World-leading expertise
in Gene and Cell Therapy
27th Annual ROTH Conference
March 2015
Disclaimer
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“presentation” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed
Cohort2 the presentationDose
Administration
months (UPDRS)
1 year (UPDRS)
2 years (UPDRS)
during
meetings or any presentation
to which this 3document
relates. 6 months (UPDRS)
Although
ensure that the facts stated
in this presentation
are
accurate and Mean
that the
opinions expressed
are fair and
Mean 27%
Mean
30%
29%
Mean 20%
1, n=3 reasonable care
1x has been taken to Original
Max.
up to 30%
Max. up to 50%
Max. up to 44%
up to 30%
reasonable, the contents of this presentation have not been formally
verified
by Oxford BioMedica
plc (the “Company”)
or any other Max.
person.
Accordingly,
no representation or warranty, expressed or implied, is made as to the fairness, accuracy, completeness or correctness of the information and opinions
Mean 34%
29%in this presentation
2, n=3 in this presentation,
2x
Original
contained
and no reliance
should be placed onMean
such 28%
information or opinions.
Further, the Mean
information
is not
Max. up to 53%
Max. up to 53%
up to 56%
complete and may be changed. Neither the Company nor any of its
respective members,
directors, officers orMax.
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
Mean 26%
3, n=3
2x
Enhanced
presentation.
This presentation does not constitute an offer to sell or a solicitation of offers to buy Ordinary Shares in the Company (the “Securities”). The Securities
have not been registered under the US Securities Act of 1933, as amended (the “Securities Act”), or qualified for sale under the law of any state or other
jurisdiction of the United States of America and may not be offered or sold in the United States of America except pursuant to such registration or an
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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.
By participating in this presentation you agree to be bound by the foregoing restrictions and the other terms of this disclaimer.
2
Overview
•
OXB is a unique and sector-leading gene/cell therapy business
•
Proprietary technologies
Cohort2
Dose
1, n=3
2, n=3
Administration
3 months (UPDRS)
6 months (UPDRS)
2 years (UPDRS)
•
Mean
27%
Mean 30%
1x delivery platform
Original based on
Gene
lentiviral
vector
IP
Max. up to 30%
Max. up to 50%
Mean 29%
Mean 20%
Max. up to 44%
Max. up to 30%
•
Mean 28%
Original
5T4 2x
antigen/antibody
in immunotherapy
Max. up to 53%
Mean 34%
Mean 29%
-
Max. up to 53%
Max. up to 56%
3, n=3
•
Pipeline 2xof product development
candidates
Mean
26%
Enhanced
-
•
Business model
•
1 year (UPDRS)
-
•
Development of gene/cell therapy products for out-licence or continuing in-house
•
Licence revenues from IP and products
•
Revenues from lentiviral vector manufacture and process development
-
IP and technical capabilities validated by
•
Lentiviral vector IP – Novartis, GSK
•
Product licences – Sanofi (StarGen™/UshStat®)
•
Process development – Novartis, ImmuneDesign, others
•
Potential to be cash positive by end-2016
•
Experienced management team
3
Corporate history and financing
• Spun out of Oxford University in 1996
Cohort2
Dose
Administration
3 months (UPDRS)
6 months (UPDRS)
1 year (UPDRS)
2 years (UPDRS)
1, n=3
1x
Original
Mean 27%
Mean 30%
Mean 29%
Mean 20%
Max. up to 30%
Max. up to 50%
Max. up to 44%
Max. up to 30%
Mean 34%
Mean 29%
-
Max. up to 53%
Max. up to 56%
Mean 28%
Original
•2, n=3London2x Stock Exchange
Max. up to 53%
3, n=3
Mean 26%
Enhanced
• AIM2xIPO – December
1996
• Admitted to Main Market – April 2001 (Ticker OXB.L)
-
• Market capitalisation - $327m (March 3, 2015)
• Daily liquidity - $600k (average last 3 months)
4
Gene therapy & cell therapy
•
Treating disease by altering genes/DNA in patients cells
•
Cohort2
1, n=3
•
Dose
Administration
3 months (UPDRS)
6 months (UPDRS)
1 year (UPDRS)
Most commonly
used viral
vectors - Lentivirus
or Adeno-Associated
virus
(AAV)
Mean
27%
Mean
30%
Mean
29%
1x
Original
Cells modified in vivo or ex vivo
2, n=3
•
3, n=3•
Max. up to 30%
Mean 28%
2x
Original
In vivo – gene therapy, Lenti or AAV based
vectors
Max. up to 53%
2 years (UPDRS)
Mean 20%
Max. up to 50%
Max. up to 44%
Max. up to 30%
Mean 34%
Mean 29%
-
Max. up to 53%
Max. up to 56%
Ex vivo2x– cell therapy (e.g.
bone marrow
stem
Mean
26%cells, T- cells), only Lenti
Enhanced
- 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
5
Gene therapy & cell therapy
In vivo
Cohort2
Dose
Administration
3 months (UPDRS)
1, n=3
1x
Original
Mean 27%
ProSavin® (Parkinson’s disease)
2, n=3
3, n=3
2x
2x
Original
Enhanced
Ex vivo
6 months (UPDRS)
1 year (UPDRS)
2 years (UPDRS)
Mean 30%
Mean 29%
Mean 20%
Max. up to 30%
Max. up to 50%
Max. up to 44%
Max. up to 30%
Mean 28%
Mean 34%
Mean 29%
-
Max. up to 53%
Max. up to 53%
Max. up to 56%
Mean 26%
-
-
-
RetinoStat® (Wet AMD)
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.
