What Is Pozelimab?
Pozelimab is an investigational medication currently being studied in clinical trials. It is a type of drug that is administered as a subcutaneous (SC) injection. While the specific mechanism of action is not detailed in the available trial descriptions, it is being investigated for its potential therapeutic effects in various conditions. Pozelimab is being studied both as a standalone treatment and in combination with another investigational drug, Cemdisiran.
Clinical trials for Pozelimab began in 2019, with the latest trial starting in 2025. A total of 18 trials involving 1,903 participants have been conducted or are ongoing. These trials are exploring Pozelimab's use in conditions such as Paroxysmal Nocturnal Hemoglobinuria, Geographic Atrophy, and various neuromuscular and inflammatory disorders.
Uses and Conditions Under Study
Pozelimab is currently under investigation for several conditions, with clinical trials sponsored primarily by Regeneron Pharmaceuticals. The drug is being studied for its potential to address a range of rare and chronic diseases.
- Blood Disorders: Pozelimab is being investigated for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare, acquired, life-threatening blood disorder characterized by the destruction of red blood cells. This drug is being studied for its potential to manage symptoms or progression of PNH in 7 trials.
- Gastrointestinal Disorders: The medication is also being explored for CD55-deficient Protein-losing Enteropathy (CHAPLE), a rare genetic disorder that causes severe gastrointestinal issues and protein loss. Pozelimab is being studied for this condition in 3 trials.
- Eye Conditions: Pozelimab is under investigation for Age-related Macular Degeneration (AMD) and Geographic Atrophy (GA), both progressive eye diseases that can lead to vision loss. These conditions are being studied across 3 trials to assess the drug's potential to slow disease progression.
- Neuromuscular and Inflammatory Conditions: Several trials are exploring Pozelimab for Generalized Myasthenia Gravis, a chronic autoimmune neuromuscular disease, as well as Idiopathic Inflammatory Myopathies and Sporadic Inclusion Body Myositis (sIBM), which are inflammatory muscle disorders. These conditions are being studied in 3 trials.
- Healthy Volunteers: Pozelimab has also been administered to healthy volunteers in 3 trials. These studies typically aim to understand the drug's safety, how it is absorbed, distributed, metabolized, and excreted by the body (pharmacokinetics), and how it affects the body (pharmacodynamics) in individuals without the target disease.
Dosing
Pozelimab is administered as a subcutaneous (SC) injection. The specific dosage forms studied include "INJECTION" and combinations with Cemdisiran, such as "Pozelimab + Cemdisiran Combo" and "Pozelimab and Cemdisiran."
In some studies, participants received Pozelimab 200mg/Cemdisiran 200 mg via subcutaneous injections. The frequency of administration has varied across trials, with some protocols specifying injections "every 4 weeks" (Pozelimab Q4W + Cemdisiran) and others "every 2 weeks" (Pozelimab Q2W + Cemdisiran). Various treatment groups, such as "PNH Transition Patients" and "C5 Polymorphism Patients," have been studied to assess the drug's effects in specific patient populations. The exact dosing regimen and duration of treatment are determined by the specific clinical trial protocol.
Side Effects
In clinical trials, specific individual side effects for Pozelimab were not consistently reported with frequencies across all studies. However, overall rates of treatment-emergent adverse events (TEAEs) were documented.
In an open-label study (NCT04209634) involving patients with CD55-deficient protein-losing enteropathy (CHAPLE disease), all 10 participants experienced at least one TEAE. Of these, 2 participants experienced mild TEAEs, 5 experienced moderate TEAEs, and 3 experienced severe TEAEs. No participants developed anti-drug antibodies to Pozelimab in this study.
For patients with paroxysmal nocturnal hemoglobinuria (PNH) receiving combination treatment with Pozelimab and Cemdisiran in an open-label study (NCT04811716):
- 66.7% of participants receiving Pozelimab every 2 weeks (Q2W) plus Cemdisiran experienced TEAEs. These included 16.7% mild and 16.7% moderate events.
- 41.7% of participants receiving Pozelimab every 4 weeks (Q4W) plus Cemdisiran experienced
Currently Recruiting Trials
Pozelimab is currently being investigated in several clinical trials for various conditions, often in combination with another experimental drug, cemdisiran. These studies aim to understand its safety and effectiveness for patients who might benefit from this treatment.
One Phase 3 study, NCT07154745, is evaluating a combination of pozelimab and cemdisiran in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) whose current treatment is not working efficiently. This study aims to find a better treatment approach for PNH and plans to enroll 35 participants.
For pediatric patients, a Phase 4 study, NCT07142343, is focusing on the safety of pozelimab in children aged 1 to 5 years with CHAPLE disease. This very rare hereditary condition is the focus for 5 young participants.
Adults with Geographic Atrophy (GA), a condition affecting the retina, may be eligible for a Phase 1 study, NCT07230834. This study is researching intravitreal pozelimab and is recruiting 54 participants.
Another large Phase 3 study, NCT06541704, is investigating subcutaneously administered pozelimab in combination with cemdisiran, or cemdisiran alone, for adult participants with Geographic Atrophy (GA) caused by Age-related Macular Degeneration (AMD). This study has a target enrollment of 975 participants.
An Early Phase 1 study, NCT06479863, is evaluating the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with Sporadic Inclusion Body Myositis (sIBM). This trial is recruiting 10 participants.
Finally, a long-term Phase 3 study, NCT05744921, is assessing the safety and effectiveness of the pozelimab and cemdisiran combination in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study aims to enroll 202 participants.
Where to Participate
Clinical trials for pozelimab are being conducted across a wide geographic area, with 151 sites located in 121 cities across 33 states. This broad reach aims to make participation accessible to a diverse group of patients.
Top locations for participation include:
- Austin, Texas (6 sites)
- San Antonio, Texas (4 sites)
- Dallas, Texas (4 sites)
- New York, New York (4 sites)
- Los Angeles, California (3 sites)
- Sacramento, California (3 sites)
- Portland, Oregon (3 sites)
- Chicago, Illinois (3 sites)
- Phoenix, Arizona (3 sites)
- Beverly Hills, California (2 sites)
Participants generally need to be between 1 and 85 years of age, and all genders are welcome. Healthy volunteers are not being recruited for these studies, as the focus is on patients with specific medical conditions, including children.
Development Timeline
The development journey for Pozelimab began on December 24, 2019, with the initiation of its first clinical trial. Since then, a total of 18 trials have been launched, aiming to enroll 1,903 participants across various phases. Regeneron Pharmaceuticals has been the primary driver of this research, sponsoring 17 of these studies, with the Austin Neuromuscular Center sponsoring one.
Pozelimab's pipeline has shown significant expansion over time. Initially, research focused on conditions such as IBS-C and hyperphosphatemia. The program quickly broadened to include a range of other conditions. Key areas of expansion have included eye conditions like Geographic Atrophy (GA) and Age-related Macular Degeneration (AMD), as well as rare diseases such as Generalized Myasthenia Gravis, Sporadic Inclusion Body Myositis (sIBM), CD55-Deficient Protein-Losing Enteropathy, and CHAPLE Disease. The latest trial is projected to conclude on November 17, 2025, marking continued progress in understanding pozelimab's potential. The trials span various stages, including 6 in Phase 3, 4 in Phase 1, and others in Phase 2, Early Phase 1, and Phase 4, reflecting a comprehensive development strategy.