Mesoblast Limited · ASX:MSB
Mesoblast — Shareholder Meeting (November 2014)
34 slides from Mesoblast's shareholder meeting presentation, Nov 25, 2014
Slide 1: Annual General Meeting
For personal use only Annual General Meeting Melbourne 25 November 2014
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: Shareholder value and ownership
Shareholder value and ownership For personal use only Market valuation Issued shares (30 September 2014) ~321 million Current share price (market close, 24 November 2014) A$4.14 Market capitalization ~A$1.32 billion Cash reserves (30 September 2014) A$170.5 million Shareholder ownership (30 Septembe
Slide 4: Significant achievements to date
Significant achievements to date For personal use only Successful translation of Mesenchymal Lineage Adult Stem Cell (MLC) based technology platforms for multiple indications Portfolio of allogeneic regenerative medicine product candidates, with five in Phase 3 trials/Phase 3 ready Well charac
Slide 5: 2014 FY highlights
2014 FY highlights Commencement of Phase 3 trial of Mesenchymal Precursor Cell (MPC) product candidate MPC- For personal use only 150-IM in NYHA class II/III heart failure; actively recruiting across multiple North American sites NIH and MSB agree on 120 patient MPC-150-IM trial in advanced / NY
Slide 6: New framework for expedited Japanese approval of regenerative medical products
New framework for expedited Japanese approval of regenerative medical products For personal use only The Pharmaceuticals, Medical Devices and Other Therapeutic Legislative framework* Products Act (PMD Act) takes effect 25 November 2014 in Japan Establishes a framework for expedited approval in J
Slide 7: Objectives and Strategy for Japan
Objectives and Strategy for Japan For personal use only Objectives To capitalize on clinical data generated to date, our strong intellectual property portfolio, and manufacturing know how for accelerated product regulatory approvals in Japan To generate nearer term revenues in the world’s second
Slide 8: Our competitive strengths
Our competitive strengths For personal use only 1 Disruptive technology platform 2 Broad portfolio of distinct late-stage product candidates 3 Scalable manufacturing capabilities 4 Intellectual property leadership 5 Strategic alliances 6 Experienced management team 7
Slide 9: 1. Disruptive technology platform
1. Disruptive technology platform For personal use only Mesenchymal Lineage Adult Stem Cell (MLCs) technology platform – Highly purified, immunoselected Stro-1/Stro-3 positive Mesenchymal Precursor Cells (MPCs), earliest precursors – Culture-expanded Mesenchymal Stem Cells (MSCs), progeny of earli
Slide 10: Focus on bringing late stage products to market
Focus on bringing late stage products to market For personal use only Products with mid term revenue opportunity MPC-06-ID: Chronic Discogenic Low Back Pain MPC-150-IM: Heart Failure MSC-100-IV: GVHD Robust Development Portfolio Progressing to next decision point Targeted for partnership
Slide 11: 2. Broad portfolio of distinct late-stage clinical product candidates
2. Broad portfolio of distinct late-stage clinical product candidates Product Filed for For personal use only Candidates Programs Preclinical Phase 2 Phase 3 approval Tier I MPC-06-ID Chronic Discogenic Low Back Pain (CDLBP) Class II/III Congestive Heart Failure (CHF) MPC-150-IM Class IV CHF M
Slide 12: MPC-06-ID: Chronic Discogenic Low Back Pain – Market opportunity
MPC-06-ID: Chronic Discogenic Low Back Pain (CDLBP) – Market opportunity MPC-06-ID is in development for the treatment of chronic discogenic low back pain (CDLBP) lasting >6 months For personal use only as a result of moderate degenerative intervertebral disc disease Market opportunity Gap in treatm
Slide 13: MPC-06-ID: CDLBP 12-month Phase 2 Results - greater reduction in pain
MPC-06-ID: CDLBP 12-month Phase 2 Results - MPCs show greater reduction in pain Phase 2 trial: 100 patients with >6 months of discogenic low back pain failing other therapies were For personal use only evaluated in a randomized, placebo controlled trial that compared saline, HA, HA + 6 million MPC,
Slide 14: MPC-06-ID: CDLBP 12-month Phase 2 results - greater reduction in pain
MPC-06-ID: CDLBP 12-month Phase 2 results - MPCs show greater reduction in pain MPC groups have a greater proportion of patients with at least a 50% improvement For personal use only in back pain or minimal/no residual back pain (VAS <20) at 12 months relative to controls Proportion of patients with
Slide 15: MPC-06-ID: CDLBP Phase 2 results - improvement in function
MPC-06-ID: CDLBP 12 month Phase 2 results - MPCs show improvement in function For personal use only • Oswestry Disability Index (ODI) is a % of patients with improvement in ODI at 12 months standardized measure of function and 90% was evaluated at 3, 6 and 12 months 80% MCID 70% • Minimally clinical
Slide 16: MPC-06-ID: CDLBP – Composite endpoint for Phase 3
MPC-06-ID: CDLBP – Composite endpoint for Phase 3 Treatment options for CDLBP must address both decreased pain and improved function, defined as MCID in For personal use only both VAS and ODI 1. A responder to treatment is defined as meeting both VAS and ODI MCID. % Responders positive at both 6 and
Slide 17: MPC-06-ID: CDLBP - Phase 3 clinical program
MPC-06-ID: CDLBP - Phase 3 clinical program For personal use only Positive End of Phase 2 meeting with FDA supports progression into Phase 3 Phase 3 study design will measure composite endpoint of decreased pain and improved function over 12 months Expected Phase 3 trial initiation end of 2014
