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EyeDNA Looking to Carve Out Regulatory Path for Retinitis Pigmentosa Gene Therapy

NEW YORK – EyeDNA Therapeutics on Tuesday said it will discuss the latest data on its investigational gene therapy, HORA-PDE6B, for PDE6b-mutated retinitis pigmentosa with regulators and define a regulatory path forward for the agent.

Retinitis pigmentosa is a rare, inherited, degenerative eye disease. It is a genetically heterogenous condition that can be caused by abnormalities in more than 80 genes. Approximately 4 percent of patients with retinitis pigmentosa have PDE6b genetic mutations, and HORA-PDE6B is designed to deliver a non-mutated PDE6b gene to target retinal cells to generate a functional protein.

At a medical meeting earlier this week, EyeDNA, a subsidiary of Paris-based Coave Therapeutics, reported positive 24-month follow-up data from a Phase I/II study of HORA-PDE6b involving 17 evaluable patients with retinitis pigmentosa caused by bi-allelic mutations in the PDE6b gene.

Researchers observed improvements in vision for patients who received the high and low dose of the gene therapy. In the low-dose group, in particular, five-year follow-up data showed that vision in patients' treated eyes stabilized, as measured by Best Corrected Visual Acuity scores. In comparison, in the untreaded eye, BCVA scores consistently declined. A full-field stimulation test of blue light assessing rod function suggested that light perception also improved in patients' treated eyes.

Patients tolerated both doses of HORA-PDE6b well. There were five eye-related serious adverse events in patients that were potentially due to the investigational treatment, and two of these were resolved. 

EyeDNA is also testing HORA-PDE6b in a cohort of up to six younger patients 13 to 17 years old, who have earlier stage disease. Three patients are enrolled so far in this cohort.

"The positive 24-month follow-up data presented confirm results from the previous interim analysis of the trial conducted at the 12-month follow-up point and support preparation for a registrational trial for HORA-PDE6b in PDE6b RP patients," the company said in a statement. "Further discussions with health authorities in the US and Europe are planned to define the optimal path to making HORA-PDE6b available to PDE6b RP patients."

EyeDNA is developing and commercializing HORA-PDE6b with Théa Open Innovation. EyeDNA is leading global development efforts and holds commercial rights to the product in the US, Japan, South Korea, China, and other territories outside Europe. In Europe and certain other countries, Coave and TOI are codeveloping the gene therapy under a license and development agreement, and TOI holds exclusive commercialization rights to HORA-PDE6b in these territories.