empasiprubart (ARGX-117) / argenx, Broteio - LARVOL DELTA

ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod (clinicaltrials.gov) - P2 | N=52 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

Design of a Phase 2a Study to Evaluate the Safety, Efficacy, and Tolerability of Intravenous Empasiprubart as an Add-On Therapy to Intravenous Efgartigimod in Adult Participants With Generalized Myasthenia Gravis (AAN 2026) - "Results of this study will provide proof-of-concept data on empasiprubart as an add-on therapy to efgartigimod for adults with AChR-Ab+ gMG."

Clinical • P2a data • CNS Disorders • Myasthenia Gravis

Empasiprubart vs Immunoglobulin in Chronic Inflammatory Demyelinating Polyradiculoneuropathy: EMVIGORATE Phase Three Study Design (AAN 2026) - "The complement system plays a key role in promoting macrophage-mediated demyelination... This Phase 3 study will compare the efficacy and safety of empasiprubart IV and IVIg in CIDP, focusing on functional ability, muscle strength/function, gait performance, and patient-reported outcomes."

Pain

Empasiprubart Versus Placebo in Chronic Inflammatory Demyelinating Polyradiculoneuropathy: EMNERGIZE Phase Three Study Design (AAN 2026) - "This Phase 3 study will compare the efficacy and safety of empasiprubart versus placebo in patients with CIDP, focusing on functional ability, muscle strength/function, clinical status, gait impairment, and patient-reported outcomes."

Clinical • Immunology • Pain

Industry Therapeutic Update from argenx: Pushing boundaries in CIDP: Pioneering innovations for patient care (AAN 2026) - "Sponsored by argenx. Learning Objectives CIDP ITU- In this CIDP-focused symposium, we are looking to educate HCPs current and future management of CIDP.Objectives will include:-Exploring the lived experience and clinical burden of CIDP from the patient perspective, highlighting unmet needs and anchoring scientific content-Understanding the complex and heterogeneous pathophysiology of CIDP and the rationale for FcRn blockade and complement inhibition-Discussing current therapies, biomarkers, and ongoing clinical trials for efgartigimod alfa and empasiprubart in CIDP"

Clinical

ARGX-999-2-MG-20001: MP: ADAPT Forward - Master protocol of a platform study to evaluate the safety and efficacy of multiple regimens in participants with Myasthenia Gravis. nnISA1: ADAPT Forward 1 - ISA1 a study to evaluate empasiprubart IV as add-on therapy to efgartigimod IV in participants with AChR-Ab seropositive generalized myasthenia gravis with a partial clinical response to... (clinicaltrialsregister.eu) - P1/2 | N=48 | Not yet recruiting | Sponsor: Argenx

New P1/2 trial • CNS Disorders • Myasthenia Gravis

ARGX-117-2402: A study to assess the efficacy and safety of empasiprubart in adults with CIDP (clinicaltrialsregister.eu) - P2/3 | N=86 | Not yet recruiting | Sponsor: Argenx

New P2/3 trial • Pain

ARGX-117-2401: A study to assess efficacy and safety of empasiprubart versus IVIg in adults with CIDP ("emvigorate") (clinicaltrialsregister.eu) - P2/3 | N=108 | Recruiting | Sponsor: Argenx

New P2/3 trial • Pain

Development of an anti-rat complement C2 antibody that improves renal outcome in a rat kidney transplant model. (PubMed, Front Immunol) - "The anti-rat C2 antibody improved kidney function and health in the rats within the first 2 weeks post-transplantation. Our study shows the successful development of an analogue of empasiprubart that can be used in preclinical in vivo disease models and highlights the potential of C2-blocking as a therapeutic strategy for preventing delayed graft function following kidney transplantation."

Journal • Preclinical • Cardiovascular • Reperfusion Injury • Transplantation

ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod (clinicaltrials.gov) - P2 | N=70 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis (clinicaltrials.gov) - P=N/A | N=70 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Myasthenia Gravis

FcRn inhibitors: A new era in neurotherapeutics. (PubMed, Handb Clin Neurol) - "Traditional immune-based therapies for autoimmune disorders, such as steroidal and nonsteroidal immunosuppressants, intravenous and subcutaneous immunoglobulin therapies, and B cell- and complement-targeting therapies, can be associated with significant side-effects and lack of efficacy in some patients...The critical role of FcRn in the pathogenesis of autoimmune disorders makes it an ideal target for novel treatments. Here, we discuss the FcRn receptor, current treatments, and recent clinical developments of FcRn-targeting treatments for autoimmune neurologic disorders."

Journal • Review • CNS Disorders • Immunology • Myasthenia Gravis • Myositis • Oncology • Pain • CD8

Immunotherapies in chronic immune-mediated neuropathies. (PubMed, Handb Clin Neurol) - "Among the latter, efgartigimod has recently been approved for the treatment of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Also rituximab, an anti-CD20 monoclonal antibody mostly used in antimyelin-associated glycoprotein (MAG) antibody neuropathy and in autoimmune nodo-paranodopathies, has been tested both in open and double-blind studies in CIDP, whereas ARGX-117, an antibody targeting the C2 fraction of the complement, is under investigation in multifocal motor neuropathy. In anti-MAG antibody neuropathy, Bruton's tyrosine kinase inhibitors, zanubrutinib and acalabrutinib, are currently tested either as a single agent or in combination with rituximab."

