Egetis Therapeutics Ab · OMXSTO:EGTX
Egetis Therapeutics Ab — Investor Presentation (December 2020)
41 slides from Egetis Therapeutics Ab's investor presentation, Dec 15, 2020
Slide 1: Corporate presentation
Corporate presentation Creating a new specialised late-stage orphan drug development company
Slide 2: Disclaimer
Disclaimer The information contained in this presentation is strictly confidential. Accordingly, the information included herein may not be referred to, quoted or otherwise disclosed by you, neither directly or indirectly nor wholly or partly. By reviewing this information, you are acknowledging t h
Slide 3: Agenda
Agenda 1. Creating a new specialised late-stage orphan drug development company 2. Emcitate® - clinical development programme 3. Aladote® - clinical development programme 4. Commercial opportunity and path to market 5. Summary A. Appendix Corporate presentation | Egetis Therapeutics | 2020-12-15 3
Slide 4: Creating a new specialised late-stage orphan drug development company
1. Creating a new specialised late-stage orphan drug development company
Slide 5: New specialised late-stage orphan drug development company
New specialised late-stage orphan drug development company 1 Dedicated orphan drug development company with two late-stage orphan drug assets: Aladote® and Emcitate® 2 Highly attractive orphan drug segment with potential USDbn market opportunities 3 Clear path to market with plan to launch in EU and
Slide 6: The combination will drive synergies
The combination will drive synergies PledPharma and Rare Thyroid Therapeutics merge 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 strat
Slide 7: The acquisition and rights issue
The acquisition and rights issue 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 Rare Thyroid Therapeutics – Subscription
Slide 8: Orphan drug segment – a highly attractive opportunity
Orphan drug segment – a highly attractive opportunity • Less extensive clinical trials 1 Orphan drug designation is awarded to products targeting limited disease • Agile and faster development process populations1 Development • Lower costs • Lower development risk 2 More than 7,000 known rare diseas
Slide 9: Two highly promising orphan drug candidates in one company
Two highly promising orphan drug candidates in one company Emcitate® – Therapy for genetic disturbance Aladote® – Prevents acute liver injury in thyroid hormone signalling with life-long severe disability caused by paracetamol poisoning • Lead candidate for addressing MCT8 deficiency, a condition •
Slide 10: Late-stage orphan drug pipeline addressing billion dollar markets
Late-stage orphan drug pipeline addressing billion dollar markets Candidate Pre-clinical Phase I Phase II Phase III Approval Rare Thyroid’s candidate Emcitate® Phase IIb/III Emcitate® Aladote® Phase IIb/III *PledOx® Phase III PledPharma’s candidate Aladote® * PledOx® did not meet the efficacy endpoi
Slide 11: Upcoming pipeline milestones
Upcoming pipeline milestones • FPFV pivotal • LPFV pivotal • 12m interim analysis • Filing EU/US • First EU/US approval Emcitate® Phase IIb/III early Phase IIb/III early Phase IIb/III early and launch intervention trial intervention trial intervention trial 2020 2021 2022 2023 2024 • Regulatory inte
Slide 12: Emcitate® - clinical development programme
2. Emcitate® - clinical development programme Corporate presentation | Egetis Therapeutics | 2020-12-15 12
Slide 13: MCT8 deficiency a detrimental condition with significant unmet medical need
MCT8 deficiency a detrimental condition with significant unmet medical need • Genetic disorder resulting in impaired thyroid hormone trafficking across cellular membranes What is MCT8 deficiency? • Mutation located to the X chromosome, affecting only males • Estimated prevalence of 1:70,000 males •
Slide 14: 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 15: Overview of completed Phase IIb
Overview of completed Phase IIb Primary objective and • Evaluate the efficacy and safety of oral administration results of tiratricol in male patients with MCT8 deficiency of all ages Secondary • Highly significant primary outcome objective and results • Significant and clinically relevant effects o
Slide 16: Consistent and highly significant results in completed Phase IIb trial
Consistent and highly significant results in completed Phase IIb trial 10 T0 8 T12 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% CI) p-value Serum T3 (nmol/L) 4.97 (± 1.55) 1.82 (± 0.69) -3.15 (-3.62, -2.68) <0.0001 We
Slide 17: Indication of positive effect on neurocognitive development
Indication of positive effect on neurocognitive development in the youngest patients 40 T0 T12 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 Corporate presentation | Egetis Therapeutics | 2020-12-15 17
Slide 18: Planned Phase IIb/III early intervention trial design
Planned Phase IIb/III early intervention trial design First patient dosed Primary objective • Improvement of neurocognitive development Secondary • Achievement of motor milestones (e.g. hold head, sit independently) objective • Confirm findings from Triac I Trial in youngest age group • An open labe
Slide 19: Emcitate® clinical development timeline
Emcitate® clinical development timeline Acquisition of all rights and assets relating to Teatrois in France CTA approvals in EU Triac I Trial completed IND submission • Interim (12 month) IND open (SMP) analysis EMA Protocol AssistanceEU FPFV2 pivotal Phase • Filing EU MAA and IIb/III early US NDA i
Slide 20: Aladote® - clinical development programme
3. Aladote® - clinical development programme Corporate presentation | Egetis Therapeutics | 2020-12-15 20
