Oxford BioMedica Receives Agreement from FDA and ANSM to Resume Recruitment into Ocular Clinical Trials
-- Newly developed, state-of-the-art analytical methods confirm the integrity and quality of Oxford BioMedica's LentiVector® platform products --
Oxford, UK - 17 October 2013: Oxford BioMedica plc ("Oxford BioMedica" or "the Company") (LSE: OXB), the leading gene-based biopharmaceutical company, today announces that it has received agreement from the US Food and Drug Administration (FDA) and the French regulatory agency, ANSM, to resume recruitment into the RetinoStat® Phase I, StarGen™ Phase I/IIa and UshStat® Phase I/IIa studies using the existing clinical trial material.
In June 2013, Oxford BioMedica announced that it had voluntarily paused recruitment into the aforementioned studies, as a precautionary measure, whilst the Company investigated the detection of very low concentrations of a potential impurity in its clinical trial material derived from a third party raw material. Oxford BioMedica has since performed extensive characterisation studies using its newly developed, state-of-the-art analytical methods to identify the impurity as highly fragmented DNA derived from foetal bovine serum (FBS), the most widely-used growth supplement for cell culture media. In light of these findings, Oxford BioMedica remains convinced of the safety, integrity and quality of its LentiVector® platform products and no safety concerns relating to any of the ocular products have been identified in any pre-clinical and clinical data generated to date.
Following the submission of a comprehensive data package to FDA and ANSM, Oxford BioMedica has received agreement from both agencies to resume recruitment into its ocular clinical trials using the existing clinical trial material. The Company will continue to use highly sensitive, state-of-the-art analytical methods to ensure the quality and integrity of its lentiviral vector products and will work with FDA and ANSM to define the necessary specifications for future batches of clinical trial material.
Oxford BioMedica is now working closely with the clinical trial centres to obtain the necessary ethics committee approvals in order to resume recruitment into the ocular clinical studies.
John Dawson, Chief Executive Officer of Oxford BioMedica, said: "We value our relationships with the regulatory authorities and are pleased that, on the basis of our extensive technical investigations to demonstrate the integrity of our products, FDA and ANSM agree with our proposal to resume treating patients in our ocular trials as soon as possible.
"We place the highest importance on safety, and our analytical methods and quality assurance processes are continuously evolving to ensure that we remain at the forefront of gene therapy development and manufacture. I am confident that, with significant opportunities ahead such as the recently-announced AMSCI project win, Oxford BioMedica will continue to lead the way in delivering novel gene therapies to patients."
For further information, please contact:
Oxford BioMedica plc:
Lara Mott, Head of Investor Relations and Corporate Communications
Tel: +44 (0)1865 783 000
Mary-Jane Elliott/Emma Thompson/Matthew Neal
Consilium Strategic Communications
Tel: +44 (0)20 7920 2354
Notes to editors
1. About 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, Novartis, 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 licence 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 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.
6. 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.
This information is provided by RNS