Cynata Therapeutics Limited · ASX:CYP
Cynata Therapeutics — Non-Deal Roadshow (November 2024)
40 slides from Cynata Therapeutics's roadshow presentation, Nov 12, 2024
Slide 1: ASX Announcement: Updated Investor Presentation
ASX ANNOUNCEMENT 12 November 2024 Updated Investor Presentation Melbourne, Australia; 12 November 2024: Cynata Therapeutics Limited (ASX: “CYP”, “Cynata”, or the “Company”), a clinical-stage biotechnology company specialising in cell therapeutics, is pleased to release an updated investor presentati
Slide 2: Pioneering the Next Generation of Cellular Therapies
For personal use only A Clinical Stage Company Pioneering the Next Generation of Cellular Therapies Investor Presentation 12 November 2024
Slide 3: Important information
Important information Summary information This Presentation contains summary information about Cynata Therapeutics Limited and its subsidiaries Forward-looking statements (CYP, or Cynata) which is current as at 12 November 2024. This Presentation should be read in This Presentation contains certain
Slide 4: Corporate overview
Corporate overview Shareholder distribution Largest shareholders 13.1% For personal use only 13% 25% Bioscience Managers is an international healthcare investment firm Bioscience Managers 10% headquarter in Melbourne that finances and enables innovative Fidelity science and technology with the poten
Slide 5: Target indications
Target indications Indication Trial phase Upcoming catalysts* Market opportunity For personal use only Acute Graft vs Host Disease Enrolment completion – Q4 2024 (aGvHD) Phase 2 US$600m1 ongoing Results – 2H 2025 FDA Orphan Designation Cynata Funded & Managed Phase 1 Diabetic Foot Ulcers (DFU) ongoi
Slide 6: Introduction to MSCs
Introduction to MSCs What are Mesenchymal Stem Cells (MSCs)?1 • MSCs are cells that naturally occur in the human body, which have key unique properties which make them ideal for potentially treating various diseases: For personal use only • Anti-inflammatory properties – important as many diseases a
Slide 7: Why MSCs?
Why MSCs? Mesenchymal Stem Cells For personal use only Key Unique Properties¹ 1 2 3 Tissue Repair & Anti-Inflammatory Immunomodulatory Regeneration Importance: Inflammation and inappropriate immune responses contribute to many diseases/medical disorders, and often lead to tissue damage. Consequently
Slide 8: Conventional MSC manufacturing process
Conventional MSC manufacturing process Isolation of Each donor Culture expansion of MSCs Many donors MSCs from Patients provides cells from each donation each donation Donor 1 Donor 2 For personal use only Donor 3 Donor 4 Standard Donor x Process¹ MSCs must be isolated New donors must be Extensive c
Slide 9: The problem
The problem Traditional manufacturing methods used to produce MSCs can encounter the following challenges: Consistency issues Inconsistent MSCs produce inconsistent & unreliable results For personal use only Potency issues Different potency levels in MSCs produce inconsistent & unreliable results Sc
Slide 10: The solution: Cynata's iPSC-based Cymerus manufacturing platform
For personal use only The solution: Cynata’s revolutionary iPSC-based Cymerus manufacturing platform
Slide 11: Cymerus process
Cymerus process One iPSC Vial of iPSCs iPSC expansion Formation of Patients donor bank from bank and differentiation MSCs For personal use only One donor Cymerus Process iPSCs are culture Blood donation from a Cells from same iPSC expanded, then turned All batches of Cymerus single donor was used ba
Slide 12: Summary of Manufacturing Challenges
Summary of Manufacturing Challenges High-level comparison of manufacturing challenges for producing MSCs Traditional MSC Manufacturing Process For personal use only Traditional Cymerus MSCs Platform Conventional donor-derived MSCs are difficult to produce at scale, and since they are derived from di
Slide 13: Current State of Play
