Egetis Therapeutics Ab · OMXSTO:EGTX
Egetis Therapeutics Ab — Investor Presentation (August 2026)
102 slides from Egetis Therapeutics Ab's investor presentation, August 2026
Slide 1: We Care for the Rare: Corporate presentation
WE CARE FOR THE RARE Corporate presentation August 2026 Emcitate® (tiratricol) launched in Germany in May 2025 FDA PDUFA date September 28, 2026 For investors and analysts only ©2026 Egetis Therapeutics. All rights reserved. 1
Slide 2: Disclaimer
Disclaimer Tiratricol is an investigational drug and has not been approved by the U.S. Food and Drug Administration (FDA). Its safety and efficacy have not been established in the U.S. THIS PRESENTATION AND ITS CONTENTS ARE NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, IN WHOLE OR IN PART, DIRECTLY
Slide 3: An integrated orphan drug company
WE CARE FOR THE RARE 1. An integrated orphan drug company, focusing on late-stage development and commercialization
Slide 4: Egetis: building an orphan drug commercial stage company
Egetis: building an orphan drug commercial stage company Focus on Emcitate® (tiratricol) for the treatment of MCT8 deficiency 1 Supplied to over 230 patients in over 25 countries including US, EU Strong regulatory status The first and only approved drug for the treatment of MCT8 deficiency BTD Break
Slide 5: Strong Execution in 2025-26 Positions Egetis for Emcitate U.S. Launch
Strong Execution in 2025-26 Positions Egetis for Emcitate U.S. Launch in 2026 ✓ Japan – Development plan alignment with PMDA EC approves Emcitate ✓ Completion and Acceptance Expected launch of as first and only FDA Breakthrough Positive Phase 3 ReTRIACt withdrawal of NDA Emcitate in the U.S. treatme
Slide 6: MCT8 deficiency results in dysfunctional thyroid hormone trafficking
MCT8 deficiency results in dysfunctional thyroid hormone trafficking Median life expectancy of 35 years with 30% of patients dying in childhood MCT8 deficiency key features Estimated incidence: 1 per 70k male births Median onset of symptoms: 4 months Median age of diagnosis: 10 months Median life ex
Slide 7: Emcitate (tiratricol) mechanism of action
Emcitate® (tiratricol) mechanism of action with clear scientific and mechanistic rationale Cell membrane • Tiratricol is a small molecule, thyroid hormone receptor agonist Tiratricol in action Tiratricol • Unlike T3, tiratricol can cross cellular membranes without a TR functional MCT8 transporter T3
Slide 8: Tiratricol treatment associated with survival benefits
Tiratricol treatment in patients with MCT8 deficiency has been shown to be associated with survival benefits In the Triac Trial I, tiratricol reached target level serum T3 & improvements Tiratricol has been shown to be in clinically relevant outcome measures associated with a 3x lower risk of mortal
Slide 9: Emcitate Approved in EU and Launched in Germany; NDA accepted
Emcitate® (tiratricol) Approved in EU and Launched in Germany NDA accepted March 27, 2026; Priority Review; PDUFA Sep 28, 2026 Robust dataset in an ultra-rare genetic disease; all FDA-required studies completed EMC cohort U.S. Expanded Triac Trial I Triac Trial II Survival study ReTRIACt study Acces
Slide 10: Tiratricol's Effect on T3 Lowering May Explain Mortality Benefit
Tiratricol’s Effect on T3 Lowering May Explain Mortality Benefit Emcitate Source for evidence of (tiratricol) efficacy T3 Triac Trial I, Triac Trial II, ReTRIACt, EMC Cohort Study Triac Trial I, Immune Body weight HR BP Arrhythmia EMC Cohort system Study RPP Triac Trial I, ReTRIACt Infection Sudden
Slide 11: Emcitate launch by Egetis and partners
Emcitate® (tiratricol) launch by Egetis and partners Executing the US & European market preparations and launches through the Egetis team To optimize the launch, we will focus our own resources on US and Europe Optimizing additional Japan license deal with countries through partners Fujimoto Türkiye
Slide 12: ETA Recommends Emcitate as Long-Term Therapy
European Thyroid Association (ETA) Recommends Emcitate (tiratricol) as Long-Term Therapy for MCT8 Deficiency ETA recommends the use of tiratricol as long-term therapy for all patients with MCT8 deficiency, and for certain patients with RTHβ Inaugural 2024 Guidelines were commissioned by the Executiv
