Acucela to Present ACU-4429 Phase 1 Data at the 10th International Symposium on Ocular Pharmacology and Therapeutics
Comprehensive Phase 1a and b Findings for Novel, Oral Compound for
Dry AMD to be Highlighted
Seattle,
Washington and Vienna, Austria (November 30, 2011)
– Acucela Inc., a Washington State-based clinical-stage
biotechnology company focused on developing new treatments for
blinding eye diseases, today announced that data for ACU-4429, the
Company’s novel, small-molecule visual cycle modulator (VCM)
for the treatment of dry age-related macular degeneration (dry
AMD), will be highlighted at the 10th International Symposium on
Ocular Pharmacology and Therapeutics (ISOPT) being held in Vienna,
Austria from December 1-4, 2011. John W. Chandler, M.D.,
Acucela’s vice president clinical affairs, will present:
“Progress in the Development of ACU-4429 for the Treatment of
Dry AMD” during the Retina: Dry AMD portion of the ISOPT
program on December 1, 2011, at 4:40 p.m. local time.
Dr. Chandler will discuss comprehensive data from the Phase 1a and
1b clinical studies of ACU-4429. The Phase 1a study assessed the
safety, tolerability, pharmacokinetics and pharmacodynamics of a
single dose administration of ACU-4429 in healthy volunteers aged
55-80. In this single-center, randomized, double-masked,
placebo-controlled, dose-escalation study, oral administration of
ACU-4429 was well-tolerated and a dose-dependent modulation of the
visual cycle was demonstrated using electroretinography (ERG), an
established eye test. Importantly, these data marked the first time
that a non-retinoid therapeutic in an oral pill form has
effectively targeted the visual cycle in a dose-dependent
manner.
The Phase 1b study assessed the safety, tolerability and
pharmacokinetics of daily doses of ACU-4429 for 14 days in 40
healthy volunteers aged 26-55. This study found that ACU-4429 was
well tolerated for 14 days at doses up to 40 mg per day.
“We are pleased to present the full results of our Phase 1b
study for ACU-4429 for the first time at a major medical
meeting,” stated Dr. Chandler. “Together, these Phase 1
studies show that ACU-4429 is well-tolerated and demonstrated its
ability to slow the visual cycle and potentially decrease
accumulation of toxic by-product in the retina. These findings
continue to illustrate the potential for ACU-4429 in the treatment
of dry AMD, for which there is no currently approved
therapy.”
Acucela to Present ACU-4429 Phase 1 Data at the 10th International
Symposium on Ocular Pharmacology and Therapeutics-Page 2
Initial data from these Phase 1 clinical trials have been presented
at other key scientific meetings and were published earlier this
year in Retina: The Journal of Retinal and Vitreous Diseases.
ACU-4429 is currently being studied in a Phase 2 multicenter,
randomized clinical trial in patients with dry AMD and the Company
expects to announce data from that trial in 2012.
AMD is a chronic degenerative disease of the retina and is the most
common cause of permanent visual loss among the elderly population.
AMD is classified into two forms: the wet (neovascular) form and
the dry (non-neovascular) form. According to Frost & Sullivan,
dry AMD is the most common form, representing about 90 percent of
all cases, and approximately 15 percent of those who suffer from
AMD develop intermediate or advanced dry AMD, typically exhibited
with geographic atrophy, with the risk of developing advanced dry
AMD increasing with age. Frost & Sullivan estimates that
the prevalence of wet AMD and advanced dry AMD in the United States
was 1.44 million and 0.72 million in 2009 and will be 1.61 million
and 0.81 million, respectively, by 2016.
About
ACU-4429
ACU-4429 utilizes Acucela’s proprietary visual cycle
modulation (VCM) technology, and is designed to prevent or inhibit
the generation of toxic by-products of the visual cycle that can
lead to degenerative eye conditions like dry AMD. In 2010, ACU-4429
was granted Fast Track designation by the U.S. Food and Drug
Administration. Data indicate that ACU-4429 slows the rod visual
cycle, resulting in decreased accumulation of a toxic by-product
that is the precursor of lipofuscin, which are deposits of toxic
substances. The chronic accumulation of lipofuscin has been
implicated in degenerative retinal diseases. ACU-4429 is
administered to patients as an oral, daily pill rather than by
injection into the eye, which is typical of many current eye
therapeutics for retinal disorders. Acucela has forged a strategic
partnership with Otsuka Pharmaceutical, Co. Ltd. to co-develop
ACU-4429 in dry AMD as well as other potential indications in North
America.
About
Acucela Inc.
Acucela Inc. (www.acucela.com) is a clinical-stage
biotechnology company focused on leveraging promising science in
visual cycle modulation (VCM) to develop new methods for treating
blinding eye diseases that affect tens of millions of people
worldwide. The Company’s orally delivered VCM
compounds, which selectively target cells within the retina to
protect visual acuity, have the potential to treat dry AMD, for
which there is no currently approved treatment, retinopathy of
prematurity, Stargardt disease and diabetic retinopathy.
ACU-4429 for dry AMD is being co-developed by Acucela and Otsuka
Pharmaceutical Co., Ltd. in North America, and the companies are
also co-developing rebamipide for dry eye and OPA-6566 for glaucoma
in the United States. Acucela was founded by Ryo Kubota,
M.D., Ph.D., a pioneer in ophthalmology and the discoverer of the
gene that causes glaucoma.
Posted: December 2011
