Guildford, UK, 1 September 2011: ReNeuron provides an update on progress with the PISCES Phase I clinical trial of its ReN001 stem cell therapy for disabled stroke patients
The Company is pleased to report that the independent Data Safety Monitoring Board (DSMB) for the clinical trial has recommended that the trial advances to the evaluation of a higher dose of ReN001. In arriving at this recommendation, the DSMB reviewed safety data from the first dose cohort of three patients treated with ReN001. The first patient treated in the cohort was assessed at nine months post-treatment, the second patient at six months and the third patient at three months.
No cell-related adverse events have been reported in the clinical trial and data from the laboratory safety tests, neurological examinations and neurofunctional tests conducted thus far indicate that the ReN001 treatment is safe and well-tolerated at the initial dose. Although preliminary in nature, these data have also enabled some early progress to be made regarding the secondary objective of the trial, namely the evaluation of appropriate clinical measurements for use in the design of future proof-of-concept clinical trials with ReN001.
As previously reported, the Company expects that the next dose cohort of three further patients will have been treated by the end of this year assuming no significant recruitment delays. The remaining dose cohorts in the PISCES trial are expected to be treated in 2012, at which point ReNeuron intends to have discussed and agreed its subsequent clinical development strategy for ReN001 with the relevant regulatory authorities both in the UK and beyond.
The PISCES study (Pilot Investigation of Stem Cells in Stroke) is the world’s first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients. Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world. The trial is being conducted in Scotland at the Institute of Neurological Sciences, Southern General Hospital, Greater Glasgow and Clyde NHS Board. In this Phase I single administration dose escalation study, the ReN001 stem cell therapy is being administered to a total of 12 stroke patients who have been left disabled by an ischaemic stroke, the most common form of the condition. The aim of the study is to test the safety and tolerability of the treatment in progressive doses while evaluating efficacy measures for the design of future clinical trials with ReN001, including structural and functional MRI imaging measures as well as a number of tests of sensory, motor and cognitive functions. The Principal Investigator for the trial is Professor Keith Muir, SINAPSE Professor of Clinical Imaging, Centre for Stroke Research, Institute of Neuroscience & Psychology at the University of Glasgow. Patients in the clinical trial will be monitored for two years, with longer term follow-up procedures in place thereafter.
Professor Muir will present further data regarding progress with the PISCES trial at the Stroke Association’s 6th UK Stroke Forum Conference in Glasgow from 29 November to 1 December 2011.
Professor Muir commented:
“We are pleased that there have been no safety issues from the first dose cohort in the PISCES trial and we look forward to evaluating further patients at a higher dose. ReN001 has the potential to address a very significant unmet medical need in disabled stroke patients and I am pleased that our team is involved in this pioneering clinical trial.”
Michael Hunt, Chief Executive Officer of ReNeuron, added:
“We are delighted that the DSMB have given a favourable recommendation to proceed to a higher dose in the PISCES stroke trial. This represents an important milestone for the trial and the preliminary data from the trial also add value to our other therapeutic programmes using the CTX neural stem cell line that forms the basis of the ReN001 stroke treatment. We look forward to providing further updates on the PISCES clinical trial in due course.”
Michael Hunt, Chief Executive Officer – ReNeuron +44 (0) 1483 302560
Dr John Sinden, Chief Scientific Officer – ReNeuron
Lisa Baderoon, Mark Court, Isabel Podda +44 (0) 20 7466 5000
Antony Legge, Oliver Rigby +44 (0) 20 7776 6550
Daniel Stewart & Company plc (Nomad and Joint Broker)
James Gallagher, Steve Waterman +44 (0) 20 3206 7000
Matrix Corporate Capital LLP (Joint Broker)
Ross Barker, Media Relations Officer +44 (0) 141 330 3535
University of Glasgow
Approximately 150,000 people suffer a stroke in the UK each year. The vast majority of these strokes are ischaemic in nature, caused by a blockage of blood flow in the brain (as opposed to a haemorrhagic or bleeding stroke).
Approximately one half of all stroke survivors are left with permanent disabilities as a result of the damage caused to brain tissue arising from the stroke. The annual health and social costs of caring for these patients is estimated to be in excess of £5 billion in the UK, with stroke patients estimated to be occupying at least 25 per cent of long term hospital beds.
The only current treatment for ischaemic stroke patients occurs in the acute phase of the condition (within several hours of the stroke), when anti-clotting agents are administered to dissolve the clot causing the blockage in blood flow to the brain. Only a small proportion of patients get to the hospital in time to be treated in this way.
