Biomarker & Intervention Discovery

PCOS (Polycystic Ovary Syndrome)

This page lists 11 druggable gene targets, 42 approved or trial-tested drug candidates and 15 recent clinical trials for PCOS (Polycystic Ovary Syndrome), 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 remissionPCOS is a lifelong endocrine disorder; complete spontaneous resolution rare; some phenotypic improvement with…
Best-intervention remissionWeight loss of 5-10% restores ovulation in 55-90% of anovulatory PCOS women; metformin restores ovulation in 30-50%…
Gap sizeLarge — lifestyle modification and metformin are evidence-based first-line but most patients receive OCPs (treating…
Primary barrierframing/practice
Full remission profile →

Remission & chronicity

Spontaneous remission
PCOS is a lifelong endocrine disorder; complete spontaneous resolution rare; some phenotypic improvement with menopause; fertility returns in 50-70% with ovulation induction; metabolic features persist
Best-intervention remission
Weight loss of 5-10% restores ovulation in 55-90% of anovulatory PCOS women; metformin restores ovulation in 30-50%; letrozole (ovulation induction): 27% live birth rate per cycle (PPCOS II); lifestyle modification: 50-80% improvement in menstrual regularity
Gap size
Large — lifestyle modification and metformin are evidence-based first-line but most patients receive OCPs (treating symptoms only, not the underlying metabolic disorder)
Primary barrier
framing/practice
OCP (oral contraceptive pill) is most commonly prescribed for PCOS, treating symptoms (irregular periods, hirsutism) without addressing the underlying insulin resistance and metabolic risk; OCP does not treat infertility and may worsen insulin sensitivity; the PCOS field has lacked a clear pharmacotherapy standard

Biomarker Targets & Therapeutic Agents

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

AMH 7AR 994CYP17A1 124CYP19A1 187FSHR 108GLP1R 316INSR 245LHCGR 78PPARG 274PRKAA1 204SHBG 107
Therapeutic Agent Effect Direction Evidence Tier Potency Source
THERAPEUTIC GONADOTROPIN ? unclear Mechanistic
ETHINYL ESTRADIOL ? unclear Mechanistic
DIETHYLSTILBESTROL ? unclear Mechanistic
CHORIOGONADOTROPIN ALFA ? unclear Mechanistic
TESTOSTERONE ? unclear Mechanistic
THERAPEUTIC HORMONE ? unclear Mechanistic
TRETINOIN ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
AMI-1 ? unclear Mechanistic
DIBEKACIN ? unclear Mechanistic
CARBONYL CYANIDE M-CHLOROPHENYLHYDRAZONE ? unclear Mechanistic
TRIPARANOL ? unclear Mechanistic
DODINE ? unclear Mechanistic
TANNIC ACID ? unclear Mechanistic
DEFLAZACORT ? unclear Mechanistic
CHEMBL:CHEMBL1235854 ? unclear Mechanistic
ERGONOVINE ? unclear Mechanistic
AMPEROZIDE HYDROCHLORIDE ? unclear Mechanistic
ATROPINE METHYLBROMIDE ? unclear Mechanistic
ESTRADIOL VALERATE ? unclear Mechanistic
BERBERINE CHLORIDE ? unclear Mechanistic
NITRALIN ? unclear Mechanistic
DEVAZEPIDE ? unclear Mechanistic
MOCLOBEMIDE ? unclear Mechanistic
GLPG0492 ~ modulates Mechanistic
DEUTENZALUTAMIDE ↓ inhibits Mechanistic
PRUXELUTAMIDE ↓ inhibits Mechanistic
HEPTANE ? unclear Mechanistic
THONZONIUM BROMIDE ? unclear Mechanistic
FENHEXAMID ? unclear Mechanistic
LEMILDIPINE ? unclear Mechanistic
(R)-BICALUTAMIDE ? unclear Mechanistic
MORICIZINE HYDROCHLORIDE ? unclear Mechanistic
METIPRANOLOL ? unclear Mechanistic
FLUROCHLORIDONE ? unclear Mechanistic
PYRIDOSTIGMINE BROMIDE ? unclear Mechanistic
FLUTAMIDE ↓ inhibits Mechanistic 154.0 nM (IC50)
CHEMBL:CHEMBL224318 ? unclear Mechanistic
CHEMBL:CHEMBL423081 ? unclear Mechanistic
PREDNISOLONE ? unclear Mechanistic 2760.0 nM (Ki)
ORBIFLOXACIN ? unclear Mechanistic
PROGESTERONE ? unclear Mechanistic 8.5 nM (Ki)
FLUDIOXONIL ? unclear Mechanistic
CHEMBL:CHEMBL1784521 ? unclear Mechanistic
FLUMETHASONE ? unclear Mechanistic
SERTRALINE HYDROCHLORIDE ? unclear Mechanistic
MELOXICAM ? unclear Mechanistic
CHEMBL:CHEMBL44011 ? unclear Mechanistic

