Guildford, UK, 29 August 2013: ReNeuron is pleased to announce that its ReN003 retinal stem cell therapy candidate for retinitis pigmentosa has achieved a significant regulatory milestone.
The therapy has been granted Orphan Drug Designation in both Europe and the US by the European Commission and the Food and Drug Administration, respectively.
Orphan Drug Designation is typically granted to treatments that provide significant benefit to patients with rare diseases that are life-threatening or chronically debilitating. Treatments with this designation benefit from significant commercial and regulatory advantages such as market exclusivity for 10 years from approval in the disease concerned, against other treatments offering no greater therapeutic advantage.
ReNeuron is using its proprietary human retinal progenitor cells as the basis of its ReN003 therapeutic candidate targeting retinitis pigmentosa, a group of hereditary diseases of the eye that lead to progressive loss of sight due to cells in the retina becoming damaged and eventually dying. Retinitis pigmentosa affects approximately 4 in 10,000 people in the EU and US.
The late pre-clinical development of the ReN003 programme also benefits from a recently awarded £0.8 million Biomedical Catalyst grant from the UK Technology Strategy Board. The Company is targeting an initial Phase I/II clinical trial with ReN003 in the UK and the US, in retinitis pigmentosa patients, with clinical trial applications expected to be filed in the middle part of 2014.
Michael Hunt, Chief Executive Officer of ReNeuron, said:
“We are delighted to have received Orphan Drug Designation for our ReN003 stem cell therapy candidate for retinitis pigmentosa from both the European Commission in Europe and the FDA in the US. Orphan status diseases are an increasing area of therapeutic and commercial focus by the mainstream pharmaceutical industry due to the distinct regulatory and market exclusivity advantages that Orphan Drug Designation confers. This fact bodes well for the commercial potential of our ReN003 therapy.”
- ReNeuron +44 (0) 1483 302560
Michael Hunt, Chief Executive Officer
Dr John Sinden, Chief Scientific Officer
- Buchanan +44 (0) 20 7466 5000
Mark Court, Fiona Henson, Sophie Cowles
Cenkos Securities +44 (0) 20 7397 8900
Stephen Keys, Adrian Hargrave (NOMAD and Broker)
Andy Roberts (Sales)
ReNeuron is a leading, clinical-stage stem cell business. Its primary objective is the development of novel stem cell therapies targeting areas of significant unmet or poorly met medical need.
ReNeuron has used its unique stem cell technologies to develop cell-based therapies for significant disease conditions where the cells can be readily administered “off-the-shelf” to any eligible patient without the need for additional immunosuppressive drug treatments. ReNeuron’s lead candidate is its ReN001 stem cell therapy for the treatment of patients left disabled by the effects of a stroke. This therapy is currently in clinical development. The Company is also developing stem cell therapies for other conditions such as critical limb ischaemia, a serious and common side-effect of diabetes, and blindness-causing diseases of the retina such as retinitis pigmentosa.
ReNeuron has also developed a range of stem cell lines for non-therapeutic applications – its ReNcell® products for use in academic and commercial research. The Company’s ReNcell®CX and ReNcell®VM neural cell lines are marketed worldwide under license by USA-based Merck Millipore.
ReNeuron’s shares are traded on the London AIM market under the symbol RENE.L. Further information on ReNeuron and its products can be found at www.reneuron.com.
This announcement contains forward-looking statements with respect to the financial condition, results of operations and business achievements/performance of ReNeuron and certain of the plans and objectives of management of ReNeuron with respect thereto. These statements may generally, but not always, be identified by the use of words such as “should”, “expects”, “estimates”, “believes” or similar expressions. This announcement also contains forward-looking statements attributed to certain third parties relating to their estimates regarding the growth of markets and demand for products. By their nature, forward-looking statements involve risk and uncertainty because they reflect ReNeuron’s current expectations and assumptions as to future events and circumstances that may not prove accurate. A number of factors could cause ReNeuron’s actual financial condition, results of operations and business achievements/performance to differ materially from the estimates made or implied in such forward-looking statements and, accordingly, reliance should not be placed on such statements.