Research getting ready to enter into phase 3 for a new drug to help those with dry macular degeneration.

Apellis sets up another phase 3 program for lead drug APL-2

by Phil Taylor 

It’s been a good summer for Apellis, which has just reported positive mid-stage data for its lead candidate in age-related macular degeneration (AMD) a couple of weeks after it raised $60 million in a series E round.

With the additional funds (PDF) and phase 2 data in hand, Apellis can now accelerate plans to start a phase 3 program for lead candidate APL-2 in the dry form of AMD, which could be a second and much larger late-stage indication for the complement C3 inhibitor after rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH).

Top-line results from the newly reported FILLY trial show that APL-2 met its primary objective of holding back the progression of geographic atrophy (GA), an advanced form of AMD, compared to a sham therapy.  

Giving the drug by intravitreal injection once a month cut progression by 29% over 12 months, while dosing every month reduced it by 20%. Moreover, zeroing in on the second half of that period revealed an even stronger effect, with GA progression cut 47% and 33% compared to sham.

Apellis’ CEO Cedric Francois, M.D., Ph.D., said the company is “very excited” by the data and intends to move forward with phase 3 “as soon as possible.”

While there are numerous treatments for the wet form of AMD, dry AMD with GA is currently an untreatable condition, according to David Boyer, M.D., of Retina-Vitreous Associates Medical Group, who said: “the reduction of the progression of atrophy in this trial offers new hope for vision maintenance for our patients.” It’s worth noting that dry AMD also accounts for 85% to 90% of all AMD cases, but tends to progress more slowly.

The new data are a big step toward fulfilling Apellis’ ambition to develop a series of indications for its C3 inhibitor beyond PNH in a market currently monopolized by mid-cap biotech Alexion’s C5-targeting Soliris (eculizumab), which made its debut way back in 2003….

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Source: Fierce Biotech