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A Clinical Stage Company Pioneering the Next Generation of Cellular Therapies Investor Webinar 6 February 2025
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Summary information This Presentation contains summary information about Cynata Therapeutics Limited and its subsidiaries (CYP, or Cynata) which is current as at 5 February 2025. This Presentation should be read in conjunction with CYP’s other periodic and continuous disclosure information lodged with the Australian Securities Exchange (ASX), which are available at www.asx.com.au. Not an offer This Presentation is not a prospectus, product disclosure statement or other offering document under Australian law (and will not be lodged with the ASIC) or any other law. This Presentation is for information purposes only and is not an invitation or offer of securities for subscription, purchase or sale in any jurisdiction. The release, publication or distribution of this Presentation (including an electronic copy) outside Australia may be restricted by law. If you come into possession of this Presentation, you should observe such restrictions. Any non-compliance with these restrictions may contravene applicable securities laws. Not investment advice This Presentation does not constitute investment or financial product advice (nor tax, accounting or legal advice) or any recommendation by CYP or its advisers to acquire CYP securities. This Presentation has been prepared without taking account of any person’s individual investment objectives, financial situation or particular needs. Before making an investment decision, prospective investors should consider the appropriateness of the information having regard to their own investment objectives, financial situation and needs and seek legal, accounting and taxation advice appropriate to their jurisdiction. CYP is not licensed to provide financial product advice in respect of CYP securities. Investment risk and past performance An investment in CYP securities is subject to known and unknown risks, some of which are beyond the control of CYP and its directors. CYP does not guarantee any particular rate of return or performance of CYP. Past performance cannot be relied upon as an indicator of (and provides no guidance as to) future CYP performance including future share price performance. Financial data All financial information in this Presentation is in Australian currency (A$) unless otherwise stated. This Presentation contains historical financial information based on financial information that has been disclosed to the ASX. Any discrepancies between totals and sums of components in tables and figures in this Presentation are due to rounding. Forward-looking statements This Presentation contains certain ‘forward looking statements’, which can generally be identified by the use of forward looking words such as ‘expect’, ‘anticipate’, ‘likely’, ‘intend’, ‘should’, ‘could’, ‘may’, ‘predict’, ‘plan’, ‘propose’, ‘will’, ‘believe’, ‘forecast’, ‘estimate’, ‘target’, ‘outlook’, ‘guidance’, ‘potential’ and other similar expressions. The forward looking statements contained in this Presentation are not guarantees or predictions of future performance and involve known and unknown risks and uncertainties and other factors, many of which are beyond the control of CYP, its directors and management, and may involve significant elements of subjective judgment and assumptions as to future events which may or may not be correct. There can be no assurance that actual outcomes will not differ materially from these forward looking statements. A number of important factors could cause actual results or performance to differ materially from the forward looking statements. No representation or warranty, express or implied, is made as to the accuracy, likelihood of achievement or reasonableness of any forecasts, prospects, returns or statements in relation to future matters contained in this Presentation. The forward looking statements are based on information available to CYP as at the date of this Presentation. Except as required by law or regulation (including the ASX Listing Rules), CYP and its directors, officers, employees, advisers, agents and intermediaries undertake no obligation to provide any additional or updated information whether as a result of new information, future events or results or otherwise. You are strongly cautioned not to place undue reliance on forward-looking statements. Industry and Market data Certain market and industry data used in connection with this Presentation may have been obtained from research, surveys or studies conducted by third parties, including