BioPharm Insight Tivorsan in planning stages for Phase I program of Duchenne’s therapeutic - CEO 2012-12-03 Tivorsan Pharmaceuticals has begun planning the design of its Phase I Duchenne’s muscular dystrophy (DMD) program for biglycan, said Dr Joel Braunstein, director, acting CEO. The study will include both healthy volunteers and boys with DMD, he added. Braunstein declined to provide a timeframe for Phase I initiation. Tivorsan will seek to work with a close group of clinical advisors and one or more specialized centers that have the infrastructure necessary to conduct a study in boys with DMD, the CEO said. The Phase I trial will enroll a meaningful number of patients and assess the drug’s effects on a variety of functional measures and biomarkers, Braunstein added. Final plans have not yet been made, though Braunstein indicated the company anticipates a clinical development program that seeks to understand the effects of biglycan in younger and older DMD patients as well as those with a variety of dystrophin mutations. Currently, the privately held Providence, Rhode Island-based firm is focused on optimizing the scale-up manufacturing process, the CEO said. Tivorsan is working with a contract manufacturing organization for the necessary CMC (chemistry, manufacturing and control) work, he indicated, though declined to provide more details on the group. The company conducts certain CMC activities internally and also has a close relationship with professor of neuroscience Justin Fallon’s research group/laboratory at Brown University, Rhode Island. Tivorsan may look to work with preclinical contract research organizations with specialized experience in areas such as toxicology work, pharmacokinetics, biodistribution and assay development. Biglycan regulates the expression of utrophin and other dystrophin-associated protein complex (DAPC) components, according to company information. It is designed to help compensate for loss of dystrophin by recruiting utrophin to the muscle cell membrane and stabilizing the DAPC, Braunstein said. Utrophin is a protein with similar structure and function to dystrophin, which is highly expressed during fetal development but then mostly disappears from muscle when normal adult dystrophin protein takes over, he explained. A treatment that restores utrophin in the muscle cell membrane when dystrophin is absent has potential to prevent muscular dystrophy pathology, Braunstein noted. The therapy is administered through systemic injection, he said. Follow-on financing activities to a Series A from 2011 are in progress by the company. The firm has explored funding with certain VC groups, angel investors as well as large and specialty biopharma companies in the space.
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BioPharm Insight Tivorsan is not working with a financial adviser at the present time. The company closed a Series A during 4Q11. Investors and the amount were not disclosed, though one investor was LifeTech Development Partners, an affiliate group that Braunstein is a part of. Tivorsan has also received support from philanthropic groups such as Parent Project Muscular Dystrophy, Muscular Dystrophy Association, Charley’s Fund and Nash Avery Foundation. by Anusha Kambhampaty in New York
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