Mesoblast Limited · ASX:MSB
Mesoblast — Q1 FY2019 Earnings Presentation
39 slides from Mesoblast's earnings presentation, Nov 16, 2018
Slide 1: Operational Highlights and Financial Results for the Quarter Ended September 30, 2018
For personal use only Operational Highlights and Financial Results for the Quarter Ended September 30, 2018 November 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: Premier Global Cellular Medicines Company
Premier Global Cellular Medicines Company For personal use only Disruptive Technology Industrial Scale Multiple Revenue Platform1 Manufacturing Generating Products & Phase 3 Assets Immuno-selected, culture Unique cell properties enable 2 approved products expanded cellular medicines large scal
Slide 5: Disruptive cellular medicine technology
Disruptive cellular medicine technology For personal use only STRO-1+ Mesenchymal Precursor Cells (MPCs) are at the apex of the hierarchy of mesenchymal lineage cells STRO-1/STRO-3 immuno-selection provides a homogeneous population of MPCs with receptors that respond to activating inflammation a
Slide 6: Commercial Translation Capabilities
Commercial Translation Capabilities Technology positioned for scalable, industrialized manufacturing For personal use only Immune privileged nature of mesenchymal lineage cells enables allogeneic “off the shelf” product candidates Culture expansion scalable to produce anticipated commercial quan
Slide 7: Global IP Estate Provides Substantial Competitive Advantage
Global IP Estate Provides Substantial Competitive Advantage For personal use only ~800 Patents and patent applications (69 Patent families) across all major jurisdictions Covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells Diseases All Tier 1 &
Slide 8: Commercial Products and Clinical Pipeline
Commercial Products and Clinical Pipeline Using Mesoblast’s Intellectual Property and Technology Platform For personal use only PLATFORM PRODUCT THERAPEUTIC AREA APPROVAL COMMERCIAL RIGHTS MARKETED MSC TEMCELL® Acute GVHD 1st allogeneic regen med approved in Japan Japan (Bone Marrow) HS Inj1 MSC (
Slide 9: Strategic Cardiovascular Partnership in China
Strategic Cardiovascular Partnership in China For personal use only Tasly Pharmaceutical Group has exclusive rights and will fund all development, manufacturing and commercialization activities in China for MPC-150-IM for the treatment or prevention of chronic heart failure and MPC-25-IC for the t
Slide 10: Financials
For personal use only Financials | 10 | 10
Slide 11: Significant Increase in Revenue
Significant Increase in Revenue For personal use only Revenue for the quarter ending September 30, 2018 (US$m) September 30, September 30, For the quarter ending $ Change % Change 2018 2017 Milestone revenue 10.5 0.5 10.0 NM Commercialization revenue 1.0 0.6 0.4 66% Interest revenue 0.2 0.1 0.1 93%
Slide 12: Loss After Tax
Loss After Tax Profit and Loss for the quarter ending September 30, 2018 (US$m) For personal use only September 30, September 30, For the quarter ending $ Change % Change 2018 2017 Total revenue 11.6 1.2 10.5 NM Research and development (18.5) (15.4) (3.1) 20% Manufacturing (4.3) (0.9) (3.4) NM Mana
Slide 13: Consistent Operating Cash Outflows
Consistent Operating Cash Outflows Cash flow highlights (US$m) For personal use only September 30, September 30, For the quarter ending $ Change % Change 2018 2017 `Operating net cash outflows (19.5) (20.4) 0.9 (4%) Investing net cash (outflows)/inflows - (0.6) 0.6 (100%) Financing net cash inflows
Slide 14: Cash Position Strengthened through Strategic Transactions
Cash Position Strengthened through Strategic Transactions Balance sheet cash (US$m) For personal use only September 30, June 30, $Change 2018 2018 Reported Cash on Hand 55.1 37.8 17.3 NovaQuest financing agreement - 39.0 (39.0) Tasly strategic partnership 40.0 40.0 - Pro forma cash on hand 95.1 116.
