What changed in this filing

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Item 1. Business

2026-03-19 compared with 2025-03-18 · 37 added, 37 removed, 307 unchanged (19% of the section changed)

Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases.

We currently have seven commercial rare disease products:

INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone.

We have six additional product candidates in late-stage development:

ET-400, ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector.

We currently have eight commercial rare disease products:

INCRELEX®, ALKINDI SPRINKLE®, KHINDIVI TM , GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone.

We have five additional product candidates in late-stage development:

ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector.

INCRELEX® – This biologic product was approved by the FDA in August 2005 as a treatment for children who suffer from severe primary insulin-like growth factor 1 deficiency (SPIGFD).

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ALKINDI SPRINKLE® is protected by three issued patents that extend to 2032, 2033, and 2034.

KHINDIVI TM – This product was approved by the FDA in May 2025 as a replacement therapy in pediatric patients five years of age and older for adrenocortical insufficiency.

KHINDIVI is the only FDA-approved oral solution formulation of hydrocortisone.

It comes in a 1mg/ml strength designed to eliminate the need to split or crush tablets, and to offer simple and accurate dosing specifically tailored to each patient’s needs.

It does not require refrigeration, mixing, or shaking – it is a ready-to-use oral liquid solution.

KHINDIVI is designed to offer administration simplicity and dosing accuracy, and to provide a therapy option for patients who have difficulty swallowing tablets or with special administration needs, such as patients with a gastric tube.

GALZIN® is FDA-approved as a maintenance treatment for patients with Wilson disease who have been initially treated with a chelating agent.

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We offer the product through our Eton Cares patient support program that provides high-touch, personalized service tailored for rare disease patients and their providers.

PKU GOLIKE ® - In March 2024, w e acquired the U.S.

PKU GOLIKE ® - In March 2024, we acquired the U.S.

rights to PKU GOLIKE, which is a next generation medical formula product engineered with the patent protected, pharmaceutical grade Physiomimic™ technology for the dietary management of phenylketonuria (“PKU”) under medical supervision.

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We acquired the product in October 2023 and launched the product in February 2024.

ET-400 - We submitted to the FDA a New Drug Application (“NDA”) for the product in 2024, which was originally scheduled with a Prescription Drug User Fee Amendments (“PDUFA”) goal date of February 28, 2025.

On February 6, 2025, we were notified by the FDA that it had applied a three-month extension to the original PDUFA goal date of February 28, 2025.

The new PDUFA goal date is now May 28, 2025.

ET-600 – ET-600 is under development for the treatment of central diabetes insipidus.

We expect to submit an NDA for the product in 2025, which could allow for an approval and launch of the product in 2026.

ET-600 – In July 2025, ET-600’s NDA was accepted for review by the FDA and assigned a PDUFA target action date of February 25, 2026.

The Company has scheduled the production of launch inventory for the first quarter of 2026 in preparation for a commercial launch shortly after the anticipated approval.

The Company recently held an ET-600 advisory meeting with leading healthcare practitioners within the pediatric endocrinology community.

Amglidia ® - In November 2024, we entered into a licensing agreement with AMMTeK., pursuant to which we have agreed to acquire the U.S.

rights to Amglidia® (glyburide oral suspension).

rights to Amglidia® (glyburide oral suspension), which we also have a license to the issued patent on Amglidia®.

Amglidia® is being developed for the treatment of neonatal diabetes mellitus, a rare condition estimated to impact approximately 300 patients in the U.S.

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AMMTeK has conducted a post-approval study tracking five years of real-world safety and efficacy in European patients, which will be used to support Eton’s NDA submission.

ET-700 – We are developing this zinc extended release product for the treatment of Wilson disease.

We plan to hold a meeting with the FDA in 2025 to discuss the product’s clinical pathway.

ET-700 – We are scheduled to begin its proof-of-concept positron emission tomography ("PET") study during 2026.

The study is expected to validate the efficacy of the Company’s propriety, patent-pending extended-release formulation, and if positive, would support the initiation of a dose ranging and pivotal clinical trial in late 2026 or early 2027.

We have also submitted a patent for ET-700, which is currently pending.

