Trofolastat (MIP-1404) is a small-molecule inhibitor of PSMA. 99mTc-trofolastat (99mTc-MIP-1404), a small-molecule inhibitor of prostate-specific membrane antigen, shows high potential to detect prostate cancer (PCa) noninvasively using SPECT.

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.

TAK-448 is an investigational oligopeptide analog of kisspeptin and a potent agonist of the GPR54 receptor. In animals, acute TAK-448 administration stimulates luteinizing hormone (LH)/follicle-stimulating hormone release, whereas continuous subcutaneous exposure rapidly down-regulates the pituitary-gonadal axis, with rapid reduction of testosterone levels in a dose-dependent manner. TAK-448 has exhibited potent antitumor activity in rat androgen-dependent prostate cancer models. In accordance with the T reductions, TAK-448 treatment showed also more rapid reduction in plasma prostate-specific antigen(PSA) levels. TAK-448 had been in phase II clinical trials for the treatment of prostate cancer. However, this research has been discontinued.