Oxford Biomedica Regulatory News (OXB)

Oxford BioMedica Receives Funding Award from the Foundation Fighting Blindness for ongoing UshStat® Phase I/IIa study

-- Award of US$125,000 will support a second clinical site in Paris, France --

Oxford, UK - 23 July 2012: Oxford BioMedica plc ("Oxford BioMedica" or "the Company") (LSE: OXB), the leading gene-based biopharmaceutical company, today announces that the US non-profit organisation, the Foundation Fighting Blindness (FFB), has granted an award of US$125,000 to the Company via its translational research arm, the Foundation Fighting Blindness Clinical Research Institute (FFBCRI) formerly known as the National Neurovision Research Institute (NNRI). The FFB was an early funding collaborator of Oxford BioMedica's pre-clinical ocular programmes and this award furthers the Company's long-standing relationships with FFB and FFBCRI. The award will support the opening of a second clinical site in Paris, France for the ongoing UshStat® Phase I/IIa study, currently underway in the US.

UshStat® is a novel gene-based treatment for Usher syndrome type 1B, designed and developed by Oxford BioMedica using its proprietary LentiVector® platform technology, and is the third ocular product to enter clinical development under the Company's collaboration with Sanofi (EURONEXT: SAN and NYSE: SNY). In February 2012, Oxford BioMedica initiated an open label, dose escalation Phase I/IIa study in the US led by Professor Richard Weleber at the Oregon Health & Science University's Casey Eye Institute. The ongoing study will enrol up to 18 patients and will evaluate three dose levels for safety, tolerability and aspects of biological activity. Initial safety data from this study are expected in H2 2012.

Oxford BioMedica plans to open the second clinical site at Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts in Paris, France with Professor José-Alain Sahel as Principal Investigator. Subject to receiving regulatory approval, the second clinical site is anticipated to open in early 2013, from which point patients could be treated in parallel at both sites.

Dr. Stephen Rose, chief research officer at the Foundation Fighting Blindness, said: "We are very pleased to be supporting the UshStat® trial in Paris. Professor José-Alain Sahel's team is one of the best retinal research groups in the world. They will do an outstanding job evaluating UshStat®'s potential for saving vision in people affected by a particularly challenging condition."

Professor José-Alain Sahel of Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts Paris, France commented: "We are very grateful to FFB for this support and are committed to testing this innovative approach with our partners, such as Pr Christine Petit (Pasteur Institute) who first identified disease-causing mutations in this gene. Our collaboration with Oxford BioMedica, the Portland team and FFB, already implemented with the ongoing StarGen™ trial, holds major promise for our patients."

Stuart Naylor, Chief Scientific Officer of Oxford BioMedica, said: "Having worked with FFB since 2003, our strong relationship has underpinned the success we have had in bringing novel ocular gene therapies into clinical development. With no available treatments for patients with Usher syndrome type 1B, we are grateful for FFB's support to boost this programme with an additional clinical site."

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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 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. Usher syndrome type 1B and UshStat®

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® uses the Company's LentiVector® 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® 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® has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits.

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 diseases. For further information please visit www.blindness.org. 

5. Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts

The Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts is one of the largest University Eye Hospitals worldwide. Among the 160,000 patients seen yearly, thousands of persons affected with hereditary retinal conditions are followed in a dedicated National Reference Center leading to testing innovative therapies within the Clinical Investigation Center. Translational research is conducted both in the hospital and the Institut de la Vision (250 researchers) with support from national, European and industrial sources (e.g. Sanofi-Fovea) and from Foundation Fighting Blindness (Paris Research Center for the Study of Retinal Degenerative Diseases).

6. 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.