Showing top 40 of 994 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PACLITAXEL PROTEIN-BOUND ? unclear Mechanistic
ORTERONEL ↓ inhibits Mechanistic
ENZALUTAMIDE ? unclear Mechanistic
GALETERONE ↓ inhibits Mechanistic
ABIRATERONE-C6 OXIME ↓ inhibits Mechanistic
ABIRATERONE ACETATE ↓ inhibits Mechanistic 17.0 nM (IC50)
SEVITERONEL ? unclear Mechanistic
LEVOKETOCONAZOLE ↓ inhibits Mechanistic
LAPITERONEL ? unclear Mechanistic
YXG-158 ↓ inhibits Mechanistic
ABIRATERONE ? unclear Mechanistic 4.0 nM (IC50)
CHEMBL138580 ↓ inhibits Mechanistic 1800.0 nM (IC50)
CHEMBL310690 ↓ inhibits Mechanistic 160.0 nM (IC50)
CHEMBL341591 ↓ inhibits Mechanistic 400.0 nM (IC50)
CHEMBL2111948 ↓ inhibits Mechanistic 1520.0 nM (IC50)
CHEMBL3137965 ↓ inhibits Mechanistic 74.0 nM (IC50)
CHEMBL2112781 ↓ inhibits Mechanistic 1250.0 nM (IC50)
CHEMBL357811 ↓ inhibits Mechanistic 27.0 nM (IC50)
CHEMBL2112888 ↓ inhibits Mechanistic 260.0 nM (IC50)
CHEMBL2111947 ↓ inhibits Mechanistic 540.0 nM (IC50)
CHEMBL3137967 ↓ inhibits Mechanistic 5.0 nM (IC50)
CHEMBL2112782 ↓ inhibits Mechanistic 970.0 nM (IC50)
CHEMBL432751 ↓ inhibits Mechanistic 3.0 nM (IC50)
CHEMBL2112772 ↓ inhibits Mechanistic 1520.0 nM (IC50)
CHEMBL310315 ↓ inhibits Mechanistic 13.0 nM (IC50)
CHEMBL82926 ↓ inhibits Mechanistic 47.0 nM (IC50)
CHEMBL2112775 ↓ inhibits Mechanistic 2500.0 nM (IC50)
KETOCONAZOLE ↓ inhibits Mechanistic 740.0 nM (IC50)
CHEMBL3085257 ↓ inhibits Mechanistic 1800.0 nM (IC50)
CHEMBL2111951 ↓ inhibits Mechanistic 290.0 nM (IC50)
CHEMBL419743 ↓ inhibits Mechanistic 400.0 nM (IC50)
CHEMBL2112522 ↓ inhibits Mechanistic 21.0 nM (IC50)
CHEMBL2112517 ↓ inhibits Mechanistic 42.0 nM (IC50)
CHEMBL2112524 ↓ inhibits Mechanistic 100.0 nM (IC50)
CHEMBL2112526 ↓ inhibits Mechanistic 24.0 nM (IC50)
CHEMBL2112533 ↓ inhibits Mechanistic 400.0 nM (IC50)
CHEMBL2112519 ↓ inhibits Mechanistic 430.0 nM (IC50)
CHEMBL325307 ↓ inhibits Mechanistic 50.0 nM (IC50)
CHEMBL114864 ↓ inhibits Mechanistic 150.0 nM (IC50)
CHEMBL115944 ↓ inhibits Mechanistic 150.0 nM (IC50)

