World-leading expertise in Gene and Cell Therapy 2014 Preliminary Results – March 2015

Embed Size (px)

Citation preview

  • Slide 1
  • World-leading expertise in Gene and Cell Therapy 2014 Preliminary Results March 2015
  • Slide 2
  • 2 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhancedMean 26% --- 2 Forward-looking statements This presentation does not constitute an offer to sell or a solicitation of offers to buy Ordinary Shares (the Securities). Although reasonable care has been taken to ensure that the facts stated in this presentation are accurate and that the opinions expressed are fair and reasonable, the contents of this presentation have not been formally verified by Oxford BioMedica plc (the Company) or any other person. Accordingly, no representation or warranty, expressed or implied, is made as to the fairness, accuracy, completeness or correctness of the information and opinions contained in this presentation, and no reliance should be placed on such information or opinions. Further, the information in this presentation is not complete and may be changed. Neither the Company nor any of its respective members, directors, officers or employees nor any other person accepts any liability whatsoever for any loss howsoever arising from any use of such information or opinions or otherwise arising in connection with this presentation. This presentation may contain forward-looking statements that reflect the Company's current expectations regarding future events, its liquidity and results of operations and its future working capital requirements. Forward-looking statements involve risks and uncertainties. Actual events could differ materially from those projected herein and depend on a number of factors, including the success of the Company's development strategies, the successful and timely completion of clinical studies, securing satisfactory licensing agreements for products, the ability of the Company to obtain additional financing for its operations and the market conditions affecting the availability and terms of such financing.
  • Slide 3
  • 3 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 3 Operational Highlights IP, technology and manufacturing capability is validated by Novartis Major new licensing and manufacturing contract signed with Novartis in October 2014 Worth up to $90 million over the next three years with royalties on CTL-019 Pipeline advanced Four clinical programmes in active development and two being prepared for Phase I/II trials RetinoStat recruitment completed in Phase I trial New CART-T 5T4 programme initiated in-house, combining both OXBs LentiVector and 5T4 technology platforms 2.2 million grant received from the TSB (now Innovate UK) to fund a Phase I/II clinical trial of OXB-102 Sanofi licensed global rights to StarGen and UshStat across all ocular indications Revenues increased Licensing revenues increased to 5.1 million (2013: 1.0 million) Manufacturing revenue increased to 7.7 million (2013: 2.6 million) R&D collaboration revenue of 0.8 million (2013: 1.7 million)* *Representing residual revenue under the 2009 Sanofi agreement
  • Slide 4
  • 4 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 4 Financial Highlights Total revenues of 13.6 million (excluding grants) in 2014 (2013: 5.4 million) Total revenues include profit-generating revenues of 7.7 million (2013: 2.6 million) Cash used in operations before capital expenditure of 7.4 million (2013: 13.0 million) Cash burn (net cash used in/generated from operations plus sales and purchases of non-current assets and interest received) of 11.6 million (2013: 11.9 million) 14.2 million cash balance at end 2014 (2.2 million at the start of the year) Balance sheet strengthened via fundraising in June raising 20.1 million (net)
  • Slide 5
  • 5 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 5 Oxford BioMedicas business model Research & Development Proprietary gene and cell therapy pipeline + Revenues Government funding Licence feesMilestonesRoyalties Manufacturing and process development OXB Solutions Contracts for lentiviral vector manufacture and process development IP Ownership Key IP makes OXB an essential partner for companies wanting to commercialise lentiviral vector based products +
  • Slide 6
  • 6 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 6 Gene therapy & cell therapy Treating disease by altering genes/DNA in patients cells Most commonly used viral vectors - Lentivirus or Adeno-Associated virus (AAV) Cells modified in vivo or ex vivo In vivo gene therapy, Lentiviral or AAV based vectors Ex vivo cell therapy (e.g. bone marrow stem cells, T cells), only Lentiviral based vectors Potential for one shot treatment giving long-term or permanent efficacy Explosion of interest in gene and cell therapy in last 2-3 years, e.g. In vivo Avalanche Biotech, Dimension Therapeutics, GenSight, NightstaRx, Spark Therapeutics, Voyager Therapeutics Ex vivo Bellicum Pharmaceuticals, Bluebird Bio, Juno Therapeutics, Kite Pharma, Novartis Collaborations Amgen/Kite Pharma, Astellas/Harvard Medical School, GSK/Adaptimmune, Lilly/Immunocore, Pfizer/Cellectis, Sanofi/Voyager OXB now attracting significant attention from leading players in this field
  • Slide 7
  • 7 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 7 Gene therapy & cell therapy In vivo ProSavin (Parkinsons disease) RetinoStat (Wet AMD) Ex vivo 1.OXB produces GMP lentiviral vector encoding CAR targeting CD19 2.White blood cells isolated from patients 3.Vector used to transduce expanded T-cells 4.The modified T-cells are infused back into the patient 5.Once inside the patient, the T-cells multiply, hunt cancer cells and destroy them.
