CTP-656 is a deuterium-modified form of ivacaftor, an FDA-approved drug for the treatment of cystic fibrosis. CTP-656 is jointly developed by Concert Pharmaceuticals and Vertex Pharmaceuticals, and is believed to have higher metabolic stability, lower toxic byproducts and increased half-life compared to the original. CTP-656 acts as a potentiator of is a cystic fibrosis transmembrane conductance regulator (CFTR) protein. CTP-656 is investigated in phase 2 clinical trials for the treatment of cystic fibrosis.

Xanomeline (LY-246,708) is an orthosteric muscarinic acetylcholine receptor (mAChR) agonist, often referred to as M1/M4-preferring. It is also known to act as a M5 receptor antagonist. Xanomeline was studied in clinical trials phase I in schizophrenia. In Phase II clinical trials in Alzheimer’s patients, xanomeline significantly improved several measures of cognitive function, yet produced unwanted side effects that limited patient compliance. The side effects seem to be associated with rapid metabolism of the alkyloxy side chain following oral administration, resulting in a nonselective, yet active compound with limited therapeutic utility. Despite a second Phase II clinical trial with a patch formulation, the liabilities of xanomeline still outweigh its benefits.

Gavinostat is an orally bioavailable hydroxymate inhibitor of histone deacetylase (HDAC) with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities. Gavinostat inhibits class I and class II HDACs, resulting in an accumulation of highly acetylated histones, followed by the induction of chromatin remodeling and an altered pattern of gene expression. At low, nonapoptotic concentrations, this agent inhibits the production of pro-inflammatory cytokines such as tumor necrosis factor- (TNF-), interleukin-1 (IL-1), IL-6 and interferon-gamma. It is currently in phase 2 trials for Myeloproliferative disorders, Polycythaemia vera and Phase III for Duchenne muscular dystrophy announced. In clinical trials of givinostat as a salvage therapy for advanced Hodgkin's lymphoma, the most common adverse reactions were fatigue, mild diarrhea or abdominal pain, moderate thrombocytopenia, and mild leukopenia.

SSR-125543 is a potent, selective, and orally active corticotropin-releasing factor 1 receptor (CRF1) antagonist (Ki value of 2nM). SSR-125543 attenuates long-term cognitive deficit induced by acute inescapable stress in mice, independently from the hypothalamic pituitary adrenal axis. SSR-125543 prevents stress-induced cognitive deficit associated with hippocampal dysfunction. SSR-125543 had been in phase II clinical trials by Sanofi for the treatment of Post-traumatic stress disorder. It is also in phase I trials for the treatment of anxiety. The compound had also been in phase II clinical trials for the treatment of major depression. However, in 2011, the research was discontinued.

Flurpiridaz F-18, a fluorine 18-labeled agent was developed by Lantheus Medical Imaging for the diagnosis of coronary artery disease (CAD), the most common form of heart disease, which affects an estimated 15.5 million Americans 20 years of age or older. Flurpiridaz F 18 is an investigational positron emission tomography (PET) myocardial perfusion imaging (MPI) agent. GE Healthcare has launched a second Phase III clinical trial for the use of Flurpiridaz 18F Injection in PET myocardial perfusion imaging (MPI) to detect CAD.

Elafibranor (GFT505) is an agonist of the peroxisome proliferator-activated receptor-α and peroxisome proliferator-activated receptor-δ. GFT505 has an active metabolite, GFT1007, and both have potent agonist activity for PPAR-a and to a lesser extent for PPAR-d. Elafibranor improves insulin sensitivity, glucose homeostasis, and lipid metabolism and reduces inflammation. Elafibranor (GFT505) reverses nonalcoholic steatohepatitis (NASH) to prevent fibrosis progression. With an outstanding safety and tolerance profile, elafibranor provides NASH patients with needed cardio-protective benefits. Elafibranor is currently being evaluated in the clinical Phase 3 study RESOLVE-IT. The safety profile of GFT505 from the completed clinical trials appears satisfactory with no indication of PPAR-g agonist effects such as edema or body weight gain.