Sequana Medical N.V · EURONEXT:SEQUA
Sequana Medical — Investor Presentation (June 2023)
55 slides from Sequana Medical's investor presentation, June 2023
Slide 1: Pioneers in the treatment of fluid overload
Pioneers in the treatment of fluid overload Liver disease, Heart failure & Cancer Investor presentation – June 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: Leading player in the treatment of fluid overload
Leading player in the treatment of fluid overload • Proprietary technologies meeting large and unmet clinical needs • Key clinical problem in liver disease, heart failure, renal failure and cancer • We are not replacing diuretics – we are targeting those patients for whom they are not effective • Di
Slide 4: Strong outlook for value drivers
Strong outlook for value drivers North American alfapump study – POSEIDON Liver disease / Q4 ‘22: Q2 ‘23: Q3‘23: Q4 ‘23: NASH Primary endpoint Additional data Data patient FDA submission readout at EASL preference study for US approval H2 H1 H2 2022 2023 2023 Q4 ‘22: Q2 ‘23: Heart failure Q2 ‘23:
Slide 5: alfapump
alfapump® Proven step change in the treatment of liver refractory ascites
Slide 6: alfapump
alfapump Eliminating fluid from the peritoneal cavity – working in partnership with the bladder Fully implanted Easy implantation Long-term implantation & catheter Automatic operation patency Wireless battery charging Moves up to 4 litres / day Settings wirelessly adjusted Virtually non-clogging Rem
Slide 7: 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 Typical patient life(1) Viral Alcoholic Liver infections “Normal Life” Disease (Hepatitis B & C) ~5 days Non-Alcoholic Steatohepatitis De
Slide 8: Limitations of existing therapies
Limitations of existing therapies Drainage (Large Volume Transjugular Intrahepatic Paracentesis / LVP) Portosystemic Shunt (TIPS) Painful, Poor Quality of Complications, Life, Short Term Benefit Contraindications Permanent Catheter System Liver transplantation External Catheter, High Cost, Risk for
Slide 9: alfapump strong clinical and economic rationale
alfapump strong clinical and economic rationale Over 950 implants and hundreds of years of patient experience • Reduced burden of disease • Improved patient quality of life • Cost savings for hospitals and payers Estimated treatment cost / patient*: LVP: ~$66K alfapump: ~$37K Breakthrough Device Des
Slide 10: POSEIDON – North American pivotal study
POSEIDON – North American pivotal study Pivotal Cohort of 40 patients implanted with the alfapump alfapump Primary Secondary Enrolment implantation endpoint endpoints 3 months 3 months 3 months // 18 months // Pre-implant Post-implant Post-implant Long-term follow-up observation stabilisation
Slide 11: POSEIDON – successful North American pivotal study
POSEIDON – successful North American pivotal study 40 patients with recurrent or refractory ascites due to liver cirrhosis implanted with the alfapump Primary effectiveness endpoints exceed predefined thresholds for study success • 100% median per-patient reduction in therapeutic paracentesis (p<0.0
Slide 12: Well positioned for successful US commercialization
Well positioned for successful US commercialization Publications & US patient US filing & presentations preference study approval Submit POSEIDON data for Survey study to quantify patients’ PMA filing planned for Q4 2023 presentation at medical liver preferences for alfapump, including FDA approval
Slide 13: Large and growing North American patient population
Large and growing North American patient population NASH is forecast to drive significant growth for many years – and changing attitudes to cirrhosis Patients with recurrent or Over Over 6 – 7% CAGR refractory ascites 75,000 170,000 due to liver cirrhosis 2025 2035 Market potential growing to over $
Slide 14: 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 125 adult centres, of which 90 cover 95% of transplants ~50 commercial team initially Source: Organ Procurement & Transplantation Network, US Dept of HHS (optn.tran
Slide 15: DSR
DSR® Disease-modifying heart failure drug therapy
Slide 16: 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 17: Direct Sodium Removal (DSR)
Direct Sodium Removal (DSR) 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 removed from body to restore
Slide 18: DSR – disease-modifying heart failure drug therapy
DSR – disease-modifying heart failure drug therapy RED DESERT and SAHARA deliver clinical proof-of-concept with long-lasting clinical benefits Clinically meaningful decongestion and durable improvements in cardio-renal health Safe, effective and rapid elimination of fluid overload / restoring euvo
Slide 19: Long-term & major reduction in loop diuretic dosing
Long-term & major reduction in loop diuretic dosing Clear demonstration of improvement in cardiovascular & renal health – driving improved clinical outcomes 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† 80
Slide 20: DSR 2.0 has improved therapeutic and safety profile
DSR 2.0 has improved 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 Favora
Slide 21: MOJAVE – Phase 1/2a randomized controlled US study
