Veopoz (pozelimab-bbfg) / Regeneron, Alnylam, Sanofi - LARVOL DELTA

Targeted complement inhibition with pozelimab in children with CD55 deficiency. (PubMed, BMJ Case Rep) - "At 3-month follow-up, both children remained in clinical remission without further need for albumin or immunoglobulin infusions. These cases highlight the importance of considering genetic causes in children with refractory PLE and demonstrate the effectiveness of early targeted complement inhibition in CHAPLE syndrome."

Journal • Pediatrics • CD55

ACCESS-EXT: A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works (clinicaltrials.gov) - P3 | N=202 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: Feb 2029 ➔ Oct 2028 | Trial primary completion date: Feb 2029 ➔ Oct 2028

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

SIENNA: A Study Investigating Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Adult Participants With Geographic Atrophy (clinicaltrials.gov) - P3 | N=975 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: May 2032 ➔ Apr 2033 | Trial primary completion date: Nov 2027 ➔ Jul 2028

Trial completion date • Trial primary completion date • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Macular Degeneration • Ophthalmology • Retinal Disorders

A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (clinicaltrials.gov) - P3; N=210; Not yet recruiting; Sponsor: Regeneron Pharmaceuticals

Clinical • Combination therapy • New P3 trial • CNS Disorders • Myasthenia Gravis

NIMBLE: A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=235 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: May 2027 ➔ Mar 2028 | Trial primary completion date: Aug 2024 ➔ Aug 2025

Combination therapy • Trial completion date • Trial primary completion date • CNS Disorders • Myasthenia Gravis

A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=210 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Not yet recruiting ➔ Recruiting | Trial primary completion date: Feb 2024 ➔ Aug 2024

Combination therapy • Enrollment open • Trial primary completion date • CNS Disorders • Myasthenia Gravis

NIMBLE: A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=335 | Recruiting | Sponsor: Regeneron Pharmaceuticals | N=235 ➔ 335 | Trial completion date: Mar 2028 ➔ Mar 2029 | Trial primary completion date: May 2025 ➔ May 2026

Enrollment change • Monotherapy • Trial completion date • Trial primary completion date • CNS Disorders • Myasthenia Gravis

NIMBLE: A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=235 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial primary completion date: Aug 2025 ➔ May 2025

Combination therapy • Monotherapy • Trial primary completion date • CNS Disorders • Myasthenia Gravis

NIMBLE: A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=335 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial primary completion date: May 2026 ➔ Jul 2025

Monotherapy • Trial primary completion date • CNS Disorders • Myasthenia Gravis

NIMBLE: A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=288 | Active, not recruiting | Sponsor: Regeneron Pharmaceuticals | Recruiting ➔ Active, not recruiting | Trial completion date: Mar 2029 ➔ Nov 2028

Enrollment closed • Monotherapy • Trial completion date • CNS Disorders • Myasthenia Gravis

An si-RNA‑Based Complement Pathway Inhibitor for gMG: Efficacy, Safety, and Impact on Quality of Life/MGC from the NIMBLE Trial (ICNMD 2026) - P3 | "Cemdisiran, both as monotherapy and in combination with pozelimab, significantly improves clinical signs and symptoms in people with gMG and was generally well-tolerated. Quarterly subcutaneous administration of cemdisiran provides effective symptom relief and enhanced quality of life, with partial complement inhibition."

Clinical • HEOR • CNS Disorders • Infectious Disease • Meningococcal Infections • Myasthenia Gravis • Pneumonia • Respiratory Diseases • Septic Shock

Efficacy and cumulative safety of cemdisiran in adults with generalised myasthenia gravis (gMG): results from the phase 3 NIMBLE trial (EAN 2026) - "The phase 3 NIMBLE trial evaluated subcutaneous cemdisiran (siRNA inhibiting hepatic C5 production) and subcutaneous pozelimab (monoclonal antibody targeting C5) as monotherapies or in combination in patients with gMG. Quarterly, subcutaneous cemdisiran was generally well-tolerated and achieved significant improvement in symptoms with partial complement inhibition among patients with gMG."

Clinical • P3 data • CNS Disorders • Infectious Disease • Meningococcal Infections • Myasthenia Gravis • Pneumonia • Respiratory Diseases • Septic Shock

COMPARISONS OF TERMINAL COMPLEMENT INHIBITION WITH POZELIMAB + CEMDISIRAN VERSUS RAVULIZUMAB IN PARTICIPANTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA: PHARMACODYNAMIC RESULTS FROM COHORT A (EHA 2026) - "Summary/Conclusion Combination therapy produced more rapid and durable effects across CH50 and AH50 versus ravulizumab in participants with PNH. These data indicate earlier onset and superior complement pathway control with the combination therapy compared to ravulizumab, supporting its potential to provide uninterrupted terminal complement activity suppression over 26 weeks."