Showing top 40 of 124 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
AZALANSTAT ↓ inhibits Mechanistic 7.6 nM (Ki)
NARINGENIN ? unclear Mechanistic
TESTOLACTONE ↓ inhibits Mechanistic 35000.0 nM (Ki)
ATROPINE ? unclear Mechanistic
FADROZOLE ↓ inhibits Mechanistic
EXEMESTANE ↓ inhibits Mechanistic
AMINOGLUTETHIMIDE ↓ inhibits Mechanistic 600.0 nM (Ki)
LIOTHYRONINE ? unclear Mechanistic
RECOMBINANT TUMOR NECROSIS FACTOR FAMILY PROTEIN ? unclear Mechanistic
IBUPROFEN, SODIUM SALT ? unclear Mechanistic
LIAROZOLE ? unclear Mechanistic
CHEMBL:CHEMBL1077603 ? unclear Mechanistic
AZTREONAM ? unclear Mechanistic
GONADOTROPIN-RELEASING HORMONE ANALOG ? unclear Mechanistic
ISOPROTERENOL ? unclear Mechanistic
CAPTOPRIL ? unclear Mechanistic
TAMOXIFEN ? unclear Mechanistic
LETROZOLE ↓ inhibits Mechanistic
ANASTROZOLE ↓ inhibits Mechanistic
ATAMESTANE ? unclear Mechanistic
FORMESTANE ? unclear Mechanistic 410.0 nM (IC50)
PHYTOESTROGEN ? unclear Mechanistic
FADROZOLE HYDROCHLORIDE ↓ inhibits Mechanistic
PHENYTOIN SODIUM ? unclear Mechanistic
ANABOLIC STEROID ? unclear Mechanistic
TESTOSTERONE ENANTHATE ? unclear Mechanistic
CHEMBL:CHEMBL572637 ? unclear Mechanistic
ADENOVIRUS VECTOR ? unclear Mechanistic
ISOPROPYL ALCOHOL ? unclear Mechanistic
BIOFLAVONOID ? unclear Mechanistic
ALENDRONATE SODIUM ? unclear Mechanistic
CAPECITABINE ? unclear Mechanistic
GENISTEIN ? unclear Mechanistic
NONSTEROIDAL ANTIINFLAMMATORY DRUG ? unclear Mechanistic
ENVIRONMENTAL ESTROGEN ? unclear Mechanistic
ANDROSTENEDIONE ? unclear Mechanistic
OPIATE ? unclear Mechanistic
FLUOROURACIL ? unclear Mechanistic
NORENDOXIFEN ↓ inhibits Mechanistic
ENDOXIFEN ↓ inhibits Mechanistic

Showing top 40 of 187 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
FOLLICLE STIMULATING HORMONE ? unclear Mechanistic
CORIFOLLITROPIN ALFA ↑ activates Mechanistic
UROFOLLITROPIN ↑ activates Mechanistic
FOLLITROPIN ↑ activates Mechanistic
[125I]FSH (HUMAN) ↑ activates Mechanistic
FSH DEGLYCOSYLATED Α/Β ↓ inhibits Mechanistic
SURAMIN ? unclear Mechanistic
CHORIONIC GONADOTROPIN ? unclear Mechanistic
FOLLITROPIN ALFA ↑ activates Mechanistic
CHORIOGONADOTROPIN ALFA ? unclear Mechanistic
THYROTROPIN ALFA ? unclear Mechanistic
FOLLITROPIN BETA ↑ activates Mechanistic
FOLLITROPIN DELTA ↑ activates Mechanistic
CHEMBL50556 ↑ activates Mechanistic 260.0 nM (EC50)
CHEMBL417173 ↑ activates Mechanistic 2500.0 nM (EC50)
CHEMBL47120 ↑ activates Mechanistic 3900.0 nM (EC50)
CHEMBL50278 ↑ activates Mechanistic 3000.0 nM (EC50)
CHEMBL279759 ↑ activates Mechanistic 25.0 nM (EC50)
CHEMBL296449 ↑ activates Mechanistic 68.0 nM (EC50)
CHEMBL49308 ↑ activates Mechanistic 120.0 nM (EC50)
CHEMBL45976 ↑ activates Mechanistic 400.0 nM (EC50)
CHEMBL46216 ↑ activates Mechanistic 9.7 nM (EC50)
CHEMBL47838 ↑ activates Mechanistic 900.0 nM (EC50)
CHEMBL48375 ↑ activates Mechanistic 8.9 nM (EC50)
CHEMBL47953 ↑ activates Mechanistic 1.8 nM (EC50)
CHEMBL295895 ↑ activates Mechanistic 1.2 nM (EC50)
CHEMBL417003 ↑ activates Mechanistic 1200.0 nM (EC50)
CHEMBL47420 ↑ activates Mechanistic 3300.0 nM (EC50)
CHEMBL300022 ↑ activates Mechanistic 3.0 nM (EC50)
CHEMBL49608 ↑ activates Mechanistic 7.2 nM (EC50)
CHEMBL45782 ↑ activates Mechanistic 5000.0 nM (EC50)
CHEMBL47059 ↑ activates Mechanistic 14.0 nM (EC50)
CHEMBL48030 ↑ activates Mechanistic 3.6 nM (EC50)
CHEMBL49540 ↑ activates Mechanistic 1600.0 nM (EC50)
CHEMBL45770 ↑ activates Mechanistic 1.7 nM (EC50)
CHEMBL433263 ↑ activates Mechanistic 2.7 nM (EC50)
CHEMBL47500 ↑ activates Mechanistic 100.0 nM (EC50)
CHEMBL44663 ↑ activates Mechanistic 150.0 nM (EC50)
CHEMBL298686 ↑ activates Mechanistic 2300.0 nM (EC50)
CHEMBL432883 ↑ activates Mechanistic 9000.0 nM (EC50)

