aTyr Pharma Inc. (NASDAQ:ATYR) secured a formal agreement with the U.S. Food and Drug Administration (FDA) regarding the Phase 3 study protocol for its lead therapeutic candidate, efzofitimod.
Following this regulatory alignment, the company prepares to initiate study-related activities across the U.S. and Europe in the fourth quarter of 2026.
Phase 3 Trial Design And Endpoints
The upcoming Phase 3 clinical trial aims to assess both the overall safety and effectiveness of efzofitimod in individuals suffering from moderate to severe forms of pulmonary sarcoidosis.
Pulmonary sarcoidosis is an inflammatory disease that causes tiny clumps of immune cells called granulomas to form in the lungs and chest lymph nodes.
Stretching over a 54-week period, researchers plan to divide up to 372 enrolled patients into two parallel cohorts. Participants will receive either a placebo or 5.0 mg/kg of efzofitimod administered intravenously every three weeks, totaling 17 doses.
Throughout the study’s duration, patients will maintain their existing background treatments, including stable doses of daily oral corticosteroids and immunosuppressants.
Investigators will evaluate the primary endpoint by tracking the change in forced vital capacity (FVC, a measure of lung function) from the patient’s baseline at week 48.
Additionally, the key secondary endpoint will measure shifts in the King’s Sarcoidosis Questionnaire (KSQ)-Lung score at the 48-week mark.
Data Backing and Funding Needs
Subgroup data derived from the earlier Phase 3 EFZO-FIT study thoroughly support the newly aligned clinical protocol.
The data revealed that patients presenting with restrictive lung disease who received the 5.0 mg/kg dose experienced clinically meaningful improvements in both FVC and multiple patient-reported outcomes, including KSQ-Lung scores, when compared to the placebo group.
To execute the planned Phase 3 development of efzofitimod for pulmonary sarcoidosis, aTyr Pharma must secure additional capital.
In August, the company trimmed its pipeline to focus its resources on efzofitimod and targeted pipeline development to conserve capital. aTyr Pharma reduced its workforce by around 60%.
Cash, cash equivalents, restricted cash, and available-for-sale investments as of June 30 were $58.9 million, sufficient to fund the company’s current operations into late 2028.
ATYR Price Action: aTyr Pharma shares were down 6.08% at $0.35 at the time of publication on Thursday. The stock is trading near its 52-week low of $0.35, according to Benzinga Pro data.
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