ABIONYX Pharma SA · EURONEXT:ABNX
ABIONYX Pharma SA — Cerenis Therapeutics — Investor Presentation (May 2018)
33 slides from ABIONYX Pharma SA's investor presentation, May 2018
Slide 1: Corporate Presentation | May 2018
Corporate Presentation | May 2018
Slide 2: Disclaimer
Disclaimer This document has been prepared by Cerenis Therapeutics (the "Company") and is for information purposes only. The information and opinions contained in this document are provided as of the date of this document only and may be updated, supplemented, revised, verified or amended, and thus
Slide 3: Why invest in CERENIS?
Cerenis” THERAPEUTICS Wd Why invest in CERENIS? << | | _n — = >< —> 7
Slide 4: An Experienced Management Team
An Experienced Management Team Jean-Louis DASSEUX, PhD, MBA Founder and CEO More than 30 years of experience in the pharmaceutical industry (Pfizer, Esperion Therapeutics, Fournier Laboratories) A leading world expert in lipid metabolism, atherosclerosis and cardiovascular diseases Inventor of
Slide 5: Target Conditions with High Unmet Medical Need
Target Conditions with High Unmet Medical Need CER-001 HDL Deficiencies due to genetic defects (FPHA) HDL Mimetic CER-209 NAFLD/NASH and Associated Atherosclerosis P2Y13r Agonist HDL PLATFORM Targeted HDL Drug Delivery in Immuno-Oncology and Chemotherapy A LISTED COMPANY WITH SUBSTANTIAL POTENTIAL I
Slide 6: Multiple Value-Creating Catalysts over Next 9 months
Multiple Value-Creating Catalysts over Next 9 months Preclinical Phase I Phase II Phase III Market Programs in development Genetic HDL deficiencies Filing for Market CER-001 (FPHA1) Two orphan disease designations2 TANGO Phase III results 4Q2018 Approval end 2019 NAFLD/NASH and CER-209 Atheroscleros
Slide 7: A Solid IP Covering Composition of Matter and Methods of Use
A Solid IP Covering Composition of Matter and Methods of Use 16 patent families protecting compounds, indications and manufacturing/diagnostic methods PRODUCT INDICATION MANUFACTURING/DIAGNOSTIC Family 1: CER-001, charged lipoproteins and their uses Family 2: Manufacturing methods for reconstituted
Slide 8: CER-001: a drug for treating orphan diseases
CER-001: a drug for treating orphan diseases
Slide 9: Value proposition CER-001
Value proposition CER-001 In the short term: a drug for treating orphan diseases 1. HDL deficiency, a major unmet medical need 2. Two orphan designations for apoA-I and ABCA deficiency 3. A potential of value creation in the short term with an ongoing phase III study (TANGO) 4. A manufacturing proce
Slide 10: HDL therapy: promising treatments for removing cholesterol
HDL therapy: promising treatments for removing cholesterol Fundamental role of HDL A major epidemiological in removing cholesterol study on HDL1 At each LDL level, it is the HDL level that determines Incidence of cardiovascular events (per 1,000) over 10 years the cardiovascular risk 350 An HDL
Slide 11: The key advantage of HDL therapy for FPHA
The key advantage of HDL therapy for FPHA FPHA: genetic defect affecting HDL synthesis Mobilization of HDL cholesterol in the blood1 CERENIS’ solution restores the blood’s ability to 50 mobilize cholesterol into HDL to facilitate its elimination 40 (mg/dl) HDL (mg/dl) 30 2 orphan drug designations
Slide 12: Confirmed universal proof of concept amongst those deficient in HDL
Confirmed universal proof of concept amongst those deficient in HDL The Phase II study showed: Reduction of the vascular wall thickness Efficacy on carotid atherosclerosis1 Behaves like a natural HDL + 20% Eliminates cholesterol Average thickness of the carotid wall (%) Patient Reduces plaque
Slide 13: TANGO: a phase III in FPHA (HDL deficiencies)
TANGO: a phase III in FPHA (HDL deficiencies) The TANGO study should show: A reduction in coronary plaque in the carotid Basic 1st MRI 2nd MRI 3rd MRI MRI follow-up follow-up follow-up RANDOMIZATION SELECTION Placebo END OF Confirmatio (n=10) STUDY n and/or Genetic tests CER-001 (n=20) Induction M
Slide 14: Cerenis holds the proprietary manufacturing process for apoA-I and HDL particles
Cerenis holds the proprietary manufacturing process for natural recombinant human apoA-I and HDL particles 1 2 3 4 PRODUCTION OPTIMIZATION ASSEMBLY LOADING Secretion of the apoA- Synthesis of suitable Assembled via a Active drug I protein via a phospholipids natural process encapsulated in HDL mamma
Slide 15: CER-209: Major Potential in the Treatment of Patients with NAFLD/NASH and Atherosclerosis
CER-209: Major Potential in the Treatment of Patients with NAFLD/NASH and Atherosclerosis
Slide 16: NAFLD/NASH and Atherosclerosis
NAFLD/NASH and Atherosclerosis • Non-alcoholic fatty liver disease (NAFLD) has become one of the most frequent chronic liver diseases in the Western society and its prevalence is likely to rise even further. • Pathogenesis of NAFLD results from disturbed lipid homeostasis and excessive accumulation
Slide 17: CER-209, a potential breakthrough treatment for NASH and Atherosclerosis
CER-209, a potential breakthrough treatment for Nonalcoholic Steatohepatitis (NASH) and Atherosclerosis Unique HDL therapy enables to address NAFLD/NASH and Atherosclerosis CER-209 is the first in class drug candidate of oral P2Y13 receptor agonists. CER-209, a new-patented molecule coming from Ce
