Apellis Pharmaceuticals hasn't yet filed its eye drug pegcetacoplan with the FDA, but it's revving up its awareness work now.
To gauge patient sentiment about the degenerative disease it treats—geographic atrophy (GA), which can lead to permanent vision loss—Apellis turned to The Harris Poll. The survey found that 68% of patients with the condition believe their visual decline has affected their independence and quality of life more than they had expected.
Meanwhile, the vast majority of GA patients said the disease has negatively affected specific aspects of their everyday lives such as the ability to read (96%), drive (95%) and travel (88%). The survey covered about 200 older people in North America, Western Europe and Australia.
Most importantly for Apellis’ future marketing plans, three out of four of the surveyed patients (76%) thought their vision loss was "a natural part of aging" before they were diagnosed. That misconception “reinforces the opportunity for broad disease education,” Apellis said.
That will be a big pitch for Apellis, which will jump into a brand-new market if pegcetacoplan wins approval. On one hand, that's great: no rivals to battle for market share. But it's also tough, because Apellis will have to build awareness from scratch with doctors and patients who aren't used to having a drug treatment to prescribe or use.
The survey showed patients are ready to be educated: 91% said they “wish to be empowered with more information and options to take control over their disease.”
“Quality of life is slipping away much faster than anticipated for an overwhelming number of people with geographic atrophy, so it is important for patients to know they are not alone,” said Stacy Pagos Haller, president and CEO of the BrightFocus Foundation, which helps fund research and spread awareness for eye diseases. "Our hope is that these results spark a public dialogue about the heavy toll … on patients’ well-being and encourage people to actively advocate for their eye health.”
GA is an advanced form of dry age-related macular degeneration (AMD). It happens in regions of the retina where cells waste away and die, and it eventually leads to blind spots in one or both eyes. Around 1 million people in the U.S. are estimated to have the disease.
The active ingredient in Apellis’ drug is already approved as Empaveli, which treats paroxysmal nocturnal hemoglobinuria, a rare blood disease. Apellis reformulated the therapy from an injection under the skin to a version injected into the eye.
It’s delivered a mixed bag in recent trials: Last year, the biotech unveiled results from two phase 3 trials, known as OAKS and DERBY, which tested pegcetacoplan as a monthly and every-other-month treatment in 1,258 patients with GA stemming from AMD. Investigators weighed efficacy by measuring change in the total area of GA lesions from baseline.
In the OAKS trial, pegcetacoplan hit its primary goal, reducing GA lesion growth by 22% in the monthly arm and 16% in the every-other-month group compared with placebo at the 12-month mark. But the drug fell short of statistical significance in DERBY, reducing lesions by just 12% and 11%, respectively.
But Apellis got better news from longer-term follow-up. The drug bested placebo by larger margins in both trials after that longer period, giving the biotech confidence to go for a green light.
The company has said it plans to ask the FDA for priority review in the second quarter, so, if all goes smoothly, it could be looking at an approval before year-end.
Other drugmakers are in the race for GA treatments, though, including Iveric Bio, which has a candidate in Zimura (avacincaptad pegol) in phase 3 testing with results due this year.
Other programs include NGM Biopharmaceuticals’ NGM621 in phase 2 and Catalyst Bio/Biogen’s pegylated CB 2782 in early development.