Things are slotting nicely into place for ReNeuron (LON:RENE), the developer of novel stem cell therapies targeting areas of unmet or poorly met medical need.
Earlier this week the company announced the appointment of Olav Hellebø, a highly experienced pharmaceutical executive, as its new chief executive officer (CEO), to spearhead the group’s next phase of development.
Happily, previous CEO Michael Hunt is remaining on board as chief financial officer, thus ensuring his skill and expertise remain on tap.
Hellebø has a significant amount of industry experience, including senior positions within Clavis Pharma, where he was the CEO, and UCB Pharma, where he was head of global biologics; he also had stints at Novartis and Schering Plough.
“His appointment strongly complements existing management,” City firm Cenkos noted.
With just over £20mln in cash in its coffers at the end of March following its well-supported fund raising last year, the focus is now very much on its development programmes, rather than proceeding with half an eye on the need to raise more cash at some indeterminate point in the future.
The company has three candidates undergoing clinical trials: ReN001, for stroke disability; ReN003, for retinitis pigmentosa (an inherited blindness causing disease that leads to diminishing tunnel vision; and ReN009, for critical limb ischaemia (a lack of blood flow that endangers all or part of the leg).
The nature of drug development means there are no sure things, but thus far the signs are looking good for all three.
With ReN001, clearance has been given to proceed into phase II testing, where the company will be hoping for a repeat of the sort of improvements many patients saw in the phase I trial.
The company said four out of 11 disabled stroke patients showed a one grade improvement on the modified Rankin scale, a consistent approach to scoring patient recovery, after 12 months on a dose escalation study.
“The 4/11 rate is somewhat indicative of what we’re looking to achieve in the phase II stage as well. So, that is the sort of response rate that would certainly get us to where we need to be in the phase II protocol that we’ve recently commenced,” Hunt told investors earlier this year.
“We draw great encouragement from these data. Certainly the treatment appears to be safe, within the context of this patient group, and we’ve seen some evidence of sustained neurological improvement in these patients on the measures that were taken,” he added.
ReN009 is from the same development platform as ReN001, and is designed to treat critical limb ischaemia (CLI), a condition that is common in diabetics that can lead to gangrene and amputation.
The market for ReN009 in the US alone has been independently estimated at between US$1bn and US$2bn.
The phase I trial for ReN009 will be a nine patient dose escalation study, and the first patient has started receiving doses.
Hunt said ReNeuron is hopeful that the company can run through this study quite quickly.
ReN003, meanwhile, is based around a different cell type to ReN001 and ReN009: human retinal progenitor cells.
The market size is smaller than for CLI, but is not negligible, having been estimated at between US$200mln and US$400mln in the US alone.
The drug has already received orphan drug designation in the US and the European Union, which is the regulator’s way of saying “no one else seems inclined to address this medical condition, so we had better fast track the drug’s passage through the regulatory labyrinth”.
A phase I/II clinical trial filing is anticipated early next year in the US.
It is not just a busy time ahead for ReNeuron, either; Hunt speaks of “the demographic time bomb” in the developed world, and a of a growing realisation that regenerative medicine approaches have the potential to really make an impact in a world where people live long enough to develop more ailments.
“Cell therapy approaches are moving out of early stage development, into phase II, into phase III in a number of cases,” Hunt said at a briefing for investment analysts.
Looking further down the road, the company is very excited about its exosome platform; thanks to the wonders of the Internet I can inform you that exosomes are found in all body fluids, and represent a new way of thinking about cell signalling.
You don’t necessarily need to understand the science; the key point is that the platform exploits existing neural stem cell technology to broaden the therapeutic pipeline beyond cell-based programmes.
Multiple new patent applications have been filed by ReNeuron, which means the pipeline should be flowing for many years to come.
Year to date the shares are up almost 30%, with the stock putting on a spurt in the last month, helped by news that star stock picker Neil Woodford has upped his stake to 18% through his Woodford Investment Management vehicle.
Meanwhile, broker Cenkos Securities believes the company is at a pivotal point in its history.
“We reiterate our BUY rating and see an important number of value inflection points between now and the end of 2015, when six-month follow-up clinical data from the Phase II stroke trial for ReN001 should become available. ReNeuron is about to enter the most exciting phase in the company's history,” the broker asserts.