Apellis Shares Rise After Saying Co-Treating With Eculizumab and APL-2 Shows Improvement

Apellis Pharmaceuticals (APLS) shares rose as much as 4.1% in Tuesday trading after the clinical-stage biopharmaceutical company provided an update on its US-based phase 1b Pharoah trial for patients with paroxysmal nocturnal hemoglobinuria (PHN).

Shares rose 2.8% to $19.90 at 2:04 pm ET in New York on Tuesday.

The Pharoah trial is evaluating treatment with APL-2, a novel inhibitor of complement factor C3, in patients on treatment with eculizumab (Soliris) who are severely anemic and transfusion-dependent. Four patients out of six reported an improvement.

The company said in a statement that patients enrolled in the trial, which is for people suffering from paroxysmal nocturnal hemoglobinuria (PNH), were initially co-treated with APL-2 and eculizumab, with the potential for discontinuation of eculizumab at the discretion of the treating physician. Two of the six patients were removed from treatment with APL-2 due to pregnancy and BMI-associated comorbidities.

Four of the six patients continued in the study for more than 32 weeks.

In these four patients, co-treatment with APL-2 resulted in an improvement of hemoglobin levels and other markers for anemia and none of the four patients required a transfusion during the co-treatment period with eculizumab, which ranged from 17 to 20 months.

“All four patients have improved hemoglobin and reticulocytes as compared to the baseline established with eculizumab monotherapy and have achieved lactate dehydrogenase (LDH) levels below the upper limit of normal (ULN),” the company said in the statement.