Slide 15: Diverse Pipeline of Cellular Medicines
For personal use only Diverse Pipeline of Cellular Medicines | 15
Slide 16: MSC-100-IV (remestemcel-L) for Steroid-Refractory Acute GVHD
For personal use only MSC-100-IV (remestemcel-L) for Steroid-Refractory Acute Graft vs Host Disease | 16 | 16
Slide 17: Remestemcel-L: Market Opportunity for Acute GVHD
Remestemcel-L: Market Opportunity for Acute Graft Versus Host Disease (aGVHD) For personal use only aGVHD is a life-threatening complication that occurs in ~50% of patients receiving Burden of allogeneic bone marrow transplants (BMT)1 Illness Steroid-refractory aGVHD is associated with mortality
Slide 18: Remestemcel-L: Phase 3 Trial Operational Update
Remestemcel-L: Phase 3 Trial Operational Update For personal use only Phase 3 study evaluated remestemcel-L in 55 children to improve overall response rate and survival − 89% of children had grade C/D disease, the most severe form and historically associated with up to 95% mortality Study succes
Slide 19: GVHD Pathway to Market
GVHD Pathway to Market For personal use only Regulatory Preparations for Biologics License Application (BLA) filing underway FDA meetings and BLA filing (Q4 CY18 – Q1 CY19) Fast Track designation allows eligibility for priority review and rolling BLA review process Commercial Parallel track
Slide 20: MPC-150-IM for Chronic Heart Failure
For personal use only MPC-150-IM for Chronic Heart Failure | 20 | 20
Slide 21: MPC-150-IM: Targeting Patients with Advanced and End-stage Heart Failure
MPC-150-IM:Targeting Patients with Advanced and End-stage Heart Failure For personal use only Common Treatment Pathway in Progressive Heart Failure1 Mesoblast Target Market: Early ACEI or ARB Advanced Statins and End-Stage HF patients3 Beta blockers Re-vascularization or valvular surgery New
Slide 22: MPC-150-IM: Adjunctive Therapy to Improve Clinical Outcomes in LVAD Patients
MPC-150-IM: Adjunctive Therapy to Improve Clinical Outcomes in LVAD Patients For personal use only In the USA, there are approximately 250,000–300,000 patients annually who Burden of suffer from advanced systolic heart failure (NYHA Class IIIb–IV). 1 Illness Despite optimal medical therapy (excl
Slide 23: INTERMACS Adverse Event Rates in LVAD Patients
INTERMACS* Adverse Event Rates in LVAD Patients: Most Common Cause of Non-surgical Hospitalization is Major GI Bleeding1 For personal use only *Interagency Registry for Mechanically Assisted Circulation (INTERMACS): Events per 100 Patient-Months in the First 12 Months Post-Implant, based on 7,286 pa
Slide 24: Proposed Pathway of Angiogenesis and Non-surgical GI Bleeding
Proposed Pathway of Angiogenesis and Non-surgical GI Bleeding During CF-LVAD For personal use only Altered Shear Stress vWF Degradation Angiopoietin- Factors (HIP) Inducible Hypoxia Hypoxia Angiogenesis Angiodysplasia Splanchnic Inflammation: Hypoperfusion IL-6, IL-1, TNFα An integrating explanation
Slide 25: MPCs Reduced Major GI Bleeding in Patient Pilot Trial
MPCs Reduced Major GI Bleeding in 30 Patient Pilot Trial1 For personal use only Cumulative Incidence for Control, 0.4 (95% CI, 0.10, 0.69) Cumulative Incidence for MPC, 0.1 (95% CI, 0.02, 0.28) Gray’s Test for Equality of CIF, P = 0.041 Readmitted Population Days to First Non-Surgical Major Bleeding
Slide 26: The 21st Century Cures Act
The 21st Century Cures Act (Cures Act) For personal use only Legislation for An Expedited Approval Path for Cellular Medicines Designated as Regenerative Medicine Advanced Therapies (RMAT) Cellular medicines may be designated as regenerative advanced therapies, if they are intended to treat, modif
