Japan : Victoria Business Forum

Transcription

1 Japan : Victoria Business Forum Silviu Itescu Chief Executive 25 August 2015

2 CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This presentation, including any comments made during or following the presentation, may contain forward-looking statements that are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. These statements may relate to, but are not limited to: expectations regarding the safety or efficacy of, or potential applications for, Mesoblast's adult stem cell technologies; expectations regarding the strength of Mesoblast's intellectual property, the timeline for Mesoblast's regulatory approval process, and the scalability and efficiency of manufacturing processes; expectations about Mesoblast's ability to grow its business and statements regarding its relationships with Teva Pharmaceutical Industries Ltd, JCR Pharmaceuticals Co., Ltd, and Lonza and future benefits of those relationships; statements concerning Mesoblast's share price or potential market capitalization; and statements concerning Mesoblast's capital requirements and ability to raise future capital, among others. Actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Factors and risks that may cause Mesoblast's actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, include, without limitation: risks inherent in the development and commercialization of potential products; uncertainty of clinical trial results or regulatory approvals or clearances; government regulation; the need for future capital; dependence upon collaborators; and protection of our intellectual property rights, among others. Accordingly, you should not place undue reliance on these forward-looking statements. 2

3 Mesoblast Competitive Strengths 1 Disruptive platform based on allogeneic, off-the-shelf adult stem cells 2 Substantial intellectual property covering products, uses, and manufacturing 3 Five product candidates that are in active Phase 3 programs or are Phase 3-ready 4 Scalable manufacturing capabilities 5 Strategic collaborations with industry leaders 6 Experienced management team 3

4 Paracrine Rationale for Effects Developing of Mesenchymal Our Mesenchymal Lineage Lineage Cells Adult Stem Cells (MLCs)* Traditional biological and chemical based therapies generally target single pathophysiological pathways. MLCs impact multiple downstream pathways and have the potential attributes for regeneration and repair: Detect injury and inflammation Respond to local stimuli and signals from the injured tissue Release a wide range of biomolecules (growth factors, chemokines, enzymes etc.) MLCs release multiple factors involved in: Cell survival and proliferation Cell and tissue repair Stimulation of mature blood vessel growth and reversal of endothelial dysfunction Inhibition or modulation of damaging inflammatory and immune responses Tissue remodelling by reduction in scar formation and fibrosis *MLCs comprise Mesenchymal Precursor Cells (MPCs ) and culture-expanded Mesenchymal Stem Cells (MSCs) 4

5 MLC Product Development Strategy Multiple mechanisms of action may facilitate therapeutic effects in diseases with high unmet needs Proprietary manufacturing processes utilized to create distinct product candidates with specific technical attributes Independent, Distinct Product Candidates Initial focus on advanced disease populations with commensurate pricing and potential for label expansion Allows for distinct product candidate reimbursement, commercialization and partnering strategies 5

6 Five Product Candidates in Active Phase 3 Programs or Phase 3-ready 6

7 New framework for Expedited Japanese Approval of Regenerative Medicines Legislative framework* The Pharmaceuticals, Medical Devices and Other Therapeutic Products Act (PMD Act) came into effect 25 November 2014 in Japan Established a framework for expedited approval in Japan for regenerative medical products Key Takeaways Conditional product approvals will be based on existing Phase 2 trial results demonstrating probable efficacy and safety with bridging studies in Japanese patients Conditional approvals will allow sales of each product candidate for up to 7 years Conditionally approved products will be covered by health insurance Conditional approvals will cover allogeneic cell therapy product candidates manufactured under GMP outside of Japan; and Full approval is expected to require further confirmation of safety and efficacy in a larger population. With late-stage clinical product candidates in multiple indications, we believe we are well positioned to take advantage of this new legislative framework in Japan 7 *PMDA slide presentation - 5 th Asian Cellular Therapy Organization Conference, Osaka; 9 November 2014

