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Acucela Announces Publication Assessing the Role of Emixustat Hydrochloride in Retinal Degeneration Treatment

SEATTLE–()–Acucela Inc. (“Acucela”), a clinical-stage ophthalmology company and wholly-owned subsidiary of Kubota Pharmaceutical Holdings Co., Ltd. (Tokyo 4596), today announced the publication of an article concerning the company’s investigational drug candidate, emixustat hydrochloride (emixustat), in the scientific journal Drug Discovery Today.

The published article, “Pharmacotherapy for Metabolic and Cellular Stress in Degenerative Retinal Diseases,” can be accessed at: For additional information about Drug Discovery Today, please visit:

Authors of the paper explored the cellular and metabolic stressors associated with degenerative retinal diseases and the potential role of visual cycle modulation with emixustat in the treatment of these diseases. The accumulation of toxic visual cycle byproducts and retinal hypoxia are stressors associated with retinal diseases such Stargardt disease (STGD) and diabetic retinopathy (DR), respectively. Emixustat’s mechanisms of action and preclinical data supporting the role of emixustat in the treatment of these diseases is detailed.

Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela Inc., said, “I am pleased that this article on the role of emixustat in treating retinal degenerations has been published in the journal Drug Discovery Today. This publication supports the rationale for investigating emixustat in the treatment of Stargardt disease and diabetic retinopathy. Advancing our clinical programs is what we are focusing on and we will continue to actively seek for partnership opportunities.”

Emixustat has been formulated for oral administration and is currently being investigated in a multi-center Phase 3 clinical trial to determine if emixustat reduces the rate of macular atrophy progression, in comparison to placebo, in subjects with Stargardt disease. Secondary objectives include assessing changes in visual function parameters such as BCVA (best-corrected visual acuity) letter score and reading speed ( identifier: NCT03772665).

About Emixustat Hydrochloride

Emixustat modulates the visual cycle by inhibiting a critical enzyme of this pathway, retinal pigment epithelium protein 65 (RPE65). The visual cycle is the process by which vitamin A is recycled in the eye; vitamin A is crucial to the visual process. Slowing the visual cycle reduces the availability of vitamin A derivatives (11-cis- and all-trans-retinal) to form precursors of toxic A2E and related compounds. In addition, reducing the availability of 11-cis-retinal decreases retinal metabolic demands under dark conditions. Emixustat when delivered orally was found to be generally well tolerated in human clinical studies with delayed dark adaptation being the most common adverse event. Acucela is exploring emixustat’s potential to stop or slow the progression of vision loss in patients diagnosed with Stargardt disease in an ongoing clinical study.

About Acucela Inc.

Acucela Inc. is a 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. Acucela’s development pipeline include drug candidates for the treatment of diabetic retinopathy, Stargardt disease, and optogenetics-based gene therapy for the treatment of retinitis pigmentosa. The company is also developing a handheld OCT device for the monitoring of neovascular retinal diseases, to be used directly by patients.;

Cautionary Statements:

“Acucela”, the Acucela logo and “Kubota” are registered trademarks or trademarks of Acucela Inc. or Kubota Pharmaceutical Holdings in various jurisdictions.


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