Alexion Pharmaceuticals (ALXN +8.4%) is up on more than 50% higher volume in reaction to Novartis’ (NVS -0.1%) announcement of positive results from an open-label, Phase 2 clinical trial evaluating investigational oral treatment LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
Patients receiving LNP023 as an add-on to Alexion’s Soliris (eculizumab) experienced significant reductions in lactate dehydrogenase levels, a biomarker of intravascular hemolysis, compared to eculizumab alone.
Specifically, LNP023 increased hemoglobin (Hb) by a clinically relevant 2.87 g/dL. 80% (n=8/10) achieved Hb levels greater than 12.00 g/dL (normal range: 13.5 – 17.5 g/dL for men and 12.0 – 15.5 g/dL for women) without blood transfusions. All required transfusions beforehand.
After at least six months of stable LNP023 add-on therapy, at investigators’ discretion, 70% (n=7/10) discontinued eculizumab and continued receiving LNP023 as monotherapy. All maintained Hb levels with no changes in biomarkers of disease activity and no symptoms of breakthrough hemolysis.
LNP023 is a factor B inhibitor of the alternative complement pathway, acting upstream of the C5 terminal pathway where eculizumab works.
PNH is a rare blood disorder characterized by the destruction of red blood cells (hemolytic anemia), blood clots (thrombosis) and impaired bone marrow function.
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