Positive DSMB Review of Ongoing Clinical Studies

RNS Number : 5269J

Oxford Biomedica PLC

08 August 2012

 

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

-- Treatment of final RetinoStat^® patient cohort underway --

Oxford, UK - 08 August 2012: 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)

DSMB highlights of ongoing StarGen™ Phase I/IIa study

·      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.

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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]

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. 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.

 

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