Company is poised to begin phase IIb efficacy trial in 2017
California-based regenerative medicine company jCyte has completed enrollment in a phase I/IIa trial to study the safety of its stem cell therapy candidate for retinitis pigmentosa (RP). The trial included 28 patients with advanced RP, eight of whom have completed the one-year study. Early safety results have been promising.
“We have successfully completed four DSMB (Data Safety Monitoring Board) reviews,” said jCyte co-founder Henry Klassen, MD, PhD. “So far, trial participants have had no significant side effects, with good tolerance of the injected cells. We are quite gratified by the results.”
The company’s investigational therapy, called jCell, uses injected retinal progenitor cells, which are intended to rescue dying retinal cells (rods and cones) and possibly regenerate new ones. The non-surgical treatment requires a single intravitreal injection, which can be performed in an ophthalmologist’s office under local anesthesia.
Retinitis pigmentosa is an incurable eye disease that destroys retinal cells and ultimately leads to blindness. It is a genetic condition that generally strikes people in their teens. Many patients are blind by the time they are 40. Worldwide, almost 1.5 million people suffer from RP, making it the leading cause of inherited blindness. Currently, there are no effective treatments.
The ongoing trial is being conducted at the Gavin Herbert Eye Institute at the University of California, Irvine and Retina Vitreous Associates in Los Angeles and has received significant support from the California Institute for Regenerative Medicine (CIRM).
As the safety trial winds up, jCyte has begun planning a phase IIb trial, which they hope to begin in 2017.
“I look forward to the next stage of development towards commercialization,” says jCyte CEO Paul Bresge. “We never lose sight of our singular goal: to ultimately deliver this much-needed therapy to patients.”
Bresge encourages RP patients who wish to participate in future trials to visit http://www.jcyte.com.