Mesoblast Limited · ASX:MSB
Mesoblast — H1 FY2018 Earnings Presentation
41 slides from Mesoblast's earnings presentation, Feb 28, 2018
Slide 1: Operational Highlights and Financial Results for the Half Year Ended December 31, 2017
For personal use only Operational Highlights and Financial Results for the Half Year Ended December 31, 2017 February 2018 Nasdaq: MESO ASX: MSB
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
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 Disruptive Cellular Technology Platform Commercial Translation Capabilities Advanced Pipeline of Cellular Medicines Targeting Serious or Life-Threatening Conditions with Unmet Needs |4
Slide 5: Disruptive Cellular Medicine Platform
Disruptive Cellular Medicine Platform1-4 For personal use only Mesenchymal Lineage Cells (MLCs) have unique receptors that respond to activating inflammatory and damaged-tissue signals In response to these signals, MLCs secrete a diverse variety of biomolecules responsible for immunomodulation a
Slide 6: Commercial Translation Capabilities: Scalable, Industrialized Manufacturing
Commercial Translation Capabilities: Technology Positioned for Scalable, Industrialized Manufacturing For personal use only Immune privileged nature of MLCs enables allogeneic “off the shelf” product candidates Culture expansion scalable to produce commercial quantities of potent and reproducibl
Slide 7: Portfolio of Advanced Product Candidates: Three Tier Product Candidates in Phase 3
Portfolio of Advanced Product Candidates: Three Tier 1 Product Candidates in Phase 3 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) HF1 MPC MPC-06-
Slide 8: The 21st Century Cures Act: Expedited Approval Path for RMAT
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 9: Mesoblast Received FDA RMAT Designation For MPC-150-IM
Mesoblast Received FDA RMAT Designation For MPC-150-IM for Heart Failure Patients With Left Ventricular Assist Devices (LVADs) For personal use only RMAT designation grant was based on the completed study data set and related analyses of a 30-patient randomized, blinded, placebo controlled trial i
Slide 10: Intellectual Property: An Extensive Portfolio
Intellectual Property: An Extensive Portfolio Covering Mesenchymal Lineage Precursors and Progeny For personal use only • Composition of Matter, Manufacturing, and Therapeutic Applications of Potent Immuno-selected mesenchymal lineage precursor and stem cells • 800 Patents and patent applications ac
Slide 11: Patent Settlement and License Agreement With TiGenix
Mesoblast Concluded Patent Settlement and License Agreement With TiGenix For personal use only Mesoblast granted TiGenix exclusive access to certain of its patents to support global commercialization of the adipose-derived mesenchymal stem cell (MSC) product Cx601 limited to the local treatment of
Slide 12: Diverse Pipeline of Cellular Medicines
For personal use only Diverse Pipeline of Cellular Medicines | 11
Slide 13: Acute Graft vs Host Disease
For personal use only Acute Graft vs Host Disease Remestemcel-L (MSC-100-IV) for Steroid-Refractory aGVHD | 13
Slide 14: Remestemcel-L: Market Opportunity for aGVHD
Remestemcel-L (MSC-100-IV): Market Opportunity for aGVHD For personal use only Steroid-refractory aGVHD patients have mortality rates as high as 95%1 Burden of Refractory aGVHD is associated with significant extended hospital stay costs2 Illness aGVHD - a severe immunological reaction occurrin
Slide 15: Remestemcel-L for aGVHD: Product Development Strategy
Remestemcel-L for aGVHD: Product Development Strategy For personal use only 1. Target pediatric patients with SR-aGVHD first Extensive safety and efficacy data generated and published in children with SR-aGVHD1 High economic burden in treatment of children with SR-aGVHD Fast-track designation
Slide 16: Pediatric Trial GVHD001 Completed Enrollment
