argenx has announced the discontinuation of the Phase III UplighTED trials of efgartigimod subcutaneous (SC) (efgartigimod alfa and hyaluronidase-qvfc) in adult patients with moderate to severe thyroid eye disease (TED).
The discontinuation follows advice from an Independent Data Monitoring Committee (IDMC), which recommended halting the studies for futility after reviewing interim data from a pre-specified interim analysis.
This recommendation was based on an analysis of unblinded data from those who had completed 24 weeks in the Phase III trials.
However, the therapy demonstrated an encouraging tolerability and safety profile, and no new safety signals were observed.
The company plans to perform a comprehensive analysis of the trial data after the study closes out and the database is locked.
This analysis aims to improve understanding of the study results and identify biological insights that could inform future research in TED.
The company also noted that data from the discontinued studies will be presented at an upcoming medical meeting.
The Phase III placebo-controlled, double-masked, multi-centre, randomised trials are aimed at assessing the safety, efficacy, pharmacokinetics, tolerability, pharmacodynamics, and immunogenicity of efgartigimod PH20 SC administered via prefilled syringe in those with TED.
Participants in the studies had active, moderate-to-severe TED linked to autoimmune thyroid conditions, including Graves' disease or Hashimoto's thyroiditis.
They were randomised into a 2:1 ratio and given either the therapy or placebo PH20 SC during the double-blinded treatment phase.
The percentage of participants who were proptosis responders at week 24 is the primary goal.
Key secondary endpoints included changes in proptosis measurement in the study eye from baseline to week 24, changes in the total Graves' Orbitopathy Quality of Life (GO-QoL) score from baseline to week 24, and the percentage of subjects with resolution of diplopia during the same period.
argenx chief medical officer Luc Truyen said: “We are disappointed the studies did not meet our desired outcome, and we especially empathise 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. This approach is fundamental to our disciplined and responsible stewardship of investment and resources in our clinical development programmes.”