Showing top 40 of 108 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
LINAGLIPTIN ? unclear Mechanistic
FRONIGLUTIDE ? unclear Mechanistic
CHEMBL:CHEMBL576809 ? unclear Mechanistic
2-METHOXYESTRADIOL ? unclear Mechanistic
SURVODUTIDE ↑ activates Mechanistic
LIRAGLUTIDE ↑ activates Mechanistic
GLUCAGON-LIKE PEPTIDE 1-(7-36) AMIDE ↑ activates Mechanistic
CHEMBL:CHEMBL530609 ? unclear Mechanistic
PRAMLINTIDE ? unclear Mechanistic
CHEMBL:CHEMBL580076 ? unclear Mechanistic
PEGAPAMODUTIDE ↑ activates Mechanistic
TRICLABENDAZOLE ? unclear Mechanistic
CHEMBL:CHEMBL597244 ? unclear Mechanistic
CHEMBL:CHEMBL600734 ? unclear Mechanistic
CHEMBL:CHEMBL1422704 ? unclear Mechanistic
CHEMBL:CHEMBL1390716 ? unclear Mechanistic
AMORFRUTIN A ? unclear Mechanistic
CHEMBL:CHEMBL317115 ? unclear Mechanistic
FENBENDAZOLE ? unclear Mechanistic
CHEMBL:CHEMBL585444 ? unclear Mechanistic
MITONAFIDE ? unclear Mechanistic
HOMIDIUM BROMIDE ? unclear Mechanistic
GLUCAGON-LIKE PEPTIDE 1-(7-37) ↑ activates Mechanistic
DANUGLIPRON ↑ activates Mechanistic
CHEMBL:CHEMBL1543865 ? unclear Mechanistic
CHEMBL:CHEMBL532452 ? unclear Mechanistic
AVEXITIDE ↓ inhibits Mechanistic
CHEMBL:CHEMBL548286 ? unclear Mechanistic
CHEMBL:CHEMBL1270168 ? unclear Mechanistic
CHEMBL:CHEMBL591834 ? unclear Mechanistic
CHEMBL:CHEMBL581152 ? unclear Mechanistic
CHEMBL:CHEMBL605708 ? unclear Mechanistic
LASALOCID A SODIUM ? unclear Mechanistic
SAXAGLIPTIN ANHYDROUS ? unclear Mechanistic
CHEMBL:CHEMBL86464 ? unclear Mechanistic
CHEMBL:CHEMBL1509103 ? unclear Mechanistic
CHEMBL:CHEMBL261693 ? unclear Mechanistic
ALBUTEROL SULFATE ? unclear Mechanistic
CHEMBL:CHEMBL1569494 ? unclear Mechanistic
GLUTAZUMAB ↑ activates Mechanistic

Showing top 40 of 316 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
LINSITINIB ↓ inhibits Mechanistic
GW441756X ? unclear Mechanistic
INSULIN GLARGINE ↑ activates Mechanistic
INSULIN ASPART PROTAMINE, HUMAN ↑ activates Mechanistic
INSULIN, ISOPHANE ↑ activates Mechanistic
INSULIN ASPART PROTAMINE RECOMBINANT ↑ activates Mechanistic
MITOGLITAZONE ? unclear Mechanistic
HESPERADIN ↓ inhibits Mechanistic
INSULIN, NEUTRAL ↑ activates Mechanistic
INSULIN TREGOPIL ↑ activates Mechanistic
AGT-182 ? unclear Mechanistic
LINIFANIB ? unclear Mechanistic
TOPIRAMATE ? unclear Mechanistic
INSULIN, REGULAR, HUMAN ↑ activates Mechanistic
SP-600125 ? unclear Mechanistic
NVP-TAE684 ? unclear Mechanistic
INSULIN HUMAN ZINC ↑ activates Mechanistic
INSULIN ICODEC ↑ activates Mechanistic
CERITINIB ↓ inhibits Mechanistic
BMS-536924 ↓ inhibits Mechanistic
CYC-116 ? unclear Mechanistic
INSULIN SUSP ISOPHANE SEMISYNTHETIC PURIFIED HUMAN ↑ activates Mechanistic
COMPOUND 15 [PMID: 24900237] ↓ inhibits Mechanistic
INSULIN, PORCINE ? unclear Mechanistic
BMS-754807 ↓ inhibits Mechanistic
TESOFENSINE ? unclear Mechanistic
CYCLOPHOSPHAMIDE ANHYDROUS ? unclear Mechanistic
CENISERTIB ? unclear Mechanistic
INSULIN HUMAN ↑ activates Mechanistic
NVP-TAE 226 ↓ inhibits Mechanistic
INSULIN PURIFIED PORK ↑ activates Mechanistic
FLUOROURACIL ? unclear Mechanistic
INSULIN SUSP ISOPHANE BEEF ↑ activates Mechanistic
RECOMBINANT HUMAN KALLIKREIN-1 ? unclear Mechanistic
INSULIN LISPRO ↑ activates Mechanistic
GÖ 6976 ? unclear Mechanistic
MYRICETIN ↓ inhibits Mechanistic 2600.0 nM (Ki)
INSULIN ZINC SUSP RECOMBINANT HUMAN ↑ activates Mechanistic
INSULIN ZINC, EXTENDED ↑ activates Mechanistic
TAMATINIB ? unclear Mechanistic

