Egetis Therapeutics Ab · OMXSTO:EGTX

Egetis Therapeutics Ab — Investor Day (December 2024)

149 slides from Egetis Therapeutics Ab's investor day presentation, Dec 18, 2024

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 4: Agenda: Egetis Investor Day, December 18, 2024
Agenda: Egetis Investor Day, December 18, 2024 Time (CET/ET) Subject Presenter(s) 15:00/9.00am Welcome & corporate update Nicklas Westerholm, CEO 15:10/9.10am MCT8 deficiency: recent advances with tiratricol Prof. Edward Visser, Erasmus Medical Center, NL 15:35/9.35am Q&A Visser & Westerholm 15:45/9
Slide 5: Several important milestones over the last 6 months
Several important milestones over the last 6 months Jun Jun Jul Aug Sep Sep Nov Dec 2024 2024 2024 2024 2024 2024 2024 2024 Promising Triac Trial II ETA guidelines • Survival Patent Secured SEK Response EMA EMA approval Innovative results diagnosis and abstract/data application to 300m Directed D180
Slide 6: ETA recommends tiratricol as long-term therapy for all patients with MCT8 deficiency
European Thyroid Association (ETA) recommends tiratricol as long-term therapy for all patients with MCT8 deficiency Jun Jun Jul Aug Sep Sep Nov Dec Promising Triac Trial II results ETA guidelines diagnosis • Survival abstract/data Patent application to the Secured SEK 300m Response EMA D180 EMA appr
Slide 7: Tiratricol treatment in MCT8 deficiency is associated with survival benefits
Tiratricol (Emcitate®) treatment in patients with MCT8 deficiency is associated with survival benefits Jun Jun Jul Aug Sep Sep Nov Dec Promising Triac Trial II results ETA guidelines diagnosis • Survival abstract/data Patent application to the Secured SEK 300m Response EMA D180 EMA approval Innovati
Slide 8: Egetis submits patent application to the USPTO
Egetis submits patent application to the USPTO Jun Jun Jul Aug Sep Sep Nov Dec Promising Triac Trial II results ETA guidelines diagnosis • Survival abstract/data Patent application to the Secured SEK 300m Response EMA D180 EMA approval Innovative and management of – ETA US PTO - “Processes of Direct
Slide 9: Egetis carried out directed share issuances amounting to SEK 300 million
Egetis carried out directed share issuances amounting to SEK 300 million (approximately USD 30 million) Jun Jun Jul Aug Sep Sep Nov Dec Promising Triac Trial II results ETA guidelines diagnosis • Survival abstract/data Patent application to the Secured SEK 300m Response EMA D180 EMA approval Innovat
Slide 10: Egetis receives positive CHMP opinion
Egetis receives positive CHMP opinion Jun Jun Jul Aug Sep Sep Nov Dec Promising Triac Trial II results ETA guidelines diagnosis • Survival abstract/data Patent application to the Secured SEK 300m Response EMA D180 EMA approval Innovative and management of – ETA US PTO - “Processes of Directed Share
Slide 11: 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 12: Key upcoming milestones 2025-2026
Key upcoming milestones 2025-2026 Emcitate® 2025-2026 • EU approval and launch • Japan – Development plan agreed with PMDA MCT8 • Topline results ReTRIACt for US NDA • US Patent granted - Processes and compounds deficiency • Filing US NDA – priority review • US approval and launch • Middle East & No
Slide 13: Agenda: Egetis Investor Day, December 18, 2024
Agenda: Egetis Investor Day, December 18, 2024 Time (CET/ET) Subject Presenter(s) 15:00/9.00am Welcome & corporate update Nicklas Westerholm, CEO 15:10/9.10am MCT8 deficiency: recent advances with tiratricol Prof. Edward Visser, Erasmus Medical Center, NL 15:35/9.35am Q&A Visser & Westerholm 15:45/9
Slide 14: MCT8 deficiency: recent advances with tiratricol (Triac)
MCT8 deficiency: recent advances with tiratricol (Triac) Edward Visser Erasmus MC, Rotterdam, The Netherlands
Slide 15: Disclosure
Disclosure Erasmus MC receives royalties and service fees from Egetis Therapeutics (no personal benefits)
Slide 16: Outline
Outline Brief context of thyroid hormone signaling MCT8 deficiency: key features & mechanisms of disease Triac Trial I Real world data QoL, survival data & Triac Trial II
Slide 17: Outline
Outline Brief context of thyroid hormone signaling MCT8 deficiency: key features & mechanisms of disease Triac Trial I Real world data QoL, survival data & Triac Trial II
Slide 18: Thyroid gland produces thyroid hormones
Thyroid gland produces thyroid hormones
Slide 19: Thyroid gland produces thyroid hormones
Thyroid gland produces thyroid hormones
