Elamipretide or SS-31 (D-Arg-2', 6'-dimethyltyrosine-Lys-Phe-NH2) is a mitochondria-targeted antioxidant. Elamipretide, is a peptide compound that readily penetrates cell membranes, and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin. In preclinical or clinical studies, elamipretide increases mitochondrial respiration, improves the electron transport chain function and ATP production and reduces formation of pathogenic ROS levels. This elamipretide-cardiolipin association has been shown to normalize the structure of the inner mitochondrial membrane, thereby improving mitochondrial function. Functional benefit is achieved through improvement of ATP production and interruption and potential reversal of damaging oxidative stress. Stealth BioTherapeutics is investigating elamipretide in late stage clinical studies in three primary mitochondrial diseases—primary mitochondrial myopathy, Barth syndrome and Leber’s hereditary optic neuropathy – as well as an earlier stage clinical study in dry age-related macular degeneration.

Toreforant (JNJ-38518168) is an orally active, selective histamine H4 receptor antagonist. Toreforant was investigated as an agent for the treatment of active rheumatoid arthritis. While some improvement in rheumatoid arthritis signs/symptoms through week 12 was observed in the phase IIa study, no efficacy was observed with toreforant at lower doses in a subsequent phase IIb study. Toreforant has been tested in clinical studies in patients with asthma and psoriasis. Toreforant failed to provide therapeutic benefit in population of patients with uncontrolled, eosinophilic, persistent asthma. Toreforant efficacy at 30 and 60 mg was greater than placebo but did not meet predefined success criterion in patients with moderate-to-severe psoriasis.

Pantetheine is the mercaptoethyl conjugated amide analog of pantothenic acid (Vitamin B5), an intermediate in the production of coenzyme A by the body. Pantetheine is part of two larger compounds (coenzyme A and acyl-carrier protein) that promote a large number of metabolic reactions essential for the growth and well-being of animals. Pantetheine has been found to ameliorate symptoms in various disease models but specifically in Pantothenate Kinase-Associated Neurodegeneration (PKAN). Pantetheine is usually administered in its disulfide form (i.e. pantethine) since pantethine is commercially available and is reduced to pantetheine in biological systems and pantethine was hydrolyzed to pantetheine and pantothenic acid prior to absorption. The applicability and efficacy of pantethine (therefore also pantetheine) as a clinical therapeutic however is hampered since both forms can be degraded by pantetheine present in the body.

Adomeglivant, also known as LY2409021, is a potent and selective glucagon receptor antagonist. Adomeglivant lowers blood glucose in healthy people and in those with type 2 diabetes. Blockade of glucagon signalling in patients with type 2 diabetes is well tolerated and results in substantial reduction of fasting and postprandial glucose with minimal hypoglycaemia, but with reversible increases in aminotransferases. Adomeglivant had been in phase II clinical trials by Eli Lilly for the treatment of type 2 diabetes mellitus. However, this research has been discontinued.

Sevitropium is a bronchospasmolytic agent.