Last updated on April 17, 2014 at 10:53 EDT

Oxford BioMedica Announces US IND Approval for Novel Ocular Product in Usher Syndrome Type 1B

October 18, 2011

OXFORD, England, October 18, 2011 /PRNewswire/ –

— Third ocular product partnered with Sanofi approved to enter clinical
development –

Oxford BioMedica plc (“Oxford BioMedica” or “the Company”) (LSE: OXB),
the leading gene-based biopharmaceutical company, today announces that the
US Food and Drug Administration (FDA) has approved its Investigational New
Drug (IND) application for the Phase I/IIa clinical development of
UshStat(R), a novel gene-based treatment for Usher syndrome type 1B.
UshStat(R) was designed and developed by Oxford BioMedica using the
Company’s proprietary LentiVector(R) platform technology and is the third
programme to enter clinical development under the Phase I/II ocular
collaboration agreement signed with Sanofi in April 2009.

The approval of the IND follows the decision by the US Recombinant DNA
Advisory Committee (RAC) to approve the UshStat(R) Phase I/IIa protocol in
May 2011. The open label, dose escalation Phase I/IIa study will enrol up to
18 patients with Usher syndrome type 1B at the Oregon Health and Science
University’s Casey Eye Institute, Portland, Oregon. The study, led by
Professor Richard Weleber, will evaluate three dose levels for safety,
tolerability and aspects of biological activity and is expected to be
initiated by the end of 2011.

Usher syndrome is the most common form of deaf-blindness which affects
approximately 30,000-50,000 patients in the US and Europe. One of the most
common subtypes is Usher syndrome type 1B. The disease is caused by a
mutation of the gene encoding myosin VIIA (MY07A), which leads to
progressive retinitis pigmentosa combined with a congenital hearing defect.
UshStat(R) uses the Company’s LentiVector(R) platform technology to deliver
a corrected version of the MYO7A gene to address the vision loss associated
with the disease. On the basis of pre-clinical data, it is anticipated that
a single application of UshStat(R) to the retina could provide long-term or
potentially permanent stabilisation of vision. There are currently no
approved treatments available for Usher syndrome type 1B. UshStat(R) has
received European and US Orphan Drug Designation which brings development,
regulatory and commercial benefits.

John Dawson, Chief Executive Officer of Oxford BioMedica, said: “This is
the third ocular IND approval that Oxford BioMedica has received from the US
regulatory agencies over the last 12 months which represents an exceptional
achievement for our R&D and regulatory teams. The continued progress of our
ocular programmes partnered with Sanofi will further support the development
path for other LentiVector(R) platform products. With no approved treatment
available for patients, we look forward to bringing UshStat(R) into Phase
I/IIa clinical development later this year.”

Professor Richard Weleber, Principal Investigator at the Casey Eye
Institute, commented: “We are delighted to be partnering with Oxford
BioMedica in the design and conduct of this; the first trial of gene
replacement for retinitis pigmentosa associated with myosin 7A-deficient
type I Usher syndrome. As such, this trial represents a major milestone in
the history of Usher syndrome. We conclude that the gene replacement therapy
that will be evaluated in this trial has the potential to provide a
substantial, durable benefit for the vision of these patients.”

Dr Stephen Rose, Chief Research Officer of the Foundation Fighting
Blindness, an early funding collaborator of Oxford BioMedica’s pre-clinical
ocular programme, added: “The IND approval for UshStat(R) is great news for
people affected by a particularly devastating condition. UshStat(R) will be
the first vision treatment for any type of Usher syndrome to move into a
human study and, as a corrective gene therapy, it holds potential to halt
the disease in its tracks.”

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(R)
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 with
current partners and licensees including Sanofi, Pfizer, GlaxoSmithKline,
MolMed, Sigma-Aldrich, Biogen Idec, VIRxSYS, Emergent BioSolutions and
ImaginAb. Further information is available at


2. LentiVector(R) gene delivery technology

Oxford BioMedica’s LentiVector(R) gene delivery 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.

3. Oxford BioMedica’s agreement with Sanofi

Under the terms of the agreement signed with Sanofi in April 2009,
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(R) for “wet” age-related macular degeneration,
StarGen(TM) for Stargardt disease, UshStat(R) for Usher syndrome 1B and
EncorStat(R) for corneal graft rejection. Oxford BioMedica will receive
committed funding of up to US$24 million over the initial phase of
development. 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.

4. 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

        For further information, please contact:

        Oxford BioMedica plc:
        Lara Mott, Head of Corporate Communications
        Tel: +44-(0)1865-783-000

        Media Enquiries:
        Mary Clark/Emma Thompson/Claire Dickinson
        Tel: +44-(0)20-7920-2342

SOURCE Oxford BioMedica plc

Source: PR Newswire