Cynata Therapeutics Limited · ASX:CYP
Cynata Therapeutics — Shareholder Meeting (November 2018)
45 slides from Cynata Therapeutics's shareholder meeting presentation, Nov 15, 2018
Slide 1: A Next Generation Stem Cell Therapeutics Company
For personal use only A Next Generation Stem Cell Therapeutics Company Cynata Therapeutics Limited (ASX: CYP) – AGM Presentation 15 November 2018
Slide 2: Important Information
Important Information For personal use only This presentation has been prepared by Cynata Therapeutics Limited. (“Cynata” or the “Company”) based on information available to it as at the date of this presentation. The information in this presentation is provided in summary form and does not contain
Slide 3: Agenda
Agenda For personal use only 1 2018 Highlights and strategy 2 Graft vs. Host Disease (GvHD) update 3 Critical Limb Ischemia (CLI) approach 4 Pre-clinical programme overview 5 Outlook and next steps 6 Appendix – corporate overview www.cynata.com 3
Slide 4: 2018 Highlights and strategy
For personal use only (1) 1 2018 Highlights and strategy
Slide 5: Significant progress in 2018
Significant progress in 2018 For personal use only Meaningful impact on Selected next clinical Completed GvHD trial patient’s lives trial candidate • Completed first in-human trial of • Completed data collection for • Critical Limb Ischaemia (CLI): major CymerusTM MSCs, treating 15 Phase 1 trial in
Slide 6: Investment Summary: a Phase II-ready biotech with a scalable proprietary platform
Investment Summary: a Phase II-ready biotech with a highly scalable, proprietary platform for producing commercial quantities of allogeneic MSCs For personal use only Scalable, globally Cymerus platform enables production of high quality Mesenchymal Stem Cells at scale applicable technology Full
Slide 7: Goal: new generation of allogeneic MSC cell therapeutics
Cynata’s goal is to develop a new generation of highly potent allogeneic MSC cell therapeutics in areas of high unmet clinical need For personal use only Phase I completed, Phase II planning Preclinical data underway Fujifilm licence option Licence available GvHD A ‘hub and spoke’ business model I
Slide 8: The MSC Ecosystem
The MSC Ecosystem For personal use only Pharma companies • Typically seeking to build an MSC program Contract Manufacturers • Often seeking to in-licence (CMOs) innovative therapies that can be • Manufacture on behalf of manufactured at scale clients – they do not have • Have resources and expertise
Slide 9: Strong clinical pipeline supports commercial objectives
Strong clinical pipeline and program supports Cynata’s commercial objectives For personal use only New enhanced pipeline and clear pathway to commercialisation Direct path to market in Japan following Phase II US$300m1 GvHD Fujifilm holds a licence option for development and commercialisation of
Slide 10: Executing on a clear scientific and commercial vision
Cynata is executing on a clear scientific and commercial vision and continually assesses pathways to maximise shareholder value For personal use only Fujifilm holds a licence option for development and Multiple options to create commercialisation of Cynata’s MSCs for GvHD shareholder value Exercise
Slide 11: Cynata is currently commercialising its Cymerus platform
Cynata is currently commercialising its Cymerus platform For personal use only • Cynata is commercialising a platform technology with multiple potential commercial avenues; its business model is to secure licensees to progress clinical trials and commercialise its MSC technology in a range of indica
Slide 12: Graft vs Host Disease (GvHD)
For personal use only 2 Graft vs Host Disease (GvHD) Study update and next steps 12
Slide 13: Clinical Protocol: CYP-GvHD-P1-01
Clinical Protocol: CYP-GvHD-P1-01 Overview of clinical trial protocol For personal use only Population: ~16 Adults with steroid-resistant acute GvHD Primary Evaluation Period Visit 1 2 3 4 5 6 7 8 Days after first dose 0 3 7 14 21 28 60 100 Cohort A Cohort B n=8 DSMB n = 8 (7 treated)* 1 x 106 cells
Slide 14: Summary of Key Results
Summary of Key Results Excellent results in Phase 1 GvHD clinical trial, a clear validation of Cynata’s MSCs and the For personal use only Cymerus platform All endpoints achieved Cohort A Cohort B Pooled Cohort A Cohort B Pooled Endpoint (28 days) (28 days) (28 days) (100 days) (100 days) (100 day
Slide 15: GvHD Response – By Subject
GvHD Response – By Subject Substantial improvement in GvHD grades observed with the majority of patients reporting a For personal use only Complete Response 4 3 GvHD grade 2 1 0 Patient # A1 A2 A3 A4 A5 A6 A7 A8 B1 B2 B3 B4 B5 B6 B7 Grade change -1 -2 -1 -3 -1 -2 -3 -3 -2 -3 -3 -2 0 -2 -1 Best P C C
