Slides
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2025 INVESTOR DAY March 18, 2025
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Special note regarding forward-looking statements Safe Harbor 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: • our future financial and operating results; • our intentions, expectations and beliefs regarding anticipated growth, market penetration and trends in our business; • the timing and success of our plan of commercialization; • our ability to successfully develop and clinically test our product candidates; and • our ability to file for FDA approval of our product candidates through the 505(b)(2) regulatory pathway. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including those described in “Risk Factors” section of the Registration Statement on Form S-1 filed Eton with the Securities and Exchange Commission on September 25, 2018. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this document may not occur and actual results could differ materially and adversely from those an ticipated or implied in our forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although we believe that the expectations reflected in our forward-looking statements are reasonable, we cannot guarantee that the future results, levels of activity, performance or events and circumstances describe d in the forward-looking statements will be achieved or occur. This document contains only basic information concerning Eton. Because it is a summary it does not contain all of the informatio n you should consider before investing. Before you invest, you should read the prospectus in that registration statement and other documents Eton has filed with the SEC for more complete information about Eton and this offering. You may get these documents for free by visiting EDGAR on the SEC Web site at www.sec.gov. Alternatively, Eton, any underwriter or any dealer participating in the offering will arrange to send you the prospectus if you request it by calling National Securities Corporation toll- free at 1-800-832-6084. Disclaimer: Products discussed in this presentation are under development and not yet approved by the FDA. The information presented here is to the best of the company's current knowledge and assumptions, which may be different from the labeling and conditions provided by the FDA upon approval, which appr oval itself is not guaranteed. In no event should this information be construed as marketing or promotion, or providing directions for use of any product, all of which are continge nt on FDA approval.
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Agenda • Reviewing Eton’s Transformational Six Months and Forward Vision • Record Fourth Quarter Financial Results • Multi-lever Increlex Growth Strategy • Adrenal Insufficiency Franchise Outlook • Unlocking Wilson Disease Opportunity • ET-600 Clinical Results and Near-Term NDA Submission • Two New Pipeline Candidates • 2025 Financial Outlook • Q&A
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Today’s Speakers SEAN BRYNJELSEN Chief Executive Officer JAMES GRUBER Chief Financial Officer DAVID KREMPA Chief Business Officer IPEK ERDOGAN-TRINKAUS Chief Commercial Officer
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Reviewing Eton’s Transformational Six Months
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To build a leading ultra-rare disease company with a relentless commitment to patients and innovative therapies. Our Vision Patients First Ultra-Rare Focus Clearing the path for better patient access and therapy experience The Greatest Number of New Therapies Achievable Rapidly growing our portfolio to bring more innovative treatments to patients Making the greatest impact where it matters the most – and too rare for big pharma
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OCT 3 Signed Increlex acquisition Expanded portfolio, delivered record revenue, strong commercial launch momentum Transformational 6 Months NOV 12 Reported record 3Q product revenue and reached GAAP profitability NOV 25 Acquired Amglidia DEC 19 Closed acquisition of Increlex DEC 30 Acquired Galzin Launched new metabolic sales force JAN 2 MAR 3 Galzin commercial launch MAR 14 Positive ET-600 clinical study results MAR 18 Reported record Q4 product sales Acquired 2 High Value Commercial Products (Increlex®, Galzin®) Added 3 Attractive Pipeline Assets (Amglidia®, ET-700, ET-800) Exceptional Execution Across All Functions
