Nipocalimab

As of early May, there were more than 10,000 patients on Vyvgart around the world. As argenx homes in on the next indication for its pipeline-in-a-product Vyvgart (efgartigimod), the company is preparing for the next leg of its commercial journey. Next month, on July 16, argenx will host an R&D day, during which the company will unveil its new program dubbed Vision 2030: Taking Breakthrough Science to 50,000 patients. As part of Vision 2030, argenx will touch on its continuing commitment to tackle severe autoimmune diseases with Vyvgart, which won its first approval in generalized myasthenia gravis (gMG) in late 2021. The company also plans to highlight its next potential commercial product empasiprubart—an antibody designed to inhibit the C2 protein that’s in early testing for multifocal motor neuropathy (MMN) and other diseases—as well as its broader antibody-based pipeline. With Vyvgart in particular, argenx aims to highlight its plans to broaden the drug’s foothold in gMG, its next potential nod in chronic inflammatory demyelinating polyneuropathy (CIDP), and the company’s wider plans to move into a new wave of indications. Additionally, argenx says it plans to “invest in its organic innovation engine to bring forward new first-in-class pipeline candidates.” "argenx today is better positioned than ever before to deliver on our commitment to transform the autoimmunity treatment landscape for patients,” Tim Van Hauwermeiren, CEO of argenx, said in a statement. “We have grown from our R&D roots over the last five years into a true global innovator, pioneering novel targets into a robust pipeline and delivering safe and effective first-in-class medicines to more than 10,000 patients around the world.” Taking a closer look at the company’s efgartigimod plans, argenx will talk up the potential to expand Vyvgart in MG through studies including the seronegative form of the disease. Roughly 15% of generalized MG patients are estimated to be seronegative, which could provide a meaningful addition to the drug’s current green light, Van Hauwermeiren told Fierce during a recent interview. Aside from myasthenia gravis, argenx will also tout results from its phase 2/3 Vyvgart study in immune-mediated myopathies, plus topline phase 2 data in Sjogren’s disease. Over the weekend, Johnson & Johnson presented positive results on its antibody nipocalimab in patients with primary Sjogren’s disease. As a member of the same class of FcRn receptor-targeting drugs that Vyvgart belongs to, nipocalimab’s trial results “do appear to support the FcRN antagonism approach in [Sjogren’s] and argenx’s decision to advance efgartigimod into a Phase III trial based on an analysis of the results from the RHO study,” William Blair’s Matt Phipps and Myles Minter wrote in a note to clients Monday. Argenx will also flaunt its next potential “pipeline-in-a-product” opportunity with empasiprubart, highlighting phase 2 data in MMN, as well as unveiling a preview of phase 2 studies in delayed graft function and dermatomyositis and introducing a fourth indication. Lastly, argenx will go over how it plans to scale its global commercial footprint to support Vision 2030. As of early May, there were more than 10,000 patients on Vyvgart around the world. Argenx is currently marketing Vyvgart for gMG in the U.S., Canada and multiple European countries, as well as China and Japan. The company also has Vyvgart distribution deals in places like the Middle East and Eastern Europe. Argenx will not chart any organizational changes as part of its new 2030 growth plan, a company spokesperson confirmed over email.  At the same time it teased its Vision 2030, argenx also revealed that it’s stopping the development of efgartigimod in post-COVID-19-mediated postural orthostatic tachycardia syndrome (PC-POTS) after its phase 2 ALPHA study found that treated patients had no clinically meaningful improvement compared to those on placebo on a measure of symptoms. While the discontinuation may read negative, "we did not explicitly ascribe any value to this indication in our model," analysts at Leerink Partners wrote in a note to clients, noting they remain confident in Vyvgart's "strong commercial execution." The company has reason to be optimistic, too, as Vyvgart nears its next potential indication in CIDP by the end of the week. The drug, which boasts positive phase 3 data in the rare autoimmune disease, is up for an FDA decision in CIDP on June 21. That new indication could represent another "multi-billion dollar commercial opportunity," the Leerink team added.  CIDP is a disease of the peripheral nervous system that causes fatigue, muscle weakness and loss of feeling in patients’ arms and legs. That weakness can worsen over time or come and go, with the potential for symptoms to significantly impair a person’s ability to function on a day-to-day basis. If approved, Vyvgart will go up against Takeda’s subcutaneous immune globulin treatment HyQvia, which won its own CIDP nod in January.

