Egetis Therapeutics Ab · OMXSTO:EGTX

Egetis Therapeutics Ab — Investor Presentation (March 2025)

95 slides from Egetis Therapeutics Ab's investor presentation, March 2025

Slide 1: Corporate presentation March 2025
WE CARE FOR THE RARE Corporate presentation March 2025 An integrated orphan drug company, focusing on late-stage development for commercialization ©2025 Egetis Therapeutics. All rights reserved. 1
Slide 2: Disclaimer
Disclaimer THIS PRESENTATION AND ITS CONTENTS ARE NOT FOR RELEASE, PUBLICATION OR DISTRIBUTION, IN WHOLE OR IN PART, DIRECTLY OR INDIRECTLY, IN OR INTO OR FROM THE UNITED STATES OF AMERICA, CANADA, AUSTRALIA, JAPAN OR ANY JURISDICTION WHERE SUCH DISTRIBUTION IS UNLAWFUL. IMPORTANT: You must read the
Slide 3: Content
Content 1. An integrated orphan drug company, focusing on late-stage development for commercialization 2. Emcitate® - MCT8 deficiency a. Overview of MCT8-deficiency b. Clinical experience with Emcitate c. Regulatory pathways in EU and US d. Commercial opportunity e. Partnerships 3. Emcitate® - Poten
Slide 4: An integrated orphan drug company, focusing on late-stage development for commercialization
WE CARE FOR THE RARE 1. An integrated orphan drug company, focusing on late-stage development for commercialization
Slide 5: 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 6: An integrated orphan drug company, focusing on late-stage development for commercialization
An integrated orphan drug company, focusing on late-stage development for commercialization Dedicated orphan drug company 1 Two late-stage assets: Emcitate and Aladote* 2 Emcitate approved in EU in February 2025 for MCT8 deficiency Pivotal trial for Emcitate NDA is ongoing 3 Highly attractive orphan
Slide 7: 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 8: Pipeline overview
Pipeline overview Emcitate – European Commission approval Feb 13, 2025 Candidate Preclinical Phase I Phase II/III MAA/NDA Comments Emcitate EU Approved - EC approval received Feb 13, 2025 MCT8 deficiency Filed Oct 2023 - First launch in EU planned in Q2 2025 Emcitate US ReTRIACt - Ongoing, pivotal,
Slide 9: Emcitate® Overview
Emcitate® Overview Lead candidate for addressing MCT8 deficiency, a condition with high unmet medical need and no available treatment • Triac Trial I (Phase IIb) completed with significant & clinically relevant effects on T3 levels and chronic thyrotoxicosis • Erasmus Medical Center cohort study con
Slide 10: Several important milestones for Egetis 2024-2025 YTD
Several important milestones for Egetis 2024-2025 YTD Jul Aug Aug Sep Sep Dec Feb 2024 2024 2024 2024 2024 2024 2025 ETA guidelines Emcitate Post-hoc Patent Secured SEK Positive CHMP European recommend all associated with analysis application to 300m Directed opinion Commission full patients with su
Slide 11: 2.a Overview of MCT8 deficiency
2.a Overview of MCT8 deficiency 11
Slide 12: MCT8 deficient patients
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 13: 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 14: Natural history study revealed poor survival with a high prevalence of treatable underlying risk factors
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 15: 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 16: 2.b Clinical experience with Emcitate
2.b Clinical experience with Emcitate
Slide 17: Orphan drug candidate
Orphan drug candidate 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 thyroid hormo
Slide 19: 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 20: 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 21: 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 2
Slide 22: Long-term efficacy and safety of Emcitate® in MCT8 deficiency patients
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 deficiency ‒ Median baseline
Slide 23: 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 ©2025 Egetis Therapeutics. All rights reserved.
Slide 24: 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 ©2025 Egetis Therapeutics. All rights reserved. 24 © 2024 Egetis Therapeutics. All rights reserved.
Slide 25: 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 26: 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 27: 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 28: Tiratricol (Emcitate®) treatment is associated with survival benefits