Journal • Review • Complement-mediated Rare Disorders • Immunology • Pain

ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis (clinicaltrials.gov) - P=N/A | N=70 | Not yet recruiting | Sponsor: argenx

New trial • CNS Disorders • Myasthenia Gravis

ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod (clinicaltrials.gov) - P2 | N=70 | Not yet recruiting | Sponsor: argenx

New P2 trial • CNS Disorders • Myasthenia Gravis

argenx to Highlight Key Data and Breadth of Immunology Innovation at 2025 AANEM Annual Meeting and MGFA Scientific Session (GlobeNewswire) - "Results from the Phase 3 ADAPT SERON study evaluating VYVGART for the treatment of adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody negative (AChR-Ab seronegative) show clinically meaningful improvements in disease activity across all three subtypes (triple negative, MuSK, and LRP4-Ab seropositive); ADAPT Jr interim results assess age-appropriate dosing, safety, and clinical effect of VYVGART in juvenile gMG...VYVGART Hytrulo CIDP data highlight functional improvement, long-term safety, and real-world characteristics of patients, underscoring the need for early treatment with effective and well-tolerated therapies; Two Phase 3 study designs for empasiprubart in CIDP – EMVIGORATE and EMNERGIZE – demonstrate argenx’s commitment to advancing innovative therapies and transforming outcomes for patients."

Clinical data • Clinical protocol • Immunology • Myasthenia Gravis

emnergize: A Study to Assess the Efficacy and Safety of Empasiprubart in Adults With CIDP (clinicaltrials.gov) - P3 | N=160 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting

Enrollment open • Pain

empacific: A Study to Evaluate Safety and Efficacy of Empasiprubart in Adults With Dermatomyositis (clinicaltrials.gov) - P2 | N=3 | Active, not recruiting | Sponsor: argenx | Recruiting ➔ Active, not recruiting | N=42 ➔ 3 | Trial completion date: Jan 2028 ➔ Nov 2026 | Trial primary completion date: Jul 2025 ➔ Nov 2026

Enrollment change • Enrollment closed • Trial completion date • Trial primary completion date • Dermatomyositis • Immunology • Myositis

ARGX-117-2401: A Study to Assess Efficacy and Safety of Empasiprubart Versus IVIg in Adults With CIDP (clinicaltrials.gov) - P3 | N=218 | Recruiting | Sponsor: argenx | Not yet recruiting ➔ Recruiting | Trial completion date: May 2030 ➔ Sep 2030 | Initiation date: May 2025 ➔ Aug 2025 | Trial primary completion date: May 2027 ➔ Sep 2027

Enrollment open • Trial completion date • Trial initiation date • Trial primary completion date • Pain

Empasiprubart in Improving Allograft Function in Recipients of a Deceased Donor Renal Allograft at Risk for Delayed Graft Function: A Phase 2 Trial in Progress (WTC 2025) - P2 | "VARVARA is a phase 2, multicenter, randomized, double-blinded, placebo-controlled trial to examine the efficacy, safety, and tolerability of empasiprubart in improving allograft function in recipients of a deceased donor renal allograft at risk for DGF."

P2 data • Cardiovascular • Inflammation • Nephrology • Reperfusion Injury

Randomized phase I trial outcomes show safe and sustainable inhibition of classical and lectin complement pathways by empasiprubart. (PubMed, Nat Commun) - P1 | "Single and multiple ascending doses of empasiprubart are generally safe and well tolerated. Overall, our results reveal in atomic detail the mechanism of empasiprubart and demonstrate that it is a first-in-class anti-C2 therapeutic antibody for use in complement-mediated diseases."

Clinical • Journal • P1 data

emnergize: A Study to Assess the Efficacy and Safety of Empasiprubart in Adults With CIDP (clinicaltrials.gov) - P3 | N=160 | Not yet recruiting | Sponsor: argenx

New P3 trial • Pain

VARVARA: ARGX-117 in Deceased Donor Kidney Transplant Recipients at Risk for Delayed Graft Function (clinicaltrials.gov) - P2 | N=103 | Active, not recruiting | Sponsor: argenx | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2026 ➔ Jul 2026 | Trial primary completion date: Apr 2026 ➔ Nov 2025

Enrollment closed • Trial completion date • Trial primary completion date • Transplantation

Safety, tolerability, and efficacy of empasiprubart in adults with dermatomyositis (EMPACIFIC): A phase 2, randomized, double-blind, placebo-controlled, multicenter study (EULAR 2025) - P2 | "This phase 2 study will evaluate the safety, tolerability, and efficacy of empasiprubart in adults with DM."

Clinical • P2 data • Cardiovascular • Complement-mediated Rare Disorders • Dermatology • Dermatomyositis • Immunology • Myositis • Pain

ARDA+: A Clinical Trial to Investigate the Long-term Safety and Tolerability, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 in Adults With Multifocal Motor Neuropathy (clinicaltrials.gov) - P2 | N=51 | Active, not recruiting | Sponsor: argenx | Trial completion date: Sep 2026 ➔ Sep 2029 | Trial primary completion date: Sep 2026 ➔ Sep 2029

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Pain