Showing top 40 of 245 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHORIONIC GONADOTROPIN ↑ activates Mechanistic
LUTROPIN ALFA ↑ activates Mechanistic
CHORIOGONADOTROPIN ALFA ? unclear Mechanistic
HCG ↑ activates Mechanistic
UROFOLLITROPIN ↑ activates Mechanistic
RELUGOLIX ? unclear Mechanistic
[125I]LH ↑ activates Mechanistic
LH (MENOTROPINS) ↑ activates Mechanistic
GTX-758 ? unclear Mechanistic
GONADOTROPIN, CHORIONIC ↑ activates Mechanistic
CHEMBL:CHEMBL448753 ? unclear Mechanistic
CTP-37-DT VACCINE ? unclear Mechanistic
[125I]CHORIONIC GONADOTROPIN (HUMAN) ↑ activates Mechanistic
CHEMBL:CHEMBL505670 ? unclear Mechanistic
CHEMBL:CHEMBL429095 ? unclear Mechanistic
FOLLICLE STIMULATING HORMONE ? unclear Mechanistic
GOSERELIN ? unclear Mechanistic
CHEMBL378091 ↑ activates Mechanistic 1900.0 nM (EC50)
CHEMBL212815 ↑ activates Mechanistic 1200.0 nM (EC50)
CHEMBL211129 ↑ activates Mechanistic 1500.0 nM (EC50)
CHEMBL377769 ↑ activates Mechanistic 800.0 nM (EC50)
CHEMBL377399 ↑ activates Mechanistic 800.0 nM (EC50)
CHEMBL211871 ↑ activates Mechanistic 1100.0 nM (EC50)
CHEMBL211405 ↑ activates Mechanistic 300.0 nM (EC50)
CHEMBL246321 ↓ inhibits Mechanistic 0.311 nM (Ki)
CHEMBL429108 ↑ activates Mechanistic 80.0 nM (EC50)
CHEMBL393847 ↑ activates Mechanistic 190.0 nM (EC50)
CHEMBL439392 ↑ activates Mechanistic 150.0 nM (EC50)
CHEMBL247700 ↑ activates Mechanistic 170.0 nM (EC50)
CHEMBL269261 ↑ activates Mechanistic 12100.0 nM (EC50)
CHEMBL247945 ↑ activates Mechanistic 500.0 nM (EC50)
CHEMBL397316 ↑ activates Mechanistic 250.0 nM (EC50)
CHEMBL245663 ↑ activates Mechanistic 2300.0 nM (EC50)
CHEMBL474041 ↓ inhibits Mechanistic 2300.0 nM (IC50)
CHEMBL1651721 ↑ activates Mechanistic 1.0 nM (EC50)
CHEMBL1651723 ↑ activates Mechanistic 10.0 nM (EC50)
CHEMBL1651823 ↑ activates Mechanistic 27.0 nM (EC50)
CHEMBL1651824 ↑ activates Mechanistic 43.0 nM (EC50)
CHEMBL1651825 ↑ activates Mechanistic 61.0 nM (EC50)
CHEMBL1651826 ↑ activates Mechanistic 55.0 nM (EC50)

Showing top 40 of 78 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
GANCAONIN L ? unclear Mechanistic
MITOGLITAZONE ? unclear Mechanistic
BADGE ↓ inhibits Mechanistic
OLSALAZINE SODIUM ↑ activates Mechanistic
CELECOXIB ? unclear Mechanistic
LY-510929 ↑ activates Mechanistic 10.0 nM (IC50)
TROGLITAZONE ↑ activates Mechanistic 302.0 nM (Ki)
BENZBROMARONE ? unclear Mechanistic
APIGENIN ? unclear Mechanistic
CHIGLITAZAR ↑ activates Mechanistic
LEVOTHYROXINE ? unclear Mechanistic
NALED ? unclear Mechanistic
CLX-0921 ? unclear Mechanistic
MBX-2044 ~ modulates Mechanistic
DICHLOROPHEN ? unclear Mechanistic
LANIFIBRANOR ↑ activates Mechanistic
TRIFLOXYSTROBIN ? unclear Mechanistic
PROCHLORAZ ? unclear Mechanistic
CHEMBL:CHEMBL599165 ? unclear Mechanistic
NAVEGLITAZAR ↑ activates Mechanistic
RAGAGLITAZAR ↑ activates Mechanistic
ALEGLITAZAR ↑ activates Mechanistic
TREPROSTINIL ? unclear Mechanistic
GW7845 ↑ activates Mechanistic
METHOTREXATE ? unclear Mechanistic
FENOFIBRIC ACID ? unclear Mechanistic
ATX08-001 ~ modulates Mechanistic
JB991 ? unclear Mechanistic
AD-5061 ↑ activates Mechanistic
GW409544 ↑ activates Mechanistic
[125I]SB-236636 ↑ activates Mechanistic
ROSIGLITAZONE MALEATE ↑ activates Mechanistic
RIVOGLITAZONE ↑ activates Mechanistic
MESALAMINE ↑ activates Mechanistic
BALSALAZIDE DISODIUM ↑ activates Mechanistic
2-PHENYLPHENOL ? unclear Mechanistic
CHEMBL:CHEMBL184450 ? unclear Mechanistic
ABIETIC ACID ? unclear Mechanistic
CHEMBL:CHEMBL2208196 ? unclear Mechanistic
ASPIRIN ? unclear Mechanistic

