Acucela announced that on January 25, 2017, the first patient was enrolled in a study to evaluate Acucela’s leading drug candidate, emixustat hydrochloride, in subjects with macular atrophy secondary to Stargardt disease.
This multicenter, randomized, masked phase 2a study is designed to evaluate the pharmacodynamics, safety, and tolerability of emixustat in subjects with macular atrophy secondary to Stargardt disease. Approximately 30 subjects will be enrolled at 4 to 6 clinical sites in the United States. Subjects will be randomly assigned to one of three treatment arms in a 1:1:1 ratio. Treatment arms include: emixustat 2.5 mg, emixustat 5 mg, and emixustat 10 mg. Subjects will orally take study drug once daily in the evening for 1 month.
“This is an important therapeutic development for patients with Stargardt Disease,” Dr. Hendrik Scholl from the University Hospital in Basel Switzerland and Study Director of the Stargardt Disease Natural History Study (PROGSTAR) sponsored by the Foundation Fighting Blindness (FFB), said in a company news release. “Emixustat addresses a well-understood mechanism that leads to toxic accumulation of material underneath the retina linked to visual loss in Stargardt disease and therefore is a promising compound for this debilitating disease.”
Additionally, Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela stated that “Stargardt disease represents a serious sight threatening unmet medical need, and we are pleased to start our phase 2a study of emixustat in subjects with Stargardt disease.”
“We applaud Acucela’s evaluation of emixustat for people with Stargardt disease, a devastating form of inherited macular degeneration, which causes significant central vision loss in young people, and for which there are currently no therapies,” said Patricia Zilliox, PhD, chief drug development officer, FFB-CRI. “Preserving vision by slowing degeneration would be a major benefit for those affected.”
The FDA granted orphan drug designation to emixustat for the treatment of Stargardt disease.