Date: 2015-05-05
Type of information: Clinical trial authorization
phase: 1-2
Announcement: clinical trial authorization
Company: Reneuron (UK)
Product: human Retinal Progenitor Cell (hRPC) therapy
Action
mechanism: cell therapy
Disease: retinitis pigmentosa
Therapeutic area: Genetic diseases - Ophtalmological diseases - Rare diseases
Country: USA
Trial
details: This is a first-in-human, dose escalation study in which participants with retinitis pigmentosa will receive a single subretinal injection of hRPC cells in one eye to evaluate safety and tolerability. Participants will be followed for one year to evaluate the safety and tolerability of hRPC Additional testing will seek to establish any preliminary efficacy from hRPC. (NCT02464436)
Latest
news: * On May 5, 2015, ReNeuron announced that it has received regulatory approval from the FDA to commence a Phase I/II clinical trial in the US with its human Retinal Progenitor Cell (hRPC) therapy candidate for retinitis pigmentosa (RP). The Company announced on 13 April 2015 that it had filed an Investigational New Drug (IND) application with the FDA to commence the Phase I/II clinical trial. The Company has since received notification from the FDA that the review of the application is complete and that ReNeuron is free to begin clinical studies. ReNeuron’s cell therapy candidate for RP has already been granted Orphan Drug Designation in both Europe and the US by the European Commission and the FDA, respectively. * On April 13, 2015, ReNeuron announced that it has filed an Investigational New Drug (IND) application with the FDA to commence a Phase I/II clinical trial with its human Retinal Progenitor Cell (hRPC) therapy candidate for retinitis pigmentosa. 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.
The 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. Preparations for the study have commenced and ReNeuron expects the study to begin in the second half of this year.