Slide 13: A phased EU launch through in-house commercial organization
A phased EU launch through in-house commercial organization started in Germany in May 2025 Launch execution in 2 waves, starting with EU4 Deliver the Emcitate clinical and economic value proposition in P&R processes, outlining: Wave 1 • MCT8 deficiency and its rarity Germany, France, Italy, Spain &
Slide 14: Europe & International
Europe & International Q2 revenue MSEK 17.4, +23% (CER) vs Q2 2025 and +30% vs Q1 2026; momentum building on German reimbursement conclusion and broader geographic reach Germany • Price negotiations with GKV-Spitzenverband concluded in Q2 2026 • Continued disease awareness, physician engagement and
Slide 15: U.S. Launch Readiness: Building Momentum Toward Launch
U.S. Launch Readiness: Building Momentum Toward Launch The Team is built and launch infrastructure is being activated Patient support program Market access Patient support capabilities and operating model Payer strategy, value communication, and account established coverage in place Experienced Team
Slide 16: U.S. Launch Readiness: Continuity of Care for EAP Patients
U.S. Launch Readiness: Continuity of Care for EAP Patients is Our Top Priority Tiratricol EAP continuity of care — ensuring Patient-level transition planning uninterrupted treatment Confirm the pathway and treating physician for each EAP patient at approval Enrollment & prescriber readiness A coordi
Slide 17: US: Annual Treatment Costs and Strength of Evidence
US: Annual Treatment Costs and Strength of Evidence Representative analogues Product Disease Estimated avg. annual treatment cost (WAC) Impact of strength of evidence on price Oxlumo® Primary ~$623K Biologic hyperoxaluria type 1 • Morbidity-driven disease burden, supported by Hypophosphatasia surviv
Slide 18: Resistance to Thyroid Hormone Type Beta (RTHβ)
Resistance to Thyroid Hormone Type Beta (RTHβ) Potential indication expansion for Emcitate into larger non-overlapping patient population Characteristics of RTHβ Emcitate as potential treatment for RTH-β • Caused by mutations in thyroid hormone receptor beta (TRβ)1 • Emcitate efficacious in restorin
Slide 19: Strong financial foundation for strategic execution
Strong financial foundation for strategic execution Solid cash position Directed share issue April 2026 of SEK 350m (USD 38m) • Cash position June 30, 2026: SEK 378 million • Oversubscribed with participation from new & existing investors • Number of outstanding shares: 464,947,224 • Market Cap: ~SE
Slide 20: Financial Overview – Second Quarter 2026
Financial Overview – Second Quarter 2026 • Total revenue ‒ Q2-2026 of 17.4 MSEK vs. 14.5 MSEK for Q2-2025, +23% YoY CER. • Cost of goods sold impacted by intangible R&D depreciation ‒ During Q2-2026 R&D depreciation of 10.1 MSEK is included in COGS ‒ Excluding the depreciation Gross profit would hav
Slide 21: Strong Execution in 2025-26 Positions Egetis for Emcitate U.S. Launch
Strong Execution in 2025-26 Positions Egetis for Emcitate U.S. Launch in 2026 ✓ Japan – Development plan alignment with PMDA EC approves Emcitate ✓ Completion and Acceptance Expected launch of as first and only FDA Breakthrough Positive Phase 3 ReTRIACt withdrawal of NDA Emcitate in the U.S. treatme
Slide 22: Pipeline Overview
Pipeline Overview Emcitate® (tiratricol) – Launched in Germany May 2025; U.S. PDUFA date September 28, 2026 Candidate Preclinical Phase I Phase II / III MAA / NDA Comments Emcitate EU EC approval received Feb 12, 2025 Launched MCT8 deficiency Launched in Germany May 1, 2025 NDA accepted Mar 27, 2026
Slide 23: Building a sustainable orphan drug company
Building a sustainable orphan drug company • Successfully develop Emcitate for EU & US approvals in 2025/26 and potentially Aladote post 2026 • Commercialize Emcitate and Aladote through an inhouse organization in Europe/ North America and partnerships in RoW • Realize the full potential of our prod
Slide 24: Appendix 1: Overview of MCT8 deficiency
Appendix 1 Overview of MCT8 deficiency 24
Slide 25: MCT8 deficiency results in dysfunctional thyroid hormone trafficking