Current State of Play For personal use only Demonstrated Opportunity Traditional MSCs can be Test the Confirm MSCs have manufacturing manufactured potential of consistent demonstrated = inconsistent consistently consistent MSCs can huge potential MSCs now at scale MSCs treat new diseases Cymerus Pro
Slide 14: Graft Versus Host Disease (aGvHD)
For personal use only Graft Versus Host Disease (aGvHD) An opportunity based on Compelling clinical data
Slide 15: Bone marrow transplants & GvHD
Bone marrow transplants & GvHD Bone marrow transplant (also known as blood stem cell transplant) • The procedure replaces blood stem cells in people whose bone marrow has been For personal use only destroyed by large doses of chemotherapy or radiotherapy • Bone marrow transplants can be curative for
Slide 16: Acute graft versus host disease (aGvHD)
Acute graft versus host disease (aGvHD) >38,000 Up to 9,500 ~35-50% Almost all 2-year survival allogeneic <50% respond steroid- develop receive rate in SR- For personal use only transplants* to steroids4 resistant cases aGvHD2,3 steroids aGvHD: <20%5 per year1 per year * “Allogeneic” means cells com
Slide 17: Phase 1 clinical trial – results (aGvHD)
Phase 1 clinical trial – results (aGvHD) Complete 53% Overall 87% 2yr 60% response response survival For personal use only (8 out of 15 showed (13 out of 15 improved (9 out of 15 alive no signs of aGvHD) by at least 1 Grade) after 2 years) Outstanding response rates and overall survival Sustained ou
Slide 18: CYP-001 vs other treatments in SR-aGvHD
CYP-001 vs other treatments in SR-aGvHD Overall Response Overall Survival Overall Response Day 28 Day 56-601 18 months • Between Day 28 and Day 56-60, the For personal use only Overall Response Rate (ORR) for 80% both RUX and BAT decreased ▲6% markedly, while the ORR for CYP-001 70% marginally incre
Slide 19: Scientific and regulatory recognition
Scientific and regulatory recognition Scientific: Publications For personal use only • Cynata was published in two editions of the highly prestigious Nature Medicine Journal following its Phase I trial results Cynata Featured on front-page of Nature Regulatory: Orphan Drug Designation Benefits inclu
Slide 20: aGvHD | Phase 2 clinical trial
aGvHD | Phase 2 clinical trial Indication High risk acute graft versus host disease (aGvHD)1 For personal use only Product CYP-001 (Cymerus iPSC-derived MSCs for intravenous infusion) • Randomised, double-blind, placebo-controlled trial Study Design • ~60 adults (steroids + CYP-001 vs steroids + pla
Slide 21: CYP-006TK for Diabetic Foot Ulcers
For personal use only CYP-006TK for Diabetic Foot Ulcers
Slide 22: Diabetic foot ulcers (DFU)
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 For personal use only • 20% of patients who develop DFU will require an amputation1 • Multi-disciplinary team can be req
Slide 23: Diabetic foot ulcer examples
Diabetic foot ulcer examples For personal use only 22
Slide 24: MSCs in DFU
MSCs in DFU MSCs have demonstrated strong success in pre-clinical DFU models Key findings 100 Silicone patch seeded with MSCs from various sources in preclinical model of diabetic wounds For personal use only 90 86% 91% • Primary outcome measured was extent of wound surface Percentage re-epitheliali
Slide 25: Cynata's MSC product for DFU
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 For personal use only Plasma polymer Cymerus MSCs treated surface Apply active Wound
Slide 26: DFU | Phase 1 clinical trial
DFU | Phase 1 clinical trial Indication Non-healing diabetic foot ulcers (DFU) For personal use only CYP-006TK (Novel silicone dressing seeded with Cymerus iPSC-derived MSCs) Product • Randomised controlled trial in ~30 adults • Patients randomised to receive either standard of care or CYP-006TK for
Slide 27: DFU | timeline
