Mesoblast Limited · ASX:MSB
Mesoblast — Shareholder Meeting (October 2015)
37 slides from Mesoblast's shareholder meeting presentation, Oct 22, 2015
Slide 1: Annual General Meeting
For personal use only Annual General Meeting Melbourne 22 October 2015
Slide 2: Cautionary Note Regarding Forward-Looking Statements
For personal use only CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation, including any comments made during or following the presentation, may contain forward-looking statements that are inherently subject to risks and uncertainties, some of which cannot be predicted or quantifi
Slide 3: Our Competitive Strengths
Our Competitive Strengths For personal use only 1 Disruptive platform based on allogeneic, “off-the-shelf” adult stem cells 2 Five product candidates that are in active Phase 3 programs or are Phase 3-ready 3 Substantial intellectual property covering products, uses, and manufacturing 4 Scalable man
Slide 4: Financials
Financials For personal use only Total shares outstanding (30 June 2015) ~337.0 million Issued shares in FY15 (including 4.5% equity stake to Celgene ~15.4 million Corporation) Current share price (market close, 21 October 2015) A$3.40 Market capitalization (market close, 21 October 2015) ~A$1.15 bi
Slide 5: Lead Product Candidates and Achievements over past months
Lead Product Candidates and Achievements over past 12 months MSC-100-IV/TEMCELL® HS Inj. - Acute Graft Versus Host Disease (aGVHD) For personal use only Our licensee, JCR Pharmaceuticals Co. Ltd., received full approval from the Japanese Ministry of Health, Labour and Welfare for TEMCELL® HS Inj.,
Slide 6: Lead Product Candidates and Achievements over past months (2)
Lead Product Candidates and Achievements over past 12 months (2) Biologic Refractory Rheumatoid Arthritis (RA) - 1st cohort fully For personal use only enrolled, 2nd cohort actively recruiting Anti-Inflammatory Portfolio - a Major Phase 2a trial in diabetic kidney disease demonstrated Emerging p
Slide 7: Disruptive Technology Platform
1 Disruptive Technology Platform For personal use only Multiple, Diverse Mechanisms Allogeneic Use MLCs from a single healthy donor can be Mesenchymal Lineage Adult Stem expanded to thousands of doses Cells (MLCs), immunoselected precursors and progeny Isolation from diverse tissue sources (bo
Slide 8: MLC Product Development Strategy
1 MLC Product Development Strategy For personal use only Multiple mechanisms of action may Proprietary manufacturing processes facilitate therapeutic effects in diseases utilized to create distinct product with high unmet needs candidates with specific technical attributes Independent, Distinct Prod
Slide 9: Five product candidates in active Phase 3 programs or Phase 3-ready
2 Five product candidates in active Phase 3 programs or Phase 3-ready For personal use only A B C D 9
Slide 10: Extensive Intellectual Property Portfolio
3 Extensive Intellectual Property Portfolio – 661 patents across 72 families# For personal use only Composition of matter (COM) Specific therapeutic Complementary technologies and manufacturing process applications and additional candidates 141 patents or patent applications 382 patents or patent ap
Slide 11: Scalable Manufacturing Capabilities: Partnership with Lonza
4 Scalable Manufacturing Capabilities: Partnership with Lonza For personal use only Manufacturing objectives Distinct manufacturing processes for each product Commercial scale processes with batch-to-batch consistency and reproducible release criteria Ensure commercial product supply is aligne
Slide 12: Update on Scalable Manufacturing Capabilities and Strategy
pdate on Scalable Manufacturing Capabilities and Strategy 4 Significant Progress in Manufacturing For personal use only 1 2 3 4 5 Key stages in manufacturing and distribution Procure bone Create master Expand to Formulate, Distribute process marrow from cell banks therapeutic package and healthy, yo
Slide 13: Strategic Collaborations with Industry Leaders
5 Strategic Collaborations with Industry Leaders For personal use only Manufacturing Development Commercialization Clinical Heart CHF program – development failure MPC-150-IM Global rights Additional CV & CNS Manufacturing programs R&D aGVHD Japan aGVHD rights Japan Japan Manufacture
