Tivorsan Pharmaceuticals is pioneering a novel approach to treating all forms of Duchenne Muscular Dystrophy (DMD) and other progressive neuromuscular disorders. Its lead candidate is recombinant human Biglycan (rhBGN), an extracellular matrix protein the company says upregulates utrophin, nNOS and other dystrophin-associated proteins and modulates MuSK activity at the neuromuscular synapse. In preclinical tests in the most widely used DMD mouse model, systemically delivered rhBGN reportedly improved muscle structure, reduced degeneration, and activated a dystrophin-independent membrane stabilization program. The company highlights that the active form of rhBGN lacks complex GAG side chains, which simplifies manufacture and may mitigate off-target effects. Tivorsan positions Biglycan as potentially synergistic with other DMD approaches such as exon skipping. The company recently raised approximately $1.8 million in private financing to fund R&D of rhBGN, working capital and general corporate purposes, and to bridge to a proposed Series D convertible preferred round expected to close in the second quarter. Tivorsan plans an IND submission and initiation of Phase 1 clinical trials in early 2017.

Total raised

$2M

Funding rounds

1

Latest round

Equity

Latest activity

Feb 2016