Eton Pharmaceuticals, Inc. · NASDAQ:ETON
Eton Pharmaceuticals — Investor Day (March 2025)
57 slides from Eton Pharmaceuticals's investor day presentation, Mar 18, 2025
Slide 1: 2025 Investor Day
elon PHARMACEUTICALS 2025 INVESTOR DAY March 18, 2025
Slide 2: Safe Harbor
Safe Harbor ‘Special note regarding forwarc-looking statements ‘Ths documan contains formardsocing statements concenng Eton Pharmaceutical neo the"Cempany "wa "us anor). The wotds lr” may “wi “potty “tetas coiuaantpte“ntand “cold woul “pet plan “epact and salar expressions that coneayucany ef fre ee
Slide 3: Agenda
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 Candi
Slide 4: Today's Speakers
Today’s Speakers SEAN BRYNJELSEN JAMES GRUBER DAVIDKREMPA _IPEK ERDOGAN-TRINKAUS Chief Executive Officer Chief Financial Officer Chief Business Officer Chief Commercial Officer
Slide 5: Reviewing Eton's Transformational Six Months
Reviewing Eton’s Transformational Six Months elon PHARMACEUTICALS,
Slide 6: 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 Theraples Achieveble Clearing the path for Making the greatest Rapidly growing our better patient access and impa
Slide 7: Transformational Months
Transformational 6 Months Expanded portfolio, delivered record revenue, strong commercial launch momentum Acquired 2 High Added 3 Attractive Value Commercial Pipeline Assets Exceptional Products (Amglidia®, Beaten Across (Increlex®, Galzin®) ET-700, ET-800) ec Ore Reported vcard 30 Acauied atin prod
Slide 8: Three-Pillar Growth Strategy
3 Pillar Growth Strategy Eton was successfully built with a three-pillar strategy and is [ze a nell positioned to sustain the momentum Extemal Acquisitions ‘And Licensing Organic Growth of Internal Pipeline Existing Products Products ‘Track Record Track Record 1G straight quarters of sequential Deve
Slide 9: 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 Me
Slide 10: Built one of the broadest ultra rare disease portfolios in the industry
Built one of the broadest ultra rare disease portfolios in the industry Increlex® - Severe Primary Insulin-Like Growth Factor 1 Deficiency v3 ‘ BAM Nitisinone Hereditary Tyrosinemia Type 1 (HT-1) ) /: WATE £T-700-Wison disease V: WME PEDIATRIC ENDOCRINOLOGY Wm ETABOLIC ADDED IN THE LAST 6 MONTHS isc
Slide 11: Demonstrated strong performance, with a roadmap for the next wave of growth
Demonstrated strong performance, with a roadmap for the next wave of growth $40 $35 & sa Clear path to $100M+ of near-term 925 revenue with the additions of Increlex, Galzin and ET-400. $20 sis S10 $5 $0
Slide 12: Q4 2024 & Full Year 2024 Results
Q4 2024 & Full Year 2024 Results elon PHARMACEUTICALS,
Slide 13: Straight Quarter of Product Sales Growth
16 Straight Quarter of Product Sales Growth +59% Revenue Growth over 04 2023, (24 2020 01 2021 42 2021 032021 042021 01 2022 02 2022 03 2022 042022 01 2023 02 2023 08 2023 04 2028 01 2024 02 2024 032024 4 2024 elon Revenue $mallons
Slide 14: Q4 2024 Financial Results
Q4 2024 Financial Results REVENUE GROSS PROFIT R&D EXPENSES ‘SGBA EXPENSES OPERATING INCOME NET INCOME $7.3 $3.5 $ Millions $11.6 $6.5 59% 78% NA 47% N/A NA sion
Slide 15: Increlex
Increlex PHARMACEUTICALS,
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 due to low insulin ike growth factor 1 (IGF-1) levels despite normal or elevated growth hormon A
Slide 17: Increlex Historic Global Revenue
Increlex Historic Global Revenue Areturn to 185 US active Q would produce estimated US revenue of ~$50 million =e) 3M Ne éfon
Slide 18: Insights from thought leaders shaping Increlex relaunch and expansion
Insights from thought leaders shaping Mattes Increlex relaunch and expansion Held advisory board with 7 US key opinion leaders in pediatric endocrinology in January + Why low penetration? Low awareness among pediatricians resulting in delayed diagnosis Many causes of short stature > IGF-1 deficiency
Slide 19: Increlex Brand Strategy
Increlex Brand Strategy eae Eton’s commercial strategy is poised to drive significant market penetration E Ee chonce ihe nai base Drive patient awareness Establish Eton as the gs ‘and engagement best-in-class partner for SPIGFD Heo aston at tte Esso rice ee pallets fer ‘and support SPIGFD embrace Rx
