ACP-105 is a novel non-steroidal SARM (Selective Androgen Receptor Modulator).

S-40503 is an orally active and selective androgen receptor modulator. S-40503 significantly increases the bone mineral density (BMD) and biomechanical strength of femoral cortical bone.

LG-121071 is an orally active and selective androgen receptor modulator with a Ki of 17 nM.

TFM-4AS-1 is a selective androgen receptor modulator (SARM), and a potent androgen receptor (AR) ligand with an IC50 of 38 nM. TFM-4AS-1 is a gene-selective agonist.

SARM1 Human Pre-designed siRNA Set A contains three designed siRNAs for SARM1 gene (Human), as well as a negative control, a positive control, and a FAM-labeled negative control.

SARM1-IN-3 (Example 30) is a SARM1 inhibitor. SARM1-IN-3 can be used in the study of chemotherapy induced peripheral neuropathy (CIPN).

SARM1-IN-2 (Example 82) is a SARM1 inhibitor with an IC50 value of <1 μM. SARM1-IN-2 inhibits axonal regrowth, leading to only 6% regrowth after 72 h of exposure. SARM1-IN-2 can be used for the research of axonal degeneration.

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)

YAP-TEAD-IN-1 TFA is a potent and competitive peptide inhibitor of?YAP-TEAD interaction (IC50=25 nM). YAP-TEAD-IN-1 TFA is a 17mer peptide and shows a higher the binding affinity to TEAD1 (Kd=15 nM) than YAP (50-171) (Kd= 40 nM).

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.

CMP-5 HClis a potent, specific, and selective PRMT5 inhibitor, while displays no activity against PRMT1, PRMT4, and PRMT7 enzymes. CMP-5 dihydrochloride selectively blocks S2Me-H4R3 by inhibiting PRMT5 methyltransferase activity on histone preparations. CMP-5 dihydrochloride prevents EBV-driven B-lymphocyte transformation but leaving normal B cells unaffected.

cmp2 is a selective TRPC6 activator suitable for treatment of synaptic deficiency in Alzheimer’s disease hippocampal neurons,Cmp2 selectively activates TRPC6 but not structurally related TRPC3 and TRPC7. Cmp2 exhibits synaptoprotective properties in culture and slices and penetrates the BBB. In vivo study indicated cmp2 (10 mg/kg I.P.) reversed deficits in synaptic plasticity in the 5xFAD mice.

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.

PTC-028 is an orally bioavailable compound that decreases BMI-1 levels by posttranslational modification

BML-286 (Compound 3289-8625) is a small molecule inhibitor of the PDZ domain of dishevelled (Dvl) with KD of 10.6 uM, competitively inhibits the Wnt signaling. Compound 3289-8625 (3 uM) inhibits Wnt signaling, effectively reduces luciferase activity by about 2-fold 293 cell line stably transfected with a luciferase reporter. Compound 3289-8625 is cell-permeable and 3289-8625 (10 uM) blocks Wnt signaling in Xenopus, inhibits Wnt pathway responses in culture and in vivo. Compound 3289-8625 suppresses cell proliferation and reduces β-catenin level in prostate cancer PC-3 cells, decreases the levels of β-catenin in both cytosolic fraction and membrane fraction. Compound 3289-8625 sensitized A2780/Taxol cells to paclitaxel.