Slide 20: Thyroid gland produces thyroid hormones
Thyroid gland produces thyroid hormones
Slide 21: Thyroid gland produces thyroid hormones
Thyroid gland produces thyroid hormones
Slide 22: T3 target cell
T3 target cell
Slide 23: T3 target cell: hormones enter
T3 target cell: hormones enter
Slide 24: T3 target cell: hormones enter by transporter proteins
T3 target cell: hormones enter by transporter proteins
Slide 25: T3 regulates developmental and metabolic process via its receptor
T3 regulates developmental and metabolic process via its receptor
Slide 26: MCT8 is a key transporter
MCT8 is a key transporter
Slide 27: Thyroid hormone signaling disorders
Thyroid hormone signaling disorders
Slide 28: Outline
Outline Brief context of thyroid hormone signaling MCT8 deficiency: key features & mechanisms of disease Triac Trial I Real world data QoL, survival data & Triac Trial II
Slide 29: MCT8 deficiency: developmental & metabolic disorder
MCT8 deficiency: developmental & metabolic disorder
Slide 30: MCT8 deficiency: developmental & metabolic disorder
MCT8 deficiency: developmental & metabolic disorder Tachycardia Frequent No head control infections Epilepsy Hypotonia Dystonia Sleep Scoliosis disturbance Hypokinesia Low muscle mass Wheel chair bound Low body weight & feeding problems Friesema, Lancet 2004; Dumitrescu, AJHG 2004; Groeneweg, Lancet
Slide 31: MCT8 deficiency: mechanisms of disease
MCT8 deficiency: mechanisms of disease
Slide 32: MCT8 deficiency: mechanisms of disease
MCT8 deficiency: mechanisms of disease
Slide 33: High mortality rate
High mortality rate patients events 100 145 32 Overall survival (%) 50 0 0 20 40 60 80 Age at last follow-up (in years) Individuals at risk Total 145 68 23 8 4 Groeneweg, Lancet Diab Endocrinol 2020
Slide 34: Therapy: dual action
Therapy: dual action Increase thyroid hormone action Reduce thyroid hormone action
Slide 35: T3 analog tiratricol (Triac) – principle in MCT8 defective cells
T3 analog tiratricol (Triac) – principle in MCT8 defective cells T3 Triac (TA3)
Slide 36: Is Triac effective in patients with MCT8 deficiency?
Is Triac effective in patients with MCT8 deficiency? Neurological phenotype Triac Trial II Triac Metabolic phenotype Triac Trial I
Slide 37: Outline
Outline Brief context of thyroid hormone signaling MCT8 deficiency: key features & mechanisms of disease Triac Trial I Real world data QoL, survival data & Triac Trial II
Slide 38: Triac Trial I: international phase 2 trial
Triac Trial I: international phase 2 trial Baseline (T0) Dose-finding N=46 1 yr Median age 7.1 yrs (range 0.8 – 66.8) Stable dose End-study (T12) Long-term 3 yr treatment extension period
Slide 39: Primary outcome: T3 concentrations normalize
Primary outcome: T3 concentrations normalize 10 8 T3 (nmol/l) 6 4 Reference 2 interval 0 Patients Groeneweg, Lancet Diab Endocrinol 2019
Slide 40: Secondary outcomes: body weight improves
Secondary outcomes: body weight improves 2 A Change in body weight to age z score at month 12 Patients Reference in 0 healthy children Body weight (z score) -2 -4 -6 -8 -10 Groeneweg, Lancet Diab Endocrinol 2019
Slide 41: Secondary outcomes: heart rate improve & PACs subside
Secondary outcomes: heart rate improve & PACs subside A Change in body weight to age z score at month 12 160 10000 140 1000 Heart rate (bpm) PAC (per 24h) 120 100 100 10 80 1 60 0.10 Patients T0 T12 Groeneweg, Lancet Diab Endocrinol 2019
Slide 42: Secondary outcomes: biochemical markers improve
Secondary outcomes: biochemical markers improve 400 T0 T12 300 SHBG (nmol/L) 200 100 0 Patients Groeneweg, Lancet Diab Endocrinol 2019
Slide 43: Outline
Outline Brief context of thyroid hormone signaling MCT8 deficiency: key features & mechanisms of disease Triac Trial I Real world data QoL, survival data & Triac Trial II
Slide 44: Triac: real world data
Triac: real world data
Slide 45: Real world data: long-term reduction T3
Real world data: long-term reduction T3 10 A Change in body weight to age z score at month 12 Baseline Last visit 8 T3 (nmol/L) 6 4 2 0 Patients Van Geest, JCEM 2022
Slide 46: Real world data: sustained improvement of body weight
Real world data: sustained improvement of body weight 2 Body weight-for-age (Z score) A Change in body weight to age z score at month 12 Reference in 0 healthy children -2 -4 -6 Natural history in untreated MCT8 deficiency -8 -10 0 5 10 15 20 Age (years) Van Geest, JCEM 2022
Slide 47: Outline
