Sequana Medical N.v · EURONEXT:SEQUA
Sequana Medical — Investor Presentation (September 2023)
56 slides from Sequana Medical's investor presentation, September 2023
Slide 1: Pioneers in the treatment of fluid overload
Pioneers in the treatment of fluid overload Transforming lives in liver disease, heart failure & cancer Investor presentation – September 2023 Euronext: SEQUA.BR
Slide 2: Disclaimers
Disclaimers Important Notice IMPORTANT: You must read the following before continuing. The following applies to this document, the oral presentation of the information in this document by Sequana Medical NV (the "Company") or any person on behalf of the Company, and any question-and-answer session t
Slide 3: Fluid overload – large markets with strong growth
Fluid overload – large markets with strong growth Diuretic-resistance is common and current treatment options are extremely limited alfapump in liver disease DSR in heart failure • Market growing to over $2.5 billion by 2035(1) • Multi-billion market opportunity • FDA breakthrough device / Approved
Slide 4: Strong outlook for key value drivers
Strong outlook for key value drivers North American alfapump study – POSEIDON Liver disease / Q2 ‘23: H2 ‘23: Q4 ‘23: H2 ‘24: NASH Additional data Patient preference PMA submission Anticipated FDA at EASL & Matched registry* to FDA approval data 2023 2024 Q2 ‘23:Mid ‘23: Heart failure Q4 ‘23: H1‘
Slide 5: alfapump
alfapump® Proven step change in the treatment of liver refractory ascites
Slide 6: Refractory ascites – key complication of liver cirrhosis
Refractory ascites – key complication of liver cirrhosis Fatty liver disease / NASH is driving dramatic growth and change in attitudes to liver cirrhosis patients Viral infections (Hepatitis B & C) Non-Alcoholic Steatohepatitis Alcoholic Liver Disease (NASH/MASH) Liver Ascites Refractory cirrhosis A
Slide 7: Clear limitations of existing treatment options
Clear limitations of existing treatment options Little innovation or new development – transplant is a partner not competitor Paracentesis (LVP / drainage) Permanent Catheter System Painful, burdensome, short term benefit, QoL impact External Catheter, Risk for Infections / Blockage “Normal Life” TI
Slide 8: alfapump – continuous ascites removal to the bladder
alfapump – continuous ascites removal to the bladder Fully implanted automatic device for long term implantation Wireless battery charging Breakthrough Device Designation Settings wirelessly adjusted Remote data monitoring Moves up to 4 litres / day Over 1,000 systems implanted Strong IP barriers th
Slide 9: Large and strongly growing North American market
Large and strongly growing North American market NASH is forecast to drive significant growth for many years – and is changing attitudes to cirrhosis 3-5 paracentesis Priority market / year ~$500MM in 2025: 12+ paracentesis 78,000 / year patients with >$2.5 Bn recurrent or 6-7% CAGR refractory ascit
Slide 10: POSEIDON – Successful North American pivotal study
POSEIDON – Successful North American pivotal study Pivotal Cohort of 40 patients with recurrent or refractory ascites due to liver cirrhosis alfapump Primary Enrolment implantation endpoint 3 months 3 months 3 months Pre-implant observation Post-implant stabilisation Post-implant observation Primary
Slide 11: POSEIDON – strong clinical profile of alfapump
POSEIDON – strong clinical profile of alfapump Primary and key secondary endpoints presented by Prof. Wong, PI, at leading international liver congress Effective in control of ascites, “virtually eliminating needle paracentesis” NASH is already a key driver of decompensated cirrhosis Safety in li
Slide 12: Attractive pricing with derisked reimbursement
Attractive pricing with derisked reimbursement Existing DRG payment and breakthrough device designation de-risk reimbursement of alfapump Coding – Strong existing position with potential for further upside • Existing US hospital DRG payment for alfapump procedure of $60-70K in target hospitals* • Su
Slide 13: US – Go direct to liver transplant centers
US – Go direct to 90 liver transplant centers Highly efficient approach to target doctors and patients – driven by treatment guidelines Cover 95% of liver transplants ~50 person commercial team Source: Organ Procurement & Transplantation Network, US Dept of HHS (optn.transplant.hrsa.gov) 13 13
Slide 14: DSR
DSR® Disease-modifying heart failure drug therapy
Slide 15: Congestion is key driver of morbidity & hospitalization
