Avenue Therapeutics to develop add-on Pompe disease treatment
Company secures patents related to clenbuterol, temporarily renamed ATX-04
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Avenue Therapeutics has secured rights to develop clenbuterol, an experimental add-on treatment for Pompe disease that has already shown promise in early clinical testing.
The company announced it has entered into an exclusive worldwide license agreement with Duke University for patents and technical expertise related to the experimental treatment, which it will be developing under the temporary name ATX-04.
“ATX-04 represents an asset with a favorable risk profile, supported by compelling human clinical data generated at Duke demonstrating functional, biochemical, and molecular benefit in Pompe disease,” Alexandra MacLean, MD, Avenue’s CEO, said in a company press release. “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 [enzyme replacement therapy].”
ATX-04 could bolster efficacy of available ERTs
Pompe disease is a genetic disorder characterized by a deficiency of acid alpha-glucosidase (GAA), an enzyme needed to break down the complex sugar glycogen. In the absence of GAA enzyme, glycogen builds up to toxic levels in cells, particularly muscle cells, causing damage that ultimately drives disease symptoms. Enzyme replacement therapy (ERT), in which a functional version of the GAA enzyme is administered into the bloodstream, is currently the standard treatment for Pompe.
ATX-04 belongs to a class of medications called beta-2 adrenergic agonists. These therapies work by activating a specific molecular receptor on muscle cells. Medications in this class are currently used mainly to help manage certain respiratory diseases, because they can ease airflow obstruction and improve respiratory function.
ATX-04 isn’t approved for lung diseases in the U.S., though it is authorized for this use in certain other countries. Avenue noted that it plans to use established data in other diseases to help streamline the clinical development of ATX-04 in Pompe disease.
In addition to its effects on respiratory muscles, ATX-04 has been shown to have anabolic effects on muscle tissue, which may include muscle growth, enhanced protein production in muscle, and increased muscle fiber size. ATX-04 has also been shown to help promote the formation of lysosomes, the cellular compartment where the GAA enzyme is normally active, and to help the GAA enzyme travel more efficiently inside cells. As such, it may offer benefits for Pompe patients by reducing glycogen accumulation and bolstering the efficacy of available ERTs.
Researchers at Duke previously conducted a Phase 1/2 clinical trial (NCT01942590) that tested ATX-04 in 17 patients with late-onset Pompe disease who were on a stable ERT regimen. Results from the yearlong study showed ATX-04 was generally well tolerated, with no serious side effects reported, and that patients tended to experience improvements in measures of walking ability and muscle strength.
Under the new licensing agreement, Duke will receive an initial upfront payment from Avenue, with the potential for additional payments as developmental milestones are met. Financial details of the deal were not disclosed.
