(Reuters) – Apellis Pharmaceuticals Inc said on Tuesday its experimental drug for a blood disorder showed greater improvement in patients’ hemoglobin levels than Alexion Pharmaceuticals Inc’s Soliris, in a late-stage study.
The study tested Apellis’ pegcetacoplan in 80 paroxysmal nocturnal hemoglobinuria (PNH) patients who remained anemic despite taking Soliris. About half of them were even dependant on blood transfusions to maintain optimal hemoglobin levels.
The 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.
PNH is a rare, life-threatening disorder associated with abnormally low hemoglobin levels due to the destruction of oxygen-carrying red blood cells, making patients dependant on frequent blood transfusions.
About 70% of PNH patients show low hemoglobin levels despite treatment with Soliris and 36% require one or more transfusions a year, according to Apellis. 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 will be rapidly adopted by patients who have seen an inadequate response to Soliris and next-generation product Ultomiris, Cowen analyst Phil Nadeau said ahead of the trial results.
Nadeau expects APL-2 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.
Th drug, which targets an immune pathway known as the complement system, is also being tested in other indications such as geographic atrophy and complement-associated kidney diseases.
Pegcetacoplan was as effective as Soliris in reducing patient dependence on blood transfusions, a secondary goal of the study, Apellis said.
Reporting by Trisha Roy and Manojna Maddipatla in Bengaluru; Editing by Shinjini Ganguli