Remestemcel-L (MSC-100-IV): Phase 3 Pediatric Trial GVHD001 Completed Enrollment as First-line Therapy in aGVHD After Failing Steroids For personal use only Multi-center, Single-Arm, Open-Label to evaluate efficacy and safety to day 100 (GVHD001) and from day 100 to day 180 (GVHD002) 55 pediatri
Slide 17: Protocol GVHD001: Primary Efficacy Outcome
Protocol GVHD001: Primary Efficacy Outcome Overall Response at Day 28 was 69%, p=0.0003 For personal use only 80% 70% 60% 50% 40% 30% 20% 10% 0% 69% Overall Response rate at Day 28 (29% CR + 40% PR) p-value calculated from the binomial distribution, under the assumption of a 0.45 success rate un
Slide 18: Protocol GVHD001: Results of Safety and Mortality
Protocol GVHD001: Results of Safety and Mortality For personal use only Remestemcel-L (MSC-100-IV) infusions were well tolerated The incidence of adverse events in the trial was consistent with that expected from the underlying disease state and in line with previous use of remestemcel-L (MSC-10
Slide 19: Protocol GVHD001: Summary and Conclusions
Protocol GVHD001: Summary and Conclusions This Phase 3 study evaluated allogeneic mesenchymal stem cells (MSCs), remestemcel-L (MSC-100-IV), for the For personal use only treatment of steroid-refractory acute graft-versus-host disease intended to improve overall response rate in pediatric subjects
Slide 20: Remestemcel-L: Commercialization Plans
Remestemcel-L (MSC-100-IV): Commercialization Plans Clinical For personal use only Successful primary endpoint at Day 28 - Completed Day 100 survival (Q2 CY2018) Day 180 safety and survival (Q3 CY2018) Manufacturing Commercial Readiness Market preparation Potential for Commercial Pri
Slide 21: MPC-150-IM Chronic Heart Failure Program
For personal use only MPC-150-IM Chronic Heart Failure (CHF) Program | 21 | 21
Slide 22: MPC-150-IM: Targeting Patients with Worsening HF
MPC-150-IM: Targeting Patients with Worsening HF Despite Optimal Standard of Care For personal use only Common Treatment Pathway in CHF1 MPC-150-IM Target Use Early MPC-150-IM targets HFrEF ACEI or ARB NYHA Advanced-stage HF Statins and End-stage HF Beta blockers Re-vascularization or valvul
Slide 23: MPC-150-IM: Product Development Strategy Following RMAT Designation
MPC-150-IM: Product Development Strategy Following RMAT Designation for Heart Failure Patients With Left Ventricular Assist Devices (LVADs) For personal use only Leverage data for potential near term market entry opportunity for MPC- 150-IM in end-stage heart failure patients with LVADs Broaden
Slide 24: MPC-150-IM: Class IV Market Opportunity
MPC-150-IM: Class IV Market Opportunity For personal use only 250K – 300K patients/yr suffer from advanced systolic HF (NYHA Class IV)1 Burden of 50k patients/yr have end-stage heart failure Despite optimal medical therapy, 1-year mortality exceeds 50% in end-stage Illness heart failure patien
Slide 25: MPC-150-IM: Phase 2b Trial in End-Stage Heart Failure with LVADs
MPC-150-IM: Phase 2b Trial Evaluating 150M MPCs in End-Stage Heart Failure Patients with LVADs The 159-patient, double-blind, placebo-controlled 2:1 randomized trial, is evaluating the safety and For personal use only efficacy of injecting MPC-150-IM into the native myocardium of LVAD recipients
Slide 26: MPC-150-IM: Class III Heart Failure Market Opportunity
MPC-150-IM: Class III Heart Failure Market Opportunity Globally, 17-45% of heart failure patients die within 1 year of hospital admission For personal use only Burden of Majority die within 5 years of admission1 Illness MPC-150-IM to target advanced HFrEF NYHA Class II-III with the objective o
Slide 27: MPC-150-IM: Operational Update for Phase 3 Trial in NYHA Class II-III
MPC-150-IM: Operational Update for Phase 3 Trial in NYHA Class II-III Advanced CHF Patients For personal use only Trial has enrolled more than 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 pati
