— Third ocular product partnered with Sanofi approved to enter clinical development —
Oxford, UK – 18 October 2011: Oxford BioMedica plc (“Oxford BioMedica” or “the Company”) (LSE: OXB), the leading gene-based biopharmaceutical company, today 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 UshStat®, a novel gene-based treatment for Usher syndrome type 1B. UshStat® was designed and developed by Oxford BioMedica using the Company’s proprietary LentiVector® platform technology and is the third programme to enter clinical development under the Phase I/II ocular collaboration agreement signed with Sanofi in April 2009.
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. UshStat® has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits.
John Dawson, Chief Executive Officer of Oxford BioMedica, said: “This is the third ocular IND approval that Oxford BioMedica has received from the US regulatory agencies over the last 12 months which represents an exceptional achievement for our R&D and regulatory teams. The continued progress of our ocular programmes partnered with Sanofi will further support the development path for other LentiVector® platform products. With no approved treatment available for patients, we look forward to bringing UshStat®into Phase I/IIa clinical development later this year.”
Professor Richard Weleber, Principal Investigator at the Casey Eye Institute, commented: “We are delighted to be partnering with Oxford BioMedica in the design and conduct of this; the first trial of gene replacement for retinitis pigmentosa associated with myosin 7A-deficient type I Usher syndrome. As such, this trial represents a major milestone in the history of Usher syndrome. We conclude that the gene replacement therapy that will be evaluated in this trial has the potential to provide a substantial, durable benefit for the vision of these patients.”
Dr Stephen Rose, Chief Research Officer of the Foundation Fighting Blindness, an early funding collaborator of Oxford BioMedica’s pre-clinical ocular programme, added: “The IND approval for UshStat® is great news for people affected by a particularly devastating condition. UshStat® will be the first vision treatment for any type of Usher syndrome to move into a human study and, as a corrective gene therapy, it holds potential to halt the disease in its tracks.”
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