Argenx SE announced it will present extensive clinical trial and real-world data for its FcRn-blocking therapies VYVGART® (efgartigimod alfa-fcab) and VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) at the upcoming American Academy of Neurology (AAN) Annual Meeting in San Diego from April 5-9, 2025.
The presentations will showcase what the company describes as the largest safety dataset on FcRn blocking therapies, demonstrating consistent and favorable safety profiles for both treatments across multiple autoimmune indications, including generalized myasthenia gravis (gMG) and chronic inflammatory demyelinating polyneuropathy (CIDP).
"Our goal is to help people living with rare autoimmune diseases feel and function the way they did before experiencing life with a debilitating condition," said Luc Truyen, M.D., Ph.D., Chief Medical Officer at argenx. "This year at AAN, we are sharing more evidence demonstrating the long-term benefits of VYVGART for patients living with gMG and CIDP."
Long-term Efficacy and Safety in gMG
New data from the ADAPT-NXT trial investigating alternative dosing regimens of VYVGART demonstrated sustained clinical improvements through 126 weeks of treatment. The study explored both biweekly and every three-week dosing schedules, showing that patients maintained minimal symptom expression (MSE) with consistent long-term safety.
Additionally, analyses from the ADAPT-SC+ trial showed that VYVGART Hytrulo maintained consistent safety results and sustained efficacy through nine cycles of treatment. This represents the largest long-term dataset of any FcRn blocker in gMG patients.
A comparative effectiveness study being presented at the meeting indicates that among emerging immunomodulatory therapies for gMG, Fc receptor blockers—particularly VYVGART—demonstrate a more favorable benefit-risk profile compared to other treatment options.
Advancing Treatment for CIDP Patients
For CIDP patients, interim results from the open-label extension ADHERE+ trial will be presented in an oral session. These findings build upon previous data supporting the long-term efficacy and safety of VYVGART Hytrulo, with particular emphasis on functional improvement.
The company will also present details on a Phase 4 open-label trial investigating the transition from intravenous immunoglobulin (IVIg) to VYVGART Hytrulo in CIDP patients. This study examines the effectiveness and safety of switching patients from stable IVIg doses to VYVGART Hytrulo within one week after the last IVIg dose.
Individualized Treatment Approaches
The breadth of data being presented supports argenx's strategy of offering individualized treatment approaches for patients with rare autoimmune diseases. The company's presentations will highlight how different dosing regimens can be tailored to patient needs while maintaining efficacy.
"Our breadth of data continues to support VYVGART as a leading biologic. It has a proven ability to achieve minimal symptom expression for gMG patients and reduce CIDP symptoms quickly while providing improved functional ability, all with a favorable safety profile," Dr. Truyen added.
Expanding Research Pipeline
Beyond its approved therapies, argenx will also present first-in-human data on ARGX-119, an agonist antibody targeting muscle-specific kinase (MuSK). This experimental therapy is being developed for neuromuscular diseases and represents an expansion of the company's autoimmune portfolio.
The presentations will include data on pharmacokinetics, safety, tolerability, and immunogenicity of ARGX-119, providing early insights into this novel therapeutic approach.
Clinical Impact for Healthcare Providers
For healthcare providers treating patients with gMG and CIDP, the data being presented offers valuable insights into treatment sequencing and long-term management strategies.
One poster presentation will examine changes in nonsteroidal immunosuppressive treatment usage before and after efgartigimod initiation in myasthenia gravis patients, potentially informing treatment algorithms. Another will analyze hospitalization outcomes after efgartigimod initiation, addressing important healthcare utilization metrics.
The company will also present data on COVID-19 vaccination response in participants across clinical trials investigating efgartigimod PH20 SC, addressing an important clinical consideration for immunocompromised patients.
Market Position and Future Direction
These presentations collectively reinforce argenx's leadership position in FcRn blocking therapies and its commitment to expanding treatment options for patients with rare autoimmune diseases. The company's focus on long-term data and real-world evidence supports its goal of establishing VYVGART as a foundational therapy across multiple indications.
With both intravenous and subcutaneous formulations now available, and data supporting various dosing regimens, argenx continues to build a comprehensive treatment platform that can be tailored to individual patient needs and preferences.
The AAN Annual Meeting represents a significant opportunity for argenx to engage with neurologists and researchers to advance the scientific understanding of FcRn blockade and its potential across multiple autoimmune conditions.