Slide 18: MPC-150-IM: Congestive Heart Failure (CHF) – Market opportunity
MPC-150-IM: Congestive Heart Failure (CHF) – Market opportunity For personal use only MPC-150-IM is in development for New York Heart Association (NYHA) class II to IV congestive heart failure Market opportunity Gap in treatment options Targeted physician population 5.1 million people in the US ar
Slide 19: MPC-150-IM CHF - Optimal dose identified in Phase 2
MPC-150-IM Congestive Heart Failure (CHF)- Optimal dose identified in Phase 2 Phase 2 top line results For personal use only Overall, results obtained in patients with CHF show positive effects on clinical outcomes, including left ventricular remodeling and functional exercise capacity. The 150 mill
Slide 20: MPC-150-IM: CHF – Phase 3 trial in NYHA Class II/III HF
MPC-150-IM: MPC-150-IM: Congestive Congestive Heart Heart Failure Failure (CHF) (CHF) – Phase – in 3 trial Phase NYHA2II/III Results HF Class II/III HF For personal use only MPC-150-IM: Congestive • Phase 3 trial is being conductedHeart by our Failure (CHF) partner Teva and is– actively Phaseenrolli
Slide 21: MPC-150-IM: Congestive Heart Failure (CHF) – Trials underway
MPC-150-IM: Congestive Heart Failure (CHF) – Trials underway For personal use only New York Heart Association (NYHA) classification of heart failure NYHA Class I NYHA NYHA Class II NYHA Class III Class 32% 29% 30% IV 9% Phase 3 CHF TEVA NIH LVAD Trial 1730 patients 120 patients NYHA Class II/III P
Slide 22: MSC-100-IV: Graft vs Host Disease – Market opportunity
MSC-100-IV: Graft vs Host Disease – Market opportunity MSC-100-IV is in development for pediatric and adult patients with acute Graft Versus Host Disease (GVHD) For personal use only following allogeneic hematopoietic stem cell transplant (HSCT) who have failed to respond to steroid treatment Market
Slide 23: MSC-100-IV: Graft vs Host Disease – Evidence of efficacy
MSC-100-IV: Graft vs Host Disease – Evidence of efficacy For personal use only Pediatric Patients 1 Adult Patients 2 - In a randomized, placebo-controlled Phase 3 trial, MSC-100- - Expanded Access Program in US has treated in excess of 200 IV significantly improved overall responses in the adult sub
Slide 24: MSC-100-IV/JR-031: Graft vs Host Disease – Product launch plans
MSC-100-IV/JR-031 : Graft vs Host Disease – Product launch plans For personal use only • JCR Pharmaceuticals Co Ltd., our GVHD partner in Japan, filed for regulatory approval for JR-031 in Japan in September 2014 and was granted orphan drug priority review • North America • Canada • Manufacturing -C
Slide 25: MPC-300-IV for treatment of Chronic Inflammatory Diseases
MPC-300-IV for treatment of Chronic Inflammatory Diseases For personal use only eg , IDO eg , IDO 24
Slide 26: MPC-300-IV for treatment of Chronic Inflammatory Diseases
MPC-300-IV for treatment of Chronic Inflammatory Diseases For personal use only Phase 2 clinical program in 138 patients across three chronic inflammatory conditions to evaluate safety, efficacy and durability of effect following IV administration of a single MPC- 300-IV dose 60 patients, type 2 d
Slide 27: MPC-300-IV for Diabetic Kidney Disease – Market opportunity
MPC-300-10-IV for Diabetic Kidney Disease – Market opportunity Targeted physician population For personal use only Market opportunity Gap in treatment options • 40-50% of all diabetes patients (19 • Standard of care (SOC) Renin • Patients with Stages 3B-4 are million in 2013 in US) develop Angiotens
Slide 28: MPC-300-IV: Biologic Refractory Rheumatoid Arthritis – Market opportunity
MPC-300-IV: Biologic Refractory Rheumatoid Arthritis– Market opportunity For personal use only Market Opportunity Gap in Treatment Options Targeted Physician Population 1.3M Americans afflicted with RA1 Despite standard of care using Rheumatologists pharmacological treatments – General interni
Slide 29: 3. Scalable manufacturing capabilities to enable commercial scale production
3. Scalable manufacturing capabilities to enable commercial scale production 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, young quantities cryopr
Slide 30: 4. Extensive intellectual property portfolio
4. Extensive intellectual property portfolio covering over 60 patent families For personal use only Composition of matter and Specific therapeutic Complementary technologies manufacturing process applications and additional candidates 139 patents or patent applications 337 patents or patent applicat
Slide 31: 5. Build strategic partnerships
5. Build strategic partnerships For personal use only Manufacturing Development Commercialization Heart Quintiles Failure Teva EDB GVHD MSB MSB Japan JCR Lonza Back Pain NIH Partner Options 30
Slide 32: 6. Experienced management team
6. Experienced management team Location For personal use only Industry background Qualification level Function 31 Building an experienced team for late-phase clinical execution and market launch
Slide 33: Upcoming milestones
Upcoming milestones Product 2014 2015 2016 2017 2018 candidates Programs Anticipated milestones For personal use only Phase 3 IND clearance in Q4 2014 MPC-06-ID Chronic discogenic Phase 3 enrollment complete in low back pain, or mid-2016 CDLBP Phase 3 interim analysis mid-2016 Phase 3 top li
Slide 34: Questions / Thank you
For personal use only Questions / Thank you 33