MOJAVE – Phase 1/2a randomized controlled US study DSR 2.0 evaluated in diuretic-resistant chronic heart failure patients with persistent congestion Safety of DSR 2.0 demonstrated in GLP animal and Phase 1 clinical studies US IND cleared by FDA 4 weeks 3 months DSR therapy (up to daily) Safety fol
Slide 22: MOJAVE – initial data expected in Q4 2023
MOJAVE – initial data expected in Q4 2023 Top-line data in mid 2025 intended to deliver the clinical data package for partnering 2023 2024 2025 Non-randomized cohort Randomized cohort (N=3) (N=30) INDbyclearance FDA DSMB approval Initial Interim Top-line data* data data Endpoints • Safety: rate of
Slide 23: Multi-billion market opportunity for DSR
Multi-billion market opportunity for DSR Delivering value through reduced hospitalization and improved survival • ~400K chronically congested HF patients hospitalized per year in the US and EU (“frequent flyers”) • High cost patients with major burden on healthcare systems, payors and patients • Val
Slide 24: Outlook
Outlook Strong near term value drivers with clear long term potential
Slide 25: Strong outlook for value drivers
Strong outlook for value drivers North American alfapump study – POSEIDON Liver disease / Q4 ‘22: Q2 ‘23: Q3‘23: Q4 ‘23: NASH Primary endpoint Additional data Data patient FDA submission readout at EASL preference study for US approval H2 H1 H2 2022 2023 2023 Q4 ‘22: Q2 ‘23: Heart failure Q2 ‘23:
Slide 26: 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 27: Contact info
Contact info [email protected] +32 498 053579 www.sequanamedical.com
Slide 28: Back-up
sequanamedical
Slide 29: 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 29 29
Slide 30: 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 31: 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 32: POSEIDON: Pivotal cohort
POSEIDON: Pivotal cohort More than 1/3 of patients implanted with the alfapump had NASH or combined NASH etiology 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
Slide 33: POSEIDON: Primary effectiveness endpoints met
POSEIDON: Primary effectiveness endpoints met Results substantially exceeded the predefined thresholds for study success Pivotal Cohort N = 40 %* p-value** 1. Frequency of Therapeutic Paracentesis (TP) a. median per-patient ratio 100% reduction p<0.001 b. mean per-patient ratio 82% reduction Not app
Slide 34: 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 35: POSEIDON: Observed data through day 180 post-implant
POSEIDON: Observed data from patients completing alfapump therapy through day 180 post-implant* Pivotal Cohort N = 26 % 1. Frequency of TP a. median per-patient ratio 100% reduction b. mean per-patient ratio 93% reduction 2. Proportion of patients with a 50% reduction in number of TP post- vs pre- 9
Slide 36: 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. 36
Slide 37: POSEIDON Roll-In Cohort: Patient profile
POSEIDON Roll-In Cohort: Patient profile 26 patients with recurrent or refractory ascites N. American patients are Age (mean) 63 y treated early in MELD score (mean ± SD) 10.3 ± 3.9 their disease Cirrhosis etiology - Alcohol - 50.0% NASH is - NASH - 23.1% becoming a major - NASH / Alcohol - 3.8% dri
Slide 38: 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 39: POSEIDON Roll-In Cohort: Improvement in quality of life maintained
POSEIDON Roll-In Cohort: Clinically important improvement in quality of life maintained up to 12 months Physical Component Score Higher is better SF-36 General health- survey questionnaire Total Score Ascites Q Specific health- survey questionnaire for ascites Lower is better 39 39 * Clinically impo
Slide 40: SF-36 Physical Component Score is an independent predictor of mortality
SF-36 Physical Component Score is an independent predictor of mortality* 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 Physica
Slide 41: Interim POSEIDON: 70% survival at 12 months
Interim POSEIDON: 70% survival at 12 months* Compares favourably to published literature Published literature cited in AASLD practice guidelines: survival rate for refractory ascites patients of only 50% at 12 months1 *Preliminary survival rate analysis of Roll-In Cohort (25 March 2022) 41 41 Source
Slide 42: 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) 42 42
Slide 43: 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) Source: Organ Procurement & Transplantation Network, US Dept of HHS (optn.transplant.hrsa.gov) 43 43
Slide 44: 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 45: 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 45 45
Slide 46: 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 47: 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 48: 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 49: 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 50: 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 51: 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 52: 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 53: 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 54: 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 55: 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