PK/PD data • Cardiovascular • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Thrombosis

A novel systemic approach to geographic atrophy with pozelimab in combination with cemdisiran or cemdisiran alone: The phase 3 SIENNA study design (ARVO 2026) - P3 | "Completion of enrollment in cohort A is expected in Q1 2026, and initial results are expected by the end of 2026. Conclusions SIENNA will provide important information on the efficacy, safety, and tolerability of a systemic approach for complete versus partial inhibition of C5 for GA treatment."

Combination therapy • P3 data • Age-related Macular Degeneration • Dry Age-related Macular Degeneration • Ophthalmology

A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently (clinicaltrials.gov) - P3 | N=35 | Recruiting | Sponsor: Regeneron Pharmaceuticals

Trial initiation date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

ACCESS-1: A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment (clinicaltrials.gov) - P3 | N=202 | Active, not recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: Jan 2027 ➔ Oct 2026 | Trial primary completion date: Jan 2027 ➔ Oct 2026

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Rapid and Sustained Efficacy of Subcutaneously Administered Cemdisiran Without Complete Complement Blockade in Patients with Generalized Myasthenia Gravis (gMG): Primary Efficacy and Safety Results from the Phase Three NIMBLE Trial (AAN 2026) - P3 | "Objective:Report on phase 3 NIMBLE trial (NCT05070858) comparing subcutaneous cemdisiran or pozelimab plus cemdisiran with placebo in patients with gMG.Background:gMG is characterized by complement activation at the neuromuscular junction. Although both arms demonstrated rapid and sustained improvements versus placebo and were generally well‑tolerated in patients with gMG, quarterly SC dosing of cemdisiran achieved efficacious outcomes without complete complement blockage."

Clinical • CNS Disorders • Infectious Disease • Meningococcal Infections • Myasthenia Gravis • Pneumonia • Respiratory Diseases • Septic Shock • LRP4

Efficacy and safety of cemdisiran siRNA in myasthenia gravis (NIMBLE): a double-blind, randomised, placebo-controlled, phase 3 trial. (PubMed, Lancet) - P3 | "Cemdisiran monotherapy and combination therapy were effective in the treatment of generalised myasthenia gravis, and were generally well tolerated. Subcutaneous dosing of cemdisiran, administered every 3 months, could provide a convenient treatment approach for generalised myasthenia gravis."

Journal • P3 data • CNS Disorders • Infectious Disease • Meningococcal Infections • Myasthenia Gravis • Respiratory Diseases

R3918-AMD-2501: Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants (clinicaltrialsregister.eu) - P1 | N=16 | Not yet recruiting | Sponsor: Regeneron Pharmaceuticals Inc.

New P1 trial • Age-related Macular Degeneration • Dry Age-related Macular Degeneration

Evaluating pozelimab in the treatment of CHAPLE disease. (PubMed, Immunotherapy) - "Eculizumab, a monoclonal antibody against complement component C5, initially demonstrated clinical benefit when administered on a compassionate-use basis. Pozelimab effectively inhibits terminal complement activation, leading to sustained remission of PLE, obviating the need for albumin replacement, reducing hospitalization rates, improving symptom control and nutritional status, and ultimately enhancing overall quality of life. This review highlights the evolving role of pozelimab in CHAPLE disease by discussing its mechanistic basis, emerging clinical evidence, and implications for patient-centered care."

Journal • Review • Cardiovascular • Hematological Disorders • Infectious Disease • Thrombosis • CD55

ACCESS-1: A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment (clinicaltrials.gov) - P3 | N=190 | Active, not recruiting | Sponsor: Regeneron Pharmaceuticals | Recruiting ➔ Active, not recruiting

Enrollment closed • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

R3918-PNH-2483: A Study to Evaluate how Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) whose Current Treatment is not Working Efficiently (clinicaltrialsregister.eu) - P2/3 | N=8 | Not yet recruiting | Sponsor: Regeneron Pharmaceuticals Inc.

New P2/3 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Study to Test the Safety of Pozelimab in Pediatric Participants 1 to 5 Years of Age With a Rare Disease Called CHAPLE (Complement Hyperactivation, Angiopathic Thrombosis, Protein-losing Enteropathy) Disease (clinicaltrials.gov) - P4 | N=5 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Trial completion date: Aug 2029 ➔ Jan 2030 | Trial primary completion date: Aug 2029 ➔ Jan 2030

Trial completion date • Trial primary completion date • Cardiovascular • Hematological Disorders • Pediatrics • Rare Diseases • Thrombosis • CD55

A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently (clinicaltrials.gov) - P3 | N=35 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

VIENNA: Intravitreal (IVT) Pozelimab for Geographic Atrophy (GA) in Adult Participants (clinicaltrials.gov) - P1 | N=54 | Recruiting | Sponsor: Regeneron Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Age-related Macular Degeneration • Dry Age-related Macular Degeneration