Mesoblast Limited · ASX:MSB
Mesoblast — Q3 FY2020 Earnings Presentation
32 slides from Mesoblast's earnings presentation, May 28, 2020
Slide 1: Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases
Global Leader in Allogeneic Cellular Medicines for Inflammatory Diseases Financial and Operational Highlights for the Third Quarter Ended March 31, 2020 ASX: MSB; Nasdaq: MESO
Slide 2: Cautionary Note Regarding Forward-Looking Statements
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, levels of activity, perf
Slide 3: Our Mission
Our Mission Mesoblast is committed to bringing to market innovative cellular medicines to treat serious and life-threatening illnesses |3
Slide 4: Allogeneic Cellular Medicines for Inflammatory Diseases
Allogeneic Cellular Medicines for Inflammatory Diseases Innovative Technology Lead Product Pipeline of Phase 3 Platform Candidate Product Candidates Allogeneic mesenchymal precursor RYONCILTM (remestemcel-L) BLA Lifecycle expansion of cells (MPCs) and their progeny, filed with US FDA for pedia
Slide 5: Platform Technology – Mechanism of Action
Platform Technology – Mechanism of Action Our cellular therapies are activated by multiple inflammatory cytokines through surface receptors, resulting in orchestration of an anti-inflammatory cascade IL-10 Breg TH1 Mesenchymal Proliferation Cytokine secretion Proliferation Precursor/Stem Cell IDO, P
Slide 6: Pipeline of Phase 3 Product Candidates
Pipeline of Phase 3 Product Candidates MESOBLAST PRODUCT THERAPEUTIC PHASE 1/2 PHASE 3 REGISTRATION COMMERCIAL COMMERCIAL CANDIDATE AREA RIGHTS PARTNERS RYONCILTM Acute GVHD - Pediatric (Remestemcel-L) Global ex-Japan Acute GVHD - Adult Pediatric & adult Chronic GVHD systemic Global inflammatory Acu
Slide 7: Commercial Scale Manufacturing Capability
Commercial Scale Manufacturing Capability Manufacturing Remestemcel-L Scalable allogeneic “off-the-shelf” cellular platforms Manufacturing meets stringent criteria of international regulatory agencies Robust quality assurance processes ensure final product with batch-to-batch consistency and r
Slide 8: Global IP Estate Provides Substantial Competitive Advantage
Global IP Estate Provides Substantial Competitive Advantage Extensive patent portfolio with protection extending through 2040 in all major markets Over 1,000 patents and patent applications (68 patent families) across all major jurisdictions Therapeutic Areas Covers composition of matter, manu
Slide 9: Financials
Financials
Slide 10: Financial Highlights
Financial Highlights First Nine Months FY2020 Compared to First Nine Months FY2019 113% increase in total revenue to US$31.5m from US$14.8m 81% growth in commercialization revenue from sales of TEMCELL to US$5.9m from US$3.3m 127% increase in milestone revenues from strategic partnerships to U
Slide 11: Success of TEMCELL by Mesoblast Licensee JCR Informs Potential US Market for RYONCIL
Continued Growth in Revenues from Sales of TEMCELL in Japan for SR-aGVHD TEMCELL® ANNUAL REVENUE FROM TEMCELL ROYALTIES IN JAPAN ACUTE GRAFT VERSUS HOST DISEASE + OTHER INDICATIONS 8.0 JCR Pharmaceuticals has exclusive rights to Mesoblast’s 7.0 7.6 MSC technology for acute GVHD in Japan 6.0 US$7
Slide 12: Substantial Increase in Revenues and Reduced Loss After Tax
Substantial Increase in Revenues and Reduced Loss After Tax March 31, March 31, Profit and Loss for the nine months ending (US$m) 2020 2019 Commercialization revenue 5.9 3.3 Milestone revenue 25.0 11.0 Interest revenue 0.5 0.5 Total Revenue 31.5 14.8 Research and development (40.9) (48.4) Manufactur
Slide 13: Strengthened Balance Sheet After Capital Raise
Strengthened Balance Sheet After Capital Raise Cash on hand at March 31, 2020 was US$60.1m Pro forma cash on hand is approximately US$150m, with the additional US$90m capital raised in May 2020 Up to an additional US$67.5 million may be available through existing financing facilities and strat
Slide 14: RYONCIL (remestemcel-L): Acute Graft Versus Host Disease
RYONCIL (remestemcel-L): Acute Graft Versus Host Disease
Slide 15: Acute Graft Versus Host Disease (aGVHD)
Acute Graft Versus Host Disease (aGVHD) Significant market opportunity for RYONCIL aGVHD is a life-threatening complication that occurs in ~50% of patients receiving Burden of allogeneic bone marrow transplants (BMTs)1 Illness Steroid-refractory aGVHD is associated with mortality rates as high a
Slide 16: Grade C/D GVHD has Significantly Worse Survival than Grade A/B
Grade C/D GVHD has Significantly Worse Survival than Grade A/B IBMTR severity grade Glucksberg severity grade | 16
Slide 17: RYONCIL: Phase 3 Trial compared to MAGIC Database
RYONCIL: Phase 3 Trial compared to MAGIC Database Improved Day 28 Overall Response and Day 100 Survival relative to matched controls A comparative analysis performed between Mesoblast’s open-label Phase 3 study and contemporaneous controls receiving institutional standard of care Phase 3 trial o
