This document contains forward-looking statements concerning Eton Pharmaceuticals, Inc. ("Eton", the "Company," "we," "us," and "our"). The words "believe," "may," "will," "potentially," "estimate," "continue," "anticipate," "intend," "could," "would," "project," "plan," "expect" and similar expressions that convey uncertainty of future events or outcomes are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements concerning the following ...

Core Insights - Eton Pharmaceuticals has received FDA approval for KHINDIVI, the first and only hydrocortisone oral solution, aimed at pediatric patients with adrenocortical insufficiency [1][3][9] - The commercial launch of KHINDIVI is expected in the week of June 2, with projected combined peak sales of KHINDIVI and ALKINDI SPRINKLE exceeding $50 million annually [1][6] Product Details - KHINDIVI is formulated as a 1mg/ml oral solution, designed for easy dosing without the need for refrigeration or mixing, making it suitable for patients with swallowing difficulties [2][4] - The product is indicated for replacement therapy in pediatric patients aged five years and older with adrenocortical insufficiency [9] Market Context - There are over 5,000 pediatric patients in the U.S. diagnosed with adrenal insufficiency, highlighting a significant market opportunity for KHINDIVI [6] - Eton's existing commercial team will promote KHINDIVI alongside ALKINDI SPRINKLE, providing additional treatment options for patients and caregivers [5][7] Strategic Partnerships - KHINDIVI will be available exclusively through Anovo, a specialty pharmacy, which will also manage the Eton Cares Program to assist patients with prescription fulfillment and financial support [7][8] Company Overview - Eton Pharmaceuticals focuses on developing treatments for rare diseases and currently has eight commercial products, including KHINDIVI and ALKINDI SPRINKLE [41]

Company Overview - Eton Pharmaceuticals, Inc is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases [3] - The company currently has seven commercial rare disease products: INCRELEX, ALKINDI SPRINKLE, GALZIN, PKU GOLIKE, Carglumic Acid, Betaine Anhydrous, and Nitisinone [3] - Eton has six additional product candidates in late-stage development: ET-400, ET-600, Amglidia, ET-700, ET-800, and ZENEO hydrocortisone autoinjector [3] Upcoming Events - Eton's CEO, CFO, and Chief Business Officer will participate in two upcoming investor conferences [1] - The 25th Annual B. Riley Securities Investor Conference is scheduled for May 22, 2025, at 11:15 am ET in Marina Del Rey, California [5] - The 22nd Annual Craig-Hallum Institutional Investor Conference will take place on May 28, 2025, in Minneapolis, Minnesota [5]