Slide 14: Portfolio Overview
14 For personal use only Portfolio Overview
Slide 15: MSC-100-IV / TEMCELL HS Inj.: Acute GVHD Market Opportunity
A MSC-100-IV / TEMCELL® HS Inj. : Acute Graft vs Host Disease – Market Opportunity MSC-100-IV / TEMCELL® HS Inj. is targeting pediatric and adult patients with acute Graft Versus Host Disease For personal use only (aGVHD) following allogeneic Bone Marrow Transplant (BMT). ~30,000 allogeneic BMTs p
Slide 16: MSC-100-IV: Phase 2 Trial in Children with SR-aGVHD
A MSC-100-IV: Phase 3 Trial in Children with Steroid Refractory Acute Graft vs Host Disease (SR-aGVHD) For personal use only MSC-100-IV in Children with SR-aGVHD who failed MSC-100-IV as first line therapy in children with multiple other modalities SR-aGVHD (n = 102) Open-label (n = 58) Response Ran
Slide 17: MSC-100-IV / TEMCELL HS Inj.: Product Launch Plans in aGVHD
A MSC-100-IV / TEMCELL® HS Inj. : Product Launch Plans in aGVHD Japan (TEMCELL® HS Inj.): For personal use only • Our licensee, JCR Pharmaceuticals Co. Ltd., received full approval from the Japanese Ministry of Health, Labour and Welfare for TEMCELL® HS Inj., the first industrially manufactured stem
Slide 18: MPC-150-IM: Congestive Heart Failure Market Opportunity
B MPC-150-IM: Congestive Heart Failure (CHF) – Market Opportunity MPC-150-IM is in development for patients with New York Heart Association Class II-IV CHF For personal use only 5.7m patients (2% of the population) diagnosed with CHF in the US1 870,000 new cases diagnosed in the US each year1 Ma
Slide 19: MPC-150-IM: Phase 2 Trial Results in CHF Identifies Optimal Dose
B MPC-150-IM: Phase 2 Trial Results in CHF Identifies Optimal Therapeutic Dose For personal use only Phase 2, randomised placebo controlled trial in 60 patients with Class II / III CHF and LVEF<40% – Placebo vs. 25, 75, 150 M MPCs injected by endomyocardial catheter – All doses were feasible and s
Slide 20: MPC-150-IM: Phase 2 Benefit on LV Remodeling in Advanced Heart Failure
B MPC-150-IM: Phase 2 Therapeutic Benefit on LV Remodeling is Evident in Patients with Advanced Heart Failure1 with LVESV >100ml 1 For personal use only Baseline LVESV > 100 mL Baseline LVESV > 100 mL Baseline LVESV > 100ml P < 0.02 (Month 6 – Baseline; mL) P < 0.05 (Month 6 – Baseline; mL) P < 0.03
Slide 21: MPC-150-IM: Phase 2 Benefit on LV Remodelling in Advanced Heart Failure
B MPC-150-IM: Phase 2 Therapeutic Benefit on LV remodelling is evident in Patients with Advanced Heart Failure For personal use only All of the HF-MACE seen over 36 months in the Phase 2 trial occurred in control patients with baseline LVESV > 100 ml; the annualized HF-MACE rate was 24% in this gr
Slide 22: MPC-150-IM: Phase 3 Pathway to Potential Approval
B MPC-150-IM: Phase 3 Pathway to Potential Approval in Patients with Advanced Heart Failure For personal use only An ongoing Phase 3 trial, using a time-to-first-event analysis of HF-MACE as the primary endpoint, is being conducted in approximately 1,165 patients, down from 1,730 following FDA agr
Slide 23: MPC-06-ID: Chronic Low Back Pain Market Opportunity
C MPC-06-ID: Chronic Low Back Pain due to Degenerative Disc Disease – Market Opportunity MPC-06-ID is in development for the treatment of chronic low back pain (CLBP) lasting >6 For personal use only months as a result of moderate degenerative intervertebral disc disease Over 5.7m patients in the
Slide 24: MPC-06-ID: 12 Month Phase 2 Trial Design
C MPC-06-ID: 12 Month Phase 2 Trial Design For personal use only 100 patients with >6 months of CLBP due to DDD and unresponsive to conservative therapies were evaluated in a randomized, placebo controlled trial Visual Analog Scale (VAS) scored from 0-100 evaluated at 1,3, 6 and 12 months – Mini
Slide 25: MPC-06-ID: Pain and Function Responder Rates at Both 6 & 12 Months
C MPC-06-ID: Pain and Function Responder Rates at Both 6 & 12 Months – Phase 2 Data For personal use only Pain Responders at Both 6 & 12 Months Functional Responder at Both 6 & 12 Months 70.0% 70.0% 59.3%a,b 60.0% 60.0% 50.0%x 48.3%y 50.0% 50.0% 44.8%c % Responder Rate % Responder Rate 40.0% 40.0% 3
Slide 26: MPC-06-ID: Composite Endpoint for Pain and Function over 24 Months
