Significant Visual Improvement Reported in Patients with Retinitis Pigmentosa in a cell therapy clinical trial

(c) ReNeuron

ReNeuron Group plc, a UK-based company focused on development of cell-based therapeutics, has announced encouraging results from an early stage trial of its cell therapy for retinitis pigmentosa (RP), which, if approved, could be a competitor to Spark/Novartis’s Luxturna (voretigene neparvovec). Unlike the already-approved gene therapy Luxturna, ReNeuron’s cell-based therapy would not be limited to patients with a disease caused by a certain mutation.

The Company reports that all three of the first cohort of subjects in the Phase II part of the trial have reported a significant improvement in vision, on average equivalent to reading an additional three lines of 5 letters on the ETDRS eye chart, the standardized eye chart used to measure visual acuity in clinical trials.

The Phase II part of the Phase I/II trial, which uses a cryopreserved, commercially ready hRPC formulation, enrolled subjects with some retinal functionality, in contrast to the very poor vision and lack of potential for improvement of the subjects in the trial’s Phase I component.

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In the latest observations of the first Phase II cohort, at two months follow-up for one subject, and at 18 days for the other two, all three subjects have reported improved vision, with standardized eye chart testing showing objective improvement in visual acuity compared with their pre-treatment baseline vision and compared with the patients’ untreated control eye.

Obviously, these data is early and we will have to wait and watch for late results to evaluate the visual acuity in these subjects in the long term, to assess durability of effect and efficacy over a longer period of time and also in a larger number of patients.

Summary of the preliminary efficacy data from the three Phase II subjects treated to date

(visual acuity measured using the standardized ETDRS chart) 

(from ReNeuron's release)

For the first subject in the cohort, visual acuity improved in the treated eye from 9 letters at baseline to 29 letters at two months follow-up; for the second subject, visual acuity improved from 9 letters at baseline to 24 letters at 18 days follow-up; for the third subject, visual acuity improved from 32 letters at baseline to 46 letters at 18 days follow-up. The untreated control eyes did not show significant improvement (mean change from baseline -1 letter, range -5 to + 5 letters). The mean change from baseline in visual acuity for these first three subjects early on in duration of follow up is thus plus 16 letters in the study eye, compared with a mean change from baseline of minus 1 letter in the untreated control eyes. All three subjects have noted a subjective improvement in vision in their treated eye.

The Phase I/II study, which is being undertaken at two clinical sites in the US – Massachusetts Eye and Ear in Boston and Retinal Research Institute in Phoenix – is an open-label study to evaluate the safety, tolerability and preliminary efficacy of the hRPC stem cell therapy candidate in patients with advanced 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.

Dosing in the Phase I part of the study was completed last year in twelve subjects with extensive retinal damage and very limited remaining vision to establish the safety of the treatment. The safety and visual stability data from this part of the study resulted in the Data Safety Monitoring Board (DSMB) approving the transition to a commercially ready hRPC drug product formulation to be administered to patients with less retinal damage and consequently better visual potential.

Dosing of the second cohort of three Phase II subjects is expected to commence in March 2019 following DSMB review of the clinical data from the first Phase II cohort. Based on the positive early data thus far from the Phase I/II study, the Company intends to seek regulatory advice regarding the optimal clinical development path for its hRPC candidate in RP.

The Company’s RP clinical programme benefits from Orphan Drug Designation in both Europe and the US, as well as Fast Track designation from the FDA. The Company expects to report further short term read-outs in mid-2019 from the ongoing Phase I/II study.

Disclosure: Dr. Pravin Dugel, MD, Managing Partner, Retinal Consultants of Arizona, Phoenix, Arizona, and Clinical Professor, Roski Eye Institute, USC Keck School of Medicine, Los Angeles, California, and a member of our Board of Directors, is also an Investigator on this trial.

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