Slide 1: Chronic Low Back Pain: MPC-06-ID Partnership with Grünenthal
For personal use only Chronic Low Back Pain: MPC-06-ID Strategic Development & Commercialization Partnership with Grünenthal for Europe & Latin America 10 September 2019 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 bringing to market innovative cellular medicines to treat serious and life-threatening illnesses |3
Slide 4: Corporate History
Corporate History For personal use only Over a decade of scientific, manufacturing, clinical development and corporate development experience targeted at bringing to market allogeneic, off-the-shelf cellular medicines for inflammatory diseases 2004: 2013: 2015: 2017: 2019: 2019: Mesoblast founded in
Slide 5: Premier Global Cellular Medicines Company
Premier Global Cellular Medicines Company For personal use only Innovative Technology Platform1 Late Stage Pipeline Commercialization Innovative technology targets Initiated rolling filing with US Building US sales force for some of the most severe disease FDA for approval for steroid- aGVHD l
Slide 6: Commercial Scale Manufacturing Capability
Commercial Scale Manufacturing Capability For personal use only Scalable allogeneic “off-the-shelf” cellular medicine platform Manufacturing meets stringent criteria set by international regulatory agencies including FDA and EMA Robust quality assurance processes ensure final product with batc
Slide 7: Global IP Estate Provides Substantial Competitive Advantage
Global IP Estate Provides Substantial Competitive Advantage For personal use only ~995 patents and patent applications (68 patent families) across all major jurisdictions Covers composition of matter, manufacturing, and therapeutic applications of mesenchymal lineage cells Diseases Enables lic
Slide 8: Commercial and Late-Stage Product Pipeline
Commercial and Late-Stage Product Pipeline For personal use only PLATFORM PRODUCT THERAPEUTIC AREA APPROVAL COMMERCIAL RIGHTS MARKETED TEMCELL® MSC Acute Graft Versus Host Japan 1st allogeneic regen med approved in Japan (Bone Marrow) HS Inj1 Disease MSC (Adipose) Alofisel®2 Perianal Fistula 1st a
Slide 9: Key Terms of the Strategic Partnership with Grünenthal GmbH
Key Terms of the Strategic Partnership with Grünenthal GmbH For personal use only Grünenthal takes exclusive license to develop and commercialise MPC-06-ID Indication: for discogenic chronic back pain, disc degeneration and/or functional disability Territory: Europe and Latin America/Caribbean I
Slide 10: Transaction Benefits to Mesoblast
Transaction Benefits to Mesoblast Strong commercial partner For personal use only Delivers commercialization, distribution, sales & marketing Field force comprises around 1,600 people across Europe, Latin America & US – overall focus is on pain – visited nearly 300,000 stakeholders in 2018 (ph
Slide 11: Grünenthal – Global Leader in Pain Management
Grünenthal – Global Leader in Pain Management For personal use only #2 Europe1 #1 Latin America1 Overview Grünenthal is a global leader in pain management and related diseases, with 50 years in pain research A long track record of bringing innovative treatments and state-of-the-art technologies
Slide 12: MPC-06-ID for Chronic Low Back Pain – Commercial Opportunity
For personal use only MPC-06-ID for Chronic Low Back Pain due to Degenerative Disc Disease – Commercial Opportunity
Slide 13: MPC-06-ID: A New Paradigm for Treatment of Chronic Low Back Pain
MPC-06-ID: A New Paradigm for Treatment of 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 system1, including excessive use o
Slide 14: Approx. 15% of Adult Population Suffers from Chronic Lower Back Pain
Approx. 10 – 15% of Adult Population Suffers from Chronic Lower Back Pain Across the 7 Major Pharmaceutical Markets For personal use only Prevalence of Chronic Lower Back Pain (millions)1 US prevalence 33.4 million, EU5 39.4 million, Japan 14.9 million Japan, Growth in the prevalence of CLBP in
