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