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Eton was successfully built with a three-pillar strategy and is well positioned to sustain the momentum 3 Pillar Growth Strategy Track Record 16 straight quarters of sequential revenue growth (driven by Alkindi Sprinkle and Carglumic Acid) Outlook Attractive long-term growth opportunities for Increlex, Galzin, and Alkindi Sprinkle Track Record Developed ET-400 internally Outlook Additional high-value programs in late-stage development (ET-600, ET-700, ET-800, Amglidia®, Zeneo®) Track Record Executed high-ROI acquisitions of Increlex, Galzin, Carglumic Acid, and Alkindi Sprinkle Outlook Well positioned to continue acquiring strategically and financially attractive products. Strong balance sheet with significant access to capital. Organic Growth of Existing Products Internal Pipeline Products External Acquisitions And Licensing
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Eton's commercial strategy is designed to effectively meet the distinct needs of ultra-rare patient populations Concentrated prescriber bases | Small number of specialists within pediatric endocrinology and metabolics enables highly targeted, efficient sales force coverage and marketing efforts Meaningful partnerships with patient advocacy groups | Through deep collaboration, Eton supports patient and caregiver communities to drive awareness, promote education, and enhance patient resources Strategic collaboration with key thought leaders and professional societies | Advisory boards and joint initiatives to engage with healthcare professionals and societies leading therapeutic advancements Eton Cares patient support program for all Rx products | $0 co-pay for eligible patients, quick-start and bridge programs, nurse hotline, monthly patient check-in, and exclusive distribution through high-touch specialty pharmacy
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Built one of the broadest ultra rare disease portfolios in the industry 1 Alkindi Sprinkle® - Pediatric Adrenal Insufficiency Increlex® - Severe Primary Insulin-Like Growth Factor 1 Deficiency Galzin® - Wilson disease Carglumic Acid - Hyperammonemia PKU GoLike® - Phenylketonuria Betaine Anhydrous - Homocystinuria Nitisinone – Hereditary Tyrosinemia Type 1 (HT-1) ET-400 – Pediatric Adrenal Insufficiency ET-600 – Diabetes Insipidus Amglidia® - Neonatal Diabetes ET-700 - Wilson disease ET-800 - Endocrinology Zeneo® - Adrenal Crisis 2 3 4 5 6 7 8 9 10 11 12 13 CMC Development Phase III/Pivotal Study NDA Submission Commercial 13 7 6 PEDIATRIC ENDOCRINOLOGY METABOLIC ADDED IN THE LAST 6 MONTHS Disclosing for First Time Products Commercial Products Pipeline Programs
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Demonstrated strong performance, with a roadmap for the next wave of growth $3M $11M $26M $39M $0 $5 $10 $15 $20 $25 $30 $35 $40 2021 2022 2023 2024 Clear path to $100M+ of near-term revenue with the additions of Increlex, Galzin and ET-400. Note: Includes only product sales and royalty revenue. Excludes licensing revenue Product Revenue in $ millions
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Q4 2024 & Full Year 2024 Results
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16th Straight Quarter of Product Sales Growth $0.4 $0.6 $0.8 $1.1 $2.2 $2.4 $3.2 $3.5 $5.3 $6.5 $7.0 $7.3 $8.0 $9.1 $9.8 $11.6 Q4 2020 Q1 2021 Q2 2021 Q3 2021 Q4 2021 Q1 2022 Q2 2022 Q3 2022 Q4 2022 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Q1 2024 Q2 2024 Q3 2024 Q4 2024 +59% Revenue Growth over Q4 2023 Note: Includes only product sales and royalty revenue. Excludes licensing revenue Revenue in $ millions
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Q4 2024 Financial Results REVENUE Q4 2023 $7.3 Q4 2024 $11.6 Growth 59% GROSS PROFIT $3.5 $6.5 78% R&D EXPENSES $1.0 -$0.9 N/A SG&A EXPENSES $4.6 $6.7 47% OPERATING INCOME -$2.0 $0.6 N/A NET INCOME -$2.3 -$0.6 N/A $ Millions
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Increlex
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Increlex brings strong strategic alignment, with multiple growth levers to accelerate adoption and expansion SPIGFD (Severe Primary IGF-1 Deficiency) is a rare genetic disorder characterized by impaired growth due to low insulin-like growth factor 1 (IGF-1) levels despite normal or elevated growth hormone (GH) Attractive Product Profile Highly durable, complex biologic product Synergistic Commercialization 94% overlap with existing pediatric endocrinologist targets Multiple Avenues to Create Value Increase adoption with sales and marketing focus Critical unmet rare disease need with no alternative treatment options Maximizes efficiency/investment of dedicated sales force and marketing Optimize pricing while prioritizing patient affordability Broaden treatment access via US label harmonization
