OXFORD, England, June 22, 2011 /PRNewswire/ --
-RetinoStat® Progress in Phase I Study in Wet Age-Related Macular Degeneration
Oxford BioMedica plc ("Oxford BioMedica" or "the Company") (LSE: OXB), a leading gene therapy company, today announces that the first dose level of RetinoStat® is safe and well-tolerated at one month following treatment. RetinoStat® is a novel gene-based treatment for neovascular "wet" age-related macular degeneration (AMD), designed and developed by Oxford BioMedica using the Company's proprietary LentiVector® gene delivery technology. It is the lead programme of the ocular agreement the Company signed with Sanofi (EURONEXT: SAN andNYSE: SNY) in April 2009.
The on-going Phase I study will enrol 18 patients with wet AMD at the Wilmer Eye Institute at Johns Hopkins, Baltimore (USA). Led by Professor Peter Campochiaro, the study will evaluate three dose levels and assess safety, aspects of visual acuity and ocular physiology. Three patients received the first dose level of RetinoStat® and one-month results have been assessed by the study's independent Data Safety Monitoring Board (DSMB). First results from the study are expected to be announced in H1 2012.
Highlights of first patient cohort at one month (dose level 1)
- Favourable safety profile with no serious adverse events related to RetinoStat® or its method of administration
- No signs of inflammation in the eye
- DSMB support received to proceed to dose level 2 in the next patient cohort
John Dawson, Chief Executive Officer of Oxford BioMedica, said: "This clinical study is breaking new ground as the first to directly administer a lentiviral vector-based treatment to patients in the US and the favourable safety profile of RetinoStat® is encouraging. We continue to work closely with the regulatory bodies in order to advance our novel ocular programmes as rapidly as possible and, together with Sanofi, we look forward to further progress during 2011."
The second ocular product to enter clinical development is StarGen™, a novel gene-based treatment for Stargardt disease, and 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. StarGen™ has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits and, with no currently approved treatment, this novel product brings significant hope for the future to Stargardt patients. The next product expected to enter Phase I/II clinical development is UshStat® for the treatment of Usher syndrome type 1B in H2 2011. UshStat® has also received European and US Orphan Drug Designation.
"We are delighted to see Oxford BioMedica's lentiviral gene therapy products successfully moving into human studies,"said Stephen Rose, Ph.D., chief research officer of the Foundation Fighting Blindness, a US non-profit organisation that provided early funding for Oxford BioMedica's pre-clinical ocular programme. "The Company's innovative treatments hold great promise for saving vision in people affected by a broad range of devastating eye diseases for which current treatments are limited or non-existent."
Notes to editors
1. Oxford BioMedica®
Oxford BioMedica plc (LSE: OXB) is a biopharmaceutical company developing innovative gene-based medicines and therapeutic vaccines that aim to improve the lives of patients with high unmet medical needs. The Company's technology platform includes a highly efficient LentiVector® gene delivery system, which has specific advantages for targeting diseases of the central nervous system and the eye; and a unique tumour antigen (5T4), which is an ideal target for anti-cancer therapy. Through in-house and collaborative research, Oxford BioMedica has a broad pipeline and its partners include Sanofi, Sigma-Aldrich and Pfizer. Further information is available at http://www.oxfordbiomedica.co.uk.
2. Oxford BioMedica's agreement with Sanofi
Under the terms of the agreement signed with Sanofi 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" AMD, StarGen™ for Stargardt disease, UshStat® for Usher syndrome 1B and EncorStat® for corneal graft rejection. Oxford BioMedica granted Sanofi 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 can exercise its option to license the products and will then assume responsibility for on-going activities. Sanofi 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.
3. LentiVector® gene delivery technology
Oxford BioMedica's LentiVector® platform technology is one of the most advanced gene delivery systems currently available, which has many applications in product development and discovery research. It is the system of choice for gene-based treatments addressing chronic and inherited diseases. Oxford BioMedica has established a dominant intellectual property estate in the field of lentiviral-vector mediated gene delivery through its in-house research and from work conducted by the Company's co-founders at Oxford University.
4. Age-Related Macular Degeneration and RetinoStat®
Age-related macular degeneration (AMD) is a major cause of blindness affecting an estimated 25 to 30 million people worldwide and the incidence of AMD is expected to triple by the year 2025 (source: AMD Alliance International). Neovascular "wet" AMD accounts for the majority of all severe vision loss from the disease. 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. On the basis of pre-clinical data, it is anticipated that RetinoStat® will require only a single administration which would give the product a significant advantage in the market over currently available treatments that often require frequent, repeated administration.
Sanofi, a global and diversified healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi has core strengths in the field of healthcare with seven growth platforms: diabetes solutions, human vaccines, innovative drugs, rare diseases, consumer healthcare, emerging markets and animal health. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY).
6. Foundation Fighting Blindness
The Foundation Fighting Blindness is a publicly-supported charity raising money to fund research for macular degeneration, retinitis pigmentosa (RP), Usher syndrome, Stargardt disease and related ocular diseases.
For further information, please contact:
Oxford BioMedica plc:
Lara Mott, Head of Corporate Communications
Emma Thompson/Katja Toon/Amber Bielecka
SOURCE Oxford Biomedica Plc