A response to the Johnson & Johnson’s Sjögren's disease treatment was observed as early as Week 4 and continued to increase throughout the 24-week treatment period compared to the placebo group. The unveiling of Johnson & Johnson’s full data from the phase 2 trial of nipocalimab in Sjögren's disease (SjD) suggest that both the Big Pharma and its competitor argenx are on the right track by targeting FcRn antagonism at the automimmune disorder, according to analysts. J&J already revealed back in February that its anti-FcRn antibody nipocalimab had scored a win in a phase 2 study in SjD, but it was only at the European Alliance of Associations for Rheumatology (EULAR) 2024 Congress over the weekend that the company finally unveiled the data. The DAHLIAS study enrolled 163 patients with the autoimmune disease who received either a 5-mg/kg or a 15-mg/kg dose of intravenous nipocalimab every two weeks for 22 weeks, or placebo, alongside standard of care. The data demonstrated a more than 70% improvement in the primary endpoint of a measure of organ disease severity at 24 weeks for patients who received the 15-mg/kg dose compared to the placebo cohort, J&J explained in a presentation. A response to the treatment was observed as early as Week 4 and continued to increase throughout the 24-week treatment period compared to the placebo group. This follows a phase 3 win for nipocalimab in another autoimmune disorder called generalized myasthenia gravis (gMG) back in February. The 15-mg/kg cohort also hit secondary endpoints for SjD that involved organ assessments and physician reviews. Improvements were noted in SjD symptoms like dryness of the mouth, eye and vagina. Safety and tolerability were consistent with other clinical studies of nipocalimab. The 5-mg/kg dose didn’t have a similar impact, however, with J&J noting in its EULAR presentation that this patient cohort clung close to the placebo group in terms of the efficacy endpoints. “These data establish proof of concept for nipocalimab in Sjögren's disease and support further clinical development, which is welcome news for the approximately four million people worldwide living with this chronic, debilitating disease,” said the study’s investigator Jacques-Eric Gottenberg, M.D., Ph.D., of Strasbourg University Hospital’s Department of Rheumatology, in a June 15 release. J&J acquired nipocalimab in the $6.5 billion takeover of Momenta Pharmaceuticals in 2020. The deal secured J&J a challenger to now-approved drugs for gMG from argenx and UCB, and a chance to pursue the wide range of indications that could potentially respond to FcRn blockade. Argenx’s failures may have diluted the hype, which had promoted FcRn as the next TNF, but there remain potentially billions of dollars to play for. Argenx is set to roll out more detailed data from the phase 2 RHO study of its approved anti- FcRn antibody Vyvgart in SjD in July. In a note in response to J&J’s presentation, analysts at William Blair said the opportunity for Vyvgart in SjD will depend on the design of the subsequent phase 3 trial. “Overall, while there was not much of a dose-response on efficacy outcomes, the high dose of nipocalimab did show a rapid separation from placebo in the primary endpoint of ClinESSDAI (within four weeks) that aligned with the decrease in total IgG and anti-Ro60 and anti-La IgG autoantibodies,” the analysts said in their assessment of J&J’s data. “While the variability and placebo response rates in SjD do leave some level of risk in future phase 3 trials, the DAHLIAS results do appear to support the FcRN antagonism approach in SjD and argenx’s decision to advance efgartigimod into a phase 3 trial based on an analysis of the results from the RHO study.”