DFU | timeline Product: CYP-006TK Phase I Study Timeline Mar 22 Feb 24 Apr 24 Q4 24 / Q1 25 For personal use only Enrolment Interim Recruitment Final Commences Completes Results¹ Results Interim Results • 16 patients reviewed after 10 weeks’: Day 0 Day 28 • 8 Standard of Care (SoC) • 8 CYP-006TK • M
Slide 28: Outlook and commercial potential
For personal use only Outlook and commercial potential
Slide 29: Upcoming catalysts
Upcoming catalysts* Results of FOUR clinical trials expected between late 2024 and early 2026 Phase 2 For personal use only Enrolment Results aGvHD complete Phase 1 Results DFU Phase 3 Results osteo- Results arthritis Results Phase 1/2 First Results kidney patient (Cohort 1) transplant enrolled Star
Slide 30: Commercial attractiveness
Commercial attractiveness Proprietary Platform For personal use only • Ability to produce MSCs consistently and at scale allows for MSCs to be used in Technology multiple indications = Platform Technology appeal Platform Technology • Platform Technology allows CYP to target multiple multi-billion do
Slide 31: Summary
Summary For personal use only Platform Compelling Billion dollar Technology Clinical Data markets Proven Multiple Commercial Indications Interest Manufacturing Excellent Safety Numerous Near- Challenges Profile Term Catalysts Overcome 30
Slide 32: Contact Us
For personal use only Contact Us Cynata Therapeutics Limited Level 3, 100 Cubitt Street Cremorne Victoria 3121 Australia cynatatherapeutics info@cynata.com @cynatastemcells www.cynata.com cynata-therapeutics
Slide 33: Annexure
For personal use only Annexure
Slide 34: Board & senior management
Board & senior management Highly skilled and experienced senior leadership team with decades of experience Dr Kilian Kelly Dr Geoff Brooke Chief Executive Officer & For personal use only Independent Non-Executive Chair Managing Director • 30+ years' experience in the healthcare • 20+ years' experien
Slide 35: Advantages of iPSC-based platform
Advantages of iPSC-based platform Induced pluripotent stem cells (iPSCs): For personal use only • Mature adult cells reprogrammed to become pluripotent, which means: • Effectively limitless proliferation capacity • Potential to differentiate into any adult cell type (including MSCs) • Similar proper
Slide 36: MSC mechanisms of action
MSC mechanisms of action MSCs may exert many effects on target cells via diverse potentially-overlapping mechanisms. For personal use only Target cells include (i) donor and host immune cells, including T cells, B cells, NK cells, monocytes and dendritic cells; and (ii) host cells susceptible to dam
Slide 37: Phase 2 trial of CYP-001 in aGvHD
Phase 2 trial of CYP-001 in aGvHD Although ruxolitinib is approved for SR-aGvHD, there is a substantial unmet need for a safer and more effective Safer and more treatment option: For personal use only effective treatments for aGvHD • Phase 1 results suggest that Cymerus MSCs could achieve better res
Slide 38: Research partnerships
Research partnerships PLATFORM POTENTIAL OF CYNATA Studies conducted in partnership with leading Large body of positive preclinical data generated research groups worldwide via R&D partnerships: For personal use only • GvHD • Diabetic wounds • Critical limb ischaemia • Organ transplant rejection • O
Slide 39: OA | Phase 3 clinical trial
OA | Phase 3 clinical trial Indication Osteoarthritis (OA) of the knee (Kellgren-Lawrence Grade 2-3) For personal use only Product CYP-004 (Cymerus iPSC-derived MSCs for intra-articular injection) • Randomised, double-blind placebo-controlled trial in ~320 adults1 Study Design • Each participant rec
Slide 40: Kidney transplant | Phase 1/2 clinical trial
Kidney transplant | Phase 1/2 clinical trial Indication Prevention of kidney transplant rejection For personal use only Product CYP-001 (Cymerus iPSC-derived MSCs for intravenous infusion) • ~16 patients to receive CYP-001 after kidney transplantation: cohort 1 (n=3); cohort 2 (n=3); cohort 3 (n=10)
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