Approval Probability
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Pozelimab · 10 trials · 7 indications
An SAE is any untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect. * Is an important medical event
An AESI (serious or non-serious) is one of scientific and medical concern specific to the sponsor's product or program, for which ongoing monitoring and rapid communication by the Investigator to the sponsor can be appropriate. Such an event might warrant further investigation in order to characterize and understand it
Any untoward medical occurrence in a patient administered a study drug which may or may not have a causal relationship with the study drug.
Any untoward medical occurrence in a patient administered a study drug which may or may not have a causal relationship with the study drug.
Open Label Treatment Period (OLTP)
Normalization of serum albumin was defined as serum albumin within the normal range at least 70 percent (%) of measurements between weeks 12 and 24, and no single albumin measurement of \<2.5 grams per deciliter (g/dL) between weeks 12 and 24, and no requirement for albumin infusion between weeks 12 and 24. Improvement in the following 4 prespecified clinical outcomes that were evaluable for improvement at baseline, without worsening of the others: Daily bowel movement frequency, the presence and severity of facial edema (physician-reported), the presence and severity of peripheral edema (physician-reported), and the participant/caregiver assessment of frequency of problematic abdominal pain. Percentage of participants with active disease at baseline who achieved normalization of serum albumin and improvement in prespecified clinical outcomes at Week 24 were reported.
Part A
Part B
Part A
| Arm | Type | Description |
|---|---|---|
| Pozelimab + Cemdisiran Combo | EXPERIMENTAL | - |
| Pozelimab + Cemdisiran treatment group | EXPERIMENTAL | Randomized 1:1:1 |
| Cemdisiran monotherapy treatment group | EXPERIMENTAL | Randomized 1:1:1 |
| Placebo treatment group | PLACEBO_COMPARATOR | Randomized 1:1:1 |
| PNH Transition Patients | EXPERIMENTAL | Patients with PNH who completed treatment/ protocol requirements (as applicable) in the parent study (R3918-PNH-2021 \[NCT05133531\]) |
| C5 Polymorphism Patients | EXPERIMENTAL | Patients who have not been treated in either parent study but who have a documented complement component 5 (C5) variation rendering them refractory to eculizumab/ravulizumab. Note: Loading dose of pozelimab administered IV on Day 1. |
| Pozelimab Q4W + Cemdisiran | EXPERIMENTAL | - |
| Pozelimab Q2W + Cemdisiran | EXPERIMENTAL | - |
| Pozelimab+Cemdisiran | EXPERIMENTAL | - |
| Active PLE | EXPERIMENTAL | Patients aged 1 year and older with a clinical diagnosis of CD55-deficient PLE disease |
| Part A | EXPERIMENTAL | - |
| Part B | EXPERIMENTAL | - |
| Cohort 1 | EXPERIMENTAL | Pozelimab: Single-dose SC on day 1 |
| Cohort 2 | EXPERIMENTAL | Pozelimab: Single-dose IV on day 1 |
| Cohort 3 | EXPERIMENTAL | Pozelimab: Single-dose SC on day 29 Cemdisiran: Single-dose SC on day 1 |
| Cohort 4 | EXPERIMENTAL | Pozelimab: Single-dose SC on day 1 Cemdisiran: Single-dose SC on day 1 |
| Cohort 5 | EXPERIMENTAL | Optional Pozelimab: Single-dose SC on day 1 or day 29 Cemdisiran: Single-dose SC on day 1 |
| Cohort 6 | EXPERIMENTAL | Pozelimab: Single-dose IV on day 1 |
| Process A | EXPERIMENTAL | Randomized 1:1 |
| Process B | EXPERIMENTAL | Randomized 1:1 |
| Name | Type | Description |
|---|---|---|
| Pozelimab | DRUG | Administered per the protocol |
| Cemdisiran | DRUG | Administered per the protocol |
| Placebo | DRUG | SC injection |
Key Inclusion Criteria: 1. Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing 2. Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months 3. LDH persistently \> 1.5 × Upper Limit of Normal (ULN) in th...
Pozelimab is an investigational C5 inhibitor being studied for several conditions, including sporadic Inclusion Body Myositis, Generalized Myasthenia Gravis, Paroxysmal Nocturnal Hemoglobinuria, and Age-related Macular Degeneration (AMD), specifically Geographic Atrophy. It is also being evaluated in healthy volunteers for safety and tolerability studies.
Pozelimab targets complement component C5, acting as an inhibitor. By binding to C5, it is designed to block the complement cascade, which is involved in inflammatory and tissue-damaging processes. This mechanism is being explored across multiple disease areas, including complement-mediated disorders and geographic atrophy.
Pozelimab is developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker REGN. Regeneron is conducting clinical trials to evaluate the safety and efficacy of Pozelimab, both as monotherapy and in combination with cemdisiran, across various indications.
Pozelimab is in Phase 3 clinical development for Geographic Atrophy associated with Age-related Macular Degeneration. It has also completed Phase 1 trials in healthy volunteers. The drug is investigational and has not been approved by regulatory authorities; it continues to be evaluated in clinical studies.
Pozelimab is being studied in several clinical trials. NCT06541704 is a Phase 3 trial in Geographic Atrophy, recruiting 975 participants. NCT07230834 is a Phase 1 intravitreal trial for Geographic Atrophy. Completed Phase 1 trials include NCT04601844 and NCT04940364, both in healthy volunteers.
Pozelimab is often studied in combination with cemdisiran, and the combination is referred to as Pozelimab/Cemdisiran. While Pozelimab alone targets C5, cemdisiran is a separate investigational agent. The combination is being evaluated in clinical trials for conditions like Geographic Atrophy and Paroxysmal Nocturnal Hemoglobinuria.