Argenx stops Sjögren’s drug trial after analysis shows ‘futility’
Monitoring committee says efgartigimod study wouldn’t meet main goal
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Argenx is stopping a global clinical trial testing its drug efgartigimod against a placebo in adults with moderate to severe Sjögren’s disease after an independent analysis deemed the study unlikely to meet its main goal.
Based on a predefined interim analysis of the trial’s data, an independent data monitoring committee — a group of experts who review data from clinical studies — recommended that the study be stopped for “futility,” the company said.
The panel concluded that efgartigimod was unlikely to be superior to the placebo at reducing disease activity after 48 weeks, or about 11 months.
While efgartigimod’s safety profile was consistent with what was already known, “we are disappointed by this outcome, most of all for people living with Sjögren’s disease, who are still waiting for treatments that fundamentally change the course of their disease,” Luc Truyen, MD, PhD, chief medical officer at Argenx, said in a company press release.
“Sjögren’s Disease is one of the most [variable] and complicated diseases in immunology,” and “we will analyze these data in depth and share what we learn with the Sjögren’s community,” Truyen said.
Treatment aimed to lower antibodies by blocking receptor protein
Sjögren’s is characterized by abnormal immune reactions against healthy tissues. While the most commonly affected tissues are the glands that produce tears and saliva, the disease can also cause damage to many other parts of the body. Sjögren’s symptoms include dry eyes, dry mouth, and fatigue.
The most common self-reactive antibodies in Sjögren’s are anti-Ro (SS-A) and anti-La (SS-B).
Efgartigimod works by blocking the neonatal Fc receptor (FcRn), a protein that normally prevents circulating antibodies from being destroyed, with the aim of lowering levels of self-reactive antibodies that drive Sjögren’s.
This strategy has been proven effective for treating other autoimmune diseases, including generalized myasthenia gravis and chronic inflammatory demyelinating polyneuropathy, for which the therapy is approved in the U.S.
The medication is available in two formulations, one administered via into-the-vein (intravenous) infusion and sold as Vyvgart, and another injected under the skin (subcutaneously) and sold as Vyvgart Hytrulo.
A previous proof-of-concept Phase 2 clinical trial, RHO (NCT05817669), tested intravenous efgartigimod against a placebo in 34 adults with moderate to severe Sjögren’s for about six months. Treatment was administered once a week.
Participants completing the trial could enroll in its open-label extension study, RHO-plus (NCT06203457), in which all received intravenous efgartigimod. Pooled data from both studies showed that the therapy reduced disease activity and led to sustained clinical responses.
Based on these promising findings, Argenx launched the Phase 3 trial, called UNITY (NCT06684847), to test efgartigimod’s subcutaneous formulation in a larger group of Sjögren’s patients.
More than 600 participants were enrolled in UNITY. All tested positive for anti-Ro or anti-SSA antibodies and had moderate to severe disease activity. They were randomly assigned to receive weekly subcutaneous injections of either efgartigimod or a placebo.
The study’s main goal was to evaluate whether the therapy outperformed the placebo at reducing disease activity, as measured by the EULAR Sjögren’s Syndrome Disease Activity Index, after 48 weeks. Data from the committee’s interim analysis now showed that this goal was unlikely to be met.
“We are grateful to the patients, families, investigators and site staff who made this study possible,” Truyen said.
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