UK group Oxford BioMedica will no doubt be celebrating news that a Phase I/II clinical programme for the first of its ocular gene therapies partnered with French group Sanofi-Aventis has been given US clearance.
The US Food and Drug Administration has OK’d the Oxford, UK-based firm’s Investigational New Drug application for the Phase I/II development of the RetinoStat, following unanimous approval of the protocol by the US Recombinant DNA Advisory Committee back in September.
The Phase I/II open label study aims to enrol 18 patients with wet AMD at the Wilmer Eye Institute at Johns Hopkins, Baltimore, and is designed to evaluate three dose levels and assess safety, aspects of visual acuity and ocular physiology. The firm said it is aiming to kick off the trial by the end of the year.
The lead candidate of its ocular agreement with Sanofi, RetinoStat is a novel gene-based treatment for a major cause of blindness, neovasular ‘wet’ age-related macular degeneration, which already affects up to 30 million people around the globe and is expected to triple by 2025.
RetinoStat is designed to deliver two anti-angiogenic genes - endostatin and angiostatin - straight to the retina via Oxford BioMedica’s LentiVector gene delivery technology, and thereby preserve and even improve vision through blocking the formation of new blood vessels.
According to the firm, early indications based on preclinical data are that RetinoStat will only need to be given once, which, as it points out, would give the product a significant advantage over existing therapies that often require repeated administration.
Hailing the approval of the Phase I/II trial as “a significant milestone” for Oxford BioMedica, chief executive John Dawson said that not only will it be the first study in the US to directly administer a lentiviral vector-based treatment to patients, but that the regulatory endorsement for the 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”.
If the trial is successful, then Sanofi can step in and take over further development of the drug, as per the terms of the groups’ partnership. Under the April 2009 deal, the UK group is responsible for preclinical and initial Phase I/II studies of four lentiviral vector-based product candidates, while Sanofi holds a licence to develop the products and an option for further development, manufacture and commercialisation on a worldwide basis.
Unsurprisingly, shareholders seem pleased with news of the US trial clearance; the group’s stock had already risen 6.5% in early morning trading on the London Stock Exchange.