMA, RENAL CELL CARCINOMA, SARCOMAVascular endothelial growth factor receptor inhibitorINHIBITOR36.11875predicted
CRIDANIMODTRYP5.156.4492275032521ENDOMETRIAL NEOPLASM26.449227503252predicted
CRIDANIMODEHMT2EHMT2 BAT8 C6orf30 G9A KMT1C NG365.05000045714016.3617275032521ENDOMETRIAL NEOPLASM26.361727503252predicted
CRIDANIMODASMSMPD1 ASM56.3617275032521ENDOMETRIAL NEOPLASM26.361727503252predicted
PUMOSETRAG5HT3AHTR3A 5HT3R HTR36.5372971072386.18754GASTROESOPHAGEAL REFLUX DISEASE26.1875predicted
shortName: protein names with drug records in the ChEMBL DB; Gene names names with drug records in the ChEMBL DB; chemblActivityScoreC: potency score of the drug upon the target referred in shortName; predictionRFRegression: random forests prediction of the drug potency on SMN2 expression enhancement assay; smn2Score: experimental record of drug potency on SMN2 expression enhancement assay; countOfScores: Number of events in the DB; ActualPlusPredictedSMN2Score: predicted SMN2 score added only to rows where there are not experimental data.