Research progressing for patients with Stargardt Disease.

Acucela Initiates Phase 2a Study of Emixustat Hydrochloride Addressing Patients with Stargardt Disease

Acucela Inc. (“Acucela”), a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596) committed to translating innovation into a diverse portfolio of drugs and devices to preserve and restore vision for millions of people worldwide, announced today that on January 25, 2017, the first patient has enrolled in a study to evaluate Acucela’s leading drug candidate, emixustat hydrochloride (“emixustat”) 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 one month.

“This is an important therapeutic development for patients with Stargardt Disease,” said 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). “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, Dr. 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.”…

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Source: Business Wire