MCT8 deficiency results in dysfunctional thyroid hormone trafficking MCT8 deficiency has two co-manifestations MCT8 deficiency results in simultaneous too high and too low New Research Sheds Light on Thyroid Hormone Transport thyroid hormone levels – causing system wide issues • In 2003, MCT8 was id
Slide 26: MCT8 deficiency: a detrimental condition with significant unmet medical need
MCT8 deficiency: a detrimental condition with significant unmet medical need What is MCT8 deficiency? What does it mean? What are the challenges? How do you manage the Quick facts from natural history2 disease? • Genetic X-linked disorder ● Non-functional MCT8 protein: ● Patients appear normal at bi
Slide 27: Natural history study revealed poor survival
Natural history study revealed poor survival with a high prevalence of treatable underlying risk factors An international, retrospective, multicentre cohort study from 2014-2020 in 151 patients • 151 patients were enrolled with 73 different MCT8 (SLC16A2) mutations • Median age at diagnosis was 24.0
Slide 28: Multiple sources lead to consistent MCT8 deficiency incidence estimates
Multiple sources lead to consistent MCT8 deficiency incidence estimates Available Data Leads to Consistent Supporting our Relevant Sources & Data MCT8 Deficiency Incidence Estimates Conservative Estimate • Multiple cohorts of patients with X- linked mental retardation under study Visser et al., Clin
Slide 29: Appendix 2: Emcitate mode of action and clinical experience
Appendix 2 Emcitate (tiratricol): Mode of action and clinical experience
Slide 30: Emcitate mechanism of action
Emcitate mechanism of action with clear scientific and mechanistic rationale and established safety profile Difference normal MCT8 and deficiency of MCT8 Emcitate (tiratricol) – Addressing MCT8 deficiency • Thyroid hormone T3 requires transporters such as MCT8 • Tiratricol is a small molecule thyroi
Slide 31: Discovery of Emcitate (Triac, tiratricol)
Discovery of Emcitate (Triac, tiratricol) T3 Triac (tiratricol) 31 © 2026 Egetis Therapeutics. All rights reserved.
Slide 32: Overview of completed Phase IIb – Triac Trial I
Overview of completed Phase IIb – Triac Trial I • Evaluate the efficacy and safety of oral administration of tiratricol in male patients with MCT8 deficiency of all ages Primary objective and • Highly significant primary outcome - Change in T3 serum concentrations results • Safe and tolerable • Resu
Slide 33: Consistent, clinically relevant and highly significant results
Consistent, clinically relevant and highly significant results Triac Trial I: Reached target level serum T3 & improvements in clinically relevant outcome measures 10 T0 (Baseline) 8 T12 (12 month) p < 0.0001 Serum T3 (nmol/l) 6 4 2 Normal range 0 Endpoints Baseline mean (± SD) 12 months mean (± SD)
Slide 34: Triac Trial I: Indication of positive effect on neurocognitive development
Triac Trial I: Indication of positive effect on neurocognitive development Triac Trial II did not meet its primary endpoints 40 T0 (Baseline) T12 (12 months) 30 GMFM score (%)1) 20 10 0 # # # Patients 1.5 to <4 years 4 to <10 years ≥10 years Age at baseline Source: Groeneweg et al; Lancet D&E 2019 3
Slide 35: Real-world evidence: Long-term efficacy and safety of Emcitate
Real-world evidence: Long-term efficacy and safety of Emcitate® in MCT8 deficiency patients Published in October, 2021 • Investigator-initiated real-world cohort study at 33 sites conducted by the Erasmus Medical Center • Investigated efficacy and safety of Emcitate in 67 patients with MCT8 deficien
Slide 36: New patient cohort of equal size to the Triac Trial I
New patient cohort of equal size to the Triac Trial I Long term follow up, up to >6 years Triac Trial I N=46 Triac Trial I patients New patients Available for long-term From managed access program monitoring N=40 N=27 EMC cohort Published in JCEM N=67 36 © 2026 Egetis Therapeutics. All rights reserv
Slide 37: New cohort confirms primary endpoint results in Triac Trial I
New cohort confirms primary endpoint results in Triac Trial I Fast and durable normalization of T3 values in almost all patients p<0.0001 37 © 2026 Egetis Therapeutics. All rights reserved.
