ReNeuron files application to commence Phase I/II clinical trial with its stem cell therapy candidate for retinitis pigmentosa
Posted: 13 April 2015 |
ReNeuron has announced that it has filed an Investigational New Drug (IND) application with the United States Food and Drug Administration (FDA) to commence a Phase I/II clinical trial with its human Retinal Progenitor Cell (hRPC) therapy candidate for retinitis pigmentosa (RP).
RP is 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.
Pre-clinical studies carried out in disease models by the Company’s academic collaborators have demonstrated that, when transplanted into the retina, ReNeuron’s retinal progenitor cell technology has the potential to preserve existing photoreceptors, potentially reducing or halting further deterioration of vision. In addition, the progenitor cells have been shown to mature into functional photoreceptors that engraft into the photoreceptor layer, bringing the possibility of restored vision.
The proposed Phase I/II clinical trial will be conducted at Massachusetts Eye and Ear, Boston. The trial design is an open-label, dose escalation study to evaluate the safety, tolerability and preliminary efficacy of the hRPC stem cell therapy candidate in up to 15 patients with advanced RP. The method of administration of the hRPCs will be a single sub-retinal injection. The primary endpoint of the study is safety, with patients being followed up for 12 months post-treatment with monitoring including measurements of visual acuity.
ReNeuron expects to be able to commence the clinical trial later in 2015
ReNeuron expects to be able to commence the clinical trial in the second half of this year. ReNeuron’s cell therapy candidate for RP has been granted Orphan Drug Designation in both Europe and the US. Orphan Drug Designation is typically granted to drug programmes that potentially provide significant benefit to patients with rare diseases that are life-threatening or chronically debilitating.
Dr Eric Pierce, Director, Retinal Degenerations Service at Massachusetts Eye and Ear, and Principal Investigator for the clinical trial, commented: “We are pleased to be working with ReNeuron on this important Phase I/II clinical trial. The human Retinal Progenitor Cells being tested in the study are promising since they can make photoreceptors. The implanted cells may not only prevent degeneration of patients’ vision but may possibly restore some vision by replacing degenerated photoreceptor cells.”
Olav Hellebø, Chief Executive Officer of ReNeuron, commented: “We are delighted to have progressed our hRPC stem cell therapy candidate for RP to the point of submitting an application to conduct a clinical trial in the US. Once approved and commenced, the proposed Phase I/II clinical trial in RP patients will be the third clinical study ongoing with ReNeuron’s cell therapy candidates, alongside our Phase II clinical trial in stroke and our Phase I clinical trial in critical limb ischaemia. The proposed RP clinical trial initiates the Company’s clinical activities in the US, a very significant milestone in the development of ReNeuron as a global leader in the stem cell therapy field.”
For more information about ReNeuron, please visit www.reneuron.com.