Sephin1 is a guanabenz derivative that binds to and inhibits a regulatory subunit of the stress-induced protein phosphatase 1 (PPP1R15A), but not the constitutive PPP1R15B, and lacks beta2-adrenergic activity. Phosphorylation of eIF2α, α subunit of eukaryotic translation initiation factor 2, reduces protein synthesis and prevents the accumulation of misfolded protein in the endoplasmic reticulum (ER). PPP1R15A recruits the serine/threonine-protein phosphatase PP1 to dephosphorylate eIF2α, so inhibiting PPP1R15A activity prolongs the phosphorylation of eIF2α and aids in its prevention of the accumulation of misfolded protein. In vitro, Sephin1 protected cells from lethal protein misfolding and cytotoxic ER stress. In vivo Sephin1 prevented Charcot-Marie-Tooth 1B and ALS diseases in mice.

Cetylamine is an aliphatic primary amine that possesses surface-active properties and widely used in water treatment. Cetylamine shows moderate anti-tuberculosis activity. Cetylamine also may be used as a source of fluoride in the prevention of dental caries.

ISATORIBINE, a guanosine analog, is an immunopotentiating agent. It is a selective agonist of toll‐like receptor 7, a pattern-recognition receptor that activates the innate immune response.

Deterenol is a beta-adrenoceptor agonist. It is an effective nonmydriatic and nonmiotic hypotensive agent, which can be used in antiglaucoma treatment.

Fasidotril is a diester prodrug of the active metabolite fasidotrilat. Fasidotrilat inhibited both angiotensin I converting enzyme (ACE, EC 3.4.25.1) and neprilysin (NEP, EC 3.4.24.11, also named neutral endopeptidase, enkephalinase, or atriopeptidase) at nanomolar concentrations (Ki = 9.8 nM against ACE and 5.1 nM against NEP) Fasidotril was being developed for the treatment of myocardial infarction, congestive heart failure and myocardial infarction.

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.

Ciraparantag (PER-977, aripazine), developed by Perosphere Inc., is a small, synthetic, water-soluble, cationic molecule and can reverse the anticoagulation mediated by unfractionated heparin, low-molecular-weight heparin, factor Xa and factor IIa inhibitors, and fondaparinux. It has the potential to be a universal antidote, inhibiting nearly all anticoagulants except vitamin K antagonists and argatroban. In April 2015, ciraparantag received FDA fast-track designation as an investigational anticoagulant reversal agent. Phase I/II trials are currently underway to evaluate the safety and efficacy of PER977 in reversing anticoagulation of edoxaban, LMWH, and UFH.2.