Parent Project Muscular Dystrophy Awards $500,000 to Tivorsan Pharmaceuticals

To: HEALTH, MEDICAL AND NATIONAL EDITORS

Grant Funds Work of Tivorsan Pharmaceuticals' Team to Bring Recombinant Biglycan to the Clinic for the Possible Treatment of Duchenne Muscular Dystrophy

HACKENSACK, N.J., Feb. 21, 2012 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced today that it will award Tivorsan Pharmaceuticals (Tivorsan) a $500,000 grant to develop the company's biglycan therapeutic candidate for Duchenne muscular dystrophy.

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Dr. Justin Fallon of Brown University, the founding scientist of Tivorsan, was the recipient of PPMD's first End Duchenne Grant in 2008. Building on that work, the Tivorsan team seeks to use recombinant human biglycan (rh-BGN) to increase utrophin at the muscle cell membrane, resulting in reduced muscle damage and improved function.

Utrophin is a molecule that is related to dystrophin in structure and form and can "stand in" for dystrophin when present in greater than normal quantities. Biglycan is a naturally occurring protein made up of amino acids and carbohydrate chains that is found in large amounts on the outside of developing and regenerating muscle cells. The form of biglycan that is active therapeutically contains only simple carbohydrate side chains. For this reason, rh-BGN is straightforward to manufacture.

PPMD President and CEO Pat Furlong believes biglycan is a promising therapy candidate in Duchenne and is happy to again support this project as Tivorsan prepares to take it to human clinical trials: "Dr. Fallon's work at Brown University over the last twenty years on the role of extracellular matrix proteins in organizing the muscle cell surface, shows promising results that will directly impact everyone in the Duchenne patient community. His lab's, and now his company's, translational research in muscular dystrophy will support the development of recombinant human biglycan as a therapy for Duchenne. The potential of research like Dr. Fallon's is exactly why we established the End Duchenne Grant Award Program in 2008, and why we are honored to be part of the team bringing this work closer to trial, today."

Dr. Fallon was thrilled to receive the news that PPMD would be supporting biglycan development again. "Biglycan has the potential to slow the progression of Duchenne in every boy diagnosed--no matter his genetic mutation. Not everyone saw the potential in biglycan during our earlier efforts. But PPMD had the vision to see the promise we saw, and when they awarded us the End Duchenne Grant, it reinvigorated my lab's work. I am certain that the NIH U-01 Translational Development Award that my laboratory received in 2009 for further developing biglycan would not have been possible without PPMD's early support. Now PPMD is coming through again with the financial assistance we need to move biglycan into human clinical trials."

"We are so appreciative of Parent Project Muscular Dystrophy for their ongoing support of the work we are doing in collaboration with Dr. Fallon and his team at Brown University," said Joel B. Braunstein, M.D., co-founder and CEO of Tivorsan Pharmaceuticals. "We are making definitive progress with biglycan, and we are highly driven to bring an effective muscular dystrophy therapy to market. To our Tivorsan team, PPMD represents the voice of the Duchenne community. This support is critical to our ability to navigate through the drug development process and achieve meaningful milestones."

Ms. Furlong explained that PPMD has a long history of supporting promising therapies and taking risk on research strategies that otherwise might not get the financial backing they need. "PPMD just completed a holiday campaign raising money specifically directed towards funding this biglycan project. We see a future without Duchenne, and we believe that biglycan will play an important role. We take our responsibility to our families very seriously: to thoroughly vet and financially support any research project, any therapy that brings us one step closer to this goal. We have a Drug Development Advisory Committee comprised of the top Duchenne experts in the world that help us make each funding decision. We will continue to award grants to projects like biglycan, that treat all patients living with Duchenne, whenever the opportunity presents itself."

For more about Parent Project Muscular Dystrophy's grant program, as well as a comprehensive list of what we are funding, please visit ParentProjectMD.org/Research.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy--our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.

Everything we do--and everything we have done since our founding in 1994--helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to http://www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.

Tivorsan Pharmaceuticals

Tivorsan Pharmaceuticals is a protein therapeutics company pioneering a unique approach to treating serious neuromuscular disorders, including Duchenne and Becker muscular dystrophy.

This method, using recombinant human biglycan (rhBGN), is based on 24 years of basic science work in the Fallon laboratory at Brown University. Tivorsan was formed by Dr. Justin Fallon in collaboration with colleagues from Old Forge Holdings, LLC of Greenwich, CT and LifeTech Research, Inc, a Baltimore, MD-based technology research and development firm.

SOURCE Parent Project Muscular Dystrophy

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Parent Project Muscular Dystrophy Awards $500,000 to Tivorsan Pharmaceuticals