Resminostat (4SC-201) is a hydroxamate HDAC inhibitor (a novel class I, IIb, and IV histone deacetylase inhibitor). Resminostat inhibits proliferation of a large variety of rodent and human cancer cell lines, likely via the Akt signalling pathway. Phase I and II clinical trials have evaluated the efficacy and safety of resminostat in the treatment of relapsed or refractory Hodgkin's Lymphoma, hepatocellular carcinoma, advanced colorectal carcinoma, and in patients with advanced stage mycosis fungoides or Sezary syndrome (an aggressive form of blood cancer). Results in patients with hepatocellular carcinoma showed that resminostat (combined with sorafenib) was safe, well-tolerated and displayed early signs of efficacy.

Neridronic acid (6-amino-1-idroxyesilidene-1,1-bisphosphonate) is a nitrogen-containing bisphosphonate licensed in Italy for the treatment of osteogenesis imperfecta and Paget’s disease of bone. The pharmacodynamic profile is similar to that of other nitrogen-containing bisphosphonates and is characterized by its high affinity for bone tissue particularly at sites undergoing a process of remodeling. In growing children affected by osteogenesis imperfect, neridronic acid rapidly increases bone mineral density as measured by dual X-ray absorptiometry and this is associated with a significant decrease in fracture cumulative number. Similar results have been obtained also in newborns (<12-month-old) and in adult patients. In Paget’s disease of bone, 200 mg intravenous neridronic acid is associated with a 65% rate of full remission and a biochemical response (decrease of > 75% of bone turnover markers) in 95% of the patients. Neridronic acid treatment has been reported to be effective also in other skeletal diseases such as osteoporosis, algodystrophy, hypercalcemia of malignancy and bone metastasis. Neridronic acid has been developed only for parenteral use, and it is the only one used as the intramuscular injection. This avoids all the limitations of oral bisphosphonates and may be offered for a home treatment with simple nursing assistants

Tamibarotene (brand name: Amnolake), also called retinobenzoic acid, is orally active, the synthetic retinoid, developed to overcome all-trans retinoic acid (ATRA) resistance, with potential antineoplastic activity against acute promyelocytic leukemia (APL). Tamibarotene is a specific agonist for retinoic acid receptor alpha/beta. It is currently marketed only in Japan and early trials have demonstrated that it tends to be better tolerated than ATRA. It has also been investigated as a possible treatment for Alzheimer's disease, multiple myeloma, and Crohn's disease.

Deuterium Oxide (heavy water) is a stable, non-radioactive isotopic form of water. The heavy water is used as a moderator of neutrons in nuclear power plants. In the laboratory, heavy water is employed as an isotopic tracer in studies of chemical and biochemical processes. Deuterium oxide acts as a true temperature-modifying agent. It protects mammalian cells against hyperthermic damage. Plant cell biologists had made a similar observation. Regarding human health, deuterium oxide has a wide range of proposed applications, such as therapeutic agent against human pancreatic cancer, determination of total body water (TBW), and enhancement of thermal stability of macromolecules, cells and tissue. Other applications are: treatment of hypertension, treatment of virus-based diseases of the respiratory tract and treatment of herpes virus-based diseases of the skin.

Chloropyramine is an antagonist of H1 histamine receptors. It is indicated for the treatment of various forms of allergic reactions. Chloropyramine is a drug capable of (1) inhibiting the biochemical function of VEGFR-3 and FAK, (2) inhibiting proliferation of a diverse set of cancer cell types in vitro, and (3) reducing tumor growth in vivo.

Epalrestat is an aldose reductase inhibitor that is approved in Japan for the improvement of subjective neuropathy symptoms, abnormality of vibration sense, and abnormal changes in heart beat associated with diabetic peripheral neuropathy.

Huperzine A is a plant alkaloid derived from Club moss plant, Huperzine serrata, which is a member or the Lycopodium species. Huperzine-A is in phase III clinical trial in the USA (Alzheimer disease) and is available as a dietary supplement. It selectively and reversibly inhibits acetylcholinesterase. Huperzine A is also a NMDA receptor antagonist, which protects the brain against glutamate induced damage, and it increases nerve growth factor levels. Huperzine A is used for Alzheimer's disease, memory and learning enhancement, and age-related memory impairment. It is also used for treating a muscle disease called myasthenia gravis, for increasing alertness and energy, and for protecting against agents that damage the nerves such as nerve gases. It can cause some side effects including nausea, diarrhea, vomiting, sweating, blurred vision, slurred speech, restlessness, loss of appetite, contraction and twitching of muscle fibers, cramping, increased saliva and urine, inability to control urination, high blood pressure, and slowed heart rate. Various medications used for glaucoma, Alzheimer's disease, and other conditions (Cholinergic drugs) interacts with Huperzine A.

Racecadotril (acetorphan) is an oral enkephalinase inhibitor for use in the treatment of acute diarrhea. Racecadotril reduces hypersecretion of water and electrolytes into the intestinal lumen, by preventing the degradation of endogenous enkephalins. Treatment with racecadotril reduces the incidence and duration of acute diarrhea and reduces diarrhea-associated symptoms compared with placebo in adults. Racecadotril treatment also results in significant reductions in stool output compared with placebo in infants and young children aged 2 months to 4 years with acute diarrhea. Both rotavirus-negative and rotavirus-positive infections appear to respond to treatment in the pediatric populations investigated for this infection. Racecadotril shows similar or slightly reduced efficacy to loperamide in the treatment of diarrhea in adults and children aged up to 10 years. However, in comparative trials, racecadotril was associated with fewer adverse events than loperamide, in particular, post-treatment constipation. Racecadotril is available in France (where it was first introduced in ~1990) and other European countries (including Germany, Italy, the UK, Spain and the Czech Republic) as well as most of South America and some South East Asian countries (including China, India and Thailand), but not in the United States.

Pyronaridine was developed in China and has been registered in that country since the 1980s. Outside China, none of the existing formulations is registered because of the failure to meet international regulatory standards. Pyronaridine is generally active against chloroquine-resistant parasites. Pyronaridine has been investigated for the treatment of Malaria. Pyronaridine targets hematin. Combination of pyronaridine with artesunate was indicated for the blood-stage treatment of both strains of malaria:  P. falciparum and P. vivax.  WHO currently recommends artesunate-pyronaridine in areas where other artemisinin-based combination therapies are failing.

Oteracil is an adjunct to antineoplastic therapy, used in combination with Gimeracil and Tegafur. Gimeracil/oteracil/tegafur combination is approved for the gastric cancer treatment. Oteracil is an orotate phosphoribosyltransferase (OPRT) inhibitor that decreases the activity of 5-fluorocil (tegafur is a prodrug of 5-fluorocil) in normal gastrointestinal mucosa.