Oxford BioMedica Announces US IND Approval for Novel Ocular Product in Stargardt Disease

20 March 2011

— Second of four products in Phase I/II ocular programme partnered with sanofi-aventis approved to enter clinical development —

Oxford, UK – 21 March 2011: Oxford BioMedica plc (“Oxford BioMedica” or “the Company”) (LSE: OXB), 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/IIa clinical development of StarGen™, a novel gene-based treatment for Stargardt disease.  StarGen™ was designed and developed by Oxford BioMedica using the Company’s proprietary LentiVector® platform technology and is the second programme to enter clinical development under the Phase I/II ocular collaboration agreement signed with sanofi-aventis in April 2009.
The approval of the IND follows the decision by the US Recombinant DNA Advisory Committee (RAC) to approve the StarGen™ Phase I/IIa protocol in January 2011.  Oxford BioMedica will enrol up to 28 patients with Stargardt disease in a multinational, open label, dose escalation Phase I/IIa study with planned sites in France and the US.  The study is anticipated to be initiated in Q2 2011.  Three dose levels will be evaluated for safety, tolerability and aspects of biological activity.  In the US, the study will be led by Dr Peter Francis at the Oregon Health and Science University, Portland, Oregon.  In France, Professor Jose-Alain Sahel will lead the study at the Centre Hospitalier Nationale D’Opthalmologie des Quinze-Vingts, Paris.

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 provide long-term or potentially permanent correction.  There are currently no approved treatments available for Stargardt disease.  StarGen™ has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits.

John Dawson, Chief Executive Officer of Oxford BioMedica, said: “Following the recent initiation of the RetinoStat® Phase I study, StarGen™ will be our second ocular product, partnered with sanofi-aventis, to enter first-in-man studies in the US.  Receiving IND approval to advance StarGen™ into Phase I/IIa development represents further endorsement of our LentiVector® platform by the US regulatory agencies and, with no currently approved treatment, this novel product brings significant hope for the future to Stargardt patients.” 

Dr Stephen Rose, Chief Research Officer of the Foundation Fighting Blindness, an early funding collaborator of Oxford BioMedica’s pre-clinical ocular programme, said:  “As an initial funder of the project we are pleased to move forward with a StarGen™ Phase I/IIa clinical trial, which reinforces the exciting potential for gene therapy to treat patients with Stargardt disease and other vision-robbing retinal degenerations.  The Foundation Fighting Blindness is committed to accelerating the translation of laboratory-based research into clinical trials, ultimately getting successful treatments for rare diseases like Stargardt to the market and to patients who need them.  Our relationship with Oxford BioMedica is an excellent example of this mission in action.”

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” 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-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.