industry or general publications. Neither CYP nor its representatives have independently verified any such market or industry data provided by third parties or industry or general publications. Disclaimer To the maximum extent permitted by law, CYP and its advisers, affiliates, related bodies corporate, directors, officers, partners, employees and agents (Related Persons) exclude and disclaim all liability, including without limitation for negligence, for any expenses, losses, damages or costs arising from this Presentation or reliance on anything contained in or omitted from it. To the maximum extent permitted by law, CYP and its Related Persons make no representation or warranty, express or implied, as to the currency, accuracy, reliability or completeness of information in this Presentation and disclaim any obligation or undertaking to release any update or revision to the information in this Presentation to reflect any change in expectations or assumptions. Statements made in this Presentation are made only as at the date of this Presentation. The information in this Presentation remains subject to change without notice. 2 Important information
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Corporate overview Financial information Share price (5 February 2025) A$0.245 Shares on issue ~225m Market capitalisation ~A$55m 3 Largest shareholders Share price – calendar year 2024 10.5% Bioscience Managers is an international healthcare investment firm headquarter in Melbourne that finances and enables innovative science and technology with the potential to transform healthcare. 10% Fidelity International is a world leading investment and asset management firm, responsible for total client assets of >US$750 billion, from clients across Asia Pacific, Europe, the Middle East, South America and Canada. Top 20 shareholders hold ~47% of shares on issue 3.6% Fujifilm is a Japanese multinational conglomerate. Cynata has a strategic manufacturing partnership with Fujifilm. Cynata is an ASX-listed company (ticker CYP), founded to commercialise the novel iPSC-based Cymerus platform, for the scalable and consistent production of mesenchymal stem cell (MSC)-based therapies
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1. Global Graft versus Host Disease Market 2019-2029 (Reflects forecast market in 2026); 2. Zion Market Research, 2019 (represents global treatment market in 2025); 3. Persistence Market Research 2018 research report: “Osteoarthritis Treatment Market: Global Industry Analysis (2012-2016) and Forecast (2017-2025) (Reflect OA market by 2025); 4. Organ Transplant Immunosuppressant Drugs Market in 2026, Grand View Research, Inc.,2019 USYD = University of Sydney; NHMRC = National Health and Medical Research Council; LUMC = Leiden University Medical Center * Timing of events is approximate, based on the Company’s information as at the date of this presentation, and subject to change. Timing refers to calendar years. 4 Target indications Indication Market opportunityTrial phase Phase 2 ongoing Phase 1/2 ongoing Kidney Transplantation (managed and funded by LUMC) Phase 1 complete Diabetic Foot Ulcers (DFU) Acute Graft vs Host Disease (aGvHD) FDA Orphan Designation US$5.9bn4 US$600m1 US$11.6bn3 US$9.6bn2 Phase 3 ongoing (enrolment complete) Osteoarthritis (OA) (managed by USYD, funded by NHMRC) Cynata Funded & Managed Upcoming catalysts* Enrolment completion – H1 2025 Results – H2 2025 Results (Cohort 1) – H1 2025 Results released Dec 2024 Results – H1 2026 Partner Funded & Managed Note: Cynata retains commercial rights for both of the partner funded & managed programs
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• Phase 1 clinical trial in diabetic foot ulcer (DFU) completed • CYP-006TK demonstrated to be safe and well tolerated, with positive efficacy data indicating substantially improved wound healing for CYP-006TK compared to the standard of care control group • Phase 2 clinical trial in acute graft-versus-host disease (aGvHD) • recruitment now >40% complete, with the rate of recruitment substantially accelerating in recent months; primary results still anticipated late 2025 • Phase 1 clinical trial in kidney transplantation • first patient treated; completion of first cohort anticipated in Q1 2025 • Phase 3 clinical trial in osteoarthritis • all patients have completed study treatment; results expected in 1H 2026 • Balance sheet strengthened • $1.88m R&D Tax Incentive rebate • $8.10m institutional placement • Strong cash balance • $10.51m at end of quarter with forecast cash runway into mid 2026 5 December 2024 Quarter – Key Highlights
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CYP-006TK for Diabetic Foot Ulcers
