Vectura announces successful outcome of phase II study on inhaled parkinson’s disease product – VR040
08 Aug 2006
Vectura announces successful outcome of Phase II study on inhaled parkinson's disease product – VR040
Chippenham, UK – 8 August 2006: Vectura Group plc (LSE: VEC) ("Vectura") today announces the successful outcome of a Phase II "proof-of concept" clinical study for its product, VR040, for the treatment of induced "off" periods in patients with Parkinson's disease (PD).
The study demonstrated that VR040 was safe, well tolerated and successfully recovered patients from an induced "off" episode with a rapid onset of action, which was also durable. VR040 is Vectura's proprietary formulation of apomorphine, delivered by oral inhalation to the lungs using Vectura's Aspirair® dry powder inhaler (DPI).
PD is a progressive neurological disorder that leads to the loss of dopamine containing neurons in the basal ganglia of the brain. People with PD often experience trembling, muscle rigidity, and difficulty walking. As the disease progresses, therapeutic control diminishes and patients experience motor complications ("off" episodes) which become more frequent, and severe, and are disabling and often of sudden onset. VR040 aims to provide rapid, non-invasive relief from such symptoms.
The randomised, double-blind, placebo-controlled, four parallel group, ascending dose study was designed to assess the safety and tolerability profile of VR040. Preliminary pharmacokinetic (PK) analysis and an assessment of efficacy were also undertaken.
The study evaluated six fine particle doses (200µg, 300µg, 500µg, 750µg, 800µg and 1,200µg) and placebo in 24 idiopathic PD patients who experience "off" episodes. Each patient received one or two doses. On visiting the clinic, a patient's conventional PD treatment was withdrawn and an "off" episode induced. Safety and efficacy assessments were conducted once a patient confirmed existence of "off" status and were continued post-administration of randomised study treatment.
Recovery from "off" status was determined by patient self-assessment of disease state with the time to onset of effect and duration of effect recorded. Safety was determined via vital signs, lung function, volunteered adverse events (AE) and electrocardiograph (ECG) measurements with blood samples collected for measurement of plasma apomorphine concentration and subsequent PK analysis.
No serious or severe adverse events were reported at any dose and no patients withdrew from the study. The profile of adverse events for all treatments, including placebo, was similar with no reports of hypotension (low blood pressure) or syncope (fainting).
Pharmacokinetic analysis confirmed that maximum apomorphine plasma concentrations were measured 1-3 minutes after doses were inhaled.
Whilst the study was not powered for meaningful statistical comparisons, apomorphine-induced conversion from an "off" state was observed in 10 out of 18 patients. The median onset of therapeutic effect in responders was 10 minutes (and as rapid as 4 minutes) after inhalation of apomorphine, the effect lasting for up to 60 minutes, with a median duration of 25 minutes. Five patients converted to a full "on" state having inhaled VR040, compared with one patient following inhalation of placebo.
Dr Chris Blackwell, Chief Executive of Vectura, said:
"This study has demonstrated that VR040 can provide rapid relief from "off" episodes for patients with Parkinson's disease, within minutes of inhalation. This bodes well for future clinical development, which will now focus on determining optimal titrated doses of VR040. The funds raised in our recent share placing provide us with the opportunity to generate significant value from VR040 as we proceed through these latter stages of development."
Dr Donald Grosset, BSc (Hons), MBChB, MD, Consultant Neurologist in the Institute of Neurological Sciences, Southern General Hospital, Glasgow and Principal Investigator of the study, commented:
"This study shows that high plasma levels can be achieved very quickly with VR040, which translate into a very quick onset of benefit for patients with PD who have motor fluctuations in the more advanced stages of their condition."
Enquiries
Vectura Group plc +44 (0) 1249 667 700
Chris Blackwell, Chief Executive
Anne Hyland, Chief Financial Officer
Financial Dynamics +44 (0) 207 831 3113
David Yates/Sarah MacLeod/John Gilbert
Notes for Editors
About Parkinsons Disease ("PD") It is estimated that approximately 1 million people in Europe and 1.5m people in the US have PD approximately 10% of these experience sever on/off motor fluctuations which do not respond to other therapies apart from apomorphine.
About Vectura
Vectura's principal focus is the development of a range of inhaled drugs for the treatment both of lung diseases and other conditions where optimised delivery via the lungs can provide significant benefits, such as a rapid onset of action, improved efficacy and improved tolerability compared with current therapies.
Vectura's products combine its proprietary, innovative, pulmonary formulation and device technologies (Aspirair® GyroHaler® and PowderHale®) with existing, off-patent drugs either for use in new indications or to provide inhalation as an improved route of administration. Using drugs that have already been approved in some form in at least one major pharmaceutical market lowers the risk of product development failure compared to new chemical entities, Vectura is able to secure patent protection for its portfolio by identifying new indications for off-patent compounds and applying the Company's proprietary delivery technologies to create new methods of administration. The Company has development collaborations with a number of companies, including Boehringer Ingelheim, Novartis, GSK and Chiesi and an un-named leading international pharmaceutical company. For further information, please visit Vectura's website at www.vectura.com.
