Five years ago, the Dutch biotech argenx outlined plans to test its drug Vyvgart in more than a dozen autoimmune conditions. By removing the self-sabotaging antibodies believed to be responsible for many of those diseases, argenx believed that one medicine could quell many seemingly different conditions.
It’s a compelling hypothesis that’s still playing out, though not exactly as argenx hoped. Although the drug is now approved for two diseases in the US and a third condition in Japan, a string of clinical failures over the past year briefly dulled the company’s shimmer.
On Tuesday, its executives took to the stage in New York to make the case to investors that Vyvgart, and argenx, are just getting started. It unveiled promising results suggesting that Vyvgart improved symptoms in people with Sjögren’s disease. The company also said that its experimental drug empasiprubart, which inhibits part of the immune system known as the complement cascade, improved symptoms in almost all patients with multifocal motor neuropathy, or MMN.
With only about 30 people in each study, the Phase 2 trials were too small to determine if the effects were statistically significant. But argenx said it plans to start larger Phase 3 trials for both conditions later this year.
Both studies are emblematic of the company’s plans for growth. At the event, CEO Tim Van Hauwermeiren laid out ambitious goals for the next five years that would continue to fulfill the promise of Vyvgart and try to replicate its success with other drugs.
By 2030, Van Hauwermeiren expects argenx will have five different drugs in Phase 3 trials, a number that he acknowledged is bigger than the company’s current pipeline would suggest. He wants Vyvgart or other drugs approved for 10 diseases, up from three now. And he plans to have more than 50,000 patients on argenx drugs each year — five times the number on Vyvgart today.
“This is the company we’re going to build,” Van Hauwermeiren told investors. “It will be a continuum of innovation, from early-stage pipeline, to late-stage pipeline, to commercial.”
So far, investors seem to be buying his pitch. After the shares $ARGX slumped at the very end of 2023, they’ve bounced back and were trading up 1.9% on Tuesday.
Expanding to new diseases
On Tuesday, argenx emphasized that developing drugs with “pipeline-in-a-product” potential is a key part of its strategy. The company highlighted early work on a compound called ARGX-119 that it plans to test in at least three nervous system diseases, including ALS. Its scientists also showcased work on a successor to Vyvgart, which Van Hauwermeiren hinted would be important to maintain sales of the drug class once Vyvgart’s patent expires.
So far, most tests of Vyvgart have been in rare diseases. The upcoming studies in Sjögren’s and MMN will be important inflection points — Sjögren’s is a comparatively common autoimmune condition, and argenx estimates there are 330,000 people with the disease and 100,000 with moderate-to-severe cases.
In the company’s Phase 2 study, 45% of patients responded to Vyvgart, compared with 11% of those who got a placebo, in a common workup of the disease that measures symptoms like salivary and tear gland function.
Last month, Johnson & Johnson unveiled promising Phase 2 data of its own FcRN inhibitor — the same class as Vyvgart — in Sjögren’s. The condition is quickly shaping up to become a major competitive area in the immunology field, something that even surprises doctors who specialize in treating the condition.
“It’s amazing that we have a number of really big trials from several different pharma companies that have demonstrated improvement in systemic disease. Would I predicted that five years ago? Probably not,” Simon Bowman, a rheumatologist at the University of Birmingham said during a panel at the investor event.
And argenx’s early data in MMN are the vanguard for a potential empasiprubart franchise. On Tuesday, argenx said its drug reduced the need for standard intravenous immunoglobulin treatments in 94% of patients in one cohort of the study and 83% of patients in another.
It’s already being tested to improve the function of kidney transplants, and a proof-of-concept study in a muscle weakness condition called dermatomyositis will begin soon.
“Empasiprubart is our next pipeline-in-a-product asset, as we have currently selected four indications and much more to come in the future,” argenx research fellow Inge Van de Walle said at the event.
The fourth indication is for a condition called chronic inflammatory demyelinating polyneuropathy, or CIDP, which Vyvgart was recently approved for. Vyvgart helps improve some, but not all, patients with the disease, suggesting that there may be multiple causes of the disease, and multiple ways to treat it.