“In patients treated with C5 inhibitors alone, extravascular hemolysis (EVH) is an ongoing problem resulting in anemia with an impact on most of the clinical parameters of hemolysis. These interim findings from this importantproof-of-concept trial show that if the alternative pathway is adequately inhibited, important parameters of EVH can be improved, including and most importantly hemoglobin, transfusion, bilirubin, reticulocyte count, and FACIT fatigue scores, when added to stable optimal dose eculizumab therapy. We look forward to completing the Phase 2 trial this summer and future discussions with the regulatory authorities. After completion of regulatory discussions, we hope to initiate a Phase 3 PNH combination trial ofACH-4471 with C5 inhibitors in the first half of 2020,” stated Steven Zelenkofske, D.O. Chief Medical Officer at Achillion.
Slides from the oral presentation will be available on the Company’s website, today, May 17, at approximately 9:00 a.m. EST athttp://ir.achillion.com/events-and-presentations.
ACH-4471 Phase 2 Trial in Combination with Eculizumab
ACH-4471 is being evaluated in combination with eculizumab, an intravenous C5 inhibitor that is currently approved as monotherapy for PNH. This is a Phase 2, open-label, multiple dose trial in adult patients on stable eculizumab treatment with blood transfusion dependent anemia, defined as receiving at least one transfusion in the 12 weeks prior to the study and a hemoglobin level below 10 g/dL. In addition to their usual dose of eculizumab, patients are administeredACH-4471 orally three times a day at a dose determined by patient clinical response. The primary outcome of the trial is the change in hemoglobin at 24 weeks compared to baseline. Secondary outcomes include the number of blood transfusions required, impact on selected clinical parameters, and safety. The trial will be followed by a long-term extension phase.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is thought to be caused by a mutation resulting in the absence of receptors normally present on red blood cells (RBCs) that interact with the complement system. The complement system typically functions normally in these patients but due to the lack of key receptors, known as CD55 and CD59, on the surface of PNH RBCs, the complement system treats these cells as foreign and destroys them via hemolysis in the circulatory system (intravascular) and in the liver or spleen (extravascular). The complement alternative pathway (AP) is a critical factor in the development of extravascular hemolysis. Complement factor D is a critical protein within the amplification loop of the AP and it is believed that inhibiting it could control the AP response. Furthermore, this mechanism of action represents a potentially distinct and unique therapeutic approach for controlling intravascular and extravascular hemolysis associated with PNH.
More information is available at http://www.achillion.com/patients-and-clinicians/.
About Achillion Pharmaceuticals
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical company focused on advancing its oral small molecule complement inhibitors into late-stage development and commercialization. Research has shown that an overactive complement system plays a critical role in multiple disease conditions including the therapeutic areas of nephrology,