Beyond the acute phase, there are no existing treatments, other than preventative or rehabilitation measures, to alleviate the disabilities suffered by stroke patients who have survived their stroke.
Source: UK Stroke Association
About ReNeuron’s ReN001 stem cell therapy for stroke
ReNeuron’s ReN001 cell therapy for stroke consists of a neural stem cell line, designated CTX, which has been generated using the Company’s proprietary cell expansion and cell selection technologies and then taken through a full manufacturing scale-up and quality-testing process. As such, ReN001 is a standardised, clinical and commercial-grade cell therapy product capable of treating all eligible patients presenting.
ReN001 has been shown to reverse the functional deficits associated with stroke disability when administered several weeks after the stroke event in relevant pre-clinical models of the condition. Extensive pre-clinical testing also indicates that the therapy is safe, with the ReN001 cells eventually cleared from the body with no adverse safety effects arising.
If ultimately shown to be safe and effective clinically, ReN001 would therefore offer a significant new treatment option for stroke survivors. The therapy offers the potential for a degree of recovery of function in disabled stroke patients, resulting in greater independence and quality of life for these patients and reduced reliance on health and social care systems.
The ReN001 cells that are being used in the initial clinical trial are taken from the existing manufactured cell banks that will form the basis of the eventual marketed product. There will therefore be no need to re-derive and test new ReN001 cell lines for subsequent clinical trials or for the market – all such cells can simply be expanded from the existing banked and tested product.
About the Institute of Neurological Sciences at the University of Glasgow
The clinical Stroke Research Group of the Division of Clinical Neurosciences is based at the Institute of Neurological Sciences at the University of Glasgow and has major collaborations, internally with the Glasgow Experimental MRI Centre, with SINAPSE (Scottish Imaging Network: A Platform for Scientific Excellence), and with the Translational Medicine Research Initiative (TMRI). Around 900 patients per year are admitted through the Acute Stroke Unit, which provides stroke services to the population of south Glasgow and specialist stroke treatments for the West of Scotland.
The unit is the highest user of acute clot-busting (thrombolytic) treatment in the UK at present, and has been extensively involved in clinical trials in stroke. Major research interests include evaluation of advanced brain imaging techniques in acute stroke, development of novel brain imaging techniques, improving the use of clot-busting drug treatments in stroke, and developing trial methodology for evaluation of regenerative treatments. The group has support from the Stroke Association, the Medical Research Council, and the TMRI. Further work with regenerative strategies include collaborations with groups developing both drug-based and stem cell therapies across Europe.
ReNeuron is a leading, clinical-stage stem cell business. Its primary objective is the development of novel stem cell therapies targeting areas of significant unmet or poorly met medical need.
ReNeuron has used its unique stem cell technologies to develop cell-based therapies for significant disease conditions where the cells can be readily administered “off-the-shelf” to any eligible patient without the need for additional immunosuppressive drug treatments. ReNeuron’s lead candidate is its ReN001 stem cell therapy for the treatment of patients left disabled by the effects of a stroke. This therapy is currently in clinical development. The Company is also developing stem cell therapies for other conditions such as peripheral arterial disease, a serious and common side-effect of diabetes, and blindness-causing diseases of the retina.
ReNeuron has also developed a range of stem cell lines for non-therapeutic applications – its ReNcell® products for use in academic and commercial research. The Company’s ReNcell®CX and ReNcell®VM neural cell lines are marketed worldwide under license by USA-based Merck Millipore.
ReNeuron’s shares are traded on the London AIM market under the symbol RENE.L. Further information on ReNeuron and its products can be found at www.reneuron.com.
This announcement contains forward-looking statements with respect to the financial condition, results of operations and business achievements/performance of ReNeuron and certain of the plans and objectives of management of ReNeuron with respect thereto. These statements may generally, but not always, be identified by the use of words such as “should”, “expects”, “estimates”, “believes” or similar expressions. This announcement also contains forward-looking statements attributed to certain third parties relating to their estimates regarding the growth of markets and demand for products. By their nature, forward-looking statements involve risk and uncertainty because they reflect ReNeuron’s current expectations and assumptions as to future events and circumstances that may not prove accurate. A number of factors could cause ReNeuron’s actual financial condition, results of operations and business achievements/performance to differ materially from the estimates made or implied in such forward-looking statements and, accordingly, reliance should not be placed on such statements.