Congestion is key driver of morbidity & hospitalization Diuretic-resistance in heart failure is common and there are few effective clinical alternatives US hospitalizations HF hospitalizations annually due to ~1m 90% due to fluid overload heart failure (HF)(3) (AKA congestion)(3) c.5d Typical Annual
Slide 16: DSR (Direct Sodium Removal) – targets the key cause
DSR (Direct Sodium Removal) – targets the key cause Eliminating fluid spread across the body – working in partnership with the kidneys 1 Sodium-free DSR product 2 Sodium diffuses from 3 DSR product + extracted 4 Body eliminates free water administered to peritoneal body into DSR product sodium remov
Slide 17: Disease-modifying heart failure drug therapy
Disease-modifying heart failure drug therapy RED DESERT and SAHARA deliver clinical proof-of-concept with long-lasting clinical benefits Effective decongestion and durable improvements in cardio-renal health Safe, effective and rapid elimination of fluid overload / restoring euvolemia Considerab
Slide 18: Long-term improvement in cardio-renal status
Long-term improvement in cardio-renal status Durable and dramatic reduction in oral loop diuretic dosing as a result of improved disease status 600 ~23 months follow-up mg furosemide equivalents 500 RED DESERT 400 101-001 101-002 300 101-003 200 101-005 101-006 100 101-007 0 101-008† 800 700 ~15 mon
Slide 19: DSR 2.0 improves therapeutic and safety profile
DSR 2.0 improves therapeutic and safety profile Strong granted IP drives high margin recurring revenue stream DSR 1.0 DSR 2.0 Sodium-free D10% (off-the-shelf) Sodium-free dextrose / icodextrin (proprietary) Clinical proof-of-concept Improved therapeutic profile Rapid clinical path Favorable
Slide 20: MOJAVE – Phase 1/2a randomized controlled US study
MOJAVE – Phase 1/2a randomized controlled US study Seeking to replicate SAHARA outcomes in US study of heart failure patients with persistent congestion 4 weeks 3 months DSR therapy (up to daily) + optimized usual care vs. Safety follow-up period IV loop diuretic + optimized usual care Endpoints • S
Slide 21: MOJAVE – initial data expected in Q4 2023
MOJAVE – initial data expected in Q4 2023 First patients enrolled in non-randomized cohort 2023 2024 2025 Non-randomized cohort Randomized cohort (N=3) (N=30, DSR = 20, control = 10) INDbyclearance FDA DSMB approval Initial Interim Top-line data* data data Top-line data in mid 2025 intended to del
Slide 22: Multi-billion commercial opportunity
Multi-billion commercial opportunity ~400K chronically congested HF patients hospitalized per year in the US and EU (“frequent flyers”) EU US $45K >$9 Bn 200K 200K US annual HF DSR addressable market patients patients hospitalization cost in US per patient Potential for premium DSR pricing through r
Slide 23: Outlook
Outlook Strong near term value drivers with clear long term potential
Slide 24: Strong outlook for key value drivers
Strong outlook for key value drivers North American alfapump study – POSEIDON Liver disease / Q2 ‘23: H2 ‘23: Q4 ‘23: H2 ‘24: NASH Additional data Patient preference PMA submission Anticipated FDA at EASL & Matched registry* to FDA approval data 2023 2024 Q2 ‘23:Mid ‘23: Heart failure Q4 ‘23: H1‘
Slide 25: Leader in large and growing markets with unmet needs
Leader in large and growing markets with unmet needs alfapump® in liver disease – market potential growing to over $2.5 billion by 2035(1) • NASH is changing liver cirrhosis market and driving strong growth • Approved in EU / FDA breakthrough device status / Strong IP portfolio • North American pivo
Slide 26: Contact info
Contact info IR@sequanamedical.com +32 498 053579 www.sequanamedical.com
Slide 27: Back-up
sequanamedical
Slide 28: Sequana Medical NV
Sequana Medical NV Founded in 2006 Gent, Belgium (HQ): corporate, clinical, commercial Zurich, Switzerland: manufacturing, engineering, QA/RA ~60 employees Euronext Brussels: SEQUA 28 28
Slide 29: Strong organisation
Strong organisation Highly experienced leadership team supported by committed and well-reputed shareholders Executive team: Ian Crosbie Kirsten Van Bockstaele Oliver Gödje Dragomir Lakic Chief Executive Officer Chief Financial Officer Chief Medical Officer VP Manufacturing Gijs Klarenbeek Martijn Bl
Slide 30: Shareholders base and financial overview
Shareholders base and financial overview Ticker: SEQUA – Euronext Brussels • Outstanding shares: 28.2M • Outstanding shares corresponding to outstanding share options: 3.9M • Analysts: • Degroof Petercam – Laura Roba • Edison – Pooya Hemami • H.C. Wainwright – Yi Chen • KBC Securities – Jeroen Van d
Slide 31: POSEIDON: Pivotal cohort
