Apellis' Drug Promising for Paroxysmal Nocturnal Hemoglobinuria

By Manojna Maddipatla and Trisha Roy

January 09, 2020

(Reuters) - Apellis Pharmaceuticals Inc said on Tuesday its experimental drug for paroxysmal nocturnal hemoglobinuria (PNH) showed greater improvement in patients' hemoglobin levels than market leader Alexion Pharmaceuticals Inc's Soliris.

Apellis tested its drug, pegcetacoplan, in 80 patients who remained anemic despite taking Soliris. About half of them were dependant on blood transfusions to maintain optimal hemoglobin levels.

The late-stage trial results would help Apellis challenge Alexion's dominance in the PNH market. Alexion's Soliris and Ultomiris, the only two currently approved drugs for PNH, recorded sales of $1.08 billion in the latest September quarter.

In terms of secondary goals, however, pegcetacoplan failed to match Soliris' effectiveness in reducing lactic acid dehydrogenase (LDH) levels.

LDH levels are an indicator of hemolysis in PNH patients.

While pegcetacoplan showed positive trends on the LDH level reduction, the drug failed to show non-inferiority to Soliris, according to Apellis.

Analysts highlighted investors' concern over whether improvement in hemoglobin levels is a sufficient benefit for FDA's approval of the product.

The company, however, believes the study data makes a strong case for the drugs' approval and its superiority over Soliris.

About 70% of PNH patients show low hemoglobin levels despite treatment with Soliris and 36% require one or more transfusions a year, Apellis said. With pegcetacoplan, also called APL-2, Apellis expects to address this unmet need.

Two-thirds of the Soliris-treated PNH patients cannot climb stairs without pausing to catch their breath, Chief Executive Officer Cedric Francois told Reuters.

"It all comes down to the quality of life... When these patients go on APL-2, they can essentially have a life that is pretty much normal."

APL-2 is expected to be launched in the United States in 2021 with penetration ramping to 28% in the PNH patient market in 2025, driving $400 million in worldwide PNH sales, according to Cowen analyst Phil Nadeau.

The drug, which targets the complement system, is also being tested in other indications such as geographic atrophy and complement-associated kidney diseases.