Slide 1: Operational Highlights and Financial Results for the Year Ended June 30, 2017 (FY17)
For personal use only Operational Highlights and Financial Results for the Year Ended June 30, 2017 (FY17) 30 August 2017
Slide 2: Operational Highlights
For personal use only Operational Highlights |2
Slide 3: Our Mission
For personal use only Our Mission: Mesoblast is committed to bring to market disruptive cellular medicines to treat serious and life-threatening illnesses |3
Slide 4: Investment Proposition: Building a Leading Franchise of Cellular Medicines
Investment Proposition: Building a Leading Franchise of Cellular Medicines For personal use only A Leader in Disruptive Capability for Commercial Advanced Pipeline of Cellular Technology Translation Cellular Medicines Platform Extensive patent portfolio Scalable industrialized Three Tier 1 pro
Slide 5: The 21st Century Cures Act
The 21st Century Cures Act (“Cures Act”): Legislation for An Expedited Approval Path for Cellular Medicines Designated as For personal use only Regenerative Medicine Advanced Therapies (RMAT) Cellular medicines may be designated as regenerative advanced therapies, if they are intended to treat, mo
Slide 6: Proprietary Mesenchymal Lineage Technology Platform
For personal use only Proprietary Mesenchymal Lineage Technology Platform |6
Slide 7: Intellectual Property: An Extensive Portfolio
Intellectual Property: An Extensive Portfolio Covering Composition of Matter, Manufacturing, and Therapeutic For personal use only Applications of Potent Immuno-selected Mesenchymal Lineage Precursors and Progeny ~ 800 Patents and patent applications across 69 Patent Families. Protection across majo
Slide 8: The Mesoblast Difference: Scalable Manufacturing Capability
The Mesoblast Difference: Technology Positioned for Scalable Manufacturing Capability 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 f
Slide 9: Translating Our Science to the Clinic
Translating Our Science to the Clinic 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 Cytokines homogeneous and potent population of MLCs with receptors Chemokines Reduced Inf
Slide 10: Diverse Pipeline of Cellular Medicines
For personal use only Diverse Pipeline of Cellular Medicines | 10
Slide 11: Portfolio of Advanced Product Candidates
Portfolio of Advanced Product Candidates: Well 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 4)
Slide 12: MPC-150-IM Chronic Heart Failure (CHF) Program
For personal use only MPC-150-IM Chronic Heart Failure (CHF) Program | 12 | 12
Slide 13: MPC-150-IM: Targeting Patients with Worsening HF Despite Optimal Standard of Care
MPC-150-IM: Targeting Patients with Worsening HF Despite Optimal Standard of Care For personal use only Under Investigation in Phase 2b/3 MPC-150-IM targets MPC-150-IM targets HFrEF NYHA class IIB/III HFrEF NYHA class III/IV patients with worsening patients with worsening HF despite optimal care HF
Slide 14: MPC-150-IM: Adjunct to LVAD in NYHA Class IV/End Stage Heart Failure
MPC-150-IM: Adjunct to LVAD in NYHA Class IV/End Stage Heart Failure Commercial Landscape For personal use only Burden of Illness and Unmet Need 250K – 300K patients suffer from advanced systolic HF (NYHA Class IV)1 Despite optimal medical therapy, 1-year mortality exceeds 50% in class IV patien
Slide 15: MPC-150-IM: Targets Destination Therapy and Potential Use for Bridge to Recovery
MPC-150-IM: Targets Destination Therapy and Potential Use for Bridge to Recovery For personal use only Three Distinct Market Segments Destination Therapy Represents ~45% of Market3 Bridge to Resucue Recovery Therapy 0.3% 0.4% • Bridge to transplant (BTT) appears saturated with ~2k transplants per an
Slide 16: MPC-150-IM: Rationale for Use as Adjunct Therapy in LVAD Patients
MPC-150-IM: Rationale for Use as Adjunct Therapy in LVAD Patients For personal use only We believe MPC-150-IM has the potential to: • enhance beneficial remodelling of native heart muscle by inducing myocardial blood vessel maturation in the heart and reducing myocardial inflammation • strengthen na
Slide 17: MPC-150-IM: Bleeding is the Major Complication of CF LVADs
MPC-150-IM: Bleeding is the Major Complication of Continuous Flow (CF) LVADs For personal use only The most common cause of LVAD-related re-hospitalization, not associated with surgical procedures, is gastrointestinal (GI) bleeding Adverse Event Rates (Events per 100 Patient-Months) in the First 12
Slide 18: LVAD MPC Pilot Trial: 25M MPCs Increased Ability to be Weaned off LVADs
LVAD MPC Pilot Trial: 25M MPCs Increased Ability to be Weaned off LVADs and For personal use only Increased Short-Term Survival1 No cell-related safety events observed LVAD MPC Pilot Trial: 12 Month Survival Median time to first readmission was 91 days in the MPC group vs 51 days in the control