Slide 28: Chronic Low Back Pain Due to Disc Degeneration
For personal use only Chronic Low Back Pain (CLBP) Due to Disc Degeneration | 28 | 28
Slide 29: MPC-06-ID: Non-Opioid Alternative for Chronic Low Back Pain
MPC-06-ID: A Non-Opioid Alternative for Chronic Low Back Pain Due to Degenerative Disc Disease For personal use only Back pain causes more disability than any other condition1 Burden of Inflicts substantial direct and indirect costs on the healthcare system1, Illness including excessive use of o
Slide 30: The Opioid Epidemic
The Opioid Epidemic For personal use only 50% of opioid prescriptions are for chronic low back pain (CLBP) Over 1,000 people are treated in U.S. emergency departments everyday for misusing prescription opioids Over 33,000 people in the U.S. died of prescription opioid related overdoses in 2016
Slide 31: 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 U.S. and Australian sites Targeted to complete recruitment Q1 CY18 FDA has provided written guidance: − Use of a composite primary endpoint at 12 and 24 months is acceptable − Agreed thresholds for pain (5
Slide 32: Our inflammatory diseases portfolio (MPC-300-IV)
For personal use only Our inflammatory diseases portfolio (MPC-300-IV) ||32 32
Slide 33: MPC-300-IV: Phase 2 Clinical Data in Immune Mediated Diseases
MPC-300-IV: Being evaluated in immune mediated diseases where the cellular product candidate responds to multiple inflammatory signals by releasing factors that modulate the immune response For personal use only Phase 2 Clinical Data in Immune Mediated Diseases 60 patients, type 2 diabetes with in
Slide 34: MPC-300-IV: Phase 2 Trial in Biologic Refractory Rheumatoid Arthritis
MPC-300-IV: Phase 2 trial in biologic refractory Rheumatoid Arthritis shows early and durable effects after single dose For personal use only Infusions were well-tolerated and there were no treatment-related serious adverse events reported, with the safety profile comparable among the placebo and
Slide 35: Financials & Milestones
For personal use only Financials & Milestones | 35
Slide 36: Q2 FY18: Cash Position and Cash Flows for the Half Year
Q2 FY18: Cash Position and Cash Flows for the Half Year Ending 31 December 2017 (US$m) For personal use only 31 Dec 2017 31 Dec 2016 $Change %Change Operating net cash outflows (35.2) (46.4) 11.2 24% Investing cash outflows (0.7) (0.3) (0.4) (133%) Financing cash inflows/(outflows) 37.9 (0.1) 38.0 N
Slide 37: Q2 FY18: Profit and Loss for the Half Year
Q2 FY18: Profit and Loss for the Half Year ending 31 Dec 2017 (US$m) For the six months ending 31 Dec 2017 31 Dec 2016 $ Change % For personal use only Revenue 14.6 0.9 13.7 NM Research and Development (31.6) (29.0) (2.6) (9%) Manufacturing Commercialization (1.7) (7.1) 5.4 76% Management & Administ
Slide 38: Q2 FY18: Profit and Loss for the Half Year
Q2 FY18: Profit and Loss for the Half Year ending 31 Dec 2017 (US$m) For the six months ending 31 Dec 2017 31 Dec 2016 $ Change % For personal use only Revenue 14.6 0.9 13.7 NM Research and Development (31.6) (29.0) (2.6) (9%) Manufacturing Commercialization (1.7) (7.1) 5.4 76% Management & Administ
Slide 39: Q2 FY18: Profit and Loss for the Half Year
Q2 FY18: Profit and Loss for the Half Year ending 31 Dec 2017 (US$m) For the six months ending 31 Dec 2017 31 Dec 2016 $ Change % For personal use only Revenue 14.6 0.9 13.7 NM Research and Development (31.6) (29.0) (2.6) (9%) Manufacturing Commercialization (1.7) (7.1) 5.4 76% Management & Administ
Slide 40: Targeted Upcoming Milestones and Catalysts
Targeted Upcoming Milestones and Catalysts For personal use only MSC-100-IV for Pediatric Acute GVHD − Day 28 primary endpoint data read-out (Q1 CY18) COMPLETE − Day 100 survival data (Q2 CY18) − Day 180 safety data (Q3 CY18) MPC-06-ID for Chronic Low Back Pain − Phase 3 trial expected to comple
Slide 41: Questions?
For personal use only Questions? | 41