Tecastemizole (aka Norastemizole) is an H1 receptor antagonist that showed great promise as potential next-generation antihistamine allergy medication. Sepracor developed Norastemizole through phase III clinical trials; however, the FDA rejected the application for approval on the grounds of an unacceptable profile of adverse side-effects including phospholipidosis and cardiomyopathies in animals exposed to the drug.

BMS 599626 is a selective and efficacious inhibitor of HER1 and HER2 with IC50 of 20 nM and 30 nM. BMS-599626 is identified as an ATP-competitive inhibitor for HER1 and as an ATP-noncompetitive inhibitor for HER2. BMS-599626 inhibits the proliferation of tumor cells expressing high levels of HER1 and/or HER2, including Sal2, BT474, N87, KPL-4, HCC202, HCC1954, HCC1419, AU565, ZR-75-30, MDA-MB-175, GEO, and PC9 cells. In a phase I trial of solid tumour patients receiving BMS 599626 no doselimiting toxicities were observed during the first cycle. Grade 1 or 2 drugrelated effects were reported and included diarrhoea, nausea, vomiting, rash, fatigue, musculoskeletal pain/cramp and cough. BristolMyers Squibb discontinued development of BMS 599626 for cancer in July 2015

Sanguinarine is an extract of the bloodroot plant Sanguinaria canadensis, a member of the poppy family. It is an inhibitor of protein phosphatases PP1, PP2C and PP2B in vitro. Also inhibits mitogen-activated protein kinase phosphatase-1 (MKP-1) and other enzymes. Sanguinarine exerts a protective effect in cerebral ischemia, and this effect is associated with its anti-inflammatory and anti-apoptotic properties. It was clinically tested as an agent against gingivitis and tooth plaques.

Mertansine (Maytansine) is a 19–member ansa macrolide structure attached to a chlorinated benzenering. It was originally isolated from the shrub Maytenus ovatus. Mertansine (DM1) is a tubulin inhibitor, it inhibits the assembly of microtubules by binding to tubulin, with a linker structure can create an antibody-drug conjugate (ADC). Mertansine is a potent microtubule-targeted compound that induces mitotic arrest and kills tumor cells at sub-nanomolar concentrations. The antimitotic effect of maytansine has been attributed to its ability to inhibit microtubule assembly by binding to tubulin with a KD of ~ 1 umol/L, at or near the vinblastine-binding site. Experimental ADCs with the SPP-DM1 design include lorvotuzumab mertansine. DM1 can also be linked to an antibody using the SMCC (4-(3-mercapto-2,5-dioxo-1-pyrrolidinylmethyl)-cylohexanecarboxylic acid) linker, in which case the International Nonproprietary Name of the conjugate formed contains the word emtansine. DM1 and its attachment via these linkers result from ImmunoGen Inc research. Trastuzumab emtansine (T-DM1) is an anti-HER2/neu antibody-drug conjugate.

ORY-1001 (known as RG 6016) is a Lysine Specific Demethylase 1 (LSD1, also known as KDM1A) inhibitor (IC50 <20nM) with high selectivity against related FAD dependent aminoxidases (MAO-A/B, IL4I1, KDM1B >100uM, SMOX 7uM). LSD1 is a histone eraser enzyme that removes methyl groups, specifically mono and demethylated H3K4 and H3K9, and by doing so regulates the expression of many genes important in the onset and progression of diseases such as cancer and neurodegenerative disorders. ORY-1001 is a lead drug candidate of Oryzon Genomics S.A. under development for the treatment of acute myeloid leukemia (AML) – phase I/II and small cell lung cancer (SCLC) – phase I. Oryzon has made significant progress with ORY-1001 over the past years.

PHA-543613 was discovered by Pfizer and has been under development primarily as a potential treatment of schizophrenia. PHA-543613 acts as an agonist to the Neuronal acetylcholine receptor protein alpha-7 subunit. A single human trial was conducted in healthy human volunteers, but the compound has been studied extensively in rat models for schizophrenia as well as Parkinson's disease and Alzheimer's disease.

INCB3284 or INCB-3284 Incyte’s internally developed, oral human CCR2 antagonist for the treatment of chronic inflammations. It is in Phase IIa trial of patients with rheumatoid arthritis (RA).

Acteoside (verbsacoside) is the one of the main active phenylethanoid glycosides from Cistanche deserticola, Lantana camara and some others herbs. It is known to have antioxidant and neuroprotective activity, and herbs containing it are used to enhance memory and can be studied for the treatment of Alzheimer's disease. It is known, that amyloid fibrils accumulation in cerebral can easily lead to neurodegenerative disorders. Acteoside has been reported to inhibit Aβ42 aggregation by activating nuclear translocation of the transcription factor NF-E2-related factor 2 (Nrf2), increasing heme oxygenase-1 (HO-1) expression. It has also been shown that acteoside could decrease nitric oxide synthase (NOS) activity and caspase-3 expression. Acteoside is a natural antioxidant product unlike other anti-tumor compounds, is an inhibitor of protein kinase C (PKC). In addition Reh-acteoside, a general acteoside of Rehmannia leaves was studied in phase 2/3 clinical trials for patients with IgA nephropathy.

Olesoxime (TRO19622) a small-molecule with a cholesterol-like structure has remarkable neuroprotective properties for motor neurons in cell culture and in rodents. The biopharmaceutical company Trophos initially developed this compound. This medicine is in phase II clinical trial in treating spinal muscular atrophy and in phase I for patients with stable relapsing remitting multiple sclerosis. This drug was also investigated in phase III clinical trial for amyotrophic lateral sclerosis, but it did not demonstrate a significant increase in survival versus placebo and that study was discontinued. Preclinical studies have demonstrated that the olesoxime promoted the function and survival of neurons and other cell types under disease-relevant stress conditions through interactions with the mitochondrial permeability transition pore (mPTP).

RO-20-1724 is a potent inhibitor of Phosphodiesterase 4 (PDE4) originally developed by Roche. It showed some promise as a potential treatment for psoriasis, but it was discontinued when it could not match the efficacy of existing treatments. RO-20-1724 was also investigated as a potential treatment for asthma and septic shock.