Egetis Therapeutics Ab · OMXSTO:EGTX
Egetis Therapeutics Ab — Investor Presentation (February 2023)
77 slides from Egetis Therapeutics Ab's investor presentation, Feb 23, 2023
Slide 1: We Care for the Rare – Corporate presentation
WE CARE FOR THE RARE Corporate presentation February 2023 An integrated orphan drug company, focusing on late-stage development for commercialization
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: Agenda
Agenda 1. An integrated orphan drug company, focusing on late-stage development for commercialization 2. Emcitate® - MCT8-deficiency and clinical experience with Emcitate - Regulatory pathway to submissions in EU and US - Commercial opportunity 3. Aladote® - Paracetamol/Acetaminophen overdose and cl
Slide 4: An integrated orphan drug company
WE CARE FOR THE RARE 1. An integrated orphan drug company, focusing on late-stage development for commercialization
Slide 5: An integrated orphan drug company, focusing on late-stage development
An integrated orphan drug company, focusing on late-stage development for commercialization Dedicated orphan drug company 1 Two late-stage assets: Emcitate and Aladote Target MAA/NDA submissions: 2 Emcitate 2023 and Aladote 2025 Highly attractive orphan drug segment with potential 3 >$1Bn annual sal
Slide 6: Building a sustainable orphan drug company
WE CARE Building a sustainable orphan drug company FOR THE RARE • Successfully develop Emcitate and Aladote for market approvals in 2024 and 2025/26, respectively • Commercialize Emcitate and Aladote through an inhouse focused organization in Europe/ North America and partnerships in RoW • Realize t
Slide 7: 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 8: Pipeline overview
Pipeline overview Planned Emcitate EU and US filings in 2023 Candidate Preclinical Phase I Phase II/III MAA/NDA Comments Emcitate EU MCT8 deficiency Q2 2023 - All clinical data available for submission Emcitate US - 16 patients, 30-day randomized trial to be MCT8 deficiency mid 2023 initiated in Q1
Slide 9: Two highly promising orphan drug candidates
Two highly promising orphan drug candidates Aladote® – To prevent acute liver injury caused by Emcitate® – Therapy for MCT8 deficiency paracetamol poisoning • MCT8 deficiency affects ~1:70,000 males: high unmet medical need, no • Paracetamol poisoning is one of the most common overdoses with availab
Slide 10: Commercialisation of Emcitate & Aladote
Commercialisation of Emcitate & Aladote Commercial infrastructure build up initiated Strong success factors… …for sustainable, profitable & lean commercialisation 1 High unmet medical need without competing compounds • Building inhouse commercial capabilities for launch of Emcitate® and Aladote® in
Slide 11: MCT8-deficiency and clinical experience with Emcitate
2. MCT8-deficiency and clinical experience with Emcitate Corporate presentation | Egetis Therapeutics | 2023-02-23 11
Slide 12: 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 13: 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 14: Emcitate Overview
Emcitate® Overview Lead candidate for addressing MCT8 deficiency, a condition with high unmet medical need and no available treatment • Triac Trial I completed with significant and clinically relevant effects • Erasmus Medical Center cohort study confirms long-term efficacy and safety for up to 6 ye
Slide 15: 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 16: 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 17: Triac Trial I: Indication of positive effect on neurocognitive development
Triac Trial I: Indication of positive effect on neurocognitive development In the youngest patients which is further studied in ongoing, fully recruited, Triac Trial II 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 baselin
Slide 18: New data confirms long-term efficacy and safety of Emcitate
New data confirms 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
Slide 19: 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 named patient program monitoring N=40 N=27 EMC cohort Published in JCEM N=67 Corporate presentation | Egetis Therapeutics | 20
Slide 20: 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 Corporate presentation | Egetis Therapeutics | 2023-02-23 20
