ARGX - argenx Highlights Breadth of Autoimmune Pipeline with New Multifocal Motor Neuropathy Data at 2024 Peripheral Nerve Society Annual Meeting | Benzinga
ARDA study data show potential for empasiprubart to drive functional improvement and reduced risk of relapse for multifocal motor neuropathy (MMN) patients
ADHERE+ data show durability of functional improvements with VYVGART® Hytrulo (efgartigimod alfa and hyaluronidase-qvfc), which is FDA approved for use in adults with chronic inflammatory demyelinating polyneuropathy (CIDP)
June 25, 2024 – 4:30pm EDT
Amsterdam, the Netherlands – argenx SE ((Euronext &, NASDAQ:ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced that new data from across the company's autoimmune pipeline were presented at the 2024 Peripheral Nerve Society (PNS) Annual Meeting in Montréal, Quebec.
"argenx is on a mission to transform the treatment of severe autoimmunity," said Luc Truyen, M.D., Ph.D., Chief Medical Officer, argenx. "We have established MMN and CIDP as autoantibody-mediated diseases, and by developing novel medicines that precisely target disease biology, we are fulfilling our mission to create truly transformative outcomes for patients. We are excited to present data for our novel therapies that may offer benefits beyond symptom management – to safely help patients regain control of their lives without harsh side effects, residual impairments, or treatments dependent on high frequency infusions. argenx continues to build scientific and clinical evidence that supports the advancement of our innovative pipeline and shows the potential to expand therapeutic choices while reducing the risk of relapse and accumulating disability."
Phase 2 ARDA Data Support Empasiprubart as Novel Targeted Treatment for MMN
For the first time at a medical congress, argenx presented Cohort 1 data from the Phase 2 ARDA study, which support proof of concept for empasiprubart as a potential new treatment option for MMN, a chronic, progressive autoimmune disease with only one approved treatment option. ARDA is the largest interventional study to-date in MMN.
- Improved function and reduced risk of IVIg retreatment: Compared with placebo, treatment with empasiprubart reduced the risk of IVIg retreatment by 91% (HR: 0.09 [95% CI: 0.02–0.44]), with improvement of grip and muscle strength, and improved patients' ability to perform daily activities.
- Favorable tolerability profile: Empasiprubart was well-tolerated and most adverse events were mild or moderate.
ADHERE Oral Presentation Highlights Consistent Sustained Efficacy and Safety of VYVGART Hytrulo for Patients with CIDP
argenx also presented new data from the pivotal ADHERE and open-label extension ADHERE+ studies evaluating VYVGART Hytrulo in patients with CIDP. The ADHERE data supported the recent FDA approval of VYVGART Hytrulo as a safe and effective new treatment option for CIDP, demonstrating sustained functional benefit across all disease scores regardless of disease stage or treatment history. ADHERE met its primary endpoint (p<0.0001) demonstrating a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo. Ninety-nine percent of trial participants elected to participate in the ADHERE+ open-label extension.
- Sustained functional improvements: Demonstrated improvements in functional ability (mean aINCAT scores) on VYVGART Hytrulo from Stage A baseline were maintained through Stage B of ADHERE and to Week 24 of ADHERE+. Mean aINCAT scores improved from Run-in Period baseline, indicating some patients gain functional benefit on VYVGART Hytrulo compared to baseline assessments while on prior treatment.
- Secondary endpoints show consistent improvements across functional and strength measures: VYVGART Hytrulo-treated patients demonstrated maintenance of functional benefit in Stage B, whereas placebo patients experienced clinically meaningful worsening across all disease scores, including aINCAT, I-RODS and grip strength
- High retention and compliance in long-term extension study: 98.9% of ADHERE+ participants demonstrated treatment compliance with VYVGART Hytrulo and 86% were still ongoing at Week 24 of the OLE.
- High retention and compliance in long-term extension study: 98.9% of ADHERE+ participants demonstrated treatment compliance with VYVGART Hytrulo and 86% were still ongoing at Week 24 of the OLE.
- Favorable safety profile: Safety profile of VYVGART Hytrulo was similar between ADHERE and ADHERE+ with no increased rate of adverse events with increased exposure.
VYVGART Hytrulo was approved on June 21, 2024, for the treatment of adult patients with CIDP by the U.S Food and Drug Administration (FDA).
About Multifocal Motor Neuropathy
Multifocal motor neuropathy (MMN) is a rare, chronic autoimmune disease of the peripheral nervous system. The disease is characterized by slowly progressive, asymmetric muscle weakness mainly of the hands, forearms and lower legs. MMN is often associated with the presence of anti-GM1 IgM autoantibodies, leading to activation of the ...