Slide 15: US/EU Patient Journey
US/EU Patient Journey – 50% of Patients With Moderate-to-Severe CLBP Progress To Specialists For Diagnosis And Treatment Of Discogenic Cause For personal use only Stakeholder Diagnostic Modalities • Physical exam Patient Presents Primary Care Physician • Pain scales With Symptoms (50%) • Imaging (MR
Slide 16: Current Patient Treatment Journey for Discogenic CLBP
Current Patient Treatment Journey (US/EU) for Discogenic CLBP (DCLBP): MPC-06-ID Potential First-Line for DCLBP Refractory to Conservative Treatments For personal use only MPC-06-ID: targeting moderate-to- severe DCLBP Conservative treatments Opioid analgesics Interventional therapies Surgeries NS
Slide 17: Inflammation is at the Core of Degenerative Disc Disease
Chronic Low Back Pain Inflammation is at the Core of Degenerative Disc Disease For personal use only McCann MR and Seguin CA. Notochord Cells in Intervertebral Disc Development and Degeneration. J. Dev. Biol. 2016, 4(1), 3; doi:10.3390/jdb4010003 | 17
Slide 18: MPC-06-ID: Potential Mechanisms of Action in Inflammatory Disc Disease
MPC-06-ID: Potential Mechanisms of Action in Treating Inflammatory Disc Disease For personal use only IL-1, IL-6 IL-1β TNFα M1 TNFα MPC-06-ID Macrophage Polarization M2 Neurite ingrowth Neuropathic pain Structural integrity of annulus Proteoglycans in nucleus | 18
Slide 19: Phase 2 Clinical Trial – Randomized Patient Allocation
Phase 2 Clinical Trial – Randomized Patient Allocation For personal use only Patients with Moderate DDD Control Treatment MPC Dose being used in the Randomize being used in the Phase 3 Program Phase 3 Program Control Treatments MPC Treatments (n=40) (n=60) Saline Hyaluronic Acid 6M MPCs 18M MPCs (n=
Slide 20: Phase 2 Clinical Trial Study Population
Phase 2 Clinical Trial Clinical Study Patient Population For personal use only A prospective, multicenter, double blinded, controlled clinical study comparing two doses of immunoselected, culture- expanded, nucleated, allogeneic MPCs when combined with hyaluronic acid to two control intradiscal inje
Slide 21: Responder Criteria Used in Post-Hoc Assessment of Phase 2 Results
Phase 3 Responder Criteria Used in Post-Hoc Assessment of Phase 2 Clinical Trial Results For personal use only FDA Guidance on Minimally Important Change (MIC) for Pain and Function1 using a composite endpoint consistent with the CDRH IDE guidance for spinal systems (i.e. spine fusion & artificial d
Slide 22: Post-Hoc Phase 2 Results Provide Target Endpoints for Phase 3
Chronic Low Back Pain MPC-06-ID – Post-Hoc Phase 2 results provide target endpoints for Phase 3 trial For personal use only A: Phase 2: Treatment Success Responders1,2 B: Phase 2: Treatment Success Responders1,2 at 12 Months at both 12 & 24 Months 50.0% 50.0% 45.0% 46.6% 45.0% p = 0.032 p = 0.050 40
Slide 23: Phase 2 Clinical Trial – 36 Month Results
Phase 2 Clinical Trial – 36 Month Results MPC therapy provides durable results through 36 months For personal use only Over 36 months: − 82% of the 6 million MPC group who achieved the Phase 3 primary endpoint composite over 24 months maintained treatment success1 − 86% of the 6 million MPC group
Slide 24: MPC-06-ID – Ongoing US Phase 3 Clinical Trial
MPC-06-ID – Ongoing US Phase 3 Clinical Trial For personal use only Three-arm study comparing 6-million MPC with or without hyaluronic acid (HA)against saline control Primary efficacy endpoint agreed to with FDA: – Overall Treatment Success Composite at both 12 and 24 months as measured by: o At
Slide 25: MPC-06-ID – Development Strategy for US & Europe
MPC-06-ID – Development Strategy for US & Europe For personal use only Phase 3 trial in chronic low back pain completed enrollment in March 2018 with 404 patients randomized to receive MPC-06-ID or placebo. All patients have now completed at least 12 months of safety and efficacy follow-up. Foll
Slide 26: For personal use only
For personal use only