National Institutes of Health Awards Dr. Justin Fallon and Brown University $1.33M+ to Advance Biglycan to Clinic for Duchenne Muscular Dystrophy

Funding Supplements Tivorsan Pharmaceuticals’ Commercialization Efforts of Biglycan as Novel Disease-Modifying Therapeutic

PROVIDENCE, R.I.–(BUSINESS WIRE)– Tivorsan Pharmaceuticals announced today that its academic collaborators – Professor Fallon and his research group at Brown University – have received a generous award from the National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH). This award provides funding and support for the accelerated preclinical development of biglycan as a novel disease-modifying therapeutic for Duchenne Muscular Dystrophy (DMD). DMD is an unrelenting and progressive, genetic, muscle-wasting disorder that currently has no good treatment options.

NIH’s continued support of biglycan is only the most recent accomplishment originating from Dr. Fallon’s work. The $1.33M+ in funding is for the third year of a $5M+ multi-year Cooperative (U01) Award that Dr. Fallon and Brown received in 2009. The U01 represents a unique, peer-reviewed, milestone-driven funding mechanism. This latest award follows two successful years of preclinical progress by Dr. Fallon, his colleagues at Brown, and his corporate collaborator – Tivorsan Pharmaceuticals – to prepare a proprietary form of biglycan, known as TVN-102, for the clinic.

Very recently, Windhover – a leading provider of business intelligence to executives within the life sciences industry and an industry expert panel – selected Tivorsan’s proprietary biglycan molecule as one of 2011’s Top 10 Most Interesting “Hot Space” Projects to Watch.

Dr. Fallon, a Professor of Neuroscience affiliated with Brown’s Institute for Brain Science, Founding Scientist of Tivorsan, and Chair of Tivorsan’s Scientific Advisory Board noted, “The NIH award demonstrates strong support to the continued development of biglycan as a therapy for an insidious disease – DMD. My team and I are extremely grateful to the NIH for this funding.”

Dr. Katherine Gordon, Managing Director of Brown University’s Technology Ventures Office, said, “We are extremely proud of Dr. Fallon and his team, and we congratulate them. Brown understands the public health imperative of Dr. Fallon’s work. We appreciate the scientific community’s acknowledgement of the high impact science conducted by Dr. Fallon and his research colleagues at our University.”

Dr. Joel B. Braunstein, a Tivorsan co-founder and CEO added, “This U01 Award provides us with meaningful feedback on the design of our preclinical development program for biglycan. Federal support for our program exemplifies the strong public commitment to tackle serious unmet medical needs with promising, innovative treatments. In the months ahead, we anticipate leveraging this funding with support from private sources, which are equally committed to our cause.”

About DMD

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.

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, including a series published during Dec. 2010 in the Proceedings of the National Academy of Sciences, 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 by Dr. Fallon who came to Brown University in Providence, RI, in 1996. 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.

Contacts

Tivorsan Pharmaceuticals
Dr. Joel B. Braunstein, 410-653-9685
jbraun@tivorsan.com







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