Sameridine was developed as a compound with both local anesthetic and opioid properties (partial micro-opioid receptor agonist). This drug participated in clinical trials when administered intrathecally to provide anesthesia for surgery and extended postoperative analgesia. However, further development of this drug was discontinued.

PF-06700841 is an inhibitor of JAK1 and TYK2 kinases. PF-06700841 tosylate salt is potentially a treatment of systemic lupus erythematosus and plaque psoriasis.

Rivanicline (also known as RJR-2403 or TC-2403) is a partial neuronal nicotinic acetylcholine receptor agonist. This compound binds primarily to the α4β2 receptor subtype. Rivanicline was originally developed for Alzheimer’s disease and shows pronounced anti-amnesic and increased recognition memory in rats. Because rivanicline also inhibits Interleukin-8 production, it has been further developed as a potential anti-inflammatory treatment for ulcerative colitis (in which nicotine is of therapeutic value but has adverse events). Rivanicline effectively inhibited TNF- and LPS-induced IL-8 production in different cell types, without toxic effects. Rivanicline was also evaluated as a potential compound for the development of nicotinic therapeutics to treat neurological diseases in cases of compromised cholinergic neurotransmission.

AT-406 (DEBIO-1143, SM-406), is a potent and orally bioavailable Smac mimetic and an antagonist of the inhibitor of apoptosis proteins (IAPs). AT-406 inhibits cancer cell growth in various human cancer cell lines. It has good oral bioavailability in mice, rats, non-human primates, and dogs, is highly effective in induction of apoptosis in xenograft tumors, and is capable of complete inhibition of tumor growth. Debiopharm under a licence from Ascenta Therapeutics is developing AT-406 for the treatment of cancers.

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.