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Regeneron’s New Study on PNH Treatment: A Potential Game-Changer?

Regeneron’s New Study on PNH Treatment: A Potential Game-Changer?

Regeneron Pharmaceuticals ((REGN)) announced an update on their ongoing clinical study.

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Regeneron Pharmaceuticals is conducting a study titled A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy. The study aims to assess the effectiveness and safety of a combination of two experimental drugs, pozelimab and cemdisiran, in treating Paroxysmal Nocturnal Hemoglobinuria (PNH) in patients who have not responded well to existing C5 inhibitors.

The intervention being tested involves the administration of two drugs, pozelimab and cemdisiran, which are designed to reduce hemolysis in PNH patients. These drugs are administered according to the study protocol.

This is an interventional Phase 3 study with a single-group assignment. There is no masking involved, meaning all participants and researchers know which treatment is being administered. The primary purpose of the study is treatment-focused.

The study is not yet recruiting, with the first submission date on August 13, 2025, and the latest update on August 26, 2025. These dates are crucial as they mark the beginning of the study’s timeline and the most recent information available.

The potential market implications of this study are significant for Regeneron Pharmaceuticals. If successful, the combination therapy could enhance the company’s portfolio and influence its stock performance positively. It could also impact investor sentiment, especially in comparison to competitors offering alternative C5 inhibitors.

The study is ongoing, and further details are available on the ClinicalTrials portal.

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