Mesoblast Limited · ASX:MSB
Mesoblast — 34th Annual J.P. Morgan Healthcare Conference (January 2016)
32 slides from Mesoblast's conference presentation, Jan 14, 2016
Slide 1: Mesoblast - A Global Leader in Cell Based Medicines
For personal use only Mesoblast -‐ A Global Leader in Cell Based Medicines 34th Annual J.P. Morgan Healthcare Conference January 2016
Slide 2: Cautionary Note Regarding Forward-Looking Statements
For personal use only CAUTIONARY NOTE REGARDING FORWARD-‐LOOKING STATEMENTS This presenta3on includes forward-‐looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertain3es and other factors that may cause our actual results, l
Slide 3: Key Investment Highlights
Key Investment Highlights For personal use only 1 DisrupAve technology plaBorm: proprietary, allogeneic, “off-‐the-‐shelf” adult stem cells with predictable therapeuAc properAes 2 Established late stage porBolio of disAnct and advanced product candidates Strategic partnerships delivering clinical,
Slide 4: Focused Company With Strong Cash Reserves to Meet Key Corporate Objectives
Focused Company With Strong Cash Reserves to Meet Key Corporate ObjecAves For personal use only • Successfully completed US lisAng with access to world’s largest sophisAcated healthcare investor pool and analysts • Financing raised USD $63.5m (net proceeds) which significantly augmented exisAng cash
Slide 5: Disruptive Technology Platform
1 DisrupAve Technology PlaBorm For personal use only MulIple, Diverse Mechanisms Allogeneic Scalable Use § Mesenchymal Lineage Adult Stem § MLCs can be isolated from diverse Assue Cells (MLCs), immunoselected sources (bone marrow, adipose, dental pulp) precursors and progeny § MLCs from a single
Slide 6: Product Candidates Target Diseases with High Unmet Needs
2 Product Candidates Target Diseases with High Unmet Needs For personal use only Three Tier 1 Product Candidates in Phase 3 Programs P R O D U C T C A N D I D AT E S PROGRAMS PRECLINICAL PHASE 2 PHASE 3 A P P R O VA L TIER 1 TEMCELL HS INJ. Acute GVHD (Japan) MSC-100-IV Acute steroid-refractory GVHD
Slide 7: Product Candidate Deliverables: Anticipated Milestones 2016-2017
2 Tier 1 Product Candidate Deliverables For personal use only 2016 2017 Product Candidate Programs Anticipated Milestones Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Temcell® HS Inj. Launch in Japan US Pediatric Phase 3 Interim Analysis (IA) Top Line Results MSC-‐100-‐IV / Acute Graft Versus Host Phase 3 program comp
Slide 8: MPC-150-IM: Chronic Heart Failure - Market Opportunity
A MPC-‐150-‐IM: Chronic Heart Failure (CHF) – Market Opportunity For personal use only MPC-‐150-‐IM is in development for pa>ents with New York Heart Associa>on Class II-‐IV CHF § 5.7m paAents (2% of the populaAon) diagnosed with CHF in the US1 § 870,000 new cases diagnosed in the US each yea
Slide 9: MPC-150-IM: Phase 2 Trial Results in CHF Identify Optimal Therapeutic Dose
A MPC-‐150-‐IM: Phase 2 Trial Results in CHF IdenAfy OpAmal TherapeuAc Dose For personal use only § Phase 2, randomised placebo controlled trial in 60 paAents with Class II / III CHF and LVEF<40% – Placebo vs. 25, 75, 150 M MPCs injected by endomyocardial catheter § At 6 months: Dose-‐dependent
Slide 10: MPC-150-IM: Phase 2 Trial Results Identify Optimal Target Patients
A MPC-‐150-‐IM: Phase 2 Trial Results in CHF IdenAfy OpAmal Target PaAents, Advanced Heart Failure (Baseline LVESV>100ml)1 For personal use only with LVESV >100ml1 Change (EnAre Cohort) Change (LVESV > 100 mL) Month 6 minus Baseline Month 6 minus Baseline Control 150 M MPC ∆, Control Control 150 M
Slide 11: MPC-150-IM: Single Dose Prevents HF-MACE Over Years
A MPC-‐150-‐IM: Single Dose Prevents HF-‐MACE Over 3 Years For personal use only HF-‐MACE Kaplan-‐Meier Curve over 36 months HF-‐MACE Kaplan-‐Meier Curve over 36 months following treatment in all paIents following treatment in paIents with LVESV>100ml § Over 36 months, paAents receiving 150M