Outline Brief context of thyroid hormone signaling MCT8 deficiency: key features & mechanisms of disease Triac Trial I Real world data QoL, survival data & Triac Trial II (boring slides)
Slide 48: Triac: effects on QoL
Triac: effects on QoL Post-hoc analyses on caregiver-reported patient-centered outcome measures from TT1 Semi-structured interviews (baseline, F-U visits, EoS visit in n=40) on complex needs and daily care challenges Most prominent changes Positive: improved interaction (22/39), improved alertness (
Slide 49: Triac: effects on mortality
Triac: effects on mortality International multi-center cohort study (n=173 sites; n=48 countries; n=484 screened patients) Excluded patients (DOB < 2004, n=152; limited data, n=66; unknown LoF, n=36) Baseline characteristics with (n=111) or without (n=117) Triac similar (except untreated patients le
Slide 50: Is Triac effective in patients with MCT8 deficiency?
Is Triac effective in patients with MCT8 deficiency? Neurological phenotype Triac Trial II Triac Yes Metabolic phenotype Triac Trial I Real world data
Slide 51: Triac Trial II - background
Triac Trial II - background
Slide 52: Triac normalizes brain development in mouse model
Triac normalizes brain development in mouse model WT Mct8/Oatp1c1 DKO Mct8/Oatp1c1 DKO - Triac + Triac Kersseboom, Mol Endo 2014
Slide 53: Triac Trial I – GMFM (exploratory analysis)
Triac Trial I – GMFM (exploratory analysis) 40 T0 T12 30 GMFM score (%) 20 10 0 Patients Groeneweg, Lancet Diab Endocrinol 2019
Slide 54: Triac Trial II – in/exclusion criteria
Triac Trial II – in/exclusion criteria International multi-center open label trial Inclusion criteria Male patient with MCT8 deficiency Aged ≤ 30 months at baseline Exclusion criteria Previous Triac treatment Previous L-T4 and/or PTU treatment for > 3 months
Slide 55: Triac Trial II – outcomes
Triac Trial II – outcomes Primary outcomes GMFM-88 total score Comparison with BSID-III Gross Motor Domain historical controls Secondary outcomes Item 10 (head control) and 24 (sitting) of GMFM-88 Complete BSID-III Motor milestone responder analysis of standardized neurological examination (HINE) Pa
Slide 56: Triac Trial II – cohort
Triac Trial II – cohort Screened (n=23) Enrolled (n=22) Finalized 96w (n=21)
Slide 57: Triac Trial II – primary outcomes
Triac Trial II – primary outcomes No statistical (and clinical) relevant change in GMFM or BSID
Slide 58: Triac Trial II – secondary outcomes
Triac Trial II – secondary outcomes Strong reduction in T3 concentrations Triac well tolerated
Slide 59: Triac Trial II – conclusions
Triac Trial II – conclusions No effect on brain development with Triac in early life Confirmation of reduction in T3 concentrations Ongoing analyses (subgroups)
Slide 60: Is Triac effective in patients with MCT8 deficiency?
Is Triac effective in patients with MCT8 deficiency? No (analysis in Neurological phenotype progress) Triac Trial II Triac Yes Metabolic phenotype Triac Trial I Real world data
Slide 61: Take home message
Take home message MCT8 deficiency: hypothyroid & thyreotoxic features Triac alleviates metabolic/thyreotoxic phenotype does not improve neurodevelopment in patients < 30 months (full analysis in progress) Persani, ETJ 2024
Slide 62: Acknowledgements
Acknowledgements Bedankt Thyroid group Sandra Smits Czech Republic Gerarda Cappuccio Stan Nowak Robin Peeters Femke Aarsen Jan Lebl India Angelique Zandstra Marcel Meima Marieke van der Knoop Klara Rozenkova Anna Simon Nitash Zwaveling Nilhan Gunhanlar Hans van Toor Jana Malikova Praveen Paul Anne-M
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Slide 66: Focused on Broad Patient Access and Value Creation
Focused on Broad Patient Access and Value Creation LAUNCH VISION Everyone who can benefit from treatment, gets access to reimbursed Emcitate asap after regulatory approval CRITICAL SUCCESS FACTORS Fast and accurate diagnosis Reimbursement & physicians prescribing of affected patients EXAMPLES OF KEY
Slide 67: 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 68: Launch possible with lean & agile team
Launch 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 Key Stakeh
Slide 69: ETA recommends tiratricol as long-term therapy for all patients with MCT8 deficiency
European Thyroid Association (ETA) recommends tiratricol as long-term therapy for all patients with MCT8 deficiency PRESS • ETA recommends the use of tiratricol as long-term therapy for all patients with MCT8 deficiency, and for certain patients with RTH-beta. • Inaugural 2024 Guidelines were commis
