Annexon Receives PRIME Designation from the EMA for ANX007 for the Treatment of Geographic Atrophy
Annexon announced that the European Medicines Agency (EMA) has granted Priority Medicine (PRIME) designation to ANX007 for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The EMA granted this designation based on the phase 2 ARCHER trial data that showed a statistically significant, durable, and dose-dependent preservation of visual function in patients with GA, as well as preclinical data supporting the protective effect of ANX007 against photoreceptor damage.
The EMA’s PRIME designation provides early and proactive support to developers of promising medicines that may offer a major therapeutic advantage over existing treatments or benefit to patients without treatment options. These medicines are considered priority medicines by the EMA, whose aim is to optimize development plans and speed up evaluations so these medicines that address significant unmet medical needs can reach patients faster.
“GA is a progressive disease impacting millions of elderly people worldwide, which severely limits their independence. There is a need for a treatment that provides the benefit both patients and physicians most desire—preservation of vision,” Douglas Love, president and CEO of Annexon, said in a company news release. “The data from our phase 2 ARCHER trial were the first to show a durable and dose-dependent preservation of visual function across multiple measures, including a statistically significant impact on the best corrected visual acuity (BCVA) functional endpoint. We believe PRIME designation supports the potential for ANX007 to address this critical need. As we look ahead, our focus remains on engaging with US and EU regulatory authorities to optimally design and expedite a global pivotal phase 3 program for ANX007 and bring this novel treatment to patients as quickly as possible.”
About ANX007 and Phase 2 ARCHER Trial
ANX007 is a fragment antigen-binding (fab) antibody designed as a first-in-kind therapeutic to selectively inhibit C1q, the initiating molecule of the classical complement pathway, and a key driver of neurodegeneration. In GA, C1q binds to photoreceptor synapses early in the disease process, causing aberrant activation of the classical pathway with synapse loss, inflammation and neuronal damage that results in vision loss. Intravitreal administration of ANX007 fully stops C1q and classical pathway activation to protect photoreceptor synapses and cells essential for vision. ANX007 is the first therapeutic candidate to receive PRIME designation for the treatment of GA.
In the randomized, multicenter, double-masked, sham-controlled phase 2 ARCHER clinical trial, ANX007 demonstrated a trend on the anatomical-based primary endpoint, but showed consistent protection against vision loss in a broad population of patients with GA. Specifically, topline data reported in May 2023 and presented at the American Society of Retina Specialists (ASRS) Annual Meeting in July 2023 showed that ANX007 provided statistically significant, time and dose-dependent protection from vision loss in patients with GA, measured by best corrected visual acuity (BCVA) ≥15-letter loss, the widely accepted and clinically meaningful functional endpoint. Protection from vision loss was also shown in multiple additional prespecified measures of BCVA and visual function, including low luminance visual acuity (LLVA) and low luminance visual deficit (LLVD). ANX007 was generally well-tolerated through month 12, with no increase in choroidal neovascularization (CNV) rates between the treated and sham arms and no events of retinal vasculitis reported.
The 6-month off-treatment follow-up period of the ARCHER phase 2 trial has been completed and Annexon plans to report the final results from the study at the American Academy of Ophthalmology 2023 Annual Meeting to be held November 3-6, 2023, in San Francisco, California.