ReNeuron UK shows clinical benefit in Phase 1 stroke trial using Neural Cells

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Exciting result for ReNeuron and stroke patients...

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Interim data from clinical trial of ReNeuron

Interim data from clinical trial of ReNeuron’s stem cell therapy for stroke to be presented at leading scientific conference. Data show no safety concerns and evidence of sustained reductions in neurological impairment and spasticity

Guildford, UK, 14 June 2012: ReNeuron is pleased to announce the presentation of interim data from the PISCES (Pilot Investigation of Stem Cells in Stroke) clinical trial of its ReN001 stem cell therapy for disabled stroke patients.

In this open label, dose-ranging Phase I safety study, ReNeuron’s ReN001 stem cell therapy is being administered in ascending doses to a total of 12 stroke patients who have been left disabled by an ischaemic stroke, the most common form of the condition.

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.

To date, six patients have been treated in the PISCES stroke study, representing the first two of four dose cohorts. The interim data being presented are from the first five patients treated, at 2 x 12 month, 1 x six month and 2 x three month follow-up points.

No cell-related adverse events or adverse immune-related responses were reported in any of the patients treated to date. A number of the patients experienced minor procedure-related adverse events such as asymptomatic bleeds or superficial scalp infections at the implantation wound site.

Reductions in neurological impairment and spasticity were observed in all five patients compared with their stable pre-treatment baseline performance and these improvements were sustained in longer term follow-up.

Neurological deficits were measured using the National Institutes of Health Stroke Scale (NIHSS), a higher score representing a worse deficit. Patients are required to have a NIHSS score of at least 6 to participate in the study. The pre-treatment median score for the first five patients was 8 (range 6 to 10) and the three month post-treatment median score was 4 (range 3 to 9).

Spasticity was measured using the Summated Ashworth Scale for affected upper and lower limbs, a higher score representing a higher degree of spasticity. The pre-treatment mean score for the first five patients was 28.6 (range 12 to 55) and the three month post-treatment mean score was 21.8 (range 5 to 42).

Functional magnetic resonance imaging (fMRI) data were also collected pre- and post-treatment to identify potential biomarkers of change in neurological function in the brains of the treated patients. Some longitudinal changes in motor activation fMRI were seen, consistent with the observed improvements in neurological measures.

The interim data from the PISCES clinical trial is being presented today as a poster presentation by the clinical team at Glasgow at the 10th Annual Meeting of the International Society for Stem Cell Research in Yokohama, Japan, running from 13-16 June 2012.

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.

As announced recently, the Company expects that, subject to a continuing lack of cell-related adverse events and affirmative Data Safety Monitoring Board advice, the remaining higher dose cohorts in the PISCES study will have been treated within the next twelve months, leaving the Company on track to submit an application for a Phase II clinical study with ReN001 during the course of 2013.

Michael Hunt, Chief Executive Officer of ReNeuron, said:

“The PISCES clinical trial is primarily a safety study and we must therefore treat any of the observed early indications of functional benefit of our ReN001 stroke treatment with considerable caution at this stage. That said, we remain encouraged by the results seen in the study to date and we look forward to providing further updates on the study as the higher dose cohorts are treated and to progressing our planning for further clinical trials with ReN001.”

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 to date. The data indicate that the ReN001 treatment has a good safety profile at the doses administered thus far. The preliminary signals of potential functional benefit, whilst intriguing, will require further investigation in a suitably designed Phase II efficacy study. The clinical team looks forward to dosing patients in the remaining higher dose cohorts in the PISCES study over the coming months.”


Enquiries:

ReNeuron +44 (0) 1483 302560
Michael Hunt, Chief Executive Officer
Dr John Sinden, Chief Scientific Officer
Buchanan +44 (0) 20 7466 5000
Mark Court, Sophie Cowles

Cenkos Securities +44 (0) 20 7397 8900
Stephen Keys, Adrian Hargrave (NOMAD and Broker)
Andy Roberts (Sales)

Stuart Forsyth, Ross Barker +44 (0) 141 330 8593
Media Relations, University of Glasgow


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 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 Reneuron.

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Just approved to proceed to higher dosage by DSMB

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ReNeuron?s stroke therapy given green light by safety board - Proactiveinvestors (UK)
Stem cell specialist ReNeuron (LON:RENE) has revealed the clinical trial of its therapy for disabled stroke patients has been given the go-ahead by the independent Data Safety Monitoring Board (DSMB).

After reviewing safety data from the first two dose cohorts of six patients treated, the board has recommended that the PISCES trial of its ReN001 stem cell treatment advances to the evaluation of a higher dose in the third of four dose cohorts to be treated in the study.

ReNeuron said that two of the six trialled are through 18 month follow-up, while the rest are through 12 month, nine month, six month and three month follow-up.

The company added that none of the patients experienced any adverse side effects.

“We are delighted that the DSMB have given a favourable recommendation to proceed to the higher and penultimate dose in the PISCES stroke trial,” said ReNeuron’s chief executive Michael Hunt.

“This represents a further important milestone for the study as we make our plans for a subsequent Phase II clinical trial application with ReNeuron's ReN001 cell-based treatment for stroke disability.”

Stroke is the third largest killer and the single largest cause of adult disability in the developed world.

The PISCES study is testing the safety and tolerability of the treatment of ascending doses of the ReN001 cells in patients with moderate to severe functional neurological impairments from their strokes in what is the world’s first fully regulated clinical trial.

In June this year, the firm revealed the first five PISCES patients showed reductions in neurological impairment and spasticity and these were maintained in the longer-term.

The company expects that the remaining patients in the study – who have already been identified and evaluated as potential candidates – will be signed up by the early part of next year.

This will mean it can apply for a Phase II clinical study with ReN001 by mid-2013.

The company also announced that the first patient in this third dose cohort of three patients was successfully treated with ReN001 and discharged from hospital with no side effects.

Shares opened almost nine per cent higher to 3.5 pence each on the news.