Slide 38: Consistent, clinically relevant and highly significant results across endpoints
Consistent, clinically relevant and highly significant results across endpoints • Data confirm the positive results from previous study, Triac Trial I • Normalization of serum T3 corresponds to improvement in thyroid hormone status in end target tissues • Beneficial effects are maintained or continu
Slide 39: Triac Trial II objective and design
Triac Trial II objective and design: Triac Trial II was designed to investigate a potential additional benefit on neurocognitive development in 22 patients with MCT8 deficiency below 30 months of age treated with Emcitate® (tiratricol) during 96 weeks • Confirm findings from Triac Trial I in younges
Slide 40: ReTRIACt Trial Designed to Support NDA Submission
ReTRIACt Trial Designed to Support NDA Submission Aligned with FDA to Include ReTRIACt Data as Complementary and Not Pivotal • A 30-day, randomized placebo-controlled Patients already on therapy withdrawal study in 15 patients tiratricol tiratricol tiratricol • The study allowed inclusion of patient
Slide 41: ReTRIACt study: Results on primary endpoints
ReTRIACt study: Results on primary endpoints Largest increase R R Patients ordered by change in serum T3 (nmol/L) R during randomized treatment period* R Placebo range [0.49 : 2.08] No change Tiratricol range D [-0.24 : 0.40] Largest decrease * = change from baseline to rescue or end of randomized t
Slide 42: Triac Trial II Summary
Triac Trial II Summary • Triac Trial II results: • The numerical improvements versus baseline observed on the primary endpoints of neurocognitive development assessed by the GMFM-88 and BSID-III scales did not show a statistically significant improvement versus historical controls. • The trial confi
Slide 43: Overview of changes in clinical variables during Triac Trial I
Overview of changes in clinical variables during Triac Trial I 43 T3 within normal range T3 outside normal range © 2026 Egetis Therapeutics. All rights reserved.
Slide 44: Real-world evidence: Treatment associated with survival benefits
Real-world evidence: Tiratricol (Emcitate®) treatment in patients with MCT8 deficiency is associated with survival benefits PRESS • Abstract published ahead of the ETA/ITC Annual Meeting report that treatment with tiratricol (Emcitate®) in patients with MCT8 deficiency is associated with a 3x lower
Slide 45: Post-hoc analysis on patient-centered outcome measures
Post-hoc analysis reports effects of tiratricol on patient-centered outcome measures in patients with MCT8 deficiency • According to the Abstract, there were PRESS improvements upon tiratricol treatment reported by caregivers related to improved interaction (22/39), greater alertness (19/39), improv
Slide 46: Appendix 3: Emcitate regulatory pathways in EU and US
Appendix 3 Emcitate® - regulatory pathways in EU and US
Slide 47: Regulatory features of Emcitate for MCT8 deficiency
Regulatory features of Emcitate for MCT8 deficiency Orphan drug designation for MCT8 deficiency ODD Eligibility: Market exclusivity 10y (EU) & 7y (US) BTD Breakthrough Therapy Designation (FDA) Rare pediatric disease designation (FDA) PRV Eligibility: Priority review voucher upon approval* MAA: EU f
Slide 48: EU Commission approves Emcitate as first and only treatment
EU Commission approves Emcitate® as the first and only treatment for patients with MCT8 deficiency Jul Aug Aug Sep Sep Dec Feb ETA guidelines Emcitate associated with New post-hoc analysis Patent application to the Secured SEK 300m Positive CHMP opinion for Europen Commission full recommend all pati
Slide 49: Emcitate/tiratricol regulatory pathway in EU/US
Emcitate/tiratricol regulatory pathway in EU/US Robust data set in an ultra rare genetic disease EMC cohort Triac Trial I Natural history Triac Trial II EMC survival study ReTRIACt study N=46 N=67 N=151 N=22 N>600 N=15 • Completed 2018 • Completed 2021 • Retrospective data, • Open-label, • Basis for
Slide 50: Value enhancing key milestones 2025-2026
Value enhancing key milestones 2025-2026 Emcitate® 2025-2026 ✓ EU launch, in the first country, Germany, ✓ Completion of US NDA submission MCT8 May 1, 2025 ✓ Japan – Development plan agreed with PMDA deficiency ✓ Türkiye partnership signed with Er-Kim ✓ US Patent – Granted (composition) ✓ Break Thro
