BioWorld International Correspondent
LONDON - The UK's only stem cell therapy company, ReNeuron plc, bounced back from problems that forced it to delist from the Alternative Investment Market and go private last year, releasing new animal data and announcing a £10 million (US$18 million) funding round.
Many of ReNeuron's difficulties arose from long delays in its human neural stem cell programs that were caused by genetic instability of the cell lines. Having resolved that, the company presented preclinical efficacy data for ReN001 in a rat model of stroke at the American Neurological Association annual meeting in Toronto last week. The cells, derived from the brain cortex, improved sensory motor abilities.
"We believe we could be first into the clinic in a major indication using neural stem cells," Michael Hunt, chief operating officer, told BioWorld International. "We have moved on a lot in the past year. We have solved the problem of genetic stability and have a fully controllable system for generating cell lines that are not tumorigenic, and have stable, normal genomes, so we think we are in a good position."
ReNeuron also has positive data in animal models of Huntington's disease and dementia that will be presented later in the year.
GMP scale-up has begun, and Hunt said Guildford, UK-based ReNeuron aims to have approval for a clinical trial by the end of 2005. That will treat people who have long-term disabilities following stroke.
"We have more preclinical safety work to do, but we have got a broad idea of what is required, based on best advice," he said.
Hunt said the plan is to approach both UK and U.S. regulators "to see what sort of ride we get. There is little precedent, and until we present it is not clear what the requirements will be."
Delays with the human neural stem cell lines and the failure of two small-molecule drug programs led to ReNeuron being taken private by its venture capital backers, the Merlin Consortium, in April 2003. Hunt said Merlin has pledged to put money into the new funding round, but would not disclose the amount.
ReNeuron raised £19.5 million when it floated in November 2000. Merlin invested £5 million venture capital in the company, then paid £3.6 million to take it private.