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.

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 ALK-001 is a chemically-modified vitamin A, in which 3 hydrogen atoms have been replaced by 3 deuterium atoms at carbon number 20.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.

Tofimilast is a potent phosphodiesterase-4 (PDE4) inhibitor with low oral bioavailability and no emesis-associated behaviors in ferrets at plasma concentrations up to 152 ng/ml. Tofimilast exhibited an apparent slower absorption through the rat lung after administration as a dry powder, although absorption half-life values were <1 h and therefore the significance of a formulation effect for this compound is questionable. Tofimilast was tested as a therapeutic agent against asthma and chronic obstructive pulmonary disease but development has been discontinued due to lack of efficacy.