Stars aligning
Long-term data from the PISCES Phase I study of Reneuron Group's (LONDON:RQE) neural stem cell product ReN001 in 11 disabled stroke patients provide further evidence of safety and encouraging signals of efficacy. The results support the recent start of a 41-patient Phase II trial in stroke disability, the outcome of which by end-2015 is a major valuation inflection point. With sufficient funds (£23.5m cash at 30 Sep 2013 and £7.8m Welsh Government grant) to Q416, the next 18 months could be transformational for ReNeuron on the clinical data front.
Encouraging signs
The PISCES study treated 11 moderately to severely disabled patients (six months to five years following an ischemic stroke) with neural stem cells (2m, 5m, 10m, 20m cells) injected into the brain. Twelve-month follow-up data showed no cell-related or immunological adverse events (reported AEs related only to injection or co-morbidities) and encouraging improvements in median disability scores vs baseline after 12 months, including: NIHSS (-3 points), Barthel Index (+3), Summated Ashworth (-7), modified Rankin (-1 in 4/11 patients) and EuroQOL (+18).
PISCES-II underway
The Phase II (PISCES-II) study with ReN001 is open for recruitment, using a new frozen cell product. The single arm, open-label study will dose (20m cells) patients with stable paresis (partial paralysis) of an arm, 8-12 weeks following an ischaemic stroke. This treatment window is considered optimum in terms of ReN001’s potential efficacy. The primary endpoint is robust and non-subjective, aimed at improving the ARAT score (Action Research Arm Test) after six months. An interim analysis in mid-2015 requires two responders in the first 21 patients, before enrolling all 41 patients. An observational study may help with recruitment into this and subsequent studies.
Filling the pipeline
A nine-patient Phase I trial with ReN009 (dosed up to 80m cells) is underway for critical (lower) limb ischaemia (CLI) and positive safety data by end 2014 could enable an 80-patient, placebo-controlled Phase II study to start in 2015. ReN003 (based on human retinal progenitor cells; hRPCs) is poised to enter a Phase I/II study in 2014.
Valuation: £35m EV heavily discounts stroke potential
With £23.5m in cash as of 30 Sep 2013, ReNeuron is funded to Q416. This covers a number of clinical trial catalysts, particularly the PISCES-II data, which could re-rate the stock and offer the potential for further funding and/or partnerships.
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