Slide 27: Mucosal Bleeding in Phase 2 Trial
Mucosal Bleeding at 6 Months in Phase 2 Trial For personal use only Rate of GI/Epistaxis Bleeding MPC (n = 106) Control (n = 53) P-value Event Rate Event Rate (100-Pt-Months) (100-Pt-Months) 3.8 15.9 <0.001 | 27
Slide 28: Serious AEs & Hospitalizations in Phase 2 Study
Serious AEs & Hospitalizations at 6 Months in Phase 2 Study only MPC Control Neurological dysfunction 4.4 per 100 pt-months 3.3 Hospitalizations use 3.6 100 pt-months Right heart failure 5.3 (p=0.03) Rate per 0.07 Major infections 9.1 GI Bleeding personal 10.2 0.21 Major device malfunction 3.5 3 7.7
Slide 29: Successful Temporary Weans from LVAD Support
Successful Temporary Weans from LVAD Support For personal use only Average proportion of successful 70 66% 62%62% temporary weans: 59%58% − 61% in MPC vs. 58% in control 60 53% Proportion of Successful − RR=1.08 (95% CI 0.83-1.41; p=0.55) 50 Posterior probability that MPC 40 Weans increased like
Slide 30: Exploratory Subgroup Analyses
Exploratory Subgroup Analyses For personal use only Interaction of Rx and Pre-determined Subgroups on Wean Success Rate over 6 Months | 30
Slide 31: Conclusions
Conclusions For personal use only • The trial succeeded in achieving the clinically meaningful outcome of reduction in gastrointestinal (GI) bleeding and related hospitalizations • Results confirm the previous pilot trial, which also demonstrated significant reduction in GI bleeding and related hosp
Slide 32: MPC-150-IM: Moderate/Advanced Heart Failure Market Opportunity
MPC-150-IM: Moderate/Advanced Heart Failure Market Opportunity For personal use only ~ 8 million patients with chronic heart failure by 2030 in USA alone1 17-45% globally die within 1 year of hospital admission1 Burden of Illness/Limited Majority of advanced heart failure patients die within 5
Slide 33: MPC-150-IM: Phase 3 Program in Moderate to Advanced Heart Failure
MPC-150-IM: Phase 3 Program in Patients with Moderate to Advanced Heart Failure For personal use only More than 85% of patients enrolled in events-driven USA Phase 3 trial, targeting ~600 patients Pre-specified interim futility analysis of the efficacy endpoint in the first 270 patients was succ
Slide 34: MPC-06-ID for Chronic Low Back Pain due to Disc Degeneration
For personal use only MPC-06-ID for Chronic Low Back Pain due to Disc Degeneration | 34 | 34
Slide 35: MPC-06-ID: Chronic Low Back Pain due to Degenerative Disc Disease
MPC-06-ID: 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 Illness system,1 including excessive use of opioids in this patient popula
Slide 36: MPC-06-ID: Phase 3 Trial in Patients with Chronic Low Back Pain
MPC-06-ID: Phase 3 Trial in Patients with Chronic Low Back Pain For personal use only Phase 3 study completed enrollment in March 2018 Over 400 patients were enrolled at 48 sites across USA and Australia Patients randomized 1:1:1 to receive saline, 6-million MPCs with hyaluronic acid and 6-mil
Slide 37: Milestones
For personal use only Milestones | 37 | 37
Slide 38: CY 2018 Corporate Milestones
CY 2018 Corporate Milestones MSC-100-IV for Acute Graft versus Host Disease For personal use only Successfully met Day 28 primary end point pediatric Phase 3 trial (Q1 CY18) Day 100 survival/safety data pediatric Phase 3 trial (Q2 CY18) Day 180 survival/safety data pediatric Phase 3 trial
Slide 39: Questions?
For personal use only Questions? | 39 | 39