Galapagos selects pre-clinical candidate drug for cachexia

Belgium-based drug discovery company Galapagos NV has selected a candidate drug to enter into pre-clinical development for cachexia (loss of weight and muscle mass) . This candidate drug is a small molecule that Galapagos has developed in its Selective Androgen Receptor Modulator (SARM) program and which has demonstrated successful Proof of Concept in animal studies.

Galapagos" pre-clinical candidate G100192 binds very selectively and strongly to targeted androgen receptors, potentially enabling the candidate drug to be efficacious without cardiovascular, prostate, or virility side effects traditionally seen in androgen therapies. Galapagos aims for once-a-day oral dosing that improves muscle mass and function, with minimal effects on hormonal status of cachexia patients.

In its SARM program, Galapagos" strategy has been to pursue a first indication in cachexia, with possible second indication in osteoporosis. In February 2008, Galapagos announced that the SARM lead compound had limited bioavailability. Thereafter, its research group at the Biocitech Park in Romainville obtained a breakthrough in potency and oral bioavailability, leading to a pre-clinical candidate within 10 months.

G100192 is a novel compound for which patents are pending, providing freedom to operate. This program is an addition to the Company's portfolio of unpartnered r&d programs that address known drug targets, which also includes an integrin receptor antagonist (IRA) program in bone metastasis and Nanocort, a liposome formulation of prednisolone for acute flares in rheumatoid arthritis and multiple sclerosis.

'The G100192 candidate is a remarkable success story. From an almost completely abandoned program in February 2008, our researchers applied rational drug design to come up with a novel compound that has overcome the program hurdles of bioavailability. G100192 shows very exciting efficacy and safety,' said Onno van de Stolpe, ceo of Galapagos. 'As a consequence, this candidate in cachexia is now well positioned to address a large unmet medical need for which there is only limited competition. We are eager to progress this molecule towards the clinic.'

Cachexia is the loss of appetite, weight and muscle mass in persons who are not actively trying to lose weight; it can be a symptom of underlying illnesses such as cancer, AIDS, and age-related disorders. Cachexia physically weakens and immobilizes patients, and response to treatment is usually poor. Existing therapies for cachexia, such as steroids and cytokine inhibitors, often have serious side effects. Current estimates of the market for cachexia therapies are in the range of Euro 1 bn.