New data from Acucela’s ACU-4429 Phase 2a trial on advanced dry AMD/geographic atrophy

Acucela Inc., a clinical-stage biotechnology company focused on developing new treatments for blinding eye diseases, announced today that the first patient data from the ACU-4429 program were presented during a poster session at The Association for Research in Vision and Ophthalmology (ARVO) 2012 Annual Meeting. The presentation was made by John W. Chandler, MD, Vice President Clinical Affairs, Acucela and David G. Birch, PhD, Chief Scientific and Executive Officer, Retina Foundation of the Southwest.

ACU-4429 is Acucela's novel, small-molecule visual cycle modulator (VCM) being developed to treat dry age-related macular degeneration (dry AMD), which occurs when light-sensitive cells in the macula slowly break down, gradually blurring central vision in the affected eye. There are currently no approved treatments for dry AMD. Acucela's VCM technology relieves the metabolic stress on the retina and has been shown in preclinical studies to result in reduced accumulation of toxic byproducts (such as A2E), which is generally recognized as contributing to the progression of the disease.

The Phase 2a ENVISION (Evaluating a Novel VISION treatment for AMD) Clarity trial is evaluating the effect of ACU-4429 or placebo on rod function in patients with advanced dry AMD/geographic atrophy. Rods and cones are critical to healthy eye function. Cone cells, which allow the perception of color, have a critical role in detailed vision and central vision that is key to activities such as reading. Rod cells are more numerous than cone cells and are crucial for vision at night and in other low lighting conditions.

Dr. Chandler stated, "An effective treatment for patients who suffer from the devastating effects of advanced dry AMD is long overdue. By selectively modulating the visual cycle in rod cells, we believe that ACU-4429 protects cone cells and by extension protects visual acuity."

He continued, "These data represent an important milestone achievement for the ACU-4429 program and offer another proof point in support of the strengthened and differentiated qualities of our scientific approach in visual cycle modulation. ACU-4429 is an oral, non-retinoid therapy with a strong safety profile to date, and we believe in the therapeutic promise of ACU-4429 in advanced dry AMD. We look forward to reporting on progress later in the year."


Acucela Inc.


  1. Blaine Legault Blaine Legault United States says:

    Please let me know of any trials or new on Acu 4429.
    Thank You

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