Avenue Therapeutics (OTC: ATXI) licenses ATX-04 Pompe therapy from Duke
Rhea-AI Filing Summary
Avenue Therapeutics, Inc. entered an exclusive worldwide license agreement with Duke University for patents and know‑how covering ATX-04 (clenbuterol), a β2-adrenergic agonist in clinical development for Pompe disease. Avenue will make an upfront payment, reimburse certain patent costs, and pay development, regulatory and commercial milestones plus tiered low single-digit royalties on future net sales.
ATX-04 has human proof-of-concept data from a Duke study in Pompe patients on enzyme replacement therapy (ERT), showing improvements in six-minute walk distance, respiratory muscle strength, reduced muscle glycogen, increased GAA activity and broader gene-expression benefits, and was generally well tolerated. Avenue plans a late-stage clinical program, initially as an adjunct to ERT, and will assume Duke’s existing clinical and regulatory assets, including the IND and FDA orphan drug designation, with potential expansion into other neuromuscular indications.
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Insights
Avenue adds a late-stage rare disease asset via ATX-04 license.
Avenue Therapeutics is securing an exclusive worldwide license to ATX-04 from Duke University, targeting Pompe disease. The asset comes with human proof‑of‑concept data showing functional, respiratory, biochemical and molecular improvements on top of standard enzyme replacement therapy, and was generally well tolerated.
The agreement aligns Avenue with a late-stage, mechanism-based adjunct therapy in a rare disease setting, plus the transfer of Duke’s IND and orphan drug designation. Economics are structured as upfront, milestones and tiered low single-digit royalties, a common biopharma pattern that ties Duke’s upside mainly to development progress and eventual sales.
Future value depends on Avenue’s ability to execute the planned late-stage clinical program and satisfy diligence obligations under the license. Subsequent company filings and trial updates will clarify timing, study design and any expansion into additional neuromuscular indications beyond Pompe disease.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the
Securities Exchange Act of 1934
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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
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Securities registered pursuant to Section 12(b) of the Act: None
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| Title of Class | Trading Symbol(s) |
| Common Stock | ATXI (OTC Markets Group, Inc.) |
Indicate by check mark whether the registrant is an emerging growth
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| Item 1.01. | Entry into Material Definitive Agreement. |
On February 18, 2026, Avenue Therapeutics, Inc. (the “Company”) entered into a license agreement (the “License Agreement”) with Duke University (“Duke”), whereby the Company obtained from Duke an exclusive, worldwide license to certain patents and know-how for the development and commercialization of products, including ATX-04 (clenbuterol), for the treatment of lysosomal storage diseases, subject to customary retained rights for Duke and other non-profit or governmental institutions to use the licensed technology for non-commercial research and educational purposes. Under the License Agreement, the Company agreed to make an upfront payment and reimburse certain patent expenses to Duke, and to make development, regulatory, and commercial milestone payments upon the achievement of certain milestones. In addition, the Company is obligated to pay a tiered low single-digit royalty on future net sales of licensed products. The Company intends to advance ATX-04 through a late-stage clinical development program leveraging existing human safety and efficacy data, with an initial focus on treating Pompe disease as an adjunct to enzyme replacement therapy (“ERT”).
The License Agreement includes customary development and commercialization diligence obligations for the Company, as well as customary termination provisions, including for uncured material breach, certain insolvency-related events and specified patent challenges, and otherwise continues on a product-by-product and country-by-country basis for so long as royalties are payable.
The foregoing description of the License Agreement does not purport to be complete and is qualified in its entirety by reference to the full text of the License Agreement, which the Company expects to file as an exhibit to a subsequent filing with the Securities and Exchange Commission.
| Item 8.01 | Other Events. |
The Company issued a press release on February 23, 2026 announcing its entry into the License Agreement, a copy of which is attached as Exhibit 99.1 hereto.
| Item 9.01 | Financial Statements and Exhibits. |
(d) Exhibits
The following exhibits are furnished herewith:
| Exhibit Number |
Description | |
| 99.1 | Press Release, dated February 23, 2026 | |
| 104 | Cover Page Interactive Data File (embedded within Inline XBRL document) |
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| AVENUE THERAPEUTICS, INC. | ||
| (Registrant) | ||
| Date: February 23, 2026 | ||
| By: | /S/ DAVID JIN | |
| David Jin | ||
| Interim Principal Financial Officer and Chief Operating Officer | ||
Exhibit 99.1
Avenue Therapeutics Enters into Exclusive Worldwide License Agreement for ATX-04 for the Treatment of Pompe Disease
ATX-04 is a selective β2-adrenergic agonist with human proof-of-concept data demonstrating improved muscle function and enhanced response to enzyme replacement therapy
MIAMI, February 23, 2026 – Avenue Therapeutics, Inc. (OTC: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for rare and neurologic diseases, today announced that it has entered into an exclusive worldwide license agreement with Duke University for patents and know-how pertaining to ATX-04 (clenbuterol), a well-characterized small-molecule β2-adrenergic agonist, in clinical development for the treatment of Pompe disease.