Slide 18: CER-209 increases HDL recognition by the liver by stimulating HDL receptors
CER-209 increases HDL recognition by the liver by stimulating the activity of HDL receptors A new mechanism of action which involves the last steps of the RLT pathway Agonist activity of CER-209 on the liver P2Y13 receptors facilitates elimination of mature HDL particles loaded with lipids, thro
Slide 19: CER-209: Promotes regression of liver steatosis (NASH)
CER-209: Promotes regression of liver steatosis (NASH) (preclinical model) Vehicle CER-209 @ 300 µg/kg + 23 % - 53 % - 50 % Cerenis US Patent 8,349,833 (2013); Goffinet M. et al (2014) PLOS One *p < 0.05, **p < 0.01, ***p < 0.001. Corporate Presentation| May 2018 19
Slide 20: CER-209 also inhibits carotid atherosclerotic plaque progression
CER-209 also inhibits carotid atherosclerotic plaque progression (APOE-/- preclinical model, Flow cessation model, 15 days of treatment) 80 Total Cholesterol/carotid (nmole/mg tissue) 60 - 45% 40 20 0 0 3 10 30 75 150 300 [CER-209] (µg/kg) Corporate Presentation| May 2018 20
Slide 21: CER-209: phase I clinical trial is ongoing
CER-209: phase I clinical trial is ongoing Single Dose Tolerance study (SDT) completed No drug related safety nor tolerance issues identified ✔ The pharmacokinetics observations support CER-209 once daily oral dosing Multiple Dose Tolerance study (MDT) in subjects with a high risk of NAFLD/NASH
Slide 22: Cargomers and HDL Mimetics
CARGOMERS® AND HDL MIMETICS CERENIS’ PROPRIETARY TARGETED HDL DRUG DELIVERY
Slide 23: Value proposition CERENIS Targeted HDL Drug Delivery Platform
Value proposition CERENIS Targeted HDL Drug Delivery Platform Expanding its HDL strategy into immuno-oncology and chemotherapeutic drug delivery 1. First HDL nanoparticle delivery platform to be initially dedicated to the oncology market 2. New strategic markets and value-creation prospects: Immuno-
Slide 24: HDL particles are perfect delivery vehicles to cancer cells
HDL particles are perfect delivery vehicles able to selectively bring cell killing agents to cancer cells Tumor Drug Chemotherapy Recognition of cancer cells via Scavenger Receptor (SR-BI) HDL nanoparticles capable of transporting active ingredients SR-B1 receptor Targeted cell Essential to cell hom
Slide 25: ApoA-I with its flexible structure accommodates different drug loads
ApoA-I with its flexible structure, is a key asset to accomodate different drug loads and target different tissues Cargomer®: multimeric apoA-1 Discoidal HDL (2-6nm) (7-9nm) The ability of monomeric apoA-I to form multimeric structures, offers the opportunity to have a carrier with adaptive capaci
Slide 26: Recombinant human apoA-I Pre beta HDL targeted Paclitaxel delivery
Recombinant human apoA-I Pre beta HDL demonstrates excellent targeted Paclitaxel delivery in xenograft murine model of human breast cancer Paclitaxel in tumor (24h after treatment) Tumor volume / groups 80 1000 Excipient Abraxane® CER-Paclitaxel Paclitaxel (ng/mg tumor) 800 60 Tumor volume ( mm 3 )
Slide 27: Immuno-oncology: effects of Cargomer on melanoma
Immuno-oncology: effects of Cargomer® (apoA-I multimer) on melanoma Tumor Volume Tumor weight 2000 4000 Gp 1: Vehicle (PBS) Gp 2: TAs + Chol-CpG Gp 3: TAs + Chol-CpG + ImmunoT Gp 4: Cargomers 1:2 Gp 5: Cargomers 1:4 1500 3000 Tumor volume (mm3) Tumor weight (mg) 1000 2000 500 1000 0 0 0 5 10 15 20 2
Slide 28: HDL particles have strong advantages over existing drug delivery technologies
HDL particles have strong advantages over existing drug delivery technologies HDL particles Natural structure stabilized by apolipoproteins, particularly apolipoprotein A-I Safety and efficacy ✓ (apo A-I) and uniquely capable of delivery of biologically active molecules to tissues and circulating ce
Slide 29: First patients enter TARGET study
First patients enter TARGET study NOVEMBER 2017 Study initiated to evaluate HDL nanoparticles in patients with esophageal cancer • First ever performed clinical study testing the potential of labelled HDL to visualize tumors in cancer patients. • Aim of the TARGET study: assess the concentrations of
Slide 30: Scientific advisory board in oncology
Seasoned scientific experts and strategic pharmaceutical industry veterans join Cerenis’ scientific advisory board in oncology MAY 2018 Creation of its Scientific Advisory Board in Oncology (SAB-Oncology) with five key appointments Briggs Morrison, MD: Chairman of Cerenis’ SAB Oncology – CEO of Sy
Slide 31: Cerenis to attend 2018 HDL Workshop
Cerenis to attend 2018 HDL Workshop (adjacent to ATVB|PVD Scientific Sessions) MAY 2018 Jean-Louis Dasseux, founder and CEO of CERENIS Therapeutics, presented at the 2018 HDL Workshop adjacent to the Vascular Discovery Scientific Sessions Presentation of CERENIS Therapeutics’ strategy in targeted
Slide 32: Value proposition: why invest in CERENIS?
Value proposition: why invest in CERENIS? CER-001: major potential in the treatment of patients with HDL deficiencies due to genetic defects 1. A potential for value creation in the short term (TANGO Phase III results 4Q2018) 2. Two orphan drug designations granted 3. A high unmet medical need 4. Ap
Slide 33: Corporate presentation – May 2018
Corporate presentation – May 2018
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