6
OXB’s unique capabilities
Slide for large image
Proprietary
development
portfolio
Lentiviral vector advantages
Cell/Vector
engineering
Process
development
over AAV
• Larger therapeutic payloads
Lentiviral vector
dominant patent
estate & know-how
OXB
Solutions
• Lentiviral vectors can be
Manufacturing
capacity
used to permanently
genetically modify dividing
cells such as T-cells or stem
Clinical &
regulatory
expertise
Analytical
development
cells (unlike AAV) – used by
Proprietary
analytics
Novartis for CTL019
7
Oxford BioMedica’s business model
Cohort2
1, n=3
Dose
Research
&
1x
Development
2, n=3
3, n=3
Administration
2x
Original
Max. up to 30%
+
Original
Proprietary gene
and cell therapy
2x
pipeline
OXB
Mean 27%
Solutions
3 months (UPDRS)
Enhanced
IP Ownership
6 months (UPDRS)
1 year (UPDRS)
Mean 30%
Mean 29%
Max. up to 50%
Max. up to 44%
Mean
28%
Contracts
+
for Mean 34%
Max. up to 53%
lentiviral vector
manufacture
and Mean 26%
process
development
Max. up to 53%
2 years (UPDRS)
Mean 20%
Max. upOXB
to 30%
Key IP makes
an essential partner
Mean 29%
for companies
Max. up to 56%
wanting to
commercialise
lentiviral vector
based products
Revenues
Government
funding
Licence fees
Manufacturing and
process development
Milestones
Royalties
8
Product Portfolio
© Oxford BioMedica 2011, all rights reserved
9
Portfolio of pipeline assets (excluding those already out-licensed)
Cohort2
Dose
Product
Administration
Lentiviral
vector TECHNOLOGY
1, n=3
1x
Original
OPHTHALMOLOGY
2, n=3
6 months (UPDRS)
1 year (UPDRS)
Est.
2 years (UPDRS)
date
Mean 27%
Mean 30%
Mean 29%
Mean 20%
Max. up to 30%
Max. up to 50%
Max. up to 44%
Max. up to 30%
Indication
RetinoStat®
3 months (UPDRS)
2015
EncorStat® Enhanced Corneal graft
Meanrejection
26%
Glaucoma-GT
Chronic glaucoma
Phase
I/II preparation
FPI Phase I/II-
2016
Pre-clinical
End pre-clinical
2016
ProSavin®
OXB-102
Parkinson’s disease
Phase I/II complete
Phase I/II
preparation
FPI Phase I/II
2016
MoNuDin®
Motor neuron disease
Research
End pre-clinical
2015
TBD
TBD
Original
Mean 28%
Max. up to 53%
3, n=3
2x
CNS
NEW IDEAS
Next inflection
Phase I follow up
Phase I CSR
stage
(primary endMean 29%
Mean 34%
Max. up to 53%
Max. up to 56%
point
met)
2x
Wet AMD
Stage
Investigating several therapy areas where Lenti based vectors have
an advantage over AAV due to payload capacity
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 ongoing
End Phase II
2015/16
CAR-T 5T4
Cancer (multiple)
Pre-clinical
End pre-clinical
2016
10
5T4 technology products
TroVax®
•
•
•
•
Cancer therapies & immunotherapy market forecast to increase to $36.8 billion by 20191
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 patient’s T cells to recognise the 5T4 tumour antigen and kill the cell
expressing it
1.
Datamonitor, 2010
11
Licences to OXB’s IP and products
Cohort2
Dose
Administration
1, n=3
1x
Company
Original
Products
Lenti based vector
2, n=3
IP &
Know-how
3, n=3
Products
2x
Novartis
2x
Original
CTL019/
Other CAR-T
Enhanced
3 months (UPDRS)
Mean 27%
Terms
6 months (UPDRS)
Mean 30%
Max. up to 30%
Max. up to 50%
Mean 28%
Mean 34%
Mean 26%
-
$10m
upfront
Max. up to
53%
Max. up to 53%
Undisclosed royalties
1 year (UPDRS)
Estimated
Mean
29%
launch
date
Max. up to 44%
Mean 29%
2017Max. up to 56%
-
2 years (UPDRS)
Estimated
Mean Yr
20%
Peak
Sales
Max. up to 30%
-
“Multi billion $”
-
GSK
Up to 6 rare
orphan
diseases
Not disclosed
TBD
$10m—$50m
per product
Sanofi
StarGen™
Undisclosed development
milestones and royalties
2021
$500m
Sanofi
UshStat®
2021
$90m
5T4 Tumour antigen
IP
Pfizer
5T4 antibody
Undisclosed
2023
>$300m
IP
ImaginAb
5T4 imaging
diagnostic
Undisclosed
2024
$10m
Bavarian Nordic
PROSTVACTM
Undisclosed
2017
$60m
PrimeBoost
IP
12
Novartis
contract
© Oxford BioMedica 2011, all rights reserved
13
CTL019
•
FDA Breakthrough Therapy designation
•
Cohort2
1, n=3
•
2, n=3
•
3, n=3
•
Dose
Administration
months
(UPDRS)
6 months
(UPDRS)
1 year (UPDRS)
Designation
supports the
advancement3 of
CTL019
to help
address
the unmet
with relapsed/refractory
acute
lymphoblastic
Mean 27%
Mean
30%
Mean 29%
1x need of patients
Original
Max.
