aTyr Pharma (ATYR) Study Result summary

Study design and methodology

• Phase 3 EFZO-FIT global trial evaluated efzofitimod at 3 mg/kg and 5 mg/kg versus placebo in 268 patients with moderate to severe pulmonary sarcoidosis across the US, Europe, Japan, and Brazil over 48 or 52 weeks.

• Patients were randomized 1:1:1 to receive monthly intravenous doses for 12 months, with all participants on stable oral corticosteroids for at least 6 months.

• Steroid tapering was protocol-guided, using Patient Global Assessment and investigator input every two weeks to direct dose adjustments.

• The primary endpoint was change from baseline in mean daily oral corticosteroid dose at week 48; secondary endpoints included KSQ Lung Score, FVC, and complete steroid withdrawal.

• Baseline demographics and disease characteristics were balanced across treatment groups.

Efficacy results

• The primary endpoint was not met due to a higher than expected placebo response; mean daily OCS dose reduced to 2.79 mg for 5 mg/kg efzofitimod vs 3.52 mg for placebo (p=0.3313).

• 52.6% of patients on 5 mg/kg efzofitimod achieved complete steroid withdrawal at week 48, versus 40.2% on placebo (p=0.0919).

• KSQ Lung Score improved by 10.36 points for 5 mg/kg efzofitimod, 7.33 for 3 mg/kg, and 6.19 for placebo (p=0.0479 for 5 mg/kg vs placebo).

• 29.5% of patients on 5 mg/kg efzofitimod were both steroid-free and had ≥8-point KSQ Lung improvement, versus 14.4% for placebo (p=0.0199).

• Lung function (FVC) was maintained across all groups at week 48.

Safety and tolerability

• Efzofitimod was well tolerated at both 3 mg/kg and 5 mg/kg doses, with a safety profile consistent with previous trials.

• Adverse events were mostly mild to moderate and balanced or generally unrelated to the study drug across groups.

• Serious adverse events and discontinuations were limited and similar between groups.

• Incidence of antidrug antibodies was small and similar across groups.

• The drug demonstrated a favorable safety profile compared to standard treatments like prednisone and methotrexate.