Slide 16: Significance of Findings
Significance of Findings For personal use only Patients with steroid-resistant Overall response rate of 93% acute GvHD typically face a dire prognosis, with extremely high A meaningful impact on mortality rates patients’ lives Key implications of clinical trial results • Endpoints in this trial were
Slide 17: Competitive Position
Competitive Position Results to date suggest that CYP-001 may be superior to other treatments for steroid-resistant For personal use only acute GvHD Key advantages of the CymerusTM process SCALABILITY & Consistent product quality – single donor overcomes regulatory concerns CONSISTENCY Lower cos
Slide 18: GvHD – Next Steps
GvHD – Next Steps For personal use only • Preparation of a Clinical Study Report (CSR) • Preparation of the CSR is a complex and time consuming process Database locked Rigorous on-site and transferred to Report text then Quality control review and statisticians, who Review by key written on basis of
Slide 19: Critical Limb Ischemia (CLI)
For personal use only 3 Critical Limb Ischemia (CLI) Overview of Cynata‘s approach
Slide 20: Critical Limb Ischemia
Critical Limb Ischemia Cynata has prioritised Critical Limb Ischemia as its next indication to take to clinical trials For personal use only • Critical Limb Ischemia (CLI) is an advanced stage of peripheral artery disease (PAD) – caused by a narrowing of the arteries in the limbs ABOUT CLI • Severel
Slide 21: Scientific support and industry enthusiasm are high for MSCs in CLI
Scientific support and industry enthusiasm are high for MSCs in CLI For personal use only Physician Perspective Mechanistic “If MSCs successfully dampen Scientific Rationale • Small clinical MSC studies have shown Rationale / immune response and promote Supporting amputation free survival (AFS) and
Slide 22: CLI study follows excellent pre-clinical results
Critical Limb Ischemia clinical study follows excellent results from an earlier pre-clinical study For personal use only Mice dosed with Cymerus MSCs experienced significantly improved Results published in a peer outcomes when compared with control group reviewed journal DAY TREATED CONTROL Loss of
Slide 23: Update on CLI clinical trial plans
Update on CLI clinical trial plans For personal use only Cymerus MSC product for CLI will be known as CYP-002 Draft clinical trial protocol synopsis is now in place o Randomised, double-blind, Phase 2 study in adults with CLI Current working assumptions:* o Primary endpoint will be Amputation Free S
Slide 24: Pre-Clinical Pipeline
For personal use only 4 Pre-Clinical Pipeline Overview of Cynata‘s activities
Slide 25: Purpose of the pre-clinical program
Purpose of the pre-clinical program Pre-clinical studies are intended to provide a rational basis for investigating the potential For personal use only safety and efficacy of an experimental drug in a particular disease indication(s) Demonstrate • MSCs have already shown promising therapeutic potent
Slide 26: Pre-clinical study programme - overview
Pre-clinical study programme - overview For personal use only *Global Pre-clinical Proof of Disease market Partner trials concept Key highlights target area opportunit started completed y Study to commence to evaluate effectiveness of Cymerus MSCs in sheep with ARDS US$2.5bn ARDS in association wi
Slide 27: Pre-clinical programme—ARDS
Pre-clinical programme—Acute Respiratory Distress Syndrome (ARDS) For personal use only ~US$2.5bn by Study partner Global 20181 market opportunity ARDS is an inflammatory process leading to build-up of fluid in the lungs and respiratory failure. Existing treatment Commonly occurs in previously hea
Slide 28: Pre-clinical programme—Heart attack
Pre-clinical programme—Heart attack For personal use only ~US$18.2bn by Study partner Global 20191 market opportunity A heart attack is life-threatening event that occurs when a blood vessel supplying the heart itself is Existing treatment suddenly blocked completely, threatening to damage the hea
Slide 29: Pre-clinical programme—Brain Cancer / Glioblastoma
Pre-clinical programme—Brain Cancer / Glioblastoma For personal use only ~US$3.3bn by Study partner Global 20241 market opportunity Glioblastoma is a type of brain cancer and is the most common type of malignant brain tumor in Existing treatment adults. Current treatment consists of surgery, radio
Slide 30: Pre-clinical programme—Diabetic wounds
Pre-clinical programme—Diabetic wounds For personal use only ~US$4.9bn by Study partner Global 20241 market opportunity Existing treatment Diabetic wounds are prevalent among the 400m+ diabetics globally and a significant opportunity options/products exists to improve existing treatments and meet
