What changed in this filing

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Item 1. Business

2025-03-18 compared with 2024-03-14 · 81 added, 38 removed, 263 unchanged (31% of the section changed)

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

The Company currently has four commercial rare disease products, ALKINDI SPRINKLE® for the treatment of adrenocortical insufficiency;

Carglumic Acid for the treatment of hyperammonemia due to N-acetylglutamate synthas e (“NAGS”) deficiency ;

Betaine Anhydrous for the treatment of homocystinuria;

and Nitisinone for the treatment of hereditary tyrosinemia type 1 (HT-1).

The Company has three additional product candidates in late-stage development:

ET-400, ET-600, and ZENEO® hydrocortisone autoinjector.

ALKINDI SPRINKLE ® (hydrocortisone granules) – This product was approved by the FDA in September 2020 as a replacement therapy for Adrenocortical Insufficiency (“AI”) in children under 17 years of age.

The product is the first and only FDA-approved granule hydrocortisone formulation designed to help provide accurate dosing for newborns and children with adrenal insufficiency.

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.

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).

The product is approved in 40 territories, including the United States and the European Union.

We acquired and launched the product in December 2024.

ALKINDI SPRINKLE® – This product was approved by the FDA in September 2020 as a replacement therapy for Adrenocortical Insufficiency (“AI”) in children under 17 years of age.

The product is the first and only FDA-approved granule hydrocortisone formulation designed to help provide accurate dosing for newborns and children with AI.

We acquired U.S.

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We believe there are approximately 10,000 children currently suffering from AI in the United States.

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

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

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

It is estimated that less than 5,000 patients in the U.S.

are currently being treated for Wilson Disease.

We acquired the product in December 2024 and assumed the commercialization of the product in the U.S.

in March 2025.

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.

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.

PKU GOLIKE’s® taste-masked, odor-free coating technology is designed to provide a better taste and a superior experience compared to alternative PKU medical formulas.

In addition, PKU GOLIKE’s delayed amino acid release formulation is designed to keep patients full for a longer period of time.

Carglumic Acid Tablets – Our Carglumic Acid product is an FDA-approved generic version of Carbaglu®.

Our product is approved for the treatment of acute and chronic hyperammonemia due to NAGS deficiency.

We acquired marketing rights to the product in October 2021 and launched the product in December 2021.

Our product is approved for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate synthase (“NAGS”) deficiency .

We acquired the marketing rights to the product in October 2021 and launched the product in December 2021.

We promote the product with our internal sales force.

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

ET-400 – Eton expects to submit a new drug application for the product during 2024, which could allow for an approval and launch of the product in 2025.

ET-600 – Eton expects to submit a new drug application for the diabetes insipidus product during 2025, which could allow for an approval and launch of the product in early 2026.

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.

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).

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

The product was approved by the European Medicines Agency in 2018 and has been granted Orphan Drug Designation by the FDA.

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

ZENEO ® Hydrocortisone Autoinjector – Our ZENEO® hydrocortisone autoinjector product candidate is a proprietary needle-free autoinjector under development for the treatment of adrenal crisis.

We expect to submit a New Drug Application (“NDA”) for the product in 2025, which could allow for FDA approval in 2026.

Eton Pharmaceuticals

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Eton Category

Carglumic Acid Tablets

NAGS deficiency

Endocrinology

Severe Primary IGF-1 Deficiency

ALKINDI SPRINKLE®

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Adrenal Insufficiency

Wilson Disease

Phenylketonuria

Carglumic Acid Tablets

NAGS deficiency

Betaine Anhydrous

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Tyrosinemia Type 1

Submission Expected in 2024

Endocrinology

Adrenal Insufficiency

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

Endocrinology

Diabetes Insipidus

Submission Expected in 2025

Under Development

Endocrinology

Neonatal diabetes mellitus

Under Development

Wilson Disease

Under Development

Endocrinology

Adrenal Insufficiency

Under Development

ZENEO® Hydrocortisone

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Adrenal Crisis

Submission Expected in 2025

Under Development

Goals and Strengths

Our goal is to become a leading profitable pharmaceutical company focused on developing and commercializing treatments for rare diseases.

Our goal is to become a leading pharmaceutical company focused on developing and commercializing treatments for rare diseases.

