Dual-Action Therapies for Bone Disease

Sponsor: Velcura Therapeutics, Inc

• Project Performance Period: 11/1/2007 - 10/31/2010
• Total project (est.): $3,299,999.00
• Requested ATP funds: $2,000,000.00

The National Osteoporosis Foundation (NOF) estimates that there are 10 million patients in the U.S. who have been diagnosed with osteoporosis, and another 34 million who are at risk due to their low bone mass. Osteoporosis is responsible for 1.5 million fractures annually. Current therapies for osteoporosis focus on slowing or inhibiting the loss of bone, and few therapies focus on stimulating bone to replace lost tissue. Velcura, an early-phase biotechnology company that develops drug therapies for the treatment of osteoporosis, bone cancer bone fractures, and other bone disorders, has discovered a family of small molecules that possess characteristics of dual-action therapies, both stimulating bone formation and inhibiting loss. As irreversible inhibitors that are rapidly cleared from the blood stream, these drugs offer low systemic exposure, reduced off-target effects and extended time on target resulting in increased efficacy. This potentially makes them effective agents for cancer patients. However, such drugs potentially can cause problems when used for long-term therapy in diseases such osteoporosis. This proposal seeks to invent a new type of therapeutic molecule by converting these irreversible inhibitors to reversible ones that retain the advantages of the irreversible molecules and their dual-action effects. The invention of an effective, potent, dual-action, compound would represent a breakthrough therapy for bone disease. Innovation is required both at the level of medicinal chemistry and in the area of bone cell assay development. If successful, the therapy could have a major economic impact on the treatment of osteoporosis. Current projections indicate a $60 billion to $65 billion market worldwide for diagnosis and treatment of the disorder by 2020.

For project information:

Cindy Orlandi, (734) 250-2619
corlandi@velcura.com

ATP Project Manager

Mrunal Chapekar, (301) 975-6846
mrunal.chapekar@nist.gov