8 Our Strategy for Japan Population demographics changing - 40% of Japanese will be 65+ by 2050 Objectives To capitalize on clinical data generated to date, our strong intellectual property portfolio, and manufacturing know how to achieve accelerated product regulatory approvals in Japan To generate nearer term revenues in Japan, the world s second most established healthcare market In order to achieve our corporate objectives in Japan, we: Have completed prioritization of lead product candidates based on assessment of commercial opportunity and feasibility in Japan Will leverage existing Phase 2 clinical trial results from our lead product candidates Have engaged with product development and commercialization experts in Japan Have initiated dialog with the Japanese regulatory authority, the PMDA Are in active discussions with existing and potential commercialization partners for our product candidates in Japan 8 We believe the PMD Act will enable our cell therapy products to be made available sooner to patients with unmet medical needs, and to achieve nearer term revenues in Japan, ahead of other jurisdictions

9 MPC-150-IM: Congestive Heart Failure (CHF) Market Opportunity MPC-150-IM is in development for patients with New York Heart Association Class II-IV CHF 5.1m patients (2% of the population) diagnosed with CHF in the U.S. 1 Market opportunity 825,000 new cases diagnosed in the U.S. each year 1 Growing by 2% per annum ~1.7m CHF NYHA Class II-IV patients with low ejection fraction (<40%) in the US alone 2 Gap in treatment options Targeted physician population Class II / III CHF patients with low ejection fraction continue to be at high risk of repeated hospitalizations and mortality, despite standard of care pharmacological treatments 3 Class III / IV CHF patients only have heart transplant and mechanical support as treatment options Specialists: Targeted physician audience & commercial footprint Heart failure specialists Interventional cardiologists Cardiac surgeons We believe MPC-150-IM is positioned to fill the significant treatment gap in patients with advanced CHF 9 1. AHA statistical Update Heart disease and stroke statistics-2014 update Circulation Gurwitz JH, Magid DJ, Smith DH, et al. Contemporary Prevalence and Correlates of Incident Heart Failure with Preserved Ejection Fraction. The American journal of medicine. 2013;126(5): European Heart Journal (2012) 33, Figure 3

10 MPC-06-ID: Chronic Discogenic Low Back Pain (CDLBP) Market Opportunity MPC-06-ID is in development for the treatment of CDLBP lasting >6 months as a result of moderate degenerative intervertebral disc disease Market opportunity Prevalent CLBP population to grow to 21.9m patients in the U.S. by % of CLBP population seek treatment 1 ~40% of CLBP patients have a discogenic cause Gap in treatment options Targeted physician population For patients who fail conservative treatment (rest and analgesia) and epidural steroids, treatment options are limited to highly invasive therapies such as spinal fusion or artificial disc replacement Surgeons report ~40% of patients ultimately fail back surgery 2 Specialists: Targeted physician audience & commercial footprint Pain management specialists and anaesthesiologists Orthopedic / spine surgeons We believe MPC-06-ID is positioned to fill the significant treatment gap in patients with moderate to severe CDLBP after conservative treatment options have failed LEK & NCI opinion leader interviews, and secondary analysis 2. Shapiro CM Phys Med Rehabil Clin N Am 2014

11 MSC-100-IV: Acute Graft Versus Host Disease (GVHD) Market Opportunity MSC-100-IV /JR-031 is in development for pediatric and adult patients with acute GVHD following allogeneic hematopoietic stem cell transplant (HSCT) who have failed to respond to steroid treatment ~30,000 allogeneic HSCTs performed globally each year, 25% pediatric 1,2 Market opportunity No approved treatment options ~50% of all patients develop GVHD (Grades II-IV) 3 ~50% steroid-refractory acute (SR-a) GVHD 4 European study showed cost of treating patients with SR-a GVHD was approx US $325,000 5 Mortality can reach 85% in patients with liver & gut complications No currently approved therapies for steroid refractory patients Off-label options have mixed efficacy with high toxicity Significant need for a new treatment with a favorable risk / benefit profile Targeted physician population Highly targeted physician audience & commercial footprint for lead launch in pediatrics ~ 75 centers in the US conduct pediatric allogeneic HSCTs ~ 50% of all pediatric transplants concentrated in 15 centers & key metropolitan areas Gratwohl T el al, Quantitative and qualitative differences in use and trends of hematopoetic stem cell transplantation : a Global Observation study. Haematollogica 213;98(8) 2. CIBMTR, Decision resources GVHD Epi Nov Decision resources Niche Markets and Rare diseases: GVHD Nov Cahn et al prospective evaluation of 2 agvhd grading systems Blood Aug Svahn BM et al, Increased costs after allogeneic haematopoetic SCT are associated with major complications and re-transplantation BMT 2012; 47:

12 MSC-100-IV / JR-031 : Product Launch Plans in GVHD USA: Positive FDA meeting July 2014 clarified pathway for accelerated approval 60 patient open-label Phase 3 pediatric trial initiated Confirmatory Phase 3 adult trial design in liver / gut subset Manufacturing and commercial readiness BLA filing for pediatric registration on track Japan: JCR Pharmaceuticals Co Ltd., our GVHD partner in Japan, filed for regulatory approval for JR-031 in Japan in September 2014 and was granted orphan drug priority review MSC-100-IV/JR-031 have potential to be first allogeneic stem cell products approved in USA, Japan Halo effect for Mesoblast s commercial go-to-market capability 12

13 Portfolio Targeting Inflammatory Diseases A Major Emerging Opportunity MLCs have receptors that respond to pro-inflammatory signals, inducing release of multiple anti inflammatory mediators MLCs thereby target multiple immune pathways concurrently This should position MLC product candidates as ideal therapeutics for immune mediated diseases where multiple pathways are associated with disease activity for which there are no alternatives and/or resistance to other therapies MLCs have demonstrated favorable risk: benefit profiles in development programs, with potential to be differentiated from other biologics that have infectious or neoplastic complications Mesoblast is developing MLC product candidates to target Diabetic kidney disease Biologic-refractory rheumatoid arthritis Biologic-refractory Crohn s disease 13

14 Extensive Intellectual Property Portfolio 652 patents across 67 families Composition of matter (COM) and manufacturing process Specific therapeutic applications Complementary technologies and additional candidates patents or patent applications Valid through * MPCs 58 patents or patent applications related to MPC composition of matter or methods of isolation, expansion and manufacture of MPCs start to expire 2020* and extend to 2029* MSC s 48 granted patents or patent applications related to MSC composition of matter and manufacture of MSCs start to expire 2018* and extend to 2035* DPSCs 32 patents or patent applications start to expire 2021* and extend to 2024* In the last 12 months we have had 33 new patents granted including 9 in the US, 6 in Japan, 5 in China and 13 in other jurisdictions *Excludes possible patent term extension 378 patents or patent applications Valid through 2035* Immunologic / inflammatory disorders 99 patents or patent applications start to expire 2019* and extend to 2035* Cardiovascular disorders 67 patents or patent applications start to expire 2018* and extend to 2024* Orthopedic disorders 63 patents or patent applications start to expire 2017* and extend to 2032* Oncology / hematology 96 patents or patent applications start to expire 2019* and extend to 2030* Other therapeutic applications 53 patents or patent applications start to expire 2027* and extend to 2032* 136 patents or patent applications Valid through * Cell-based complementary technologies 62 patents or patent applications - start to expire 2017* and extend to 2030* SDF-1 26 patents or patent applications start to expire 2027* and extend to 2032* Factors and agents for cardiovascular and other fibrotic indications 48 patents or patent applications start to expire 2021* and extend to 2024*

15 Scalable Manufacturing Capabilities: Partnership with Lonza Manufacturing objectives Distinct manufacturing processes for each product Commercial scale processes with batch-to-batch consistency and reproducible release criteria Ensure commercial product supply is aligned with projected market needs 15

16 Strategic Collaborations Manufacturing Development Commercialization Manufacturing R&D Clinical research Heart failure CHF program MPC-150-IM Global rights Additional CV & CNS programs GVHD Japan Japan GVHD rights Manufacturing CHF clinical research Back pain partner options Global rights 16

17 Thank You