Slide 18: RYONCIL: Anticipated FDA Approval in 2020
RYONCIL: Anticipated FDA Approval in 2020 Results from three studies using RYONCIL in children and adults with SR-aGVHD support the FDA BLA filing − RYONCIL was used as salvage therapy in an expanded access program in 241 children with SR-aGVHD (80% Grade C/D) who failed institutional standard of
Slide 19: Remestemcel-L: Lifecycle Extension Strategy
Remestemcel-L: Lifecycle Extension Strategy US/Ex-US parallel launch adult SR-aGVHD US launch COVID-19 ARDS Label extensions Pediatric - Influenza ARDS SR-aGVHD Phase - Bacterial ARDS 3 positive results Ex-US launch - Chronic GVHD COVID-19 ARDS & - HIE Product development/ pediatric manufacturing 20
Slide 20: Remestemcel-L: Acute Respiratory Distress Syndrome (ARDS) due to COVID-19
Remestemcel-L: Acute Respiratory Distress Syndrome (ARDS) due to COVID-19
Slide 21: Overview – Remestemcel-L for COVID-19 ARDS
Overview – Remestemcel-L for COVID-19 ARDS COVID-19 is a respiratory virus with a high mortality due to a severe inflammatory condition of the lungs called acute respiratory disease syndrome (ARDS) ARDS is caused by cytokine storm in lungs of patients infected with COVID-19 and is the primary ca
Slide 22: ARDS due to COVID-19, Influenza & Bacterial Infection – Major Unmet Need
ARDS due to COVID-19, Influenza & Bacterial Infection – Major Unmet Need Normal alveolus Injured alveolus during the acute phase Acute respiratory distress syndrome (ARDS) Alveolar Sloughing of bronchial epithelium A major area of unmet medical need airspace Inactivated surfactant Multiple trigg
Slide 23: Rationale for Remestemcel-L Treatment of ARDS
Rationale for Remestemcel-L Treatment of ARDS The COVID-19 virus stimulates a cytokine storm in the lung, increasing inflammatory cytokines and biomarkers such as TNFα, IL-6, IL-8, hepatocyte growth factor, and IL-2R leading to ARDS1-3 When remestemcel-L arrives in the inflamed lung, its surface
Slide 24: Pilot Data From Emergency IND Provides Rationale for Randomized Controlled Phase 3 Trial
Pilot Data From Emergency IND Provides Rationale for Randomized Controlled Phase 3 Trial of Remestemcel-L in COVID-19 ARDS Compassionate Use Data from Emergency IND 12 patients with moderate or severe ARDS received two infusions of remestemcel-L at Mt. Sinai Hospital in New York City Nine patien
Slide 25: Phase 3 Trial of 300 Patients with ARDS due to COVID-19
Phase 3 Trial of 300 Patients with ARDS due to COVID-19 Objective: Multi-center, randomized, controlled, blinded study to assess safety and efficacy of remestemcel-L versus standard of care (SOC) treatment in subjects with moderate/severe ARDS on ventilator due to COVID-19 The trial will be cond
Slide 26: Key Milestones for Remestemcel-L in COVID-19 ARDS
Key Milestones for Remestemcel-L in COVID-19 ARDS Recruitment is expected to take three to four months Interim analyses planned which could result in stopping the trial early for efficacy or futility. First interim analysis when 30% of patients reach the primary endpoint Seek expedited regulat
Slide 27: Update on Phase 3 Product Candidates - Heart Failure - Chronic Low Back Pain
Update on Phase 3 Product Candidates - Heart Failure - Chronic Low Back Pain
Slide 28: Partnerships and License Agreements
Partnerships and License Agreements Partnerships and License Agreements Phase 3 Product Candidates Product candidates MPC-06-ID CHRONIC LOW BACK PAIN - DEGENERATIVE DISC DISEASE Strategic partnership to develop and commercialize MPC-06-ID in Europe & Latin America PREVALENCE EUROPE Mesoblast wil
Slide 29: REVASCOR for Advanced and End-Stage Heart Failure
REVASCOR for Advanced and End-Stage Heart Failure In December 2019, the Phase 3 trial in advanced heart failure surpassed the number of primary endpoint events required for trial completion − Final study visits for all surviving patients have been completed − Ongoing quality review of all data is
Slide 30: MPC-06-ID for Chronic Low Back Pain
MPC-06-ID for Chronic Low Back Pain Phase 3 trial of MPC-06-ID for chronic low back pain in 404 patients: − Final study visits for all patients have been completed − Ongoing quality review of all data is being completed at the study sites − Data readout planned for mid-2020 Continued operational
Slide 31: Major Operational Milestones for the Next 12 Months
Major Operational Milestones for the Next 12 Months Remestemcel-L for SR-aGVHD & Other Rare Diseases RYONCIL Priority Review underway with PDUFA date set for September 30, 2020 If approved, US launch of RYONCIL planned for 2020 Expand investigator-initiated clinical trials for chronic GVHD and
Slide 32: Thank You
Thank You