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Acucela (AUCL) Granted New Patent Covering Method Of Use For Emixustat Hydrochloride

9/11/2017 8:27:07 AM

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SEATTLE--(BUSINESS WIRE)--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 the United States Patent and Trademark Office (USPTO) granted U.S. Patent No. 9,737,496 B2 assigned to Acucela, adding strength to the company’s intellectual property position. The patent is significant to Acucela because the claims are directed towards methods of use of emixustat hydrochloride (“emixustat”) and related compounds for providing a therapeutic benefit for subjects suffering from Stargardt disease.

Title of invention:

Alkoxy compounds for disease treatment


Method for providing a therapeutic benefit for subjects diagnosed with Stargardt disease comprising administering to the subject emixustat or a broad number of related compounds

Patent number:

U.S. Patent No. 9,737,496 B2


Acucela Inc., Seattle, Washington

Stargardt disease (STGD) is a rare, inherited, degenerative disease of the retina affecting approximately 1 in 8,000–10,000 individuals and is the most common type of hereditary macular dystrophy.(1). The most common form of the disease is caused by a genetic mutation of the ABCA4 gene leading to the accumulation of toxic vitamin A byproducts (primarily A2E) in the retina, which results in the gradual deterioration of photoreceptors and vision. Symptoms of Stargardt disease typically appear during childhood or adolescence, but in some cases difficulty with eyesight and vision loss may not be identified until later in life.

Stargardt disease affects less than 40,000 patients in the U.S. where it is recognized as an orphan disease, subject to the Orphan Drug Act. Currently, there are no known therapies that exist to slow the advance of the disease, and it is recognized as a serious unmet medical need by the United States Foundation of Fighting Blindness and the National Eye Institute.

The FDA (U.S. Food and Drug Administration) has granted orphan drug designation to emixustat hydrochloride for the treatment of Stargardt disease. (See January 5, 2017 press release titled “Acucela Receives Orphan Drug Designation from the FDA for the Treatment of Stargardt Disease”)

Currently, one Phase 2a clinical study of emixustat in patients with Stargardt disease is underway at Acucela. This multicenter, randomized, masked 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 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.

(1) Blacharski PA. Fundus flavimaculatus. In: Newsome, DA, ed. Retinal dystrophies and degenerations. New York: Raven Press, 1988:135-159.

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 and therapeutics for the treatment of retinitis pigmentosa, proliferative diabetic retinopathy, diabetic macular edema, AMD, Stargardt disease, cataract and presbyopia. The company is also developing a monitoring device for neovascular retinal diseases, to be used directly by patients.;

Cautionary Statements

Certain statements contained in this press release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. These forward-looking statements include statements regarding our expectations related to our development plans and ability to successfully develop and commercialize our product candidates and the potential efficacy, future development plans and commercial potential of our product candidates. These statements are based on current assumptions that involve risks, uncertainties and other factors that could cause the actual results, events or developments to differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties, many of which are beyond our control, include, but are not limited to: our investigational product candidates may not demonstrate the expected safety and efficacy; our pre-clinical development efforts may not yield additional product candidates; any of our or our collaborators' product candidates may fail in development, may not receive required regulatory approvals, or may be delayed to a point where they are not commercially viable; our clinical trials could be delayed; new developments in the intensely competitive ophthalmic pharmaceutical market may require changes in our clinical trial plans or limit the potential benefits of our investigational product candidates; the impact of expanded product development and clinical activities on operating expenses; adverse conditions in the general domestic and global economic markets; as well as the other risks identified in our filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date hereof and we assume no obligation to update these forward-looking statements, and readers are cautioned not to place undue reliance on such forward-looking statements. For a detailed discussion of the foregoing risks and other risk factors, please refer to our filings with the Securities and Exchange Commission, which are available on Kubota Pharmaceutical Holdings (Acucela’s parent company) investor relations website ( and on the SEC’s website (

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

Acucela Inc.
Media Contact :
Michael Hasegawa
Senior Director, Corporate Communications
Investor Relations Contact :
John Gebhart
Chief Financial Officer

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