JCyte Licenses Ex-US Rights to Retinitis Pigmentosa Cell Therapy to Santen for $252 Million

Rare Daily Staff

JCyte entered into a licensing agreement with Japanese pharmaceutical Santen to develop and commercialize its first-in-class, experimental therapy jCell for the treatment of retinitis pigmentosa in regions including Europe, Asia, and Japan.

Retinitis pigmentosa is a rare, genetic condition that progressively destroys the rod and cone photoreceptors in the retina and is the leading cause of inheritable blindness. It often strikes people in their teens, with many patients rendered legally blind by middle age. Worldwide, an estimated 1.9 million people suffer from the disease, including approximately 100,000 people in the United States.

Under the terms of their, JCyte will receive $50 million in upfront cash, $12 million in a convertible note offering, and $190 million in clinical and sales milestones based on regulatory approval and initial sales in Europe, Asia and Japan. The total deal is valued at up to $252 million. JCyte is also entitled to receive tiered, double-digit royalty payments on net sales of jCell therapy once commercialized outside the United States.

“We intend to use the proceeds from this transaction to continue development of our lead investigational therapy jCell, to improve the lives of patients with retinitis pigmentosa, as well as other degenerative retinal diseases,” said Paul Bresge, CEO of JCyte.

jCell is a minimally-invasive, intravitreal injection, which can be performed in an ophthalmologist’s office with topical anesthetic. The entire procedure takes less than 30 minutes. The principal mechanism of action is the release of neurotrophic factors that may rescue diseased retinal cells. jCell therapy aims to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and potentially reactivated. jCell has been developed with support from the California Institute for Regenerative Medicine, which has funded preclinical development and ongoing clinical studies. JCyte has completed an evaluable phase 2b clinical trial for the treatment of retinitis pigmentosa with a crossover portion of the trial continuing.

The U.S. Food and Drug Administration has granted JCyte Regenerative Medicine Advanced Therapy designation based on early clinical data, making jCell potentially eligible for BLA priority review. In addition to RMAT, jCell has received Orphan Drug designation from the FDA and the European Medicines Agency.

Photo: Paul Bresge, CEO of JCyte