up
to
30%
Max.
up
to
50%
Max. up to 44%
leukaemia (r/r ALL)
Intensive2xguidance fromOriginal
FDA through development
Mean 28%
Mean 34%
Mean 29%
Max. up to 53%
Max. up to 53%
Max. up to 56%
Rolling review;
Mean 26%
2x
Enhanced
Expedited
approval
2 years (UPDRS)
Mean 20%
Max. up to 30%
-
•
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
14
Novartis contracts (October 2014)
•
Initial contract May 2013 – proved our capabilities
Cohort2
Dose
Administration
•1, n=3October 2014
contracts
include
1x
Original
2, n=3
3, n=3
•
6 months (UPDRS)
1 year (UPDRS)
2 years (UPDRS)
Mean 27%
Mean 30%
Mean 29%
Mean 20%
Max. up to 30%
Max. up to 50%
Max. up to 44%
Max. up to 30%
Non-exclusive licence to OXB’s lentiviral vector platform IP in oncology
Mean 28%
Mean 34%
Mean 29%
2x
Original
•
Process development collaboration
2x
•
•
•
3 months (UPDRS)
Enhanced
Max. up to 53%
Max. up to 53%
Max. up to 56%
Mean 26%
-
-
-
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
15
Summary
© Oxford BioMedica 2011, all rights reserved
16
Upcoming potential value driving news flow
Cohort2
Dose
1, n=3
1x
3, n=3
3 months (UPDRS)
6 months (UPDRS)
1 year (UPDRS)
2 years (UPDRS)
Original
Mean 27%
Mean 30%
Mean 29%
Mean 20%
RetinoStat®
2015
2, n=3
Administration
2x
2x
up to 30%
Phase I final data results expected, ready for Phase II and/orMax.
partnering
Mean
Mean 34%
Mean 29%
Original
Long term (3
year) follow up
on28%
Prosavin patients
Max. up to 30%
Max. up to 50%
Max. up to 44%
Max. up to 53%
Max. up to 53%
Max. up to 56%
Identification of new product development candidates
Mean 26%
Enhanced
Further IP licences/manufacturing/process development contracts
-
Read out from TroVax® Phase II studies
Read out from MoNuDin® preclinical results
2016
FPI 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
17
Summary
•
Lentiviral vector IP recognised by Novartis, GSK
•
Internal clinical programmes
Cohort2
Dose
Administration
3 months (UPDRS)
6 months (UPDRS)
1 year (UPDRS)
2 years (UPDRS)
1, n=3
1x
Original
Mean 27%
Mean 30%
Mean 29%
Mean 20%
Max. up to 30%
Max. up to 50%
Max. up to 44%
Max. up to 30%
Mean 29%
-
•
2, n=3
•
3, n=3
•
•
Max. up to 56%
-
-
Out-licensed clinical programmes
•
Two ophthalmology in vivo Phase I/II products licensed to Sanofi
•
Two out-licensed 5T4 antibody technology phase I studies
Further potential product development opportunities being evaluated
•
•
Three gene therapy product candidates in Phase I/II development
Mean 28%
Mean 34%
2x
Original
Max.
up to 53%
Max. up to 53%
®
Three investigator-led Phase II TroVax studies
Mean 26%
2x
Enhanced
-
Including CAR-T 5T4
Significant revenue potential from manufacturing and process development
18
Contact us
Dose
Oxford BioMedica
plc
1, Windrush
n=3
1x
Court
Transport Way
2, Oxford
n=3
2x
OX4 6LT
3, n=3
2x
United Kingdom
Cohort2
Administration
Original
6 months (UPDRS) CEO
1 year (UPDRS)
John Dawson,
Mean 27%Tim Watts,
Mean 30%
CFO Mean 29%
3 months (UPDRS)
Max. up to 30%
Original
Mean 28%Tel:
Max. up to 53%
Enhanced
Mean 26%
Max. up to 50%
Max. up to 44%
Mean 29%
+44Mean
(0)34%
1865 783 000
Max. up to 53%
Max. up to 56%
-
-
2 years (UPDRS)
Mean 20%
Max. up to 30%
-
www.oxfordbiomedica.co.uk
www.oxbsolutions.co.uk
enquiries@oxfordbiomedica.co.uk
19