Verucerfont (formerly GSK561679), a corticotropin releasing factor (CRF) receptor 1 antagonist, is being developed by GlaxoSmithKline under the license from Neurocrine Biosciences, for the treatment of post-traumatic stress disorder and congenital adrenal hyperplasia (CAH). Verucerfont is a potent, selective CRF1 receptor antagonist (IC50 for CRF1, CRF2, and CRF-BP ~ 6.1, >1000 and >1000 nM, respectively), and is orally available and brain penetrant. Verucerfont is in phase II clinical trials for the treatment of post-traumatic stress disorder or alcoholism. The compound was also in trials for the treatment of classic congenital adrenal hyperplasia (CAH). However, this research has been discontinued. In 2015, orphan drug designation was received in U.S. for the indication.

Oxamisole (previously known as PR 879-317A), a selective immunomodulatory agent that was studied for the treatment of viral infections. Experiments on mice have shown that antiviral properties of oxamisole were mediated through the immunomodulatory effects of this compound on the immune system. However, further development of this drug was discontinued.

Taziprinone was studied as an antitussive agent. Information about the current use of this compound is not available.

Lisofylline [1-(5R-hydroxyhexyl)-3,7-dimethylxanthine] is a unique metabolite of pentoxifylline. Lisofylline inhibited the generation of phosphatidic acid and free fatty acids. Lisofylline blocked the release of pro-inflammatory cytokines in oxidative tissue injury, in response to cancer chemotherapy and in experimental sepsis. Lisofylline regulates immune cell function and autoimmune response by inhibition of IL-12 signalling and cytokine production. Lisofylline may have therapeutic value in the prevention of autoimmune disorders, including Type 1 diabetes, autoimmune recurrence following islet transplantation, and in preservation of beta cell functional mass during islet isolation.

Turofexorate Isopropyl (XL335) is a potent, selective, and orally bioavailable FXR agonist. Binds to the ligand-binding domain (LBD) of human FXR. Turofexorate Isopropyl resides in a predominately hydrophobic pocket with only a few polar atoms making contact with WAY-362450. Turofexorate Isopropyl promotes transcription of the human BSEP, human SHP, and mouse IBABP genes utilizing reporter constructs with EC50 of 17, 230, and 33 nM, respectively in promoter assays. Turofexorate Isopropyl had been in phase I clinical trials for the treatment of hyperlipidemia. This compound was originally discovered by Exelixis Pharmaceuticals, then licensed to Wyeth (now a wholly-owned subsidiary of Pfizer). However, the studies were discontinued.

Soraprazan (remofuscin), a potassium-competitive acid blocker (P-CAB; formerly called an acid pump antagonist [APA]), is being developed by Katairo GmbH for the treatment of Stargardt's disease and dry age-related macular degeneration (AMD). Clinical development is underway for Stargardt's disease and dry age-related macular degeneration in the Netherlands and Germany. On 13 November 2013, orphan designation was granted by the European Commission to Katairo GmbH, Germany, for soraprazan for the treatment of Stargardt’s disease. Soraprazan is a a highly potent reversible, and fast-acting inhibitor of gastric H,K-ATPase. Soraprazan shows immediate inhibition of acid secretion in various in vitro models and in vivo and was found to be more than 2000-fold selective for H,K-ATPase over Na,K- and Ca-ATPases.

Methastyridone (MK-202), a centrally acting stimulant, was studied with chronic anergic schizophrenics. Information about the current use of this compound is not available.

Methyldesorphine is a synthetic narcotic analgesic derived from morphine. It has no medicinal value in the US. This compound is listed as a Schedule I Narcotic controlled substance under the Controlled Substances Act 1970.

Taselisib (GDC-0032) is an highly selective small-molecule inhibitor of phosphatidylinositol 3-kinase (PI3K) p110-α isoform (PIK3CA). Taselisib is designed to bind to the ATP-binding pocket of PIK3CA to potentially prevent subsequent downstream signaling. Taselisib caused a strong differential growth inhibition in carcinoma cells harbored oncogenic PIK3CA mutations. In preclinical studies, taselisib induced growth inhibition in PIK3CA-mutant xenograft mouse models. Genentech (a Roche subsidiary) is developing taselib primarily for the treatment of solid tumours.