JCyte has signed an ex-US licensing deal with Japanese ophthalmic company Santen Pharmaceutical worth up to $252m to develop and commercialise its investigational jCell therapy for a rare genetic eye disorder called retinitis pigmentosa.

According to jCyte, treatment with jCell is a minimally-invasive intravitreal injection, which can be carried out in an ophthalmologist’s office with topical anesthetic. The entire procedure is said to take less than 30 minutes, as per the biotech company, which is engaged in developing therapies for rare degenerative retinal diseases.

JCell’s main mechanism of action is the release of neurotrophic factors that can rescue diseased retinal cells. The investigational therapy has been designed to preserve vision by intervening in the disease at a time when host photoreceptors can be protected and reactivated potentially.

JCyte developed jCell with support from the California Institute for Regenerative Medicine (CIRM), which has financially helped in previous preclinical development and ongoing clinical studies.

The biotech company has completed the evaluable part of a phase 2b clinical trial of jCell in the US for the treatment of retinitis pigmentosa. The company said that the crossover portion of the trial is going on with the investigational therapy given to more than 100 patients.

JCyte secured the Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for the investigational therapy. The RMAT status has been granted based on early clinical data, which potentially makes the retinitis pigmentosa therapy candidate eligible for biologics license application (BLA) priority review.

Apart from RMAT, jCell has been granted orphan drug designation from the FDA and also the European Medicines Agency (EMA).

As per the terms of the licensing deal, jCyte will be paid $50m upfront by Santen Pharmaceutical along with $12m in a convertible note offering. The biotech company will also stand to receive $190m based on achieving clinical and sales milestones on regulatory approval and initial sales across Europe, Asia, and Japan.

jCyte will also be entitled to be paid tiered, double-digit royalty payments from the Japanese firm on net sales of jCell therapy once commercialised outside the US.

JCyte CEO Paul Bresge said: “By leveraging Santen’s large, existing global commercial and medical infrastructure in ophthalmology, as well as its commitment to commercializing cell and gene therapies, we help ensure that more patients with retinitis pigmentosa who live outside the U.S. will have access to this technology.

“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.”