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A return to 185 US active patients would produce estimated US revenue of ~$50 million Increlex Historic Global Revenue 2007 2008 2009 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 2020 2021 2022* 2023 2024* 185 US Patients at Peak 67 Active US Patients at Year End 2024 Reduction/elimination of promotional activitiesShortage in US and EU €28.3M €13.7M Note: Global sales in Euros. *2022 net revenue impacted by 3m EUR gross-to-net true up. 2024 revenue impacted by return of all channel inventory in December
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• Why low penetration? - Low awareness among pediatricians resulting in delayed diagnosis - Many causes of short stature IGF-1 deficiency not top of mind - Engrained habit of growth hormone use may not help SPIGFD patients and may result in missing the critical window of opportunity for optimal outcomes to reach their full potential - Diagnosing and continuously re-testing is challenging which leads to late therapy start and therapy ineligibility • Key unmet needs and opportunities - Need to drive awareness of SPIGFD among stakeholders (pediatricians, pediatric endocrinologists, caregivers, advocacy groups) - HCPs to continuously screen all short stature patients for SPIGFD - Strong KOL consensus on criticality of harmonizing US SPIGFD definition with EU to treat the right patient population Insights from thought leaders shaping Increlex relaunch and expansion Held advisory board with 7 US key opinion leaders in pediatric endocrinology in January
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Eton’s commercial strategy is poised to drive significant market penetration Increlex Brand Strategy Re-engage the pediatric endocrinology community HCPs to screen all short stature patients for SPIGFD HCPs to consistently use IGF-1 testing to accurately diagnose SPIGFD patients HCPs to use Increlex in all SPIGFD patients Drive patient awareness and engagement Caregivers to advocate for and support SPIGFD awareness and treatment Caregivers to advocate for access to Increlex for their SPIGFD child Establish Eton as the best-in-class partner for SPIGFD HCPs to understand and embrace Rx process, Eton Cares, and leverage Eton Cares resources Caregivers to understand and use Eton Cares, including $0 copay* *For commercially eligible patients.
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Exclusive Specialty Pharmacy • $0 copay for commercial patients • Insurance specialists, pharmacist and nurse support to assist patients and offices; direct overnight therapy delivery Strong 60-day patient transition and demand generation 67 patients transferred within 1 week in late Dec ‘24 81 active patients through mid-March Building strong momentum in the first 90 days of Increlex relaunch Drive Increlex HCP penetration and enhance access Accelerate awareness and adoption through omnichannel strategic efforts Strategic Sales Force Deployment • 11 Rare Disease Specialists with strong ped endo expertise • 695 HCP primary targets Personalized HCP Engagement and Education • HCP email campaigns • Programmatic and EHR advertising • New HCP and patient websites • Peer-to-peer education / speaker programs • HCP and patient educational resources Key Society and Patient Advocacy Partnerships • PES Silver Mission Alliance Partner (MAP) • Strong partnerships with Human Growth Foundation (HGF) and MAGIC • KOL engagement and advisory boards • Key conference presence at PES, PENS, ENDO, AAP national and regionals
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US (FDA) EUROPE (EMA) Height Index Standard Deviation Score (SDS) of ≤-3 AND IGF-1 Levels ≤-3 SDS <2.5 percentile for age and gender Estimated Patient Population with Current Indication 200 1,000+ Harmonizing US definition of SPIGFD to match EU would significantly expand the market opportunity Estimated to expand the US patient population ~5x to approximately 1,000 patients ~-1.96 SDS (2.5 percentile) -3.0 SDS (~0.13 percentile) SPIGFD Definition Source: Company estimates, graph assumes IGF-1 levels are normally distributed
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Increlex US Label Harmonization Harmonize US Increlex label definition of SPIGFD to match EU VISION Increlex global registry has monitored EU patient outcomes and safety for >350 patients over more than 15 years Existing patient registry data will be used to support Eton’s case for harmonizing the US label FDA meeting expected in mid-2025; supplement filing expected in H2 2025 Expected 6-month review for potential label revision in H1 2026
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Increlex poised for significant US revenue growth, with or without label harmonization 2023 US Revenue Q2 2025 Estimated forward run-rate Estimated market opportunity at full penetration (200 patients) Potential market opportunity with harmonized label (~1,000 patients) ~$11M ~$20M ~$55M $250M+ >2.5X GROWTH POTENTIAL REMAINING TO REACH FULL PENETRATION WITH CURRENT LABEL Source: Company Estimates
