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Batoclimab, an FcRn inhibitor, shows promise in reducing pathogenic antibodies associated with thyroid eye disease (TED), revealing potential therapeutic benefits and safety in clinical trials.

The following is a summary of “Proof-of-concept and Randomized, Placebo-controlled Trials of an FcRn Inhibitor, Batoclimab, for Thyroid Eye Disease,” published in the December 2023 issue of Endocrinology by Kahaly, et al.

The pathogenesis of thyroid eye disease (TED) involves the role of pathogenic thyrotropin receptor antibodies (TSH-R-Ab) that significantly affect the condition. Inhibiting the neonatal fragment crystallizable receptor (FcRn) has shown potential in reducing these antibodies responsible for TED’s pathological progression. For a study, researchers sought to present the inaugural clinical investigations of an FcRn inhibitor named batoclimab in patients with TED.

The research comprises proof-of-concept (POC) and randomized, double-blind placebo-controlled trials. Enrolled participants were individuals experiencing moderate-to-severe active TED. In the POC trial, the patients received batoclimab through weekly subcutaneous injections of 680 mg for the initial 2 weeks, succeeded by 340 mg for the ensuing 4 weeks. For the double-blind trial, participants were assigned randomly to receive weekly doses of batoclimab (either 680 mg, 340 mg, or 255 mg) or a placebo over a span of 12 weeks. The primary endpoints were variations from the baseline in serum anti-TSH-R-Ab and total IgG (in the POC trial) and a 12-week proptosis response (in the randomized trial).

The randomized trial was prematurely halted due to an unexpected surge in serum cholesterol levels, leading to data analysis from 65 out of the intended 77 patients. Both trials highlighted significant reductions in detrimental anti-TSH-R-Ab and total IgG serum concentrations (P < .001) upon batoclimab administration. Although no statistically significant differences emerged between batoclimab and the placebo concerning the proptosis response at the 12-week mark, noteworthy variations were evident at earlier stages. Moreover, there was a notable reduction in orbital muscle volume (P < .03) by the 12th week and an enhancement in the quality of life, specifically in the appearance domain (P < .03) by the 19th week within the 680-mg dosage group. Batoclimab demonstrated satisfactory tolerance levels, although some participants experienced temporary albumin reductions and lipid elevations, which reverted upon treatment cessation.

The outcomes shed light on the therapeutic potential and safety profile of batoclimab, advocating for its continued exploration as a viable treatment option for individuals suffering from TED.

Source: academic.oup.com/jcem/article/108/12/3122/7214053