REGN : Poze-Cemdi Combo In Phase 3 Result Shows Superior Hemolysis Control In PNH Vs. Ravulizumab

Alnylam Pharmaceuticals

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(RTTNews) - Regeneron Pharmaceuticals Inc. (REGN) announced positive updated Phase 3 data of an exploratory cohort from the ACCESS-1 trial investigating its first-in-class pozelimab and cemdisiran (poze-cemdi) combination treatment against ravulizumab, a standard-of-care complement factor 5 (C5) inhibitor, in patients with paroxysmal nocturnal hemoglobinuria.

According to the company, the Head-to-head exploratory cohort of a Phase 3 trial showed first-in-class poze-cemdi combination treatment helped patients achieve and maintain greater disease control, as measured by lactate dehydrogenase (LDH) levels, compared to standard-of-care ravulizumab.

The company noted that Five patients receiving ravulizumab did not achieve meaningful LDH control compared to one patient receiving poze-cemdi. After switching to the combination, four of the five previously treated with ravulizumab achieved LDH control.

Paroxysmal nocturnal hemoglobinuria or PNH is an ultra-rare, chronic, life-threatening complement-mediated blood disorder. People with PNH have an acquired genetic mutation in which red blood cells are destroyed (known as hemolysis) by the complement system, which is part of the innate immune system. The lysed red blood cells release lactate dehydrogenase (LDH), which is a biomarker used to measure the degree of hemolysis.

Hemolysis causes a range of symptoms including fatigue, shortness of breath, and life-threatening blood clots. Inhibition of C5, a protein involved in complement system activation, is an established treatment approach to prevent intravascular hemolysis, which occurs inside blood vessels; LDH can be used to determine the effectiveness of C5 inhibition. Addressing intravascular hemolysis is a critical treatment approach to reducing the symptoms and risk of life-threatening complications of PNH.

Pozelimab and cemdisiran are being evaluated in separate Phase 3 trials for several complement-mediated disorders, including PNH, myasthenia gravis (MG) and geographic atrophy (GA).

The pozelimab and cemdisiran combination is being developed under an agreement with Alnylam Pharmaceuticals, Inc.

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