Slide 21: Consistent 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 22: Emcitate regulatory pathway to submissions in EU and US
2. Emcitate® - regulatory pathway to submissions in EU and US Corporate presentation | Egetis Therapeutics | 2023-02-23 22
Slide 23: 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 Eligibility: Six months review of NDA & rolling submission Rare pediatric disease designation (FDA) PRV Eligi
Slide 24: Emcitate regulatory pathway to submissions in EU and US
Emcitate regulatory pathway to submissions in EU and US The first potential treatment for MCT8 deficiency, a rare genetic disease with high unmet medical need and no available treatment Included in MAA in EU in H1 2023 To be added post approval Included in NDA in US mid 2023 under the Fast Track Des
Slide 25: Egetis intends to submit MAA for Emcitate to EMA in Q2 2023
Egetis intends to submit MAA for Emcitate® to EMA in Q2 2023 based on existing clinical data • Based on regulatory interactions, Egetis concludes that available data from Triac Trial I and recently published long-term data are sufficient for a Marketing Authorisation Application (MAA) in Europe • Ha
Slide 26: Content in Emcitate MAA submission
Content in Emcitate MAA submission • Regulatory submissions in major regions contain the same type of key information on Efficacy, Safety and Quality ‒ presented in a common format (called CTD - Common Technical Document) Common technical document Key components of regulatory dossier Clinical data N
Slide 27: Treatment effects on T3 levels could provide basis for approval
Treatment effects on T3 levels in MCT8-deficiency could provide a basis for marketing approval in the US – NDA targeted in H2 2023 • FDA acknowledges that a treatment effect on T3 levels and the manifestations of chronic thyrotoxicosis in MCT8-deficiency could provide a basis for marketing approval
Slide 28: ReTRIACt: withdrawal of Emcitate in males with MCT8 Deficiency
ReTRIACt: withdrawal of Emcitate in males with MCT8 Deficiency Randomized placebo-controlled trial needed for NDA submission Primary • Proportion of participants who meet the rescue criterion (serum total T3 > ULN) endpoint during the 30-day double-blind Randomized Treatment Period Secondary • Chang
Slide 29: Controlled Study (ReTRIACt) – design agreed with FDA
Controlled Study (ReTRIACt) – design agreed with FDA Primary endpoint: Proportion of participants who meet the rescue criterion (T3>ULN) during the 30-day double-blind Randomized Treatment Period Primary endpoint n=8 tiratricol tiratricol tiratricol R placebo tiratricol n=8 Run-in period Randomized
Slide 30: Triac Trial II fully recruited
Triac Trial II fully recruited: to establish effects of early intervention on neurocognitive development Market approval not dependent on Triac Trial II data Primary • Confirm findings from Triac Trial I in youngest age group objective • Improvement in neurocognitive development as measured by GMFM1
Slide 31: Emcitate milestones and timelines
Emcitate milestones and timelines • EU & US approvals, US & EU ODD RTH-b pricing and launch US Rare Pediatric Triac Trial II fully • US Priority Review Disease Designation recruited Voucher (upon approval) Start Triac Trial II Launch preparations • Triac Trial II data 2020 2021 2022 2023 2024 • Star
Slide 32: 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 PRV Examples of PRVs sold • The FDA grants Rare Pediatric Disease designation (RPD) Seller Buyer Value Year to therapies for serious or life-thre
Slide 33: Emcitate – Commercial opportunity
2. Emcitate® - Commercial opportunity Corporate presentation | Egetis Therapeutics | 2023-02-23 33
Slide 34: Estimating 10-15k addressable patients globally
Estimating 10-15k addressable patients globally No approved treatment for MCT8 deficiency ~10-15k potential patients MCT8 deficiency prevalence MCT8 deficiency epidemiology 1:50,000-70,000 males • At least one new-born diagnosed per 140,000 live births in the Netherlands in past years, corresponding
Slide 35: 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 36: Emcitate supplied globally on a named patient basis
