The Food and Drug Administration (FDA) has granted Orphan Drug designation to TVN-102 (Tivorsan), previously referred to as recombinant human Biglycan, for the treatment of Duchenne Muscular Dystrophy (DMD).

Preclinical studies have shown that TVN-102 has improved muscle health and function in a mouse model, as well as reduced muscle cell degeneration and death. The Company intends to initiate a Phase 1 clinical trial in 2017. 

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TVN-102 is an investigational, small, naturally occurring, extracellular matrix muscle protein with a unique mechanism of action for neuromuscular disorders. It is designed to upregulate utrophin, neuronal nitric oxide synthase (nNOS) and other dystrophin-associated proteins in the muscle membrane, as well as controlling muscle specific kinase (MuSK) activity and localization at the nerve-muscle synapse. It is also thought to be synergistic with other treatment options under development for DMD, such as exon skipping.

Additionally, the active form of TVN-102 lacks the complex carbohydrate (GAG) side chains that are present in most forms of biglycan, potentially mitigating off-target effects.

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