ET-800 – We are developing this ready-to-use, injectable liquid hydrocortisone in a vial for the hospital market.

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Adrenal Insufficiency

Endocrinology

Adrenal Insufficiency

Wilson Disease

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Endocrinology

Adrenal Insufficiency

NDA Submitted (PDUFA Goal Date of May 28, 2025)

Endocrinology

Diabetes Insipidus

Under Development

PDUFA goal date of February 25, 2026

Endocrinology

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Our team has completed over 150 business development transactions throughout their careers and their industry connections and track record provide the Company with proprietary deal flow.

We typically avoid participating in broker led transactions of auction processes.

We typically avoid participating in broker led transactions or auction processes.

Regulatory expertise – our knowledge and experience in gaining FDA approval, and particularly our knowledge within the 505(b)(2) regulatory pathway, provides drug sponsors with the opportunity to leverage existing data or literature to drastically expedite drug development timelines and reduce investment.

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Sales and Marketing

We currently commercialize seven products under our own label with our internal infrastructure and sales force.

We market and sell INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone in the United States consistent with applicable laws.

We currently commercialize eight products under our own label with our internal infrastructure and sales force.

We market and sell INCRELEX®, ALKINDI SPRINKLE®, KHINDIVI TM , GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone in the United States consistent with applicable laws.

These products are distributed to patients via specialty pharmacies, which support customer service and reimbursement activities.

Research and Development

We currently have eight employees that support our product research and development (“R&D”) priorities and strategy.

We currently have twelve employees that support our product research and development (“R&D”) priorities and strategy.

In addition, we utilize external sources for various product development activities, including the resources of our product development partners for certain product candidates and through the use of contract laboratory services on a fee for service model.

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All products are manufactured in compliance with current Good Manufacturing Processes (“GMP”), and our internal quality system requires us to enter quality agreements with and audit all of our manufacturers prior to commercializing the product.

Our choice to rely on external manufacturers significantly reduces the amount of capital invested in our business and allows us the flexibility to pursue a broad range of opportunities beyond the specific capabilities of a single facility.

Our choice to rely on external manufacturers significantly reduces the amount of capital required to be invested in our business and allows us the flexibility to pursue a broad range of opportunities beyond the specific capabilities of a single facility.

Intellectual Property

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ALKINDI SPRINKLE® is protected by three issued patents that extend to 2034.

ET-400 is protected by two issued patents that extend to 2043 and there are additional patent applications related to this product under review with the U.S.

KHINDIVI TM is protected by two issued patents that extend to 2043, and there are additional patent applications related to this product under review with the U.S.

Patent and Trademark Office (“USPTO”).

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The testing and approval process require substantial time, effort, and financial resources, and we cannot be certain that any approval will be granted on a timely basis, if at all.

Preclinical development of a drug candidate can take several years to complete, with no guarantee that an IND based on those studies will become effective to even permit clinical testing to begin.

Preclinical development of a drug candidate can take several years to complete, with no guarantee that an IND application based on those studies will become effective to even permit clinical testing to begin.

Even though several of our pharmaceutical product candidates utilize active drug ingredients that are commercially marketed in the United States in other dosage forms, we need to establish safety and effectiveness of those active ingredients in the formulation and dosage forms that we are developing.

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Section 505(b)(2) New Drug Applications

We intend to submit applications for certain product candidates via the 505(b)(2) regulatory pathway.

We submit applications for certain product candidates via the 505(b)(2) regulatory pathway.

As an alternate path for FDA approval of new indications or new formulations of previously approved products, a company may submit a Section 505(b)(2) NDA, instead of a “stand-alone” or “full” NDA.

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Such reforms could have an adverse effect on anticipated revenue from product candidates that we may successfully develop and for which we may obtain regulatory approval and may affect our overall financial condition and ability to develop product candidates.

At December 31, 2024, we had 31 full-time employees, eight of whom are engaged in research and development activities, 18 are engaged in sales and marketing operations and five of whom are engaged in general corporate and strategy roles.

At December 31, 2025, we had 44 full-time employees, twelve of whom are engaged in research and development activities, twenty-four are engaged in sales and marketing operations and eight of whom are engaged in general corporate and strategy roles.

