Egetis Therapeutics Ab · OMXSTO:EGTX
Egetis Therapeutics Ab — Investor Presentation (February 2022)
63 slides from Egetis Therapeutics Ab's investor presentation, February 2022
Slide 1: Corporate presentation February 2022
Corporate presentation February 2022 A new specialised late-stage orphan drug development company
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. A new specialized late-stage orphan drug development company 2. Emcitate® - Clinical development program - Commercial opportunity 3. Aladote® - Clinical development program - Commercial opportunity 4. The Orphan drug segment and path to market 5. Summary A. Appendix Corporate presentation
Slide 4: A new specialized late-stage orphan drug development company
1. A new specialized late-stage orphan drug development company
Slide 5: A new specialized late-stage orphan drug development company
A new specialized late-stage orphan drug development company 1 Dedicated orphan drug development company with two late-stage orphan drug assets: Emcitate® and Aladote® Target MAA/NDA submissions for Emcitate in 2023 2 and for Aladote in 2024/2025 Highly attractive orphan drug segment with potential
Slide 6: 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 7: Pipeline overview
Pipeline overview Planned Emcitate EU and US filings in 2023 Candidate Preclinical Phase I Phase II/III Submission Comments Emcitate EU MCT8 deficiency H1 2023 - All clinical data available for submission Emcitate US - 16 patients, 30-day randomized trial to MCT8 deficiency mid 2023 be performed in
Slide 8: Two highly promising orphan drug candidates
Two highly promising orphan drug candidates Emcitate® – Therapy for genetic disturbance Aladote® – Prevents acute liver injury in thyroid hormone signaling with life-long severe disability caused by paracetamol/acetaminophen poisoning • Lead candidate for addressing MCT8 deficiency which affects ~1:
Slide 9: Emcitate® - clinical development program
2. Emcitate® - clinical development program Corporate presentation | Egetis Therapeutics | 2022-02-22 9
Slide 10: 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 disorder resulting in ● Absence of a functional MCT8 ● Patients appear normal a
Slide 11: 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 the MCT8 deficiency • Thyroid hormone T3 requires transporters such as MCT8 • Tiratricol is a thyroid hormone analogue
Slide 12: 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 in MCT8 defici
Slide 13: 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 14: Consistent, clinically relevant and highly significant results
Consistent, clinically relevant and highly significant results Reached target level serum T3 in completed Phase IIb trial (Triac Trial I) 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) Difference in means (95%
Slide 15: Indication of positive effect on neurocognitive development
Indication of positive effect on neurocognitive development In the youngest patients which is further studied in ongoing 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 baseline Source: Groeneweg et al; Lancet
Slide 16: New data confirms long-term efficacy and safety of Emcitate® in MCT8 deficiency patients
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 17: 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 | 202
Slide 18: 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 | 2022-02-22 18
Slide 19: 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 T3 in serum corresponds to improvement in thyroid hormone status in end target tissues • Beneficial effects are maintained or cont
Slide 20: Egetis intends to submit MAA for Emcitate® to EMA in H1 2023
Egetis intends to submit MAA for Emcitate® to EMA in H1 2023 based on existing clinical data Press release issued Dec 13, 2021 • Based on recent regulatory interactions, Egetis concludes that available data from Triac Trial I and recently published long-term data are sufficient for a Marketing Autho
Slide 21: Basis for marketing approval in the US – NDA targeted in mid 2023
Treatment effects on T3 levels in MCT8-deficiency could provide a basis for marketing approval in the US – NDA targeted in mid 2023 Press release issued Jan 18, 2022 • FDA acknowledges that a treatment effect on T3 levels and the manifestations of chronic thyrotoxicosis in MCT8-deficiency could prov
Slide 22: Controlled study agreed with the FDA to verify previous findings
