gefurulimab (ALXN1720) / AstraZeneca - LARVOL DELTA

The Role of Complement in the Pathogenesis and Treatment of Myasthenia Gravis. (PubMed, Cells) - "This has led to the development of the first C5 inhibitors approved for myasthenia gravis with AchR antibodies: eculizumab, ravulizumab, and zilucoplan. Other clinical trials are currently in progress, investigating the potential therapeutic role of other targets, including the Factor B inhibition or hepatic synthesis of the C5 protein. Other proposed potential targets that have not yet been clinically tested are also discussed in this review article."

Journal • Review • CNS Disorders • Immunology • Myasthenia Gravis

A novel dual-target gene therapy strategy design with balanced gene expressions (ASGCT 2025) - " A series of dual-target constructs expressing Gefurulimab and sCD59 were constructed (Fig.1a)... By employing the insulator framework, we identified a vector construct capable of balancing the expression of dual-target genes. Both target gene exhibited relatively high expression levels. Such a vector design can be utilized for gene therapy of multi-target diseases, such as dry AMD."

Gene therapy • Dry Age-related Macular Degeneration • Gene Therapies • Ophthalmology

PK Study of Gefurulimab SC in Healthy Chinese Adult Participants (clinicaltrials.gov) - P1 | N=8 | Completed | Sponsor: Alexion Pharmaceuticals, Inc. | Recruiting ➔ Completed

Trial completion

PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=12 | Recruiting | Sponsor: Alexion Pharmaceuticals, Inc. | Trial primary completion date: Jul 2027 ➔ Apr 2027

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

The Phase 3 PREVAIL Study Assessing the Efficacy and Safety of Subcutaneous Gefurulimab in Adults with Generalized Myasthenia Gravis: Trial in Progress (AAN 2025) - P3 | "This study examines the potential of the C5 inhibitor gefurulimab as a new treatment for patients with AChR-Ab+ gMG that can conveniently be self-administered at home through once-weekly SC injection. Results from PREVAIL will inform the clinical development program of gefurulimab."

Clinical • P3 data • CNS Disorders • Myasthenia Gravis

Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=260 | Active, not recruiting | Sponsor: Alexion Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • CNS Disorders • Myasthenia Gravis

PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=12 | Recruiting | Sponsor: Alexion Pharmaceuticals, Inc. | Trial completion date: Oct 2029 ➔ Jun 2029

Trial completion date • CNS Disorders • Myasthenia Gravis • Pediatrics

PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=12 | Recruiting | Sponsor: Alexion Pharmaceuticals, Inc. | Not yet recruiting ➔ Recruiting | Initiation date: Mar 2025 ➔ Nov 2024 | Trial primary completion date: Apr 2027 ➔ Jul 2027

Enrollment open • Trial initiation date • Trial primary completion date • CNS Disorders • Myasthenia Gravis • Pediatrics

PK Study of Gefurulimab SC in Healthy Chinese Adult Participants (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Alexion Pharmaceuticals, Inc.

New P1 trial

A Study of Subcutaneous Gefurulimab Using Prefilled Syringe Versus Autoinjector in Healthy Adult Participants (clinicaltrials.gov) - P1 | N=175 | Completed | Sponsor: Alexion Pharmaceuticals, Inc. | Active, not recruiting ➔ Completed

Trial completion

PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=12 | Not yet recruiting | Sponsor: Alexion Pharmaceuticals, Inc.

New P3 trial • CNS Disorders • Myasthenia Gravis • Pediatrics

A Study of Subcutaneous Gefurulimab Using Prefilled Syringe Versus Autoinjector in Healthy Adult Participants (clinicaltrials.gov) - P1 | N=175 | Active, not recruiting | Sponsor: Alexion Pharmaceuticals, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Apr 2025 ➔ Oct 2024 | Trial primary completion date: Apr 2025 ➔ Sep 2024

Enrollment closed • Trial completion date • Trial primary completion date

Phase 1 Study of Gefurulimab Pharmacokinetics (PK) and Safety Following Delivery Via Autoinjector in Healthy Adults (AAN 2024) - P3 | "This study will examine the PK, PD, safety, tolerability, immunogenicity, and device performance of the gefurulimab autoinjector in healthy adults. Results may support use of the autoinjector with the goals of providing convenient administration and enhancing patient experience in individuals with gMG."

