Tepotinib is an investigational small molecule inhibitor of the c-Met receptor tyrosine kinase. Alterations of the c-Met signaling pathway are found in various cancer types and correlate with aggressive tumor behavior and poor clinical prognosis. Tepotinib is a potent and selective c-Met inhibitor, >200-fold selective for c-Met than IRAK4, TrkA, Axl, IRAK1, and Mer. Tepotinib is currently in Phase I/II trials in liver cancer and lung cancer.

MK-8031 (also known as Atogepant) is piperidinonylcarboxamideazaindane derivative patented by Merck Sharp & Dohme Corp as CGRP receptor antagonist useful for prevention and treatment of Migraine. A press release in June 2018 announced positive results for MK-8031, in a Phase 2 trial of daily use for episodic migraine prevention. MK-8031appeared to show good efficacy in migraine prevention and no significant liver toxicity signal at any dose despite daily dosing for 3 months. Phase III clinical trial was initiated in 2019 and currently in progress.

Maralixibat (SHP625; formerly LUM001 or lopixibat) is a potent, apical, sodium‐dependent, bile acid transporter competitive inhibitor with minimal systemic absorption. Maralixibat works by reducing systemic levels of bile acids. In animal models of cholestasis, maralixibat blocked reabsorption of bile acids in the terminal ileum, thereby reducing enterohepatic recirculation to the liver and increasing fecal excretion of bile acids. Maralixibat is being evaluated as a treatment for children with rare cholestatic liver diseases, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Vericiguat, discovered at Bayer, is the first soluble guanylate cyclase (sGC) stimulator. Vericiguat is currently being studied in a Phase III clinical program for the treatment of heart failure with reduced ejection fraction (HFrEF).

Cabotegravir is an investigational drug that is being studied for the treatment and prevention of HIV infection. Cabotegravir belongs to a class (group) of HIV drugs called integrase inhibitors. Integrase inhibitors block an HIV enzyme called integrase. (An enzyme is a protein that starts or increases the speed of a chemical reaction.) By blocking integrase, integrase inhibitors prevent HIV from multiplying and can reduce the amount of HIV in the body. Cabotegravir does not require boosting with an additional drug. Two forms of cabotegravir are being studied: tablets that are taken by mouth (known as oral cabotegravir or oral CAB) and a long-acting injectable form that is injected into the muscle (known as cabotegravir LA or CAB LA; LA stands for "long-acting"). (A long-acting drug formulation works over a long period of time. Using this type of drug might mean that the drug could be taken less often, making a treatment or prevention regimen simpler to take.) Cabotegravir is in Phase-III clinical trials for HIV infections.

OTL-0038 or OTL-38, a fluorescent-labelled folate receptor-α (FRα) targeted imaging agent that accumulates in vivo in tumor cells expressing FR. OTL38 is currently in ongoing Phase 3 clinical trial in ovarian cancer and successfully completed phase 2 clinical trial in lung cancer. OTL38 is being evaluated for its ability to help surgeons locate and remove hard-to-find cancerous lesions that are often widespread. In 2014, the OTL-38 molecule was granted orphan drug status which can be given to the maker of a drug that treats rare conditions or diseases and offers protection from competition for a period of time. In addition, OTL-38 was studied in phase II clinical trials in the Netherlands for the diagnosis of endometriosis.

Fexinidazole is an antiparasitic drug, which is in the phase III of clinical trial for the treatment of Human African Trypanosomiasis, and in the phase II for the treatment Disease, Chagas and Visceral Leishmaniosis. However, for the Visceral Leishmaniosis, studies were terminated, due to lack of efficacy. Fexinidazole rapidly metabolized to two active metabolites, a sulfone and a sulfoxide, which prolong the pharmacological action of parent drug. These metabolites retaine trypanocidal activity but are less effective in nifurtimox-resistant lines, which can lead to the potential danger in the use of fexinidazole as a monotherapy.

Viloxazine is an antidepressant drug was used to treat patients with depression. Viloxazine inhibits noradrenaline uptake. This drug was approved in some Europe countries, but not in the USA, but then it was discontinued because of competition from other drugs. In the frame of drug repositioning, Viloxazine participated in clinical trials for the treatment of attention deficit hyperactivity disorder. Phase II of trials was successfully passed.