Argenx discontinues phase 3 UplighTED trials of efgartigimod SC in thyroid eye disease

Argenx SE has discontinued the phase 3 UplighTED studies evaluating efgartigimod subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) in adults with moderate to severe thyroid eye disease (TED).

According to the company, the trial is being discontinued based on the recommendation from an Independent Data Monitoring Committee (IDMC) to stop the trials for “futility” following its review of data from a pre-specified interim analysis. However, the company did note efgartigimod showed a favorable safety and tolerability profile, and no new safety signals were identified.

UplighTED was a randomized, double-masked, placebo-controlled, multicenter trial designed to evaluate the efficacy, safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of efgartigimod PH20 SC administered by prefilled syringe in adult participants with TED. Patients enrolled in the trial have active, moderate-to-severe thyroid eye disease associated with autoimmune thyroid conditions (Graves' disease or Hashimoto's thyroiditis).

In the trial, patients were randomized 2:1 to receive efgartigimod PH20 SC or placebo PH20 SC during the double-blinded treatment period. The primary endpoint for the trial was the percentage of participants who were proptosis responders at week 24.

The IDMC conducted a futility evaluation on unblinded data from patients completing 24 weeks in the phase 3 trial. Following closeout and database lock, the company plans to conduct a comprehensive analysis of the data to “enhance understanding of the studies’ outcomes and uncover key biological insights that may inform future research in TED.”

Luc Truyen, MD, PhD, CMO at argenx, commented on the decision in a press release from the company, saying, “We are disappointed the studies did not meet our desired outcome, and we especially empathize with patients who are living with TED and seeking new therapies for this challenging disease. We had pre-planned this futility analysis, as it provides a meaningful interim evaluation of observed patient outcomes and enables us to responsibly evaluate the study’s future likelihood of success.”

The company plans to share data from the discontinued trial at future medical meetings.

References:

1. argenx Provides Update on UplighTED Studies of Efgartigimod SC in Thyroid Eye Disease. Published December 15, 2025. Accessed December 15, 2025. https://argenx.com/news/2025/press-release-3205093.html