Treatment effects on T3 levels in MCT8-deficiency could provide a basis for marketing approval in the US Agreed with the FDA to perform a controlled study to verify previous findings • A small, 30-day, placebo-controlled study in 16 treated patients will be conducted to verify the results on T3 leve
Slide 23: Controlled Study - design
Controlled Study - design Primary endpoint: Serum T3 levels, measured as the proportion of patients meeting T3 ≥ULN* within the randomized treatment period Primary endpoint n=8 tiratricol tiratricol tiratricol R placebo tiratricol n=8 Run-in period Randomized treatment period Follow-up period Day 0
Slide 24: Emcitate regulatory pathway to submissions in EU and US
Emcitate regulatory pathway to submissions in EU and US The first potential treatment of MCT8 deficiency, a rare genetic disease with high unmet medical need and no available treatment Included in MAA in EU To be added post approval Included in NDA in US when data available Triac Trial I EMC cohort
Slide 25: Ongoing Triac Trial II: early intervention on neurocognitive development
Ongoing Triac Trial II: to establish effects of early intervention on the neurocognitive development aspects of the disease 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 a
Slide 26: Emcitate® clinical development timeline
Emcitate® clinical development timeline US ODD RTH-b Acquisition of all rights CTA approvals in EU • Complete recruitment and assets relating to IND submission (LPFV) Triac Trial II Q1 ’22 Teatrois in France IND open (SMP) • Conduct 16 pts randomized Triac Trial I completed 30 day study for US NDA U
Slide 27: Emcitate® - Commercial opportunity
2. Emcitate® - Commercial opportunity Corporate presentation | Egetis Therapeutics | 2022-02-22 27
Slide 28: 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 29: 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 30: Supporting diagnosis through disease awareness initiatives
Supporting diagnosis through disease awareness initiatives MCT8 deficiency awareness and educational activities launched through various channels • Disease awareness and educational efforts aim to ‒ increase number of physicians who understand how to diagnose and manage MCT8 deficiency ‒ speed up di
Slide 31: 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 of Emcitate’s potential to address it • NPU and compassionate use programs MEDICINES AGENCY ‒ mechanisms to allow early access to a medicine pri
Slide 32: Analogue orphan drugs priced at premium
Analogue orphan drugs priced at premium Rapid market penetration with considerable sales already 3rd year in market • Payers in general accept higher prices for orphan drugs • Emcitate fulfills these criteria, no other drugs available or compared to traditional drugs and especially if they; being de
Slide 33: 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 34: Aladote® - clinical development program
3. Aladote® - clinical development program Corporate presentation | Egetis Therapeutics | 2022-02-22 34
Slide 35: 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 36: 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 37: 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 38: 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 39: 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-22 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 40: Pivotal Phase IIb/III study for US/EU regulatory submission
Pivotal Phase IIb/III study for US/EU regulatory submission1 • Primary: Composite of ALT and INR Efficacy • Number (%) of patients that need further NAC after 21h endpoints • Length of hospital stay • Experimental biomarkers, K18, miR-122 and GLDH Patient • Increased-risk POD patients, Late arrivals
Slide 41: Aladote® clinical development timeline
Aladote® clinical development timeline US ODD granted • Orphan Drug Designation EU Results presented at Society of Toxicology, • Initiate pivotal EASL ILC and Lancet Phase IIb/III study2 EBiomedicine • Interim analysis after 50% Regulatory interactions of patients included with FDA and EMA • Recruit
Slide 42: Aladote® - Commercial opportunity
3. Aladote® - Commercial opportunity Corporate presentation | Egetis Therapeutics | 2022-02-22 42
Slide 43: Estimating at least 175k addressable patients globally
