Mesoblast Limited · ASX:MSB
Mesoblast — J.P. Morgan Healthcare Conference 2017
38 slides from Mesoblast's conference presentation, Jan 13, 2017
Slide 1: Cellular Medicines for Intractable Serious and Life-Threatening Diseases
For personal use only Cellular Medicines for Intractable Serious and Life-Threatening Diseases Dr. Silviu Itescu, Chief Executive January 2017 ASX:MSB/Nasdaq: MESO
Slide 2: Cautionary Note Regarding Forward-Looking Statements
For personal use only CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, le
Slide 3: Our Mission, Vision and Focus
For personal use only Our Mission, Vision and Focus: Mesoblast is committed to bring to market its disruptive cellular medicines to treat serious and life-threatening illnesses where there are currently no alternative treatments |3
Slide 4: Compelling Investment Proposition
Compelling Investment Proposition: Building a Leading Franchise of Cellular Medicines For personal use only Leader in Disruptive Proven Capability for Advanced Pipeline of Cellular Technology Commercial Translation Cellular Medicines Platform Extensive patent portfolio Scalable industrialized
Slide 5: The 21st Century Cures Act
The 21st Century Cures Act (“Cures Act”): Legislation for An Expedited Approval Path for Cellular Medicines Designated For personal use only as Regenerative Advanced Therapies Cellular medicines may be designated as regenerative advanced therapies, if they are intended to treat, modify, reverse, c
Slide 6: Implications of the Cures Act
Implications of the Cures Act: Mesoblast Products for Serious and Life-Threatening For personal use only Diseases Well Positioned The 21st Century Cures Act will: Shorten clinical development time Shorten timeframes to FDA approval Reduce costs of development Increase the prospect of near-te
Slide 7: Recent Corporate Development: Partnership Negotiations
Recent Corporate Development: Negotiations for a Development and Commercialization Partnership Underway For personal use only December 22, 2016 - Entered into an equity Mallinckrodt Pharmaceuticals purchase agreement with Mallinckrodt − Gains a significant opportunity to opt-in to a Pharmaceutic
Slide 8: Financial Highlights
Financial Highlights For personal use only At September 30, 2016, the Company had cash reserves of $US60.4 million In order to absorb the incremental costs of the MPC-150-IM program in advanced heart failure in FY17, the Company has executed its planned operational streamlining and re-prioritiza
Slide 9: Proprietary Mesenchymal Lineage Technology Platform
For personal use only Proprietary Mesenchymal Lineage Technology Platform |9
Slide 10: Intellectual Property is Core to Our Strategy
The Mesoblast Difference: Intellectual Property is Core to Our Strategy: For personal use only An Extensive Portfolio Covering Composition of Matter, Manufacturing, and Therapeutic Applications of Highly Potent Immuno-selected Mesenchymal Lineage Precursors and Progeny 796 Patents - 72 Patent Famili
Slide 11: Transforming the Science of Cellular Medicine into Commercial Reality
The Mesoblast Difference: Transforming the Science of Cellular Medicine into Commercial Reality For personal use only Mesenchymal lineage immuno-selected precursors and Mesenchymal progeny cells (MLCs) Lineage Cells (MLCs) STRO-1/STRO-3 immuno-selection provides a homogeneous and potent populati
Slide 12: Scalable Manufacturing For High Margin Medicines
The Mesoblast Difference: Scalable Manufacturing For High Margin Medicines For personal use only Manufacture completed for clinical supply of all current Phase 3 trials Regulatory activities ongoing to meet requirements for commercial manufacturing across product pipeline Specific formulations
Slide 13: Diverse Pipeline of Cellular Medicines
For personal use only Diverse Pipeline of Cellular Medicines | 13
Slide 14: Portfolio of Advanced Product Candidates