Slide 70: 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 71: Step-wise building team to execute on key activities 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 72: Driving disease awareness to support diagnosis of affected patients
Driving disease awareness, including educational initiatives, to support diagnosis of affected patients Meetings with MCT8 community Digital channels for broader reach • Advisory boards • Website • Caregivers • Social media • Medical Experts • Email campaigns • Congresses • Electronic Continuous Med
Slide 73: mct8deficiency.com is our central disease awareness hub
mct8deficiency.com is our central disease awareness hub Raise awareness around importance of fast and accurate diagnosis of MCT8 deficiency to ensure optimal care and management Key assets hosted on the platform:  HCP/Patient/Caregiver videos  Mode of disease video Resource centre including:  MoD
Slide 74: 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 75: 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 76: 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 77: Summary
Summary Broad patient access and value creation – building sustainable rare disease company • Preparing for launch in Europe and US by lean and agile Egetis team, other regions through partners • Disease awareness initiatives to support diagnosis of affected patients • Deliver solid value propositio
Slide 78: Launch Readiness Germany
Launch Readiness Germany Dr. med. Henna Oittinen Corbinelli, Medical Director Europe & International Raymond Francot, General Manager for DACH, Italy and Central & Eastern Europe ©2024 Egetis Therapeutics. All rights reserved.
Slide 79: Critical Elements of a successful Emcitate Launch
Critical Elements of a successful Emcitate Launch Ensure Access to Emcitate for all Eligible Patients upon obtaining European Marketing Authorization Pricing & Stakeholder Find Patients Reimbursement Engagement • Disease • Dossier Awareness • Key Opinion Development Leaders & HCPs • Diagnosis • Bene
Slide 80: Critical Elements of a successful Emcitate Launch
Critical Elements of a successful Emcitate Launch Ensure Access to Emcitate for all Eligible Patients upon obtaining European Marketing Authorization Pricing & Stakeholder Reimbursement Find Patients Engagement • Dossier • Disease Awareness • Key Opinion Development • Diagnosis Leaders and HCPs • Be
Slide 81: Benefit assessment and price negotiations follow a strict and transparent process
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 costs IQWIG
Slide 82: Opportunities for a successful AMNOG Process are identified
Opportunities for a successful AMNOG Process are identified Opportunities Implications • Implement comprehensive and sound dossier strategy • Orphan disease status offers respective argumentations supported by and aligned with EPAR/SmPC • Focus on 1st treatment for ultra-rare MCT8 deficiency, addres
Slide 83: Critical Elements of a successful Emcitate Launch
Critical Elements of a successful Emcitate Launch Ensure Access to Emcitate for all Eligible Patients upon obtaining European Marketing Authorization Pricing & Stakeholder Reimbursement Find Patients Engagement • Dossier • Disease Awareness • Key Opinion Development • Diagnosis Leaders & HCPs • Bene
Slide 84: Increasing Disease Awareness during pre-launch phase
Increasing Disease Awareness during pre-launch phase is an important success factor for successful launch of Ultra-Orphan drugs • Importance of strengthening awareness about MCT8 deficiency as a debilitating disease with distinct concomitant clinical presentations and the importance of its early tre
Slide 85: Critical Elements of a successful Emcitate Launch
Critical Elements of a successful Emcitate Launch Ensure Access to Emcitate for all Eligible Patients upon obtaining European Marketing Authorization Pricing & Stakeholder Reimbursement Find Patients Engagement • Dossier • Disease Awareness • Key Opinion Development • Diagnosis Leaders & HCPs • Bene
Slide 86: HCP Engagement Strategy
HCP Engagement 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 87: Strong German Expert support for increasing disease awareness
Strong German Expert support for increasing disease awareness Improving patient care in MCT8 deficiency Cross-functional core Expert group Experts agree that interdisciplinary clinical monitoring is needed to • Endocrinology/Pediatric Endocrinology improve standard of care in MCT8 deficiency • Pedia
