Sulfo-ara-F-NMN (CZ-48) is a mimetic of nicotinamide mononucleotide (NMN). Sulfo-ara-F-NMN acts selectively, activating SARM1 but inhibiting CD38 (IC50 around 10 μM). Sulfo-ara-F-NMN induces intracellular cyclic ADP-ribose (cADPR) production.

DSRM-3716 (DSRM3716, 5-iodoisoquinoline) is a potent and selective inhibitor of NADase SARM1 with IC50 of 75 nM, displays selectivity against various NAD metabolizing and pathway enzymes (e.g., NAMPT and NMNAT).

LGD 3303 is an androgen receptor agonist.1 It selectively binds to the androgen receptor (Ki = 0.9 nM for the human receptor) over mineralocorticoid, glucocorticoid, and progesterone receptors (Kis = 1,261, 581, and 136 nM, respectively, for the human receptors). It also selectively induces reporter transcription in a reporter assay using CV-1 cells expressing the androgen receptor (EC50 = 3.6 nM) over CV-1 cells expressing the mineralocorticoid or progesterone receptors (EC50s = 3,695 and 2,233 nM, respectively). LGD 3303 (3 mg/kg per day) increases levator ani muscle mass, but not prostate muscle mass, in orchiectomized (ORDX) male rats. It increases body weight and gastrocnemius mass, bone mineral content and density in the lumbar spine and mid-femur, and bone formation rate in ovariectomized (OVX) female rats when administered at a dose of 3 mg/kg per day. LGD 3303 (30 mg/kg per day) increases the time spent in the male area in a sexual preference test by sexually experienced, but not sexually inexperienced, estradiol benzoate- and progesterone-primed OVX female rats.

MK-3984 is a selective androgen receptor modulator (SARM). MK-3984 can be used for the research of muscle wasting associated with cancer.

CMP05 (Endosomolytic compound CMP05) is a novel endosomolytic small-molecule compound that exhibits higher transfection efficiency/ability to induce endosomal escape of oligonucleotides (ONs) than current compounds such as chloroquine.

BC-DXI-32982 is a potent inhibitor of the interaction between DX2 and KRAS4B with a half-maximal inhibitory concentration (IC50) of 0.18 μM, showing little effect on the PRKACA-PRKAR2A interaction.

CTIBD is a novel potent activator of the BKCa channel with EC50 of 3.9 uM.CTIBD showed significantly higher potency compared with three other known BKCa activators: NS 1619, NS 11021, and rottlerin.CTIBD induced a reversible potentiation of macroscopic outward currents of the BKCa channel, CTIBD activates both rSlo/rβ1 and rSlo/rβ4 coexpressed channels mainly by decreasing the closing rate of the channel.CTIBD concentration-dependently reduced ACh-induced contractions in isolated rat urinary bladder strips. CTIBD effectively restored frequent voiding contraction and lowered voiding volume without affecting other bladder function parameters in acetic acid-induced overactive bladder (OAB) model (i.p. 20 mg/kg).

Topical nonsteroidal antiandrogen (NSAA)

Ezeprogind (AZP-2006) is an orally active neurotrophic inducer. Ezeprogind targets all causes of neurodegeneration and is not only aiming at markers such as Abeta protein or tau protein. Ezeprogind is a potent neuroprotectant and can be used for the research of neurological disorders, including progressive supranuclear palsy (PSP), tauopathies, alzheimer’s and parkinson’s diseases, et al.

Z-Arg-Lys-AOMK is a potent, selective, irreversible neutral pH 7.2 inhibitor of Cathepsin B with IC50 of 13 nM, >100-fold higher potent than at pH 4.6 (IC50=1,830 nM). Z-Arg-Lys-AOMK displayed high specificity for cathepsin B compared to other lysosomal cysteine cathepsins. Z-Arg-Lys-AOMK selectively inhibits cathepsin B cleavage of peptides at neutral cytosolic pH compared to acidic lysosomal pH conditions. Z-Arg-Lys-AOMK completely inhibited cathepsin B in human neuroblastoma cell lysates at 1 uM. Z-Arg-Lys-AOMK is cell permeable and inhibits intracellular cathepsin B.

GABA-A modulator, blocking the sedative effects without lowering the convulsion threshold

ADAM9i is a specific small molecule inhibitor of ADAM9 protein with binding KD of 5.9 uM and enzyme IC50 of 5.2 uM, promotes the selective degradation of KRAS and sensitizes pancreatic cancers to chemotherapy.

HTT-D3 is a potent, CNS-penetrant, orally bioavailable huntingtin (HTT) splicing modulator, affects HTT pre-mRNA splicing and reduces HTT expression with IC50 of 8 nM.Oral administration of HTT-D3 resulted in dose-dependent and approximately equivalent mHTT protein lowering in both brain and peripheral tissues in mouse models carrying human mHTT transgene, BACHD and Hu97/18.HTT-D3 (10 mg/kg) reduced mHTT mRNA and protein levels in BACHD brain in BACHD mice.

