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Avenue Therapeutics Licenses ATX-04 for Pompe Disease

Story Highlights
  • Avenue Therapeutics licensed global rights to ATX-04 from Duke University to develop and commercialize it for Pompe disease and related neuromuscular indications.
  • Clinical data from Duke suggest ATX-04 can enhance enzyme replacement therapy in Pompe disease, potentially improving functional and respiratory outcomes for patients while bolstering Avenue’s rare disease portfolio.
  • Looking for the best stocks to buy? Follow the recommendations of top-performing analysts.
Avenue Therapeutics Licenses ATX-04 for Pompe Disease

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Avenue Therapeutics ( (ATXI) ) has issued an update.

On February 23, 2026, Avenue Therapeutics announced it had entered an exclusive worldwide license agreement with Duke University covering patents and know-how for ATX-04 (clenbuterol), a selective β2-adrenergic agonist in clinical development for Pompe disease. The deal grants Avenue global rights to develop and commercialize ATX-04 as an adjunct to enzyme replacement therapy in Pompe disease and potentially other neuromuscular indications, while transferring Duke’s clinical and regulatory assets, including an existing IND and FDA orphan drug designation.

Pompe disease is a rare inherited disorder marked by progressive skeletal and respiratory muscle weakness, and current enzyme replacement therapies still leave patients with significant morbidity. Human proof-of-concept data from a Duke-led study showed ATX-04 improved six-minute walk distance, respiratory muscle strength, glycogen burden, GAA activity, and disease-relevant gene expression, positioning Avenue to advance a late-stage, mechanism-based therapy that could enhance standard-of-care outcomes and potentially strengthen its presence in the rare neuromuscular disease market.

The most recent analyst rating on (ATXI) stock is a Hold with a $0.16 price target. To see the full list of analyst forecasts on Avenue Therapeutics stock, see the ATXI Stock Forecast page.

Spark’s Take on ATXI Stock

According to Spark, TipRanks’ AI Analyst, ATXI is a Neutral.

The score is held back mainly by weak financial quality (ongoing losses and negative free cash flow despite improvement) and very bearish technicals (price well below key moving averages with negative MACD). Valuation is constrained by a negative P/E and no dividend, while recent corporate events provide some offset through a positive asset-sale transaction and governance stability.

To see Spark’s full report on ATXI stock, click here.

More about Avenue Therapeutics

Avenue Therapeutics, Inc. is a Miami-based specialty pharmaceutical company focused on developing and commercializing therapies for rare and neurologic diseases. Founded by Fortress Biotech, Inc. and listed on the OTC market under the ticker ATXI, the company targets niche indications where existing treatments leave substantial unmet medical need.

Average Trading Volume: 5,535

Technical Sentiment Signal: Strong Sell

Current Market Cap: $477.5K

Find detailed analytics on ATXI stock on TipRanks’ Stock Analysis page.

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