Galapagos has discovered new drug targets (starting points for the development of novel drugs) using cells from patients for more than ten diseases. 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 two candidate drugs in clinical development that are based on this target discovery platform. 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 Phase I clinical trials. These results support the design of a Phase II study planned to start in the second half of 2010, where the efficacy of the novel candidate drug will be assessed in RA patients. 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. Additionally, Galapagos is developing GLPG0492, an orally available small molecule in its Selective Androgen Receptor Modulator (SARM) program, which entered Phase I clinical trials in May 2010. GLPG0492 has shown efficacy in the treatment of cachexia in pre-clinical studies, while studies to evaluate the molecule’s potential efficacy in Duchenne muscular dystrophy.

Recently, Galapagos made the strategic decision to pursue drug discovery and development in orphan diseases – diseases of high medical need, not commonly pursued by pharmaceutical companies. Cystic fibrosis (CF) is the first orphan disease in which Galapagos plans to discover, develop and launch disease-modifying medicines. This decision is based on Galapagos’ successful collaboration with the CF Foundation, where Galapagos identified the first ever disease-modifying targets for CF. With this strategy, Galapagos expects to benefit from the accelerated approval procedures and exclusive commercial rights granted to developers of orphan drugs through US and European regulatory agencies, to ultimately provide real medical benefit to this sizeable population of patients.

Galapagos is progressing one of the largest pipelines in biotech, with five clinical and over 50 small molecule discovery/pre-clinical programs.