Avenue Therapeutics licenses ATX-04 from Duke for Pompe disease treatment

Avenue Therapeutics Inc. (OTC: ATXI) announced it has entered into an exclusive worldwide license agreement with Duke University for ATX-04, a treatment for Pompe disease. The agreement covers patents and know-how for the drug, also known as clenbuterol.

ATX-04 is a selective β2-adrenergic agonist that has demonstrated improvements in muscle function in clinical studies. A study conducted at Duke University showed the treatment resulted in improvements in six-minute walk distance and increased respiratory muscle strength in Pompe disease patients receiving enzyme replacement therapy.

Pompe disease is a rare inherited disorder caused by deficiency of the enzyme acid α-glucosidase, resulting in progressive skeletal and respiratory muscle weakness. The condition affects patients across a spectrum from severe infantile-onset to later onset forms.

Clenbuterol has regulatory approvals outside the United States for respiratory diseases and is administered orally. The drug increases protein synthesis and muscle fiber size while enhancing lysosomal biogenesis and intracellular trafficking of the enzyme deficient in Pompe disease.

Under the license terms, Avenue will receive exclusive worldwide rights to develop and commercialize ATX-04 for Pompe disease and potentially other neuromuscular indications. The company will also assume control of Duke's existing clinical and regulatory assets, including the investigational new drug application and FDA orphan drug designation.

Duke University will receive an upfront payment, potential development and commercial milestone payments, plus royalties on net sales. Avenue plans to advance ATX-04 through late-stage clinical development, initially focusing on treating Pompe disease as an adjunct to enzyme replacement therapy.

"This license allows us to advance a differentiated, mechanism-based therapy with the potential to meaningfully enhance outcomes for patients receiving standard-of-care ERT," said Alexandra MacLean, Avenue's chief executive officer.

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