ReNeuron Files Application to Commence Phase I/II Clinical Trial in the US
News Apr 14, 2015
ReNeuron Group plc has announced that it has filed an Investigational New Drug (IND) application with the US 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.
ReNeuron has worked with world-leading collaborators and academic institutions in the retinal disease field to successfully take its retinitis pigmentosa programme through pre-clinical development. These include the Schepens Eye Research Institute/Massachusetts Eye and Ear (an affiliate of Harvard Medical School), UCL Institute of Ophthalmology, Moorfields Eye Hospital and the US-based Foundation Fighting Blindness, the world’s leading private source of research funding for inherited retinal diseases. The pre-clinical programme has also benefited from UK Government funding under a BioMedical Catalyst grant, awarded in 2013.
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, a world-renowned clinical centre for the treatment of retinal diseases. 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.
Subject to regulatory and local ethics approvals, 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 by the European Commission and the FDA, respectively.
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. In acknowledgment of the relatively small patient populations involved, commercial and other incentives are provided to developers of orphan drugs.
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. The work leading to this clinical trial began over 15 years ago in the laboratory of Michael Young PhD, de Gunzburg Scholar and Co Director of the Ocular Regenerative Medicine Institute at the Schepens Eye Research Institute/Massachusetts Eye and Ear and Harvard Medical School.”
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.”