For investors seeking exposure to the burgeoning field of stem cell technology, Alternative Investment Market-quoted ReNeuron (LON:RENE) looks an intriguing proposition.
The firm has the distinction of being the first, and so far only, company to have received regulatory approval for a stem cell-based clinical trial in the UK.
The development of stem cells in order to treat diseases is significant because of their ability to both renew themselves and create new cells of other kinds of tissues. So, while bone marrow transplants are already used to treat leukaemia, other stem cell therapies may one day be used to treat a wide range of diseases, including cancer and multiple sclerosis.
Despite this, stem cells have proved a highly controversial issue, mainly in relation to stem cells garnered from embryos. However, ReNeuron itself derives stem cell products from non-embryonic human tissue sources.
The company’s main current area of activity is its stem cell therapy for disabled stroke patients, which is called ‘ReN001’. Stroke is the third-largest cause of death and the single largest cause of adult disability in the developed world.
But ReNeuron is also developing stem cell therapies for a number of other conditions, as well as growing a range of stem cell lines for non-therapeutic applications.
The ReN001 therapy is based around ReNeuron’s lead neural stem cell line, designated CTX by virtue of its origin from the cortex region of the brain. The company’s other cell therapy programmes utilise either the CTX cortical cell line or separate cell lines generated from the region of the brain or body appropriate to the disease in question. These programmes are currently at the research or pre-clinical stages of development, targeting neurological and other conditions including sub-acute stroke, Alzheimer’s disease, peripheral or lower limb ischaemia and diseases of the retina.
In July, ReNeuron signed a new contract with Angel Biotechnology for manufacturing services in support of its development programme for the ReN001 stem cell therapy. The programme is expected to continue though to mid-2012, and includes the manufacture of doses for ReNeuron’s ongoing PISCES clinical trial with ReN001 in stroke.
The new contract is structured so that both firms have much more flexibility in terms of work scheduling, giving ReNeuron the ability to match the needs and opportunities of its development programmes in real time.
PISCES (Pilot Investigation of Stem Cells in Stroke) is the world’s first fully regulated clinical trial of a neural stem cell therapy for disabled stroke patients.
The PISCES trial is being conducted in Scotland at the Institute of Neurological Sciences, Southern General Hospital in Glasgow. This Phase I single administration does escalation safety study involves the ReN001 stem cell therapy being given to a total of 12 stroke patients who have been left disabled by an ischaemic stroke, the most common form of the condition.
Having begun the trial last autumn, ReNeuron has since reported that the therapy appears to be well tolerated by the first-dose cohort of three patients. All three patients were safely discharged from hospital two days after what the firm calls a “straightforward neurosurgical procedure” was used to administer the ReN001 cells.
To date, the first patient has been assessed at six months post-treatment, the second patient at three months and the third patient at one month. All three patients remain well, and although the PISCES trial is still at an early stage ReNeuron is “greatly encouraged” by the progress of the patients to date.
The independent Data Safety Monitoring Board for the PISCES trial is expected to review data from the first dose cohort by the end of this month. Assuming it gives approval for the trial to move on to a higher-dose cohort, ReNeuron expects this second cohort to have been treated by the end of this year.
The remaining dose cohorts are expected to be treated over the course of next year, by which point ReNeuron intends to have discussed and agreed a subsequent clinical development trial for ReN001 with relevant authorities in the UK and abroad.
“Ultimately, our plan is that we expect to license this, and other programmes, to partners,” says Michael Hunt, ReNeuron’s chief executive officer. “No-one has done this yet, so it is very difficult to tell what the regulator will allow. It could be as quick as three-to-five years or it could be longer than that.”
The firm’s other stem cell therapies include ReN009, targeted at peripheral arterial disease (PAD), and ReN003, its therapy programme focused on diseases of the retina.
PAD is a chronic, debilitating disease that progressively restricts blood flow in the limbs, causing cramping and chronic pain, and in rare cases it can lead to the loss of a limb. During the past year, ReNeuron’s collaborators on its ReN009 programme at Bristol Heart Institute have presented positive pre-clinical efficacy data.
Meanwhile, the firm has received guidance from regulatory authorities in both the UK and the US regarding the ReN009 programme and, based on this, it is progressing the remaining pre-clinical studies and cGMP cell manufacturing campaign necessary to complete the regulatory data package to be submitted for clinical trial approvals.
ReNeuron’s is continuing to collaborate with the Schepens Eye Research Institute at Havard Medical School – which recently announced it is to team up with the Massachusetts Eye and Ear Infirmary in Boston to create the world’s largest ophthalmology research centre – on its ReN003 programme. The two parties aim to take the programme through late pre-clinical development and into an initial clinical trial in patients suffering from retinitis pigmentosa, which is a blindness-causing disease brought about by degeneration of the photoreceptor cells in the retina.
During its financial year to the end of March 2011 ReNeuron’s revenues were £29,000 (2010: £31,000), representing royalty income from the group’s non-therapeutic licensing activities.
The loss for the year increased to £6.1 million (2010: £3.6 million before exception items), reflecting initial clinical costs in ReNeuron’s stroke programme as well as increased late pre-clinical activity on other core therapeutic programmes and non-recurring legal and professional fees.
In terms of its balance sheet, last December ReNeuron successfully raised £10 million via an oversubscribed share placing, and cash and cash equivalents at the end of March amounted to £9.7 million (March 31 2010: £5.5 million). The firm believes these funds should be sufficient to support current operations into 2012. “It’s enough to get us through to next year,” says Hunt. “But it is clear we will need to raise further funds.”
For now, ReNeuron is continuing to focus on its most-advanced therapy, ReN001, as well as working with its academic partners on ReN003 and ReN009. “We’re a company of limited resources. We’ve got 20 employees and constrained finances, so we’ve got to be mindful of that and not try to do too much,” says Hunt.
ReNeuron will be holding its annual general meeting in London on September 15 2011.