“Compound 14d” (Shionogi & Co.) is a highly potent, dose-dependent adenosine monophosphate activated protein kinase (AMPK) activator being investigated for its blood-glucose-lowering effects.

AKB-9778 is a potent, selective inhibitor of vascular endothelial protein tyrosine phosphatase (VE-PTP) with IC50 of 17 pM.

KMN-159 is a potent prostaglandin E2 type 4 (EP4) receptor agonist with Ki of 0.38 nM and shows five fold increase in potency versus KMN-80.

VU6003586 is a novel allosteric modulators of mGlu5 with EC50 of 174 nM.

DS54360155 is an orally potent analgesic without μ-opioid receptor agonist activity.

UCSF7447 is a selective and inverse agonists of melatonin receptor 1 (MT1) with EC50 of 41 nM, and shows 53-fold selectivity for human MT1 over human MT2.

UCSF3384 is a selective and inverse agonists of melatonin receptor 1 (MT1) with EC50 of 21 nM, and shows 31-fold selectivity for human MT1 over human MT2.

UCSF4226 is a selective and inverse agonists of melatonin receptor 1 (MT2) with EC50 of 6.3 nM, and shows 91-fold selectivity for human MT2 over human MT1.

HST5040 is a clinical candidate for the treatment of propionic acidemia (PA) and methylmalonic acidemia (MMA) with EC50 of 0.94 μM in P-CoA-lowering activity.

[18F]PSMA-1007, a positron emission tomography (PET) tracer, specifically targets prostate-specific membrane antigen (PSMA), which is highly expressed in prostate cancer. PSMA-PET is effective especially for regional detection of biochemical recurrence, which significantly affects patient management. Herein, we established and optimized a one-step radiolabeling protocol to separate and purify [18F]PSMA-1007 with a CFN-MPS200 synthesizer for clinical application.

SC9 is an uncompetitive inhibitor of WT dynamin-related protein 1 (Drp1) with IC50 of 270 nM. SC9 completely prevents the LPS-induced decline in cells with fused mitochondria.

N8279 (NCATS-SM8864) is a selective, brain-penetrant small molecule Gαq-biased GHSR1a agonist with binding IC50 of 1.3 uM.N8279 is nearly an order of magnitude (8.9-fold) more potent than the endogenous ligand ghrelin and is a full agonist.iCa2+ EC50 of N8279 is 41-fold more potent than its GHSR1a binding IC50, suggesting possible allosteric activity, which could be competitively inhibited by GHSR1a antagonists YIL781 and JMV2959.N8279 is a weak activator of GHSR1a-mediated, βarr2-dependent cellular responses relative to ghrelin.N8279 requires receptor sites and/or conformational states driven by the GHSR1a ECD that are distinct from ghrelin.N8279 is brain-penetrant and attenuates aberrant DAergic behavior in mice.

CRCD2 is an NT5C2 (cytosolic 5’ nucleotidase II) inhibitor with a kd of 70.9 μM. It enhances the cytotoxic effects of 6-MP and effectively reverses thiopurine resistance mediated by genetic and non-genetic mechanisms of NT5C2 activation in ALL.

SMN2 splicing modulator TEC-1 is a novel specific, CNS penetrant small molecule SMN2 splicing modulator, increases the expression level of FL-SMN2 mRNA and decreases the expression level of Δ7 mRNA.TEC-1 showed higher selectively (>60-fold) on galactosylceramidase and huntingtin gene expression compared to previously reported compounds (e.g., SMN-C3) due to off-target effects on cryptic exon inclusion and nonsense-mediated mRNA decay.TEC-1 modulates SMN2 splicing and displays disease-modifying effects in motor neurons derived from SMA patient iPSCs (GM24468).|TEC-1 rescues the phenotype in a murine model of spinal muscular atrophy (SMA).

DX314 is a potent, specific CYP26B1 inhibitor with IC50 of 108 nM, >15-fold selectivity over CYP26A1.DX314 potentiates all-trans-RA (atRA) gene expression effects in healthy and diseased reconstructed human epidermis (RHE).DX314 potentiates the effects of atRA on the expression and localization of keratin 10 (KRT10), protects barrier function in RHE.DX314 reduces comedonal number, induces epidermal thickening, and increases comedonal profile, while having no effect on transepidermal water loss (TEWL) in treated rhino mice.

C11-MRTX is a lipid-conjugated MRTX849 analogue with a 11-carbon tail. C11-MRTX is a nonaggregating potent cellular inhibitor of K-Ras(G12C).

DX-308 is a potent, selective dual CYP26A1/B1 inhibitor and retinoic acid metabolism blocking agent.DX308 does not interact with off‐target nuclear receptors or CYP450s, is not genotoxic, and is stable in skin, despite vigorous hepatic metabolism.Topical DX308 induces comedolysis and epidermal thickening without apparent adverse effects in vivo.DX308 shows potent modulation of retinoid‐responsive genes by DX308 in both healthy and keratinization disorder keratinocytes (KCs).DX 308 may present an improved therapeutic alternative for the treatment of keratinization disorders and other retinoid‐responsive skin ailments.

BFL-1 inhibitor 4E14 (4E14) is a potent, selective, covalent BFL-1 inhibitor that disrupt BH3-binding activity with IC50 of 1.3 uM, targets a unique C55 residue in the BFL-1 groove.4E14 blocks BFL-1 suppression of BAX-mediated mitochondrial apoptosis.

Fenretinide Glucuronide Monosodium Salt, is the metabolite of Fenretinide (F250000), which is a synthetic retinoid deriverative, substances related to vitamin A. They are also shown to be used for the treatment of cancer, as well as in the treatment of cystic fibrosis, rheumatoid arthritis, acne, and psoriasis.

RCS-03-104-4 is a cysteine-reactive JQ1 analogue, which potently induces BRD4 degradation with DC50 and Dmax of 79 nM and 71%, respectively. RCS-03-104-4 causes BRD4 degradation by inducing its proximity with the ubiquitin ligase activity of DCAF11.

AEP07 (AEP 07) is a selective small molecule inhibitor of PARP4 (vPARP) inhibitor with IC50 of 0.8 uM, >12-fold selective over other PARP family members.

Rv1625c agonist V-59 (V-59) is a specific small molecule agonist of the Mtb adenylyl cyclase Rv1625c, inhibits Mtb growth in macrophages (EC50=0.3 uM) in an Rv1625c-dependent mechanism.V-59 inhibits Mtb growth in cholesterol media (EC50 0.70 uM), but not in media containing the two-carbon fatty acid acetate, or in standard rich growth media.V-59 stimulates Rv1625c to produce cAMP, which is necessary for V-59 to inhibit Mtb growth.Chemically activating Rv1625c via V-59 preferentially inhibits cholesterol utilization in WT Mtb, rather than equally inhibiting all lipid utilization by the bacterium.

Foldamer 33 is a small molecule HSP110 inhibitor, directly binds to the nucleotide-binding domain (NBD) of HSP110, blocks HSP110 chaperone function and colorectal cancer growth.Foldamer 33 significantly inhibit HSP110 anti-aggregating activity with IC50 of 87.8 uM.Foldamer 33 disrupts HSP110-STAT3 interaction with IC50 of 35.9 uM in competitive BLI assays.Foldamer 33 inhibits SW480 colorectal cancer cell proliferation, and (5 mg/kg) Foldamer 33 displays an anti-tumor effect (TGI of 40% and 60%) in mice bearing a colorectal cancer.

Mal-propyl-Ala-Ala-Asn(Trt)-PAB is a hetero bifunctional cross-linker, useful in antibody drug conjugation (ADC).