Pompe disease is a rare, inherited lysosomal storage disorder caused by deficiency of the enzyme acid α-glucosidase (GAA), resulting in progressive skeletal and respiratory muscle weakness. The disease presents across a wide clinical spectrum, from severe infantile-onset to later onset forms marked by progressive muscle weakness and respiratory failure, and remains associated with significant morbidity despite available enzyme replacement therapies (ERT).
Clenbuterol is a clinically validated, orally administered selective β2-adrenergic agonist with regulatory approvals outside the United States for the treatment of respiratory diseases. The drug has well-established anabolic effects on skeletal muscle, resulting in increased protein synthesis and muscle fiber size. In addition, clenbuterol enhances lysosomal biogenesis and intracellular trafficking of GAA, the enzyme deficient in Pompe disease, leading to reduced glycogen accumulation in muscle tissue.
A clinical study conducted at Duke University in patients with Pompe disease on baseline ERT, led by Principal Investigator Dwight D. Koeberl, M.D., Ph.D., demonstrated that ATX-04 was associated with meaningful improvements across multiple clinically and biologically relevant domains. Treatment with ATX-04 resulted in improvements in six-minute walk distance, reflecting enhanced functional capacity, as well as increased respiratory muscle strength, including maximal inspiratory pressure. ATX-04 was also associated with reductions in muscle glycogen burden assessed by biopsy, increased GAA activity with improved intracellular trafficking, and broad normalization of disease-relevant gene expression. The therapy was generally well tolerated with chronic, titrated dosing. Collectively, these findings support ATX-04’s possible use as a mechanistic potentiator of ERT.
“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,” said Alexandra MacLean, M.D., Chief Executive Officer of Avenue Therapeutics. “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.”
Avenue plans to advance ATX-04 through a late-stage clinical development program leveraging existing human safety and efficacy data from other jurisdictions, with an initial focus on treating Pompe disease as an adjunct to ERT with the potential to expand into other related indications.
Under the terms of the license, Duke University has granted Avenue an exclusive, worldwide license to develop and commercialize products covered by Duke’s patents and related know-how, including ATX-04 (clenbuterol) for Pompe disease, with the potential to expand into other designated neuromuscular indications. Avenue will also assume control of Duke’s existing clinical and regulatory assets for ATX-04, including the investigational new drug (IND) application and the U.S. Food and Drug Administration (FDA) orphan drug designation for Pompe disease. Duke University will receive an upfront payment, as well as potential development, regulatory and commercial milestone payments, plus royalties on net sales.
About Avenue Therapeutics
Avenue Therapeutics, Inc. (OTC: ATXI) is a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of rare and neurologic diseases. Avenue is headquartered in Miami, FL and was founded by Fortress Biotech, Inc. (Nasdaq: FBIO). For more information, visit www.avenuetx.com.
