PTC596 is an orally active and selective B-cell-specific Moloney murine leukemia virus integration site 1 (BMI-1) inhibitor. PTC596 targets BMI1 expressed by both tumor cells and cancer stem cells (CSCs), and induces hyper-phosphorylation of BMI1, leading to its degradation. PTC596 downregulates MCL-1 and induces p53-independent mitochondrial apoptosis in acute myeloid leukemia progenitor cells.

EMI1 (EGFR MaMTH Inhibitor 1) is a novel EGFR ex19del/T790M/C797S inhibitor.EMI1, while potently reducing the interaction of EGFR triple mutant with Shc1 in our MaMTH-DS assay, did not behave as a TKI and displayed no inhibition of the kinase activity of EGFR triple-mutant protein invitro.EMI1 did, however, more strongly inhibit the viability and increase the caspase 3/7 activ-ity of PC9 EGFR ex19del/T790M/C797S triple-mutant cells than noncancerous human bronchial epithelial (HBE) cells, as well as potently reduce PC9 EGFR ex19del/T790M/C797S organ-oid viability.EMI1 had a similar inhibi-tory effect on microtubule plus-end growth in both EGFR-WT and EGFR-C797S triple-mutant cells at 50–100 nM concentration. At 1 µM concentration, EMI1 strongly depolymerized inter-phase microtubules, perturbed spindle formation and induced strong mitotic block in PC9 EGFR ex19del/T790M/C797S cells after 20 h of treatment. EMI1 inhibited interaction of both proteins with EGFR at a level similar to that observed with Shc1, indicating it is not a specific inhibitor of the EGFR-Shc1 PPI interface. Rather, the loss of interaction mediated by EMI1 appears to be due to a more general alteration in EGFR activity.EMI1 induced EGFR degradation, and inhibited the activation of EGFR, ERK, AKT and S6 in PC9-ex19del and PC9-ex19del/T790M cells.

ABBV-744 is a BDII-selective BET bromodomain inhibitor that is being investigated to treat AML and metastic castration-resistant prostate cancer.BBV-744, a highly potent and selective inhibitor of the BD2 domain of BET family proteins with drug-like properties. In contrast to the broad range of cell growth inhibition induced by DbBi, the antiproliferative activity of ABBV-744 was largely, but not exclusively, restricted to cell lines of acute myeloid leukaemia and prostate cancer that expressed the full-length androgen receptor (AR). ABBV-744 retained robust activity in prostate cancer xenografts, and showed fewer platelet and gastrointestinal toxicities than the DbBi ABBV-07514.Analyses of RNA expression and chromatin immunoprecipitation followed by sequencing revealed that ABBV-744 displaced BRD4 from AR-containing super-enhancers and inhibited AR-dependent transcription, with less impact on global transcription compared with ABBV-075. These results underscore the potential value of selectively targeting the BD2 domain of BET family proteins for cancer therapy.

Foxy-5 is a novel Wnt-5A mimicking hexapeptide that inhibits breast cancer metastasis in vivo by targeting cell motility.

FRAX1036 is a novel ATP-competitive small molecule inhibitor of group I p21-activated Kinases (PAKs).

Danicopan (ACH-4471, ACH-0144471) is a highly potent, orally active Factor D inhibitor with Kd of 0.54 nM, inhibits the proteolytic activity of purified Factor D against substrate Factor B in complex with C3b, blocking production of Bb fragment IC50 of 15 nM.

FK962 is an enhancer of somatostatin release, exerts cognitive-enhancing actions. Anti-dementia properties.

A potent, selective, non-RGD-mimetic αIIbβ3 integrin receptor antagonist with IC50 of 45 nM, with no activity on αVβ3 receptors. .

THZ1 is a novel selective and potent covalent CDK7 inhibitor with IC50(binding affinity) of 3.2 nM; inhibits Jurkat cell's proliferation with IC50 of 50 nM.

JA2131 is a selective bioavailable poly(ADP-ribose) glycohydrolase (RARG) inhibitor with IC50 of 0.4 μM.

ζ(ZETA)-Stat (1,3,6-Naphthalenetrisulfonic acid) is a novel PKC-zeta (PKC-ζ) specific inhibitor with IC50 of 5 uM, showed only 13% inhibition on PKC-I at 20 uM.

