Annexon Provides Update on ARCHER II Global Registrational Program in GA

Annexon announced patient dosing was initiated in the global pivotal phase 3 ARCHER II trial evaluating ANX007 for the treatment of geographic atrophy (GA). The company says the trial is the only GA pivotal program utilizing visual protection as the global regulatory-aligned primary endpoint.

Additionally, Annexon announced new data regarding protection of vision and vision-related retinal structure from the phase 2 ARCHER trial, supporting the disease modifying potential of ANX007 for the treatment of GA. These data were presented on July 18, 2024, at the American Society of Retina Specialists (ASRS) annual scientific meeting.

“ANX007 is the only investigational medicine for GA to date to show in a randomized clinical trial significant protection of both visual acuity and key visual structures in regions of the eye essential for vision,” Douglas Love, president and chief executive officer of Annexon, said in a company news release. “These data underscore ANX007’s potential best-in-class profile and make all the more exciting the initiation of the ARCHER II global, pivotal phase 3 study designed to treat patients impacted by this devastating disease at a vulnerable stage in their lives. Assuming success in the ARCHER II trial, ANX007 has the potential to be the first GA therapy approved to protect vision.”

ARCHER II is a global, randomized, sham-controlled phase 3 trial with topline data expected in the second half of 2026. The ANX007 registrational program has received regulatory alignment with both the FDA and the European Medicines Agency (EMA) on key study elements, including on using, for the first-time in GA, best corrected visual acuity (BCVA) protection against ≥15-letter loss as the primary outcome measure. In ARCHER II, ANX007 will be compared to sham control in a well-powered study with a robust safety database that is expected to enroll approximately 630 patients. ANX007 is the only therapeutic candidate for GA to date to receive Priority Medicine (PRIME) designation in the European Union, and has been granted Fast Track designation from the FDA.

Data highlights from the ARCHER phase 2 analyses presented at the ASRS annual meeting include:

ANX007 provided broad-based protection of vision vs. sham-treated eyes

• Statistically significant and dose dependent protection of vision measured by best corrected visual acuity (BCVA) protection against ≥15-letter loss at month 12, which represents three lines on the standard Early Treatment of Diabetic Retinopathy Study (ETDRS) eye chart and is a widely accepted clinically meaningful assessment of visual acuity (21.3% (sham) vs. 5.6% (ANX007 monthly); P=0.0021)
• Statistically significant protection of vision in low light conditions, low luminance visual acuity (LLVA) at month 12, which is an important assessment of visual performance for everyday activity (LLVA ≥15-letter loss at month 12: 20.3% (sham) vs. 7.6% (ANX007 monthly); P=0.022)

ANX007 provided structural protection in regions of the retina critical for visual acuity 

• Photoreceptors are neurons in the retina responsible for detecting light, and thus protecting their structure is essential for protecting vision
• ANX007 provided up to 60% protection of photoreceptors compared to sham within the central 1.5 mm of the fovea, as measured by ellipsoid zone (EZ) through 12 months (P=0.0319*)
• ANX007 provided 29% protection of photoreceptors across the full retinal field as measured by EZ through 12 months (P=0.017*)
• ANX007 demonstrated a trend of protection against RPE loss in the central foveal subfield, the region where RPE loss best correlates with vision loss (18% protection of RPE loss, P=0.40*). RPE loss is a measure that lags behind photoreceptor loss.