Raxatrigine also known as GSK1014802 and CNV-1014802, is a novel analgesic under development by Convergence Pharmaceuticals for the treatment of lumbosacral radiculopathy (sciatica) and trigeminal neuralgia (TGN). It is a novel state dependent small molecule sodium channel blocker that preferentially inhibits the Nav 1.7 ion channel, a therapeutic target implicated by genetics in human pain conditions. Raxatrigine is thought to penetrate the central nervous system and block Nav channels in a novel manner. CNV1014802 was granted orphan drug designation in 2013 by the US Food and Drug Administration (FDA) for the treatment of trigeminal neuralgia.

LY2606368 (Prexasertib) is a small-molecule Chk-1 inhibitors invented by Array and being developed by Eli Lilly and Company. Lilly is responsible for all clinical development and commercialization activities. LY2606368 is advancing in Phase 2 clinical trials for cancer. Prexasertib preferentially binds to and inhibits CHK1 and, to a lesser extent, inhibits CHK2. Chk-1 is a protein kinase that regulates the tumor cell's response to DNA damage often caused by treatment with chemotherapy. In response to DNA damage, Chk-1 blocks cell cycle progression in order to allow for repair of damaged DNA, thereby limiting the efficacy of chemotherapeutic agents. Inhibiting Chk-1 in combination with chemotherapy can enhance tumor cell death by preventing these cells from recovering from DNA damage.

Managlinat dialanetil (MB06322 or CS-917) is an inhibitor of fructose 1,6-bisphphosphatase. Managlinat dialanetil is a bisamidate prodrug and its activation requires a two-step enzyme catalyzed reaction. Metabasis Therapeutics Inc in collaboration with Daiichi Sankyo Co Ltd was developing managlinat dialanetil for the potential treatment of type 2 diabetes.

Nitracrine (Ledakrin, C-283) is an acridine derivative with potential cytostatic and antitumor activities. Nitracrine induces the unwinding of supercoiled DNA and binds to the DNA through intercalation, forming drug-DNA adducts and DNA interstrand crosslinks. This inhibits RNA synthesis, protein production, cell growth, DNA replication and cell proliferation; altogether, this may promote apoptosis. Since cancer cells have increased metabolism and proliferate at an increased rate, nitracrine may induce tumor cell apoptosis. The drug was used in clinics in Poland for the treatment of ovarian, colon, lung and mammary carcinomas. Some undesirable effects observed in clinics: e.g. nausea, vomiting and toxicity and mutagenicity of the drug in S. typhimurium strongly limited the therapeutic use of nitracrine and promoted a search for more suitable analogues.

GSK-2269557 (nemiralisib), a PI3Kδ inhibitor, is in development as an anti-inflammatory drug for the treatment of inflammatory airways disease. Studies in moderate or severe stable COPD patients have shown an acceptable safety and tolerability profile when GSK-2269557 was administered via inhalation. Cough was the most commonly reported adverse event.

Norbolethone is a 19-nor anabolic steroid first synthesized in 1966. During the 1960s it was administered to humans in efficacy studies concerned with short stature and underweight conditions. It has never been reported by doping control laboratories prior to 2001. Norbolethone matches the description for what is described as a "designer steroid. " In fact, Norbolethone was given in clinical trials over 30 years ago and never given the green light. No supply was ever manufactured, and no test was ever developed to detect this substance, yet ironically, it was suspected of being used in the 2000 Olympics based on blood and urine assays done by the IOC. Norbolethone was used in the treatment of idiopathic underweight, prevention of indomethacin-induced intestinal ulcers.

Hafnium oxide is a radio enhancer; its nanoparticles (NBTXR3) were designed for high dose energy deposition within cancer cells when exposed to ionizing radiation. When activated by radiotherapy (RT), NBTXR3 allows for a higher energy deposit than RT alone, yielding an increased tumoral cell death. Nanocrystals of hafnium oxide are under investigation in phase II/III in patients with locally advanced soft tissue sarcoma (STS) of the extremity and trunk wall. In addition, NBTXR3, nanoparticles are being studied in phase I/II in patients with head and neck cancer; liver cancer; prostate cancer and rectal cancer.