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Regeneron Pharma Announces Positive Phase 2 Results In Rare Blood Disorder

Regeneron Pharmaceuticals Inc. (REGN) announced data from the pozelimab Phase 2 clinical program in paroxysmal nocturnal hemoglobinuria or PNH, validating the weekly 800 mg subcutaneous dosing regimen, following an initial intravenous or IV loading dose. Pozelimab reduced the abnormal destruction of red blood cells, otherwise known as "hemolysis," with patients in the initial cohort achieving normal levels of a blood biomarker of elevated hemolysis called lactate dehydrogenase or LDH.

PNH is an ultra-rare, chronic, life-threatening disease where genetic mutations cause hemolysis, resulting in a range of symptoms including fatigue, shortness of breath and blood clots.

The results from initial 6-patient cohort showed pozelimab reduced lactate dehydrogenase to normal levels at week 8 in patients with paroxysmal nocturnal hemoglobinuria, utilizing a weekly subcutaneous dosing regimen.

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