Giddy up: Alexion challenger Apellis could race to $4B in annual sales with Empaveli, analysts say

Apellis Pharmaceuticals is off to the races with its lead C3 inhibitor Empaveli.

It’s already won the Kentucky Derby with an FDA nod for patients with the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH), and now it’s eyeing the trophy at Preakness Stakes with its soon-to-come geographic atrophy (GA) data, Jefferies analysts wrote in a note to clients on Thursday. 

While the drug's full potential has yet to be realized, its eventual winnings could make it a thoroughbred next to rival C5 inhibitors, eventually racking up over $4 billion in annual sales, the analysts said. 

That’s high talk for little Apellis, which is going toe-to-toe with industry heavyweights like Alexion’s Soliris and its follow-up Ultomiris with pegcetacoplan, sold as Empaveli. That rivalry kicked off after Apellis won a key FDA nod in May to treat adult PNH patients with its under-the-skin injection. 

RELATED: Can a small biotech challenge Alexion’s Soliris? With Empaveli’s FDA nod, Apellis can give it a try

The disease is a rare, chronic blood disorder characterized by the destruction of healthy, oxygen-carrying red blood cells. The destruction is driven by an uncontrolled immune system and defective bone marrow not making enough blood components. 

Apellis’ C3 therapy, designed to regulate that excessive immune response, targets a different enzyme than Alexion's C5 inhibitors. But up until its FDA nod, the PNH space had been dominated by Alexion’s class of drugs. To Jefferies, however, Empaveli could prove to be stiff competition and eventually split the market, scoring more than $1 billion in annual sales in the process. 

That’s because Empaveli has already topped blockbuster Soliris in a head-to-head phase 3 trial, increasing hemoglobin levels by 3.8 g/dL over the Soliris group after 16 weeks. About 70% of C5-treated PNH patients are not well managed, with roughly 30% still requiring transfusions, the Jefferies team added. 

Meanwhile, Apellis is also expecting topline data from two phase 3 studies, aptly named Derby and Oaks, studying its C3 inhibitor in GA patients. There are about 1.4 million patients experiencing the advanced form of age-related macular degeneration that leads to progressive and irreversible vision loss. 

While the GA field has had its notable failures in recent years, including Roche’s lampalizumab, Jefferies expects Apellis to pass its late-stage test and garner enough “oomph” to push it over the finish line. That indication could draw in over $3 billion in peak sales, the analysts say.

RELATED: Apellis' PNH victory lap: Newly FDA-approved Empaveli helped patients without prior Soliris treatment

There’s still room for more trophies on Apellis’ shelf, which could come if it’s able to successfully race Empaveli through additional neurology indications, Jefferies said. The company is testing the drug in a phase 2 study for patients with amyotrophic lateral sclerosis (ALS), and it expects to complete enrollment by the end of the year.