Forward-Looking Statements
This press release contains predictive or “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of current or historical fact contained in this press release, including statements that express our intentions, plans, objectives, beliefs, expectations, strategies, predictions or any other statements relating to our future activities or other future events or conditions are forward-looking statements. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “will,” “should,” “would” and similar expressions are intended to identify forward-looking statements. These statements are based on current expectations, estimates and projections made by management about our business, our industry and other conditions affecting our financial condition, results of operations or business prospects. These statements are not guarantees of future performance and involve risks, uncertainties and assumptions that are difficult to predict. Therefore, actual outcomes and results may differ materially from what is expressed or forecasted in, or implied by, the forward-looking statements due to numerous risks and uncertainties. Factors that could cause such outcomes and results to differ include, but are not limited to, risks and uncertainties arising from: the fact that we currently have no drug products for sale and that our success is dependent on our product candidates receiving regulatory approval and being successfully commercialized; the possibility that serious adverse or unacceptable side effects are identified during the development of our current or future product candidates, such that we would need to abandon or limit development of some of our product candidates; our ability to successfully develop, partner, or commercialize any of our current or future product candidates; our ability to realize the anticipated benefits of any exclusive license agreements, including the ability to successfully develop the product candidates licensed and to comply with the diligence, milestone and other obligations under such agreements; the substantial doubt raised about our ability to continue as a going concern, which may hinder our ability to obtain future financing; the significant losses we have incurred since inception and our expectation that we will continue to incur losses for the foreseeable future; uncertainty related to the timing and amounts expected to be realized from future milestone, royalty or similar future revenue streams, if at all; our need for substantial additional funding, which may not be available to us on acceptable terms, or at all, which unavailability could force us to delay, reduce or eliminate our product development programs or commercialization efforts; our reliance on third parties for several aspects of our operations; our reliance on clinical data and results obtained by third parties that could ultimately prove to be inaccurate, unreliable, or unacceptable to regulatory authorities; the possibility that we may not receive regulatory approval for any or all of our product candidates, or that such approval may be significantly delayed due to scientific or regulatory reasons; the fact that even if one or more of our product candidates receives regulatory approval, they will remain subject to substantial regulatory scrutiny; the effects of current and future laws and regulations relating to fraud and abuse, false claims, transparency, health information privacy and security, and other healthcare laws and regulations; the effects of competition for our product candidates and the potential for new products to emerge that provide different or better therapeutic alternatives for our targeted indications; the possibility that the government or third-party payors fail to provide adequate coverage and payment rates for our product candidates or any future products; our ability to establish sales and marketing capabilities or to enter into agreements with third parties to market and sell our product candidates; our exposure to potential product liability claims; risks related to the protection of our intellectual property and our potential inability to maintain sufficient patent protection for our technology and products; our ability to maintain compliance with the obligations under our intellectual property licenses and funding arrangements with third parties, without which licenses and arrangements we could lose rights that are important to our business; the fact that Fortress Biotech, Inc. controls a majority of the voting power of our outstanding capital stock and has rights to receive significant share grants annually; and those risks discussed in our filings which we make with the SEC. Any forward-looking statements speak only as of the date on which they are made, and we undertake no obligation to publicly update or revise any forward-looking statements to reflect events or circumstances that may arise after the date of this press release, except as required by applicable law. Investors should evaluate any statements made by us in light of these important factors.
Contact:
Jaclyn Jaffe
Avenue Therapeutics, Inc.
(781) 652-4500
ir@avenuetx.com
FAQ
What did Avenue Therapeutics (ATXI) announce regarding ATX-04 and Duke University?
Avenue Therapeutics announced an exclusive worldwide license agreement with Duke University for patents and know-how related to ATX-04 (clenbuterol) in Pompe disease. The deal gives Avenue rights to develop and commercialize ATX-04 globally, building on human proof-of-concept data generated in Duke’s clinical study.
What is ATX-04 and how is it intended to treat Pompe disease for Avenue Therapeutics (ATXI)?
ATX-04 is an orally administered, selective β2-adrenergic agonist being developed as a treatment for Pompe disease. It aims to enhance enzyme replacement therapy by improving muscle function, increasing respiratory strength, reducing muscle glycogen, and improving intracellular trafficking of the deficient GAA enzyme in patients.
What clinical results support Avenue Therapeutics’ (ATXI) ATX-04 program in Pompe disease?
A Duke University clinical study in Pompe patients on baseline ERT showed ATX-04 improved six-minute walk distance, increased respiratory muscle strength, reduced muscle glycogen on biopsy, increased GAA activity, and broadly normalized disease-relevant gene expression, while being generally well tolerated with chronic, titrated dosing.
What financial terms are disclosed in Avenue Therapeutics’ (ATXI) license for ATX-04?
The agreement requires Avenue to pay Duke an upfront payment, reimburse certain patent expenses, and make development, regulatory and commercial milestone payments. Duke is also entitled to tiered low single-digit royalties on future net sales of licensed products incorporating ATX-04 or related technologies.
How does Avenue Therapeutics (ATXI) plan to develop ATX-04 after licensing it from Duke?
Avenue plans a late-stage clinical development program for ATX-04, initially as an adjunct to enzyme replacement therapy in Pompe disease. The company will leverage existing human safety and efficacy data and may later expand development into other designated neuromuscular indications covered under the license.
What regulatory assets for ATX-04 will Avenue Therapeutics (ATXI) obtain from Duke University?
Under the license, Avenue will assume control of Duke’s existing clinical and regulatory assets for ATX-04, including the investigational new drug (IND) application and the U.S. FDA orphan drug designation for Pompe disease, supporting further development in this rare disorder.
Filing Exhibits & Attachments
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