POSEIDON: Pivotal cohort alfapump therapy still ongoing at day 180 post-implant Actual data used Implanted with Evaluable for primary N = 26 Enrolled in alfapump effectiveness endpoint Pivotal Cohort at 6 months N = 71 Exited study prior to N = 40 N = 40 completing 6-month Pre-specified post-implant
Slide 32: POSEIDON: Pivotal Data - Patient profile
POSEIDON: Pivotal Data - Patient profile 40 severely decompensated patients – alcohol and NASH as key drivers of cirrhosis Age (mean) 63.6 ± 9.5 yr MELD score (mean ± SD) 15.2 ± 3.8 Cirrhosis etiology* - Alcohol - 47.5% - NASH - 37.5% - Viral hepatitis - 12.5% - Others - 11.0% TP per month prior to
Slide 33: POSEIDON: Primary effectiveness endpoints exceed predefined thresholds
POSEIDON: Primary effectiveness endpoints exceed predefined thresholds for study success* • 100% median per-patient reduction in therapeutic paracentesis (p<0.001)** • vs hypothesis of at least a 50% reduction • 77% of patients with at least 50% reduction in therapeutic paracentesis (p<0.001)** • vs
Slide 34: POSEIDON: Observed data through day 180 post-implant
POSEIDON: Observed data from patients completing alfapump therapy through day 180 post-implant* Distribution of reduction in Therapeutic Paracentesis post-implant vs pre-implant (Pivotal Cohort N = 26) * These observed patient data are not part of the main primary effectiveness endpoint analysis. 34
Slide 35: POSEIDON: Primary safety endpoint in line with expectations
POSEIDON: Primary safety endpoint in line with expectations Primary safety endpoint (pivotal cohort N = 40): • Combined rate of i) open surgical re-intervention due to pump system-related AE or to restore pump functionality, ii) pump explant (without replacement) due to pump system-related AE, or ii
Slide 36: POSEIDON: Similar number of MAEs pre vs post implant
POSEIDON: Similar number of MAEs pre vs post implant 3 months pre-implant 3 months post-implant (Day -90 to Day -1) (Day 91 to Day 180) No. of subjects with No. of subjects with No. of events No. of events events events Major Adverse Events 5 3 5 4 AKI > stage 2 0 0 1 1 Hepatorenal Syndrome 0 0 1 1
Slide 37: POSEIDON: Comparable number of serious infections pre vs post implant
POSEIDON: Comparable number of serious infections pre vs post implant 3 months pre-implant 3 months post-implant (Day -90 to Day -1) (Day 91 to Day 180) No. of subjects with No. of subjects with No. of events No. of events events events All Serious Infections 2 2 3 3 Of which: Ascites-Related Seriou
Slide 38: Despite AKIs, stable kidney function over long-term
Despite AKIs, stable kidney function over long-term • AKI events post-implant were manageable 6 months post-implant (Day 0 to Day 180) No. of events No. of subjects with events AKI stage 1 16 14 AKI 1 of limited clinical relevance AKI stage 2 4 4 AKI 2 and 3: three events resolved and three events w
Slide 39: POSEIDON: QoL assessment
POSEIDON: QoL assessment Two validated QoL methodologies are part of secondary endpoints • SF-36: • General health-survey questionnaire • Endpoint: improvement* in SF-36 Physical Component Score • Subdomains of Physical Component Score: physical functioning, role physical, bodily pain, general healt
Slide 40: POSEIDON: Clinically meaningful and statistically significant improvement in QoL
POSEIDON: Clinically meaningful and statistically significant improvement in QoL SF-36 Physical Component Score (higher is better): Ascites Q Score (lower is better): * * * * 40 40 * Pre-implant = at baseline; Post-implant = at 6 months post-implantation QoL: Quality of Life
Slide 41: SF-36 Physical Component Score is an independent predictor of mortality
SF-36 Physical Component Score is an independent predictor of mortality(1) Multicenter randomized controlled study in patients with recurrent (N=164) and refractory (N=241) ascites Patients stratified by survival 1 year after follow-up Cumulative all-cause mortality for patients below or above Physi
Slide 42: POSEIDON: 70% survival at one year post-implantation
POSEIDON: 70% survival at one year post-implantation Compares favorably to published literature citing 50% survival at 1 year from diagnosis of refractory ascites(1) Note: POSEIDON study not powered for survival 42 42 Source 1: Biggins et al., Hepatology, Vol. 74, No. 2, 2021, AASLD Practice Guidanc
Slide 43: Top 60 liver transplant centers (2021)
Top 60 liver transplant centers (2021) Top 60 centers cover 80% of transplants (6,967 out of 8,685) POSEIDON center (N = 13) Source: Organ Procurement & Transplantation Network, US Dept of HHS (optn.transplant.hrsa.gov) 43 43