Emcitate supplied globally on a named patient basis The named patient use (NPU) confirms the significant unmet medical need in MCT8 deficiency and the view on how Emcitate address it • NPU and compassionate use programs MEDICINES AGENCY ‒ mechanisms to allow early access to a medicine prior to regul
Slide 37: Commercialization of Emcitate
Commercialization of Emcitate Disease area conditions provide opportunity for lean commercialization Favorable conditions for launch success Stepwise establishing inhouse commercial capabilities • Preparing for 2024 launch in US and Europe with Addressing unmet medical need organization of 40-50 emp
Slide 38: Building commercial organization for launch
Building commercial organization 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 – Leadership team brings launch skills and best practices from in total 100+ years at intern
Slide 39: 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 40: Enabling patient identification through disease awareness
Enabling patient identification through disease awareness MCT8 deficiency awareness and educational activities launched through various channels mct8deficiency.com EXHIBIT AT DISEASE AWARENESS COLLABORATION WITH OPTIMUM CHANNEL MIX SCIENTIFIC/MEDICAL AND EDUCATION PAGs & KOLs FOR MAXIMUM REACH CONFE
Slide 41: Aiming for broad access to Emcitate for affected families
Aiming for broad access to Emcitate for affected families Payer projects initiated to generate optimal reimbursed price – No families should pay out of own pocket • Payers in general accept higher prices for • Emcitate fulfills these criteria, no other drugs orphan drugs compared to traditional drug
Slide 42: Developing a clinical and economic value proposition
Developing a compelling Emcitate clinical and economic value proposition to secure reimbursement & access Key for payer assessments to describe unmet need & quantify burden of MCT8 deficiency • The impact of MCT8 deficiency on patients and caregivers is underreported • Significant clinical and econo
Slide 43: Implemented Expanded Access Program as requested by the FDA
Implemented Expanded Access Program as requested by the FDA - will Simplify Process for Accessing Emcitate • High demand for single patient INDs (Investigational New Drug) resulting in process delays • FDA Requested Expanded Access Program Transition - Simplify Process for Accessing Emcitate • Patie
Slide 44: Exclusive Distribution Model Through Speciality Pharmacy
Exclusive Distribution Model Through Speciality Pharmacy is Preferred option for Rare Disease • Insurance resolution and contracting • Prior authorization support • Appeals • Dedicated case managers • Improves patient experience and outcomes • Patient Assistance and Copay Support •Aim for no family
Slide 45: US Pricing & Reimbursement
US Pricing & Reimbursement Relatively straight forward for ultra-orphans with key focus on rarity and severity of disease Analogues selected based on: Emcitate’s value drivers confirmed by US payer research: • Rarity (ultra-orphan) • Paediatric “You have all the things “The product gets paid for “If
Slide 46: Paracetamol/Acetaminophen overdose and clinical experience with Aladote
3. Paracetamol/Acetaminophen overdose and clinical experience with Aladote Corporate presentation | Egetis Therapeutics | 2023-02-23 46
Slide 47: Paracetamol/acetaminophen poisoning
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 48: 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 49: 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 50: 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 51: Aladote demonstrates reduced liver injury 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 52: Aladote – Regulatory pathway to submissions in EU and US
3. Aladote® - Regulatory pathway to submissions in EU and US Corporate presentation | Egetis Therapeutics | 2023-02-23 52
Slide 53: 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 co
Slide 54: 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 55: 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 2023 2024 2025/26 • Start pivotal Phase • Regulatory submissions IIb/III study Europe/US • Europe/US approvals and launch • Regulatory
Slide 56: Aladote – Commercial opportunity