Slide 31: Pre-clinical programme—Coronary Artery Disease
Pre-clinical programme—Coronary Artery Disease For personal use only ~US$22.5bn by Study partner Global 20211 market opportunity CAD involves a narrowing of the coronary arteries due to a build-up of fatty deposits (plaque), also Existing treatment known as atherosclerosis, which reduces blood flo
Slide 32: Pre-clinical programme—Asthma
Pre-clinical programme—Asthma For personal use only ~US$25.6bn Study partner Global by 20241 market opportunity Existing treatment Chronic asthma is managed by steroid-type drugs (e.g. “puffers”). Severe, persistent, high-risk or options/products difficult-to-control asthma is much more challengin
Slide 33: Pre-clinical programme—Cytokine Release Syndrome
Pre-clinical programme—Cytokine Release Syndrome For personal use only ~US$4.5bn by Study partner Global 2022 (CAR-T)1 market opportunity Immunotherapies e.g. CAR-T cells are very promising cancer treatments, however their use is often Existing treatment associated with potentially fatal adverse e
Slide 34: Pre-clinical programme—Sepsis
Pre-clinical programme—Sepsis For personal use only ~US$5.9b by Study partner Global 2026 1 market opportunity Sepsis is the most common cause of death in Intensive Care Units despite management with Existing treatment antibiotics and supportive therapy. New therapies are urgently needed to addres
Slide 35: Outlook and Next Steps
For personal use only 5 Outlook and Next Steps
Slide 36: Key focus areas for next months
Key focus areas for next 12 months For personal use only Commence Phase II GvHD trial, with or without Fujifilm (noting that, while Fujifilm has not yet not exercised its license option, it has, in association with Cynata, commenced preparations for clinical trials and product manufacture) Comme
Slide 37: Key upcoming milestones
Key upcoming milestones Today For personal use only H1 CY2018 H2 CY2018 H1 CY2019 H2 CY2019 Phase I clinical trials GvHD If Fujifilm do not exercise their option, Cynata intends to progress to Phase 2 independently Fujifilm licence or with an alternative partner Completed option expires Detailed tri
Slide 38: Appendix: Corporate Overview
For personal use only 6 Appendix Corporate Overview
Slide 39: Investment Highlights
Investment Highlights For personal use only Scalable, world-first technology: Cymerus platform overcomes inherent challenges of other production methods and enables mass-production of therapeutic MSCs Phase II ready: Excellent Phase I results provide validation of Cynata’s Cymerus platform; Cyna
Slide 40: Corporate overview
Corporate overview For personal use only Company profile Share price performance (last 12 months, A$) Cynata Therapeutics is an Australian stock exchange 1.60 listed clinical-stage biotechnology company developing disruptive regenerative medicines. 1.20 +66% Financial information 0.80 +22% Share pri
Slide 41: Only platform to produce commercial quantities of MSCs from iPSCs
Cynata has the only platform in the world to produce commercial quantities of Mesenchymal Stem Cells from a single source: iPSCs For personal use only Today’s on-market MSC manufacturing Patented Cymerus Platform solution has a number of shortcomings overcomes shortcomings Sourcing cells from multip
Slide 42: MSCs are a highly potent form of stem cell attracting clinical interest
MSCs are a highly potent form of stem cell attracting significant clinical interest – and in need of a scalable commercial solution For personal use only Mesenchymal Stem Cells (MSCs) are believed to Global commercial potential, with multiple target play a vital role in repair and regeneration areas
Slide 43: Cell therapy is an active market attracting big pharma M&A interest
Cell therapy is an active market attracting big pharma M&A interest For personal use only USD USD USD 628M 307M 379M Acquired by Acquired by Acquired by March 2015 February 2016 January 20181 • Enables Fujifilm to combine • Enables Astellas to establish a leading • Extends existing partnership betwe
Slide 44: Globally experienced board and management team
Globally experienced board and management team For personal use only Dr Paul Wotton Dr Ross Macdonald Dr Stewart Washer Dr John Chiplin Mr Peter Webse Dr Kilian Kelly Chairman Managing Director Non-Executive Director Non-Executive Director Non-Executive Director Vice President, Product Chief Executi
Slide 45: Thank you for your attention
For personal use only Thank you for your attention Cynata Therapeutics Limited Contact details: Level 3 62 Lygon Street ross.macdonald@cynata.com Carlton Victoria 3053 +61 (0) 412 119343 Australia www.cynata.com
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