Oxford BioMedica Announces Ocular Clinical Programme Update
-- Positive DSMB review of first patient cohort in UshStat® Phase I/IIa Study --
-- Further encouraging data from RetinoStat® and StarGen™ clinical studies --
Oxford, UK - 20 November 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 ongoing UshStat® Phase I/IIa study by the Data Safety Monitoring Board (DSMB); an independent panel of specialists in the fields of ophthalmology, virology and vectorology. The Company also announces further data from the ongoing RetinoStat® and StarGen™ clinical studies. All three ocular gene therapies were designed and developed by Oxford BioMedica using the Company's proprietary LentiVector® gene delivery technology.
DSMB highlights of ongoing UshStat® Phase I/IIa study in Usher syndrome type 1B
· Three patients treated at dose level 1
· No serious adverse events related to UshStat® or its method of administration
· No signs of inflammation in the treated eye
· Safety profile now up to six months post-treatment
· DSMB support received to proceed to the second patient cohort (dose level 2)
Highlights of ongoing RetinoStat® Phase I study in "wet" age-related macular degeneration
· Three patient cohorts complete (n=9, ascending dose levels 1, 2 and 3), treatment of final patient cohort ongoing (n=9, confirmatory dose level) across two clinical centres
· Long-term safety profile now up to 19 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®
· Long-term protein expression1: now sustained for up to one year post-treatment at dose levels 1 and 2, and up to two months at dose level 3
· Further preliminary data continue to show a dose response, with the escalation to dose levels 2 and 3 yielding an increase in average protein expression
1. As at latest available time points
Further safety data from ongoing StarGen™ Phase I/IIa study in Stargardt disease
· 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
· No signs of inflammation in the eye
· Long-term safety profile now up to 16 months post-treatment (dose level 1)
· Treatment of third patient cohort ongoing (n=4, dose level 2)
John Dawson, Chief Executive Officer of Oxford BioMedica, said: "Given recent positive developments in the field, we believe gene therapy is the future - particularly in fighting ocular disease. We remain very encouraged by the continued progress across our ocular programmes partnered with Sanofi and, having successfully initiated three pioneering first-in-man studies, we are well-placed to leverage our expertise and exploit new opportunities utilising our proprietary LentiVector® platform technology."
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. In the US, the study is led by Professor Peter Campochiaro at the Wilmer Eye Institute at Johns Hopkins, Baltimore and by Dr Andy Lauer at the Oregon Health and Science University, Portland, Oregon.
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.
The UshStat® 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. UshStat® has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits.
For further information, please contact:
Oxford BioMedica plc:
Lara Mott, Head of Corporate Communications
Tel: +44 (0)1865 783 000
Mary Clark/Sarah Macleod/Claire Dickinson M:Communications
Tel: +44 (0)20 7920 2360
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, ImaginAb and Immune Design Corp. Further information is available at www.oxfordbiomedica.co.uk and www.oxbsolutions.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. 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.
5. 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.
6. 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 ABCA4 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 ABCA4 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.
This information is provided by RNS