Date: 2013-10-17
Type of information:
phase: 1-2a
Announcement: agreement to resume recruitment
Company: Oxford BioMedica (UK) Sanofi (France)
Product: UshStat®
Action mechanism: Usher syndrome 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.
Disease: Usher syndrome type 1B
Therapeutic area: Ophtalmological diseases
Country: USA
Trial
details: The UshStat® open label, dose escalation Phase I/IIa study will enrol up to 18 patients with Usher syndrome type 1B at the Oregon Health and Science University’s Casey Eye Institute, Portland, Oregon. The study, led by Professor Richard Weleber, will evaluate three dose levels for safety, tolerability and aspects of biological activity. UshStat® has received European and US Orphan Drug Designation .
Latest
news: * On October 17, 2013, Oxford BioMedica, a gene-based biopharmaceutical company, has announced that it has received agreement from the 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, 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.* On June 3, 2013, Oxford BioMedica, a gene-based biopharmaceutical company, has announced that it has voluntarily paused recruitment into the RetinoStat® Phase I, StarGen™ Phase I/IIa and UshStat® Phase I/IIa studies, as a precautionary measure, whilst the company investigates the recent detection of very low concentrations of potential impurities derived from a widely-used third party raw material.A new, highly sensitive test method has recently been introduced into the Company’s wide range of analytical methods and quality assurance processes used for routine batch testing. Using this method, potential impurities have been detected at very low concentrations in clinical trial material. No safety concerns relating to any of the aforementioned products have been identified in any pre-clinical and clinical data generated to date and there is no reason to believe that the favourable safety profile of these products will be affected.Such precautionary measures are routine and Oxford BioMedica is working very closely with the regulatory authorities to complete its investigations. The Company is committed to resuming the clinical trials as soon as possible and will continue to keep the market informed as appropriate.
* On November 20, 2012, Oxford BioMedica, a gene-based biopharmaceutical company, and its partner Sanofi have announced a positive interim review of the ongoing UshStat® Phase I/IIa study by the Data Safety Monitoring Board (DSMB); an independent panel of specialists in the fields of ophthalmology, virology and vectorology. DSMB highlights of ongoing UshStat® Phase I/IIa study in Usher syndrome type 1B:
• Three patients treated at dose level 1
• No serious adverse events related to UshStat® or its method of administration
• No signs of inflammation in the treated eye
• Safety profile now up to six months post-treatment
DSMB support received to proceed to the second patient cohort (dose level 2).