Slide 88: Providing Access in MENAT
Providing Access in MENAT Peter Verwaijen Global Head of Brand Strategy & Commercial Business Expansion General Manager Benelux
Slide 89: 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 90: 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 93: US regulatory pathway and the ReTRIACt study
WE CARE FOR THE RARE US regulatory pathway and the ReTRIACt study December 18, 2024 Nicklas Westerholm, CEO ©2024 Egetis Therapeutics. All rights reserved.
Slide 94: Agenda: Egetis Investor Day, December 18, 2024
Agenda: Egetis Investor Day, December 18, 2024 Time (CET/ET) Subject Presenter(s) 15:00/9.00am Welcome, CHMP opinion & corporate update Nicklas Westerholm, CEO 15:10/9.10am MCT8 deficiency: recent advances with tiratricol Prof. Edward Visser, Erasmus Medical Center, NL 15:35/9.35am Q&A Visser & West
Slide 95: Emcitate regulatory pathway in US
Emcitate regulatory pathway in 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 cohort
Slide 96: 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 97: Current status of ReTRIACt trial (as of Dec. 18, 2024)
Current status of ReTRIACt trial (as of Dec. 18, 2024) Map not to scale Addenbrooke’s Hospital CHOP Cambridge, UK Philadelphia Erasmus Medical Center Rotterdam, NL New sites opened • 18 patients have been included so far, of which 8 patients have completed the randomized phase, 1 patient in the rand
Slide 98: Agenda: Egetis Investor Day, December 18, 2024
Agenda: Egetis Investor Day, December 18, 2024 Time (CET/ET) Subject Presenter(s) 15:00/9.00am Welcome, CHMP opinion & corporate update Nicklas Westerholm, CEO 15:10/9.10am MCT8 deficiency: recent advances with tiratricol Prof. Edward Visser, Erasmus Medical Center, NL 15:35/9.35am Q&A Visser & West
Slide 99: US Launch Opportunity for Emcitate
US Launch Opportunity for Emcitate December 18, 2024 Anny Bedard, President North America Ann-Marie Redmond, Head Market Access & Pricing 99 ©2024 Egetis Therapeutics. All rights reserved.
Slide 100: Progressing Strategic Priorities to Drive Launch Success
Progressing Strategic Priorities to Drive Launch Success Key Drivers Shaping Egetis US Commercial Success Key Progress Areas Driving Impactful steps towards a successful launch Identify • Refined our understanding of MCT8 Accelerate treatment deficiency patient journey and profile for patient center
Slide 101: Empowering Patient Finding Through Collaboration, Education and Innovation
Empowering Patient Finding Through Collaboration, Education and Innovation Collaborations & Partnerships Education & Awareness Innovative Data-Driven Approach • Partnering with Advocacy Groups to • Clinical trial recruitment • Leverage advanced analytics of Real- maximize impact through aligned Worl
Slide 102: Building Momentum to Scale Patient Finding Efforts
Building Momentum to Scale Patient Finding Efforts Known 2024 >100 confirmed diagnosed 2023 MCT8 deficiency patients 2022 Accelerate patient finding Naive or treated under early access efforts by integrating advanced data-driven insights into our existing Hospitals with initiatives known MCT8 defici
Slide 103: Establishing Strong Payer Relationships to Support Broad Access
Establishing Strong Payer Relationships to Support Broad Access Across Priority Segments Anticipated Payer Mix Tricare Medicare ~2% ~6% Medicaid and Commercial Commercial ~9% Early engagement to raise awareness and education of MCT8 deficiency to support speed to coverage and Medicaid reimbursement
Slide 104: 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 105: Prioritizing Impactful Activities to Optimize Access
Prioritizing Impactful Activities to Optimize Access • Foster Key Opinion Leader (KOL) • Operationalize fit for purpose • Payer value proposition and objection champions with strong understanding provider patient experience model handler of MCT8 deficiency • Design of support services to • Real worl
Slide 106: Building Our Team, Capabilities and Infrastructure for US Launch
Building Our Team, Capabilities and Infrastructure in Stepwise Approach for a Successful US Launch Supply Chain Market Access Medical Affairs • 3PL • Patient Services • Medical Scientific • Specialty Pharmacy • Payer Engagement Liaisons • Medical Info Marketing Commercial • Brand Manager Operations
