This article is more than 5 years old. Information may no longer be current.
Acucela files investigational new drug application for novel dry AMD therapy
Click Here to Manage Email Alerts
BOTHELL, Wash. — Acucela has filed an investigational new drug application with the U.S. Food and Drug Administration to conduct a phase 1 clinical trial of its lead compound for treating dry age-related macular degeneration, the company announced in a press release.
The compound, called ACU-02, is an orally accessible small-molecule modulator of the visual cycle, which is believed to be a factor in the pathophysiology of the dry form of AMD. In preclinical trials, ACU-02 demonstrated a desirable pharmacokinetic profile, which significantly decreased the accumulation of the retinal-related toxic byproduct A2E, which is believed to damage retinal cells and contribute to vision loss in patients with AMD.
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 later this year.
"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," Ryo Kubota, MD, PhD, Acucela's chief executive officer, said in the release.