Avenue Therapeutics, Inc. (OTC: ATXI), a specialty pharmaceutical company dedicated to developing therapies for rare and neurologic diseases, has announced an exclusive worldwide licensing agreement with Duke University for patents and know-how related to ATX-04 (clenbuterol). This small-molecule β2-adrenergic agonist is currently in clinical development aimed at treating Pompe disease.
Pompe disease is a rare, inherited lysosomal storage disorder that results from a deficiency of the enzyme acid α-glucosidase (GAA). This deficiency leads to progressive skeletal and respiratory muscle weakness, manifesting in various forms from severe infantile-onset to later-onset types. Despite existing enzyme replacement therapies (ERT), the condition remains associated with significant morbidity.
Clenbuterol, which has regulatory approvals in other countries for treating respiratory diseases, is known for its anabolic effects on skeletal muscle, promoting increased protein synthesis and muscle fiber size. Moreover, it enhances lysosomal biogenesis and intracellular trafficking of GAA, thus aiding in the reduction of glycogen accumulation in muscle tissues affected by Pompe disease.
A clinical study conducted at Duke University under the guidance of Principal Investigator Dwight D. Koeberl, M.D., Ph.D., demonstrated that ATX-04 significantly improved various clinically relevant endpoints in patients on baseline ERT. Key improvements included enhanced six-minute walk distance, increased respiratory muscle strength, and reductions in muscle glycogen burden, alongside increased GAA activity and improved gene expression normalization. ATX-04 was generally well tolerated, showcasing its potential as a mechanistic potentiator of ERT.
“ATX-04 represents an asset with a favorable risk profile, supported by compelling clinical data generated at Duke, demonstrating functional, biochemical, and molecular benefits in Pompe disease,” stated Alexandra MacLean, M.D., CEO of Avenue Therapeutics. “This license allows us to advance a differentiated, mechanism-based therapy that has the potential to significantly enhance outcomes for patients undergoing standard-of-care ERT.”
Avenue Therapeutics plans to propel ATX-04 into late-stage clinical development, utilizing existing data from other regions, with an initial focus on Pompe disease as an adjunct to ERT while considering expansion into other related indications. Under the licensing agreement, Avenue has secured exclusive rights to develop and commercialize products covered by Duke's patents, including ATX-04 for Pompe disease, with possibilities for extension into other neuromuscular indications. Duke University will receive an upfront payment and may also benefit from potential milestone payments along with royalties on net sales.
For more details on Avenue Therapeutics and their initiatives, visit Inside Ticker.