Regenera Pharma has enrolled the first patient in a Phase lll trial of RPh201 in the US for the treatment of non-arteritic anterior ischemic optic neuropathy (NAION), a type of stroke of the optic nerve.
Supported by fast-track designation from the US Food and Drug Administration (FDA), the multicentre, double-blind, placebo-controlled trial aims to enrol 234 patients diagnosed with NAION at least one year prior to enrolment into the study.
During the trial, patients will be randomised and receive twice-weekly injections of RPh201 or placebo for a period of six months.
The trial’s primary endpoint is the proportion of patients with visual acuity improving by three lines or more from baseline to week 26.
Set to be conducted in 12 centres across the US, the trial is expected to provide final results in the last quarter of next year.
The Phase lll trial chair Dr Leonard Levin said: “Several thousand people have irreversible visual loss from NAION each year in the US, and the likelihood of improvement after the first six months is very low.
“This new study will help determine the potential for RPh201 to become the first drug shown to restore vision in these patients.”
RPh201 is a well-defined extract from gum mastic, which is purified, formulated and stabilised in a proprietary process developed by Regenera Pharma.
Furthermore, Regenera is conducting a Phase lla trial in Canada of RPh201 to treat patients with moderate Alzheimer-related dementia.
Regenera is expected to enrol around 45 to 81 patients for the multicentre, double-blind, placebo-controlled trial.
The enrolled patients will be randomised in a 2:1 ratio to receive twice-weekly subcutaneous injections of RPh201 or placebo over a six-month period.
The trial, which will primarily measure cognitive changes from baseline to six months, is currently dosing patients at five centres in Ontario and Quebec.
Readout of the trial is expected by the second quarter of next year.