Date: 2014-04-29
Type of information: Licensing agreement
Compound: StarGen™, UshStat®, EncorStat®, RetinoStat®
Company: Sanofi (France) Oxford Biomedica (UK)
Therapeutic area: Ophtalmalogical diseases - Genetic diseases - Rare diseases
Type agreement: licensing
Action mechanism: gene therapy 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. UshStat® : 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. EncorStat® uses LentiVector® technology to deliver the same genetic payload as RetinoStat®, endostatin and angiostatin in order to block vascularisation and to prevent graft rejection. EncorStat® is designed for ex vivo treatment of donor corneas prior to transplant. RetinoStat® delivers two anti-angiogenic genes, endostatin and angiostatin, directly to the retina and aims to preserve and improve the vision of patients through anti-angiogenesis which blocks the formation of new blood vessels.
Disease: Stargardt's disease- Usher syndrome type 1B - corneal graft rejection - wet age-related macular degeneration
Details: * On June 28, 2012, Sanofi has elected to exercise its options to acquire two exclusive worldwide licences for further development, manufacture and commercialisation of StarGen™ and UshStat®, two novel gene-based treatments designed and developed by Oxford BioMedica using its proprietary LentiVector® platform technology.
StarGen™ is currently in phase 1/2a development for Stargardt disease. UshStat® is also in phase 1/2a development for Usher syndrome type 1B. Oxford BioMedica is currently conducting these two ongoing Phase I/IIa trials for StarGen™ and Ushstat®. The companies will continue to work together to plan the next stages of development and finalise the terms of the worldwide licence agreements.
Financial terms: Under the terms of the ocular agreement signed with Sanofi in April 2009, Oxford BioMedica will receive a total option exercise payment of $3 million and is eligible for further development and commercialisation milestone payments and royalties on any future sales of the products.
Latest news: * On April 29, 2014, Oxford BioMedica has announced that it will regain the worldwide rights to RetinoStat®, a treatment for wet age-related macular degeneration. Sanofi has notified Oxford BioMedica that due to pipeline prioritisation they will not be taking up the option to continue development of RetinoStat® beyond the currently ongoing Phase I study. Sanofi has confirmed that this decision is not linked to unexpected results based on an analysis of the data from the study. Oxford BioMedica has recently announced that it had successfully completed the recruitment and dosing of the Phase I trial of RetinoStat®. Interim analysis of patient data from the ongoing open-label study, which has been designed to assess the safety and aspects of biological activity of RetinoStat® in the eye, has demonstrated safety and signs of efficacy. Indicative results from the study are expected towards the end of 2014. Oxford BioMedica has alsoc indicated that the company has previously received expressions of interest in RetinoStat® from both large pharmaceutical/biotech companies and financial investors in the event of Sanofi not exercising its option.
* On February 17, 2014, Oxford BioMedica has announced that it has concluded the terms of the development and commercialisation licence agreement with Sanofi to develop the novel gene-based medicines for the treatment of ocular diseases, StarGen™ and UshStat®. Under the terms of the licence, Sanofi has been granted broadened global rights across all ocular disease indications for StarGen™ and UshStat® and, in return, Oxford BioMedica will regain the worldwide rights to EncorStat®, a treatment for corneal graft rejection.Under the terms of the collaboration agreement signed in April 2009, Sanofi committed $24 million of development funding that was subsequently prioritised for the advancement of RetinoStat®, StarGen™ and UshStat®. In June 2012, Sanofi elected to exercise its options to acquire exclusive worldwide development and commercialisation licences for StarGen™ and UshStat® which triggered an aggregate option exercise payment of US$3 million to Oxford BioMedica. Under the new Licence Oxford BioMedica is eligible for development and commercialisation milestone payments and royalties on any future sales of the two products. The parties expect that the management of the ongoing clinical studies will be handed over from the Company to Sanofi during the first half of 2014.
EncorStat® was designed and developed by Oxford BioMedica using the Company's proprietary LentiVector® platform technology, was included in the 2009 collaboration between Sanofi and Oxford BioMedica and has successfully completed pre-clinical studies. As previously confirmed on 19 November 2013, Oxford BioMedica was awarded a £1.8 million grant by the UK's innovation agency, the Technology Strategy Board, which will facilitate the Company funding a Phase I/IIa clinical study for EncorStat® which is planned to be conducted at Moorfields Eye Hospital, London, and is expected to start recruitment in 2015.
