Positive outcome for VR040 in Parkinson?s trial 16 November 2010
Rapid onset of therapeutic benefit
Chippenham, UK – 16 November 2010: Vectura Group plc (“Vectura”; LSE: VEC) today announces positive data from a Phase II clinical study for its inhalation product, VR040, for the treatment of "off" episodes (periods of inability to control movement) in patients with fluctuating Parkinson’s disease. VR040 is Vectura's proprietary formulation of apomorphine, delivered to the bloodstream by inhalation through the lungs using the Company’s proprietary dry powder inhalation (DPI) technology. VR040 was designated an orphan drug by the European Medicines Agency (EMA) and is being developed using the EMA scientific advice procedure.
The results of the study demonstrate that VR040 improves the control of movement in patients with Parkinson’s disease. VR040 produced clinically relevant and statistically significant improvement, compared with placebo (p=0.023), of the Unified Parkinson’s Disease Rating Scale Part III (UPDRS III), a measure of a patient’s ability to control movement (see Notes for editors).
VR040 also reduced the time patients were in an “off” state by over 2 hours, a change considered by the investigators to be highly clinically relevant, although not statistically significant (p=0.078), when compared with placebo in the relatively small number of patients tested. The mean onset of therapeutic benefit was rapid, with patients converting to an “on” state in less than 9 minutes after taking VR040, with some patients responding just one minute after receiving their dose. VR040 was well tolerated with no reports of severe adverse events.
Fifty-five patients, diagnosed with fluctuating idiopathic Parkinson’s disease, were randomised to receive either placebo or VR040. Patients were rapidly titrated to a dose of VR040 that elicited a therapeutic response and continued to use this dose in the home setting to treat “off” episodes.
Dr Donald Grosset, BSc (Hons), MBChB, MD, Consultant Neurologist in the Institute of Neurological Sciences, Southern General Hospital, Glasgow, UK and Principal Investigator of the study, commented:
"The study was a first opportunity to evaluate inhaled apomorphine in the home environment with patients reporting clinically significant reductions in their mean daily off time. It is reassuring to see that the safety and therapeutic benefit of VR040 have now been reproduced across three separate trials in 126 Parkinson’s disease patients.”
Dr Chris Blackwell, Chief Executive of Vectura, added:
"This study confirms the potential therapeutic benefit of VR040 to Parkinson’s patients, and in three separate studies, that it is a safe, easy to use and non-invasive treatment. These data will also be of value in ongoing discussions with prospective licensing partners. This completes Vectura’s planned investment in the clinical development of VR040.”
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Chris Blackwell, Chief Executive
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Notes for editors
About Parkinson’s disease
Parkinson’s disease (PD) is a progressive neurological disorder that leads to the loss of dopamine-containing neurons in the basal ganglia of the brain. It is estimated that approximately one million people in Europe and 1.5 million people in the US have PD. As the disease progresses, therapeutic control diminishes and approximately 80% of patients experience motor complications (so called "off" episodes) 5–10 years post initiation of levodopa treatment. The inhaled VR040 treatment aims to provide rapid, convenient, non-invasive relief from such symptoms.
The Unified Parkinson Disease Rating Scale (UPDRS) rates a patient across 31 separate items, each with a score of 0–4. The rating scale covers three areas: mentation, behaviour and mood (4 items); activities of daily living (13 items); and motor examination (14 items). The latter component, UPDRS III, is routinely employed across clinical studies to measure changes in motor function as a result of treatment administration.
Results from previous VR040 studies
In a proof-of-concept study, six doses of VR040 and placebo were evaluated in 24 PD patients who experience "off" episodes. Five patients converted to a full "on" state having inhaled VR040, compared with one patient following inhalation of placebo.
In a study to evaluate the safety and efficacy of four different doses of VR040 up to 3.2 mg in 47 PD patients, VR040 demonstrated statistically significant improvement in the primary endpoint of mean change in UPDRS III relative to that seen with placebo (p=0.016). There was a rapid onset of action; the majority of patients achieving conversions within 10 minutes of inhaling apomorphine. Some patients reported conversion as quickly as 2 minutes after taking their medication. As in previous studies, VR040 was well tolerated with no reports of treatment-related severe adverse events and no patient withdrawals resulting from safety concerns.
Vectura Group plc develops inhaled therapies principally for the treatment of respiratory diseases. Vectura’s main products target diseases such as asthma and chronic obstructive pulmonary disease (COPD), a growing market that is currently estimated to be worth in excess of $25 billion. Vectura also develops products for other lung pathologies and non-respiratory diseases.
Vectura has six products marketed by its partners and a portfolio of drugs in clinical and pre-clinical development, some of which have been licensed to major pharmaceutical companies. Vectura seeks to develop certain programmes itself where this will optimise value. Vectura’s formulation and inhalation technologies are available to other pharmaceutical companies on an out-licensing basis where this complements Vectura’s business strategy.
Vectura has development collaborations and licence agreements with several pharmaceutical companies, including Novartis, Sandoz (the generics arm of Novartis), Baxter, GlaxoSmithKline (GSK) and Otsuka. For further information, please visit Vectura’s website at www.vectura.com.
This press release contains forward-looking statements, including statements about the discovery, development and commercialisation of products. Various risks may cause Vectura’s actual results to differ materially from those expressed or implied by the forward-looking statements, including: adverse results in clinical development programmes; failure to obtain patent protection for inventions; commercial limitations imposed by patents owned or controlled by third parties; dependence upon strategic alliance partners to develop and commercialise products and services; difficulties or delays in obtaining regulatory approvals to market products and services resulting from development efforts; the requirement for substantial funding to conduct research and development and to expand commercialisation activities; and product initiatives by competitors. As a result of these factors, prospective investors are cautioned not to rely on any forward-looking statements. We disclaim any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.
Posted: November 2010