Showing top 40 of 274 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CENISERTIB ? unclear Mechanistic
SIROLIMUS ? unclear Mechanistic
JNJ-7706621 ? unclear Mechanistic 1700.0 nM (Kd)
SAPONARIN ? unclear Mechanistic
TYROSINE KINASE INHIBITOR ? unclear Mechanistic
METFORMIN HYDROCHLORIDE ↑ activates Mechanistic
ENTRECTINIB ? unclear Mechanistic 251.19 nM (Ki)
PHENFORMIN ? unclear Mechanistic
PALBOCICLIB ? unclear Mechanistic
LY2090314 ? unclear Mechanistic
CHEMBL:CHEMBL1997335 ? unclear Mechanistic
TETRADECANOYLPHORBOL ACETATE ? unclear Mechanistic
DOVITINIB ? unclear Mechanistic 520.0 nM (Kd)
CHEMOPREVENTIVE AGENT ? unclear Mechanistic
METFORMIN ? unclear Mechanistic
SOTRASTAURIN ? unclear Mechanistic 630.96 nM (Ki)
CHEMBL:CHEMBL587615 ? unclear Mechanistic
NVP-TAE684 ? unclear Mechanistic
PD166285 ? unclear Mechanistic
LEVOTHYROXINE SODIUM ? unclear Mechanistic
TOZASERTIB ? unclear Mechanistic 1100.0 nM (Kd)
HESPERADIN ↓ inhibits Mechanistic 19.0 nM (Kd)
ACADESINE ↑ activates Mechanistic
ADENOSINE DEAMINASE INHIBITOR ? unclear Mechanistic
PENTOSTATIN ? unclear Mechanistic
GW441756X ? unclear Mechanistic
CHEMBL:CHEMBL379975 ? unclear Mechanistic
ILORASERTIB ? unclear Mechanistic
CYC-116 ? unclear Mechanistic 501.19 nM (Ki)
RG-1530 ? unclear Mechanistic
PF-562271 ? unclear Mechanistic
STREPTOZOCIN ? unclear Mechanistic
SUNITINIB ↓ inhibits Mechanistic 52.0 nM (Kd)
ALVOCIDIB ↓ inhibits Mechanistic 6600.0 nM (Kd)
STAUROSPORINE ↓ inhibits Mechanistic 9.0 nM (Kd)
VANDETANIB ↓ inhibits Mechanistic 10000.0 nM (Kd)
AST-487 ↓ inhibits Mechanistic 1400.0 nM (Kd)
RAF-265 ↓ inhibits Mechanistic 4500.0 nM (Kd)
MIDOSTAURIN ↓ inhibits Mechanistic 180.0 nM (Kd)
SU-014813 ↓ inhibits Mechanistic 100.0 nM (Kd)

