Acucela Validates Sampling Method That Could Pave Way For More Efficient Clinical Tests

Article published in Bioanalysis shows blood sampling method could be more affordable, accessible

SEATTLE--(BUSINESS WIRE)--Acucela Inc. (TOKYO:4589), a clinical-stage biotechnology company that specializes in discovering and developing novel drug candidates to potentially treat and slow the progression of sight-threatening ophthalmic diseases, has validated an analytical method that could lead to significant cost savings in the collection of blood samples as part of the company’s clinical trials of emixustat hydrochloride (emixustat), the company announced today.

“Bioanalysis of emixustat (ACU-4429) in whole blood collected with volumetric absorptive microsampling by LC-MS/MS”

Emixustat, the first internally developed compound by Acucela, is orally dosed, targets the visual cycle, and is in development for the potential treatment of geographic atrophy (GA) associated with the dry form of age-related macular degeneration (AMD), for which there is currently no U.S. Food and Drug Administration (FDA)-approved therapy available. The long-term clinical safety and efficacy of emixustat is being evaluated in an ongoing Phase 2b/3 study.

Acucela had an article published in a recent issue of Bioanalysis showing promising results for the collection of human dried blood using a newly invented, patent pending Mitra™ VAMS™ (volumetric absorptive microsampling) device from the company Neoteryx. VAMS is a novel approach for obtaining dried biological fluids for quantitative bioanalysis, without volumetric hematocrit bias, that collects a fixed amount of blood (e.g. 10 microliters) following a fingerprick or other method for obtaining low volumes.

The company’s study validated the VAMS method for the bioanalysis of emixustat in human blood. This method is being tested in bridging studies to confirm if the Mitra VAMS device can be used in late-stage clinical trials of emixustat. The VAMS collection method, using dried blood instead of wet blood, could be more efficient and economical than the traditional method of collecting plasma samples – and not just in the testing of emixustat. Since VAMS uses dried blood, shipping and handling costs of samples are much lower, and samples can be collected in more varied and remote locations than a research lab or hospital.

“Emixustat is being developed for the treatment of GA associated with dry AMD, the most common cause of blindness in the aging population, and it would be administered in oral pill form, which is less invasive than commonly explored intravitreal injection,” said Ryo Kubota, MD, PhD, Chairman, President and CEO of Acucela. “Our research published in Bioanalysis regarding blood collection has the potential to make that development process more efficient.”

The article, “Bioanalysis of emixustat (ACU-4429) in whole blood collected with volumetric absorptive microsampling by LC-MS/MS,” is in Vol. 7., No. 16 (pages 2071-2083) of Bioanalysis. An abstract can be found here.

About Acucela Inc.

Acucela Inc. (www.acucela.com or www.acucela.jp) is a clinical-stage biotechnology company that specializes in discovering and developing novel drug candidates to potentially treat and slow the progression of sight-threatening ophthalmic diseases, which could impact millions of individuals worldwide. Acucela currently has the following drug candidates in development in collaboration with Otsuka Pharmaceutical Co., Ltd.: emixustat hydrochloride, its lead investigational drug candidate for geographic atrophy associated with dry age-related macular degeneration based on Acucela’s proprietary visual cycle modulation technology; and OPA-6566, an investigational compound originating from Otsuka Pharmaceutical for the treatment of ocular hypertension and glaucoma.

Cautionary Statements

Certain statements contained in this press release are forward-looking statements made pursuant to the safe harbor provisions of 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. Words such as “will” as well as similar expressions are intended to identify forward-looking statements. These forward-looking statements include Acucela’s expectations regarding corporate development activities and the ultimate success of the enterprise. These statements involve risks and uncertainties that could cause the Company’s actual results to differ materially from those projected in forward-looking statements, including, but not limited to, the risk that our investigational product candidates will not demonstrate the expected benefits, the success of our investigational product candidates depends heavily on the willingness of our collaboration partner to continue to co-develop our investigational product candidates, the risk of delays in our expected clinical trials, the risk that new developments in the intensely competitive ophthalmic pharmaceutical market require changes in our clinical trial plans or limit the potential benefits of our investigational product candidates, and other risks and uncertainties inherent in the process of discovering and developing therapeutics that demonstrate safety and efficacy. For a detailed discussion of these and other risk factors, please refer to the Company’s filings with the Securities and Exchange Commission, which are available on the Company’s investor relations Web site (http://ir.acucela.com/) and on the SEC’s Web site (http://www.sec.gov).