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Adrenal Insufficiency Franchise Outlook
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Maximizing therapy penetration in AI – a dual product franchise strategy to address diverse HCP and patient preferences across the age continuum 2,500 patients 5,000 patients 10,000 patients Source: Company Estimates 0-4 years 8 years & under 17 years & under Adrenal Insufficiency Estimated Pediatric Prevalence in the US ET-400 Unlocks a larger share of the $200MM+ market, offering tailored accuracy and removes the burden of getting the dose ‘just right’ Lowest flexible dosing accuracy – dosing accuracy to 0.1mg Ideal for patients who cannot tolerate tablets or granules; patients with severe dysphagia or texture issues Simplicity and convenience for parents (i.e. night dosing) Suitable for G-tube/enteral feeding Alkindi Sprinkle Alkindi is the first and only hydrocortisone treatment designed to provide accurate dosing for pediatric patients with AI – addressing an unmet need for accurate, low-dose treatment, transforming standard of care Avoids compounding and crushing tablets Convenient, on-the-go form Easy and fast administration (no syringe/clean up) Four dosage options for treatment precision and weight- based titration Unlocking Greater Penetration Complementary portfolio positioning to address a larger patient population
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Alkindi Sprinkle continues growth with strong runway to capture share Progressively increased prescribing HCPs, indicating growing confidence and adoption Steady growth in active patients on therapy; Jan ‘25 second highest month in referrals since launch Robust omnichannel marketing to accelerate HCP adoption and foster patient retention 100 300 500 700 Jan - 24 Feb - 24 Mar - 24 Apr - 24 May - 24 Jun- 24 Jul - 24 Aug - 24 Sep - 24 Oct - 24 Nov - 24 Dec - 24 Jan - 25 Feb - 25 Prescribing HCPs 0 50 100 150 200 250 300 350 400 450 500 Dec-20 Feb-21 Apr-21 Jun-21 Aug-21 Oct-21 Dec-21 Feb-22 Apr-22 Jun-22 Aug-22 Oct-22 Dec-22 Feb-23 Apr-23 Jun-23 Aug-23 Oct-23 Dec-23 Feb-24 Apr-24 Jun-24 Aug-24 Oct-24 Dec-24 Feb-25 Active Patients by Month
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28.2% Oral Suspension From a compound pharmacy Crush Tablets (whole, halved, or quartered) 21.8% 20.5% Alkindi Sprinkle Solid Tablets Manually crush tablets and mix with liquid or food (homemade suspension) 29.5% ET-400 filling a critical gap for an FDA-approved therapy that offers simplicity and accuracy Source: February 2025 MAGIC Foundation Facebook survey of 156 caregivers of Adrenal Insufficiency patients four years old and younger 50% of patients report using a compounded or a homemade liquid. “How do you give your child their daily dose of hydrocortisone?”
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Upon ET-400 approval, FDA guidelines will prohibit compounded suspensions. However, ET-400’s advantages are expected to drive adoption regardless. No Tested for Quality and Potency Patient Out-of-Pocket Cost* Room Temperature $0 Formulation ET-400 Solution Compounded Suspension Suspension Shaking Required Yes FDA-Approved Yes No Yes No Varies (typically $40-80 per month) Storage Condition Varies (often refrigerated) Stability 2 Years Varies (typically 15-60 days) FDA guidelines do not allow for compounding of products that are “essentially a copy of one or more approved drugs.” 34% of compounded product tested by FDA failed potency and/or quality testing2 31% of compounded products failed potency testing. “All of the sampled products that failed potency analyses had subpotent results, indicating that the products analyzed contained less of the active ingredient(s) than expected (as described in the product’s label)” 2 *Eton to provide $0 co-pay for all patients allowed. Compounded suspensions are not typically covered by insurance Source: http s://www.fda.gov/drugs/human-drug-compounding/report-limited-fda-survey-compounded-drug-products
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ET-400 full steam ahead – Q2 PDUFA date and launch in sight • PDUFA date: May 28, 2025 • Eton IR responses completed; no deficiencies or requests outstanding • Launch product quantities on hand; commercial readiness on track • Robust omnichannel marketing launch plan effective immediately post-approval • Commercial launch planned within 1 week of PDUFA • Remain highly confident sales of Alkindi Sprinkle and ET-400 will exceed $50 million annually
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Galzin
