ReNeuron Release: Reports Positive Results In Phase II Stroke Trial
ReNeuron Group plc (AIM: RENE), a UK-based global leader in the development of cell-based therapeutics, is pleased to announce positive data from the Company’s Phase II clinical trial (PISCES II) of its CTX cell therapy candidate for stroke disability.
PISCES II is a single arm, open-label study in patients living with disability resulting from ischaemic stroke. The study’s primary endpoint was for two patients to reach a minimum two-point improvement in the grasping and lifting test, sub-test number 2, of the Action Research Arm Test (“ARAT”), at three months post-treatment. Three of the 21 patients achieved this at three, six or twelve months respectively after treatment and were within a group of four responders who also showed clinically relevant improvements on the total ARAT score of arm motor performance. Although the ARAT sub-test number 2 study endpoint was not met as some responses came later than the three-month target, the result is nonetheless highly encouraging.
Strongly positive results were also seen in the other endpoints of the study, with seven patients (33%) showing a clinically relevant improvement on the Modified Rankin Scale (a measure of disability and dependence) and eight patients (38%) showing a clinically relevant improvement on the Barthel Index (a measure of performance in activities of daily living). In total, 15 out of 21 patients had a clinically significant response on at least one efficacy measure. Improvements in the ARAT scores, Modified Rankin Scale and Barthel Index were all sustained throughout the follow up period.
As a result of the positive data from the PISCES II study, the Company intends to apply to the US and European regulatory authorities to commence a randomised, placebo-controlled, pivotal clinical trial in patients who are living with disability post-stroke.
Patients in the PISCES II study were monitored prior to treatment to ensure that their disability was stable and showing no spontaneous improvement. A dose of 20 million CTX cells was administered to the patients between two and thirteen months (median seven months) after the stroke via direct injection adjacent to the area of the brain with the stroke damage. Patients were monitored for changes in arm function as well as a range of measures to assess disability and activities of daily living. All 21 patients in the study have completed three-month follow-up, with ten patients followed for six months and three for twelve months. Further data will continue to be collected until all patients have reached 12 months post treatment.
The PISCES II study also demonstrated that the CTX treatment was well tolerated. The most common adverse events were transitory and related to the surgical procedure, such as headache and nausea. Safety and efficacy data from the study will be presented at forthcoming stroke and rehabilitation medical conferences.
Professor Keith Muir, SINAPSE Professor of Clinical Imaging, Division of Clinical Neurosciences at the University of Glasgow, and Principal Investigator of the PISCES II study, said:
“The findings of the PISCES II study are encouraging in that the CTX treatment shows improvements both in specific neurological problems, such as arm function, and also in more general disability and independence. These improvements occurred in sufficient numbers of patients to warrant further investigation in a larger, controlled clinical study. Further, the CTX treatment was well tolerated in this Phase II study, which confirms and adds to the results of the earlier Phase I clinical trial. We look forward to the opportunity to further test the efficacy of this potential new treatment for disabled stroke survivors.”
Olav Hellebø, Chief Executive Officer of ReNeuron, said:
“We are delighted that the PISCES II clinical trial has shown our CTX cell therapy candidate has the potential to become a treatment option for patients living with chronic consequences following stroke. We are particularly excited by the response rate seen on the measures relating to disability and activities of daily living, given that these are the most important for patients and their carers. These measures are also the ones viewed by regulatory authorities as most relevant for late-stage clinical development.
“We are extremely grateful to the patients who volunteered for the PISCES II study and to the stroke research teams across the UK who have been involved in this ground-breaking clinical trial.
“Based on these positive results we will move into a controlled clinical trial to further assess the efficacy of CTX treatment in a significantly larger cohort of patients. We have a strong balance sheet to fund this pivotal study as well as reaching other important milestones across our development pipeline.“
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