Acucela Announces Publication of Pre-clinical Data for Emixustat Hydrochloride

SEATTLE–(BUSINESS WIRE)–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 pre-clinical data related to the company’s investigational drug candidate, emixustat hydrochloride (emixustat), in the scientific journal Investigative Ophthalmology & Visual Science (IOVS).

The published article, “Emixustat reduces metabolic demand of dark activity in the retina,” can be accessed at: https://iovs.arvojournals.org/article.aspx?articleid=2756154. For additional information about IOVS, a journal published by the Association for Research in Vision and Opththalmology, please visit: http://www.iovs.org/.

Authors of the paper evaluated the effects of emixustat on retinal metabolism in rat models. In these animals, both oxygen consumption and ion channel activity in the retina under dark conditions, when the retina is most metabolically active, were evaluated. Emixustat treatment resulted in decreased oxygen consumption and ion channel activity. The authors note that these data indicate that emixustat can reduce metabolic and oxygen demands in the retina under dark conditions, which has implications for the treatment of ischemic retinal diseases in which hypoxia plays a prominent role, such as diabetic retinopathy.

Ryo Kubota, MD, PhD, and Chairman, President and CEO of Acucela Inc., said, “I am pleased that our pre-clinical data are now published in the journal IOVS to provide evidence that emixustat has the potential for therapeutic effects in diseases like diabetic retinopathy. We are actively seeking partnership opportunities to further advance these clinical programs.”

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 (Clinicaltrials.gov 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. https://www.acucela.com; https://www.kubotaholdings.co.jp/en/

Cautionary Statements: https://www.kubotaholdings.co.jp/en/1/index.html

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