We periodically utilize outside consultants on an as-needed basis, including medical consultants.

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Solely for convenience, the trademarks and trade names in this Annual Report are referred to without the symbols ® and ™, but such references should not be construed as any indication that their respective owners will not assert, to the fullest extent under applicable law, their rights thereto.

Recent Development

As previously reported in our Form 8-K/A filed on March 7, 2025, following the closing of our acquisition of the INCRELEX® product on December 19, 2024, the Company’s advisors determined that, under relevant accounting rules and interpretations, the acquisition must be accounted for by the Company as a business combination rather than an asset purchase.

As a result of this determination, the Company is required to file an amendment to the Original Report to provide separate audited financial statements and unaudited pro forma financial information for INCRELEX®, specified by Item 9.01 of Form 8-K, no later than 71 calendar days after the date that the Original Report on Form 8-K was required to be filed with the SEC, which was March 7, 2025.

While the Company is required to file audited financial statements of INCRELEX® for the years ended December 31, 2023 and 2022 and the nine months ended September 30, 2024 and 2023, as well as unaudited proforma financial information for the years ended December 31, 2024 and 2023, INCRELEX® was accounted for as a single product within Ipsen's consolidated financial statements and not as a separate business.

Historical financial statements for the Increlex product do not exist, and as a result, the Company believes that it will be unable to create the required financial statements.

Accordingly, the Company has filed a request with the SEC that the SEC waive the aforementioned financial statement filing requirement.

Risk Factors Summary

You should carefully consider the risks set forth in the section of this Annual Report of Form 10-K entitled “Risk Factors” beginning on page 10 of this Annual Report, including, but not limited to, the following:

We may have significant research, regulatory and development expenses as we advance our product candidates.

The Inflation Reduction Act and related Medicare reforms may adversely affect the prices we realize and the demand for our products.

Our participation in U.S.

government price reporting and discount programs (including the Medicaid Drug Rebate Program, 340B, VA/FSS and TRICARE) could adversely affect the net prices we realize and expose us to significant liabilities.

Third-party coverage and reimbursement and health care cost containment initiatives, including PBM practices, formulary controls and utilization management may constrain our future revenues.

We may operate or support patient services and assistance programs, and government scrutiny of these activities could require us to curtail such programs, reducing patient access and adversely affecting demand for our products.

If we obtain or seek marketing authorizations and commercialization outside the United States, we may be subject to pricing controls and health technology assessment processes that could delay commercialization and reduce net prices.

We may need to grow the size of our organization, and we may experience difficulties in managing this growth.

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Sales of counterfeits of any of our product candidates, as well as unauthorized sales of any of our product candidates, may have adverse effects on our revenues, business and results of operations and damage our brand and reputation.

We have entered into several arrangements with related parties for the development and marketing of certain product candidates and these arrangements present potential conflicts of interest.

We face competition from other biotechnology and pharmaceutical companies and our operating results will suffer if we fail to compete effectively.

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Significant additional labeling or warning requirements or limitations on the availability of our products may inhibit sales of affected products.

Current and future legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our product candidates and affect the prices we may obtain.

We are subject to Drug Supply Chain Security Act requirements, and failure to comply could disrupt our distribution and result in enforcement action.

Changes in U.S.

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political landscape may adversely affect our business, results of operations, financial condition, and prospects.

If we market any of our products or product candidates in a manner that violates health care fraud and abuse laws, or if we violate government price reporting laws, we may be subject to civil or criminal penalties.

If we market any of our products or product candidates in a manner that violates health care fraud and abuse laws, we may be subject to civil or criminal penalties.

We may not be able to establish agreements with third parties with whom we wish to collaborate and, if we are able to establish them, we may not be able to establish them on commercially reasonable terms, which could result in alterations or delays of our development and commercialization plans.

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Clinical drug development involves a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results.

Third-party coverage and reimbursement and health care cost containment initiatives and treatment guidelines may constrain our future revenues.

We are subject to extensive laws and regulations related to data privacy, and our failure to comply with these laws and regulations could harm our business.

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Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.

What changed 10-K

Item 1. Business