ReNeuron Group Plc (LON:RENE) has delivered encouraging results from its phase II clinical study treating stroke patients with its CTX stem cells.
Its strategy is to develop cell lines for "off the-shelf" therapies to treat serious conditions where there is unmet medical need.
Three programmes have clinical trials currently ongoing: CTX for strokes: CTX for critical limb ischaemia; and a retinitis pigmentosa programme.
Stroke trial results encouraging
The latest results from the PISCES II stroke trial, led by Professor Keith Muir of Glasgow University, monitored improvement in arm movement as measured by the ARAT sub-test number two.
The hope was to see discernible change within three months in two of the 21 patients receiving treatment.
As it turned out three patients registered improvement – though two of them were outside the timescale set by researchers.
Even so, ReNeuron called the results “highly encouraging”.
On other disability improvement measures the treatment appeared to have an impact on a greater number of patients.
ReNeuron said it now intends to apply to the US and European regulatory authorities to begin a randomised, placebo-controlled, pivotal clinical trial in patients who are living with disability post-stroke.
A “delighted” Olav Hellebø, chief executive, said: “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.
Other trials progressing
First safety data is also expected early next year in the CTX phase I trial for critical limb ischaemia.
Also ongoing is the group's retinitis pigmentosa programe. This is examining treatment for a group of diseases which lead to blindness.
The firm recently kicked off a phase I/II clinical trial in the US and initial short-term safety and tolerability data from the phase I part in the first nine patients is expected in early 2017.
Pre-clinical data recently published on the human retinal progenitor cells (hRPCs) used on rodent models showed well for long-term efficacy and safety.
"The hRPCs were well tolerated, safe and preserved retinal structure and vision up to six months post-injection in the studies," the firm revealed.
"The cells also survived and integrated into both damaged and normal neural retina without adverse affects.
“These findings suggest that, as well as treating later stage retinal disease, the hRPCs can be deployed in less affected regions of the retina at earlier stages of retinal disease or even in normal regions of peripheral retina.”
ReNeuron chief executive Olav Hellebø said: "We are pleased to see the publication of further positive pre-clinical data from our hRPC retinal stem cells in a quality peer-reviewed scientific journal."
Funded for four programmes
A fourth programme is its exosome nanomedicine platform - a new nanomedicine targeting cancer, which is a potential new delivery system in gene therapy, where promising pre-clinical data had been shown.
Last year, ReNeuron carried out a huge £68.4mln funding to enable it to work simultaneously on all of its programmes.
"Success in any single one of these is worth a multiple of our current market cap," Hellebø highlighted at the time.