Apellis Pharmaceuticals Provides Update on Phase 1b Open Label Study of APL-2 in PNH Patients Not Previously Treated with Eculizumab

Apellis Pharmaceuticals (Nasdaq:APLS), a clinical-stage biopharmaceutical company focused on the development of novel therapeutic compounds for the treatment of autoimmune and inflammatory diseases, today announced interim data from its Phase 1b PADDOCK trial for patients with paroxysmal nocturnal hemoglobinuria (PNH).  The ongoing trial is evaluating the treatment with APL-2, a novel inhibitor of complement factor C3, in patients not previously treated with eculizumab (Soliris^TM).

As quoted in the press release:

The Company previously reported that APL-2 reduced lactate dehydrogenase (LDH) and increased hemoglobin (Hgb) levels in three patients not previously treated with eculizumab after daily subcutaneous administration of 270 mg of APL-2 for 28 days in the trial.  Based on the safety and activity observed in these patients, the administration period was extended from 28 days to one year and the number of patients to be enrolled in the PADDOCK trial was increased.  As of April 13, 2018, the data cut-off date for this interim analysis, six more patients have been treated with APL-2 at the 270 mg/day dose for a minimum of 28 days.

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