Lestaurtinib (CEP-701, KT-5555) is an orally bio-available polyaromatic indolocarbazole alkaloid derived from K-252a. Lestaurtinib is a multi-targeted tyrosine kinase inhibitor which has been shown to potently inhibit FLT3 at nanomolar concentrations in preclinical studies, leading to its rapid development as a potential targeted agent for treatment of AML. Phase I studies have shown lestaturtinib to be an active agent particularly when used in combination with cytotoxic drugs. Currently, Phase II and Phase III studies are underway aiming to establish the future of this agent as a treatment option for patients with FLT3-ITD AML.

INCB-039110 (also known as Itacitinib) is an orally bioavailable inhibitor of Janus-associated kinase 1 (JAK1) with potential antineoplastic activity patented by Incyte Corporation for treatment of autoimmune disorders and cancer. Upon oral administration of NCB-039110, this agent selectively inhibits the phosphorylation and activity of JAK1, which may result in inhibition of JAK1-mediated signaling, induction of apoptosis, and reduction of cell proliferation in JAK1-expressing tumor cells. INCB-039110 in combination with nab-paclitaxel plus gemcitabine demonstrated an acceptable safety profile with clinical activity seen in patients with advanced solid tumors including pancreatic cancers. Based on the potential for additive or synergistic effects, NCB-039110 is currently being explored in combination with immunotherapeutic agents including the anti-programmed cell death protein 1 antibody and small molecule inhibitors.

FIN-6 (PATIDEGIB, IPI-926), a semisynthetic derivative of alkaloid cyclopamine, is a G protein-coupled receptor Smoothened (Smo) inhibitor with antineoplastic activity. Smo is a key signaling transmembrane protein in the Hedgehog signaling pathway which plays an important role in the proliferation of neuronal precursor cells in the developing cerebellum and other tissues. FIN-6 (PATIDEGIB, IPI-926) is under development for Gorlin syndrome, basal cell carcinomas, and other potential indications.

F-901318 (Olorofim) is an orotomide antifungal drug. An investigation into the mechanism of action of F-901318 found that it acts via inhibition of the pyrimidine biosynthesis enzyme dihydroorotate dehydrogenase (DHODH) in a fungal-specific manner. Olorofim is being developed by F2G for the treatment of mycoses.