GUILDFORD, UK I 28 May 2013 I ReNeuron provides an update on progress with the PISCES clinical trial of its ReN001 stem cell therapy for disabled stroke patients.
Interim data from the first nine patients treated in the PISCES study are being presented by the clinical team from Glasgow’s Southern General Hospital at the 22nd European Stroke Conference, taking place in London this week.
There were no cell-related or immunological adverse events reported in any of the patients treated. Sustained reductions in neurological impairment and spasticity were observed in most patients compared with their stable pre-treatment baseline performance, reflected in the summary evaluation scores below:
• National Institutes of Health Stroke Scale (neurological deficit): improved by median 1 point at 1 month post-treatment (n=9) and 3 points at 1 year (n=5)
• Barthel Index (functional outcome): improved by median 1 point at 3 months (n=8) and 4 points at 1 year (n=5)
• Modified Rankin Score (disability and handicap): improved by median 1 grade at 1 year (n= 5)
• Summated Ashworth scores for affected limbs (spasticity): improved by mean 4.5 at 3 months (n=9) and 7.2 at 1 year (n=5).
Since the above data were collated, the remaining patients in the PISCES study have been treated, with no subsequent cell-related or immunological adverse events reported. As previously announced, the Company has cleared all points arising from the regulatory review of its proposed UK multi-site Phase II clinical trial to examine the efficacy of ReN001 in patients disabled by an ischaemic stroke. The Company is also pleased to announce that this Phase II study has been adopted by the NHS National Institute for Health Research Stroke Research Network (SRN). This important endorsement will enable the Company to work closely with the SRN to optimise performance against defined targets regarding site set-up, patient recruitment and monitoring activities across the various sites participating in the study.
As previously reported, the Company will seek final regulatory and ethical approvals for the Phase II stroke study by submitting a data package including three month follow-up data on the final dose cohort in the PISCES study to the UK regulatory authorities in early July and, assuming approvals are granted, expects to commence recruitment into the Phase II study shortly thereafter.
Michael Hunt, Chief Executive Officer of ReNeuron, said:
“The PISCES study continues to yield encouraging results. Assuming the remaining required short-term follow up data confirm the good safety profile of the treatment, we will be able to move the ReN001 therapy confidently into Phase II clinical development, as planned, later this year. This will represent yet another important milestone for ReNeuron, maintaining the Company’s pre-eminent position in the development of a neural stem cell-based treatment for disabled stroke patients.”
Professor Keith Muir, SINAPSE Professor of Clinical Imaging, Division of Clinical Neurosciences at the University of Glasgow, and Principal Investigator of the PISCES study, said:
“We remain pleased and encouraged by the data emerging from the PISCES study. The data to date identify no safety issues with the ReN001 treatment. The evidence of functional improvement requires further investigation in a suitably designed Phase II efficacy study and we look forward to being a principal clinical site in that study when it commences.”
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About the PISCES clinical trial
The PISCES study 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.
The primary aim of the study is to test the safety and tolerability of the treatment in ascending doses of the ReN001 cells, in patients with moderate to severe functional neurological impairments resulting from their stroke. The secondary aim of the study is to evaluate efficacy measures for the design of future clinical trials with ReN001, including imaging measures as well as a number of tests of sensory, motor and cognitive functions.
About stroke
Approximately 150,000 people suffer a stroke in the UK each year and approximately 800,000 in the US. 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 and over $70 billion in the US, 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; American 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, in both acute and long term safety studies.
If ultimately shown to be safe and effective clinically, ReN001 would therefore offer a ground-breaking 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 PISCES 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.
About ReNeuron
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 critical limb ischaemia, a serious and common side-effect of diabetes, and blindness-causing diseases of the retina such as retinitis pigmentosa.
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.
SOURCE: ReNeuron
