PROVIDENCE, R.I.--(BUSINESS WIRE)--Tivorsan Pharmaceuticals, Inc., a biotechnology company that is pioneering a novel approach to treating all forms of Duchenne Muscular Dystrophy (DMD) and other serious, debilitating neuromuscular disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead investigational drug, human recombinant Biglycan, for the treatment of DMD.
“We’re excited to receive Orphan Drug Designation for human recombinant Biglycan and realize this important step toward our goal of bringing a much-needed treatment to patients with DMD,” said Jim Connolly, CEO of Tivorsan. “The Tivorsan team is executing on key activities to advance the development of our lead form of Biglycan, which is known as TVN-102, and we remain on track with a planned IND submission and initiation of Phase 1 clinical trials in 2017.”
FDA Orphan Drug Designation provides special status to novel drugs and biologics that diagnose, treat or prevent rare or orphan diseases and disorders that affect fewer than 200,000 patients in the United States. The designation provides the drug developer with certain regulatory and financial incentives, including a seven- year period of U.S. marketing exclusivity, as well as tax credits for clinical research costs, assistance in clinical trial design and regulatory proceedings, and a waiver of FDA’s drug filing fees.
About Tivorsan Pharmaceuticals
Tivorsan is a biotechnology
company developing novel treatment approaches to treat a series of rare
and orphan diseases, including all forms of DMD and Becker’s Muscular
Dystrophy (BMD), as well as other progressive, life threatening
neuromuscular disorders such as Congenital Muscular Dystrophy, ALS,
Spinal Muscular Atrophy and Myasthenia Gravis. The company’s lead
candidate, TVN-102, is based on scientific work from the Fallon
Laboratory at Brown University, which was led by Justin Fallon, Ph.D.,
scientific founder of Tivorsan.
About TVN-102
TVN-102, which was previously referred to as
recombinant human Biglycan, is a small, naturally occurring
extracellular matrix protein found in muscle. It has a unique mechanism
of action, that of upregulating utrophin, nNOS (neuronal nitric oxide
synthase) and other dystrophin-associated proteins in the muscle
membrane, as well as controlling MuSK (muscle specific kinase) activity
and localization at the nerve-muscle synapse. This unique and
multi-faceted mechanism provides a strong foundation and platform for
the development of a series of therapies for neuromuscular disorders
with significant unmet medical need. Tests in the most widely used mouse
model of DMD have shown that systemically delivered TVN-102 upregulates
utrophin and activates the dystrophin-independent membrane stabilization
program. TVN-102 has been shown to improve muscle health and function in
a mouse model. Mice treated with TVN-102 also show reduced muscle cell
degeneration and death.
TVN-102 targets all forms of DMD, regardless of mutation status. In addition, TVN-102 should be synergistic with other therapeutic approaches that are in development for DMD, such as exon skipping. The proposed synergy should be supported by TVN-102’s upregulation of utrophin, as well as its targeting of nNOS, a key enzyme that is functionally compromised in DMD and BMD.
Importantly, the active from of TVN-102 lacks the complex carbohydrate (GAG) side chains that are present in most forms of biglycan. This simplified structure greatly facilitates the manufacture of TVN-102 and mitigates off-target effects.
