Efzofitimod, a biologic immunomodulator, reduces steroid dependency and improves lung function in pulmonary sarcoidosis, offering hope for a novel treatment.

In an early phase 1b/2a multiple ascending dose study the biologic efzofitimod significantly reduced steroid dependency and improved lung function in patients with pulmonary sarcoidosis. This breakthrough offers hope for a novel steroid-sparing agent with potential benefits for patients suffering from this rare condition. These findings were presented at the 2023 ERS International Congress.

Efzofitimod, a groundbreaking biologic immunomodulator, has recently undergone evaluation in a phase 1b/2a multiple ascending dose study aimed at addressing the needs of patients suffering from chronic pulmonary sarcoidosis. This debilitating condition often requires high doses of steroids for symptom management. The study’s objective was to determine whether efzofitimod could reduce the steroid dose while maintaining symptom control and lung function.

The current study involved 37 patients who were randomly assigned to receive treatment with efzofitimod or placebo. The placebo and subtherapeutic efzofitimod arm (1 mg/kg) from the previous study were pooled. Patients received six intravenous doses over a 24-week period, with the goal of tapering oral steroid use to 5 mg/day by week 8. Three main endpoints were investigated: steroid tapering, lung function improvement, and patient-reported outcomes (PROs). Inclusion criteria encompassed patients aged 18-75, weight between 40 kg and 160 kg, documented sarcoidosis, lung involvement, specific symptom severity, and a willingness to attempt steroid taper.

The data revealed compelling evidence of efzofitimod’s efficacy. Notably, 7.7% of patients in the therapeutic group relapsed, compared with 54.4% in the placebo/subtherapeutic group. This marked difference demonstrates efzofitimod’s potential to reduce steroid dependency (P=0.017).

Moreover, lung function, as measured by FVC, significantly improved in the therapeutic group (P=0.035). PROs, assessed through King’s Sarcoidosis Questionnaire—Lung (KSQ-L) scores, also favored the therapeutic group. Over half (52.9%) of the patients in this group exhibited a significant and clinically relevant increase (≥12), three times the minimal clinically important differences (MCID), compared with only 15.0% in the subtherapeutic group (P=0.032).

Composite endpoints combining steroid tapering without worsening in FVC or PROs further supported efzofitimod’s potential benefits. Adverse events were also monitored closely, with notable mentions including the development of antibodies, infusion-related reactions, and newly occurring malignancies.

This study presents compelling evidence of efzofitimod’s effectiveness in reducing steroid dependency and improving lung function and PROs in patients with pulmonary sarcoidosis. These findings provide proof-of-concept and helped lay the groundwork for the phase 3 EFZO-FIT Study, which will aim to confirm and expand upon these promising results.

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