Clinical • P1 data • PK/PD data • CNS Disorders • Myasthenia Gravis • Pain

A Study of Subcutaneous Gefurulimab Using Prefilled Syringe Versus Autoinjector in Healthy Adult Participants (clinicaltrials.gov) - P1 | N=174 | Recruiting | Sponsor: Alexion Pharmaceuticals, Inc.

New P1 trial

Characterization of the bispecific VHH antibody gefurulimab (ALXN1720) targeting complement component 5, and designed for low volume subcutaneous administration. (PubMed, Mol Immunol) - "Gefurulimab also inhibits complement activity supported by the rare C5 allelic variant featuring an R885H substitution in the MG7 domain. Taken together, these data suggest that gefurulimab may be a promising candidate for the potential treatment of complement-mediated disorders."

Journal

Safety and Pharmacokinetic Study of Subcutaneous ALXN1720 in Participants With Proteinuria (clinicaltrials.gov) - P1b | N=13 | Completed | Sponsor: Alexion | Active, not recruiting ➔ Completed

Trial completion • Renal Disease

Safety and Pharmacokinetic Study of Subcutaneous ALXN1720 in Participants With Proteinuria (clinicaltrials.gov) - P1b | N=12 | Active, not recruiting | Sponsor: Alexion | Recruiting ➔ Active, not recruiting

Enrollment closed • Renal Disease

Safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of subcutaneous and intravenous ALXN1720 in healthy volunteers: a phase 1, randomized, double-blind, placebo-controlled, single and multiple ascending dose study (AAN 2023) - P1 | "ALXN1720 exposure was dose-proportional and was associated with corresponding decreases in serum free C5 concentrations. Immunogenicity was low."

Clinical • P1 data • PK/PD data

Study design and methodology of the PREVAIL trial: a phase 3, randomized, double-blind, placebo-controlled study of the safety and efficacy of subcutaneous ALXN1720 in adults with generalized myasthenia gravis (AAN 2023) - P3 | "Results PREVAIL is currently active and recruiting patients in multiple countries. Conclusions PREVAIL will assess safety and efficacy of SC ALXN1720 in patients with AChR Ab+ gMG."

Clinical • P3 data • CNS Disorders • Myasthenia Gravis

Safety and Pharmacokinetic Study of Subcutaneous ALXN1720 in Participants With Proteinuria (clinicaltrials.gov) - P1b | N=12 | Recruiting | Sponsor: Alexion Pharmaceuticals | Trial primary completion date: Jan 2023 ➔ Apr 2023

Trial primary completion date • Renal Disease

Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=254 | Recruiting | Sponsor: Alexion Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

Safety and Efficacy of ALXN1720 in Adults With Generalized Myasthenia Gravis (clinicaltrials.gov) - P3 | N=200 | Not yet recruiting | Sponsor: Alexion Pharmaceuticals

New P3 trial • CNS Disorders • Myasthenia Gravis

Safety and Pharmacokinetic Study of Subcutaneous ALXN1720 in Participants With Proteinuria (clinicaltrials.gov) - P1b | N=12 | Recruiting | Sponsor: Alexion Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Renal Disease

Safety and Pharmacokinetic Study of Subcutaneous ALXN1720 in Participants With Proteinuria (clinicaltrials.gov) - P1b | N=12 | Not yet recruiting | Sponsor: Alexion Pharmaceuticals

New P1 trial • Renal Disease

Study of Subcutaneous and Intravenous ALXN1720 With and Without rHuPH20 in Healthy Subjects (clinicaltrials.gov) - P1 | N=97 | Completed | Sponsor: Alexion Pharmaceuticals | Recruiting ➔ Completed

Trial completion