Maralixibat (SHP625; formerly LUM001 or lopixibat) is a potent, apical, sodium‐dependent, bile acid transporter competitive inhibitor with minimal systemic absorption. Maralixibat works by reducing systemic levels of bile acids. In animal models of cholestasis, maralixibat blocked reabsorption of bile acids in the terminal ileum, thereby reducing enterohepatic recirculation to the liver and increasing fecal excretion of bile acids. Maralixibat is being evaluated as a treatment for children with rare cholestatic liver diseases, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

MK-3118 is an orally active inhibitor of fungal β-(1,3)-glucan synthase patented by Merck Sharp & Dohme Corp for the treatment of fungal infections. MK-3118 demonstrated enhanced efficacy for most C. albicans and C. glabrata ER isolates relative to caspofungin. MK-3118 showed no or poor activity against Mucoromycotina and Fusarium spp. However, MK-3118 was highly active against Paecilomyces variotii and was the only compound displaying some activity against notoriously pan-resistant Scedosporium prolificans.

Cyclic pyranopterin monophosphate is a precursor to molybdenum cofactor, which is required for the enzyme activity of sulfite oxidase, xanthine dehydrogenase/oxidase, and aldehyde oxidase. The drug is being investigated in severe molybdenum cofactor deficiency type A. In a clinical study, intravenous administration of the drug resulted in greatly improved neurodevelopmental outcome when started sufficiently early.

KD025 is an orally available, selective small molecule inhibitor of ROCK2 (Rho-associated coiled-coil kinase 2), a molecular target in multiple autoimmune, fibrotic and neurodegenerative diseases. KD025 is the only ROCK2-specific inhibitor in the clinical trials. KD025 down-regulates the IL-17 and IL-21 secretion in human PBMCs, and leads to down-regulation of STAT3 phosphorylation, IRF4, and RORγt expression in CD4+ T cells. Kadmon Pharmaceuticals initiated phase II clinical trials of KD025 for the treatment of Graft-versus-host disease; Idiopathic pulmonary fibrosis; Plaque psoriasis.

OTL-0038 or OTL-38, a fluorescent-labelled folate receptor-α (FRα) targeted imaging agent that accumulates in vivo in tumor cells expressing FR. OTL38 is currently in ongoing Phase 3 clinical trial in ovarian cancer and successfully completed phase 2 clinical trial in lung cancer. OTL38 is being evaluated for its ability to help surgeons locate and remove hard-to-find cancerous lesions that are often widespread. In 2014, the OTL-38 molecule was granted orphan drug status which can be given to the maker of a drug that treats rare conditions or diseases and offers protection from competition for a period of time. In addition, OTL-38 was studied in phase II clinical trials in the Netherlands for the diagnosis of endometriosis.

Fexinidazole is an antiparasitic drug, which is in the phase III of clinical trial for the treatment of Human African Trypanosomiasis, and in the phase II for the treatment Disease, Chagas and Visceral Leishmaniosis. However, for the Visceral Leishmaniosis, studies were terminated, due to lack of efficacy. Fexinidazole rapidly metabolized to two active metabolites, a sulfone and a sulfoxide, which prolong the pharmacological action of parent drug. These metabolites retaine trypanocidal activity but are less effective in nifurtimox-resistant lines, which can lead to the potential danger in the use of fexinidazole as a monotherapy.

Voclosporin (trans-ISA247) is a Cyclosporin A derivative and immunosuppressive compound currently being investigated for the treatment of psoriasis, lupus nephritis and for the prevention of organ rejection in kidney transplant patients. An animal study showed that a lower blood level of Voclosporin was able to produce a greater or similar inhibition of lymphocyte proliferation, expression of T-cell activation surface antigens, and T-cell cytokine production compared to Cyclosporin A. Voclosporin has been shown to be an efficacious and safe immunosuppressant in phase IIb and phase III trials in renal transplant recipients and in plaque psoriasis patients. In clinical trials, Voclosporin added to standard-of-care induction therapy for lupus nephritis increases complete renal remission (CRR) rates, but higher rates of adverse events including death were observed.

Selpercatinib (LOXO-292, ARRY-192) is a potent and specific RET (c-RET) inhibitor that was granted accelerated FDA approval on May 8, 2020, for specific RET-driven cancer indications. It is currently marketed under the brand name RETEVMO™ by Loxo Oncology Inc.