Egetis Therapeutics Ab · OMXSTO:EGTX
Egetis Therapeutics Ab — Investor Presentation (July 2021)
52 slides from Egetis Therapeutics Ab's investor presentation, Jul 7, 2021
Slide 1: Corporate presentation July 2021
Corporate presentation July 2021 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. A new specialised late-stage orphan drug development company 2. Emcitate® - Clinical development programme - Commercial opportunity 3. Aladote® - Clinical development programme - Commercial opportunity 4. The Orphan drug segment and path to market 5. Summary A. Appendix Corporate presentat
Slide 4: A new specialised late-stage orphan drug development company
1. 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: Emcitate®and Aladote® Highly attractive orphan drug segment with potential 2 >$1Bn annual sales opportunity 3 Clear path to market approval in EU and US with
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: 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 Emcitate® Phase IIb/III Aladote® Phase IIb/III *PledOx® Phase III * Egetis Therapeutics has decided to park the development of PledOx following the POLAR results. Our partner
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 signalling with life-long severe disability caused by paracetamol/acetaminophen poisoning • Lead candidate for addressing MCT8 deficiency, a condition • P
Slide 9: Emcitate® - clinical development programme
2. Emcitate® - clinical development programme Corporate presentation | Egetis Therapeutics | 2021-07-07 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: Overview of completed Phase IIb
Overview of completed Phase IIb • 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 • Results published in
Slide 13: Consistent, clinically relevant and highly significant results
Consistent, clinically relevant and highly significant results 44 out of 48 patients reached target level serum T3 in completed Phase IIb trial 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
Slide 14: Indication of positive effect on neurocognitive development in the youngest patients
Indication of positive effect on neurocognitive development in the youngest patients which is further studied in ongoing Phase IIb/III trial 40 T0 (Baseline) T12 (12month) 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; Lan
Slide 15: Ongoing Phase IIb/III early intervention trial design
Ongoing Phase IIb/III early intervention trial design Patient recruitment progresses according to plan and expected to be completed in Q4 2021 Primary • Confirm findings from Triac I Trial in youngest age group objective • Improvement in neurocognitive development as measured by GMFM1) and BSID-III2
Slide 16: Emcitate® clinical development timeline
Emcitate® clinical development timeline Acquisition of all rights and assets relating to CTA approvals in EU Teatrois in France 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 17: Emcitate® - Commercial opportunity
2. Emcitate® - Commercial opportunity Corporate presentation | Egetis Therapeutics | 2021-07-07 17
Slide 18: 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 19: 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 20: Analogue orphan drugs priced at > $250k – 400k per patient
Analogue orphan drugs priced at > $ 250k – 400k per patient Rapid market penetration with considerable sales already 3rd year in market • Payers in general accept higher prices for orphan drugs • Emcitate fulfills these criteria and can become unique, compared to traditional drugs and especially if
Slide 21: Emcitate® commercial opportunity
Emcitate® commercial opportunity – Addressing unmet needs in ultra rare diseases create substantial opportunity • Significant unmet needs for patients suffering from MCT8 deficiency, i.e. no satisfactory treatments and heavy disease burden ~10-15k potential patients MCT8 deficiency prevalence 1:50,0
Slide 22: 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 23: Aladote® - clinical development programme
3. Aladote® - clinical development programme Corporate presentation | Egetis Therapeutics | 2021-07-07 23
Slide 24: 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 25: 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 26: 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 27: 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 28: Aladote® demonstrates consistent results of reduced liver injury
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 29: 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 30: Aladote® clinical development timeline
Aladote® clinical development timeline US ODD granted Results presented at • Interim analysis after Society of Toxicology, 50% of patients EASL ILC and Lancet included EBiomedicine • Recruitment completed Regulatory interactions with FDA and EMA 2018 2019 2020/21 2022/23 2023/24 20321 Phase Ib/IIa s
Slide 31: Aladote® - Commercial opportunity
3. Aladote® - Commercial opportunity Corporate presentation | Egetis Therapeutics | 2021-07-07 31
Slide 32: 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 33: 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 34: 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 35: 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 36: The orphan drug segment and path to market
4. The orphan drug segment and path to market Corporate presentation | Egetis Therapeutics | 2021-07-07 36
Slide 37: 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 38: 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 | 2021-07-
Slide 39: 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 40: Summary
5. Summary Corporate presentation | Egetis Therapeutics | 2021-07-07 40
Slide 41: 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/acetaminophen poisoning • Lead candidate for addressing MCT8 deficiency,
Slide 42: 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 43: Upcoming pipeline milestones
Upcoming pipeline milestones ✓ FPFV pivotal • LPFV pivotal • 12m interim analysis • Filing EU/US • EU approval and launch Emcitate® Phase IIb/III early Phase IIb/III early Phase IIb/III early • US approval and launch intervention trial intervention trial intervention trial • US Rare Pediatric Diseas
Slide 44: 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® Highly attractive orphan drug segment with potential 2 >$1Bn annual sales opportunity 3 Clear path to market approval in EU and US wit
Slide 45: Appendix
A. Appendix Corporate presentation | Egetis Therapeutics | 2021-07-07 45
Slide 46: 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 47: 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 48: 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 49: 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 50: 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 51: Share Register
Share Register Ten largest shareholders Capital Votes Num. of shares Verified CETOROS AB (Peder Walberg) 19,3% 19,3% 31 858 414 2020-12-31 AVLA HOLDING AB (Kennet Roth) 10,0% 10,0% 16 572 442 2021-03-31 FJÄRDE AP-FONDEN 8,7% 8,7% 14 311 300 2021-03-31 CIDRO FÖRVALTNING AB+ PETER LINDELL 7,4% 7,4% 12
Slide 52: Thank you!
Thank you! Egetis Therapeutics egetis.com Corporate presentation | Egetis Therapeutics | 2021-07-07 52
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