Slide 12: MPC-150-IM: Phase 3 Trial Recruiting Well and Targets Advanced Heart Failure
A MPC-‐150-‐IM: Phase 3 Trial RecruiAng Well and Targets Advanced Heart Failure where Medical Need is Greatest For personal use only § PaAents with large baseline LVESV and advanced heart failure are at highest risk of HF-‐MACE § For these paAents exisAng therapies are inadequate and economic b
Slide 13: MPC-150-IM: Reduced Size of Phase 3 Program Following FDA Discussions
A MPC-‐150-‐IM: Reduced Size of Phase 3 Program Following FDA Discussions For personal use only Following the preliminary responses from FDA in Dec 2015, the ongoing phase 3 program is planned to be opAmized as follows: § The current Phase 3 trial size will be reduced from 1,165 to approximately
Slide 14: MPC-06-ID: Chronic Low Back Pain - Market Opportunity
B MPC-‐06-‐ID: Chronic Low Back Pain due to DegeneraAve Disc Disease – Market Opportunity For personal use only MPC-‐06-‐ID is in development for the treatment of chronic low back pain (CLBP) las>ng >6 months as a result of moderate degenera>ve intervertebral disc disease § Over 5.7m paAents in
Slide 15: MPC-06-ID: Phase 2 Trial Design
B MPC-‐06-‐ID: 12 Month Phase 2 Trial Design For personal use only § 100 paAents with >6 months of CLBP due to DDD and unresponsive to conservaAve therapies (including opioids and epidural steroids) were evaluated in a randomized, placebo controlled Phase 2 trial § Visual Analog Scale (VAS) scor
Slide 16: MPC Groups Have Greater Proportion of Patients with at Least 50% Pain Reduction
B MPCs Groups Have a Greater ProporAon of PaAents Through 24 Months with at Least 50% Pain ReducAon Than Controls For personal use only 6 & 12Months 12 & 24 Months 50% ReducAon in VAS LBP with No IntervenAon 50% ReducAon in VAS LBP with No IntervenAon 70.0% 70.0% 59.3%a,b 60.0% 60.0% p = 0.016 50.0%
Slide 17: MPC Groups: 15 Point Improvement in Function (ODI) With No Intervention
B MPCs Groups Have a Greater ProporAon of PaAents Through 24 Months with at Least 15 Point Improvement in FuncAon (ODI) With No IntervenAon Compared to Controls For personal use only 6 & 12 Months 12 & 24 Months 15pt Improvement in ODI with No IntervenAon 15pt Improvement in ODI with No IntervenAon
Slide 18: MPC-06-ID: Composite Endpoint for Both Pain and Function Over 24 Months
B MPC-‐06-‐ID: Composite Endpoint for Both Pain and FuncAon Over 24 Months – Phase 2 Data For personal use only % PaIents with treatment success % PaIents 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% % Respon
Slide 19: MPC-06-ID: Phase 3 Pathway to Potential Approval
B MPC-‐06-‐ID: Phase 3 Pathway to PotenAal Approval in PaAents with CLBP due to Moderate DegeneraAve Intervertebral Disc Disease For personal use only § Phase 3 trial is enrolling well – The study will enroll ~330 paAents – Primary efficacy endpoint is a composite of ≥ 50% pain improvement (VAS) an
Slide 20: MSC-100-IV / TEMCELL HS Inj.: Acute Graft vs Host Disease - Market Opportunity
C MSC-‐100-‐IV / TEMCELL® HS Inj. : Acute Grad vs Host Disease – Market Opportunity For personal use only MSC-‐100-‐IV / TEMCELL® HS Inj. is targe>ng pediatric and adult pa>ents with acute GraS Versus Host Disease (aGVHD) following allogeneic Bone Marrow Transplant (BMT). § ~30,000 allogeneic B
Slide 21: MSC-100-IV: Phase 3 Trial in Children with Steroid Refractory aGVHD
C MSC-‐100-‐IV: Phase 3 Trial in Children with Steroid Refractory Acute Grad 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 mulIple other modaliIes SR-‐aGVHD (n = 102) Open-‐label (n =
Slide 22: MSC-100-IV / TEMCELL HS Inj.: Product Launch Plans in aGVHD
