Slide 1: Financial Results for the Year Ended 30 June 2016
For personal use only Financial Results for the Year Ended 30 June 2016 August 2016
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
Agenda For personal use only FINANCIAL RESULTS TECHNOLOGY REVIEW & OPERATIONAL UPDATE TIMELINES 3
Slide 4: Financial Results
Agenda For personal use only FINANCIAL RESULTS 4
Slide 5: Financial Highlights US$m
Financial Highlights US$m For personal use only • The Company ended FY16 with cash reserves of US$80.9 million • The Company reduced its annual operating and investing cash outflows by 15% (US$16.4 million) to US$89.7 million for FY16 in comparison with FY15, primarily by decreasing Tier 2 expenditu
Slide 6: Strategic Prioritization of Core Assets to Reduce Operational Spend
Strategic Prioritization of Core Assets to Reduce Operational Spend For personal use only As previously indicated in July, the Company has initiated a number of cost reductions in order to absorb the incremental costs of the MPC-150-IM program in FY17. Specifically: • Company labor force was restruc
Slide 7: FY16 Cash Flows and Cash Position
FY16 Cash Flows and Cash Position US$m 30 June 2016 30 June 2015 $ Change % For personal use only Cash on hand 80.9 110.7 (29.8) Annual Cash flows: Operating Cash Outflows (88.0) (101.0) 13.0 13% Investing Cash Outflows (1.7) (5.1) 3.4 67% Financing Cash Inflows 62.1 45.9 16.2 35% Net decrease in ca
Slide 8: FY16 Profit and Loss (1 of 2)
FY16 Profit and Loss (1 of 2) US$m 30 June 2016 30 June 2015 $ Change % For personal use only Revenue 42.5 19.8 22.8 115% Research and Development (50.0) (62.6) 12.6 20% Manufacturing Commercialization (29.8) (23.8) (6.0) (25%) Management & Administration (22.5) (29.5) 7.0 24% Contingent Considerati
Slide 9: FY16 Profit and Loss (2 of 2)
FY16 Profit and Loss (2 of 2) US$m 30 June 2016 30 June 2015 $ Change % For personal use only Revenue 42.5 19.8 22.8 115% Research and Development (50.0) (62.6) 12.6 20% Manufacturing Commercialization (29.8) (23.8) (6.0) (25%) Management & Administration (22.5) (29.5) 7.0 24% Contingent Considerati
Slide 10: Technology Review & Operational Update
Agenda For personal use only TECHNOLOGY REVIEW & OPERATIONAL UPDATE 10
Slide 11: Prudent Use of Cash Reserves Delivers Substantial Progress
Prudent Use of Cash Reserves Delivers Substantial Progress Corporate Focus For personal use only • US listing and financing completed in difficult market conditions • Prudent use of cash used to advance Tier 1 product candidates (CHF, RA, CLBP and aGVHD) • Expanding US investor base provides additio
Slide 12: Product Delineation Using Mesenchymal Lineage Cells Provides Diversified Pipeline
Product Delineation Using Mesenchymal Lineage Cells Provides Diversified Pipeline For personal use only Multiple mechanisms of action (MoA) Proprietary manufacturing processes may facilitate therapeutic effects in utilized to create distinct product diseases with high unmet needs candidates with spe
Slide 13: Phase Results Across Multiple Disease States Suggest a Shared MoA
Phase 2 Results Across Multiple Disease States Suggest a Shared MoA For personal use only • Consistent signals of efficacy seen in Phase 2 trials for our Tier 1 product candidates in hardest to treat patient segments across multiple disease states (CHF, RA, CLBP, aGVHD) • These observations suggest
Slide 14: Shared Immunomodulatory MoA Across Multiple Disease States
Shared Immunomodulatory MoA Across Multiple Disease States Inflammation induces production of immuno-modulatory factors by For personal use only Mesenchymal Precursor Cells (MPCs), which regulate multiple immune pathways concurrently MPCs have receptors that respond to inflammatory signals, result
Slide 15: Diversified Pipeline of Product Candidates for High Unmet Needs
Diversified Pipeline of Product Candidates for High Unmet Needs First Product on market - Three Tier 1 Product Candidates in Phase 3 For personal use only 15
Slide 16: MPC-150-IM: Chronic Heart Failure – Market Opportunity
MPC-150-IM: Chronic Heart Failure (CHF) – Market Opportunity MPC-150-IM is in development for patients with New York Heart Association Class II-IV CHF For personal use only 5.7m patients (2% of the population) diagnosed with CHF in the US1 915,000 new cases diagnosed in the US each year1 Market
Slide 17: MPC-150-IM: Phase Results Show Promise in Prevention of HF-MACE
MPC-150-IM: Phase 2 Results Show Promise in the Prevention of HF-MACE* HF-MACE Kaplan-Meier Curve over 36 months HF-MACE Kaplan-Meier Curve over 36 months For personal use only following treatment in all patients following treatment in patients with LVESV>100ml Over 36 months, patients receiving 1
