Date: 2015-05-13
Type of information: Publication of results in a medical journal
phase: preclinical
Announcement: publication of results in Plos One
Company: Acucela (USA - MA)
Product: emixustat hydrochloride
Action mechanism:
Disease: degenerative retinal diseases
Therapeutic area: Ophtalmological diseases
Country:
Trial details:
Latest
news: * On May 13, 2015, Acucela, a clinical-stage biotechnology company that specializes in discovering and developing novel drug candidates to potentially treat and slow the progression of sight-threatening ophthalmic diseases, announced the publication of pre-clinical data related to the company’s investigational drug candidate, emixustat hydrochloride (emixustat), in PLOS ONE. The published article is entitled “Visual Cycle Modulation as an Approach Toward Preservation of Retinal Integrity,” and can be accessed at: http://dx.plos.org/10.1371/journal.pone.0124940. Authors of the paper provide biochemical and electrophysiological data in mouse models of retinal disease to show that emixustat produces a dose-dependent, reversible suppression of both visual chromophore biosynthesis and rod photoreceptor activity. In these models, emixustat treatment was shown to protect photoreceptors from light-mediated damage, reduce levels of toxic retinal fluorophores, such as A2E, and preserve the retinal vasculature during periods of hypoxia. The authors note that these findings could be relevant for the development of potential treatments for certain degenerative retinal diseases. Emixustat has been formulated for oral administration and is currently being investigated in a multi-center Phase 2b/3 clinical trial to evaluate safety and efficacy in patients with geographic atrophy (GA) associated with dry age-related macular degeneration (AMD).