Portfolio of Advanced Product Candidates: Ideally Positioned for the 21st Century Cures Act Environment For personal use only Commercialization Product Therapeutic Area Pre-Clinical/ Platform Candidate Pre-IND Phase 2 Phase 3 Approval Partnering1 Advanced (Class 3) HF MPC MPC-150-IM End Stage (Class
Slide 15: MPC-150-IM Chronic Heart Failure Program
For personal use only MPC-150-IM Chronic Heart Failure (CHF) Program Multi-billion dollar blockbuster potential | 15
Slide 16: MPC-150-IM: Targets Serious Complications of Heart Failure
MPC-150-IM: For personal use only Targets the Most Serious and Life-Threatening Complications of Heart Failure Significant Burden of Illness and Unmet Need Globally, 17-45% of heart failure patients die within 1 year of hospital admission Majority of these patients die within 5 years of admissio
Slide 17: CHF Program: Phase 2 Randomized Placebo Controlled Trial
Chronic Heart Failure (CHF) Program: Phase 2 Randomized Placebo Controlled Trial in 60 Patients HF Class II/III and LVEF<40% For personal use only Objectives: LVESV Month 6 - Baseline LVEDV Month 6 - Baseline − Identify a dose response and an optimal therapeutic dose − Identify optimal target popu
Slide 18: CHF Program: Benefit on LV Remodeling in LVESV >100ml
Chronic Heart Failure (CHF) Program: Phase 2 Therapeutic Benefit on LV Remodeling is Amplified in Subjects with LVESV >100ml For personal use only Placebo corrected benefit of 150M cell dose on cardiac volumes and ejection fraction at 6 months was greatest in patients with more advanced heart fail
Slide 19: CHF Program: Durable Protection Against HF-MACE
Chronic Heart Failure (CHF) Program: Phase 2 Clinical Results Show A Single High-dose Injection May be Sufficient For Durable For personal use only (36 Months) Protection Against HF-MACE1 in Patients With Advanced Heart Failure % HF-MACE Kaplan-Meier Curve over HF-MACE Kaplan-Meier Curve over 36 mon
Slide 20: MPC-150-IM: Phase 3 Trial Targets Advanced Heart Failure
MPC-150-IM: Phase 3 Trial Targets Advanced Heart Failure For personal use only Patients with large baseline LVESV and advanced heart failure are at highest risk of HF-MACE – Have increased likelihood of having recurrent HF hospitalizations – Existing therapies are inadequate and economic burden is
Slide 21: MPC-150-IM: Phase 3 Trial Operational Update
MPC-150-IM: Phase 3 Trial Operational Update For personal use only The trial’s primary endpoint is a comparison of recurrent heart failure-related major adverse cardiovascular events (HF-MACE) in advanced CHF patients receiving either MPC-150-IM by catheter injection into the left ventricular hear
Slide 22: Chronic Low Back Pain Due to Disc Degeneration
For personal use only Chronic Low Back Pain (CLBP) Due to Disc Degeneration Multi-billion dollar blockbuster potential | 22
Slide 23: MPC-06-ID: Alternative to Invasive Surgery and Opioid Use
MPC-06-ID: Alternative to Invasive Surgery and Opioid Use for Chronic Low Back Pain Patients For personal use only Significant Burden of Illness and Unmet Need Back pain causes more disability than any other condition1 Inflicts substantial direct and indirect costs on the healthcare system1, inc
Slide 24: MPC-06-ID: Phase 2 Trial Results
MPC-06-ID: Phase 2 Trial Results Support Phase 3 Program1 For personal use only General Overview 100 patients with >6 months of CLBP due to DDD and unresponsive to conservative therapies (incl. opioids and epidural steroids) were evaluated in a randomized, placebo controlled Phase 2 trial Safety
Slide 25: MPC-06-ID: Data Support Composite Endpoint in Phase 3
MPC-06-ID: Phase 2 Data Support Composite Pain and Function Endpoint in Ongoing Phase 3 Study For personal use only - Consistent with Potential as an Alternative to Surgery and Opioid Use for CLBP Patients % Patients with Treatment % Patients with Treatment Treatment Success Composite Success at 6 &