Slide 107: Delivering Impact at Launch
Delivering Impact at Launch Critical Priorities 2025 • ReTRIACt results and NDA submission • Patient readiness for treatment at launch • Strong HCP commitment • Committed and active patient advocacy community • Maximized access • Purposed built organization 107 ©2024 Egetis Therapeutics. All rights
Slide 109: Resistance to Thyroid Hormone β and the Unmet Medical need
Resistance to Thyroid Hormone β and the Unmet Medical need Professor Aled Rees Consultant Endocrinologist Cardiff University United Kingdom 18th December 2024
Slide 110: Resistance to Thyroid Hormone BETA RTHβ
Resistance to Thyroid Hormone BETA = RTHβ
Slide 111: Endocrinology
Endocrinology Understand how hormones work – in health and disease
Slide 112: Overview
Overview 1. Overview of Thyroid Hormone 2. What is Resistance to Thyroid Hormone β? 3. Diagnosis of Resistance to Thyroid Hormone β 4. Effects of Resistance to Thyroid Hormone β 5. Treatment options, unmet needs 6. Future research priorities
Slide 113: Overview of Thyroid Hormone
Overview 1.Overview of Thyroid Hormone
Slide 114: Thyroid Hormone Production
Thyroid Hormone production Thyroid Hormone Production Thyroid Stimulating Hormone (TSH) + Thyroid Hormones T4 and T3
Slide 115: Thyroid Hormone Production: The Feedback Loop
Thyroid Hormone production Thyroid Hormone Production: “The Feedback Loop” Thyroid Stimulating Hormone (TSH) - + Thyroid Hormones T4 and T3
Slide 116: Thyroid Hormone Production: The Feedback Loop Example
ThyroidThyroid HormoneHormone Production:production “The Feedback Loop” Example Thyroid Stimulating Hormone (TSH) - + Thyroid Hormones T4 and T3 Overactive Thyroid Example levels Normal Levels Gland TSH LOW <0.03 0.27-4.2 T4 HIGH 45 12-22 T3 HIGH 22 3.1-6.8
Slide 117: Normal Thyroid Hormone action
Normal Thyroid Hormone action TRH Muscle Metabolic Rate TSH Bone Bone maturation Bone turnover Growth T3 T4 CNS Brain development Myelination Liver SHBG production Heart Cholesterol metabolism Inotropic effects Adapted from Moran C. Best Pract Res Clin Endocrinol Chronotropic effects Metab. 2015. PM
Slide 118: But how do Thyroid Hormones actually work?
Thyroid But howHormone Action do Thyroid II Hormones actually work? To answer this we need to go inside the cell Nucleus
Slide 119: Thyroid Hormone Action II
Thyroid But how Hormone do Thyroid Action Hormones II work? actually Thyroid Hormone Receptor DNA (genes) Genes switched OFF Cell Nucleus Any cell
Slide 120: Thyroid Hormone Action II
Thyroid But how Hormone do Thyroid Action Hormones II work? actually Thyroid Hormone Receptor Thyroid T3 T3 Hormone Present T4 DNA (genes) Genes switched ON Cell Nucleus Any cell
Slide 121: Thyroid Hormone Receptors – Two Forms
Thyroid Hormone Receptors – TWO FORMS TRH Muscle Metabolic Rate 𝛽𝛽 >𝛼𝛼 𝛼𝛼>𝛽𝛽 TSH Bone Bone maturation Bone turnover 𝛼𝛼>𝛽𝛽 Growth T3 T4 𝛼𝛼>𝛽𝛽 CNS Brain development Myelination 𝛽𝛽 >𝛼𝛼 𝛼𝛼>𝛽𝛽 Liver SHBG production Heart Cholesterol metabolism Inotropic effects Adapted from Moran…
Slide 122: Overview: What is Resistance to Thyroid Hormone β?
Overview 1. What is Thyroid Hormone, and what does it do? 2.What is Resistance to Thyroid Hormone β? 3. How might Resistance to Thyroid Hormone affect me?
Slide 123: RTHβ: Thyroid Receptor β is Resistant to Thyroid Hormone
RTH𝛽𝛽: Thyroid Receptor β is Resistant to Thyroid Hormone Resistant pathway TRH Sensitive pathway Hypothalamus Muscle 𝛽𝛽 >𝛼𝛼 Pituitary thyrotrophs 𝛼𝛼>𝛽𝛽 TSH Bone Thyroid gland 𝛼𝛼>𝛽𝛽 T3 T4 𝛼𝛼>𝛽𝛽 CNS 𝛽𝛽 >𝛼𝛼 𝛼𝛼>𝛽𝛽 Liver Heart Adapted from Moran C. Best Pract Res Clin…
Slide 124: How is Resistance to Thyroid Hormone β diagnosed?
Overview 1. What is Thyroid Hormone, and what does it do? 2. What is Resistance to Thyroid Hormone? 3.How is Resistance to Thyroid Hormone 𝛽𝛽 diagnosed? 4. How might Resistance to Thyroid Hormone affect me? 5. Research
Slide 125: The Feedback Loop in RTHβ
“The Feedback Loop” in RTHβ 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 126: RTHβ: Abnormal Thyroid blood tests
RTH𝛽𝛽: Abnormal Thyroid blood tests Resistant pathway TRH Sensitive pathway Hypothalamus Muscle 𝛽𝛽 >𝛼𝛼 Pituitary thyrotrophs 𝛼𝛼>𝛽𝛽 TSH Bone High FT4 Thyroid High FT3 gland Normal range TSH Goitre 𝛼𝛼>𝛽𝛽 70% T3 T4 𝛼𝛼>𝛽𝛽 CNS 𝛽𝛽 >𝛼𝛼 𝛼𝛼>𝛽𝛽 Liver Heart Adapted from Moran C. Best…
Slide 127: DNA testing for RTHβ
DNA testing for RTHβ “THRB” DNA Gene Blood test Cell
Slide 128: Inheritance of RTHβ
