Pexiganan is a 22-amino-acid synthetic cationic peptide. It is an analog of magainin 2, which is a host defense peptide isolated from frog skin. The drug is thought to act by disturbing the permeability of the cell membrane or cell wall. Pexiganan exhibited in vitro broad-spectrum antibacterial activity when it was tested against 3,109 clinical isolates of gram-positive and gram-negative, anaerobic and aerobic bacteria. It is currently in phase 3 clinical trials as a topical antimicrobial agent for the treatment of mild infections associated with diabetic foot ulcers. In vitro data for pexiganan acetate suggest that the drug does have hemolytic activity at concentrations relevant for antibacterial activity. In association with tigecycline, pexiganan administration could overcome antibiotic resistance and increase the effectiveness of treatment against P. aeruginosa sepsis.

Prazarelix is gonadorelin (GnRH) antagonist. It is primarily used in assisted reproduction to control ovulation. The drug works by blocking the action of GnRH upon the pituitary, thus rapidly suppressing the production and action of luteinizing hormone (LH) and follicle-stimulating hormone (FSH).

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 ALK-001 is a chemically-modified vitamin A, in which 3 hydrogen atoms have been replaced by 3 deuterium atoms at carbon number 20.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

Rosiptor (AQX-1125) is a once daily, orally administered small molecule being developed by Aquinox Pharmaceuticals for the treatment of asthma, bladder pain syndrome/interstitial cystitis (IC/BPS) and chronic prostatitis/chronic pelvic pain syndrome. AQX-1125 is the only clinical-stage, orally administered, SHIP1 activator. AQX-1125 significantly reduces the late response to allergen challenge, with a trend to reduce airway inflammation. AQX-1125 was safe and well tolerated and merits further investigation in inflammatory disorders. AQX-1125 has been used in trials studying the treatment of COPD, atopic dermatitis, interstitial cystitis, and bladder pain syndrome. However, the development has been discontinued.