Monepantel (trade name Zolvix) is a new oral anthelmintic drug from a group of amino-acetonitrile derivatives with broad-spectrum activity against gastrointestinal nematodes of sheep, including adults and L4 larvae of the most important species. Monepantel have a novel mode of action involving a unique, nematode-specific clade of nicotinic acetylcholine receptor (nAChR) subunit ACR-23. Monepantel acts as a positive allosteric modulator of this receptor subunit, which is forced to open on stimulus but cannot close again, resulting in a constant uncontrolled flux of ions and finally in a depolarization of muscle cells leading to paralysis, spasmodic contractions of the anterior portion of the pharynx and ultimately death of adult nematodes. Monepantel-exposed C. elegans also exhibited molting defects and large vacuoles, characteristic for necrosis, are developed. Some nematode species lack ACR-23/MPTL-1 and predicted them to be Monepantel insensitive. Caenorhabditis nematodes equipped with ACR-23/MPTL-1 receptor subunits were found susceptible to Monepantel, whereas Pristionchus pacificus, closely related to these worms but lacking an ACR-23/MPTL-1 homolog, was tolerant. Monepantel shows an excellent tolerability in mammals and it is also active against multidrug-resistant parasites, indicating that its molecular target is absent or inaccessible in the host and is different from those of the classic anthelmintics. Safety pharmacological studies in rats indicate that MOP does not exert negative effects at a dose of 2000 mg/kg. Unfortunately, recent studies from New Zealand and Australia have reported that the efficacy of this new anthelmintic has declined markedly. Lack of efficacy of MOP was confirmed in the slaughtered sheep in which burdens of T. circumcincta and T. colubriformis showed no significant reductions. Whilst these two parasites now appear solidly resistant, the status of O. venulosum remains less clear.

Zaldaride is a calmodulin antagonist known to produce inhibition of calmodulin-dependent voltage-gated ion channels including those of Ca2 , Na , and K . Zaldaride was also observed to inhibit nicotinic acetylcholine receptor (nAChR) channel currents. Zaldaride has been studied in clinical trials as a potential treatment for travelers diarrhea.

CV-205502 (Quinagolide, Norprolac), a small molecule, dopamine 2-receptor agonist was developed by Novartis for the treatment of prolactinoma and hyperprolactinaemia. It is is a long-acting dopamine agonist with potent D2 and weak D1 activity. Quinagolide exists as a racemate and its relevant clinical activity is mediated predominantly by the (-) enantiomer. It is typically present in the hydrochloride salt form and is marketed as oral tablets under the brand name Norprolac contained as a racemate. Quinagolide is currently available in several countries including Canada, but not approved for treatment in the United States. Owing to its dopaminergic action, the drug exerts a strong inhibitory effect on the secretion of the anterior pituitary hormone prolactin, but does not reduce normal levels of other pituitary hormones. Long-term treatment with Norprolac was found to reduce the size or limit the growth of prolactin-secreting pituitary macroadenomas. In some patients the reduction of prolactin secretion may be accompanied by short- lasting, small increases in plasma growth hormone levels, the clinical significance of which is unknown. As a specific inhibitor of prolactin secretion with a prolonged duration of action, Norprolac has been shown to be effective and suitable for once-a- day oral treatment of patients presenting with hyperprolactinaemia and its clinical manifestations such as galactorrhoea, oligomenorrhoea, amenorrhoea, infertility and reduced libido.

Fadrozole (CGS 16949A) is a tetrahydroimidazole-pyridine derivative is a non-steroidal inhibitor of aromatase. In the third phase of clinical trials it was shown, that this drug has good therapeutic effect as a second-line treatment in postmenopausal women with metastatic breast cancer.

Tropisetron (Tropisetron-AFT) is a potent and selective serotonin 3 (5-hydroxytryptamine3; 5-HT3) receptor antagonist with antiemetic properties, probably mediated via antagonism of receptors both at peripheral sites and in the central nervous system. Surgery and treatment with certain substances, including some chemotherapeutic agents, may trigger the release of serotonin from enterochromaffin-like cells in the visceral mucosa and initiate the emesis reflex and its accompanying feeling of nausea. Tropisetron (Tropisetron-AFT) selectively blocks the excitation of the presynaptic 5-HT3 receptors of the peripheral neurons in this reflex, and may exert additional direct actions within the CNS on 5-HT3 receptors mediating the actions of vagal input to the area postrema.

