Galicaftor (ABBV-2222/GLPG-2222) is an orally bioavailable small molecule that functions as a CFTR corrector, demonstrating potential for therapeutic development in cystic fibrosis research.

Sufugolix(TAK-013) is a non-peptide, orally-active, selective antagonist of the gonadotropin-releasing hormone receptor (GnRHR) (IC50 = 0.1 and 0.06 nM for affinity and in vitro inhibition, respectively).

Mavacamten（MYK-461）is a small molecule that reduces contractility by decreasing the ATPase activity of the cardiac myosin heavy chain.

GSK-2881078 is an orally active and nonsteroidal selective androgen receptor modulator (SARM) which act as partial AR agonists in androgenic tissues while mainly as complete AR agonists in synthetic metabolic tissues,induces AR-mediated transcriptional activation in PC3(AR)2 cells (EC50 = 3.99 nM) and the effect can be inhibited by the non-steroidal AR antagonist Bicalutamide. GSK-2881078 can be used in research of muscle weakness and cachexia associated with both chronic and acute illness.

Ligandrol (LGD-4033) is a novel nonsteroidal, oral SARM that binds to androgen receptor with high affinity (Ki of 1 nM) and selectivity.

SAR-100842 (SAR100842) is a potent, selective, orally available antagonist of the Lysophosphatidic Acid 1 Receptor (LPA1 receptor) with IC50 of 31 nM in β-arrestin assays.

Trofinetide (NNZ-2566) is an analog of a molecule which is derived from IGF-1 and occurs naturally in the brain. IGF-1 is a growth factor stimulated by growth hormone.

MGL-3196 is a potent and highly selective thyroid hormone receptor β agonist in clinical trials for the treatment of dyslipidemia.

GBT-440(Voxelotor) is a novel small molecule hemoglobin modifier which increases hemoglobin oxygen affinity.

Leniolisib (CDZ173) is a potent and selective PI3Kδ inhibitor currently in phase II/III clinical trials for the treatment of immunodeficiency disorders.

Octreotide acetate, a long-acting synthetic analog of native somatostatin, inhibits growth hormone, glucagon, and insulin more potently.

CWHM-12 a novel small molecule inhibitor of αV integrins with IC50s of 1.8/0.8/1.5/0.2 nM for αvβ1/αvβ3/αvβ8; less potency on αvβ5(IC50=61 nM) and on inhibition on αIIbβ3/α2β1/α10β1.

A nontoxic heat shock protein (HSP) coinducer and potentiator of the heat shock response.

TD139 is a novel high-affinity inhibitor of the galectin-3 carbohydrate binding domain with a Kd of 14 nM.

Emixustat hydrochloride strongly inhibits 11-cis-retinol production with IC50 values of 232 ± 3 nM.

Ibiglustat (Genz-682452,SAR402671), is a potent and selective Glucosylceramide synthase inhibitor and ceramide glucosyltransferase inhibitor.

CP-547632 is as a potent inhibitor of the VEGFR-2 and basic fibroblast growth factor (FGF) kinases (IC50 11 and 9 nM, respectively).

Tropifexor (LJN452) is a highly potent non-bile Acid FXR Agonist for the Treatment of Cholestatic Liver Diseases and Nonalcoholic Steatohepatitis (NASH).

Tecovirimat (ST-246, Tpoxx) is a potent, orally bioavailable, first-in-class inhibitor of orthopoxvirus egress with EC50 of 0.01-0.6 uM against a panel of Orthopoxviruses; ST-246 is active against multiple orthopoxviruses, including vaccinia, monkeypox, c

Sivelestat(ONO5046; LY544349; EI546) is a competitive inhibitor of human neutrophil elastase(IC50 = 44 nM; Ki=200 nM); also inhibited leukocyte elastase obtained from rabbit, rat, hamster and mouse.

Senicapoc(ICA17043) is a potent and selective Gardos channel blocker; blocked Ca(2+)-induced rubidium flux from human RBCs/ inhibited RBC dehydration with IC50s of 11 nM/30 nM, respectively. IC50 value: 11/30 nM [1] Target: Gardos channel in vitro: I

Selonsertib（GS-4997）is an orally bioavailable inhibitor of apoptosis signal-regulating kinase 1 (ASK1), with potential anti-inflammatory, antineoplastic and anti-fibrotic activities.

SAR 1118 is a potent tetrahydroisoquinoline (THIQ)-derived LFA-1/ICAM-1 antagonist for treating dry eye.

Rapamycin (Sirolimus, AY-22989, WY-090217) is a specific mTOR inhibitor with IC50 of ~0.1 nM. -

Oltipraz is a potent Nrf2 activator and a potent inducer of Phase II detoxification enzymes, most notably glutathione-S-transferase (GST). Phase 3.

ND-630 is an acetyl-CoA carboxylase (ACC) inhibitor; inhibits human ACC1 and ACC2 with IC50 values of 2.1 and 6.1 nM, respectively.

JNJ-10229570 (JNJ10229570) is a novel potent, selective melanocortin receptor MC1R and MC5R antagonist with binding IC50 of 270 nM and 200 nM, respectively.

Ivacaftor (VX-770, Kalydeco) is a potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM, respectively.

INT-747(Obeticholic acid; 6-ECDCA) is a potent and selective FXR agonist(EC50=99 nM) endowed with anticholestatic activity.

Ilaprazole (IY-81149) is a proton pump inhibitor; inhibits H+/K+-ATPase with an IC50 of 6.0 μM.