Tiratricol (Emcitate®) treatment in patients with MCT8 deficiency is associated with survival benefits 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
Slide 29: Post-hoc analysis reports effects of tiratricol on patient-centered outcome measures
Post-hoc analysis reports effects of tiratricol on patient-centered outcome measures in patients with MCT8 deficiency Jul Aug Aug Sep Sep Dec Feb ETA guidelines Emcitate associated with Post-hoc analysis Patent application to the Secured SEK 300m Positive CHMP opinion for Europen Commission full rec
Slide 30: 2.c Emcitate® - regulatory pathways in EU and US
2.c Emcitate® - regulatory pathways in EU and US
Slide 31: 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) Fast Fast track designation (FDA) track Rare pediatric disease designation (FDA) PRV Eligibility: Priority review voucher upon approval* MAA MAA: EU
Slide 32: EU Commission approves Emcitate® as the first and only treatment for patients with MCT8 deficiency
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 33: Emcitate regulatory pathway in EU & US
Emcitate regulatory pathway in EU & US Robust data set in an ultra rare genetic disease EMC cohort Triac Trial I Natural history Triac Trial II Survival study ReTRIACt Trial study N=46 N=67 N=151 N=22 N~228 N=16 • Completed 2018 • Completed 2021 • Retrospective data, • Open-label, • Retrospective co
Slide 34: ReTRIACt: withdrawal of Emcitate in males with MCT8 Deficiency
ReTRIACt: withdrawal of Emcitate in males with MCT8 Deficiency Pivotal randomized placebo-controlled trial for NDA submission • Proportion of participants who meet the rescue criterion (serum total T3 > ULN) Primary during the 30-day double-blind Randomized Treatment Period endpoint • Change in card
Slide 35: Design of the ReTRIACt clinical trial
Design of the ReTRIACt clinical trial Requested by the FDA • A 30-day, randomized placebo-controlled withdrawal study in 16 patients Primary endpoint Patients already on therapy n=8 • Design agreed with FDA tiratricol tiratricol tiratricol • The study allows for inclusion of patients that are alread
Slide 36: Current status of ReTRIACt trial (as of Feb 26, 2025)
Current status of ReTRIACt trial (as of Feb 26, 2025) Map not to scale Addenbrooke’s Hospital CHOP Cambridge, UK Philadelphia Erasmus Medical Center Rotterdam, NL New sites opened in 2024 • 19 patients have been included so far, of which 11 patients have completed the randomized phase and 3 patients
Slide 37: Upcoming value enhancing key milestones 2025-2026
Upcoming value enhancing key milestones 2025-2026 Emcitate® 2025-2026 • EU launch, in the first country, Germany, • Japan – Development plan agreed with PMDA MCT8 during the second quarter of 2025 • US Patent granted - Processes and compounds deficiency • Topline results ReTRIACt for US NDA • US app
Slide 38: 2.d Emcitate® - Commercial opportunity
2.d Emcitate® - Commercial opportunity
Slide 39: 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 40: Emcitate supplied globally in managed access programs
Emcitate supplied globally in managed access programs Managed access programs confirm the significant unmet medical need in MCT8 deficiency and the view on how Emcitate addresses it • Managed access programs MEDICINES AGENCY ‒ mechanisms to allow early access to a medicine prior to regulatory market
Slide 41: 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 42: Preparing for Emcitate launch by Egetis and partners
Preparing for Emcitate 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 (> 70% of sales for most ultra-orphans) Optimizing additional Japan license deal with cou
Slide 43: Step-wise building team to execute on key activities at the right time for launch success
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 44: 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 45: 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 46: Deliver solid Emcitate clinical and economic value proposition to enable reimbursement & broad access
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 47: European Thyroid Association (ETA) recommends tiratricol as long-term therapy
European Thyroid Association (ETA) recommends tiratricol as long-term therapy for all 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 r
Slide 48: Phased EU launch: Germany first
