Geclosporin (Cyclosporine) is an immunosuppressive agent, that is used to prevent rejection of a transplanted organ by the body. Geclosporin is isolated from a fungus, Beauveria nivea, and was first discovered in 1970. By suppressing the immune system, this drug prevents white blood cells from rejecting the transplanted organ. Geclosporin primarily does this by suppressing T cells and T cell cytokine production, but also acts in other ways, for example by inhibiting growth and activation of B cells and antigen presenting cells, and by reducing antibody production. Geclosporin is usually combined with other compounds, and has been studied as potential treatment for a large range of disorders. It is used for the treatment of several other conditions, such as severe recalcitrant plaque psoriasis, severe active rheumatoid artritis, and to prevent rejection of donor cells as a result of bone marrow transplantation. Relapse after discontinuation of this compound is to be expected, and therefore, patients should receive maintenance therapy at the lowest effective dosage. The most common adverse events are hypertrichosis, gingival hyperplasia, and neurological and gastrointestinal effects. Renal dysfunction is also possible, but irreversible damage is rare.

G749 is a Fms-like tyrosine receptor kinase 3 (FLT3) inhibitor and a promising next-generation drug candidate for the treatment of relapsed and refractory acute myeloid leukemia (AML) patients with various FLT3-ITD/FLT3-TKD mutants that shows the ability to overcome drug resistance. It demonstrated potent and sustained inhibition of the FLT3 wild type and mutants including FLT3-ITD, FLT3-D835Y, FLT3-ITD/N676D, and FLT3-ITD/F691L in cellular assays. G749 retained its inhibitory potency in various drug-resistance milieus such as patient plasma, FLT3 ligand surge, and stromal protection. It also displayed potent antileukemic activity in bone marrow blasts from AML patients regardless of FLT3 mutation status, including those with little or only minor responses to AC220 or PKC412. Oral administration of G749 yielded complete tumor regression and increased life span in animal models.

Filgotinib (GLPG0634) is a highly selective JAK1 inhibitor. GLPG0634 is a promising drug candidate for the future treatment of autoimmune and inflammatory disorders. It is in phase III clinical trials (initiated mid-2016) for the treatment of rheumatoid arthritis, Crohn's disease and ulcerative colitis. Most common adverse events observed were infections, gastrointestinal disorders and nervous system disorders.

N-[2-[(2-amino-2-oxoethyl)-[3-(2-oxopyrrolidin-1-yl)propyl]amino]-2-oxoethyl]-2-[2-(2-fluorophenyl)ethylamino]-N-(2-methylpropyl)acetamide (BN201) is a small peptide molecule, a first-in-class neuroprotective compound. BN201 promotes the survival of cultured neural cells when subjected to oxidative stress or when deprived of trophic factors. BN201 promotes neuronal differentiation, the differentiation of precursor cells to mature oligodendrocytes in vitro, and the myelination of new axons. BN201 modulates several kinases participating in the insulin growth factor 1 pathway including serum-glucocorticoid kinase and midkine, inducing the phosphorylation of NDRG1 and the translocation of the transcription factor Foxo3 to the cytoplasm. In vivo, BN201 prevents axonal and neuronal loss, and it promotes remyelination in models of multiple sclerosis, chemically induced demyelination, and glaucoma. Bionure, a spin-off from Hospital Clínic de Barcelona that is based in California, is developing BN201 for multiple sclerosis, acute optic neuritis (AON) and glaucoma. BN201 was granted with orphan designation status for optic neuritis by the FDA. Optic neuritis is often an early sign of multiple sclerosis. The efficacy, safety, and capacity of the drug to cross the blood-brain barrier have been demonstrated in animal models, but the drug has not yet entered clinical testing.