Oxford BioMedica plc, a leading gene therapy company, announces that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for the phase I/II clinical development of RetinoStat, a novel gene-based treatment for neovascular “wet” age-related macular degeneration (AMD).  RetinoStat was designed and developed by Oxford BioMedica using the  company's proprietary LentiVector gene delivery technology and is the lead programme of the ocular agreement the company signed with sanofi-aventis in April 2009.
 
The approval of the IND follows the decision by the US Recombinant DNA Advisory Committee (RAC) to unanimously approve the RetinoStat phase I/II protocol in September 2010.  The open label dose escalation and safety phase I/II study will enrol 18 patients with wet AMD at the Wilmer Eye Institute at Johns Hopkins, Baltimore (USA).  The study, led by Professor Peter Campochiaro, will evaluate three dose levels and assess safety, aspects of visual acuity and ocular physiology.  Subject to receiving local approvals, the study is anticipated to be initiated by the end of December 2010.

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 will 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.

John Dawson, Chief Executive Officer of Oxford BioMedica, said: “Receiving both FDA and RAC approval to advance RetinoStat into phase I/II development is a significant milestone for Oxford BioMedica and is a result of the outstanding effort from our R&D and regulatory teams.

“Not only will this be the first US clinical study to directly administer a lentiviral vector-based treatment to patients, we believe this regulatory endorsement for our platform technology will also support the development path for a number of other lentiviral vector-based products in our pipeline - including our Parkinson’s disease product, ProSavin, which we intend to take into the US for phase II development.  Together with our partner, sanofi-aventis, we look forward to progressing all four of our gene-based ocular products into clinical development by the end of 2011.”

Under the terms of the agreement signed with sanofi-aventis 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” AMD, StarGen for Stargardt disease, UshStat for Usher syndrome 1B and EncorStat for corneal graft rejection.  Oxford BioMedica granted sanofi-aventis 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-aventis can exercise its option to license the products and will then assume responsibility for on-going activities.  Sanofi-aventis also has rights to broaden its license to develop the four products in additional indications, and has rights of first refusal to license other lentiviral vector-based products for the treatment of ocular diseases.

Oxford BioMedica 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 the highly efficient LentiVector gene delivery technology, 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.