Slide 21: Paracetamol poisoning
Paracetamol poisoning – no adequate treatment for increased-risk patients What is • Minimum toxic dose of paracetamol in adults is only 7.5g paracetamol • Risk factors include malnutrition, alcoholism and consumption of other medications poisoning? • Paracetamol poisoning can lead to acute liver fai
Slide 22: 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 23: Overview of completed Phase Ib/IIa
Overview of completed Phase Ib/IIa Primary outcome • Safety and tolerability of Aladote® co-treatment with NAC Secondary • Alanine transaminase (ALT), international normalised ratio (INR), keratin-18, outcomes caspase-cleaved keratin-18 (ccK18), microRNA-122 and glutamate dehydrogenase (GLDH) • An o
Slide 24: Positive proof-of-principle Phase Ib/IIa results
Positive proof-of-principle Phase Ib/IIa results Safety & tolerability Liver injury – ALT1 pre-defined secondary outcome NAC + 2 NAC + 5 NAC + 10 NAC + 2 NAC + 5 NAC + 10 % of patients needing µmol/kg µmol/kg µmol/kg µmol/kg µmol/kg µmol/kg additional NAC infusions Event NAC alone Aladote Aladote Al
Slide 25: Aladote® demonstrates consistent results of reduced liver injury
Aladote® demonstrates consistent results of reduced liver injury as measured by exploratory biomarkers K18 ccK18 miR-22 • K18 and its caspase cleaved form ccK18, is a measure of cell death and correlate with peak ALT activity during the hospital stay • miR-122 is a liver specific early marker (micro
Slide 26: Pivotal Phase IIb/III study for US/EU regulatory submission
Pivotal Phase IIb/III study for US/EU regulatory submission1 Patient population • Increased-risk POD patients, Late arrivals (>8h) requiring treatment with NAC NAC regimes • Licensed 21 hr NAC regime Initiation of randomized • IV (bolus) as soon as possible after randomization and after starting tre
Slide 27: Aladote® clinical development timeline
Aladote® clinical development timeline US ODD granted Results presented at Society of Toxicology, EASL ILC and Lancet EBiomedicine • Interim analysis Regulatory interactions • Regulatory with FDA and EMA submissions EU/US 2018 2019 2020/21 2022/23 2023/24 20321 Phase Ib/IIa study fully Regulatory in
Slide 28: Commercial opportunity and path to market
4. Commercial opportunity and path to market Corporate presentation | Egetis Therapeutics | 2020-12-15 28
Slide 29: Orphan drugs provide an attractive return on investment
Orphan drugs provide an attractive return on investment 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% NON ORPHAN 5.4 y
Slide 30: Emcitate® and Aladote® – alleviating societal burden
Emcitate® and Aladote® – alleviating societal burden Emcitate® Aladote® • All MCT8 patients have significant neurocognitive disability • In the US the annual cost in 2010 was estimated at USD 1,059m from early childhood and typically require constant, life-long to treat patients with POD3 supportive
Slide 31: Orphan drug pricing landscape
Orphan drug pricing landscape 2018 US pricing of non-oncology orphan drugs with remaining protection >600 000 700 000 Brineura Strensiq 600 000 US Revenue per patient per year (USD) 500 000 Soliris Spinraza 400 000 300 000 Emcitate® 200 000 Average price of USD 150 000 100 000 Neulasta Aladote® 0 0
Slide 32: Late-stage orphan drug pipeline – a billion USD opportunity
Late-stage orphan drug pipeline – a billion USD opportunity Emcitate® Aladote® Target population Pricing assumption Target population Pricing assumption 10-15,000 200-400,000 ~135,000 1:70.000 males affected1, 1.5bn Hospital admissions POD ~5,000 people with access to USD/per patient per year patien
Slide 33: Late-stage orphan drug pipeline – a billion USD opportunity
Late-stage orphan drug pipeline – a billion USD opportunity 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 Centrali
Slide 34: Summary
5. Summary Corporate presentation | Egetis Therapeutics | 2020-12-15 34
Slide 35: New specialised late-stage orphan drug development company
New specialised late-stage orphan drug development company 1 Dedicated orphan drug development company with two late-stage orphan drug assets: Aladote® and Emcitate® 2 Highly attractive orphan drug segment with potential USDbn market opportunities Clear path to market with plan to launch in EU and U
Slide 36: Appendix
A. Appendix Corporate presentation | Egetis Therapeutics | 2020-12-15 36
Slide 37: Leadership team
Leadership team Nicklas Westerholm Stefan Carlsson CEO CMO • Took office in June 2017 and has previously worked in the AstraZeneca Group since 1995 • Took office as CMO in November 2017. Med Dr Gothenburg University, where he in several global roles in various business areas, most recently as VP Pro
Slide 38: Board of directors
Board of directors Håkan Åström Gunilla Osswald Chairman of the board Board member • Board member since: 2011 • Board member since: 2017 • Other assignments: Chairman of the boards of directors of • Ph.D. in biopharmacy and pharmacokinetics Affibody Holding AB, Tubulus RP Förvaltning AB and MedCore
Slide 39: Scientific advisory board
Scientific advisory board Established for Aladote® Dr. Richard C. Dart • Ph.D., Chair of the Department of Medical Social Sciences at Northwestern University Feinberg School of Medicine in Chicago, USA. Expert in evaluations of patient-reported outcomes in clinical trials. Professor Laura James • MD
Slide 40: Share Register
Share Register TO BE UPDATED Corporate presentation | Egetis Therapeutics | 2020-12-15 40
Slide 41: Thank you!
Thank you! Egetis Therapeutics egetis.com Corporate presentation | Egetis Therapeutics | 2020-12-15 41
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