3. Aladote® - Commercial opportunity Corporate presentation | Egetis Therapeutics | 2023-02-23 56
Slide 57: 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 58: 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 59: 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 60: The attractiveness of the orphan drug segment
4. The attractiveness of the orphan drug segment Corporate presentation | Egetis Therapeutics | 2023-02-23 60
Slide 61: 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 62: 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. Corporate presentation | Egetis Therapeutics | 2023-02-
Slide 63: Summary
5. Summary Corporate presentation | Egetis Therapeutics | 2023-02-23 63
Slide 64: EGTX – a de-risked biotech with substantial unlocked potential
EGTX – 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, Shi
Slide 65: Two highly promising orphan drug candidates
Two highly promising orphan drug candidates Aladote® – To prevent acute liver injury caused by Emcitate® – Therapy for MCT8 deficiency paracetamol poisoning • MCT8 deficiency affects ~1:70,000 males: high unmet medical need, no • Paracetamol poisoning is one of the most common overdoses with availab
Slide 66: Upcoming pipeline milestones
Upcoming pipeline milestones • FPI ReTRIACt* trial for US NDA • EU approval and launch • Results ReTRIACt* for US NDA • US approval and launch Emcitate® ✓ US & EU ODD RTH-b • Filing EU MAA Q2 ‘23 • US Rare Pediatric Disease Priority Review Voucher ✓ Recruitment completed in Triac Trial II, Q2 2022 •
Slide 67: An integrated orphan drug company, focusing on late-stage development
An integrated orphan drug company, focusing on late-stage development for commercialization Dedicated orphan drug company 1 Two late-stage assets: Emcitate and Aladote Target MAA/NDA submissions: 2 Emcitate 2023 and Aladote 2025 Highly attractive orphan drug segment with potential 3 >$1Bn annual sal
Slide 68: Appendix
A. Appendix Corporate presentation | Egetis Therapeutics | 2023-02-23 68
Slide 69: Discovery of Emcitate (Triac, tiratricol)
Discovery of Emcitate (Triac, tiratricol) T3 Triac (tiratricol)
Slide 70: 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 71: Leadership team with global experience & proven track record
Leadership team with global experience & proven track record Nicklas Westerholm Anny Bedard CEO President Egetis North America • Joined 2017 • Joined 2022 • AstraZeneca 1995-2017 • Commercial leadership roles at Shire and Sarepta • VP Late-stage development CVMD • Executive Officer & VP Japan Operat
Slide 72: Board of directors
Board of directors Thomas Lönngren Gunilla Osswald Chair of the board Board member • Board member since 2017 • Board member since 2021 • PhD in biopharmacy and • MSc in social and regulatory pharmacy and a pharmacokinetics degree in Pharmacy, University of Uppsala. • Other assignments: CEO BioArctic
Slide 73: Share Register and Market Cap
Share Register and Market Cap • Cash position: SEK 128M (~EUR 12M)* + SEK 196M (~EUR 18M)** • Number of outstanding shares: 249.6M • MCap: ~SEK 1,3 billion*** • Listing venue: Nasdaq Stockholm Main Market The shareholder list doesn’t yet reflect changes from the Directed Share Issue Jan 25, 2023 Sou
Slide 74: Acquisition of Rare Thyroid Therapeutics
Acquisition of Rare Thyroid Therapeutics on 5 November 2020
Slide 75: The combination will drive synergies
The combination will drive synergies PledPharma and Rare Thyroid Therapeutics merged to launch a new company + = PledPharma Rare Thyroid Therapeutics Synergistic orphan drug focus • Team with profound late-stage drug • Team with strong track-record of identifying • 2020 accelerated PledPharma’s stra
Slide 76: The acquisition and rights issue
The acquisition and rights issue Institutional investor base broadened Acquisition Rights issue On 5 November 2020, PledPharma Successfully raised SEK 250 million in oversubscribed rights issue acquired all outstanding common shares (c. SEK 200m) and utilized overallotment option (c. SEK 50m) in Rar
Slide 77: Thank you!
Thank you! Egetis Therapeutics egetis.com Corporate presentation | Egetis Therapeutics | 2023-02-23 77
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