Verinurad (RDEA3170) is a selective uric acid reabsorption inhibitor in clinical development for the treatment of gout and asymptomatic hyperuricemia. Verinurad specifically inhibits URAT1 with a potency of 25 nM. High affinity inhibition of uric acid transport requires URAT1 residues Cys-32, Ser-35, Phe-365 and Ile-481. Unlike other available uricosuric agents, the requirement for Cys-32 is unique to verinurad. Verinurad doses as low as 2.5 mg produce significant sUA lowering in humans, and this greater reduction in sUA may lead to improved outcomes and medical benefits for patients with gout. Verinurad in monotherapy studies has been associated with increased urinary uric acid concentrations and low rates of serum creatinine (sCr) elevation. Verinurad combined with febuxostat decreased sUA dose-dependently while maintaining uric acid excretion similar to baseline. All dose combinations of verinurad and febuxostat were generally well tolerated.

Mitometh, a methylated homolog of mitotane possesses an adrenalytic activity and has been studied for the treatment of adrenal cancer. Experiments on animals and on human cell lines have shown that mitometh was not effective in the therapy of adrenal cancer.

Novartis Oncology (previously Novartis) is developing nazartinib (formerly EGF 816), a third generation mutant-selective tyrosine kinase inhibitor (TKI) of epidermal growth factor receptor (EGFR), for the treatment of solid malignancies, with a focus on non-small cell lung cancer. Nazartinib is a covalent mutant-selective EGFR inhibitor, with Ki and Kinact of 31 nM and 0.222 min−1 on EGFR(L858R/790M) mutant, respectively. Upon oral administration, nazartinib covalently binds to and inhibits the activity of mutant forms of EGFR, including the T790M EGFR mutant, thereby preventing EGFR-mediated signaling. This may both induce cell death and inhibit tumor growth in EGFR-overexpressing tumor cells. EGFR, a receptor tyrosine kinase mutated in many tumor cell types, plays a key role in tumor cell proliferation and tumor vascularization. EGF816 preferentially inhibits mutated forms of EGFR including T790M, a secondarily acquired resistance mutation, and may have therapeutic benefits in tumors with T790M-mediated resistance when compared to other EGFR tyrosine kinase inhibitors. As this agent is selective towards mutant forms of EGFR, its toxicity profile may be reduced as compared to non-selective EGFR inhibitors which also inhibit wild-type EGFR.

L-DEPRENYL-D2 C-11 is a selective irreversible inhibitor of monoamine oxidase B (Mao-B) which labels functionally active enzyme. It is used as a positron emission tomography radioligand for measurement of the Mao-B activity in vivo brain.

Cesium Cation CS-131 is an unstable radioisotope of cesium (Cs) with radiocytotoxic application. Cs-131 is a gamma photon-emitting radionuclide with high energy and a relatively short half-life of 9.7 days. Intraoperative cesium-131 (Cs-131) brachytherapy performed at the time of neurosurgical resection may represent an excellent salvage treatment option.

Mallinckrodt (previously Sucampo Pharmaceuticals) is developing nobiprostolan (RK 023), a topical therapy for male pattern baldness (androgenetic alopecia) and hypotrichosis. The drug is a physiologically active fatty acid derivative. Nobiprostolan is in Phase IIa clinical trials for the treatment of male pattern baldness.

Merck was developing MK 8245, an orally active inhibitor of stearoyl CoA desaturase for the treatment of type-2 diabetes mellitus. MK-8245 is a liver-targeting inhibitor of stearoyl-CoA desaturase (SCD) with IC50 of 1 nM for human SCD1 and 3 nM for both rat SCD1 and mouse SCD1, with anti-diabetic and anti-dyslipidemic efficacy. It is in Phase 2 clinical studies for type 2 diabetes mellitus.