Slide 26: MPC-06-ID: Phase 3 Trial Operational Update
MPC-06-ID: Phase 3 Trial Operational Update For personal use only The 24-month results from the Company’s 100-patient Phase 2 trial of MPC-06-ID for treatment of CLBP were presented at the 24th Annual Scientific Meeting of the Spine Intervention Society; received the 2016 Best Basic Science Abstra
Slide 27: Acute Graft vs Host Disease (aGVHD)
For personal use only Acute Graft vs Host Disease (aGVHD) Our nearest-term revenue product candidate: MSC-100-IV for steroid-refractory aGVHD | 27
Slide 28: MSC-100-IV: Acute Graft vs Host Disease
MSC-100-IV: Acute Graft vs Host Disease Serious and Life-Threatening Complication of Bone Marrow Transplants For personal use only Significant Burden of Illness and Unmet Need aGVHD - a severe immunological reaction occurring in BMT patients Steroid-refractory aGVHD (SR-aGVHD) patients have mort
Slide 29: MSC-100-IV: Prior Clinical Results Support Phase 3 in Children
MSC-100-IV: Prior Clinical Results1 Support Ongoing Phase 3 Trial in Children with For personal use only Steroid Refractory Acute GVHD (SR-aGVHD) MSC-100-IV in Children with SR-aGVHD MSC-100-IV as first line therapy who failed multiple other modalities in children with SR-aGVHD - Survival of Pediatr
Slide 30: Acute GVHD: Product Development Strategy
Acute Graft vs Host Disease: Product Development Strategy For personal use only Pediatric GVHD0001/GVHD002: Phase 3 study ongoing, ~40 sites planned1 Multi-center, single-arm, open-label to evaluate efficacy and safety to day 100 (study 001) and from day 100 to day 180 (study 002) At least 60 pe
Slide 31: GVHD001: Successful Interim Futility Analysis
GVHD001: Successful Interim Futility Analysis in 4Q CY16 For personal use only Predefined Bayesian futility rule that determined the predictive probability of success using the primary endpoint of Day 28 overall response Method determined the likelihood of obtaining a statistically significant t
Slide 32: Inflammatory Diseases Portfolio (MPC-300-IV)
For personal use only Our inflammatory diseases portfolio (MPC-300-IV) is a highly attractive emerging opportunity Multi-billion dollar blockbuster potential | 32
Slide 33: MPC-300-IV: Biological Refractory Rheumatoid Arthritis
MPC-300-IV: Biological Refractory Rheumatoid Arthritis (RA) For personal use only Significant Burden of Illness and Unmet Need RA is associated with multiple co-morbidities and psychosocial impairments1 Mesoblast development program targets the biologic refractory population2 − ~1/3 of RA patien
Slide 34: MPC-300-IV: Phase 2 Efficacy Responses at Week 12
MPC-300-IV: Biological Refractory RA Phase 2 Efficacy Responses at Week 12 For personal use only All Subjects Subgroup with prior use of 1-2 Biologics P=value P=value Placebo 1M/kg 2M/kg 2M=kg vs. Placebo 1M/kg 2M/kg 2M=kg vs. placebo placebo N=16 N=16 N=16 N=9 N=10 N=11 ACR70 0% 20% 27% 0.04 0% 20%
Slide 35: MPC-300-IV: ACR Responses Compared to Published Comparators
MPC-300-IV: Biological Refractory RA Phase 2 ACR Responses Compared to Published Comparators at Week 12 For personal use only ACR70 @ 3 Months 40% Marketed Pipeline 36% 35% 30% 27% 25% 20% 22% 15% 14% 10% 5% 11% 6% 0% Inhibitors of IL-6, MPC 2m/kg CD80/CD86, CD20, JAK 1 Jak Inhibitors2 MPC 2m/kg (1-
Slide 36: Upcoming Planned Milestones and Catalysts
Upcoming Planned Milestones and Catalysts Over the Next 12 Months For personal use only MPC-150-IM − Phase 3 interim analysis for Class II/III (1Q CY17) − Phase 2B complete trial enrollment for Class IV (Mid-17) − Phase 2B data read-out Class IV (4Q CY17) MSC-100-IV − Phase 3 complete trial enro
Slide 37: Cellular medicines offer unique solutions to intractable medical challenges
For personal use only Cellular medicines offer unique solutions to intractable medical challenges | 37
Slide 38: Thank You
For personal use only Thank You | 38