Phased EU launch: Germany first Pricing & Reimbursement (P&R) strategy execution in 2 waves, starting with EU4 EC Decision Timelines P&R process for Wave 1 similar ultra-orphans Initiated right after pending EC decision Germany: 1 year, but immediate Germany, France, Spain & Italy funded access in p
Slide 49: 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 • Support clinical studies and basi
Slide 50: Our Expanded Access Program is a vital step on our path to commercialization in the US
Our Expanded Access Program is a vital step on our path to commercialization in the US Tiratricol (Emcitate) Expanded Access Program sites A significant asset to both the patients and Egetis launch readiness • Provided early and sustainable access to therapy • Expose physicians to Emcitate prior to
Slide 51: Disease awareness activities in the US are bearing fruit
Disease awareness activities in the US are bearing fruit Known confirmed 2024 >100 diagnosed MCT8 deficiency 2023 patients in the US 2022 Accelerate patient finding Naive or treated under early access efforts by integrating advanced data-driven insights into our existing Hospitals with initiatives k
Slide 52: Balancing Annual Treatment Costs and Broad Access
Balancing Annual Treatment Costs and Broad Access Analogues Access Product Disease Estimated annual Less restrictive treatment cost (WAC) • Prior Authorization to label Skyclarys® Friedreich ataxia • Genetic Test Attestation/documentation ~$400K Small molecule • Specialist prescribing Procysbi® Neph
Slide 53: 2.e Emcitate partnerships
2.e Emcitate partnerships
Slide 54: Advancing rest of world with 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 55: The MENAT-region
The MENAT-region Opportunity for patient access based on EMA approval in the Middle-East, North-Africa and Turkey • MENAT-region has a large population with well established healthcare systems • EMA approval allows for access in some of the countries without the need for national regulatory submissi
Slide 56: Egetis’ approach to the MENAT-region
Egetis’ approach to the MENAT-region Serving patients in the MENAT-region by working together with local partners • Given that Europe and the US are the priorities for Egetis together with the need for local resources in the MENAT-region, Egetis is currently identifying strategic partners for collab
Slide 57: Potential for indication expansion into RTH-beta
3. Potential for indication expansion into RTH-beta
Slide 58: Resistance to Thyroid Hormone type Beta (RTH-β)
Resistance to Thyroid Hormone type Beta (RTH-β) Potential indication expansion for Emcitate into non-overlapping patient population Characteristics of RTH-β Emcitate as potential treatment for RTH-β • Caused by mutations in thyroid hormone receptor • Emcitate efficacious in restoring signaling in ma
Slide 59: “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
Slide 60: Increased Mortality RTHβ
Increased Mortality RTH𝛽 2.84 3.49 6.35 Welsh cohort 55 patients RTH Beta 2750 Age and sex matched controls Median age 1st event 56 vs 67 ©2025 Egetis Therapeutics. All rights reserved. 60 Okosieme, Lancet Diabetes Endocrinology 2023. PMID 37475119
Slide 61: 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 ©2025 Egetis
Slide 62: Financials
4. Financials
Slide 63: Egetis secured long-term financing of SEK 462m and added top-tier US specialist investor as largest shareholder
Egetis secured long-term financing of SEK 462m and added top-tier US specialist investor as largest shareholder Announcement published on October 10, 2023 • Unique combined long-term financing, comprising SEK 172m private placement at a premium and SEK 290m debt financing ‒ First in its class in a S
Slide 64: Egetis carried out directed share issuances amounting to SEK 300 million
Egetis carried out directed share issuances amounting to SEK 300 million (USD 30 million) Announcement published on September 30, 2024 PRESS • Led by Frazier Life Sciences with a USD 10 million investment. • Supported by international and Swedish specialist healthcare funds. • Subscription price at
Slide 65: FDA granted Rare Pediatric Disease designation to Emcitate®
FDA granted Rare Pediatric Disease designation to Emcitate® US Rare Pediatric Disease Priority Review Voucher (PRV) provides a ~$100m opportunity Overview of PRV Examples of PRVs sold Seller Buyer Value Year • The FDA grants Rare Pediatric Disease designation (RPD) to therapies for serious or life-t