Slide 20: Building strong momentum in the first days of Increlex relaunch
Building strong momentum in the increlex first 90 days of Increlex relaunch patente and fies ‘tong 6-day patent and demand georaton ¥ 61 patont vane + 81 eetve patients through mid March Increlex Hcp | and adoption through omnichannel enhance | strategic access efforts e ee e Foundation (HEP an MAGI
Slide 21: Harmonizing US definition of SPIGFD to match EU would expand the market
* increlex: 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 1.96 808 @25percentitey © usiroa —- @ evaore ema) = Height index Standard Deviation Seore (SDS) of 3 anes
Slide 22: Increlex US Label Harmonization
e ‘increlex: Increlex US Label Harmonization VISION Harmonize US Increlex label definition of SPIGFD to match EU increlex 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
Slide 23: Increlex poised for significant US revenue growth
* inerelex: Increlex poised for significant US revenue growth, iewles with or without label harmonization i Potential market opportunity seanMae with harmonized label (1000 patients) Estimated market opportunity at full penetration >2.5x GROWTH POTENTIAL (200 patients) REMAINING TO REACH FULL PENETR
Slide 24: Adrenal Insufficiency Franchise Outlook
Adrenal Insufficiency Franchise Outlook elon PHARMACEUTICALS,
Slide 25: Maximizing therapy penetration in AI - a dual product franchise strategy
Maximizing therapy penetration in Al - a dual product franchise strategy to address diverse HCP and patient preferences across the age continuum oy senate . eae . ee i ¥ Aleodit the fst aod only *¥ Unlocks larger sharoof the hyerocontsone teatment ‘2004 marke, offering designed to provide accurate ‘
Slide 26: Alkindi Sprinkle continues growth with strong runway to capture share
Alkindi Sprinkle continues growth with Sa strong runway to capture share Active Paints by Month Proserbing HCPS
Slide 27: ET-400 filling a critical gap for an FDA-approved therapy
ET-400 filling a critical gap for an FDA-approved therapy that offers simplicity and accuracy Alkindi Sprinkle Oral Suspension From compound pharmacy “How do you give your child their daily dose of hydrocortisone?” 50% of patients report usinga compounded or homemade liquid. Solid Tablets (hol, halv
Slide 28: ET-400 vs compounded suspensions
ET-l) 400 Upon ET-400 approval, FDA guidelines will prohibit compounded suspensions. However, ET-400's advantages are expected to drive adoption regardless. FDA guidelines do not allow for compounding of products that Eoemilitlon are “essentially a copy of one or more approved drugs." ‘Shaking Requi
Slide 29: ET-400 full steam ahead - Q2 PDUFA date and launch in sight
ET-400 full steam ahead — BE 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-ap
Slide 30: Galzin
Galzin PHARMACEUTICALS,
Slide 31: Galzin represented a compelling acquisition opportunity to Eton
Galzin represented a compelling acquisition Galzin 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 Prodvet hs eked patient suport sees for acsess and aterdebdny Low
Slide 32: 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 Galzin (ine acetate) capsules 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-ap
Slide 33: 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. 42% of patients have difficulty finding drug at local pharmacy Eton Cares . baal Patient Support for Galzin® an 48%have rouble geting theirmesication so copay Galzin CEE 1S ¥ Direc
Slide 34: ET-700 zinc extended release for Wilson disease
ET-700 + Zinc extended release developed specifically for Wilson disease patients 140,000 + Reduces dosing burden (current products are 3x daily) ; potential to reduce GI side effects canneaue + Patent filed; FDA meeting expected in Q2 2025 patient population + Potential NDA filing in 2027 + Clear p
Slide 35: Galzin Brand Strategy
Galzin Galzin Brand Strategy Sele Unlocking growth acceleration ~ increase therapy adoption and o O eak access barriers Ensure smooth transition of Accelerate Galzin adoption Increase WD diagnosis rates ‘existing patients on Galzin ‘through awareness through targeted initiatives ‘and education Galzi
Slide 36: Galzin acquisition to launch in record time
