Guildford, UK, 14 August 2012: ReNeuron provides an update on progress with the PISCES clinical trial of its ReN001 stem cell therapy for disabled stroke patients.
In this open label, dose-ranging Phase I safety study, taking place in Scotland, 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 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 the third of four dose cohorts to be treated in the study. In arriving at this recommendation, the DSMB reviewed safety data from the first two dose cohorts of six patients treated with ReN001. Of these patients, two are through 18 month follow-up, one is through 12 month follow-up, one is through 9 month follow-up, one is through 6 month follow-up and one is through three month follow-up. No cell-related adverse events or adverse immune-related responses have been reported in any of the patients treated to date.
The Company is also pleased to report that the first patient in this third dose cohort of three patients has now been successfully treated with ReN001 and discharged from hospital with no acute safety issues arising.
The PISCES study is the world’s first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients. 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.
In June of this year, interim data from the PISCES study from the first five patients treated was presented by the Glasgow clinical team at the 10th Annual Meeting of the International Society for Stem Cell Research (ISSCR) in Yokohama, Japan. 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.
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 patients in the PISCES study will have been recruited and treated by early 2013, leaving the Company on track to submit an application for a Phase II clinical study with ReN001 by mid-2013. These remaining patients have already been identified and evaluated as potentially eligible for treatment in the PISCES study, subject to further screening and consenting visits.
Michael Hunt, Chief Executive Officer of ReNeuron, said:
“We are delighted that the DSMB have given a favourable recommendation to proceed to the higher and penultimate dose in the PISCES stroke trial. 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.”
Michael Hunt, Chief Executive Officer
Dr John Sinden, Chief Scientific Officer +44 (0) 1483 302560
Mark Court, Fiona Henson, Sophie Cowles +44 (0) 20 7466 5000
Stephen Keys, Adrian Hargrave (NOMAD and Broker)
Andy Roberts (Sales) +44 (0) 20 7397 8900
Stroke is the third largest cause of death and the single largest cause of adult disability in the developed world. 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.
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.