- New data from a clinical study of Apellis Pharmaceutical Inc's experimental eye drug APL-2 appeared to support previous results, driving shares in the biotech higher on Thursday and in early Friday trading.
- APL-2, as the drug is known, had already demonstrated a significant reduction in the growth of lesions associated with geographic atrophy (GA) in patients with age-related macular degeneration (AMD). Results from a six-month follow-up period further suggested that effect was due to the drug's biological activity.
- During the six months when no treatment was administrated, GA lesion growth rates between previously treated patients and those who received sham were similar — indicating the statistically significant effect observed in the primary analysis was due to treatment.
Geographic atrophy, an advanced form of age-related macular degeneration, leads to central blind spots and permanent vision loss. It affects around a million people across the U.S., who have no currently approved treatments to try.
Previously released results from the 12-month FILLY Phase 2 study showed APL-2 met its primary endpoint, significantly reducing the rate of GA lesion growth. Monthly injections reduced growth rate by 29%, while alternate month injections led to a more modest reduction in growth rates of 20%.
After 12 months of treatment, patients were followed for another six months without further administration of the drug. Growth rates were still reduced versus sham treatment, although the effect was more in line with the control than during the on-treatment period.
Apellis sees the updated data as supportive of the drug's biological activity.
"We are pleased that the 18-month data support that the statistically significant effect we observed in the first 12 months of the study was the result of the biological activity of APL-2," said company CEO Cedric Francois in a statement.
Looking ahead, Apellis plans to soon begin identical Phase 3 studies comparing multiple intravitreal injections of APL-2 in patients with GA. Like the FILLY study, the late-stage trials will test the drug's effect on GA lesion growth at 12 months.
Geographic atrophy has proven a a tough test for drug developers. Following past clinical setbacks from other companies, Roche AG's hopeful lampalizumab recently failed its second Phase 3 study in GA. The company later confirmed it would not seek U.S. approval in that indication.