BUSINESS WIRE
Orally-Available Small Molecule for Dry Form of AMD
Acucela Inc., a clinical-stage biotechnology company focused on developing therapies for blinding eye diseases, announced today that it has dosed the first subject in a Phase I trial for its lead compound ACU-02. The single-site trial is enrolling healthy normal volunteers in a double-masked, placebo-controlled, single ascending-dose study to evaluate the safety, tolerability and pharmacokinetics of ACU-02.
ACU-02 is a novel, orally available small molecule being developed for the treatment of the dry form of age-related macular degeneration (AMD). ACU-02 is a potent modulator of the visual cycle that acts by interrupting the pathophysiology of AMD by significantly reducing the accumulation of the retinal related toxic by-product A2E which is believed to damage retinal cells, thus leading to the decrease or loss of vision in AMD patients.
“We believe that ACU-02 provides significant promise for the millions of patients suffering from AMD, and we hope to be able to someday provide these patients with a treatment that is delivered in the form of a pill rather than an injection into the eye,” said Ryo Kubota, M.D., Ph.D., Acucela’s chief executive officer. “AMD is the leading cause of vision loss for people over the age of 50, and dry AMD accounts for approximately 90 percent of all cases. More than 29 million people are currently afflicted with AMD worldwide, and with the increase in the aging population, that number is expected to double in the next 20 years, so a safe and effective therapy for this disease is greatly needed. There are currently no approved therapies for dry AMD.”
About Acucela
Acucela Inc. is focused on developing new drug therapies for eye diseases, and particularly neurodegenerative retinal diseases such as macular degeneration. The company has proprietary disease-specific assays and technologies to identify and develop compounds that may safely and effectively treat retinal diseases. The company’s novel approaches have significant therapeutic potential to treat retinal diseases such as Age-related Macular Degeneration (AMD) and Stargardt disease. The Bothell-based, privately held biotechnology company was founded in 2002. For more information, please visit http://www.acucela.com