Slide 18: MPC-150-IM: Phase 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 • For these patients existing therapies are inadequate and economic burden is greatest • To confirm that MPC-150-IM reduces HF
Slide 19: MPC-150-IM: Phase Trial Operational Update
MPC-150-IM: Phase 3 Trial Operational Update • Phase 3 trial for 600 patients with advanced heart failure is recruiting well For personal use only across North American sites; as of June over 240 patients • After reviewing all clinical data for the first 175 patients in April 2016, the trial’s Data
Slide 20: MPC-300-IV: Biologic Refractory RA – Market Opportunity
MPC-300-IV: Biologic Refractory Rheumatoid Arthritis (RA) – Market Opportunity Ongoing randomized, controlled Phase 2 Trial in 48 patients with biologic refractory rheumatoid For personal use only arthritis, comparing two doses of MPC-300-IV against placebo There are 5.3 million prevalent cases in
Slide 21: MPC-300-IV: Study Design for Phase Trial in Biologic Refractory RA
MPC-300-IV: Study Design for Phase 2 Trial in Biologic Refractory RA For personal use only Phase 2 double-blind, randomized, placebo-controlled, dose-escalating study comparing a single MPC infusion of either 1M/kg or 2M/kg against placebo in 48 patients with active RA and inadequate response to a
Slide 22: MPC-300-IV: Summary of Key Efficacy Responses in Biologic Refractory RA
MPC-300-IV: Summary of Key Efficacy Responses at Week 12 in Biologic Refractory RA All Subjects Subgroup with Prior Use of 1-2 For personal use only Biologics Placebo 1M/kg 2M/kg p-value Placebo 1M/kg 2M/kg p-value 2M/kg vs 2M/kg vs placebo placebo N=16 N=16 N=16 N=9 N=10 N=11 ACR70 0% 20% 27% 0.04
Slide 23: MPC-300-IV: One Dose Provides Efficacy in the Biologic Refractory Population
MPC-300-IV: One Dose Provides Compelling Efficacy at 12 Weeks in the Biologic Refractory Population ACR70 @ 3 Months For personal use only 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 (1-2 bi
Slide 24: MPC-300-IV: Conclusions
MPC-300-IV: Conclusions For personal use only Biologic refractory rheumatoid arthritis (RA) population accounts for approximately one-third of all RA patients who have received anti-TNF or other biologic agents, is the hardest to treat, and requires new therapies that are both effective and safe,
Slide 25: MPC-300-IV for Biologic Refractory RA: Pathway Forward
MPC-300-IV for Biologic Refractory RA: Pathway Forward For personal use only Potential Phase 3 clinical development program in Biologic Refractory RA for MPC- 300-IV is likely to include: – Short duration to primary endpoint (12-24 weeks) – Powering of trial size guided by treatment benefit observ
Slide 26: MPC-06-ID: Chronic Low Back Pain – Market Opportunity
MPC-06-ID: Chronic Low Back Pain Due to Disc Degeneration (CLBP) – Market Opportunity MPC-06-ID is in development for the treatment of CLBP lasting >6 months as a result of For personal use only moderate degenerative disc disease Prevalent CLBP population to grow to 21.9m patients in the U.S. by 2
Slide 27: MPC-06-ID: Phase Trial Operational Update
MPC-06-ID: Phase 3 Trial Operational Update For personal use only • Current 360 patient Phase 3 trial is recruiting well across US sites • FDA has provided written guidance: – Use of a composite primary endpoint is acceptable for approval – Agreed thresholds for pain (50% decrease in VAS) and functi
Slide 28: MSC-100-IV / TEMCELL HS Inj.: Acute Graft vs Host Disease
MSC-100-IV / TEMCELL® HS Inj. : Acute Graft vs Host Disease MSC-100-IV / TEMCELL® HS Inj. is targeting pediatric and adult patients with acute Graft Versus Host Disease (aGVHD) following allogeneic Bone Marrow Transplant (BMT) For personal use only ~30,000 allogeneic BMTs performed globally (~20K
Slide 29: Manufacturing Achievements
Manufacturing Achievements Manufacture completed for clinical supply of all For personal use only current Phase 3 trials Regulatory activities ongoing to meet requirements for commercial manufacturing across product pipeline Specific formulations defined for product delineation In-house prop
Slide 30: Timelines
30 For personal use only Agenda TIMELINES
Slide 31: Tier Product Candidate Deliverables (Calendar Year)
Tier 1 Product Candidate Deliverables (Calendar Year) 2016 2017 For personal use only Product Candidate Programs Milestones Q1 Q2 Q3 Q4 Q1 Q2 Q3 Q4 Phase 3 Enrollment Complete Class II and III Heart Failure MPC-150-IM Interim Results Class IV Heart Failure Requiring Phase 2b trial results LVAD Top l