Tivorsan Pharma Meets Development Milestones with DMD Treatment Research

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Duchenne muscular dystrophy

Tivorsan Pharmaceuticals, Inc. (Tivorsan), a protein therapeutics company with a focus on developing treatments for neuromuscular disorders such as muscular dystrophy, recently announced the company has achieved a fourth set of pivotal milestones under a series of research grants from Parent Project Muscular Dystrophy (PPMD) and Muscular Dystrophy Association (MDA). These achievements mark the release of more funding from these grants, in fulfillment of PPMD’s pledge of $565,000, and the MDA’s pledged $1 million.

Aside from additional funding, these crucial milestones demonstrate Tivorsan’s efforts to advance recombinant human biglycan for Duchenne Muscular Dystrophy (DMD). It is a disease-modifying, naturally occurring protein derived from the extracellular matrix of cardiac and skeletal muscle cells, known to play a chief role in muscular health and function. The team of researchers at Tivorsan, along with the founding scientist himself, Professor Justin Fallon from Brown University, created a recombinant form of this protein, dubbed, TVN-102, which has been observed to have therapeutic properties in animal models of DMD or the mdx mice.

“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,” commented Dr. Joel B. Braunstein, the company’s President and Co-Founder. “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.”

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Before Tivorsan met the fourth set of milestones, they were able to design a series of complex techniques meant to facilitate the process of large-scale manufacturing for the experimental drug, which will prove vital in advancing TVN-102 to bigger, more intensive clinical testing. The company was also able to observe, during a pilot manufacturing run, that TVN-102 had enhanced utrophin levels in cells sampled from patients with DMD, which can compensate for the lack of dystrophin.

“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,” said Pat Furlong, the Founding President and CEO of Parent Project Muscular Dystrophy.

Dr. Valerie Cwik, the 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.”