NI-42 is a potent, selective and orally active BRPF1 bromodomain inhibitor with IC50 of 7.9 nM, with excellent selectivity over nonclass IV BRDs; weakly inhibits BRPF2 (BRD1), BRPF3, BRD9 and BRD4 (BD1) with IC50 of 48, 260, 310 and 4500 nM respectively, and no activity for ATAD2A/2B (IC50>100 uM); shows a modest (GI50=1-10 uM) and selective inhibition of proliferation of certain cancer lines, particularly those lines exhibiting monocytic lineage differentiation; a novel, structurally orthogonal chemical probe for the BRPFs suitable for cellular and in vivo studies.

A potent, orally bioactive 20S proteasome inhibitor with IC50 of 1.3 nM (inhibition of proteasomal chymotrypsin-like proteolytic activity).

A novel anti-TB agent that has an MIC range of 0.06-0.5 ug/mL..

OGG1 inhibitor O8 is a potent, selective inhibitor of 8-oxoguanine DNA glycosylase 1 (OGG1) with IC50 of 0.22 uM, displays no significant activity against other DNA Glycosylases (NEIL1, NTH1, Fpg, IC50>50 uM)..

MYC inhibitor DC-34 is a potent small molecule that selectively inhibits MYC at the transcriptional level only when a G-quadruplex (G4) is present in the promoter, binds to MYC G4 with Kd of 9.4 uM.

Dopamine D2 and 5-HT1A partial agonist in development as a potential treatment for schizophrenia and other psychotic indications; Pharmacoloy profile makes it an atypical antipsychotic and a new approach for the treatment of schizophrenia.

A homoisoflavanone found in Caesalpinia sappan, a potent, selective IMPDH2 inhibitor that covalently binds to Cys140 with Kd of 3.944 nM, almost 10 times lower than the Kd of binding to IMPDH1.

R enantiomer of Hydroxychloroquine. Hydroxychloroquine is a potent drug for the COVID-19 trement.

BKI-1369 is a bumped kinase inhibitor (BKI). BKI-1369 increases human Ether-a-go-go-related gene (hERG)-inhibitory activity with an IC50 of 1.52 μM. BKI-1369 reduces the parasite burden and diseases severity in the gnotobiotic pig model. BKI-1369 has been well characterized for potency, stability, metabolism, toxicity, pharmacokinetics and is potent against C. parvum in infected mice and calves.

BF 227 is a candidate for an amyloid imaging probe for PET, with a Ki of 4.3 nM for Aβ1-42 fibrils.

BNC105 is a novel tubulin polymerization inhibitor that selectively disrupts tumor vasculature and displays single-agent antitumor efficacy.

SBI-553 is a potent and brain penetrant NTR1 allosteric modulator with EC50 of 0.34μM.

Ibrolipim (NO-1886) is an orally active lipoprotein lipase (LPL)-promoting agent. Ibrolipim decreases plasma triglycerides, increases high-density lipoprotein cholesterol levels. Ibrolipim has renoprotective and hypolipidemic effects.

AUT-1 is a novel specific, cell permeant modulator of Kv3 channels with EC50 of 4.7 and 4.9 uM for Kv3.1b and Kv3.2a, respectively.

Nortadalafil is demethyl Tadalafil, which is a PDE5 inhibitor, currently marketed in pill form for treating erectile dysfunction (ED) under the name Cialis; and under the name Adcirca for the treatment of pulmonary arterial hypertension. IC50 value: Ta

Mefuparib(CVL218) hydrochloride is a potent, highly selective, competitive PARP1/2 inhibitor with IC50 of 3.2/1.9 nM, respectively.CVL218 was more potent than Remdesivir in blocking Coronavirus infection of cells and equally as potent as Remdesivir in blocking replication of virus once it has entered the cells.

Estrogen receptor modulator 1 (compound 18) is an orally active and selective estrogen receptor modulator (SERM), with a pIC50 of 0.46. Estrogen receptor modulator 1 induces regression of Tamoxifen-resistant, hormone independent xenograft tumors.

Xanthotoxol is a coumarin and a major component in C. monnieri that has diverse biological activities.

NS-3-008 is a novel small-molecule transcriptional inhibitor of G0s2 (G0/G1 switch 2) with IC50 of 2.25 uM.