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.

ACT-462206 is a new, potent, and selective dual orexin receptor antagonist that inhibits the stimulating effects of the orexin peptides at both the orexin 1 and 2 receptors. ACT-462206 shows anxiolytic-like properties in rodents without affecting cognition and motor function. It is therefore a potential candidate for the treatment of insomnia and was in phase I of clinical trial, but this research has been discontinued.

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.

Alkeus Pharma's lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to prevent the formation of these toxic vitamin A dimers in the eye. ALK-001 is a chemically-modified vitamin A, in which 3 hydrogen atoms have been replaced by 3 deuterium atoms at carbon number 20. Replacing the retina's vitamin A with ALK-001 slows the formation of toxic vitamin A dimers. ALK-001 is in phase II clinical trials for the treatment of Stargardt's disease. The compound was co-developed by Alkeus Pharmaceuticals and Columbia University.

DSM265 is an experimental antimalarial that selectively inhibits the parasite dihydroorotate dehydrogenase (DHODH). DSM265 is the first DHODH inhibitor to reach clinical development for treatment of malaria. DSM265 is highly selective toward DHODH of the malaria parasite Plasmodium, efficacious against both blood and liver stages of P. falciparum, and active against drug-resistant parasite isolates. The only possible DSM265-related adverse event was a moderate transient elevation in serum bilirubin.

GNF-156 (ganaplacide or KAF-156) is an antimalarial agent that is part of the imidazolopiperazine family. It exerts activity against pre-erythrocytic liver stages, asexual and sexual blood stages. An improvement compared to existing antimalarial drug combinations is that this compound shows promising single-dose antimalarial activity, and no serious safety and tolerability concerns in humans are known so far. Phase II clinical trials have been completed for GNF-156. Its potential is also being investigated in combination with lumefantrine (an aryl-amino alcohol) in LUM-KAF156.