BUSINESS WIRE
Orally-available Small Molecule for Dry Form of AMD
BOTHELL, Wash.--(BUSINESS WIRE)--Acucela Inc., a clinical-stage biotechnology company focused on
developing therapies for blinding eye diseases, announced today that it
has filed an investigational new drug (IND) application to conduct a
Phase 1 clinical trial for its lead compound ACU-02 with the U.S. Food
and Drug Administration (FDA). The filing follows a successful pre-IND
meeting in Washington D.C. where the company reviewed its current
pre-clinical efficacy and safety data as well as development plans with
FDA.
ACU-02 is an orally available small molecule modulator of the visual
cycle, which is thought to play a key role in the pathophysiology of the
dry form of age-related macular degeneration (AMD), a disease which
afflicts over 29 million patients worldwide. In preclinical testing
ACU-02 was found to be a potent modulator of the visual cycle with
desirable pharmacokinetic profile, which significantly decreased the
accumulation of the retinal related toxic by-product A2E that is
believed to damage retinal cells, leading to the decrease or loss of
vision in AMD patients. Acucela plans to initiate dosing of healthy
normal volunteers in a double-masked, placebo-controlled, single
ascending-dose study to evaluate the safety, tolerability, and
pharmacokinetics of ACU-02 before the end of the second quarter of this
year.
“We believe ACU-02 is a unique,
first-in-class, potent, small molecule which holds significant promise
for the millions of patients suffering the debilitating effects of dry
AMD,” said Ryo Kubota, MD, Ph.D., Acucela’s
chief executive officer. “Given the fact that
this is a non-retinoid compound, we believe that it will have a better
side effect profile, and additionally, this product candidate is
delivered orally—an administration method
which we believe would be popular with patients given that current AMD
treatments are delivered by injection.”
About Dry Age-Related Macular Degeneration
Age-related macular degeneration afflicts over 29 million people
worldwide and is segmented into “dry”
and “wet” forms of
the disease. Dry AMD accounts for approximately 90 percent of the total
AMD population, and unlike wet AMD, there are no approved therapies for
the disease, which is currently the leading cause of vision loss in
people over the age of 50.
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 and injuries. The company’s
novel approaches have significant therapeutic potential to treat retinal
diseases such as Age-related Macular Degeneration (AMD) and Stargardt
disease, which affect 50 million people worldwide. The Bothell-based,
privately-held, biotechnology company was founded in 2002. For more
information, please visit http://www.acucela.com.