US-based clinical-stage ophthalmology company Acucela, a subsidiary of Kubota Pharmaceutical, has begun enrolment for its Phase IIa study of emixustat hydrochloride (emixustat) to treat Stargardt disease.

Vitamin A is recycled in the eye through a process known as the visual cycle.

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Emixustat controls the visual cycle by preventing a key enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65).

A slowed visual cycle limits availability of vitamin A derivatives to formation of toxic vitamin A byproducts such as A2E and related compounds.

"This is an important therapeutic development for patients with Stargardt Disease."

The Phase IIa study will be conducted as a multicentre, randomised, masked trial intended to determine pharmacodynamics and the safety and tolerability of emixustat in subjects diagnosed with macular atrophy secondary to Stargardt disease.

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The study will enrol about 30 patients across four to six clinical sites in the US who will be randomly assigned to one of three treatment arms in a 1:1:1 ratio.

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The subjects will receive an oral administration of dosages of emixustat in 2.5mg, 5mg, and 10mg on a once-daily basis in the evening for one month.

University Hospital in Basel Switzerland and Stargardt Disease Natural History Study (PROGSTAR) study director Dr Hendrik Scholl said: “This is an important therapeutic development for patients with Stargardt Disease.

“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.”