Tivorsan Pharmaceuticals closes private placement funds to support R&D of Biglycan for Duchenne Muscular Dystrophy

Providence, RI, February 5, 2016 – 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 it has raised approximately $1.8 million in private financing from a leading institutional investor, as well as other existing and new accredited investors.

The financing will be used to fund research and development of Tivorsan’s lead compound, recombinant human Biglycan, for the treatment of DMD and other neuromuscular disorders, and for working capital and general corporate purposes. In addition, the financing will serve as a bridge to the Company’s proposed Series D convertible preferred financing, which is expected to close in the second quarter.

“We are pleased to complete this important financing and appreciate the support and participation of new and existing investors,” said Jim Connolly, CEO of Tivorsan. “The financing will allow us to continue our critical development efforts of Biglycan for our lead indication in DMD, including a planned IND submission and initiation of Phase 1 clinical trials in early 2017.”

T.R. Winston & Company served as placement agent to Tivorsan in the financing.

About Tivorsan Pharmaceuticals
Tivorsan is a biotechnology company developing novel treatment approaches to treat a series of rare diseases including all forms of DMD and Becker’s Muscular Dystrophy (BMD), as well as other progressive, life threatening neuromuscular disorders, including Congenital Muscular Dystrophy, ALS, Spinal Muscular Dystrophy and Myasthenia Gravis. The Company’s lead candidate, recombinant human Biglycan, is based on the scientific work from the Fallon Laboratory at Brown University as led by Justin Fallon, Ph.D., scientific founder of Tivorsan.

About Biglycan

Biglycan is a small, naturally occurring extracellular matrix protein naturally found in muscle. It has a unique Mechanism of Action: upregulating utrophin, nNOS 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 to develop a series of therapies for a range of neuromuscular disorders with significant unmet medical need. Tests in the most widely used mouse model of DMD have shown that systemically-delivered recombinant human biglycan (rhBGN) upregulates utrophin and activates the dystrophin-independent membrane stabilization program. Biglycan has been shown to improve muscle health, structure and health in this mouse model and indicates that Biglycan treated mice have healthier skeletal muscle that undergoes less death and degeneration.

Biglycan targets all forms of DMD, regardless of mutation status. In addition, biglycan should be synergistic with other therapeutic approaches that are in development for DMD, such as exon skipping. The proposed synergy should be supported by biglycan’s upregulation of utrophin, as well as its targeting of nNOS (neuronal nitric oxide synthase), a key enzyme that is functionally compromised in Duchenne and Becker Muscular Dystrophy.

Importantly, the active form of rhBGN lacks the complex carbohydrate (GAG) side chains that are present in most forms of biglycan. This simplified structure greatly facilitates rhBGN manufacture and mitigates off-target effects.

About T.R. Winston & Company
T.R. Winston & Company (“TRW”), is a full-service investment, corporate and merchant banking firm. TRW is dedicated to serving corporate and institutional clients, family offices and high-net-worth individuals in the areas of public and private financing, asset management, mergers and acquisitions, health care regulatory, reimbursement and policy advisory services, and financial restructurings. In addition, we also offer our clients trading and prime services.

Forward-Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, regarding the research, development and potential commercialization of pharmaceutical products. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of the statements, and could cause actual outcomes and results to differ materially from current expectations. Tivorsan is providing this information as of the date of this press release and does not undertake any obligation to update forward-looking statements as a result of new information, future events or otherwise.

Contacts:

Jim Connolly for Tivorsan Pharmaceuticals, Inc.

610-659-8082
jconnolly@tivorsan.com

Investors:

Rob Kristal, CFA, for Tivorsan Pharmaceuticals, Inc.

646-760-2300
rk@trwinston.com







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