MSC-‐100-‐IV / TEMCELL® HS Inj. : Product Launch Plans in aGVHD For personal use only Japan (TEMCELL® HS Inj.): 2016 Expected Revenues • Our licensee JCR PharmaceuAcals Co. to launch TEMCELL® HS Inj. in Japan for adult and pediatric aGVHD in Q1 2016 • Japan’s NaAonal Health Insurance (NHI) set rei
Slide 23: Anti-Inflammatory Portfolio - a Major Emerging Opportunity
D AnA-‐Inflammatory PorBolio -‐ a Major Emerging Opportunity For personal use only Inflamma>on induces produc>on of immuno-‐modulatory factors by MLCs, which regulate mul>ple immune pathways concurrently § MLCs have receptors that respond to pro-‐ inflammatory signals, resulAng in release of anA i
Slide 24: MPC-300-IV for Treatment of Chronic Inflammatory Diseases
D MPC-‐300-‐IV for Treatment of Chronic Inflammatory Diseases For personal use only § Biologic-‐refractory rheumatoid arthriIs, 48 paIents – Ongoing – Randomized, placebo controlled, dose-‐escalaAng study – First dose / cohort 1 is fully enrolled – Topline data expected Q1 2016 – Clinical objecA
Slide 25: MPC-300-IV: Biologic Refractory Rheumatoid Arthritis - Market Opportunity
D MPC-‐300-‐IV: Biologic Refractory Rheumatoid ArthriAs (RA) – Market Opportunity For personal use only Ongoing randomized, controlled Phase 2 Trial in 48 pa>ents with biologic refractory rheumatoid arthri>s, comparing two doses of MPC-‐300-‐IV against placebo § 1.7m paAents with RA in the US1
Slide 26: MPC-300-IV: Diabetic Nephropathy - Market Opportunity
D MPC-‐300-‐IV: DiabeAc Nephropathy (DN) – Market Opportunity For personal use only § ~7.1 million paAents with DiabeAc Nephropathy in the US1 § ~1.96 million paAents with Chronic Kidney Disease (CKD) Stage 3b-‐4 (GFR 20-‐45 ml/min / 1.73 Market m2) DiabeAc Nephropathy3,4 opportunity § ~200,0
Slide 27: Strategic Collaborations with Industry Leaders
3 Strategic CollaboraAons with Industry Leaders For personal use only Manufacturing Development CommercializaIon § Clinical Heart § CHF program – development failure MPC-‐150-‐IM § Global rights § AddiAonal CV & CNS § Manufacturing programs § R&D aGVHD § Japan aGVHD rights Japan § Japan Ma
Slide 28: Scalable Manufacturing Capabilities: Partnership with Lonza
4 Scalable Manufacturing CapabiliAes: Partnership with Lonza For personal use only Manufacturing objecIves § DisAnct manufacturing processes for each product § Commercial scale processes with batch-‐to-‐batch consistency and reproducible release criteria § SubstanAal advances made in developmen
Slide 29: Extensive Intellectual Property Portfolio - Patents Across 72 Families
5 Extensive Intellectual Property PorBolio – 661 Patents Across 72 Families# For personal use only ComposiIon of maqer (COM) Specific therapeuIc Complementary technologies and manufacturing process applicaIons and addiIonal candidates 141 patents or patent applica>ons 382 patents or patent applica>on
Slide 30: Experienced Management Team
6 Experienced Management Team For personal use only Silviu Itescu – MBBS, FRACP CEO and Managing Director • Mesoblast Founder, physician scienAst in the fields of stem cell biology, autoimmune diseases, organ transplantaAon, and heart failure. • Previously: Faculty member Columbia University, USA; Un
Slide 31: Experienced Board of Directors
6 Experienced Board of Directors For personal use only Brian Jamieson, FCA Non-‐ExecuAve Chairman Silviu Itescu -‐ MBBS, FRACP Former Chief ExecuAve of a major Australian Law Firm Minter Ellison, Chief CEO and Managing Director ExecuAve Officer at KPMG Australia, KPMG Board Member in Australia and a
Slide 32: Key Investment Highlights
Key Investment Highlights For personal use only 1 DisrupAve technology plaBorm: proprietary, allogeneic, “off-‐the-‐shelf” adult stem cells with predictable therapeuAc properAes 2 Established late stage porBolio of disAnct and advanced product candidates Strategic partnerships delivering clinical,
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