Tazadolene was developed as a novel non-opioid analgesic with antidepressant properties. Experiments on rodents have revealed that unique analgesia properties of tazadolene was due to the ability of this compound to activate both serotonergic and alpha 2 adrenergic antinociceptive systems. Information about the current use of this drug is not available.

KW-2478 is a novel and potent non-ansamycin inhibitor of heat shock protein 90 designed to overcome the limitations, including low water solubility and hepatotoxicity, of 17-allylamino-17-demethoxygeldanamycin (17-AAG). KW-2478 exerts a strong antitumor activity against multiple myeloma (MM) cells with various chromosomal translocations. KW-2478 inhibits cell growth and apoptosis associated with Hsp90 client protein degradation. Recent study results have revealed that KW-2478 is able to deplete Hsp90 client Cdk9 and the phosphorylated 4E-BP1, a transcriptional kinase and a transcription inhibitor respectively, leading to reduced expression of FGFR3, c-Maf, and cyclin D1. KW-2478 suppresses tumor growth and induces the degradation of client proteins in tumors in NCI-H929 s.c. inoculated model at doses of 100 mg/kg or more. KW-2478 reduces both serum M protein and MM tumor burden in the bone marrow in OPM-2/GFP i.v. inoculated mouse model at doses of 100 mg/kg.

Zaurategrast (CDP323) is an ethyl ester prodrug of CT7758, a potent carboxylic acid antagonist of integrin alpha4-beta1 (α4β1) or very late antigen 4 (VLA4). CDP323 was under development with UCB and Biogen Idec for the treatment of multiple sclerosis. Its development was discontinued in 2009 based on inadequate interim efficacy data in a phase II clinical trial.

Vapendavir (BTA-798) is an orally active capsid-binding inhibitor. It is a potent, orally bioavailable, broad spectrum inhibitor of the large group of HRVs. Vapendavir binds tightly to VP1s canyon and disrupts the ability of the capsid to bind to a specific cell surface receptor. This further inhibits the release of the viral RNA into the cell cytoplasm during the viral uncoating process (removing the lipid bilayer), thereby disrupting viral replication. Vapendavir is designed to be dosed orally. Vapendavir is in phase II clinical trials for the treatment of rhinovirus-induced aggravation of pre-existing asthma or COPD. Vapendavir has shown significant results in proof-of-concept studies. Thus far, from the Phase 1/2 studies conducted, vapendavir has demonstrated a desirable clinical pharmacology profile with high bioavailability, linear pharmacokinetic profile, remained unaffected by concomitant medications, and was not exclusively metabolized.

CPP-115 is a gamma-aminobutyric acid aminotransferase activator, developed by Catalyst Pharmaceutical Partners, Inc. The compound does not exhibit other GABAergic or off-target activities and is rapidly and completely orally absorbed and eliminated. In rats, CPP-115 produced inhibition of cocaine-induced increases in extracellular dopamine and in synaptic dopamine in the nucleus accumbens. CPP-115 decreased spasms in the multiple-hit rat model of infantile spasms. CPP-115 was found to have high therapeutic potential for the treatment of cocaine addiction and for a variety of epilepsies, has successfully completed a Phase I safety clinical trial, and was found to be effective in the treatment of infantile spasms (West syndrome).

CH5183284/Debio 1347 is selective and orally available fibroblast growth factor receptors: FGFR1, FGFR2, and FGFR3 inhibitor that has a unique chemical scaffold. By interacting with unique residues in the ATP-binding site of FGFR1, FGFR2, or FGFR3, CH5183284/Debio 1347 selectively inhibits there receptors, but does not inhibit kinase insert domain receptor (KDR) or other kinases. CH5183284/Debio 1347 will provide therapeutic opportunities for patients who have FGFR genetic alterations and patients with acquired resistance to existing FGFR selective inhibitors that contain the common methoxy moieties.

Halofuginone is a low molecular weight quinazolinone alkaloid, and a potent inhibitor of collagen alpha1(I) and matrix metalloproteinase 2 (MMP-2) gene expression. Halofuginone also effectively suppresses tumor progression and metastasis in mice. Halofuginone is a potent inhibitor of collagen a1(I) and matrix metalloproteinase 2 (MMP-2) gene expression. Halofuginone also suppresses extracellular matrix deposition and cell proliferation. Also was shown that halofuginone increased apoptosis in α smooth muscle actin- and prolyl 4-hydroxylase β-expressing cells in mdx diaphragm and in myofibroblasts, the major source of extracellular matrix. The profound antitumoral effect of halofuginone is attributed to its combined inhibition of tumour-stromal support, vascularization, invasiveness, and cell proliferation. HT-100 (delayed-release halofuginone), currently in clinical phase 1b/2a in five U.S. hospitals, is a small molecule drug candidate taken orally for the treatment of Duchenne muscular dystrophy (DMD) patients primarily through its ability to reduce fibrosis and inflammation and promote muscle fiber regeneration. The medicine candidate has been granted orphan drug designation in the U.S. and the EU — meaning it has been commercially undeveloped due to its limited profitability — and fast-track designation in the U.S. — an FDA process that aims to facilitate the development and patients’ reach to novel therapies for unmet medical needs.

Yohimbinic acid, also known as yohimbic acid is an indole alkaloid, which was isolated from dried roots of Rauwolfia serpentina. Yohimbinic acid is a potent inhibitor of a human DNA Topoisomerase I and can inhibit cancer cells growth.

Epetraborole (AN3365 or GSK2251052) is a novel boron-containing antibiotic that inhibits bacterial leucyl tRNA synthetase. It is active against Gram-negative bacteria, including Enterobacteriaceae bearing NDM-1 and KPC carbapenemases, as well as P. aeruginosa. Epetraborole was under development for the treatment of Gram-negative bacterial infections.