Slide 51: Appendix 4: Emcitate Commercial opportunity
Appendix 4 Emcitate® - Commercial opportunity
Slide 52: Emcitate alleviating patient and societal burden
Emcitate®– alleviating patient and societal burden Aiming to provide value for both patients and society MCT8 deficiency is a detrimental condition with significant unmet medical need and no approved therapy • Median life-expectancy of MCT8 patients is 35 years1 Patients • Patients underweight for a
Slide 53: Tiratricol supplied globally in managed access programs
Tiratricol supplied globally in managed access programs Managed access programs confirm the significant unmet medical need in MCT8 deficiency • Managed access programs MEDICINES AGENCY ‒ mechanisms to allow early access to a medicine prior to regulatory marketing approval ‒ granted to pharmaceutical
Slide 54: Commercialization possible with lean & agile team
Commercialization possible with lean & agile team Unique setting for Emcitate in MCT8 deficiency Seizing opportunity for cost-effective value creation Addressing unmet medical need • Targeted stakeholder interactions • Efficiency gains through global-country team coordination No competition External
Slide 55: Emcitate launch by Egetis and partners
Emcitate® (tiratricol) launch by Egetis and partners Executing the US & European market preparations and launches through the Egetis team To optimize the launch, we will focus our own resources on US and Europe Optimizing additional Japan license deal with countries through partners Fujimoto Türkiye
Slide 56: Step-wise building team to execute on key activities
Step-wise building team to execute on key activities at the right time for launch success Key projects driven by recognized industry talents recruited to the Egetis Commercial & Medical Affairs Team – Core team brings launch skills and best practices from in total 150+ years at international compani
Slide 57: Focusing on Critical Areas for Launch Success
Focusing on Critical Areas for Launch Success Aiming to Improve the Lives of MCT8 Deficiency Patients and their Caregivers IDENTIFY PATIENTS ENSURE ACCESS Boost disease awareness, educate on Preparing for broad access to Emcitate as disease*, diagnosis and newborn soon as possible after marketing sc
Slide 58: Expanding disease awareness momentum
Expanding disease awareness momentum Amplified by External Efforts Constructive dialogues at Scientific community Great work ongoing by several scientific congresses generating more data patient advocacy groups Example from Annual Meeting of the European Thyroid Association Van der Most, F. et al. T
Slide 59: Deliver solid Emcitate clinical and economic value proposition
Deliver solid Emcitate clinical and economic value proposition to enable reimbursement & broad access Key for payer assessments to describe burden of disease, unmet need & benefit of treatment High burden of MCT8 Significant unmet medical Emcitate benefit validated by deficiency need physicians and
Slide 60: ETA Recommends Emcitate as Long-Term Therapy
European Thyroid Association (ETA) Recommends Emcitate (tiratricol) as Long-Term Therapy for MCT8 Deficiency ETA recommends the use of tiratricol as long-term therapy for all patients with MCT8 deficiency, and for certain patients with RTHβ Inaugural 2024 Guidelines were commissioned by the Executiv
Slide 61: A phased EU launch starting in Germany
A phased EU launch through in-house commercial organization starting in Germany May 1 Pricing & Reimbursement (P&R) strategy execution in 2 waves, starting with EU4 Deliver the Emcitate clinical and economic value proposition in P&R processes, outlining: Wave 1 • MCT8 deficiency and its rarity Franc
Slide 62: Germany: Benefit assessment and price negotiations
Germany: Benefit assessment and price negotiations for new drugs follow a strict and transparent process AMNOG Process is well-defined and led by G-BA for benefit assessment and by GKV for price negotiations 4 parties involved in AMNOG process: For orphan drugs: population size and annual therapy co
Slide 63: Germany Launch Strategy