Inheritance of RTHβ Unusually inherited in an autosomal dominant pattern So for a father/mother with RTHβ, each of their children has a 1 in 2 chance of having the condition 1 in 20,000 to 40,000 Males = Females Can be diagnosed at any age Can cause many symptoms Some people have no symptoms Symptom
Slide 129: Effects of Resistance to Thyroid Hormone β
Overview 1. What is Thyroid Hormone, and what does it do? 2. What is Resistance to Thyroid Hormone? 3.How is Resistance to Thyroid Hormone diagnosed? 4. Effects of Resistance to Thyroid Hormone 𝛽𝛽 5. Research
Slide 130: RTHβ: Summary of Features
RTH𝛽𝛽: Summary of Features Resistant pathway TRH Sensitive pathway Hypothalamus Muscle Raised Metabolic Rate 𝛽𝛽 >𝛼𝛼 Pituitary thyrotrophs 𝛼𝛼>𝛽𝛽 Failure to Thrive in Childhood TSH Bone High FT4 Thyroid Low BMD High FT3 gland Delayed bone age 29-47% Normal range TSH Goitre 𝛼𝛼>𝛽𝛽 Short…
Slide 131: Increased Mortality RTHβ
Increased Mortality RTH𝛽𝛽 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 Okosieme, Lancet Diabetes Endocrinology 2023. PMID 37475119
Slide 132: Features of RTHβ: focus on heart
Features of RTHβ: focus on heart Atrial Fibrillation (Risk is increased)
Slide 133: Features of RTHβ: focus on heart
Features of RTHβ: focus on heart Heart Failure (Risk is increased)
Slide 134: Monitoring of RTHβ
Both Children and adults Monitoring of RTHβ Children only Adults only How? How often? Clinical assessment; symptoms Clinical Examination (weight, Blood Pressure, goitre, heart exam) Annual Growth, school performance, hearing, behaviour Fasting bloods for Cholesterol and Diabetes tests Blood tests An
Slide 135: Overview: Treatment options, Unmet Needs
Overview 1. What is Thyroid Hormone, and what does it do? 2. What is Resistance to Thyroid Hormone? 3.How is Resistance to Thyroid Hormone diagnosed? 4. Treatment options, Unmet Needs 5. Research
Slide 136: RTHβ: Treatment
RTH𝛽𝛽: Treatment TRH Hypothalamus Current treatment options are not optimal Pituitary 𝛽𝛽 >𝛼𝛼 TSH thyrotrophs Conventional Treatments for an Overactive High FT4 Thyroid Thyroid Gland not recommended High FT3 gland Normal range TSH Goitre 70% - Anti-thyroid drugs - Surgery to remove Thyroid…
Slide 137: RTHβ: Treatments
RTH𝛽𝛽: Treatments Resistant pathway TRH Sensitive pathway Hypothalamus Muscle Raised Metabolic Rate 𝛽𝛽 >𝛼𝛼 Pituitary thyrotrophs 𝛼𝛼>𝛽𝛽 Failure to Thrive in Childhood TSH Bone High FT4 Thyroid Low BMD High FT3 gland Delayed bone age 29-47% Normal range TSH Goitre 𝛼𝛼>𝛽𝛽 Short stature…
Slide 138: Tiratricol
Tiratricol Tiratricol Thyroid Stimulating Hormone (TSH) Thyroid Hormones T4 and T3 Tiratricol = Tri-iodothyroacetic Acid = TRIAC = “Emcitate”
Slide 139: Tiratricol
Tiratricol • Managed access programme at Cambridge • 8 adults with RTH𝛽𝛽 treated with Tiratricol monotherapy • Mean age 36 years • Duration of treatment 13-143 months (mean 40 months)
Slide 140: 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) 2 fT4 (pmol/L) A7 A7 30 A8 A8 20 1 10 0 0 Baseline 1 Nadir 2 Baseline 1 Nadir2
Slide 141: Symptoms on Tiratricol Treatment
Symptoms on Tiratricol Treatment Hyperthyroid Symptom Score 25 A1 A2 Mean HSS Score ↓ 8 Points A3 20 A4 A5 A6 15 HSS SCORE A7 A8 10 5 0 Baseline Nadir fT4 Timepoint
Slide 142: Resting Energy Expenditure
Resting Energy Expenditure 3,5 3 2,5 Baseline Resting Energy Expenditure Nadir 2 1,5 1 (Z score) 0,5 0 A7 A1 A4 A8 A2 A3 A6 A5 -0,5 -1 -1,5
Slide 143: Overview: Future Research Priorities
Overview 1. What is Thyroid Hormone, and what does it do? 2. What is Resistance to Thyroid Hormone? 3. Ho 4. w might Resistance to Thyroid Hormone affect me? 6. Future Research Priorities
Slide 144: Patient Webinar Feedback
Patient Webinar Feedback Treatments Health Registry Understanding Heart Health
Slide 145: Tack Thank you Diolch
Tack Thank you Diolch
Slide 148: Agenda: Egetis Investor Day, December 18, 2024
Agenda: Egetis Investor Day, December 18, 2024 Time (CET/ET) Subject Presenter(s) 15:00/9.00am Welcome, CHMP opinion & corporate update Nicklas Westerholm, CEO 15:10/9.10am MCT8 deficiency: recent advances with tiratricol Prof. Edward Visser, Erasmus Medical Center, NL 15:35/9.35am Q&A Visser & West
Slide 149: Concluding remarks
Concluding remarks • Egetis – a de-risked biotech with substantial unlocked potential ‒ Strong data in clinical trials, demonstrating significant effects on key clinical outcomes ‒ Already passed most of typical drug development risks ‒ Significant market opportunity • CHMP opinion for Emcitate® (ti