Slide 44: Top 61 – 90 liver transplant centers (2021)
Top 61 – 90 liver transplant centers (2021) 30 centers cover 15% of transplants (1,347 out of 8,685) 44 44 Source: Organ Procurement & Transplantation Network, US Dept of HHS (optn.transplant.hrsa.gov)
Slide 45: Leading experts as Heart Failure Scientific Advisors
Leading experts as Heart Failure Scientific Advisors Dr. Maria Rosa Costanzo Dr. Wilson Tang Medical Director of the Edward Center for Professor of Medicine at Cleveland Clinic Lerner Advanced Heart Failure College of Medicine at Case Western Reserve Medical Director Heart Failure Research for the U
Slide 46: Persistent congestion and Worsening renal function
Persistent congestion and Worsening renal function Persistent congestion and worsening renal function are key targets in decompensated heart failure Wattad et al, American Journal of Cardiology, 2015: interaction between worsening renal function and persistent congestion in acute decompensated heart
Slide 47: DSR therapy treatment overview
DSR therapy treatment overview DSR Drug Step 1: Peritoneal catheter placement bag Step 2: DSR treatment episode 1. Infusion of DSR Drug 2. 24 hour dwell 3. Drainage DSR • Week 1: 5x DSR therapy Drug • Week 2-4: 3x/week DSR therapy Step 3: Catheter removal DSR Drug + sodium drain bag 47 47
Slide 48: RED DESERT: The first repeated DSR therapy study
RED DESERT: The first repeated DSR therapy study Repeated dose proof-of-concept study of DSR in stable heart failure patients on high dose diuretics alfapump + surgical start DSR therapy discharged from end DSR port implantation in hospital hospital therapy 2 weeks 2 weeks 4 weeks DSR therapy Contin
Slide 49: RED DESERT: Successful proof-of-concept study
RED DESERT: Successful proof-of-concept study 8 euvolemic HF patients on high dose diuretics treated with DSR 3x per week up to 6 weeks Highly effective management of fluid and sodium balance • Generally safe and well tolerated; no clinically relevant hyponatremia Significant improvement in cardio-r
Slide 50: RED DESERT: Highly effective management of fluid & sodium
RED DESERT: Highly effective management of fluid & sodium No loop diuretics required during study despite mean baseline dose of >300 mg/day furosemide equivalents Ejection Fraction (%) NT-proBNP (pg/mL) Daily Dose of loop diuretics (mg)** During DSR Treatment Subject At baseline At baseline At basel
Slide 51: SAHARA: Ph. 2a in target patient population
SAHARA: Ph. 2a in target patient population Decompensated heart failure patients with persistent congestion on high dose diuretics Patient Start DSR Euvolemic End DSR enrolment therapy state therapy Phase 1: Phase 2: Up to 3 x 2 weeks 16 weeks alfapump + port Intensive DSR therapy Maintenance DSR th
Slide 52: SAHARA: Expanding into decompensated patients
SAHARA: Expanding into decompensated patients 10 evaluable diuretic-resistant HF patients with persistent congestion on 2-6 weeks of intensive DSR therapy1 Safely, effectively and rapidly eliminate persistent congestion & restore euvolemia • All patients achieved euvolemia within one week of intensi
Slide 53: Consistently improved NYHA class
Consistently improved NYHA class RED DESERT SAHARA 4IV 4 IV 3 III 3III NYHA Class NYHA Class 2 II 2II 1I 1I 0 0 101-001 101-002 101-003 101-005 101-006 101-007 101-008 01-01 01-03 01-04 01-05 01-06 01-08 01-09 01-10 01-11 01-12 • at screening • post DSR* * Post DSR = end of phase 1 (6w) in RED DESER
Slide 54: Strong reduction in predicted mortality
Strong reduction in predicted mortality Over 75% reduction in predicted one-year mortality based on Seattle Heart Failure Model* • Seattle Heart Failure Model is a highly validated model to predict survival in heart failure • Validated in approx. 10,000 heart failure patients in over 46 countries wi
Slide 55: SAHARA: Safety
SAHARA: Safety No clinically relevant changes in serum sodium levels or progressive hyponatremia • 3 SAEs in 3 patients: • Blocked peritoneal catheter (phase 2) 2 in 2 patients DMC: related to study device but unrelated to implant procedure or treatment DMC: unrelated to study device, • Stable angin
Slide 56: Evaluating potential for DSR in renal failure
Evaluating potential for DSR in renal failure Complementary opportunity leveraging heart failure programme capabilities • Like heart failure, kidney failure / dialysis is one of the leading burdens for healthcare systems and carries a high mortality / morbidity burden • Hemodialysis seeks to tackle
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