Slide 1: Operational Highlights and Financial Results for the Half Year Ended December 31, 2016
For personal use only Operational Highlights and Financial Results for the Half Year Ended December 31, 2016 February 2017 |1
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: Agenda
For personal use only Agenda • Strategic Update • Financial Results • Operational Update • Timelines |3
Slide 4: Strategic Update
For personal use only Strategic Update |4
Slide 5: 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 |5
Slide 6: Compelling Investment Proposition: Building a Leading Franchise of Cellular Medicines
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 7: 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 8: The 21st Century Cures Act: Expedited Approval Path for Cellular Medicines
The 21st Century Cures Act (“Cures Act”): Legislation for An Expedited Approval Path for Cellular Medicines Designated as For personal use only Regenerative Advanced Therapies Cellular medicines may be designated as regenerative advanced therapies, if they are intended to treat, modify, reverse, c
Slide 9: Recent Corporate Development: Negotiations for Development and Commercialization Partnership
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 10: Financial Results
For personal use only Financial Results | 10
Slide 11: H1 FY 2017: Financial Highlights
H1 FY 2017: For personal use only Financial Highlights US$m At December 31, 2016, cash reserves were US$55.6 million after adjusting for US$21.7 million from the equity purchase agreement with Mallinckrodt announced on December 23, 2016; proceeds received on January 6, 2017 The Company executed
Slide 12: FY 2017: Cash Position
FY 2017: For personal use only Cash Position (US$m) 31 Dec 2016 30 Sep 2016 30 Jun 2016 Reported Cash on hand 33.9 60.4 80.9 Equity purchase agreement 21.7 - - Adjusted Cash on hand 55.6 60.4 80.9 Adjusted Cash on 31 December 2016 equals Reported Cash plus US$21.7 million cash received on 6 Januar
Slide 13: Operational Streamlining: Update
Operational Streamlining: For personal use only Update (US$m) From August 2016, the Company executed a range of cost reduction initiatives in order to offset the incremental costs of the MPC-150-IM program in FY17. $11.5 million of savings have been generated by these initiatives is the first six mo
Slide 14: Operating Cash Flows Reduced by Operational Streamlining
FY 2017: For personal use only Operating Cash flows reduced by Operational Streamlining (US$m) 31 Dec 2016 31 Dec 2015 $ Change Operating Cash flows for the first six months Payment to Suppliers and Employees (47.3) (51.9) 4.6 Commercialization revenue received 0.6 - 0.6 Milestone revenue received -
Slide 15: Operational Highlights
For personal use only Operational Highlights | 15
Slide 16: MPC-150-IM Chronic Heart Failure Program
For personal use only MPC-150-IM Chronic Heart Failure (CHF) Program Multi-billion dollar blockbuster potential | 16
Slide 17: 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 die within 5 years of admission1 MPC-150-IM to
Slide 18: MPC-150-IM: Phase 3 Trial Operational Update
MPC-150-IM: Operational Update for Phase 3 trial of MPC-150-IM in New York Heart For personal use only Association (NYHA) Class II-III advanced CHF patients This trial’s primary endpoint is a comparison of recurrent heart failure-related major adverse cardiovascular events (HF-MACE) in advanced CH
Slide 19: MPC-150-IM: Phase 2b Trial in End-Stage Heart Failure with LVAD
MPC-150-IM: Operational Update for Phase 2b trial in patients with end-stage heart failure who For personal use only have received a left ventricular assist device (LVAD) This program is sponsored and funded by the United States National Institutes of Health (NIH). Enrollment of this trial total
Slide 20: 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 | 20
Slide 21: MPC-300-IV: Biological Refractory Rheumatoid Arthritis Market Landscape
MPC-300-IV: Biological Refractory Rheumatoid Arthritis (RA) Market Landscape For personal use only Market Size There are approx 6.0 million prevalent cases in the US, Japan, and EU5, with 2.9 million in the US alone in 20161,2 In 2016, sales of RA agents, predominantly TNF inhibitors exceeded $1
Slide 22: MPC-300-IV: Phase 2 Study Design
MPC-300-IV: Phase 2 Study design For personal use only Phase 2 double-blind, randomized, placebo-controlled, dose-escalating study comparing a single infusion of two MPC doses (1M/kg and 2M/kg) with placebo in patients with active RA Inadequate response to at least 1 anti-TNF +/- other biologics
Slide 23: MPC-300-IV: Phase 2 Trial Results
MPC-300-IV: Phase 2 Trial Results For personal use only The safety profile over 39 weeks was comparable among the placebo and both MPC treatment groups, with no cell-related serious adverse events Both MPC doses outperformed placebo at week 39 in each of ACR20/50/70 responses, as well as by medi
Slide 24: Summary of Key Efficacy Responses: All Subjects
Summary of Key Efficacy Responses at 3 and 9 Months: All Subjects For personal use only Week 12 Week 39 Placebo 1M/kg 2M/kg Placebo 1M/kg 2M/kg N=16 N=16 N=16 N=16 N=16 N=16 ACR20 50% 47% 53% 36% 69% 57% ACR50 19% 27% 40% 14% 31% 21% ACR70 0% 20% 27%* 0% 23% 21% ACR-N median 20% 11% 28% 9% 27% 27% A
Slide 25: Summary of Key Efficacy Responses: Prior Use of 1-2 Biologics
Summary of Key Efficacy Responses at 3 and 9 Months: Sub-group with Prior Use of 1-2 Biologics For personal use only Week 12 Week 39 Placebo 1M/kg 2M/kg Placebo 1M/kg 2M/kg N=9 N=10 N=11 N=9 N=10 N=11 ACR20 33% 60% 55% 22% 67% 60% ACR50 11% 30% 55% 0% 33% 30% ACR70 0% 20% 36% 0% 22% 30% ACR-N median
Slide 26: 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 | 26
Slide 27: 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 28: 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 29: Acute Graft vs Host Disease: MSC-100-IV
For personal use only Acute Graft vs Host Disease (aGVHD) Our nearest-term revenue product candidate: MSC-100-IV for steroid-refractory aGVHD | 29
Slide 30: 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 31: 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 32: GVHD001: Successful Interim Futility Analysis in 4Q CY16
GVHD001: Successful Interim Futility Analysis in 4Q CY16 For personal use only Predefined Bayesian futility rule determined predictive probability of success using the primary endpoint of Day 28 overall response Method determined the likelihood of obtaining a statistically significant treatment
Slide 33: Timelines
For personal use only Timelines | 33
Slide 34: 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 35: Thank you
For personal use only Thank you | 35