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Galzin represented a compelling acquisition opportunity to Eton Need for Improved Patient Experience Product has lacked patient support services for access and affordability Low Awareness and Education Lack of promotion has led to low appreciation of Galzin’s advantages vs chelators and unapproved OTC Complex Barrier to Entry Due to site of action in the intestine, blood levels are not indicative of zinc bioavailability Underdiagnosed Condition Diagnosis rates expected to increase with greater prevalence of genetic testing and newborn screening Reinvestment Opportunity Opportunity for re-investment in Wilson disease to bring innovation (ET-700) Wilson disease is a rare genetic disorder causing excessive copper accumulation, leading to hepatic and neurological symptoms
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Galzin’s target market opportunity is multiples of the product’s current usage Est. 10,000 US Wilson disease Patients (1 in 30,000 incidence) ~2,000 Diagnosed and Actively Treated ~800 on Zinc Therapy ~200-300 Galzin® Galzin has demonstrated superior efficacy, but lack of promotion, access challenges, high co-pays, and a supply shortage drove majority of zinc patients to use OTC products rather than FDA-approved Galzin ® Note: Patient totals estimated based on literature, company estimates, IQVIA data, and physician surveys
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2025 commercial strategy tackles key patient and provider needs, while pipeline focus is to enhance therapy experience. ACCESS Historic Status 42% of patients have difficulty finding drug at local pharmacy 48% have trouble getting their medication approved by insurance Eton Solution Eton Cares Patient Support for Galzin® $0 co-pay Direct overnight shipments Prior authorization support Free drug for all uninsured or under-insured patients, including MedicareAFFORDABILITY 40% of patients pay >$50/month (16% pay >$200/month) DOSING BURDEN 3x/day dosing, requires fasting 1-2 hours prior to each dose 48% of patients miss a dose at least 1x/week ET-700 extended release to provide Less frequent dosing Less GI impact SIDE EFFECTS High rate of GI discomfort and discontinuation ET -700 Source: “Adherence with medical management in patients with Wilson disease”. University of Miami and Wilson Disease Association patient survey. https://wilsondisease.org/wp-content/uploads/2021/11/WD-Adherence-WDA-2020.pdf
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10,000 Estimated US patient population $100M+ Estimated peak sales potential 2027 Estimated NDA submission ET-700 • Zinc extended release developed specifically for Wilson disease patients • Reduces dosing burden (current products are 3x daily) ; potential to reduce GI side effects • Patent filed; FDA meeting expected in Q2 2025 • Potential NDA filing in 2027 • Clear path to $100+ million of peak sales after approval (500-1,000+ patients) Milestone Status Formulation √ Patent Filing √ FDA Meeting Q2 2025 Manufacturing Production 2025 Clinical Study 2026 NDA Submission 2027 FDA-Approved Wilson Disease Products Product Estimated Annual Cost* Syprine $310,498 Cuvrior $292,803 Cuprimine $382,359 Galzin $4,139 * Annual WAC prices. Assumes 1,000mg daily dose for Syprine and Cuprimine, 1,200mg for Cuvrior, and 150mg for Galzin
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Galzin Brand Strategy Ensure smooth transition of existing patients on Galzin Accelerate Galzin adoption through awareness and education Increase WD diagnosis rates through targeted initiatives HCPs to become aware about Galzin acquisition and Eton’s sales force and patient support programs Patients to understand new ways to access Galzin and Eton Cares offerings Wilson Disease Association and COEs to collaborate with Eton on ensuring continuity of care for Galzin patients HCPs and patients to differentiate Galzin from OTC supplements HCPs to prescribe Galzin for all WD patients on zinc therapy Patients to broaden their WD knowledge and advocate for access to Galzin Initiating partnerships to expand genetic / biomarker testing Leveraging AI-driven predictive tools and EHR campaigns to support patient discovery Educating targeted general physician community on WD and early symptom patterns to increase early diagnosis Unlocking growth acceleration – increase therapy adoption and break access barriers Galzin relaunched under Eton’s label March 3rd Expect nearly all patient conversions to occur by Q3
