Eton Pharmaceuticals, Inc. · NASDAQ:ETON

Eton Pharmaceuticals — Piper Sandler 37th Annual Healthcare Conference 2025

31 slides from Eton Pharmaceuticals's conference presentation, Dec 4, 2025

  1. Slide 1: Piper Sandler 37th Annual Healthcare Conference

    elon PHARMACEUTICALS Piper Sandler 37'* Annual Healthcare Conference December 2025

  2. Slide 2: Safe Harbor

    Safe Harbor Special note regarding forward-looking statements wa “pte” the "Company "wes" and our), The words “bee "may “estate "ortiuearespale tend” “coul "woul "projet" "epee our fate nae and operating rests: ‘etemngadcto pnel cneiten 1osscestay deep sn esata our poke and “Sr yee FDn opener pro ca

  3. Slide 3: Our Vision

    Our Vision To build a leading ultra-rare disease company with a relentless commitment to patients and innovative therapies. Go The Greatest Number of Patients First Ultra-Rare Focus New Therapies Achievable Clearing the path for Making the greatest Rapidly growing our better i therapy experience the

  4. Slide 4: Experienced Leadership Team

    Experienced Leadership Team Baxter 1}! Horizon Giseiy David Krempa Ipok Erdogan-Trinkaus Danka Radosavijevic Gsacenr — Elarvbot —-Stolmar

  5. Slide 5: Built one of the broadest ultra rare disease portfolios in the industry

    Built one of the broadest ultra rare disease portfolios in the industry EAB ALKINDI SPRINKLE? - Pediatric Adrenal Insufficiency EM KHINDIVI™- Pediatric Adrenal Insufficiency n efon

  6. Slide 6: Pillar Growth Strategy

    3 Pillar Growth Strategy Eton is well positioned for long-term growth w a Organic Growth of Internal Pipeline External Acquisitions Existing Products Products And Licensing Track Record Track Record Track Record revenue growth internally bal INCRELEX, GALZIN, Carglumic ‘Aci and ALKIND| SPRINKLE Outl

  7. Slide 7: Eton's commercial strategy is designed to effectively meet the distinct needs of ultra-rare patient populations

    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 Mean

  8. Slide 8: Track record of commercial execution and revenue growth

    Track record of commercial execution and revenue growth Exceeded $80 million revenue run rate in Q3 2025 Clear path to $100M+ Product Revenue in milions g $80 ‘of near-term revenue with currently $70 approved products. $60 $50 $30 $20 s10 $3 p 11 — 202 2022

  9. Slide 9: Strong Outlook: potential to generate >$500M in combined peak sales

    Strong Outlook: Current portfolio and pipeline has the potential to generate >$500M in combined peak sales Zl Increlex U.S. Label Harmonization KHINDIVI Indication Age Expansion & wd Other inctelex 9%. /khindvi Galziry Ceramic Metabolic (cmon ope / eames Acid Products Peak Net Sales Potential $300M+

  10. Slide 10: Financial Outlook

    Financial Outlook elon PHARMACEUTICALS,

  11. Slide 11: Eton has delivered significant revenue growth with minimal SG&A increase

    Eton has delivered significant revenue growth with minimal SG&A increase e expected to g} Despite major investment in 2 cantly slower than revenue SGRA expen: >900% produet revenue ‘growth since Q1 2022 2025 SG&A Investments, ‘expected to be flat or beter inarial ‘Additional corporate headcount to ‘

  12. Slide 12: Gross Margin — Favorable Tailwinds

    Gross Margin — Favorable Tailwinds Product mix shifting favorably; expecting ~70% adjusted gross margin in Q4 2025 and >75% adjusted gross margin by 2028 275% 70% + Addition of high gross margin products 66.4% INCRELEX and GALZIN in 2025 65% 42% Continued growth of higher margin ALKINDI SPRINKLE and

  13. Slide 13: Near-Term Milestones

    Near-Term Milestones ET-600 PDUFA Date (Feb 25", 2026) Potential initiation of Increlex label harmonization study (FDA meeting Dec 2025, planned study initiation in H1 2026) KHINDIVI label expansion submission (anticipated for Q2 2026) ET-700 pilot study (planned initiation Q1 2026, results expected

  14. Slide 14: Appendix

    Appendix PHARMACEUTICALS,

  15. Slide 15: INCRELEX

    INCRELEX PHARMACEUTICALS,

  16. Slide 16: INCRELEX brings strong strategic alignment, with multiple growth levers

    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 h

  17. Slide 17: Harmonizing US definition of SPIGFD to match EU would expand the market opportunity

    “increlex: fom 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 ~-196S08 (25 percentile) Us (FDA) @ europe (ema) £ < Height Index Standard Deviation Score (SOS) of =-3 a