Egetis Therapeutics — Investor Day (December 2024) on 6ix

Egetis Therapeutics Ab — Investor Day (December 2024)

Slide 1: We Care for the Rare - Investor Day

Slide 2: Disclaimer

Slide 3: Press

Slide 4: Agenda: Egetis Investor Day, December 18, 2024

Slide 5: Several important milestones over the last 6 months

Slide 6: ETA recommends tiratricol as long-term therapy for all patients with MCT8 deficiency

Slide 7: Tiratricol treatment in MCT8 deficiency is associated with survival benefits

Slide 8: Egetis submits patent application to the USPTO

Slide 9: Egetis carried out directed share issuances amounting to SEK 300 million

Slide 10: Egetis receives positive CHMP opinion

Slide 11: Building a sustainable orphan drug company

Slide 12: Key upcoming milestones 2025-2026

Slide 13: Agenda: Egetis Investor Day, December 18, 2024

Slide 14: MCT8 deficiency: recent advances with tiratricol (Triac)

Slide 15: Disclosure

Slide 16: Outline

Slide 17: Outline

Slide 18: Thyroid gland produces thyroid hormones

Slide 19: Thyroid gland produces thyroid hormones

Slide 20: Thyroid gland produces thyroid hormones

Slide 21: Thyroid gland produces thyroid hormones

Slide 22: T3 target cell

Slide 23: T3 target cell: hormones enter

Slide 24: T3 target cell: hormones enter by transporter proteins

Slide 25: T3 regulates developmental and metabolic process via its receptor

Slide 26: MCT8 is a key transporter

Slide 27: Thyroid hormone signaling disorders

Slide 28: Outline

Slide 29: MCT8 deficiency: developmental & metabolic disorder

Slide 30: MCT8 deficiency: developmental & metabolic disorder

Slide 31: MCT8 deficiency: mechanisms of disease

Slide 32: MCT8 deficiency: mechanisms of disease

Slide 33: High mortality rate

Slide 34: Therapy: dual action

Slide 35: T3 analog tiratricol (Triac) – principle in MCT8 defective cells

Slide 36: Is Triac effective in patients with MCT8 deficiency?

Slide 37: Outline

Slide 38: Triac Trial I: international phase 2 trial

Slide 39: Primary outcome: T3 concentrations normalize

Slide 40: Secondary outcomes: body weight improves

Slide 41: Secondary outcomes: heart rate improve & PACs subside

Slide 42: Secondary outcomes: biochemical markers improve

Slide 43: Outline

Slide 44: Triac: real world data

Slide 45: Real world data: long-term reduction T3

Slide 46: Real world data: sustained improvement of body weight

Slide 47: Outline

Slide 48: Triac: effects on QoL

Slide 49: Triac: effects on mortality

Slide 50: Is Triac effective in patients with MCT8 deficiency?

Slide 51: Triac Trial II - background

Slide 52: Triac normalizes brain development in mouse model

Slide 53: Triac Trial I – GMFM (exploratory analysis)

Slide 54: Triac Trial II – in/exclusion criteria

Slide 55: Triac Trial II – outcomes

Slide 56: Triac Trial II – cohort

Slide 57: Triac Trial II – primary outcomes

Slide 58: Triac Trial II – secondary outcomes

Slide 59: Triac Trial II – conclusions

Slide 60: Is Triac effective in patients with MCT8 deficiency?

Slide 61: Take home message

Slide 62: Acknowledgements

Slide 63: Investor Day - Q&A

Slide 64: Global Launch Preparations

Slide 65: 65

Slide 66: Focused on Broad Patient Access and Value Creation

Slide 67: Preparing for Emcitate launch by Egetis and partners

Slide 68: Launch possible with lean & agile team

Slide 69: ETA recommends tiratricol as long-term therapy for all patients with MCT8 deficiency

Slide 70: Emcitate supplied globally in managed access programs

Slide 71: Step-wise building team to execute on key activities for launch success

Slide 72: Driving disease awareness to support diagnosis of affected patients

Slide 73: mct8deficiency.com is our central disease awareness hub

Slide 74: Expanding disease awareness momentum

Slide 75: Deliver solid Emcitate clinical and economic value proposition

Slide 76: Phased EU launch: Germany first

Slide 77: Summary

Slide 78: Launch Readiness Germany

Slide 79: Critical Elements of a successful Emcitate Launch