We believe we are unique in the pharmaceutical industry in our ability to identify, acquire, and advance products through the development and regulatory process.

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Sales and Marketing

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

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

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.

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

Research and Development

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

We currently have eight 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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ALKINDI SPRINKLE® is protected by three issued patents that extend to 2034.

ET-400 is protected by an issued patent that extends to 2043, and there are additional patent applications related to this product and ET-600 under review with the U.S.

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.

Patent and Trademark Office (“USPTO”).

In June 2021, we acquired U.S.

marketing rights from Crossject to the ZENEO® hydrocortisone autoinjector.

ET-600 is protected by an issued patent that extends to 2044 and there are additional patent applications related to this product under review with the USPTO.

We intend to seek patent protection on our internally developed products as circumstances warrant.

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Phase 1 clinical trials are conducted in small groups of healthy volunteers to assess safety of various dosing regimens and pharmacokinetics.

After a safe dose has been established, in Phase 2 clinical trials the drug is administered to a limited patient population with the target disease or condition to identify possible adverse events and safety risks, to conduct a preliminary evaluation of the efficacy of the product candidate in treating the targeted disease or condition.

After a safe dose has been established, in Phase 2 clinical trials the drug is administered to a limited patient population with the target disease or condition to identify possible adverse events and safety risks, and to conduct a preliminary evaluation of the efficacy of the product candidate in treating the targeted disease or condition.

Phase 3 clinical trials are usually multi-center, double-blind controlled trials in larger numbers of subjects to assess as fully as possible both the safety and effectiveness of the drug, establish the overall risk-benefit of the product candidate, and provide adequate information for the labeling of the product.

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If an NDA submission is accepted for filing, the FDA begins an in-depth review of the NDA.

Under the Prescription Drug User Fee Act (“PDUFA”) the FDA’s goal is to complete its initial review and respond to the applicant within ten months of submission, unless the application relates to an unmet medical need, or is for a serious or life-threatening indication, in which case the goal may be within six months of NDA submission.

Under a PDUFA, the FDA’s goal is to complete its initial review and respond to the applicant within ten months of submission, unless the application relates to an unmet medical need, or is for a serious or life-threatening indication, in which case the goal may be within six months of NDA submission.

However, the review process and the target response date under PDUFA may be extended if the FDA requests or the NDA sponsor otherwise provides additional information or clarification regarding information already provided in the NDA.

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For example, if we change the manufacturer of a product or our API, the FDA may require stability or other data from the new manufacturer.

Such data will take time and are costly to generate, and the delay associated with generating these data may cause interruptions in our ability to meet commercial demand, if any.

Such data will take time and are costly to generate, and the delay associated with generating this data may cause interruptions in our ability to meet commercial demand, if any.

While physicians may use products for indications that have not been approved by the FDA, we may not label or promote the product for an indication that has not been approved.

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Healthcare Reform

In the United States and some foreign jurisdictions, there have been, and we expect there will continue to be, a number of legislative and regulatory changes to the health-care system that could prevent or delay marketing approval pharmaceutical products, restrict or regulate post-approval activities and affect our ability to profitably sell our product candidates.

In the United States and some foreign jurisdictions, there have been, and we expect there will continue to be, a number of legislative and regulatory changes to the health-care system that could prevent or delay marketing approval of pharmaceutical products, restrict or regulate post-approval activities and affect our ability to profitably sell our product candidates.

Among policy makers and payors in the United States and elsewhere, there is significant interest in promoting changes in healthcare systems with the stated goals of containing healthcare costs, improving quality and expanding access.

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These reductions went into effect in April 2013 and, due to subsequent legislative amendments to the statute, will remain in effect through 2030 unless additional action is taken by Congress.

● On April 13, 2017, CMS published a final rule that gives states greater flexibility in setting benchmarks for insurers in the individual and small group marketplaces, which may have the effect of relaxing the essential health benefits required under the Health Care Reform Law for plans sold through such marketplaces.

● On April 13, 2017, the Centers for Medicare and Medicaid Services (“CMS”) published a final rule that gives states greater flexibility in setting benchmarks for insurers in the individual and small group marketplaces, which may have the effect of relaxing the essential health benefits required under the Health Care Reform Law for plans sold through such marketplaces.

● On May 30, 2018, the Right to Try Act, was signed into law.

