PROVIDENCE, R.I.--(BUSINESS WIRE)--Tivorsan Pharmaceuticals, Inc. (Tivorsan) today announced that it met its fourth (4th) set of critical milestones under its existing grants from Parent Project Muscular Dystrophy (PPMD) and Muscular Dystrophy Association (MDA). The achievement of these milestones triggers additional payments to Tivorsan pursuant to the Company’s $565,000 grant from PPMD and $1,000,000 grant from MDA.
The milestones reflect important progress in Tivorsan’s ongoing efforts to develop recombinant human biglycan as a disease-modifying treatment candidate for Duchenne muscular dystrophy (DMD). Biglycan is a naturally occurring protein found in the extracellular matrix of skeletal and cardiac muscles. It localizes to the outside of developing and regenerating muscle cells, where it plays a number of important roles to promote muscle health and function. The Tivorsan team including its founding scientist, Professor Justin Fallon of Brown University, has developed a recombinant form of biglycan known as TVN-102. TVN-102 has shown itself to be therapeutically active in the standard animal model of DMD, known as the mdx mouse.
In the latest set of milestones that Tivorsan achieved to trigger the additional payments by PPMD and MDA, the Company created a suite of sophisticated methods that will aid the process of scale-up manufacturing for TVN-102. This development work is necessary to facilitate the production of TVN-102 for use in clinical trials. In addition, Tivorsan demonstrated that using TVN-102 made from a pilot manufacturing process, the drug demonstrated increased utrophin levels in human cells obtained through biopsy from boys with DMD.
Stated Dr. Joel B. Braunstein, Tivorsan’s President and Co-Founder, “We are very delighted with our recent accomplishments. These activities bring us closer to having an optimized manufacturing process that we will be able to rely upon for making large amounts of protein in preparation of human clinical studies. Further, the demonstrated increases in utrophin levels that we can observe from applying TVN-102 directly to cells obtained from boys with DMD underscores the drug’s therapeutic potential. Recombinant biglycan has potential to treat all boys with DMD, regardless of their dystrophin mutation status. It accomplishes this by way of its utrophin-based mechanism of action. We are deeply appreciative of PPMD’s and MDA’s continued support, and acknowledgement by scientific peers that we are making important strides toward bringing TVN-102 closer to clinical testing.”
Pat Furlong, the Founding President and CEO of Parent Project Muscular Dystrophy (PPMD), commented, “We are pleased with the progress of this project. Duchenne muscular dystrophy represents a critical unmet medical need and the more options we have in the pipeline the better.”
Further, Valerie Cwik, M.D. Executive Vice President & Chief Medical and Scientific Officer for the Muscular Dystrophy Association added, “At MDA, we’re driven by our passion to create a world free of the harmful effects of muscle disease. There is an unmet need for the treatment of Duchenne muscular dystrophy and MDA is pleased to see the progress made by Tivorsan Pharmaceuticals. MDA remains a champion for treating DMD. Tivorsan’s biglycan drug has the potential to effectively attract the utrophin protein to where it could have a clinically beneficial effect in muscle cells.”
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 also regulates the expression of nNOS (neuronal nitric oxide synthase) at the muscle membrane. Biglycan likely acts through a 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, Becker Muscular Dystrophy (BMD), various congenital muscular dystrophies (CMD), and amyotrophic lateral sclerosis (ALS). The approach, 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.