Slide 1: 2014 Results & Corporate strategy
For personal use only 2014 Results & Corporate strategy Focus on late stage product development 26 August 2014
Slide 2: Cautionary Note Regarding Forward-Looking Statements
For personal use only 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 quantifi
Slide 3: Significant achievements to date
Significant achievements to date For personal use only Successful translation of three cell-based technology platforms for multiple applications Robust IP portfolio of >60 patent families provides long-term commercial protection for mesenchymal lineage cells Portfolio of allogeneic regenerativ
Slide 4: 2014 highlights
2014 highlights For personal use only FDA clears 1730 patient Mesenchymal Precursor Cell (MPC) Phase 3 trial in NYHA class II/III heart failure; actively recruiting across multiple North American sites NIH and MSB agree on 120 patient MPC trial in advanced / NYHA class IV heart failure End of
Slide 5: Financial Results
Financial Results AUDm 30 June 14 30 June 13 Change % For personal use only Revenue from Continuing Operations 26.0 28.8 (2.8) (10) Commercialization revenue 16.4 18.3 (1.9) (10) Interest revenue 9.6 10.5 (0.9) (9) Other Income 11.1 5.9 5.2 88 Research & development tax incentive 8.6 5.9 2.7 46 Writ
Slide 6: Operating Cash Burn Rate
Operating Cash Burn Rate AUDm 30 June 2014 30 June 2013 For personal use only Cash on hand * 196.4 315.3 Operating Cash Burn 81.9 54.1 Adjustments: Manufacturing timing of payments (4.6) 4.6 One-off settlement of a provision (5.9) - Corporate taxation refunds 6.8 3.3 Normalized Operating Cash Burn 7
Slide 7: Corporate Strategic Imperatives
Corporate Strategic Imperatives For personal use only 1 Clinically differentiated products 2 Focus on bringing late stage products to market 3 Enable manufacturing scale up to meet demands of portfolio Leverage expanding talent base to continue to establish a culture of shared 4 leadership and accou
Slide 8: 1. Clinically differentiated products
1. Clinically differentiated products For personal use only Development of product-specific intellectual property Targeting of multiple indications based on sound science and unmet medical need Establishment of product specific pricing based on value based outcomes Parallel licensing/partner
Slide 9: 2. Focus on bringing late stage products to market
2. Focus on bringing late stage products to market For personal use only Products with mid term revenue streams MSC: GVHD – “Halo” niche product near to market, premium priced, PYS ˃$250m MPC: Chronic Discogenic Low Back Pain – low dose, large market, PYS USA ˃$2bn MPC: Heart Failure – large m
Slide 10: Robust Development Portfolio of Tier 1 and Tier 2 products
Robust Development Portfolio of Tier 1 and Tier 2 products For personal use only 9
Slide 11: MSC-100-IV: Graft vs Host Disease – Market Opportunity
MSC-100-IV: Graft vs Host Disease – Market Opportunity MSC-100-IV is in development for pediatric and adult patients with acute Graft versus Host Disease (GVHD) For personal use only following allogeneic hematopoietic stem cell transplant (HSCT) who have failed to respond to steroid treatment Market
Slide 12: MSC-100-IV: Graft vs Host Disease – Evidence Of Efficacy
MSC-100-IV: Graft vs Host Disease – Evidence Of Efficacy For personal use only Pediatric Patients 6 Adult Patients 7 - Expanded Access Program in US has treated in excess of 200 - In a randomized, placebo-controlled Phase 3 trial, MSC- patients 100-IV significantly improved overall responses in the
Slide 13: MSC-100-IV: Graft vs Host Disease – Product Launch
MSC-100-IV : Graft vs Host Disease – Product Launch For personal use only • JCR Pharmaceuticals plans to file for registration H2 2014 • Japanese launch 2015 • Canadian launch 2016 • New Zealand launch 2016 • Positive FDA meeting June 2014 - pathway for accelerated approval clarified • 60 patient op
Slide 14: MPC-06-ID: Chronic Discogenic Low Back Pain – Market Opportunity
MPC-06-ID: Chronic Discogenic Low Back Pain (CDLBP) – Market Opportunity MPC-06-ID is in development for the treatment of chronic low back pain (CLBP) lasting >6 months For personal use only as a result of moderate degenerative intervertebral disc disease Market Opportunity Gap in Treatment Options
