Oxford BioMedica Announces Ocular Programme Update

By Oxford Biomedica Plc, PRNE
Tuesday, June 21, 2011

OXFORD, England, June 22, 2011 -


Progress in Phase I Study in Wet Age
Macular Degeneration


Oxford BioMedica plc (”Oxford BioMedica” or “the Company”) (LSE:
OXB), a leading gene therapy company, today announces that the
first dose level of RetinoStat® is safe and
well-tolerated at one month following treatment.
 RetinoStat® is a novel gene-based treatment for
neovascular “wet” age-related macular degeneration (AMD), designed
and developed by Oxford BioMedica using the Company’s proprietary
LentiVector® gene delivery technology.  It is the
lead programme of the ocular agreement the Company signed with
Sanofi (EURONEXT: SAN and NYSE: SNY) in April 2009.

The on-going Phase I study will enrol 18 patients with wet AMD
at the Wilmer Eye Institute at Johns Hopkins, Baltimore (USA).
 Led by Professor Peter Campochiaro, the study will evaluate
three dose levels and assess safety, aspects of visual acuity and
ocular physiology.  Three patients received the first dose
level of RetinoStat® and one-month results have been
assessed by the study’s independent Data Safety Monitoring Board
(DSMB).  First results from the study are expected to be
announced in H1 2012.

Highlights of first patient cohort at
one month (dose level 1)

  • Favourable safety profile with no serious adverse events
    related to RetinoStat® or its method  of
  • No signs of inflammation in the eye
  • DSMB support received to proceed to dose level 2 in the next
    patient cohort

John Dawson, Chief Executive Officer of Oxford BioMedica,
This clinical study is breaking new ground as
the first to directly administer a lentiviral vector-based
treatment to patients in the US and the favourable safety profile
of RetinoStat
® is encouraging.  We
continue to work closely with the regulatory bodies in order to
advance our novel ocular programmes as rapidly as possible and,
together with Sanofi, we look forward to further progress during

The second ocular product to enter clinical development is
StarGen™, a novel gene-based treatment for Stargardt disease, and
the first patient in the Phase I/IIa study was treated in the US in
June 2011 at the Oregon Health & Science University’s Casey Eye
Institute.  StarGen™ has received European and US Orphan Drug
Designation which brings development, regulatory and commercial
benefits and, with no currently approved treatment, this novel
product brings significant hope for the future to Stargardt
patients.  The next product expected to enter Phase I/II
clinical development is UshStat® for the treatment of
Usher syndrome type 1B in H2 2011.  UshStat® has
also received European and US Orphan Drug Designation.

We are delighted to see Oxford
s lentiviral gene therapy products
successfully moving into human studies,
said Stephen
, Ph.D., chief research officer of the Foundation Fighting
Blindness, a US non-profit organisation that provided early funding
for Oxford BioMedica
s pre-clinical ocular
The Companys innovative
treatments hold great promise for saving vision in people affected
by a broad range of devastating eye diseases for which current
treatments are limited or non-existent.

Notes to editors

1. Oxford BioMedica®

Oxford BioMedica plc (LSE: OXB) is a biopharmaceutical company
developing innovative gene-based medicines and therapeutic vaccines
that aim to improve the lives of patients with high unmet medical
needs.  The Company’s technology platform includes a highly
efficient LentiVector® gene delivery system, which has
specific advantages for targeting diseases of the central nervous
system and the eye; and a unique tumour antigen (5T4), which is an
ideal target for anti-cancer therapy.  Through in-house and
collaborative research, Oxford BioMedica has a broad pipeline and
its partners include Sanofi, Sigma-Aldrich and Pfizer.
 Further information is available at href="www.oxfordbiomedica.co.uk/">www.oxfordbiomedica.co.uk.

2. Oxford BioMedicas agreement with

Under the terms of the agreement signed with Sanofi in April
, Oxford BioMedica is responsible for the pre-clinical and
initial Phase I/II studies of four lentiviral vector-based product
candidates in the field of ophthalmology: RetinoStat®
for “wet” AMD, StarGen™ for Stargardt disease, UshStat®
for Usher syndrome 1B and EncorStat® for corneal graft
rejection.  Oxford BioMedica granted Sanofi a license to
develop the products and an option for further development,
manufacture and commercialisation on a worldwide basis.  At
any time prior to or within a defined period after completion of
each Phase I/II study, Sanofi can exercise its option to license
the products and will then assume responsibility for on-going
activities.  Sanofi also has rights to broaden its license to
develop the four products in additional indications, and has rights
of first refusal to license other lentiviral vector-based products
for the treatment of ocular diseases.

3. LentiVector® gene delivery

Oxford BioMedica’s LentiVector® platform technology
is one of the most advanced gene delivery systems currently
available, which has many applications in product development and
discovery research.  It is the system of choice for gene-based
treatments addressing chronic and inherited diseases.  Oxford
BioMedica has established a dominant intellectual property estate
in the field of lentiviral-vector mediated gene delivery through
its in-house research and from work conducted by the Company’s
co-founders at Oxford University.

4. Age-Related Macular Degeneration and

Age-related macular degeneration (AMD) is a major cause of
blindness affecting an estimated 25 to 30 million people worldwide
and the incidence of AMD is expected to triple by the year 2025
(source: AMD Alliance International).  Neovascular “wet” AMD
accounts for the majority of all severe vision loss from the
disease.  RetinoStat® delivers two anti-angiogenic
genes, endostatin and angiostatin, directly to the retina and aims
to preserve and improve the vision of patients through
anti-angiogenesis which blocks the formation of new blood vessels.
 On the basis of pre-clinical data, it is anticipated that
RetinoStat® will require only a single administration
which would give the product a significant advantage in the market
over currently available treatments that often require frequent,
repeated administration.

5. Sanofi

Sanofi, a global and diversified healthcare leader, discovers,
develops and distributes therapeutic solutions focused on patients’
needs. Sanofi has core strengths in the field of healthcare with
seven growth platforms: diabetes solutions, human vaccines,
innovative drugs, rare diseases, consumer healthcare, emerging
markets and animal health. Sanofi is listed in Paris (EURONEXT:
SAN) and in New York (NYSE: SNY).

6. Foundation Fighting Blindness

The Foundation Fighting Blindness is a publicly-supported
charity raising money to fund research for macular degeneration,
retinitis pigmentosa (RP), Usher syndrome, Stargardt disease and
related ocular diseases.

For further information, please contact:

Oxford BioMedica plc:
Lara Mott, Head of Corporate Communications

Tel: +44(0)1865-783-000

Media Enquiries:
Emma Thompson/Katja Toon/Amber Bielecka

Tel: +44(0)20-7920-2342


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