aTyr Pharma says FDA aligned on 372‑patient sarcoidosis trial
The new Phase 3 study shifts its primary measure to lung function after an earlier miss, but aTyr says it needs more money to run it.
Adrian Kessler
A planned 372-patient Phase 3 trial puts aTyr Pharma, Inc. NASDAQATYR back on a path to test efzofitimod in pulmonary sarcoidosis after the company reported Thursday that the U.S. Food and Drug Administration aligned with its protocol. The 54-week study shifts to lung function as its main measure, but aTyr says it needs more capital to carry it out.
The Sept. 24 release calls for a randomized comparison of efzofitimod and placebo, with change in forced vital capacity, a measure of lung function, as the primary endpoint at week 48. Change in the King's Sarcoidosis Questionnaire-Lung score at week 48 is the key secondary endpoint, and aTyr expects to begin study-related activities in the fourth quarter of 2026.
aTyr is a San Diego clinical-stage developer of biologic therapies for inflammatory and fibrotic lung diseases.
Shares closed Wednesday at US$0.38 on the Nasdaq at 4:00:01 p.m. EDT, according to the NASDAQATYR quote. That price predates the Thursday morning announcement and does not show a trading reaction to it.
Lung Function Becomes Primary Endpoint
Patients with restrictive lung disease on stable background treatment would be split evenly between intravenous efzofitimod at 5.0 mg/kg and placebo, dosed every three weeks for 17 doses. The protocol covers people taking no more than 5.0 mg of daily oral corticosteroid and/or an immunosuppressant, with background treatment held steady.
The earlier EFZO-FIT Phase 3 study used steroid reduction as its main measure and missed that endpoint in 268 patients, aTyr reported on Sept. 15, 2025. Participants received one of two efzofitimod doses or placebo. At week 48, the higher-dose group's mean daily steroid dose fell to 2.79 mg against 3.52 mg for placebo, with p=0.3313.
Across that full study, predicted forced vital capacity fell 1.81 percentage points in the higher-dose arm and 2.11 points on placebo, with p=0.7875. aTyr described both active doses as generally well tolerated. In a post hoc subgroup with restrictive lung disease, aTyr reported an FVC benefit against placebo but gave no numerical effect size for that subgroup in its Sept. 24 release.
The FDA alignment is a company-reported agreement on the new protocol. It is not an approval decision, and the new trial still has to produce evidence on efficacy and safety.
The company reported US$58.9 million in cash, restricted cash and investments at June 30, 2026. Its Aug. 7 forecast put the runway for current operations into late 2028; the planned Phase 3 trial requires financing beyond that plan.
“Future development of efzofitimod in the planned Phase 3 study in pulmonary sarcoidosis will require the Company to obtain additional capital through equity or debt offerings, grant funding, collaborations, strategic partnerships and/or licensing arrangements,” aTyr said in its Sept. 24 release.
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Respiratory Peers Face Different Tests
Roivant Sciences Ltd. NASDAQROIV stopped developing namilumab for pulmonary sarcoidosis after its 107-patient Phase 2 study missed its primary endpoint, the company said on Dec. 3, 2024. Gossamer Bio, Inc. NASDAQGOSS submitted a seralutinib application to the FDA in September 2026 for pulmonary arterial hypertension, a different lung disease; its Sept. 23 release says the agency must still decide whether to accept the application for filing.
Read more: Gossamer Bio submits seralutinib NDA after Phase 3 endpoint miss
The SPDR S&P Biotech ETF stood at US$155.35 at 12:20:02 p.m. EDT Wednesday, according to its NYSEARCAXBI quote. That sector price also predates aTyr's news.
aTyr plans to initiate study-related activities in the fourth quarter of 2026, with U.S. and European regulatory submissions its stated focus.