Vildagliptin, previously identified as LAF237, is a new oral anti-hyperglycemic agent (anti-diabetic drug) of the new dipeptidyl peptidase-4 (DPP-4) inhibitor class of drugs. Vildagliptin inhibits the inactivation of GLP-1 and GIP by DPP-4, allowing GLP-1 and GIP to potentiate the secretion of insulin in the beta cells and suppress glucaon release by the alpha cells of the islets of Langerhans in the pancreas. It is currently in clinical trials in the U.S. and has been shown to reduce hyperglycemia in type 2 diabetes mellitus. While the drug is still not approved for use in the US, it was approved in Feb 2008 by European Medicines Agency for use within the EU and is listed on the Australian PBS with certain restrictions. Vildagliptin is marketed under the trade names Galvus, Zomelis.

Piperylone is a non-narcotic analgesic and antispasmodic agent.

ABP-688 C-11 (8 (3-(6-methyl-pyridin-2-ylethynyl)-cyclohex-2-enoneO-11C-methyl-oxime), a noncompetitive and highly selective antagonist for the metabotropic glutamate receptor subtype 5 (mGluR5). It is a promising PET ligand for the quantification of mGlu5 receptors in humans and animals. It readily crosses the blood-brain barrier. The temporal and spatial changes in mGluR5 availability suggest ABP-688 C-11 PET imaging in epilepsy provide abnormal glutamatergic network during epileptogenesis. ABP-688 C-11 was used in receptors imaging in Major Depressive disorder, chronic neuropathic pain, anxiety and other brain disease investigations.

ABL-001 (asciminib), a potent and selective allosteric tyrosine-protein kinase ABL1 inhibitor that is undergoing clinical development testing in patients with Chronic myeloid leukemia (CML) and Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia. is a tyrosine-protein kinase ABL1 inhibitor. In contrast to catalytic-site ABL1 kinase inhibitors, ABL001 binds to the myristoyl pocket of ABL1 and induces the formation of an inactive kinase conformation. ABL001 and second-generation catalytic inhibitors have similar cellular potencies but distinct patterns of resistance mutations, with genetic barcoding studies revealing pre-existing clonal populations with no shared resistance between ABL001 and the catalytic inhibitor nilotinib. ABL001 was tested on mice with a particularly aggressive type of CML. The combination of ABL001 and nilotinib led to complete disease control and eradicated CML xenograft tumors without recurrence after the cessation of treatment. ABL001 is being tested in clinical trials for treatment of CML and Philadelphia Chromosome-positive Acute Lymphoblastic Leukemia alone and in combination with niotinib, imatinib or dasatinib.

Osilodrostat (INN, USAN) (developmental code name LCI-699) is an orally active, non-steroidal corticosteroid biosynthesis inhibitor which is under development by Novartis for the treatment of Cushing's syndrome and pituitary ACTH hypersecretion (a specific subtype of Cushing's syndrome). Osilodrostat specifically acts as a potent and selective inhibitor of aldosterone synthase (CYP11B2) and at higher dosages of 11β-hydroxylase (CYP11B1). Osilodrostat decreases plasma and urinary aldosterone levels and rapidly corrects hypokalemia, in patients with primary aldosteronism and hypertension. At doses ≥1 mg o.d. Osilodrostat markedly increases 11-deoxycortisol plasma levels and blunts ACTH-stimulated cortisol release in ≈20% of patients, consistent with the inhibition of CYP11B1. In patients with resistant hypertension, Osilodrostat produces a non-significant reduction in blood pressure, possibly due to the increase in 11-deoxycortisol levels and the stimulation of the hypothalamic-pituitary-adrenal feedback axis. Because of the lack of selectivity, poor antihypertensive effect, and short half-life, the development of Osilodrostat as antihypertensive was halted. As of 2017, Osilodrostat is in phase III and phase II clinical trials for the treatment of pituitary ACTH hypersecretion and Cushing's syndrome, respectively.