Regulatory Update Provides More Clarity on Efzofitimod in Pulmonary Sarcoidosis

研报英文原文证据摘录

Regulatory Update Provides More Clarity on Efzofitimod in Pulmonary Sarcoidosis

C O M PA N Y N O T E

M a y 1 1 , 2 0 2 6

...benefit among pulmonary sarcoidosis patients with a non-restrictive phenotype, but it showed

a clinically meaningful benefit among those with a restrictive phenotype (+124 mL at Day

365). Hence, this validates selection of this pulmonary sarcoidosis subpopulation for C-006 in

order to maximize efzofitimod's benefit. Moreover, Exhibit 2 reviews the proposed inclusion/

exclusion criteria in C-006 in order to target pulmonary sarcoidosis patients with a restrictive lung

phenotype on stable background treatment.

Exhibit 1 - Clinically Meaningful FVC Benefit was Observed in Patients with Restrictive

Lung Disease in Ph3 EFZO-FIT

Source: ATYR Company Materials

Exhibit 2 - Efzofitimod's Ph3 Proposed Inclusion and Exclusion Criteria

Details on efzofitimod's optimized Ph3 (C-006) trial design based on FDA feedback.

Accordingly, ATYR reviewed the proposed trial design for efzofitimod's additonal Ph3 that

incorporates key takeaways from FDA engagement. Specifically, key optimizations when

compared to Ph3 EFZO-FIT include (Exhibit 3): 1) primary endpoint is FVC at Week 48 (vs

steroid reduction at Week 48 in Ph3 EFZO-FIT); 2) enrollment of pulmonary sarcoidosis patients

with a restrictive lung phenotype and stable background treatment (vs all lung phenotypes with

forced steroid taper in Ph3 EFZO-FIT); and 3) 5.0 mg/kg Q3W dosing (vs 5.0 mg/kg Q4W

in Ph3 EFZO-FIT). With this, Exhibit 4 details the trial design schematic of a 48-week (up-

to 54-week) Ph3 assessing efzofitimod vs placebo in n=~372 moderate-to-severe pulmonary

sarcoidosis patients with restrictive disease and primary endpoint of change from baseline in

FVC (mL) at Week 48.