Oxford BioMedica Announces Positive DSMB Review of Ongoing RetinoStat® and StarGen™ Clinical Studies

Description

Oxford BioMedica Announces Positive DSMB Review of Ongoing RetinoStat® and StarGen™ Clinical Studies PR Newswire OXFORD, England, August 8, 2012 OXFORD, England, August 8, 2012 /PRNewswire/ -- ®-- Treatment of final RetinoStat patient cohort underway -- Oxford BioMedica plc ("Oxford BioMedica" or "the Company") (LSE: OXB), the leading gene-based biopharmaceutical company, and its partner Sanofi (EURONEXT: SAN and NYSE: SNY) today announce a positive interim review of ®the RetinoStat Phase I study in neovascular "wet" age-related macular degeneration (AMD) and the StarGen™ Phase I/IIa study in Stargardt disease by the Data Safety Monitoring Board (DSMB); an independent panel of specialists ®in the fields of ophthalmology, virology and vectorology. RetinoStat and StarGen™ were designed and developed by Oxford BioMedica using the ®Company's proprietary LentiVector gene delivery technology. ®DSMB highlights of ongoing RetinoStat Phase I study Nine patients treated to date (n=3 at each of dose levels 1, 2 and 3) ®No serious adverse events related to RetinoStat or its method of administration Long-term safety profile now up to 18 months post-treatment (dose level 1) Successful retinal transduction, as shown by substantial increase in expression and secretion of endostatin and angiostatin proteins measured in the anterior chamber of the ®eye following a single administration of RetinoStat .

Informations

• Publié par: oxford-biomedica-plc
• Nombre de lectures: 42
• Langue: English

Extrait

Oxford BioMedica Announces Positive DSMB
Review of Ongoing RetinoStat® and StarGen™
Clinical Studies
PORX FNOeRwDs,w iErnegland, August 8, 2012

OXFORD, England
,
August 8, 2012
/PRNewswire/ --
-- Treatment of final RetinoStat
®

patient cohort underway --
Oxford BioMedica plc ("Oxford BioMedica" or "the Company") (LSE: OXB), the
leading gene-based biopharmaceutical company, and its partner Sanofi
(EURONEXT: SAN and NYSE: SNY) today announce a positive interim review of
the RetinoStat
®
Phase I study in neovascular "wet" age-related macular
degeneration (AMD) and the StarGen™ Phase I/IIa study in Stargardt disease by
the Data Safety Monitoring Board (DSMB); an independent panel of specialists
in the fields of ophthalmology, virology and vectorology. RetinoStat
®
and
StarGen™ were designed and developed by Oxford BioMedica using the
Company's proprietary LentiVector
®
gene delivery technology.
DSMB highlights of ongoing RetinoStat
®

Phase I study
Nine patients treated to date (n=3 at each of dose levels 1, 2 and 3)
No serious adverse events related to RetinoStat
®
or its method of administration
Long-term safety profile now up to 18 months post-treatment (dose level 1)
Successful retinal transduction, as shown by substantial increase in expression and
secretion of endostatin and angiostatin proteins measured in the anterior chamber of the
eye following a single administration of RetinoStat
®
. So far, expression is sustained for
up to 12 months post-treatment at dose level 1 (n=3) and up to six months post-
treatment at dose level 2 (n=3)
DSMB support received to proceed to final patient cohort (n=9, confirmatory dose level)

sDtSudMyB highlights of ongoing StarGen™ Phase I/IIa

Eight patients treated at dose level 1 to date (n=4 severe level of disease, n=4 less
severe)
No serious adverse events related to StarGen™ or its method of administration
Long-term safety profile now up to 12 months post-treatment (dose level 1)
DSMB support received to proceed to third patient cohort (n=4, dose level 2)

John Dawson, Chief Executive Officer of

Oxford

BioMedica, said:

"The
continued progress of our ophthalmology portfolio, supported by another
positive DSMB review, is encouraging

-

particularly given that early
RetinoStat
®

data demonstrate sustained therapeutic protein expression in the
eye following a single administration. The favourable safety profile of our novel

ocular gene therapies further supports the wider LentiVector
®

platform safety
package with over 33 patients treated to date across the ocular and Parkinson's
disease programmes."
The RetinoStat
®
open label, dose escalation Phase I study will enrol 18 patients
with "wet" AMD and will evaluate three dose levels to assess safety and aspects
of ocular physiology. The study is led by Professor Peter Campochiaro at the
Wilmer Eye Institute at Johns Hopkins,
Baltimore
(
USA
) and Oxford BioMedica
has opened a second clinical site at the Oregon Health and Science University,
Portland
with Dr Andy Lauer as principal investigator. Further results from this
study are expected in Q4 2012.
The StarGen™ open label, dose escalation Phase I/IIa study will enrol up to 28
patients and will evaluate three dose levels for safety, tolerability and aspects
of biological activity. In the US, the study is led by Professor David Wilson at the
Oregon Health and Science University,
Portland, Oregon
. In France, Professor
Jose-Alain Sahel leads the study at Centre Hospitalier National d'Ophtalmologie
des Quinze-Vingts,
Paris
. StarGen™ has received European and US Orphan
Drug Designation which brings development, regulatory and commercial
benefits. Further results from this study are expected in Q4 2012.

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 with current partners and
licensees including Sanofi, Pfizer, GlaxoSmithKline, MolMed, Sigma-Aldrich,
Biogen Idec, Emergent BioSolutions and ImaginAb. Further information is
available at http://www.oxfordbiomedica.co.uk.
2. LentiVector
®

gene delivery technology
Oxford BioMedica's LentiVector
®
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
®
for "wet" age-related macular degeneration, StarGen™ for

Stargardt disease, UshStat
®
for Usher syndrome 1B and EncorStat
®
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. On
29 June 2012
,
Sanofi elected to exercise its options to acquire two exclusive worldwide
licences for further development, manufacture and commercialisation of
StarGen™ and UshStat
®
. Oxford BioMedica is currently conducting the two
ongoing Phase I/IIa trials for StarGen™ and UshStat
®
. The companies will
continue to work together to plan the next stages of development and finalise
the terms of the worldwide licence agreements.
4. Age-Related Macular Degeneration and RetinoStat
®
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
®
may
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. Stargardt disease and StarGen™

Stargardt disease is the most common juvenile degenerative retinal disease
which affects approximately 80-100,000 patients in the US and
Europe
. The
disease is caused by a mutation of the ABCR gene which leads to the
degeneration of photoreceptors in the retina and vision loss. StarGen™ uses
the Company's LentiVector
®
platform technology to deliver a corrected version
of the ABCR gene. On the basis of pre-clinical data, it is anticipated that a
single application of StarGen™ to the retina could potentially either provide
long-term or permanent correction. There are currently no approved
treatments available for Stargardt disease.
For further information, please contact:

Oxford BioMedica plc:
Lara Mott, Head of Corporate Communications
Tel: [phone]

Media Enquiries:
Mary Clark/Sarah Macleod/Claire Dickinson M:Communications
Tel: [phone]