NEW YORK – Coave Therapeutics on Wednesday released preliminary results from a Phase I/II trial of its gene therapy for retinitis pigmentosa, which it says support advancing the program to a registration trial.
The French biotechnology company shared 12-month data from an ongoing Phase I/II trial of CTx-PDE6b, a gene therapy to treat patients with retinitis pigmentosa, a rare, inherited, and degenerative eye disease that can be caused by mutations in various genes. Coave's gene therapy, which is designed for patients with certain mutations in the PDE6b gene, aims to deliver a functional copy of PDE6b into the subretinal space and stabilize or halt retinal degeneration through production of functional PDE6b proteins in photoreceptive rod cells.
In the Phase I/II trial, 17 patients have received CTx-PDE6b at two ascending doses in one eye, while the other eye served as an untreated control. Six patients with less advanced disease and who received the higher dose seemed to benefit from the gene therapy and performed well in multiple visual function tests.
"These initial findings provide strong support for expanding this Phase I/II study to include patients with less advanced disease while we begin our preparations for a registrational trial with CTx-PDE6b," Coave CEO Rodolphe Clerval said in a statement.
The 17 patients who have received CTx-PDE6b to date in the Phase I/II trial have been adults who tend to have more advanced disease. The firm has received regulatory permission to enroll six more patients between 13 and 25 years old with earlier-stage disease into this study.
Coave leads global development of CTx-PDE6b and has commercial rights in the US, Japan, South Korea, China, and other regions outside Europe. Coave's codevelopment partner, Théa Open Innovation, another French firm, is developing the gene therapy in Europe and certain other countries.