Slide 66: Share Register, Cash and Market Cap
Share Register, Cash and Market Cap • Cash position December 31, 2024: SEK 351 million • Number of outstanding shares: 359,238,126 • Market Cap: ~SEK 1.6 billion* • Listing venue: Nasdaq Stockholm, Main Market • Ticker: EGTX Source: Monitor by Modular Finance. Compiled and processed data from variou
Slide 67: Egetis submits patent application to the USPTO
Egetis submits patent application to the USPTO 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 patients survival benefits reports effects of US PTO - “
Slide 68: Summary
5. Summary
Slide 69: Egetis – a de-risked biotech with substantial unlocked potential
Egetis – a de-risked biotech with substantial unlocked potential • Late stage biotech “under the radar”, developing the first therapy for a devastating genetic disorder ‒ Strong team with established track record in the orphan drug space, including SOBI, Alexion, Biomarin, Biogen, Vertex, Sarepta, S
Slide 70: Upcoming value enhancing key milestones 2025-2026
Upcoming value enhancing key milestones 2025-2026 Emcitate® 2025-2026 • EU launch, in the first country, Germany, • Japan – Development plan agreed with PMDA MCT8 during the second quarter of 2025 • US Patent granted - Processes and compounds deficiency • Topline results ReTRIACt for US NDA • US app
Slide 71: An integrated orphan drug company, focusing on late-stage development for commercialization
An integrated orphan drug company, focusing on late-stage development for commercialization Dedicated orphan drug company 1 Two late-stage assets: Emcitate and Aladote* 2 Emcitate approved in EU in February 2025 for MCT8 deficiency Pivotal trial for Emcitate NDA is ongoing 3 Highly attractive orphan
Slide 72: Appendix
6. Appendix
Slide 73: 6.a The attractiveness of the orphan drug segment
6.a The attractiveness of the orphan drug segment
Slide 74: 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 75: 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. 75 ©2025 Egetis Therapeutics. All rights reserved.
Slide 76: 6.b Leadership Team and Board of Directors
6.b Leadership Team and Board of Directors
Slide 77: Leadership team with global experience & proven track record
Leadership team with global experience & proven track record Nicklas Westerholm Desiree Luthman Katayoun Welin-Berger, PhD CEO VP Regulatory Affairs VP Technical Operations • Joined 2017 • Joined 2023 • Joined 2023 • AstraZeneca 1995-2017 • Global regulatory professional, >25y experience • VP Operat
Slide 78: Board of directors
Board of directors Mats Blom Thomas Lönngren Chair of the board since 2024 Board member since 2021 • Shares in Egetis: 3,134,762 • Shares in Egetis: 283,158 • BA, Business Administration & Economics, Lund University; • MSc in social and regulatory pharmacy and a degree in MBA, IESE University of Nav
Slide 79: Termination of discussions regarding a potential acquisition of the Company
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 80: 6.c Paracetamol/Acetaminophen overdose and clinical experience with Aladote
6.c 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 81: 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 82: Paracetamol/acetaminophen 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 83: 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 84: 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 85: 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 86: Aladote® demonstrates consistent results of reduced liver injury as measured by exploratory biomarkers
Aladote® demonstrates consistent results of reduced liver injury as measured by exploratory biomarkers K18K18 ccK18 ccK18 miR-122 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
Slide 87: 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 88: 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 89: 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 90: 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 91: Aladote® - Commercial opportunity
3. Aladote® - Commercial opportunity
Slide 92: 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 93: 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 94: 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