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7 Diabetic foot ulcers (DFU) • Open sore or wound that develops in patients with diabetes • Very difficult to heal, which can lead to serious complications such as serious infection • 20% of patients who develop DFU will require an amputation1 • Multi-disciplinary team can be required to treat: GP, endocrinologist, podiatrist, wound care nurse, vascular surgeon and infectious disease specialist • Cost to treat DFU in the US can exceed US$60,000 per patient (depending on severity)2 • Annual costs to US public and private payers estimated to be US$9 – 13 billion per year2 Diabetes is the fastest growing public health concern worldwide4 ~38 million Americans have diabetes5 Up to 34% of those with diabetes will develop a foot ulcer1 20% of patients with DFU will require amputation of the foot or limb1 150,000+ amputations per year in the US due to DFU6 Estimated costs to US public and private payers US$9–13 billion per year2 1. McDermott et al. Diabetes Care. 46:209–221 (2023). 2. Raghav et al. Ther Adv Endocrinol Metab. 9(1) 29-31 (2018). 3. Hicks et al. J Vasc Surg. 67:1455-62 (2018). WIfI = Wound, Ischemia, and foot Infection3 4. Hossain et al. Health Sci Rep. 7(3):e2004 (2024). 5. American Diabetes Association: https://diabetes.org/about-diabetes/statistics/about-diabetes 6. American Diabetes Association: https://diabetes.org/advocacy/amputation-prevention-alliance
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8 Cynata’s MSC product for DFU • Cynata has developed a proprietary wound dressing using MSCs (“CYP-006TK”) • CYP-006TK utilises a proprietary surface-coating, optimised for the delivery of MSCs directly to the wound Cymerus MSCs Plasma polymer treated surface Wound dressing Apply active wound dressing
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9 DFU | Phase 1 clinical trial Indication Non-healing diabetic foot ulcers (DFU) Product CYP-006TK (novel silicone dressing seeded with Cymerus iPSC-derived MSCs) Study Design • Randomised controlled trial in ~30 adults • Patients randomised to receive either standard of care (SOC) or CYP-006TK for 4 weeks, followed by SOC • SOC treatment = current best practice as determined by investigator (e.g. conventional wound dressings etc) • Primary objective was safety; efficacy measures included wound healing, pain and quality of life Study Conduct • Clinical sites in Australia (Adelaide and Perth) • Patient enrolment complete (April 2024) • All patient visits complete (September 2024) Results Final results released in December 2024 For further information: https://clinicaltrials.gov/study/NCT05165628
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Safety and tolerability 10 Primary Objective Phase 1 clinical trial of CYP-006TK in DFU successfully achieved its primary objective: – safe and well-tolerated – no participants withdrew from the trial due to adverse events – no suspected serious adverse reactions were reported
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11 Change in wound surface area • Substantial mean reduction (improvement) in wound surface area at both 12 & 24 weeks, in both mm2 and percentage terms CYP-006TK • Mean increase (deterioration) in wound surface area at both 12 & 24 weeks, in mm 2 terms • Increase in mm2 terms combined with moderate reduction in percentage terms indicates that larger wounds were less likely to heal Standard of Care Reduction in wound size (Improvement) Increase in wound size (Deterioration) 12w 24w 12w 24w
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12 Larger wounds* (measuring >200 mm²) • Mean reduction in wound surface area was similar in larger wounds to when all wounds were included • Substantial improvement in large wounds is especially encouraging as larger DFU are more likely to lead to an amputation¹ CYP-006TK • Extent of mean increase (deterioration) was greater in larger wounds than when all wounds were included • Mean change by percentage was markedly worse in larger wounds than in all wounds Standard of Care Reduction in wound size (Improvement) Increase in wound size (Deterioration) * A total of 19 participants had wounds measuring <200 mm² at baseline (nine in the CYP-006TK group; ten in the control group) 1. Pickwell K, et al. Diabetes Care. 2015;38(5):852-7. 12w 24w 12w 24w
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Ongoing clinical trials - leveraging the unique potential of Cymerus MSCs