Showing top 40 of 204 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CHEMBL:CHEMBL464631 ? unclear Mechanistic
NORGESTREL ? unclear Mechanistic 1.23 nM (Kd)
SOMATROPIN (RECOMBINANT DNA ORIGIN) ? unclear Mechanistic
BUSULFAN ? unclear Mechanistic
DROLOXIFENE ? unclear Mechanistic
INSULIN, REGULAR, HUMAN ? unclear Mechanistic
ENZYME INHIBITOR ? unclear Mechanistic
ANTICONVULSANT AGENT ? unclear Mechanistic
ADJUVANT ? unclear Mechanistic
LISINOPRIL ANHYDROUS ? unclear Mechanistic
STANOLONE ↓ inhibits Mechanistic 3.8 nM (IC50)
CHEMBL194392 ↓ inhibits Mechanistic 22400.0 nM (IC50)
CHEMBL193200 ↓ inhibits Mechanistic 13600.0 nM (IC50)
CHEMBL218958 ↓ inhibits Mechanistic 48230.0 nM (IC50)
CHEMBL216530 ↓ inhibits Mechanistic 11140.0 nM (IC50)
CHEMBL216660 ↓ inhibits Mechanistic 10350.0 nM (IC50)
CHEMBL386857 ↓ inhibits Mechanistic 950.0 nM (IC50)
ESTRADIOL ↓ inhibits Mechanistic 50.0 nM (IC50)
TESTOSTERONE ↓ inhibits Mechanistic 14.0 nM (IC50)
CHEMBL265939 ↓ inhibits Mechanistic 2187.76 nM (Kd)
NARINGENIN ↓ inhibits Mechanistic 954.99 nM (Kd)
CHEMBL265940 ↓ inhibits Mechanistic 831.76 nM (Kd)
CHEMBL259383 ↓ inhibits Mechanistic 588.84 nM (Kd)
CHEMBL259997 ↓ inhibits Mechanistic 2187.76 nM (Kd)
CHEMBL406776 ↓ inhibits Mechanistic 407.38 nM (Kd)
CHEMBL259384 ↓ inhibits Mechanistic 107.15 nM (Kd)
GENISTEIN ↓ inhibits Mechanistic 39810.72 nM (Kd)
CHEMBL408649 ↓ inhibits Mechanistic 28840.32 nM (Kd)
CHEMBL265178 ↓ inhibits Mechanistic 10964.78 nM (Kd)
ALDOSTERONE ↓ inhibits Mechanistic 4786.3 nM (Kd)
CHEMBL408778 ↓ inhibits Mechanistic 2454.71 nM (Kd)
CHEMBL259327 ↓ inhibits Mechanistic 2137.96 nM (Kd)
4-NONYLPHENOL ↓ inhibits Mechanistic 1202.26 nM (Kd)
CHEMBL409022 ↓ inhibits Mechanistic 1148.15 nM (Kd)
ESTRADIOL BENZOATE ↓ inhibits Mechanistic 1148.15 nM (Kd)
ETIOCHOLANOLONE ↓ inhibits Mechanistic 707.95 nM (Kd)
HYDROCORTISONE ↓ inhibits Mechanistic 630.96 nM (Kd)
MEDROXYPROGESTERONE ACETATE ↓ inhibits Mechanistic 630.96 nM (Kd)
NANDROLONE ↓ inhibits Mechanistic 501.19 nM (Kd)
CORTICOSTERONE ↓ inhibits Mechanistic 457.09 nM (Kd)

Showing top 40 of 107 agents ranked by evidence tier.


Clinical Trials

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

NCT07342946 Phase: N/A Started: 2026-12-20

This study will look at whether a reduced-calorie Mediterranean-style eating plan, together with a synbiotic supplement, can improve health measures and quality of life in women who have polycystic ovary syndrome (PCOS) and are overweight or have obesity. Participants will be assigned by chance (lik...

NCT03864068 Phase: 2 Started: 2020-01-24

To determine if Inositol, a dietary supplement, will improve ovarian and adrenal androgen excess in women with Polycystic Ovarian Syndrome(PCOS).

NCT06836128 Phase: 3 Started: 2025-03-01

The study aims to evaluate the effects of combination of metformin with NAC in PCOS on biochemical and hormonal parameters.

NCT06143527 Phase: 1 Started: 2023-11-16

This trial will study the safety and efficacy of cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cells delivered intravenously for the treatment of Polycystic Ovary Syndrome.

NCT07770711 Phase: N/A Started: 2026-08-13

Polycystic ovary syndrome (PCOS) is a common endocrine disorder frequently associated with obesity, insulin resistance (IR), and metabolic dysfunction. Although lifestyle modification is recommended as first-line therapy, the optimal dietary approach for women with PCOS remains uncertain. This rando...

NCT07873294 Phase: N/A Started: 2025-08-01

Polycystic ovary syndrome (PCOS) is one of the most common endocrine and metabolic disorders affecting women of reproductive age. Its prevalence varies considerably according to the population studied and the diagnostic criteria applied. A recent global systematic review and meta-analysis estimated ...

NCT07872501 Phase: N/A Started: 2025-01-01

This study will compare the effectiveness of clomiphene citrate alone with clomiphene citrate combined with metformin for treating anovulation and infertility in women with polycystic ovary syndrome (PCOS). Women with PCOS who have difficulty becoming pregnant because they do not ovulate regularly w...

NCT06896981 Phase: 2/3 Started: 2025-01-01

Polycystic Ovary Syndrome (PCOS) is a common endocrine disorder that is frequently associated with metabolic features, including obesity. Improvement of metabolic manifestations helps in the control of symptoms, including subfertility. Metformin has been commonly used to decrease insulin resistance ...

