Resiniferatoxin (RTX or RTX-107) is a vanilloid derived from a cactus-like plant (Euphoria resiniferous) and has anti-inflammatory activity. This compound is an agonist of the transient receptor potential vanilloid 1 (TRPV1). Resiniferatoxin produces analgesia by desensitizing the TRPV1 receptor. Findings of several studies have suggested a potential therapeutic use of the anti-inflammatory effect of resiniferatoxin. Phase I and II clinical trials have been completed or are underway, evaluating the safety and efficacy of resiniferatoxin in pain-related disorders such as osteoarthritis and cancers.

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 ALK-001 is a chemically-modified vitamin A, in which 3 hydrogen atoms have been replaced by 3 deuterium atoms at carbon number 20.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