aTyr Pharma Provides Update on Timing of Regulatory Response for Planned Phase 3 Study of Efzofitimod in Pulmonary Sarcoidosis
aTyr Pharma (Nasdaq: ATYR) updated the expected timing for U.S. FDA feedback on the protocol for its planned Phase 3 study of efzofitimod in chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease.
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Rhea-AI Summary
aTyr Pharma (Nasdaq: ATYR) updated the expected timing for U.S. FDA feedback on the protocol for its planned Phase 3 study of efzofitimod in chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease. Based on FDA feedback, the company anticipates a regulatory response by mid-September 2026.
The planned 54-week, global, randomized, double-blind, placebo-controlled trial is expected to enroll up to approximately 372 patients on stable low-dose corticosteroids and/or background immunosuppressants. Patients will receive either 5.0 mg/kg efzofitimod or placebo intravenously every three weeks. The primary endpoint is change from baseline in forced vital capacity at week 48, with a key secondary endpoint of change in King’s Sarcoidosis Questionnaire-Lung score at week 48.
Details
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On Sep 2, the first trading day after this news, ATYR closed 4.72% above the previous close. Our momentum scanner recorded 4 alerts for this stock that day. Relative volume reached 3.4x the daily average during tracking.
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Key Figures
- FDA response timing
- mid-September 2026
- Response on submitted Phase 3 protocol
- Study duration
- 54 weeks
- Planned Phase 3 pulmonary sarcoidosis study
- Efzofitimod dose
- 5.0 mg/kg
- Intravenous dosing every 3 weeks
- Total doses
- 17 doses
- Planned 54-week study
- Planned enrollment
- up to approximately 372 patients
- Patients with symptomatic pulmonary sarcoidosis and restrictive lung disease
- Background corticosteroid limit
- ≤ 5.0 mg daily
- Stable oral corticosteroid dose permitted in the study
- Primary endpoint timing
- week 48
- Change from baseline in forced vital capacity
Previous Clinical trial Reports
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Subgroup analysis reported FVC and patient-reported outcome improvements.
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Prior study missed its primary endpoint despite secondary endpoint improvements.
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EFZO-FIT missed its primary endpoint for oral corticosteroid reduction.
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The last patient visit was completed in the global EFZO-FIT study.
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Interim analysis reported clinically important improvement in three of four treated patients.
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Key Terms
pulmonary sarcoidosis medical
restrictive lung disease medical
forced vital capacity medical
double-blind technical
placebo-controlled technical
immunomodulator medical
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Company anticipates response from FDA by mid-September 2026 on protocol submitted for planned Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis with restrictive lung disease.
SAN DIEGO, Sept. 01, 2026 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: ATYR) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today provided an update on the expected timing of a response from the U.S. Food and Drug Administration (FDA) related to the protocol the Company submitted in June 2026 for a planned Phase 3 study of efzofitimod in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease. Based on feedback from the FDA, the Company anticipates receiving a response by mid-September 2026.
The planned Phase 3 trial is expected to be a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with moderate to severe pulmonary sarcoidosis. The 54-week study will consist of two parallel cohorts randomized equally to either 5.0 mg/kg efzofitimod or placebo dosed intravenously once every 3 weeks for a total of 17 doses. The study is intended to enroll up to approximately 372 patients with symptomatic pulmonary sarcoidosis with restrictive lung disease who are receiving a stable dose of ≤ 5.0 mg daily oral corticosteroid and/or a background immunosuppressant. All background treatment will remain stable throughout the duration of the study. The primary endpoint of the study will be change from baseline in forced vital capacity at week 48 and the key secondary endpoint will be change from baseline in the King’s Sarcoidosis Questionnaire-Lung score at week 48.
About Efzofitimod
Efzofitimod is a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. Efzofitimod is currently being investigated in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD, and aTyr recently submitted a protocol to the FDA for a global Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.
About aTyr
aTyr is a clinical stage biotechnology company leveraging evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. tRNA synthetases are ancient, essential proteins that have evolved novel domains that regulate diverse pathways extracellularly in humans. aTyr’s discovery platform is focused on unlocking hidden therapeutic intervention points by uncovering signaling pathways driven by its proprietary library of domains derived from all 20 tRNA synthetases. aTyr’s lead therapeutic candidate is efzofitimod, a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease, a group of immune-mediated disorders that can cause inflammation and progressive fibrosis, or scarring, of the lungs. For more information, please visit www.atyrpharma.com.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are usually identified by the use of words such as "anticipate," “believes,” “can,” “could,” “designed,” “expects,” “intends,” “may,” “plans,” “potential,” “upcoming,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by such safe harbor provisions for forward-looking statements and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements include, among others, statements regarding our continued development of efzofitimod in pulmonary sarcoidosis; timelines and plans with respect to certain regulatory and development milestones, activities and goals, including the potential receipt of a response from the FDA by mid-September 2026 on a protocol submitted to the FDA in June 2026 for a planned Phase 3 study of efzofitimod in pulmonary sarcoidosis; the proposed design of our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, including the dosing regimen, enrollment expectations, targeted endpoints, and strategy to focus on a more limited patient population; and our interpretation of the results of the Phase 3 EFZO-FIT™ study and the meaning of those interpretations for our planned Phase 3 study. These forward-looking statements also reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects, as reflected in or suggested by these forward-looking statements, are reasonable, we can give no assurance that the plans, intentions, expectations, strategies or prospects will be attained or achieved. All forward-looking statements are based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. Furthermore, actual results may differ materially from those described in these forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, uncertainty related to interactions with the FDA in general, uncertainty regarding geopolitical and macroeconomic events, risks associated with the discovery, development and regulation of efzofitimod, the risks associated with targeting a more limited patient population in our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, the risk that we or future partners may cease or delay preclinical or clinical development activities for efzofitimod for a variety of reasons (including difficulties or delays in patient enrollment in planned clinical trials), the possibility that future collaborations could be terminated early, and the risk that we may not be able to raise the additional funding required for our business and product development plans, as well as those risks set forth in our most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q and in our other SEC filings. Except as required by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
Contact:
Ashlee Dunston
Sr. Director, Investor Relations and Public Affairs
adunston@atyrpharma.com
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