Fluasterone is a fluorinated derivative of an endogenous steroid hormone androstenolone (dehydroepiandrosterone, DHEA). According to in vivo studies, fluasterone possess endocrinologic effects manifested in increased estrous cycle length and decreased the weights of the uterus, prostate, seminal vesicles, and testes. The mechanism of action of fluasterone is not yet fully elucidated, but most likely involve inhibition of glucose-6-phosphate hydrogenase. Fluasterone was developed by Aeson Therapeutics and was investigated in the late 1990s for the treatment of asthma, cancer, cardiovascular and metabolic disorders. The development of fluasterone for discontinued, probably due to a combination of low potency and insufficient oral bioavailability. Later, the development of fluasterone was continued by the company SteroTherapeutics. In 2018 the FDA has granted an orphan drug designation for fluasterone for the treatment of nonalcoholic fatty liver disease, nonalcoholic steatosis, and hyperglycemia in patients with Cushing’s syndrome.

Isobutyramide (VX-366) is an orally available branched chain amide that may offer an alternative to current treatments for beta-hemoglobinopathy. Isobutyramide has been shown to increase fetal hemoglobin (HbF) in patients with beta-hemoglobinopathies. Isobutyramide was originated at the Children's Hospital Oakland Research Institute which, in 1993, granted exclusive worldwide rights for the agent to Vertex. However, development of Isobutyramide has been discontinued for sickle cell anaemia and thalassaemia.

SC-41930 has been shown to be a specific LTB4 receptor antagonist both in vitro and in vivo. SC-41930 can produce significant anti-inflammatory effects. The anti-inflammatory activity of SC-41930 could be attributed to postreceptor inhibition of inflammatory mediator production by human neutrophil and other cells in addition to antagonism of human neutrophil LTB4 receptors.

Gossypol is a substance that is found in the cotton plant. It is removed from the seeds and used for medicine. Gossypol is effective as a nonhormonal male contraceptive; however, it has been documented to have irreversible effects on male fertility. Gossypol is reported to exhibit antioxidant, anticancer, antivirus, antiparasitic, and antimicrobial properties and lower plasma cholesterol. Nausea, emesis, anorexia, diarrhea, altered taste sensation, small intestine obstruction, and fatigue have been recorded in clinical trials as adverse reactions. Large amounts of gossypol can decrease potassium levels in the body. Low potassium levels can increase the side effects of digoxin.

DMP-777 (also termed L-694458) a cell permeant b-lactam inhibitor of a human leukocyte elastase was developed for treatment cystic fibrosis, juvenile rheumatoid arthritis.

Leupeptin is produced by various species of Actinomycetes. It strongly inhibits proteolysis by plasmin, trypsin and papin. Leupeptin is well absorbed through oral route. Leupeptin has been known to cause various neuropathological changes in vivo resembling those of aging or neurodegenerative processes in the human brain, including the accumulation of neuronal processes and neuronal cytoskeletal abnormalities leading to neurofibrillary tangle (NFT)-like formations. In in vitro experiments, leupeptin protects the heart from myocardial stunning. Leupeptin was found to inhibit tumorigenesis in mouse skin induced by a single, noncarcinogenic dose of 7,12- dimethylbenz(a)anthracene followed by repeated application of croton oil. Tumors that had already been induced were scarcely affected by leupeptin.

Etiocholanedione is a natural metabolite of dehydroepiandrosterone with potent antiobesity activity. Etiocholanedione has been identified as a metabolite of an altered androgen metabolism that eventually leads hepatocellular carcinoma to impaired hormone responsiveness in human. Etiocholanedione has been identified as a metabolite of 17alpha-hydroxyprogesterone in some patients affected by congenital adrenal hyperplasia, although it doesn't appear to account for the masculinization observed in congenital hyperplasia.

Cyclocreatine is a nearly planar creatine analog. Cyclocreatine is an efficient substrate for creatine kinase, but its phosphorylated form is a poor phosphate donor in comparison with creatine phosphate. It exerts antitumor, antiviral, neuroprotective activity.

Allopurinol riboside is a metabolite of allopurinol, a xanthine oxidase inhibitor indicated for the management of patients with leukemia, lymphoma, and solid tumor malignancies who are receiving cancer therapy which causes elevations of serum and urinary uric acid levels and who cannot tolerate oral therapy. Allopurinol riboside is not an inhibitor of xanthine oxidase. Allopurinol riboside is commonly thought to be directly synthesized by purine nucleoside phosphorylase (PNP) in vivo. Allopurinol riboside competitively inhibits the action of PNP on inosine in vitro. Allopurinol riboside potently inhibits growth in vitro of promastigotes of Leishmania species. Patients with American cutaneous leishmaniasis who received allopurinol riboside had clinical improvement. In addition, allopurinol riboside demonstrated some effectivity against Trypanosoma cruzi infections in animals.