Tivorsan’s Biglycan Granted Orphan Drug Designation for Treatment of Duchenne MD

Tivorsan’s lead investigational therapy for the treatment of Duchenne muscular dystrophy (DMD) has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (U.S. FDA).

The human recombinant Biglycan (TVN-102), a natural component of muscles, activates a utrophin-based compensatory pathway that can potentially overcome damage caused by the lack of dystrophin in DMD patients.

DMD is an X-linked genetic disorder that results from mutations in the gene encoding the protein dystrophin.

According to Tivorsan, systemically-administered Biglycan improves muscle health, structure and function in dystrophin-deficient mice, with no signs of toxicity. The results indicate that mice treated with Biglycan have healthier, better functioning skeletal muscle.

“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,” Jim Connolly, Tivorsan’s chief executive officer, said in a press release. “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.”

IND refers to the FDA’s Investigational New Drug application process by which the pharmaceutical company could be permitted to ship the experimental drug across state lines for the clinical trial, before the final drug approval occurs.

TVN-102 is a small, natural occurring extracellular matrix protein with a unique mechanism of action that upregulates utrophin, neuronal nitric oxide synthase (nNOS), and controls MuSK (muscle specific kinase) activity and localization at the nerve-muscle synapse, and other dystrophin-associated proteins in the muscle membrane.  TVN-102 should be used in combination with other therapies, such as exon skipping, currently under development.

The proposed synergy should be supported by TVN-102’s upregulation of utrophin, as well as its targeting of nNOS, an important enzyme that is functionally compromised in DMD patients. Additionally, the simplified structure of TVN-102 reportedly mitigates undesirable effects.

U.S. FDA’s Orphan Drug Designation provides special status to novel drugs and biologics that diagnose, treat or prevent rare or orphan diseases that affect fewer than 200,000 patients in the United States, and that are deemed less profitable for pharmaceutical companies to develop. The special status provides the drug developer with certain regulatory and financial incentives in order to accelerate access to patients who will benefit from the new drugs.