NCT05680805 Phase: 4 Started: 2023-05-27

The aim of this study is to see the associations of metabolic responses of metformin with single nucleotide polymorphisms (SNPs) (rs628031 and rs2282143) of solute carrier family 22 member 1 (SLC22A1) gene in women with polycystic ovary syndrome (PCOS). This prospective clinical study will be conduc...

NCT07585396 Phase: 2/3 Started: 2024-04-10

Polycystic ovary syndrome (PCOS),is a complex neuro-endocrine disorder affecting approximately 5% to 10% of women in reproductive age.Inositols are considered insulin sensitizers, as they modulate the members of insulin signaling pathways.Thiss study compares between the effect of inositol and metfo...

NCT04257500 Phase: 4 Started: 2020-09-03

A prospective study to determine the metabolic effects of the contraceptive vaginal ring among overweight and obese women with polycystic ovary syndrome (PCOS). We will recruit a total of 40 participants and study use of the vaginal ring over a 4-month period.

NCT06519656 Phase: 1/2 Started: 2024-10-21

The goal of this clinical trial is to learn the efficacy of Mazdutide in treating obese female adults diagnosed with polycystic ovary syndrome (PCOS). The main question it aims to answer is: Does Mazdutide lower the free androgen index (FAI) in obese female adults with PCOS? Participants will take M...

NCT06418347 Phase: 3 Started: 2024-04-01

This research study aims to investigate the effect of adding Alpha lipoic acid (ALA) supplement to letrozole treatment in infertile women with polycystic ovary syndrome (PCOS). The study will include 150 PCOS participants seeking fertility treatment. They will be randomly divided into two groups - c...

NCT07811856 Phase: 4 Started: 2026-08-01

Polycystic ovary syndrome (PCOS) is a common condition associated with metabolic abnormalities, including insulin resistance, impaired glucose regulation, and abnormal lipid levels. Metformin is commonly used to improve metabolic outcomes in women with PCOS, while lifestyle modification and behavior...

NCT05972928 Phase: 2/3 Started: 2024-08-07

The Study aims to determine the proposed positive influence of Sitagliptin in manipulating hormonal , metabolic and inflammatory parameters in the treatment of Polycystic ovary syndrome and subsequent infertility


Agents Found By Disease-Level Search

Searched directly from “PCOS (Polycystic Ovary Syndrome)” 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).

42 agents shown (approved or with trial evidence) — 33 not found by the gene-target search above.

Metformin

Phase 3 · 10 trials · source: ClinicalTrials.gov, Open Targets
NCT03792984 NCT00159536 NCT06015594

Letrozole

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT06418347 NCT02389088

leuprolide acetate

· 2 trials · source: ClinicalTrials.gov
NCT00004311 NCT00349258

hCG

· 2 trials · source: ClinicalTrials.gov
NCT01815138 NCT01815138

Cetrorelix acetateNOT in gene-target list

Phase 3 · 2 trials · source: ClinicalTrials.gov, Open Targets
NCT01185704 NCT01185704

Alpha lipoic acidNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06418347

Vitamin D3NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03792984

Calcium CarbonateNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03792984

Cinnamon Cap 500 MGNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03778099

OCP + MetforminNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03229057

Anthropometric and physical examinationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03841981

Indirect calorimetry, accelerometer and seven-day dietary recallNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03841981

Biochemical, hormonal and metabolic phenotypingNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03841981

Sonographic studiesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03841981

Cardiovascular autonomic function studiesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03841981

Oral smear and feces specimenNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT03841981

ECoFamNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07129434

Escitalopram 20 mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05840692

Pioglitazone 30 Mg Oral TabletNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07472179

metformin 500 mg thrice dailyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07472179

degarelix (long acting GnRH antagonist)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01709942

cetrorelix 0.25mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01709942

spironolactone

Phase 2/3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00004311

SIMVASTATIN

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT00575601

Mazdutide

Phase 1/2 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT06519656

Adolescent PCOS group receiving oral contraceptivesNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07127458

Human Chorionic Gonadotropin (hCG)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04071574

GonadotropinsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04071574

GNRH-A TriptorelinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04071574

GnRH antagonistNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04071574

Resveratrol (1000mg twice a day), Myoinositol 1000mg (Twice a day)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04867252

Metformin (500 mg Twice a day), Pioglitazone (15 mg Twice a day)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04867252

liraglutide

Phase 3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT05965908

Clomifene citrate ( clomid 50 mg )NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02544776

AmlodipineNOT in gene-target list

Unknown · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT02544776

Myo-inositolNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05678114

Progesterone

Phase 2/3 · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT06812559

GnRH antagonist (Cetrorelix)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06812559

Letrozole 2.5mgNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06403488

VipdometNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02683226

IncresyncNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02683226

recombinant human chorionic gonadotropinNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00989781

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