ReNeuron Group has reported positive long-term efficacy data from the ongoing Phase I/II clinical trial of human retinal progenitor cell (hRPC) stem cell therapy in patients with retinitis pigmentosa (RP).
RP is a group of hereditary eye diseases that cause progressive loss of sight and blindness. hRPC therapy is intended to preserve existing photoreceptors to reduce or potentially stop further vision loss.
In October last year, interim efficacy data from the trial revealed sustained clinically relevant visual acuity improvements, compared to baseline, in a group of participants.
According to the latest long-term data, the meaningful clinical effect from the therapy candidate continued at all time points through to twelve months following treatment.
ReNeuron Group CEO Olav Hellebø said: “We remain greatly encouraged by the data from the Phase I/IIa clinical study of our hRPC cell therapy candidate in patients with RP.
“The longer-term follow-up data are particularly noteworthy, demonstrating that the therapy appears to maintain its beneficial effects out to at least one year post-treatment.”
The company has filed a protocol amendment with the US Food and Drug Administration (FDA) to expand the Phase I/IIa trial.
The aim is to enrol up to an additional of nine patients in the Phase IIa part of the study. New participants will be treated with a dose of two million hRPC cells, which will be compared to the one million cells dose used in the trial so far.
The updated protocol will enable a greater range of pre-treatment baseline visual acuity. Data from the expanded segment is expected to be reported later this year.
ReNeuron has also submitted an application to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) to open the trial to the Oxford Eye Hospital.