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14 aGvHD | Phase 2 clinical trial Indication High risk acute graft versus host disease (aGvHD)1 Product CYP-001 (Cymerus iPSC-derived MSCs for intravenous infusion) Study Design • Randomised, double-blind, placebo-controlled trial • ~60 adults (steroids + CYP-001 vs steroids + placebo) • Primary objective is to assess efficacy of CYP-001 based on Overall Response Rate at Day 28 Study Conduct • Conducted under IND from US FDA • Clinical sites in USA, Europe and Australia • First patient enrolled in March 2024; enrolment >40% complete2 • Aiming to complete patient enrolment in H1 2025 Results Results anticipated in H2 2025 (primary evaluation) 1. Trial is recruiting patients with High Risk newly diagnosed aGvHD (risk assessed based on refined Minnesota criteria), which means patients are not yet eligible to receive ruxolitinib. This is earlier in treatment pathway than completed Phase 1 trial, which was conducted in patients with steroid-resistant aGvHD. For further information see: https://clinicaltrials.gov/study/NCT05643638 2. As of December 2024 Quarterly Activity Report, released to ASX on 30 January 2025
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15 OA | Phase 3 clinical trial Indication Osteoarthritis (OA) of the knee (Kellgren-Lawrence Grade 2-3) Product CYP-004 (Cymerus iPSC-derived MSCs for intra-articular injection) Study Design • Randomised, double-blind placebo-controlled trial in ~320 adults1 • Each participant receives 3 injections over 12 months; follow-up of 24 months from first dose • Co-primary endpoints are reduction of knee symptoms and measure of cartilage loss Study Conduct • Trial conducted by University of Sydney, funded by Australian Government NHMRC grant, while Cynata retains commercial rights, with clinical centres in Sydney and Hobart • Patient enrolment complete (November 2023) • Patient treatment complete (November 2024) • Last patient last visit expected ~November 2025 Results • Results anticipated in H1 2026 1. Clinical trial entitled Stem Cells as a symptom – and strUcture-modifying Treatment for medial tibiofemoral OsteoaRthritis: a randomised placebo-controlled trial (SCUlpTOR). NHMRC: National Health and Medical Research Council For further information: https://www.anzctr.org.au/Trial/Registration/TrialReview.aspx?id=379726&isReview=true
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16 Kidney transplant | Phase 1/2 clinical trial Indication Prevention of kidney transplant rejection Product CYP-001 (Cymerus iPSC-derived MSCs for intravenous infusion) Study Design • ~16 patients to receive CYP-001 after kidney transplantation: cohort 1 (n=3); cohort 2 (n=3); cohort 3 (n=10) • Trial will evaluate safety (all cohorts) and efficacy of MSCs in facilitating reduction of calcineurin inhibitors (anti-rejection medication; Cohort 3) Study Conduct • Trial conducted and funded by Leiden University Medical Center (LUMC), Netherlands, while Cynata retains commercial rights • Patient enrolment commenced in Q4 2024, with first patient treatment completed in Dec 2024 Results Outcome of Cohort 1 anticipated in H1 2025
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Outlook and commercial potential
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JP Morgan BioWeek/Biotech Showcase San Francisco, January 2025 Company presentation and partnering meetings Advanced Therapies Congress London, March 2025 Company presentation and partnering meetings BIO International Boston, June 2025 Partnering meetings BIO Japan, RM Japan Yokohama, October 2025 Partnering meetings • Upcoming catalysts will accelerate and broaden partnering discussions • We attend leading conferences in our sector, to tell our story and open new discussions • Following on from multiple events earlier this year, selected key events going forward include: • We will also attend further key events in the sector (ARM, ISCT, ISSCR) and in the regions 18 Industry connections
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19 Important new publication https://doi.org/10.1038/s41536- 024-00382- y
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20 Upcoming catalysts* DFU results announced Dec 2024; results from THREE further trials expected by 1H 2026 * Timing of events is approximate, and subject to changebased on the Company’s information as at the date of this presentation, Results Results Start of enrolment Q1 2025 Q2 2025 Q3 2025 Q4 2025 Q1 2026 Q2 2026 Phase 1/2 kidney transplant Phase 3 osteo- arthritis Phase 2 aGvHD Phase 1 DFU Results announced – Dec 2024 Enrolment complete Results Results Results (Cohort 1)
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Contact Us Cynata Therapeutics Limited Level 3, 100 Cubitt Street Cremorne Victoria 3121 Australia info@cynata.com www.cynata.com cynatatherapeutics @cynatastemcells cynata-therapeutics