Estimating at least 175k addressable patients globally Annual number of POD (paracetamol/acetaminophen overdose) cases hospitalized and receiving i.v. antidote. (NAC currently the only option) POD epidemiology Europe 89,000 cases/year of paracetamol overdose in the US and 105,000 USA >120k cases/yea
Slide 44: 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 toget
Slide 45: 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 46: Aladote® commercial opportunity
Aladote® commercial opportunity – Addressing unmet needs in antidote market create substantial opportunity • POD is a life-threatening condition with remaining Annual number of POD cases hospitalized and receiving i.v. antidote medical needs (NAC currently the only option) • No effective treatments
Slide 47: The orphan drug segment and path to market
4. The orphan drug segment and path to market Corporate presentation | Egetis Therapeutics | 2022-02-22 47
Slide 48: 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 49: 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 | 2022-02-
Slide 50: Commercialisation of Emcitate & Aladote
Commercialisation of Emcitate & Aladote Strong success factors… …for sustainable, profitable & lean commercialisation 1 High unmet medical need without competing compounds • Plan to build inhouse commercial capabilities for launch of Emcitate® and Aladote® in EU and US 2 Centralized, focused target
Slide 51: Summary
5. Summary Corporate presentation | Egetis Therapeutics | 2022-02-22 51
Slide 52: Two highly promising orphan drug candidates
Two highly promising orphan drug candidates Emcitate® – Therapy for genetic disturbance Aladote® – Prevents acute liver injury in thyroid hormone signaling with life-long severe disability caused by paracetamol/acetaminophen poisoning • Lead candidate for addressing MCT8 deficiency which affects ~1:
Slide 53: Late-stage orphan drug pipeline, $1Bn+ annual sales opportunity
Late-stage orphan drug pipeline, $1Bn+ annual sales opportunity Analogue benchmarks indicate substantial market potential Emcitate® Aladote® Addressable Analogue pricing Target population Pricing assumption patients1 > 10,000 > $250,000 > 175,000 $5,000 Estimate of addressable patients globally with
Slide 54: Upcoming pipeline milestones
Upcoming pipeline milestones ✓ US ODD RTH-b ✓ Fast Track Designation US • EU approval and launch • Complete recruitment (LPFV) Triac Trial II Q1 ’22 ✓ Data published confirms • US approval and launch Emcitate® • Conduct 16 pts randomized 30 day study for US NDA long-term efficacy and • US Rare Pedia
Slide 55: A new specialized late-stage orphan drug development company
A new specialized late-stage orphan drug development company 1 Dedicated orphan drug development company with two late-stage orphan drug assets: Emcitate® and Aladote® Target MAA/NDA submissions for Emcitate in 2023 2 and for Aladote in 2024/2025 Highly attractive orphan drug segment with potential
Slide 56: Appendix
A. Appendix Corporate presentation | Egetis Therapeutics | 2022-02-22 56
Slide 57: Leadership team
Leadership team Nicklas Westerholm Kristina Sjöblom Nygren CEO CMO • Took office in June 2017 and has previously worked in the AstraZeneca Group since 1995 • Took office in May 2020 and has previously worked as CMO and Head of in several global roles in various business areas, most recently as VP Pr
Slide 58: Board of directors
Board of directors Thomas Lönngren Gunilla Osswald Chairman of the board Board member • Board member since: 2021 • Board member since: 2017 • MSc in social and regulatory pharmacy and a degree in • Ph.D. in biopharmacy and pharmacokinetics Pharmacy, University of Uppsala. • Other assignments: CEO Bi
Slide 59: Share Register and Market Cap
Share Register and Market Cap • Cash position: SEK 144M (~EUR 14M)* • Number of outstanding shares: 165M • MCap: ~SEK 900M** • Listing venue: Nasdaq Stockholm Main Market Source: Monitor by Modular Finance. Compiled and processed data from various sources, including Euroclear, Morningstar and the Sw
Slide 60: Acquisition of Rare Thyroid Therapeutics on 5 November 2020
Acquisition of Rare Thyroid Therapeutics on 5 November 2020
Slide 61: 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 62: 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 63: Thank you!
Thank you! Egetis Therapeutics egetis.com Corporate presentation | Egetis Therapeutics | 2022-02-22 63
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