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Congressional Inquiries and proposed and enacted federal and state legislation designed to, among other things, bring more transparency to product pricing, reduce the cost of prescription drugs under Medicare, review the relationship between pricing and manufacturer and patient programs, and reform government program reimbursement methodologies for products.

In July 2021, President Biden signed an Executive Order affirming the administration’s policy to (i) support legislative reforms that would lower the prices of prescription drug and biologics, including by allowing Medicare to negotiate drug prices, by imposing inflation caps, and, by supporting the development and market entry of lower-cost generic drugs and biosimilars;

In July 2021, President Biden signed an Executive Order affirming the administration’s policy to (i) support legislative reforms that would lower the prices of prescription drugs and biologics, including by allowing Medicare to negotiate drug prices, by imposing inflation caps, and, by supporting the development and market entry of lower-cost generic drugs and biosimilars;

and (ii) support the enactment of a public health insurance option.

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Pursuant to court order, the removal and addition of the aforementioned safe harbors were delayed and recent legislation imposed a moratorium on implementation of the rule until January 1, 2032.

Although a number of these and other proposed measures may require authorization through additional legislation to become effective, and the Biden administration may reverse or otherwise change these measures, both the Biden administration and Congress have indicated that they will continue to seek new legislative measures to control drug costs.

Although a number of these and other proposed measures may require authorization through additional legislation to become effective, and the Trump administration may reverse or otherwise change these measures.

In August 2022, President Biden signed the Inflation Reduction Act (“IRA”) which provides for (i) the government to set or negotiate prices for select high-cost Medicare Part D (beginning in 2026) and Medicare Part B drugs (beginning in 2028) that are more than nine years (for small-molecule drugs) or 13 years (for biological products) from their FDA approval, (ii) manufacturers to pay a rebate for Medicare Part B and Part D drugs when prices increase faster than inflation beginning in 2022 for Medicare Part D and 2023 for Medicare Part B drugs, and (iii) Medicare Part D redesign which replaces the current coverage gap provisions and establishes a $2,000 cap for out-of-pocket limits costs for Medicare beneficiaries beginning in 2025, with manufacturers being responsible for 10% of costs up to the $2,000 cap and 20% after that cap is reached.

Implementation of the IRA is expected to be carried out through upcoming actions by regulatory authorities, the outcome of which is uncertain.

Implementation of the IRA is expected to be carried out through actions by regulatory authorities, the outcome of which is uncertain.

In August 2023, the Biden Administration published the first ten medicines subject to the Medicare Drug Price Negotiation program.

In addition, there will be a new Medicare manufacturer discount program agreement expected to be signed in March 2024 that will change our discounting obligations for all medicines in Medicare, with few exceptions, beginning in 2025.

The Medicare Drug Price Negotiation Program is currently subject to legal challenges and therefore, the outcome of the Program remains uncertain.

We continue to evaluate the impact of the IRA on our business, operations and financial condition and results as the full effect of the IRA on our business and the pharmaceutical industry remains uncertain.

Eton signed a program agreement in February 2024 with an effective date of January 1, 2025 that could change our discounting obligations for all medicines in Medicare, however the impact to our business is minimal since Eton products are prescribed to very few Medicare patients.

Should the program change such that it significantly increases our discounting obligations, or should the number of our products prescribed to Medicare patients increase substantially, it could have a material adverse effect on our net revenues, financial condition, results of operations or prospects.

At the state level, legislatures have increasingly passed legislation and implemented regulations designed to control pharmaceutical and biological product pricing, including price or patient reimbursement constraints, discounts, restrictions on certain product access and marketing cost disclosure and transparency measures, and, in some cases, designed to encourage importation from other countries and bulk purchasing.

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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.

We currently have 30 full-time employees, 9 of whom are engaged in research and development activities, 16 are engaged in sales and marketing operations and 5 of whom are engaged in general corporate and strategy roles.

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.

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

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We rely significantly on information technology and any failure, inadequacy, interruption or security lapse of that technology, including any cybersecurity incidents, could harm our ability to operate our business effectively.

Use of artificial intelligence-based software by us or third parties with which we contract, may lead to the release of confidential information which may impact our ability to realize the benefits of our intellectual property.

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.

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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.

Changes in U.S.

trade policy, threats of international tariffs, and changes to the U.S.

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.

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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