Benznidazole (Ro 07-1051) is an antiparasitic medication, with an IC50 of 20.35 μM for Colombian T. cruzi strains, and has been used in the treatment of Chagas disease.

CO23 is a selective thyroid hormone receptor (TR) α agonist and used for growth and development regulation. CO23 was able to be transported through the blood-brain barrier. CO23 (ip; 0.04-5.0 nmol/g) has ≈50% cholesterol reduction with 0.8 nmol/g dose and higher doses are equally effective. CO23 has no effect at the lowest dose of 0.04 nmol/g and increases Dio1 mRNA 5-, 10-, and 15-fold at the doses of 0.8, 2.5, and 5 nmol/g, respectively.

ZK53 is a selective activator of mitochondrial caseinolytic protease P (HsClpP). It exhibits an EC50 of 1.37 μM for α-casein hydrolysis by HsClpP. ZK53 treatment allosterically activates HsClpP to uncontrollably degrade the essential mitochondrial proteins and is inactive toward bacterial ClpP proteins.

Novel Eph antagonist, inhibiting Eph/ephrin interactions, enhancing glucose-stimulated insulin secretion (GSIS), and acting as a hypoglycemic agent

ZH97 is a selective and covalent small-molecule inhibitor of BFL-1 protein with binding Ki of 0.41 uM, >200-fold selectivity over MCL-1.ZH97 does not bind to BCL-xL, BCL-2, TEAD2, TEAD4, MDM2, MDMX, BRD2 BD1, BRD2 BD2, BRD3 BD1, BRD3 BD2, BCL9, PD1/PD-L1 (Ki>400 uM).ZH97 modifies BFL-1 at the C55 residue, blocks BFL-1/BID interaction in vitro.ZH97 dose-dependently increased cytoplasmic cytochrome c level in U937 cells, has cell growth inhibition with IC50 of 2.8 uM, 5.5 uM, 7.7 uM, 6.8 uM, and 3.9 uM in U937, Kasumi-1, K562, MM.1S, and MV4-11 cell lines, respectively.ZH97 inhibits BFL-1/PUMA interaction in cell lysate and is effective in cancer cells that harboring highexpression level of BFL-1.

HDL376 is a scavenger receptor class B type I (SR-BI) inhibitor. HDL376 directly inhibits SR-BI-mediated lipid transport in cells and in liposomes reconstituted with purified SR-BI (IC50 = 0.22 μM). HDL376 can be used for the research of atherosclerotic coronary artery disease.

AZD1897 is a PIM1, PIM2, and PIM3 inhibitor with IC50 values of less than 3 nM for these three PIM kinases. AZD1897 exhibits anticancer activity and synergistically inhibits the activity of acute myeloid leukemia (AML) cells in combination with Capivasertib (HY-15431). This synergistic inhibitory effect is achieved through the inhibition of the mTOR and MCL1 pathways.

SID 7969543 is a selective SF-1 (steroidogenic factor 1, NR5A1) inhibitor with an IC50 of 760 nM. SID 7969543 inhibits SF-1-triggered luciferase expression with IC50 of 30 nM. SF-1 is a transcription factor belonging to the nuclear receptor superfamily.

Zagociguat is the stimulator of soluble guanylate cyclase. Zagociguat increases nitric oxide (NO) signaling leading to an increase in cyclic guanosine monophosphate production. Zagociguat has the potential for the research of noncentral nervous system (CNS) disorders.

Isradipine(Dynacirc) is a calcium channel blocker with an IC50 of 34±8 μM.

MAGL-IN-1 is a potent, selective, reversible and competitive inhibitor of MAGL, with an IC50 of 80 nM. MAGL-IN-1 exhibits anti-proliferative effects against human breast, colorectal, and ovarian cancer cells. MAGL-IN-1 blocks MAGL in cell-based as well as in vivo assays.

A potent HCV NS5B RNA polymerase inhibitor.

MW01 is a specific inhibitor for DNA damage-induced NF-κB activity, inhibits CLK2 and CLK4 with IC50 of 360 and 14.1 nM, does not inhibit CLK3.

MTB-9655 is a novel, potent and selective Acetyl-Coenzyme A Synthetase 2 (ACSS2) inhibitor, which is currently in Phase I clinical investigation on patients with tumors with high levels of ACSS2.

(S)-CKi-1 is a selective, covalent inhibitor of creatine kinases (CKs), inhibits recombinant creatine kinase B (CKB) phosphotransfer activity with IC50 of 0.6 uM, depletes creatine phosphagen energetics in cells.

DS-2330 is a phosphorous lowering agent for treating hyperphosphatemia in chronic kidney disease..

Balsalazide Disodium is the disodium salt form of balsalazide, an aminosalicylate and oral prodrug that is enzymatically cleaved in the colon to produce the anti-inflammatory agent mesalazine. Mesalazine acts locally on the mucosa of the colon where it diminishes inflammation by blocking the production of arachidonic acid metabolites and other inflammatory agents. Balsalazide disodium is used to treat chronic inflammatory bowel disease.