Financial Data and Key Metrics Changes - The company's first quarter revenue was $17.3 million, a 117% increase from $8 million in the first quarter of 2024 [35] - Product sales reached $14 million, up 76% from $8 million in the prior year, driven by increased sales of Alkindi Sprinkle and the addition of Incralyx [36] - Adjusted gross profit was $12 million, or 69.5% of total revenue, compared to $5.2 million or 65.6% in the prior year [36] - Adjusted EBITDA for the first quarter was $3.7 million, compared to $500,000 in the prior year [39] - The company reported a net loss of $1.6 million for the quarter, compared to a net loss of $800,000 in the prior year [40] Business Line Data and Key Metrics Changes - Alkindi Sprinkle has shown strong growth for 17 consecutive quarters, with an increase in new patient referrals in 2025 [15] - Incralyx has seen rapid patient acquisition, reaching over 90 active patients shortly after its relaunch, with expectations to reach 100 by year-end [8][12] - Galzin, acquired for Wilson's disease treatment, is expected to generate significant revenue as the company addresses access and affordability issues [21][22] Market Data and Key Metrics Changes - The company is focusing on pediatric endocrinology, leveraging its existing sales force and infrastructure to drive growth in this segment [6][15] - The market for severe primary IGF-1 deficiency is estimated to have around 200 patients in the U.S., with potential for significant growth through increased awareness and screening [6][12] - The Wilson disease market is estimated to impact approximately 10,000 people in the U.S., with only about 2,000 diagnosed and treated, indicating a substantial unmet need [19][20] Company Strategy and Development Direction - The company aims to become a leading player in the ultra-rare disease market, with a strong focus on product launches and pipeline development [30][31] - The strategy includes harmonizing U.S. and EU labels for Incralyx to expand treatment opportunities significantly [10][12] - The company is also advancing new product candidates like ET-700 and ET-800, targeting unmet needs in rare disease treatments [24][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's growth trajectory, expecting to exit 2025 with an annual revenue run rate of approximately $80 million [36] - The management team is optimistic about the upcoming product launches and the potential for significant revenue growth from the pipeline [31] - The company is not concerned about tariff impacts, as most products are produced domestically, and any European purchases represent less than 5% of forecasted revenue [33][34] Other Important Information - The company has made significant investments in infrastructure to support growth, including a dedicated metabolic sales team and commercial activities for product relaunches [37][38] - The company expects to maintain adjusted gross margins of approximately 70% for the full year 2025, with long-term goals exceeding 75% by 2028 [36] Q&A Session Summary Question: Update on Incralyx FDA meeting - The meeting request has been submitted, with expectations for a follow-up in July and a submission for label change in Q3 [42] Question: Recent interactions with FDA regarding ET-400 - The company is on track for approval within two weeks, with final labeling discussions underway [44] Question: Weekly Incralyx prescription trends - The company has significantly increased the number of patients, with expectations to exceed revenue goals this year [54][55] Question: Expected timing for ET-400 commercial revenue - The launch is anticipated around June, with revenue expected to ramp up in Q3 [56][57] Question: Commentary on tariff risk exposure - The company does not foresee a meaningful impact from tariff proposals, as it primarily sells domestically [60][62] Question: Feedback on Galzin relaunch - The relaunch has been well-received, with a strong patient uptake and zero copay for patients [68][70]

Financial Data and Key Metrics Changes - The company's first quarter revenue was $17.3 million, a 117% increase compared to $8 million in the first quarter of 2024 [37] - Product sales reached $14 million, up 76% from $8 million in the same quarter last year, driven by increased sales of Alkindi Sprinkle and the addition of Incralyx [38] - Adjusted gross profit was $12 million, or 69.5% of total revenue, compared to $5.2 million or 65.6% in the prior year [39] - Adjusted EBITDA for the first quarter was $3.7 million, compared to $500,000 in the prior year [43] - The total net loss was $1.6 million, compared to a net loss of $800,000 in the prior year [43] Business Line Data and Key Metrics Changes - The acquisition and relaunch of Incralyx and Dalzan contributed to strong growth in existing products [6] - Alkindi Sprinkle has shown consistent growth for 17 consecutive quarters, with an increase in new patient referrals in 2025 [16] - The company expects to reach approximately 80,000,000 annual revenue run rate by the end of 2025 [38] Market Data and Key Metrics Changes - The company is focusing on pediatric endocrinology, leveraging its existing sales force and commercial infrastructure to raise awareness of severe primary IGF-1 deficiency [8] - The market for ET400 is estimated to have around 5,000 adrenal insufficiency patients under nine years of age, presenting a compelling opportunity [18] Company Strategy and Development Direction - The company aims to become a leading player in the ultra-rare disease market, with a focus on expanding its product portfolio through acquisitions and licensing [31] - The strategy includes harmonizing U.S. and EU labels for Incralyx to expand treatment opportunities significantly [13] - The company is also developing ET700, an extended-release version of Galzin, to address dosing challenges and improve patient outcomes [24] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory of existing products and the potential for new product approvals in the near future [6] - The company is optimistic about the upcoming launch of ET400, expecting rapid uptake and significant revenue impact in subsequent quarters [60] - Management noted that the tariff situation is not a major concern, as most products are produced in the U.S. and the impact of European purchases is minimal [34][35] Other Important Information - The company has made significant investments in infrastructure to support growth, including a dedicated metabolic sales team and commercial activities for product relaunches [41] - The company reported a cash balance of $17.4 million and generated $2.1 million in operating cash flow during the quarter [43] Q&A Session Summary Question: Update on Incralyx label expansion meeting with FDA - The meeting request has been submitted, and the company expects to have it in the coming weeks, likely in July [45] Question: Recent interactions with FDA regarding ET400 - The company is on track for approval within two weeks, with the final step being labeling discussions [47] Question: Weekly Incralyx prescription trends - The company has significantly increased the number of patients and expects to meet or exceed revenue goals this year [56] Question: Expected timing for commercial revenue from ET400 - The launch is expected around the approval date, with revenue anticipated in Q3 [59] Question: Feedback on Galzin relaunch - The relaunch has been well-received, with a strong uptake and zero copay for patients, improving access significantly [70]

