argenx Showcases Autoimmune Pipeline with New MMN Data at 2024 PNS Annual Meeting
June 25, 2024 – Amsterdam, the Netherlands – argenx SE, a global immunology company, has released new data from its autoimmune pipeline presented at the 2024 Peripheral Nerve Society (PNS) Annual Meeting in Montréal, Quebec. The findings highlight potential advancements in the treatment of multifocal motor neuropathy (MMN) and chronic inflammatory demyelinating polyneuropathy (CIDP).
According to Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx, the company is dedicated to transforming the treatment landscape for severe autoimmunity. By targeting disease biology with novel medicines, argenx aims to offer benefits that extend beyond symptom management. The recent data underscore the potential of argenx’s therapies to help patients regain control of their lives without harsh side effects or frequent infusions.
Phase 2 ARDA Study
The Phase 2 ARDA study is a major interventional trial for MMN, a progressive autoimmune disease with limited treatment options. Empasiprubart, a new treatment under investigation, demonstrated significant promise.
Improved Function and Reduced Risk of IVIg Retreatment: Empasiprubart reduced the risk of IVIg retreatment by 91% compared to placebo, with enhanced grip and muscle strength, and improved daily activity performance.
Tolerability: The treatment was well-tolerated, with most adverse events being mild or moderate.
ADHERE and ADHERE+ Studies
New data from the ADHERE and its extension ADHERE+ studies regarding VYVGART Hytrulo for CIDP were also presented. These studies supported the recent FDA approval of VYVGART Hytrulo as a new treatment for CIDP, showcasing its sustained efficacy and safety.
Sustained Functional Improvements: Patients treated with VYVGART Hytrulo maintained functional improvements throughout the study duration. The mean scores on key functional measures such as aINCAT remained stable or improved, indicating sustained benefits from the treatment.
Secondary Endpoints: Consistent improvement across various functional and strength measures was observed. Patients treated with VYVGART Hytrulo maintained functional benefits, whereas placebo-treated patients experienced clinical deterioration.
High Retention and Compliance: The ADHERE+ study showed a high retention rate with 86% of participants continuing treatment at Week 24, and 98.9% demonstrating compliance.
Safety Profile: The safety profile of VYVGART Hytrulo was consistent between the ADHERE and ADHERE+ studies, without an increased rate of adverse events over time.
About Multifocal Motor Neuropathy (MMN)
MMN is a rare chronic autoimmune disease affecting the peripheral nervous system, characterized by progressive muscle weakness predominantly in the hands, forearms, and lower legs. High-dose IVIg is the only approved treatment, yet disease progression remains a challenge, underlining the need for new therapeutic options.
About Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
CIDP is a severe autoimmune condition of the peripheral nervous system, leading to muscle weakness, fatigue, and sensory loss in the limbs. Without treatment, it can severely limit one’s ability to perform daily activities, with many requiring mobility aids like wheelchairs.
Phase 2 ARDA Study Design
The Phase 2 ARDA study is a randomized, double-blind, placebo-controlled trial evaluating the safety, efficacy, and tolerability of empasiprubart in MMN patients. The study involves a pre-randomization IVIg dependency period, followed by a 16-week double-blind treatment phase. Safety and tolerability are the primary endpoints, with additional measures including time to IVIg retreatment and various clinical efficacy scores.
ADHERE Trial Design
The ADHERE trial is a comprehensive study assessing VYVGART Hytrulo for CIDP. It involved a run-in period followed by a randomized, placebo-controlled phase. The primary endpoint was the time to first relapse, with patients showing clinical improvement on various scales advancing to the randomized phase.
About Empasiprubart and VYVGART Hytrulo
Empasiprubart is a humanized monoclonal antibody targeting C2 to inhibit specific complement pathways, potentially applicable across multiple autoimmune indications. VYVGART Hytrulo, combining efgartigimod alfa with hyaluronidase for subcutaneous administration, reduces circulating IgG levels and is FDA-approved for CIDP treatment.
Argenx continues to build a robust pipeline of innovative therapies aimed at severe autoimmune diseases, showcasing commitment to improving patient outcomes through scientific and clinical excellence.
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