Oxford, UK – 13 July 2009: Oxford BioMedica (LSE: OXB), a leading gene therapy company, today announces an update on the Phase I/II study of its novel gene therapy, ProSavin, for the treatment of Parkinson’s disease. Patients treated at the first dose level have maintained their improvement in motor function for one year, with an average improvement of 29%. Analogous investigator assessments of patients in the second cohort treated at a higher dose level have achieved similar benefit at three months, and the first patient to reach their six-month assessment has demonstrated further improvement. The Data Monitoring Committee supported Oxford BioMedica’s proposal to proceed to a third dose level incorporating the Company’s new administration technology.
The ongoing Phase I/II study is designed to evaluate the safety and efficacy of ProSavin in patients with mid-stage Parkinson’s disease (PD) who are experiencing reduced benefit on L-DOPA ‘equivalent’ therapy. ProSavin is administered directly into the striatum of the brain using a well established surgical technique. The first stage of the study is a dose escalation in cohorts of three patients at each dose level and, to date, six patients have been treated. Three patients that received the first dose level (1x) have reached their one-year assessments and three patients at the second dose level (2x) have been assessed at three months post treatment. Motor function is assessed according to the Unified Parkinson’s Disease Rating Score (UPDRS) in patients’ ‘off state’ (i.e. after withdrawal of PD medication).
ProSavin has been safe and well tolerated in all patients, with no serious adverse events and no evidence of immunotoxicity. All patients have reduced or maintained their PD medication relative to baseline.
One year after their treatment, patients at the 1x dose have maintained their improved motor function with an average increase of 29%. All patients have enhanced their quality of life, based on a standard measure of clinical benefit that is recorded by the patient using a questionnaire known as PDQ-39. The average increase in patients’ PDQ-39 score was 42% at one year.
At three months, patients at the 2x dose have increased motor function with an average improvement of 28% and the first of these patients to be assessed at six months has shown further benefit. All patients in the second cohort have experienced a better quality of life at three months with an average improvement of 33%.
The study’s independent Data Monitoring Committee (DMC) has reviewed the data, as required by the study protocol, and supported the Company’s proposal to proceed directly to a third dose level that is five-fold higher than the first dose level. Oxford BioMedica plans to follow the DMC’s recommendation and incorporate its new delivery technology for the administration of the 5x dose level of ProSavin. The new technique reduces the surgical time, facilitates higher dosing and has the potential to provide better reproducibility as study centres expand and thus accelerate clinical development timelines. A protocol amendment for the new technology is being prepared, which will be submitted and reviewed by the French healthcare regulatory agency, AFSSAPS. The Company aims to complete the submission before the end of the third quarter of 2009.
The Principal Investigator, Professor Stéphane Palfi from the Henri Mondor Hospital in Paris, commented on the results: “The new data are highly promising, demonstrating long-lasting benefit that is comparable in some patients to the current alternative of Deep Brain Stimulation. If the higher dose levels of ProSavin can provide even greater efficacy, then ProSavin would represent a breakthrough treatment for patients who currently have limited options when they fail on current medications.”
John Dawson, Oxford BioMedica’s Chief Executive Officer, added: “We are delighted by the safety and efficacy data emerging from this first-in-man study of ProSavin. In parallel with the ongoing trial, we are designing protocols for the next stage of development, which we will present to both the FDA and EMEA. Discussions with prospective partners are also progressing well. Our objective is to complete the current study in the second half of 2010 and advance into larger studies with a partner as soon as possible.”
1. Oxford BioMedica
Oxford BioMedica (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 gene delivery system (LentiVector®), 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. Partners include sanofi-aventis, Sigma-Aldrich and Wyeth. Technology licensees include Biogen Idec, GlaxoSmithKline, Merck & Co and Pfizer. Further information is available at www.oxfordbiomedica.co.uk
2. ProSavin®
ProSavin is Oxford BioMedica’s novel gene-based therapeutic for the treatment of Parkinson’s disease. The product is administered directly to the striatum in the brain. It delivers three genes required to convert cells that normally do not produce dopamine into cells that do, thereby replacing the dopamine synthesising cells lost during the course of the disease. ProSavin utilises Oxford BioMedica’s proprietary LentiVector® system to deliver the genes AADC (aromatic amino acid decarboxylase), TH (tyrosine hydroxylase) and CH1 (GTP-cyclohydrolase 1). These genes reprogramme transduced cells to manufacture and secrete dopamine.