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Exclusive Specialty Pharmacy • $0 copay for commercial patients • Enhanced patient assistance program for uninsured or underinsured patients, including Medicare • Quick start program to start patients in as little as 24 hours • Insurance specialists/pharmacists to assist patients Existing pharmacies continue to dispense until inventory depletion – expect full patient transition over 3-6 months Galzin acquisition to launch in record time: a playbook in agile commercialization Drive Galzin HCP penetration and enhance access Accelerate awareness and adoption through omnichannel strategic efforts Strategic Sales Force Deployment • 5 Rare Disease Specialists • 267 HCP targets including COEs, current prescribers, potential writers with high Wilson disease patient population • Inside clinical sales specialist targeting additional 300 HCPs • Eton and Optime Care press releases • HCP email campaigns – Feb and Mar • New HCP and patient websites • HCP and patient educational resources • Wilson Disease Association outreach on Day 1 • First key liver conference presence in March • KOL engagement and advisory board DAY 1 LAUNCH IMPERATIVES
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Metabolic Products
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Metabolic portfolio remains a steady source of cash flow, enabling strategic acquisitions that will drive Eton’s future growth Continued financial contribution • Indication – Hyperammonemia • US patient population - ~100 • Lower cost alternative to very expensive branded therapy • High-touch Eton Cares patient support HCP interest growing for Golike with metabolic sales force Unified call points, driving incremental revenue Betaine Anhydrous Nitisinone Capsules • Indication – Phenylketonuria (PKU) • US patient population - ~8000 • Proprietary technology offering taste and efficacy advantages; new clinical data and TYR line extension • Indication – Homocystinuria | Tyrosinemia type 1 • US patient population – 1000-2000 | 200-300 • Serve as strategic door opener to metabolic institutions
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ET-600
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3,000 Estimated US patient population $20-50M Estimated peak sales potential 2025 Estimated NDA submission ET-600 (desmopressin oral solution) • Positive bioequivalence (BE) study results announced March 14th • Under development for diabetes insipidus; Patent issued, expires in 2044 • Currently no FDA-approved desmopressin oral liquid formulations • NDA submission expected in April 2025 • Synergistic pediatric endocrinology call point • Pre-launch commercial readiness activities on track for Q1 launch
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Amglidia
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300 Estimated US patient population $10-30M Estimated peak sales potential 2026 Estimated NDA submission Amglidia® (glyburide oral suspension) • Under development for neonatal diabetes mellitus • Synergistic pediatric endocrinology call point • Approved in EU since 2018 • No FDA-approved oral treatments for neonatal diabetes • Granted Orphan Drug Designation by the FDA • FDA meeting scheduled for April 2025 • No milestone owed by Eton until successful FDA meeting
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Hydrocortisone Injection
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Two pipeline hydrocortisone products poised to unlock a $200M opportunity CURRENT STANDARD OF CARE RETAIL MARKET HOSPITAL MARKET Solu-Cortef® 5.8 million vials per year Lyophilized (dry powder) vial that must be reconstituted prior to administration 225,000 Vials per year Primarily used by parents, caretakers, and patients as an emergency rescue treatment for adrenal crisis ZENEO 5.5 M Vials per year Used widely in the hospital setting across a variety of indications including allergic reactions, asthma, inflammation, pain, rheumatoid arthritis ET-800 Source: market size based on IQVIA data NEW
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225,000 Current market size in units $50-100M Estimated peak sales potential 2027 Estimated NDA submission Zeneo® (hydrocortisone autoinjector) • Life changing impact to AI patients • Easy-to-use, two-step needle-free autoinjector device with 24 issued patents • Innovative ready-to-use liquid formulation removes need for reconstitution • Complexity with drug/device combination and change of CMO has led to delayed development • $100M+ opportunity at current 225,00 annual retail units Source: market size based on IQVIA data
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5,500,000 vials $102M Current market size $50-75M Estimated peak sales potential 2027 Estimated NDA submission ET-800 (hydrocortisone vial) • Leverage existing proprietary ZENEO liquid formulation to bring a superior vial product to the hospital market • Innovative ready-to-use formulation removes need for reconstitution • ET-800 would deliver a superior product to the current $102 million (5.5 million vials) hydrocortisone injection market • Patent filed (expected expiry in 2045) Milestone Status Formulation √ Patent Filing √ Pilot Batch Manufacturing √ Registration Batch Manufacturing 2025 Clinical Study 2026 NDA Submission 2027
