Regeneron’s PNH Study: A Promising Step in Rare Disease Treatment

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

Study Overview: Regeneron Pharmaceuticals is conducting a study titled An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria. The study aims to assess the long-term safety and effectiveness of the pozelimab and cemdisiran combination therapy in treating Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. This research is significant as it explores potential new treatment options for PNH patients.

Intervention/Treatment: The study tests a combination of two experimental drugs, pozelimab and cemdisiran. These drugs are designed to work together to treat PNH by potentially reducing the disease’s symptoms and improving patients’ quality of life.

Study Design: This is a Phase 3 interventional study with a non-randomized, parallel assignment model. The study is open-label, meaning no masking is involved, and its primary purpose is treatment-focused. Participants include those transitioning from a previous study and those with specific genetic variations.

Study Timeline: The study began on February 6, 2023, with the latest update submitted on August 18, 2025. These dates are crucial as they mark the study’s progression and ensure stakeholders are informed of its current status.

Market Implications: This study update could positively impact Regeneron’s stock performance by showcasing its commitment to innovative treatments for rare diseases. Investors may view this as a promising step, especially if the results demonstrate significant efficacy and safety. Competitors in the rare disease treatment space will be closely monitoring these developments.

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