Germany Launch Strategy Building strong Expert base to advance management of MCT8 deficiency MCT8 deficiency Experts • Engage experts in increasing disease awareness in Germany • Advance collaborative efforts on monitoring and treatment guidance of MCT8 deficiency • Advocate for importance of local
Slide 64: United States: Egetis Priority Market
United States: Egetis Priority Market From Regulatory Milestone to Commercial Momentum Driving Patient Identification Growth • Deep engagement in key referral centers • Strong KOL and advocacy alignment • Growing identified patient pool (>100) Expanded Access Strengthening Launch Readiness • 17 acti
Slide 65: US: Annual Treatment Costs and Strength of Evidence
US: Annual Treatment Costs and Strength of Evidence Representative analogues Product Disease Estimated avg. annual treatment cost (WAC) Impact of strength of evidence on price Oxlumo® Primary ~$623K Biologic hyperoxaluria type 1 • Morbidity-driven disease burden, supported by Hypophosphatasia surviv
Slide 66: Appendix 5: Emcitate partnerships
Appendix 5 Emcitate partnerships
Slide 67: License agreement with Fujimoto for Emcitate in Japan
Advancing rest of world with license agreement with Fujimoto for Emcitate in Japan • Highly suitable partner in Fujimoto PRESS ‒ Private company in Osaka, Japan, founded in 1933 ‒ Significant experience from successfully registering and launching medicines for Blood, Neurological and Orphan diseases
Slide 68: Emcitate launch by Egetis and partners
Emcitate® (tiratricol) launch by Egetis and partners Executing the US & European market preparations and launches through the Egetis team To optimize the launch, we will focus our own resources on US and Europe Optimizing additional Japan license deal with countries through partners Fujimoto Türkiye
Slide 69: Appendix 6: Potential for indication expansion into RTH-beta
Appendix 6 Potential for indication expansion into RTH-beta
Slide 70: Resistance to Thyroid Hormone Type Beta (RTH-β)
Resistance to Thyroid Hormone Type Beta (RTH-β) Potential indication expansion for Emcitate into larger non-overlapping patient population Characteristics of RTH-β Emcitate as potential treatment for RTH-β • Caused by mutations in thyroid hormone receptor beta (TRβ)1 • Reduced target tissue response
Slide 71: The Feedback Loop in RTH
“The Feedback Loop” in RTH Thyroid Stimulating Hormone (TSH) - + Thyroid Hormones T4 and T3 Example levels Normal Levels TSH NORMAL RANGE 4.0 0.27-4.2 T4 HIGH 45 12-22 T3 HIGH 22 3.1-6.8 71 © 2026 Egetis Therapeutics. All rights reserved.
Slide 72: Increased Mortality RTHβ
Increased Mortality RTH𝛽 2.84 3.49 6.35 Welsh cohort 55 patients RTH Beta 2750 Age and sex matched controls Okosieme, Lancet Diabetes Endocrinology 2023. PMID 37475119 Median age 1st event 56 vs 67 © 2026 Egetis Therapeutics. All rights reserved. 72
Slide 73: Thyroid Hormone Concentration on Triac Treatment
Thyroid Hormone Concentration on Triac Treatment fT4T4 Baseline to Nadir TT3 Baseline to Nadir fT4 Free Concentration Total T3 Concentration 60 A1 (LCMS) A1 A2 A2 3 50 A3 A3 A4 A4 A5 A5 40 A6 A6 TT3 (nmol/L) fT4 (pmol/L) A7 2 A7 30 A8 A8 20 1 10 0 0 Baseline 1 Nadir 2 Baseline 1 Nadir2 73 © 2026 Ege
Slide 74: Appendix 7: Financials
Appendix 7 Financials
Slide 75: Strong financial foundation for strategic execution
Strong financial foundation for strategic execution Solid cash position Directed share issue April 2026 of SEK 350m (USD 38m) • Cash position June 30, 2026: SEK 378 million • Oversubscribed with participation from new & existing investors • Number of outstanding shares: 464,947,224 • Market Cap: ~SE
Slide 76: Financial Overview – Second Quarter
Financial Overview – Second Quarter • Total revenue ‒ Q2-2026 of 17.4 MSEK vs. 14.5 MSEK for Q2-2025, +23% YoY CER. • Cost of goods sold impacted by intangible R&D depreciation ‒ During Q2-2026 R&D depreciation of 10.1 MSEK is included in COGS ‒ Excluding the depreciation Gross profit would have bee
Slide 77: FDA granted Rare Pediatric Disease designation to tiratricol
FDA granted Rare Pediatric Disease designation to tiratricol US Rare Pediatric Disease Priority Review Voucher (PRV) provides a ~$200m opportunity Overview of PRV Examples of PRVs sold • The FDA grants Rare Pediatric Disease designation (RPD) Seller Buyer Value Year to therapies for serious or life-
Slide 78: Patent Granted for MCT8 Deficiency Composition in the U.S.
