Galapagos has discovered new drug targets (human proteins that are starting points for the development of novel drugs) using human primary cells relevant for rheumatoid arthritis, osteoarthritis and osteoporosis and Alzheimer’s disease. Galapagos applies these targets to find small molecules that change the activity of these proteins and thereby can influence the course of the disease, forming the basis for the development of first-in-class medicines. This approach is expected to yield a breakthrough in treatment by stopping the disease rather than just treating the symptoms.

Galapagos has successfully entered two candidate drugs based on its target discovery platform into clinical trials. Candidate drug GLPG0259, which is being developed for the treatment of rheumatoid arthritis, demonstrated good safety in healthy volunteers and a profile that supports once-daily oral dosing in a Phase I study. A second Phase I study for candidate drug GLPG0259 is currently underway. Another novel candidate drug, GLPG0555, identified through Galapagos’ proprietary target discovery platform as part of the arthritis alliance with GlaxoSmithKline, entered Phase I clinical development in December 2009.

Additionally, Galapagos has drug discovery programs based on known modes-of-action. Nanocort®, a liposome formulation of prednisolone, is being developed for the treatment of acute multiple sclerosis and rheumatoid arthritis flares. Nanocort has demonstrated safety and a good response in a Phase I/II rheumatoid arthritis trial. After evaluating Nanocort’s product profile, Galapagos decided to pursue MS as a first indication and began a Phase II study to evaluate the efficacy and safety of Nanocort in treating MS flares in November 2009. The first Phase I trial for the cancer metastasis (GLPG0187) program showed good safety and a promising biomarker profile in healthy volunteers. In 2010 Galapagos plans to initiate a second Phase I trial for GLPG0187, including cancer patients. Galapagos has also developed a small molecule in its Selective Androgen Receptor Modulator (SARM) program, which demonstrated successful Proof of Concept in animal studies and was nominated for pre-clinical development at the end of 2008. This pre-clinical candidate is now being developed for the treatment of cachexia (loss of weight and muscle mass) and is also being investigated for its potential effectiveness in treating Duchenne muscular dystrophy.

Through a number of strategic alliances with big pharma, Galapagos has financed expansion of its R&D portfolio to more than 40 programs, including four in clinical development.