Results from initial 6-patient cohort show pozelimab reduced lactate dehydrogenase (LDH) to normal levels at week 8 in patients with paroxysmal nocturnal hemoglobinuria (PNH), utilizing a weekly subcutaneous dosing regimen
Second part of Phase 2 trial initiated; plans for Phase 3 program underway
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced topline data from the pozelimab (REGN3918) Phase 2 clinical program in paroxysmal nocturnal hemoglobinuria (PNH), validating the weekly 800 mg subcutaneous (SC) dosing regimen, following an initial intravenous (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 (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. Even with existing therapies, which require regular intravenous infusions administered at infusion centers or during a home visit by a health professional, approximately 50% of newly-treated patients do not achieve normal LDH levels.
“In our view, any new medicine for PNH must deliver real change for patients, such as more patients achieving normal LDH levels, or a reduced treatment burden that potentially allows for at-home self-administration,” said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron. “We are encouraged by these early pozelimab results, with patients achieving normal levels of LDH by week 8 using the subcutaneous dosing regimen. We look forward to speaking with regulators about the next phase of our program for these patients.”
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