Apellis' eye med application on track for next year after FDA OKs inclusion of phase 2 data

After posting mixed phase 3 readouts in September, Apellis Pharmaceuticals' investigational eye drug pegcetacoplan is poised to hit the FDA's desk in geographic atrophy with a mix of mid- and late-stage data. 

The FDA gave Apellis the OK to use safety and efficacy data from two phase 3 trials, DERBY and OAKS, plus the phase 2 FILLY trial, when it files the drug for approval in geographic atrophy (GA) secondary to age-related macular degeneration in the first half of 2022. The company said it doesn't believe it will need to run any additional studies. 

GA, which is an advanced form of AMD that can lead to vision loss and blindness in older adults, has no FDA-approved treatments. Apellis' drug targets the protein complement component 3, which the company says is the only target that can precisely control complement overactivation, which drives lesion growth.

Pegcetacoplan, the active ingredient in Apellis' rare disease med Empaveli, won approval in May in patients with the rare blood disorder paroxysmal nocturnal hemoglobinuria. 

In written feedback, the FDA told Apellis it doesn't distinguish between trial phases, so long as the studies are adequate and well-controlled. All three trials Apellis plans to use in its application package meet those standards, the agency said, according to Apellis' release. 

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Back in September, pegcetacoplan posted a win in the phase 3 OAKS study, where it slashed GA lesion growth by 22% and 16% in patients who received the drug monthly and every-other-month, respectively. In DERBY, however, pegcetacoplan only reduced lesions by 12% and 11% in the same patient cohorts. Neither study raised significant safety concerns, although the drug was linked to slightly higher rates of exudation, or emitted fluid. 

Apellis' chief medical officer Federico Grossi, M.D., Ph.D., has pointed to the drug's potential as the first FDA-approved treatment for GA secondary to age-related AMD, which can cause blindness. 

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While the "totality of the evidence" bodes well for pegcetacoplan, it was previously unclear whether the FDA would sign off on the addition of the FILLY study to Apellis' filing package. The FDA's phase 2 blessing marks "an important de-risking for the submission process," Cantor Fitzgerald analysts wrote in a note to clients Friday. The drug's efficacy across the three related studies sits at around 20% reduction in GA lesion, the analysts said. 

Some 1 million Americans and 5 million people globally suffer from GA. If pegcetacoplan can succeed where others, like Roche's complement-inhibiting drug lampalizumab, have failed, it could be looking at $3 billion in peak sales, Jefferies analysts have predicted.

Earlier this year, Apellis entered the commercial realm when pegcetacoplan scored approval in paroxysmal nocturnal hemoglobinuria. In that disease, it's challenging Alexion's big-selling Soliris and the company's follow-up Ultomiris.