Egetis Announces Grant of Patent for MCT8 Deficiency Composition in the U.S. • USPTO granted patent PRESS • Provides protection for a novel composition containing tiratricol as active ingredient • Claims cover a method of treating MCT8 deficiency, including dose regiments • Orange Book-listable with
Slide 79: Appendix 8: The attractiveness of the orphan drug segment
Appendix 8 The attractiveness of the orphan drug segment
Slide 80: Orphan drug segment – a highly attractive opportunity
Orphan drug segment – a highly attractive opportunity Shorter clinical development time1 Higher attainable prices2 Orphan drugs attractive returns5 Phase II to launch Average # of years Mean cost per patient and year (USDk) 143,4 152,2 150,9 128,1 134,5 ORPHAN 3.8 years 1.7x 10,3% 31,2 32,4 33,7 NON
Slide 81: Orphan drug segment – a highly attractive opportunity
Orphan drug segment – a highly attractive opportunity Orphan drug designation is awarded to • Less extensive clinical trials 1 products targeting limited disease populations1 Development • Agile and faster development process • Lower costs • Lower development risk 2 More than 7,000 known rare diseas
Slide 82: CAGR estimates of total pharmaceutical market vs orphan
CAGR estimates of total pharmaceutical market vs orphan The global orphan or rare disease market size was valued at an estimated USD 140 – 150 bn and is expected to grow at 10-14% CAGR over the coming five years. Source: EvaluatePharma, Pareto. 82 © 2026 Egetis Therapeutics. All rights reserved.
Slide 83: Appendix 10: Leadership Team and Board of Directors
Appendix 10 Leadership Team and Board of Directors
Slide 84: Leadership team with global experience & proven track record
Leadership team with global experience & proven track record Nicklas Westerholm Tiago Nunes, MD, PhD Katayoun Welin-Berger, PhD CEO Chief Medical Officer VP Technical Operations • Joined 2017 • Joined 2026 • Joined 2023 • AstraZeneca 1995-2017 • SVP, Clinical Development, Mirum Pharmaceuticals • VP
Slide 85: Board of directors
Board of directors Mats Blom Margarida Duarte Chair of the board since 2024 Board member since 2025 • Shares in Egetis: 3,499,762 • Shares in Egetis: 0 • BA, Business Administration & Economics, Lund University; • BS in Pharmaceutical Sciences & Executive Master’s degree MBA, IESE University of Nava
Slide 86: Termination of discussions regarding a potential acquisition
Termination of discussions regarding a potential acquisition of the Company Announcement published on May 23, 2023 • Discussions, triggered by an unsolicited approach by an external party, have taken place between certain external parties and Egetis regarding a potential acquisition of the Company •
Slide 87: Appendix 11: Paracetamol overdose and clinical experience with Aladote
Appendix 11 Paracetamol/Acetaminophen overdose and clinical experience with Aladote * In-house development of Aladote has been parked until Emcitate MCT8 deficiency submissions have been completed
Slide 88: Aladote – To prevent acute liver injury caused by paracetamol poisoning
Aladote® – To prevent acute liver injury caused by paracetamol poisoning* • Paracetamol poisoning is one of the most common overdoses with >175,000 hospital admissions globally per annum • No adequate treatment exists for increased risk patients • Orphan drug designation (ODD) granted in the US & EU
Slide 89: Paracetamol poisoning – no adequate treatment for increased-risk patients
Paracetamol/acetaminophen poisoning – no adequate treatment for increased-risk patients What is • Minimum toxic dose of paracetamol/acetaminophen in adults is only 7.5g paracetamol/ acetaminophen • Risk factors include malnutrition, alcoholism and consumption of other medications poisoning? • Parace
Slide 90: Orphan drug candidate