Core Insights - Eton Pharmaceuticals reported exceptional first-quarter results for 2025, achieving record financial performance and launching two recently acquired products, INCRELEX and GALZIN, while advancing its development pipeline [2][4][11]. Financial Performance - Eton's first-quarter 2025 net revenues reached $17.3 million, a 117% increase from $8.0 million in the same period last year, with product sales of $14.0 million reflecting a 76% growth [11][12]. - The company reported a gross profit of $9.9 million, up 97% from $5.0 million year-over-year, driven by increased product sales [13]. - Adjusted EBITDA for the first quarter was $3.7 million, compared to $0.5 million in the prior year [18]. Product Developments - The relaunch of INCRELEX, a treatment for Severe Primary IGF-1 Deficiency, has seen a significant increase in new patients since its acquisition and relaunch [5][12]. - Eton is preparing for the anticipated launch of ET-400, targeting a $200 million market opportunity for oral hydrocortisone, with a PDUFA date set for May 28, 2025 [6][12]. - The company also relaunched GALZIN for Wilson disease and announced the development of ET-700, an extended-release form of zinc acetate [7][12]. Licensing and Partnerships - Eton out-licensed the commercial rights to INCRELEX outside the U.S. to Esteve Pharmaceuticals for up to ten years, receiving a $4.3 million upfront licensing fee [8][9]. - The licensing revenue recognized during the first quarter included $1.8 million from this out-licensing agreement [11]. Cash Position and Expenses - As of March 31, 2025, Eton had cash and cash equivalents of $17.4 million, with an operating cash flow of $2.1 million generated during the quarter [21]. - Research and Development (R&D) expenses increased to $1.2 million from $0.7 million year-over-year, primarily due to the development of ET-700 and ET-800 [15]. General and Administrative (G&A) expenses rose to $9.2 million, reflecting increased advertising and promotional costs [17].

Core Viewpoint - Eton Pharmaceuticals, Inc. will report its first quarter 2025 financial results on May 13, 2025, and will host a conference call to discuss these results [1]. Company Overview - Eton Pharmaceuticals is focused on developing and commercializing treatments for rare diseases, currently offering seven commercial products: INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone [3]. - The company has six additional product candidates in late-stage development: ET-400, ET-600, Amglidia®, ET-700, ET-800, and ZENEO® hydrocortisone autoinjector [3]. Investor Relations - Investors can submit questions via email to investorrelations@etonpharma.com during the conference call [1]. - The live webcast of the conference call will be accessible on Eton's website, with an archived version available for 30 days post-event [2].

Core Points - Eton Pharmaceuticals has submitted a New Drug Application (NDA) for ET-600, an oral solution of desmopressin for treating central diabetes insipidus, with a 10-month FDA review expected and a potential launch in Q1 2026 [1][2] - If approved, ET-600 will be the only FDA-approved oral liquid formulation of desmopressin, addressing the needs of approximately 3,000 pediatric patients in the U.S. [2] - Eton holds a patent for ET-600 that expires in 2044, with additional patent applications under review [2] - The company has seven commercial rare disease products and six additional product candidates in late-stage development [3]