C MPC-06-ID: Composite Endpoint for both Pain and Function over 24 Months – Phase 2 Data For personal use only % Patients with treatment success % Patients with treatment success at 6 & 12 months at 12 & 24 months 50.0% 50.0% 44.4%a,b 45.0% 45.0% 40.0% 37.9%c 40.0% 37.0%d 35.0% 35.0% 32.1% % Respond
Slide 27: MPC-06-ID: Phase 3 Clinical Study Design
C Phase 3 Clinical Study Design For personal use only Initiated a Phase 3 program consisting of two confirmatory double-blinded, placebo controlled Phase 3 clinical trials ~330 patients per study Efficacy endpoints: Consistent with approach for approval of spinal device technologies Subjects mee
Slide 28: Anti-Inflammatory Portfolio - a Major Emerging Opportunity
Anti-Inflammatory Portfolio - a Major Emerging Opportunity For personal use only Mesenchymal Lineage Cells (MLCs*) have receptors that respond to pro- inflammatory signals, inducing release of multiple anti inflammatory mediators MLCs thereby target multiple immune pathways concurrently This s
Slide 29: MPC-300-IV for Treatment of Chronic Inflammatory Diseases
D MPC-300-IV for Treatment of Chronic Inflammatory Diseases Inflammation-dependent induction in MPC and role in regulating the function For personal use only of both Th17 cells and macrophages IL-6 e.g., , IDO e.g., , IDO 29
Slide 30: MPC-300-IV for Treatment of Chronic Inflammatory Diseases
D MPC-300-IV for Treatment of Chronic Inflammatory Diseases Biologic-refractory rheumatoid arthritis, 48 patients – Ongoing For personal use only – Randomized, placebo controlled, dose-escalating study – First dose / cohort 1 is fully enrolled – 6 month topline data expected Q4 2015 Type 2 Diabe
Slide 31: MPC-300-IV: Biologic Refractory Rheumatoid Arthritis Market Opportunity
D MPC-300-IV: Biologic Refractory Rheumatoid Arthritis (RA) – Market Opportunity Ongoing randomized, controlled Phase 2 Trial in 48 patients with biologic refractory rheumatoid For personal use only arthritis, comparing two doses of MPC-300-IV against placebo 1.7m patients with RA in the US1 Inc
Slide 32: MPC-300-IV: Diabetic Nephropathy Market Opportunity
D MPC-300-IV: Diabetic Nephropathy (DN) – Market Opportunity ~7.1 million patients with DN in the US1 For personal use only ~1.96 million patients with Stage 3b-4 (GFR 20-45 ml/min / 1.73 m2) DN3,4 Market opportunity ~200,000 incident cases per year with Stage 3b-42 Despite standard of care,
Slide 33: MPC-300-IV: Diabetic Nephropathy Phase 2 Trial Design
D MPC-300-IV: Diabetic – Phase 2 Diabetic Nephropathy Trial Design For personal use only 2012 joint workshop held by FDA and National Kidney Foundation concluded that time to 30-40% decline in GFR may be an acceptable primary endpoint for evaluating potential benefits of new therapies in advanced
Slide 34: MPC-300-IV: Diabetic Kidney Disease Phase 2 Results - Improvement in GFR at 24 weeks
D MPC-300-IV: Diabetic Kidney Disease Phase 2 Results – Improvement in GFR at 24 weeks For personal use only W e e k 2 4 e G F R C h a n g e f r o m B a s e lin e W e e k 2 4 e G F R C h a n g e f r o m B a s e lin e 2 A ll S u b je c t s B a s e lin e e G F R > 3 0 m l/ m in / 1 .7 3 m 6 6 P la c e
Slide 35: MPC-300-IV: Diabetic Kidney Disease Phase 2 Results - IL-6 Effects
D MPC-300-IV: Diabetic Kidney Disease Phase 2 Results – Treatment Related Effects on the Inflammatory Marker IL-6 and Therapeutic Response in Patients with High Baseline IL-6 Levels For personal use only MPC by Dose Group Pooled MPC B a s e lin e a t W e e k 1 2 4 Median (IQR) at Baseline: 3.4 (2.8,
Slide 36: Upcoming Milestones by Calendar Year
Upcoming Milestones by Calendar Year '15 2016 2017 2018 For personal use only Product Candidate Programs Anticipated Milestones Q4 Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 H1 H2 Temcell® HS Inj. Launch in Japan MSC-100-IV / Acute Graft Versus Host US Pediatric Phase 3 top line Results Temcell® HS Inj. Disease Interi
Slide 37: Our Competitive Strengths
Our Competitive Strengths For personal use only 1 Disruptive platform based on allogeneic, “off-the-shelf” adult stem cells 2 Five product candidates that are in active Phase 3 programs or are Phase 3-ready 3 Substantial intellectual property covering products, uses, and manufacturing 4 Scalable man