  • Slide 8
  • 8 Slide for large image 8 OXBs unique capabilities Lentiviral vector advantages over AAV Larger therapeutic payloads Lentiviral vectors can be used to permanently genetically modify dividing cells such as T-cells or stem cells (unlike AAV) used by Novartis for CTL019 Process development Lentiviral vector dominant patent estate & know-how Analytical development Proprietary analytics Manufacturing capacity Cell/Vector engineering Clinical & regulatory expertise Proprietary development portfolio OXB Solutions
  • Slide 9
  • Oxford BioMedica 2011, all rights reserved 9 Product Portfolio Oxford BioMedica 2011, all rights reserved
  • Slide 10
  • 10 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 10 Portfolio of pipeline assets (excluding those already out-licensed) ProductIndicationStageNext inflection Est. date Lentiviral vector TECHNOLOGY OPHTHALMOLOGYRetinoStat Wet AMDPhase I follow up stage (primary end point met) Phase I CSR2015 EncorStat Corneal graft rejectionPhase I/II preparationFPI Phase I/II2016 Glaucoma-GTChronic glaucomaPre-clinicalEnd pre-clinical2016 CNS ProSavin OXB-102 Parkinsons disease Phase I/II complete Phase I/II preparation FPI Phase I/II2016 MoNuDin Motor neuron diseaseResearchEnd pre-clinical2015 NEW IDEAS Investigating several therapy areas where Lenti based vectors have an advantage over AAV due to payload capacity TBD Exploring possibilities to enter cell therapy field in our own right e.g. CAR-T 5T4 5T4 TECHNOLOGY ONCOLOGY TroVax Cancer (multiple)3 x Phase II ongoingEnd Phase II2015/16 CAR-T 5T4Cancer (multiple)Pre-clinicalEnd pre-clinical2016
  • Slide 11
  • 11 5T4 technology products 1.Datamonitor, 2010 TroVax Cancer therapies & immunotherapy market forecast to increase to $36.8 billion by 2019 1 TroVax targets 5T4, onco-foetal tumour antigen expressed on surface of majority of solid tumours, stimulating immune system to destroy cancerous cells Clinical trials show safety in >500 patients; analyses show clear indication of efficacy Patients likely to respond to TroVax can be identified by a simple blood test CAR-T 5T4 A gene modified autologous T cell engineered with lentiviral vector to express an antibody against 5T4; delivered by IV infusion Acts by re-directing a patients T cells to recognise the 5T4 tumour antigen and kill the cell expressing it
  • Slide 12
  • 12 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 12 Licences to OXBs IP and products CompanyProductsTerms Estimated launch date Estimated Peak Yr Sales Lenti based vector IP & Know-how NovartisCTL019/ Other CAR-T $10m upfront Undisclosed royalties 2017Multi billion $ GSKUp to 6 rare orphan diseases Not disclosedTBD$10m$50m per product ProductsSanofiStarGenUndisclosed development milestones and royalties 2021$500m SanofiUshStat 2021$90m 5T4 Tumour antigen IPPfizer5T4 antibodyUndisclosed2023>$300m IPImaginAb5T4 imaging diagnostic Undisclosed2024$10m PrimeBoost IPBavarian NordicPROSTVAC TM Undisclosed2017$60m
  • Slide 13
  • Oxford BioMedica 2011, all rights reserved 13 Novartis contract
  • Slide 14
  • 14 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 14 CTL019 FDA Breakthrough Therapy designation Designation supports the advancement of CTL019 to help address the unmet need of patients with relapsed/refractory acute lymphoblastic leukaemia (r/r ALL) Intensive guidance from FDA through development Rolling review Expedited approval 90% of patients experienced complete remissions and sustained remissions of two years with CTL019 (The New England Journal of Medicine, October 2014) Phase II study in paediatric ALL expected to start H1 2015 CAR-T products have very substantial peak year sales potential
  • Slide 15
  • 15 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 15 Novartis contracts (October 2014) Initial contract May 2013 proved our capabilities October 2014 contracts include Non-exclusive licence to OXBs lentiviral vector platform IP in oncology Process development collaboration Arising IP owned by OXB, NVS have exclusive licence to arising IP in CAR-T cell products Initial 3 year manufacturing contract for clinical supply for NVS CTL019 programme potential to extend Financial terms include $4.3m equity investment IP licence $9.7m non-refundable upfronts Undisclosed royalties on CTL019 and other CAR-T products Manufacturing and process development Up to $76m over 3 years
