Date: 2013-10-17
Type of information: Initiation of preclinical development
phase: 1-2
Announcement: agreement to resume recruitmen
Company: Oxford Biomedica (UK) Sanofi (France)
Product: StarGen®
Action
mechanism: gene therapy. StarGen® uses the Company's LentiVector® platform technology to deliver a corrected version of the ABCR gene. Mutations of this gene lead to the degeneration of photoreceptors in the retina and vision loss. StarGen® is a gene-based therapy for the treatment of Stargardt disease. 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 Oxford Biomedica's LentiVector® gene delivery technology to deliver a corrected version of the ABCR gene. A single administration of the product directly to the retina could provide long-term or potentially permanent correction.
Disease: Stargardt's disease
Therapeutic area: Rare diseases - Ophtalmological diseases - Genetic diseases
Country: France, USA
Trial
details: 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.
There are two parts to the study. A dose-escalation phase looking at three doses of StarGen®, eight patients will be recruited at the first dose level, and four each at the next two dose levels. This will be followed by a dose confirmation phase where the highest dose that is safe and well tolerated will be examined in up to twelve patients.
Further results from this study are expected in Q4 2012.
Latest
news: 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 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.
* On November 20, 2012, Oxford BioMedica and its partner Sanofi have announced further data from the ongoing StarGen™ clinical study.
Further safety data from ongoing StarGen™ Phase I/IIa study in Stargardt disease:
• Eight patients treated at dose level 1 to date (n=4 severe level of disease, n=4 less severe)
• No serious adverse events related to StarGen™ or its method of administration
• No signs of inflammation in the eye
• Long-term safety profile now up to 16 months post-treatment (dose level 1)
• Treatment of third patient cohort ongoing (n=4, dose level 2).
* On August 8, 2012, Oxford BioMedica and Sanofi have announced a positive interim review of the StarGen™ Phase I/IIa study in Stargardt disease by the Data Safety Monitoring Board (DSMB); an independent panel of specialists in the fields of ophthalmology, virology and vectorology.
DSMB highlights of ongoing StarGen™ Phase I/IIa study:
• Eight patients treated at dose level 1 to date (n=4 severe level of disease, n=4 less severe)
• No serious adverse events related to StarGen™ or its method of administration
• Long-term safety profile now up to 12 months post-treatment (dose level 1)
DSMB support received to proceed to third patient cohort (n=4, dose level 2)
* On June 3, 2011, Oxford Biomedica has announced that the first patient in the Phase I/IIa study was treated in the US in June 2011 at the Oregon Health & Science University’s Casey Eye Institute. This gene therapy is one of the four lentiviral vector-based products included in the collaboration Oxford BioMedica has signed with Sanofi in April 2009.
* On March 21, 2011, Oxford BioMedica has announced that the 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 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. StarGen® has received European and US Orphan Drug Designation.
