After posting mixed late-stage data last fall and securing FDA support to include midstage data in its approval application for pegcetacoplan in geographic atrophy (GA), Apellis has been awaiting one last set of results before proceeding to the agency. Now, with those 18-month data in hand, the company is getting its regulatory package together for a filing next quarter.
In top-line results released in September 2021, Apellis said its C3 inhibitor pegcetacoplan, the active ingredient in rare disease drug Empaveli, met its primary endpoint in the phase 3 OAKS trial. In patients with GA secondary to age-related macular degeneration (AMD), the drug cut GA lesion growth after 12 months by 22% in a monthly treatment arm and by 16% in the every-other-month group compared with sham injections.
In the DERBY study, the drug didn't fare as well. In that phase 3 trial, the med cut GA lesion growth at 12 months by 12% and 11% in the monthly and every-other-month arms, respectively.
Now, in updated findings for the medicine's effect out to 18 months, Apellis said the OAKS GS lesion growth reduction once again came in at 22% for the monthly group and 16% for the every-other month arm. At DERBY's 18-month reading, investigators recorded a 13% reduction for the monthly group and 12% for the every-other-month patients. The company noted that the p-values for all findings were "nominal."
In the trials, the drug's effects started to appear within six months, and the data "show the potential for improving treatment effects over time," Apellis said in a Wednesday investor presentation.
“It’s a very important win for us at Apellis and a further confirmation of the efficacy profile, combined with the safety profile,” for pegcetacoplan in GA, Cedric Francois, M.D., Ph.D., the company’s co-founder and CEO, said in an interview.
Together, the phase 3 trials enrolled 1,258 adults with GA, a leading cause of blindness that afflicts more than 1 million people in the U.S. and more than 5 million globally, the company says. There are no approved treatments for GA.
Despite the mixed 12-month results, Apellis last September said it believed the data could support an FDA filing and potential approval. And in November 2021, the company said the FDA had signed off on its plan to submit data from the phase 2 FILLY trial as well.
With the 18-month data in hand, Apellis plans to submit its FDA filing in the second quarter of 2022. That could set the drugmaker up for an FDA decision by the end of the year, according to the investor presentation.
Taking OAKS, DERBY and FILLY together, Francois characterized the results as “highly clinically meaningful result.”
In the OAKS study, the drug was shown to preserve about 0.66 square millimeters of retina in GA patients over 18 months. While that space could fit “on the tip of a pencil,” that’s “actually a large area” in the retina, Francois explained.
For instance, the area of the retina that is most critical for one's ability to read an eye chart at the doctor is less than 2 square millimeters, he said.
“We believe we are on a path to having the first drug approved for this disease," Francois said.
Apellis, founded in 2009, is already a commercial drugmaker thanks to pegcetacoplan's approval last year to treat the rare blood disorder paroxysmal nocturnal hemoglobinuria. In that field, the drug is marketed as Empaveli and is challenging Alexion's successful complement inhibitors Soliris and Ultomiris. In GA, analysts have figured a regulatory win for pegcetacoplan could bring in blockbuster-level sales.