Slide 19: MPC-150-IM: Operational Update for Phase 2b Trial Evaluating 150M MPCs
MPC-150-IM: Operational Update for Phase 2b Trial Evaluating 150M MPCs in End-Stage Heart Failure Patients as Adjunct to LVAD For personal use only Study is sponsored and funded by the United States National Institutes of Health (NIH), and conducted by the NIH-funded Cardiothoracic Surgical Trials
Slide 20: MPC-150-IM: Targets Serious and Life-Threatening Complications of Heart Failure
MPC-150-IM: Targets the Serious and Life-Threatening Complications of Heart Failure For personal use only 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 target advanced
Slide 21: MPC-150-IM: Durable (36 Months) Protection Against HF-MACE
MPC-150-IM: Durable (36 Months) Protection Against HF-MACE1 in Phase 2 Trial Following Single Dose For personal use only % HF-MACE Kaplan-Meier Curve over HF-MACE Kaplan-Meier Curve over 36 months following 36 months following treatment in all patients1 treatment in patients with LVESV>100ml2 • Over
Slide 22: MPC-150-IM: Operational Update for Phase 3 Trial in NYHA Class II-III
MPC-150-IM: For personal use only Operational Update for Phase 3 Trial in NYHA Class II-III Advanced CHF Patients • Trial has enrolled 400 of approximately 600 patients • In April 2017, a pre-specified interim futility analysis of the efficacy endpoint in the Phase 3 trial's first 270 patients was s
Slide 23: Acute Graft vs Host Disease (aGVHD)
For personal use only Acute Graft vs Host Disease (aGVHD) MSC-100-IV for steroid-refractory aGVHD ||23 23
Slide 24: MSC-100-IV: Acute Graft vs Host Disease (aGVHD)
MSC-100-IV: Acute Graft vs Host Disease (aGVHD) Serious and Life-Threatening Complication of Bone Marrow Transplants For personal use only Burden of Illness and Unmet Need aGVHD - a severe immunological reaction occurring in BMT patients Steroid-refractory aGVHD (SR-aGVHD) patients have mortalit
Slide 25: MSC-100-IV for aGVHD: Product Development Strategy
MSC-100-IV for aGVHD: Product Development Strategy For personal use only Pediatric aGVHD Multi-center, single-arm, open-label, ongoing Phase 3 study in up to 60 pediatric patients with steroid- refractory aGVHD The pre-specified interim futility analysis of the trial’s primary endpoint was succe
Slide 26: Chronic Low Back Pain (CLBP) Due to Disc Degeneration
For personal use only Chronic Low Back Pain (CLBP) Due to Disc Degeneration | 26 | 26
Slide 27: MPC-06-ID: Potential Alternative to Invasive Surgery and Opioid Use
MPC-06-ID: Potential Alternative to Invasive Surgery and Opioid Use for Chronic Low Back Pain Patients For personal use only 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, inclu
Slide 28: MPC-06-ID: Phase 2 Trial Results Support Phase 3 Program
MPC-06-ID: Phase 2 Trial Results Support Phase 3 Program For personal use only 100 patients with >6 months of CLBP due to DDD and unresponsive to conservative therapies (incl. opioids and epidural steroids) were evaluated in a blinded, randomized, placebo controlled Phase 2 trial Composite Respond
Slide 29: MPC-06-ID: Phase 3 Trial Update
MPC-06-ID: Phase 3 Trial Update For personal use only A 360-patient Phase 3 trial across US and Australian sites Targeted to complete recruitment by Q4 2017 FDA has provided written guidance: - Use of a composite primary endpoint is acceptable for potential approval - Agreed thresholds for pai
Slide 30: Financial Results
For personal use only Financial Results | 30
Slide 31: Financial Highlights for the Twelve Months Ending June 30, 2017
Q4 FY 2017: Financial Highlights for the Twelve Months Ending June 30, 2017 (US$m) For personal use only At June 30, 2017, the Company had cash reserves of US$45.8 million and US$84.0 million after adjusting for proceeds from the A$50.7 million (US$40.0 million) fully underwritten entitlement offe
Slide 32: Operational Streamlining: Successfully Achieved Targeted Savings for FY17
Operational Streamlining: Successfully achieved targeted savings for FY17 (US$m) For personal use only In August 2016, the Company announced a range of cost reduction initiatives in order to save US$20-25 million of operational costs. US$20.7 million of cost savings have been achieved through these
Slide 33: Targeted Upcoming Milestones and Catalysts
Targeted Upcoming Milestones and Catalysts For personal use only MPC-150-IM − Phase 3 trial for Class II/III continues to enroll with target expected completion (2H CY18) − Phase 2B trial expected to complete enrollment (3Q CY17) for Class IV1,2 − Phase 2B data read-out Class IV (expected 1Q CY18)