Orphan drug candidate with clear scientific and mechanistic rationale Early presenters (<8h) Late presenters (>8h) are at increased-risk for liver injury NAC treatment effective against liver injury NAC treatment + Aladote® to prevent liver injury • Liver glutathione (GSH) replenished by NAC, toxic
Slide 91: Overview of completed Phase Ib/IIa
Overview of completed Phase Ib/IIa • Met primary endpoint of safety tolerability in the combination of Aladote® and NAC Primary • Results presented at the 58th Annual Meeting of the Society of Toxicology, EASL objective and ILC in April, Vienna and published in Lancet’s journal EBioMedicine in 2019
Slide 92: Positive proof-of-principle Phase Ib/IIa results
Positive proof-of-principle Phase Ib/IIa results Indicates that Aladote may reduce liver injury Safety & tolerability Liver injury – ALT1 pre-defined secondary outcome NAC + 2 NAC + 5 NAC + 10 NAC + 2 NAC + 5 NAC + 10 % of patients needing mol/kg mol/kg mol/kg mol/kg mol/kg mol/kg additional N
Slide 93: Aladote demonstrates reduced liver injury by exploratory biomarkers
Aladote® demonstrates consistent results of reduced liver injury as measured by exploratory biomarkers K18 ccK18 miR-122 10 10 10 Relative change from baseline Relative change from baseline Relative change from baseline NAC alone NAC alone NAC alone NAC and 2umol/kg NAC and 2umol/kg NAC and 2umol/kg
Slide 94: Aladote - Regulatory pathway to submissions in EU and US
3. Aladote® - Regulatory pathway to submissions in EU and US* * In-house development of Aladote has been parked until Emcitate MCT8 deficiency submissions have been completed
Slide 95: ALBATROSS: Phase IIb/III study for US/EU regulatory submission
ALBATROSS: Phase IIb/III study for US/EU regulatory submission* • Patients who have overdosed on paracetamol with increased Patient risk of liver damage due to late arrival at hospital (> 8h) who population need treatment with NAC NAC regimen • Approved 21 hours NAC regimen Treatment • 4 groups in c
Slide 96: ALBATROSS: Aladote Phase IIb/III study design
ALBATROSS: Aladote Phase IIb/III study design Seamless Phase IIb/III design Based on the acetaminophen/paracetamol levels eligible patients will be randomised in a 1:1:1:1 ratio to one of the 4 treatment arms in combination with NAC: 35 35 55 140 pts interim 250 pts 35 analysis 55 35 • Stop the stud
Slide 97: Aladote clinical development timelines
Aladote clinical development timelines Orphan Drug Designation EU • Interim analysis CTA pivotal • Recruitment completed Phase IIb/III study and topline results 2022 tbc tbc tbc • Start pivotal Phase • Regulatory submissions IIb/III study (after Europe/US Emcitate submissions have been completed) •
Slide 98: Aladote - Commercial opportunity
3. Aladote® - Commercial opportunity
Slide 99: Aladote – alleviating patient and societal burden
Aladote– alleviating patient and societal burden Aiming to provide value for both patients and society POD is a life threatening condition with remaining medical needs • POD (paracetamol/acetaminophen overdose) can lead to acute liver failure, liver transplant or death Patients • In US and UK togeth
Slide 100: Commercialisation of Aladote for high-risk POD patients
Commercialisation of Aladote for high-risk POD patients Very cost-effective since possible to launch through members of Emcitate team Favorable conditions for launch success Addressing life-threatening condition • Anologue antidotes priced at $3.5k – 50k Addressing unmet medical need • National emer
Slide 101: Analogue antidotes priced at $3.5k – 50k
Analogue antidotes priced at $ 3.5k – 50k National emergency hospital stocking guidelines - opportunity for rapid market penetration • Various antidotes, e.g. vs. drug overdosing, metal poisoning, snake bites and reversal of anticoagulant treatment effects • Limit morbidity/mortality when used withi
Slide 102: Thank you!
Thank you! Egetis Therapeutics egetis.com karl.hard@egetis.com 102
Egetis Therapeutics — Investor Presentation (August 2026) on 6ix