Slide 15: MPC-06-ID: CDLBP – Phase 2 Results
MPC-06-ID: CDLBP – Phase 2 Results Phase 2 trial: 100 patients with > 6 months of discogenic low back pain failing other therapies were evaluated in For personal use only a randomized, placebo controlled trial that compared saline, HA, HA+6M MPC, HA + 18M MPC’s injected into culprit painful disc % P
Slide 16: MPC-06-ID: CDLBP – Composite Endpoint for Phase 3
MPC-06-ID: CDLBP – Composite Endpoint for Phase 3 Treatment options for CDLBP must address both decreased pain and improved function, defined as Minimally For personal use only Important Changes – at least 30% improvement in VAS and at least 10 point improvement in Oswestry Disability Index (ODI) 10
Slide 17: MPC-150-IM: Congestive Heart Failure – Market Opportunity
MPC-150-IM: Congestive Heart Failure (CHF) – Market Opportunity For personal use only MPC-150-IM is in development for New York Heart Association (NYHA) class II to IV heart failure Market Opportunity Gap in Treatment Options Targeted Physician Population 5.1 million people in the US are Despite
Slide 18: MPC-150-IM: Congestive Heart Failure – Phase 2 Results
MPC-150-IM: Congestive Heart Failure (CHF) – Phase 2 Results The MPC dose for the Phase 3 trial was chosen on the basis of results from a 60-patient Phase 2 trial For personal use only Kaplan-Meier Analysis for Time-to-First HF-MACE 150M vs Control Treatments (36 Months Follow-up) Phase 3 primary ef
Slide 19: MPC-150-IM: Congestive Heart Failure – Trials underway
MPC-150-IM: Congestive Heart Failure (CHF) – Trials underway For personal use only New York Heart Association (NYHA) classification of heart failure NYHA Class I NYHA NYHA Class II NYHA Class III Class 32% 29% 30% IV 9% Phase 3 CHF TEVA NIH LVAD Trial 1730 patients 120 patients NYHA Class II/III P
Slide 20: 3. Objectives of commercial scale manufacturing
3. Objectives of commercial scale manufacturing For personal use only 1. Distinct manufacturing processes for each product 2. Commercial scale processes with batch-to- batch consistencies and reproducible release criteria 3. Ensure commercial product supply is aligned with projected market needs Wor
Slide 21: Manufacturing IP enhanced as well as IP for compositions and indications
Manufacturing IP enhanced as well as IP for compositions and indications For personal use only • MPCs • MSCs Composition • DPSC • Expanded HSCs of matter • SDF-1 • Isolation • Expansion • Purification • Manufacturing scale up Manufacturing • Cryopreservation process • Release testing • Storage and d
Slide 22: 4. Create a product-focused organizational structure
4. Create a product-focused organizational structure For personal use only CEO MPC: Heart MSC: GvHD MPC: CLBP Clinical Regulatory Failure Product Manufacturing Commercial Research Support* Product Head Product Head Operations & Quality Head Clinical Operations Resources Regulatory & Quality Resource
Slide 23: Build an experienced team for late-phase clinical execution and market launch
Build an experienced team for late-phase clinical execution and market launch Location For personal use only Industry Qualification Background Level Function 22
Slide 24: 5. Build strategic partnerships
5. Build strategic partnerships For personal use only Manufacturing Development Commercialisation Heart Quintiles Failure Teva EDB GVHD MSB MSB Japan JCR Lonza Back Pain NIH Partner Options 23
Slide 25: Use of financial resources
Use of financial resources For personal use only Allocation of Funds Fully fund through launch Funded to next value inflection Budgeted Life Cycle R&D Further sources of funds include: Strategic Alliances, Government Partnering, Capital Markets and Product Revenues 24
Slide 26: Outlook for next months
Outlook for next 12 months For personal use only GVHD – Unmet need with near term milestones • Pediatric trial recruitment to support BLA filing for accelerated approval in US • Parallel US trial in adults with liver/gut GVHD • Commercial manufacturing to support product launches • JCR partner to fi
Slide 27: References
References For personal use only 1. 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 2012 3. Decision resources Niche Markets and Ra
Slide 28: Questions / Thank you
For personal use only Questions / Thank you 27