  18. Slide 18: INCRELEX poised for significant US revenue growth

    & ‘increlex’ INCRELEX poised for significant US revenue growth, with or without label harmonization Potential market opportunity $250M+ with harmonized label (1000pavets) Estimated market opportunity at full penetration (200 patients) 3 2025 Estimated forward run-rate 2023 US Revenue

  19. Slide 19: Adrenal Insufficiency Franchise

    Adrenal Insufficiency Franchise (ALKINDI SPRINKLE + KHINDIVI) elon PHARMACEUTICALS,

  20. Slide 20: Dual product strategy allows patients to choose their preferred product

    Dual product strategy allows the ~10,000 pediatric adrenal insufficiency patients to choose their preferred product Mei Khindivt Sprinkle (hydrocortisone) (hydrocortisone) oral granules " mg/ml oral solution srenocotcaineutfiiency ‘Commercial Launch: November 2020 Commercial Launch: June 2025 ~ Avoi

  21. Slide 21: KHINDIVI fills a critical gap for an FDA-approved therapy

    KHINDIVI fills a critical gap for an FDA-approved therapy that offers simplicity and accuracy ALKINDI SPRINKLE Oral Suspension How do you give your Keg. From a compound pharmecy child their daily dose of sprinde hydrocortisone?” pane 9 50% of patients report using a compounded or a homemade liquid.

  22. Slide 22: FDA guidelines prohibit compounded suspensions, but KHINDIVI's advantages expected to drive adoption

    FDA guidelines prohibit compounded suspensions, but KHINDIVI's advantages are expected to drive adoption regardless of enforcement FDA guidelines do not allow for Formulation more approved drugs.” ‘Shaking Required 134% of compounded product tested by FOA atled potency and/or quality Tested for Qual

  23. Slide 23: GALZIN

    GALZIN PHARMACEUTICALS,

  24. Slide 24: GALZIN represented a compelling acquisition opportunity to Eton

    ; a Galzin GALZIN represented a compelling acquisition Lriseel ca opportunity to Eton Wilson disease is a rare genetic disorder causing excessive copper accumulation, leading to hepatic and neurological symptoms Need for Improved Patient Experience Prodthslockes patent suport secs for szess and ated

  25. Slide 25: GALZIN's target market opportunity is multiples of the product's current usage

    GALZIN’s target market opportunity is multiples of the product's current usage Est. 10,000 US Wi e Patients (1 in tne Galzin (cine acetate) capsules GALZIN has demonstrated superior efficacy, but lack of promotion, access challenges, high co-pays, and a supply shortage drove majority of zinc patient

  26. Slide 26: 2025 commercial strategy tackles key patient and provider needs

    2025 commercial strategy tackles key patient and provider needs, while pipeline focus is to enhance therapy experience. Historic Status Eton Solution Se a ACCESS. Klas ceclitaed Patient Support for GALZIN oo coronene alzin ¥ Prior authorization support (inc acetate) capsules AFFORDABILITY (16% pay >

  27. Slide 27: ET-700

    ET-700 1d specifically for patients + Patent filed; FDA meeting held Q2 2025 study + Potential NDA filing in 2027 Clear path to $100+ million of peak sales after approval (500-1,000+ patients) Reduces dosing burden (current products are 3x daily) ; potential to reduce GI side effects. Proof concept

  28. Slide 28: ET-600 (desmopressin oral solution)

    ET-600 (desmopressin oral solution) @@ Under development for diabetes insipidus; Patent issued, expires in 2044 3,000 Positive bioequivalence (BE) study results announced March 14th Estimated US NDA accepted for review, assigned February 25, 2026 PDUFA date —__ Currently no FDA-approved desmopressin

  29. Slide 29: Amglidia (glyburide oral suspension)

    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 300 Estimated US patient population 2026 Es

  30. Slide 30: Two pipeline hydrocortisone products poised to unlock a $200M opportunity

    Two pipeline hydrocortisone products poised to unlock a $200M opportunity trergeney rescue treatment or etenal cis + Two-tep needles autcnjector wih 24 issued Patents andinnovaivereeaytosse gu formulation + Estimated 2027 NDA submision + $100 million opportunity + Cumbersome: must Solu-Cortef® © be

  31. Slide 31: Non-GAAP Gross Profit Adjustments

    Non-GAAP Gross Profit Adjustments 2023 2024 Revenue (GAAP) sue 339017 haute: Licensing Revenue - $300 [ae of Royal Interests $5300 - $26,142 sos WBostof Goods Sid (GAAP) 370580 315600 Pastimes: foocs associated with icensing revenue - 3270 fonetime milestone payments -s1900 - hauistion‘elated ventor

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