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Financial Outlook
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Gross Margin – Favorable Tailwinds • Addition of high gross margin products Increlex and Galzin in 2025 • Continued growth of higher margin Alkindi Sprinkle and ET-400 • Smaller share of revenue contribution from lower-margin generic products Product mix shifting favorably; expecting ~70% adjusted gross margin in 2025 and >75% adjusted gross margin by 2028 66.4% 64.2% ~70% >75% 55% 60% 65% 70% 75% 2023 2024 2025 2028 GAAP Non-GAAP Note: Detailed adjustment calculation available in appendix
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Despite major investment in 2025, SG&A expenses are still expected to grow significantly slower than revenue Eton has delivered significant revenue growth with minimal SG&A increase $0 $2 $4 $6 $8 $10 $12 Q1 2022 Q2 2022 Q3 2022 Q4 2022 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Q1 2024 Q2 2024 Q3 2024 Q4 2024 Revenue SG&A 433 % product revenue growth since Q 122 36 % SG&A expense increase since Q 122 Second sales force (Metabolic) Increlex relaunch ET-400 launch Additional corporate headcount to support expanded portfolio Galzin relaunch 2025 SG&A Investments Product Revenue in $ millions
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Acceleration of revenue growth in 2025 Revenue ramping throughout 2025: • U.S. Increlex’s full post-transition revenue benefit starting in Q2 • Galzin post-transition revenue benefit starting in Q3 • ET-400 revenue expected to start in Q3 2021 2022 2023 2024 Exit runrate for 2025 Expect to exit 2025 at approximately a $80 million revenue run rate
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Conclusion
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Conclusion • The past six months have been truly transformational, laying a strong foundation for accelerated growth with a clear path and strategic competencies in place to reach $100M in the near term • Q4 2024 marked a record high – 16th consecutive quarter of growth, up 59% from Q4 2023 • Increlex brings strong strategic alignment, with multiple growth levers to accelerate adoption and expansion— already showing powerful early signals within the first 90 days of launch • Eton’s dual-product franchise strategy in Adrenal Insufficiency with Alkindi Sprinkle/ET-400 is expected to deliver peak sales of more than $50MM • Galzin unlocks a vast opportunity beyond current usage, with a two -pronged strategy driving near-term adoption via commercial efforts while the pipeline accelerates potential growth by enhancing therapy experience • ET-600 is a strong near-term pipeline candidate with synergistic commercial fit and positive clinical results; targeting estimated NDA submission in Q2 2025 • Amglidia aligns with the existing commercial strategy to drive incremental revenue growth, while two hydrocortisone pipeline candidates have the potential to unlock $150M across hospital and retail markets, complementing the AI portfolio • Driving significant revenue growth with minimal SG&A increase, fueled by higher -margin products, and are on track to exit 2025 at ~$80M annual revenue run rate
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Q&A
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Appendix
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Experienced Leadership Team Proven track record of successfully developing and commercializing products at industry leading companies Sean Brynjelsen Chief Executive Officer & Director James Gruber, CPA Chief Financial Officer David Krempa Chief Business Officer Ipek Erdogan-Trinkaus Chief Commercial Officer Scott Grossenbach Sr. Vice President, Sales Operations Danka Radosavljevic Sr. Vice President, Quality and Operations
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Non-GAAP Gross Profit Adjustments 2023 2024 Revenue (GAAP) $31,642 $39,011 Adjustments: Licensing Revenue -- -$500 Sale of Royalty Interests -$5,500 -- Product Sales Revenue $26,142 $38,511 Cost of Goods Sold (GAAP) $10,580 $15,600 Adjustments: COGS associated with licensing revenue -- -$270 One-time milestone payments -$1,000 -- Acquisition-related inventory step up charges -- -$429 Intangible amortization expense -$790 -$1,096 Adjusted Cost of Goods Sold (Non-GAAP) $8,790 $13,805 Adjusted Gross Profit (Non-GAAP) $17,352 $24,706 Adjusted Gross Margin (Non-GAAP) 66.4% 64.2%
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2024 Full Year Results REVENUE 2023 31.6 2024 39.0 Growth 23% GROSS PROFIT 21.1 23.4 11% R&D EXPENSES 3.3 3.3 -2% SG&A EXPENSES 18.9 22.8 20% OPERATING INCOME -1.2 -2.6 N/A NET INCOME -0.9 -3.8 N/A Year-over-year growth in revenue and gross profit fuels momentum for new product launches in 2025 $ Millions