Galzin acquisition to launch in record time: Seen a playbook in agile commercialization Dayt + ton and opine Cate pets ses LAUNCH IMPERATIVES {Pema cmpalgns ~FeD si cP and pent webskes Accelerate awareness Drive Galzin HoP and adoption Penetration | ote oeh and enhance access omnichannel strategic e
Slide 37: Metabolic Products
Metabolic Products elon PHARMACEUTICALS,
Slide 38: Metabolic portfolio remains a steady source of cash flow
Metabolic portfolio remains a steady source of cash flow, enabling strategic acquisitions that will drive Eton’s future growth = Carglumie Acid Continued HOP interest growing for Golike financial contribution with metabolic sales force + Indication - Hyperammonemia + Indication = Phenyiketonuria (PK
Slide 39: ET-600
ET-600 PHARMACEUTICALS,
Slide 40: ET-600 (desmopressin oral solution)
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 ped
Slide 41: Amglidia
Amglidia PHARMACEUTICALS,
Slide 42: Amglidia (glyburide oral suspension)
Amglidia® (glyburide oral suspension) 300 Estimated US patient population + Under development for neonatal diabetes mellitus + Synergistic pediatric endocrinology call point + Approved in EU since 2018. + No FDA-approved oral treatments for neonatal diabetes 2026 Estimated + Granted Orphan Drug Desi
Slide 43: Hydrocortisone Injection
Hydrocortisone Injection elon PHARMACEUTICALS,
Slide 44: Two pipeline hydrocortisone products poised to unlock a $200M opportunity
Two pipeline hydrocortisone products poised to unlock a $200M opportunity CURRENT STANDARD OF CARE RETAIL MARKET Primarily used by parents, | 225,000. ‘ssi an patents ‘as an emergency rescue Vials per year treatment for adrenal crisis HOSPITAL MARKET Used widely in the hospital 5.5 Meng actors geri
Slide 45: Zeneo (hydrocortisone autoinjector)
Zeneo® (hydrocortisone autoinjector) + Life changing impact to Al 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 t
Slide 46: ET-800 (hydrocortisone vial)
ET-800 (hydrocortisone vial) =z) ( 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
Slide 47: Financial Outlook
Financial Outlook elon PHARMACEUTICALS,
Slide 48: Gross Margin - Favorable Tailwinds
Gross Margin - Favorable Tailwinds Product mix shifting favorably; expecting ~70% adjusted gross margin in 2025 and >75% adjusted gross margin by 2028 275% 75% 70% peas — + Addition of high gross margin products 66.4% Increlex and Galzin in 2025 ox ere + Continued growth of higher margin Alkindi Spr
Slide 49: Eton has delivered significant revenue growth with minimal SG&A increase
Eton has delivered significant revenue growth with minimal SG&A increase estment in 202 Despite major SG&A expenses are still expected to grow significantly slower than revenue 433% product revenue growth ae 2025 SG8A Investments basicdeact Increlex relaunch
Slide 50: Acceleration of revenue growth in 2025
Acceleration of revenue growth in 2025 Expect to exit 2025 at approximately a $80 million revenue run rate Revenue ramping throughout 2025: + US. Increlex's full posttransition revenue benefit starting in Q2 + Galzin post-transition revenue benefit starting in Q3 + £T-400 revenue expected to start i
Slide 51: Conclusion
Conclusion PHARMACEUTICALS,
Slide 52: Conclusion
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 ~ 16" consecutive quarter of growth, up 59% from Q4 2023, + Increl
Slide 53: Q&A
Q&A elon PHARMACEUTICALS
Slide 54: Appendix
Appendix PHARMACEUTICALS,
Slide 55: Management team
paler Leaalensany iam cessfully developin bSse@ James Graber, CPA Dav Krempa Ipek Edogan-Trinkaus } ‘Scott Groseenbach Baxter |! HORIZON A Flavor — Stolmar aa
Slide 56: Non-GAAP Gross Profit Adjustments
Non-GAAP Gross Profit Adjustments Fens (GAAP) sae sn) hadussment: Ieensig Revene $500 Se of Royalty eres $5500 3 Product Sales Revenue 26342 ssn tof ede Sed (GARP) 310580 s15600 Admsiments: 36 ceocatd wih cesing revenue seo etme mfesone payments -sv000 = visto elated avntry stp op charges 309 Jeangb
Slide 57: 2024 Full Year Results
2024 Full Year Results $ Millions Year-overyear growth in revenue and gross profit fuels momentum for new product launches in 2025 R&D EXPENSES 33 33 2% OPERATING INCOME 12 26 N/A NET INCOME 09 38 NA elon