On Tuesday, Apellis Pharmaceutical Inc. announced that its experimental drug for curing a blood disorder has reflected great improvement in haemoglobin level of various patients. It is performing better than Soliris by market leader Alexion Pharmaceuticals Inc.
80 paroxysmal nocturnal haemoglobinuria patients suffering from anaemia while consuming Soliris were tested with Apellis’s drug, pegcetacoplan. Among these patients, around half were dependant on blood transfusion to sustain optimal haemoglobin levels.
Apellis will likely challenge the dominance of Alexion in PNH market after the results of late stage trial. The only two drugs approved for PNH are Alexion’s Ultomiris and Soliris that recorder sale of around $1.08 billion during September quarter. Apellis’s shares went up by 22% at $ 36.8 after reaching 40.7%.
Madhu Kumar, Baird analyst said that the concerns grew after pegcetacoplan failed to match Soliris in matching effectiveness by lowering dehydrogenase’s lactic acid levels.
Hemolysis breakage in red blood cells are an indicator of LDH levels in PNH patients. According to Apellis, pegcetacoplan indicated positive results on reduction of LDH level however, the drug failed to indicate non-inferiority to Soliris.
Analysts called attention of investor’s concern if mere improvement in levels of haemoglobin would justify the approval of the product by FDA.
The company believes that the study data predicts strong approval and superiority of the drug over Soliris.
Apellis said that 70% of PNH patients reflect low haemoglobin levels regardless of treatment with Soliris and around 36% are recommended to transfuse blood once or twice in a year. With pegcetacoplan, also known as APL-2, the unmet needs will be addressed.
Cedric Francois, Chief Executive Officer said that almost two third of PNH patients treated with Soliris face difficulty climbing stairs and often run out of breath but with APL-2, patients can have better chance of living a normal life.