  • Slide 16
  • Oxford BioMedica 2011, all rights reserved 16 Financial update
  • Slide 17
  • 17 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhancedMean 26% --- 17 2014 financial highlights 1 Total revenues increased to 13.6m (2013 5.4m) Recurring profit generating revenues increased to 7.7m (2013 2.6m) Cash used in operations (before capex) reduced to 7.4m (2013 13.0m) 1.Audited financial results 2.Aggregate of net cash used in operating activities, purchase of non-current assets and interest received million20142013 Revenue13.65.4 Operating loss before interest and tax(10.6)(12.8) Loss after interest and tax(8.7)(11.1) Cash burn 2 (11.6)(11.9) Cash/cash equivalents at 31 December14.22.2
  • Slide 18
  • 18 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 18 Revenues 2014 vs 2013 m
  • Slide 19
  • 19 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 19 Cash flow millions20142013 Operating loss before interest and tax(10.6)(12.8) Non-cash P&L items1.51.4 Working capital incl deferred income1.7(1.6) Cash used in operations(7.4)(13.0) R&D tax credit1.62.0 Purchase of non-current assets(5.6)(0.9) Interest paid/other (0.2) - Cash burn 1 (11.6)(11.9) Financing activities 23.6 - Increase/(decrease) in cash 2 12.0(11.9) Cash balance 2 14.22.2 1.Aggregate of net cash used in operating activities, purchase of non-current assets and interest received
  • Slide 20
  • 20 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 20 Financial outlook for 2015 Continuing to build revenues from OXB Solutions business Deliver Novartis requirements Strive to secure contracts with other companies Continue to reduce underlying operating cash burn Investing in our manufacturing capabilities Partially funded by 7.1m AMSCI grant and loan package Investing in our product candidates Potential licence income Further LentiVector technology licences Building towards royalty income when CTL-019 comes to market
  • Slide 21
  • Oxford BioMedica 2011, all rights reserved 21 Summary Oxford BioMedica 2011, all rights reserved
  • Slide 22
  • 22 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 22 Upcoming potential value driving news flow 2015RetinoStat Phase I final data results expected, ready for Phase II and/or partnering Long term (3 year) follow up on Prosavin patients Identification of new product development candidates Further IP licences/manufacturing/process development contracts Read out from TroVax Phase II studies Read out from MoNuDin preclinical results 2016FPI OXB-102 clinical programme FPI EncorStat clinical programme StarGen/UshStat development milestones Glaucoma-GT pre-clinical results CAR-T 5T4 pre-clinical results Plus Novartis newsflow on CTL019 product
  • Slide 23
  • 23 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 23 Summary & outlook OXB is a unique and sector-leading gene/cell therapy business Three-pronged strategy: 1) pipeline, 2) IP revenues, 3) OXB Solutions IP and technical capabilities validated by Novartis, GSK, Sanofi etc Valuable pipeline of product development candidates in progress 4 wholly-owned assets in Phase 1/II clinical development Out-licensed programmes in clinical trials with Sanofi, Pfizer and ImaginAb Further potential product development opportunities being evaluated including CAR-T 5T4 Significant revenue potential from Novartis including royalty stream from CTL-019 Further licensing and royalty income beyond Novartis contract to fund own pipeline Potential to be cash positive by end-2016 Commercially-driven and strong team now in place to execute OXBs strategy
  • Slide 24
  • 24 Cohort 2 DoseAdministration3 months (UPDRS)6 months (UPDRS)1 year (UPDRS)2 years (UPDRS) 1, n=31xOriginalMean 27% Max. up to 30% Mean 30% Max. up to 50% Mean 29% Max. up to 44% Mean 20% Max. up to 30% 2, n=32xOriginalMean 28% Max. up to 53% Mean 34% Max. up to 53% Mean 29% Max. up to 56% - 3, n=32xEnhanced Mean 26% --- 24 Contact us Oxford BioMedica plc Windrush Court Transport Way Oxford OX4 6LT United Kingdom www.oxfordbiomedica.co.uk www.oxfordbiomedica.co.uk www.oxbsolutions.co.uk [email protected] John Dawson, CEO Tim Watts, CFO Tel: +44 (0) 1865 783 000