— Positive DSMB review of first patient cohort in UshStat® Phase I/IIa Study —
— Further encouraging data from RetinoStat® and StarGen™ clinical studies —
Oxford, UK – 20 November 2012: Oxford BioMedica plc (“Oxford BioMedica” or “the Company”) (LSE: OXB), the leading gene-based biopharmaceutical company, and its partner Sanofi (EURONEXT: SAN and NYSE: SNY) today announce 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. The Company also announces further data from the ongoing RetinoStat®and StarGen™ clinical studies. All three ocular gene therapies were designed and developed by Oxford BioMedica using the Company’s proprietary LentiVector® gene delivery technology.
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)
Highlights of ongoing RetinoStat® Phase I study in “wet” age-related macular degeneration
- Three patient cohorts complete (n=9, ascending dose levels 1, 2 and 3), treatment of final patient cohort ongoing (n=9, confirmatory dose level) across two clinical centres
- Long-term safety profile now up to 19 months post-treatment (dose level 1)
- Successful retinal transduction, as shown by substantial increase in expression and secretion of endostatin and angiostatin proteins measured in the anterior chamber of the eye following a single administration of RetinoStat®
- Long-term protein expression1: now sustained for up to one year post-treatment at dose levels 1 and 2, and up to two months at dose level 3
- Further preliminary data continue to show a dose response, with the escalation to dose levels 2 and 3 yielding an increase in average protein expression
1.As at latest available time points
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)
John Dawson, Chief Executive Officer of Oxford BioMedica, said: “Given recent positive developments in the field, we believe gene therapy is the future – particularly in fighting ocular disease. We remain very encouraged by the continued progress across our ocular programmes partnered with Sanofi and, having successfully initiated three pioneering first-in-man studies, we are well-placed to leverage our expertise and exploit new opportunities utilising our proprietary LentiVector® platform technology.”
The RetinoStat® open label, dose escalation Phase I study will enrol 18 patients with “wet” AMD and will evaluate three dose levels to assess safety and aspects of ocular physiology. In the US, the study is led by Professor Peter Campochiaro at the Wilmer Eye Institute at Johns Hopkins, Baltimore and by Dr Andy Lauer at the Oregon Health and Science University, Portland, Oregon.
The StarGen™ open label, dose escalation Phase I/IIa study will enrol up to 28 patients and will evaluate three dose levels for safety, tolerability and aspects of biological activity. In the US, the study is led by Professor David Wilson at the Oregon Health and Science University, Portland, Oregon. In France, Professor Jose-Alain Sahel leads the study at Centre Hospitalier National d’Ophtalmologie des Quinze-Vingts, Paris. StarGen™ has received European and US Orphan Drug Designation which brings development, regulatory and commercial benefits.
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 which brings development, regulatory and commercial benefits.