ReNeuron Group has announced that positive efficacy data were presented during the weekend at the American Academy of Ophthalmology Annual Meeting (AAO) in San Francisco from the company’s phase 1/2a clinical trial of its hRPC stem cell therapy in retinitis pigmentosa (RP). Topline data from this presentation were announced by the company on October 2, 2019.
A copy of the presentation, by Pravin Dugel, MD, an investigator in the ongoing RP study, is available on the company’s website via the following link:
In the presentation given on Saturday, October 12, 2019, Dr. Dugel discussed the strong individual improvements in vision seen in the clinical trial and welcomed hRPC as a promising new potential therapy for patients with RP.
“We’re excited by the progress of ReNeuron’s hRPC therapy. From the Foundation’s perspective, any gain in vision, or even stabilization, is a major step forward for patients with RP as currently it is a condition where progressive loss of vision leads to blindness,” Benjamin R. Yerxa, PhD, Chief Executive Officer of the US-based non-profit organization Foundation Fighting Blindness, said in a company news release.
Dr. Dugel’s presentation reiterated that follow-up visual acuity data from the patients treated in the phase 2a segment of the study continue to show the hRPC therapy’s ability to deliver clinically meaningful signals of efficacy.
From a safety perspective, according to Dr. Dugel, the clinical trial data continue to show a good overall safety profile for the hRPC therapy, with no immune or cell-related adverse events reported. The isolated episodes of surgically related adverse events are consistent with those expected for sub-retinal injection procedures, some of which may be mitigated in the future with technique refinements and injection site selection.
The ongoing phase 1/2a clinical trial is an open-label study to evaluate the safety, tolerability and preliminary efficacy of ReNeuron’s hRPC stem cell therapy candidate in patients with advanced RP. To date, 22 patients have been treated in the study, consisting of 12 patients in the phase 1 segment of the study and 10 patients in the phase 2a segment of the study. Eight out of the 10 phase 2a patients treated have reached at least the 1 month follow up time point.
The company will continue to generate further longer-term follow up data from the ongoing phase 1/2a study. In parallel, the company will consult with its advisers and regulatory authorities in Europe and the U.S. in order to design and agree on the future clinical development program for its hRPC cell therapy candidate for the treatment of RP.
ReNeuron’s RP program has been granted Orphan Drug Designation in both Europe and the U.S., as well as Fast Track designation from the FDA.