Egetis Therapeutics — Investor Presentation (March 2025) on 6ix

Egetis Therapeutics Ab — Investor Presentation (March 2025)

Slide 1: Corporate presentation March 2025

Slide 2: Disclaimer

Slide 3: Content

Slide 4: An integrated orphan drug company, focusing on late-stage development for commercialization

Slide 5: Orphan drug segment – a highly attractive opportunity

Slide 6: An integrated orphan drug company, focusing on late-stage development for commercialization

Slide 7: Building a sustainable orphan drug company

Slide 8: Pipeline overview

Slide 9: Emcitate® Overview

Slide 10: Several important milestones for Egetis 2024-2025 YTD

Slide 11: 2.a Overview of MCT8 deficiency

Slide 12: MCT8 deficient patients

Slide 13: MCT8 deficiency: a detrimental condition with significant unmet medical need

Slide 14: Natural history study revealed poor survival with a high prevalence of treatable underlying risk factors

Slide 15: Multiple sources lead to consistent MCT8 deficiency incidence estimates

Slide 16: 2.b Clinical experience with Emcitate

Slide 17: Orphan drug candidate

Slide 18: Discovery of Emcitate (Triac, tiratricol)

Slide 19: Overview of completed Phase IIb – Triac Trial I

Slide 20: Consistent, clinically relevant and highly significant results

Slide 21: Triac Trial I: Indication of positive effect on neurocognitive development

Slide 22: Long-term efficacy and safety of Emcitate® in MCT8 deficiency patients

Slide 23: New patient cohort of equal size to the Triac Trial I

Slide 24: New cohort confirms primary endpoint results in Triac Trial I

Slide 25: Consistent, clinically relevant and highly significant results across endpoints

Slide 26: Triac Trial II objective and design

Slide 27: Triac Trial II Summary

Slide 28: Tiratricol (Emcitate®) treatment is associated with survival benefits

Slide 29: Post-hoc analysis reports effects of tiratricol on patient-centered outcome measures

Slide 30: 2.c Emcitate® - regulatory pathways in EU and US

Slide 31: Regulatory features of Emcitate for MCT8 deficiency

Slide 32: EU Commission approves Emcitate® as the first and only treatment for patients with MCT8 deficiency

Slide 33: Emcitate regulatory pathway in EU & US

Slide 34: ReTRIACt: withdrawal of Emcitate in males with MCT8 Deficiency

Slide 35: Design of the ReTRIACt clinical trial

Slide 36: Current status of ReTRIACt trial (as of Feb 26, 2025)

Slide 37: Upcoming value enhancing key milestones 2025-2026

Slide 38: 2.d Emcitate® - Commercial opportunity

Slide 39: Emcitate®– alleviating patient and societal burden

Slide 40: Emcitate supplied globally in managed access programs

Slide 41: Commercialization possible with lean & agile team

Slide 42: Preparing for Emcitate launch by Egetis and partners

Slide 43: Step-wise building team to execute on key activities at the right time for launch success

Slide 44: Focusing on Critical Areas for Launch Success

Slide 45: Expanding disease awareness momentum

Slide 46: Deliver solid Emcitate clinical and economic value proposition to enable reimbursement & broad access

Slide 47: European Thyroid Association (ETA) recommends tiratricol as long-term therapy

Slide 48: Phased EU launch: Germany first

Slide 49: Germany Launch Strategy

Slide 50: Our Expanded Access Program is a vital step on our path to commercialization in the US

Slide 51: Disease awareness activities in the US are bearing fruit

Slide 52: Balancing Annual Treatment Costs and Broad Access

Slide 53: 2.e Emcitate partnerships

Slide 54: Advancing rest of world with license agreement with Fujimoto for Emcitate in Japan

Slide 55: The MENAT-region

Slide 56: Egetis’ approach to the MENAT-region

Slide 57: Potential for indication expansion into RTH-beta

Slide 58: Resistance to Thyroid Hormone type Beta (RTH-β)

Slide 59: “The Feedback Loop” in RTH

Slide 60: Increased Mortality RTHβ

Slide 61: Thyroid Hormone Concentration on Triac Treatment

Slide 62: Financials

Slide 63: Egetis secured long-term financing of SEK 462m and added top-tier US specialist investor as largest shareholder

Slide 64: Egetis carried out directed share issuances amounting to SEK 300 million

Slide 65: FDA granted Rare Pediatric Disease designation to Emcitate®

Slide 66: Share Register, Cash and Market Cap

Slide 67: Egetis submits patent application to the USPTO

Slide 68: Summary

Slide 69: Egetis – a de-risked biotech with substantial unlocked potential

Slide 70: Upcoming value enhancing key milestones 2025-2026

Slide 71: An integrated orphan drug company, focusing on late-stage development for commercialization

Slide 72: Appendix

Slide 73: 6.a The attractiveness of the orphan drug segment

Slide 74: Orphan drug segment – a highly attractive opportunity

Slide 75: CAGR estimates of total pharmaceutical market vs orphan

Slide 76: 6.b Leadership Team and Board of Directors

Slide 77: Leadership team with global experience & proven track record

Slide 78: Board of directors

Slide 79: Termination of discussions regarding a potential acquisition of the Company