Tivorsan Pharmaceuticals is a biopharmaceutical company developing novel treatment approaches to address all forms of Duchenne Muscular Dystrophy (DMD) and Becker’s Muscular Dystrophy (BMD), as well as other progressive, life threatening neuromuscular disorders. The lead candidate therapeutic, recombinant human Biglycan, is based on the scientific work from the Fallon Laboratory at Brown University. The technology is exclusively licensed to Tivorsan Pharmaceuticals from Brown University.
Biglycan has a unique Mechanism of Action (MOA): up-regulating utrophin, nNOS and other dystrophin-associated proteins at the muscle membrane. Biglycan also plays an important role in neuromuscular junction stability through its interaction with Muscle Specific Kinase (MuSK). This unique and differentiated MOA provides a strong platform to develop a series of first or best in class therapies for a range of neuromuscular disorders beyond DMD and BMD, including Congenital Muscular Dystrophy, ALS, Spinal Muscular Atrophy and Myasthenia Gravis.
IND-enabling activities are currently underway and the company is targeting an IND submission and initiation of Phase 1 clinical trials in early 2017.