Galapagos collaborates with Charley’s Fund, Nash Avery Foundation to develop SARM candidate drug for Duchenne muscular dystrophy

  • Second indication for Galapagos candidate drug G100192
  • Sponsored by Charley’s Fund and Nash Avery Foundation
  • Initiation of preclinical development
Mechelen, Belgium and Great Barrington, Massachusetts, USA; 17 September 2009 – Galapagos NV (Euronext: GLPG), Charley’s Fund Inc. and the Nash Avery Foundation announced today that they will collaborate to investigate the potential effectiveness of Galapagos’ SARM candidate drug, G100192, in treating Duchenne muscular dystrophy.  G100192 is an orally-available small molecule therapeutic, which has demonstrated successful Proof of Concept in pre-clinical studies for cachexia (the loss of weight and muscle mass).
Charley’s Fund and the Nash Avery Foundation will support the costs of pre-clinical studies to assess the potential effectiveness of G100192 in treating Duchenne muscular dystrophy.  While investigating this new indication, Galapagos will continue to develop the SARM therapeutic for cachexia, and plans to initiate a Phase I clinical trial in healthy volunteers in the beginning of 2010.
“Working with Charley’s Fund, the Nash Avery Foundation and experts in the field of Duchenne gives us the opportunity to explore a second indication for G100192 and expand the potential therapeutic benefit of this candidate drug,” said Onno van de Stolpe, CEO of Galapagos. 
“The SARM therapeutic that Galapagos is developing for cachexia could be of potential benefit in treating Duchenne muscular dystrophy,” said Benjamin Seckler, M.D., President of Charley’s Fund.  “If the pre-clinical models show efficacy in treating this disease, G100192 could become one of the first small molecule candidate drugs for Duchenne – complementing the gene therapy and biologic approaches in development.”
About Duchenne muscular dystrophy
Duchenne muscular dystrophy is the most common fatal genetic disorder to affect children around the world.  Approximately one in every 3,500 boys worldwide is afflicted with Duchenne muscular dystrophy with 20,000 new cases reported each year.  It is a devastating and incurable muscle-wasting disease associated with specific inborn errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function.  Symptoms usually appear in male children before age six.  Progressive muscle weakness of the legs and pelvis eventually spreads to the arms, neck, and other areas.  By age 10, braces may be required for walking, and most patients are confined to a wheelchair by age 12.  Eventually, this progresses to complete paralysis and increasing difficulty in breathing.  The condition is terminal and death usually occurs before the age of 30.  The outpatient cost of care for a non-ambulatory Duchenne muscular dystrophy patient is among the highest of any disease.  There is currently no cure for Duchenne muscular dystrophy, but for the first time in decades, there are promising therapies in or moving into development.
About Charley’s Fund
Charley’s Fund Inc. is a not-for-profit foundation that finances therapeutics development for Duchenne muscular dystrophy.  The foundation’s mission is to expedite a treatment or cure in time to help this generation of children who suffer from Duchenne muscular dystrophy.  Charley’s Fund Inc. targets translational research – research that moves science from the lab into human clinical trials.  The 501 (c)(3) public charity was co-founded in 2004 by Benjamin Seckler, M.D. and Tracy Kramer Seckler, whose son Charley has Duchenne muscular dystrophy.  More info at:
About Nash Avery Foundation
The Nash Avery Foundation is a not-for-profit private foundation that is focused on financially supporting programs that will slow and eventually stop Duchenne muscular dystrophy.  Nash Avery Foundation was founded by Angela and Tom Wicka after their son Nash was diagnosed with DMD.  The Foundation believes that the solution in stopping this disease will come from a combination of well funded science and getting talented people focused on this disease.  More info at:
About Galapagos
Galapagos (Euronext: GLPG; OTC: GLPYY) is a drug discovery and development company with small molecule programs in bone and joint diseases, bone metastasis, cachexia, anti-infectives and metabolic diseases.  It has established risk sharing alliances with GSK, Janssen Pharmaceutica, Eli Lilly and Merck and Co.  Through an alliance with MorphoSys, Galapagos is also developing new antibody therapies in bone and joint diseases.  Its division BioFocus DPI offers a full suite of target-to-drug discovery products and services to pharmaceutical and biotech companies and to patient foundations, encompassing target discovery and validation, screening and drug discovery through to delivery of pre-clinical candidates.  Galapagos currently employs 495 people and operates facilities in six countries, with global headquarters in Mechelen, Belgium.  More info at:
Galapagos NV
Onno van de Stolpe, CEO
Tel: +31 6 2909 8028
Charley’s Fund Inc.
Benjamin D. Seckler, M.D., President
Tel: +1 413 528 5744 or +1 877 436 3363 (877-4-END-DMD)
Nash Avery Foundation
Tom C. Wicka, President and Founder
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