PROVIDENCE, R.I.--(BUSINESS WIRE)--Tivorsan Pharmaceuticals, Inc., a privately held biotechnology company developing a novel protein therapeutic to combat Duchenne Muscular Dystrophy (DMD), has been selected as one of 2011’s Windhover’s Top 10 Most Interesting “Hot Space” Projects to Watch.
Windhover is a leading provider of business intelligence to executives within the life sciences industry. An industry expert panel affiliated with Windhover selected Tivorsan’s Biglycan in the category of “Hot Space” therapeutic areas, inclusive of projects in Women’s Health, Dermatology, Bone and Muscle Disease, Ophthalmology and other high value areas. The recognition stemmed from Biglycan’s potential to address an urgent medical need through a unique mechanism of action, the progress of its development, project leadership, and potential for indication expansion and multi-level partnering.
Duchenne MD is an unrelenting, genetic muscle disorder that strikes young boys and ultimately leads to death in early adulthood. It affects approximately 1 in 3,500 newborn boys, triggered by mutations of the gene encoding the dystrophin protein. Dystrophin plays an essential role in cell signaling and preserving the structural integrity of skeletal and cardiac muscle cells. The deficiency of dystrophin in DMD leads to muscle cell damage, fatty replacement and scarring of muscle tissue, and progressive weakness. No current treatments successfully address the biologic course of the disease.
Tivorsan’s Biglycan is a proprietary form of a naturally-occurring protein that works through a compensatory pathway to restore muscle health in dystrophin-deficient muscles. In the standard mouse model of DMD, Biglycan markedly reduces muscle damage, reduces muscle degeneration, and improves muscle function. The company is actively working towards human clinical studies with a proprietary form of the protein known as TVN-102.
"We are honored to have our lead pipeline compound selected as one of Windhover's Top 10 Most Interesting," said Dr. Joel B. Braunstein, CEO and Director of Tivorsan Pharmaceuticals. "We are working to move TVN-102 through production and preclinical testing as rapidly as possible. We are hopeful to help many children and their families suffering from this tragic disease."
As a selected company, Tivorsan will present at Windhover's 6th Annual Therapeutic Area Partnerships conference November 30-December 2, 2011 in Boston, MA. Previously nominated Windhover Top 10 projects included many successfully partnered programs, including those from Portola Pharmaceuticals, Kosan Biosciences Inc., NeurogesX, Acorda Therapeutics, Isis Pharmaceuticals and Sangamo BioSciences.
About Biglycan
Biglycan regulates the expression of utrophin at the muscle cell membrane (sarcolemma). Utrophin can compensate for the loss of dystrophin. Thus, utrophin offers an alternative pathway to maintaining the integrity of the muscle cell membrane in boys with DMD. A utrophin-based therapy could potentially benefit all forms of DMD, regardless of the underlying mutation. Biglycan acts through an apparently unique mechanism of action, distinct from other treatments in development, yet potentially suitable for concurrent use with such treatments. Animal efficacy studies have shown that systemically administered recombinant biglycan is well-tolerated and remains effective for approximately three weeks following a one-time dosing. Therapeutic effects persist out to three months following dosing once every three weeks.
About Tivorsan Pharmaceuticals
Tivorsan Pharmaceuticals, Inc. is a protein therapeutics company pioneering a unique approach to treating serious neuromuscular disorders, including DMD and Becker Muscular Dystrophy (BMD). This method, using recombinant human biglycan, is based on 25 years of basic science work in the Fallon laboratory at Brown University, Providence, RI. Tivorsan was formed by Dr. Fallon in collaboration with colleagues from Old Forge Holdings of Greenwich, CT and LifeTech Research, a Baltimore, MD-based technology research and development firm. Early support for the Tivorsan program originated, in part, from Federal sources, as well as Charley’s Fund, Nash Avery Foundation, and Parent Project Muscular Dystrophy, philanthropies seeking cures for DMD.