aTyr Pharma Announces Alignment with FDA on Phase 3 Study of Efzofitimod in Pulmonary Sarcoidosis
Further development requires additional capital, which may come from financing, grants, partnerships or licensing.
Rhea-AI Summary
aTyr Pharma (ATYR) announced FDA alignment on its planned Phase 3 efzofitimod study protocol for pulmonary sarcoidosis on September 24, 2026.
The global, randomized, placebo-controlled study is designed to enroll up to approximately 372 patients with symptomatic pulmonary sarcoidosis and restrictive lung disease. Over 54 weeks, participants will receive either 5.0 mg/kg efzofitimod or placebo intravenously every three weeks for 17 doses. The primary endpoint is change in forced vital capacity, a measure of lung function, at week 48. A lung-symptom questionnaire score is a secondary endpoint at week 48.
In a subgroup analysis of the earlier Phase 3 EFZO-FIT study, patients with restrictive lung disease who received 5.0 mg/kg efzofitimod showed improvements in forced vital capacity and patient-reported outcomes compared with placebo. aTyr expects to begin study-related activities in the fourth quarter of 2026, focusing on U.S. and European regulatory submissions. Further development will require additional capital.
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News Explained
As of
Sources and calculations
- aTyr Pharma FDA alignment announcement (2026-09-24)
- aTyr 2026 second-quarter fundamentals (2026Q2)
- Available liquidity against the last reported quarterly operating outflow, in days at that rate ($16,160,000 + $41,258,000) / ($9,351,000 / 91) = 558.8 days
Details
Market move: ATYR +88.86% vs previous close. Phase 3 FDA alignment
On Sep 24, the day this news came out, the latest delayed price for ATYR is 88.86% above the previous close. Argus tracked a peak move of +82.6% during the session. Our momentum scanner has recorded 49 alerts for this stock so far that day. The latest delayed price is $0.71. Relative volume is exceptionally heavy at 19.1x the average.
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Key Figures
- Planned study enrollment
- Up to approximately 372 patients
- Planned Phase 3 study
- Study duration
- 54 weeks
- Planned Phase 3 study
- Efzofitimod dose
- 5.0 mg/kg
- Intravenous dosing in one study cohort
- Dosing schedule
- Once every 3 weeks for a total of 17 doses
- Efzofitimod cohort
- Primary endpoint timing
- Week 48
- Change from baseline in FVC
- Key secondary endpoint timing
- Week 48
- Change from baseline in KSQ-Lung score
- Study-related activities
- Fourth quarter of 2026
- Expected initiation timing
Previous Clinical trial Reports
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Updated expected timing for FDA protocol feedback to mid-September 2026.
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Reported subgroup FVC and patient-reported outcome benefits; submitted proposed Phase 3 protocol.
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EFZO-FIT missed its primary steroid-dose endpoint; company planned FDA engagement on next steps.
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Key Terms
fvc medical
double-blind medical
placebo-controlled medical
corticosteroid medical
immunosuppressant medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
Global Phase 3 study will evaluate efzofitimod in patients with pulmonary sarcoidosis with restrictive lung disease utilizing FVC as primary endpoint.
Company expects to initiate study-related activities in the fourth quarter of 2026.
SAN DIEGO, Sept. 24, 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 announced that it has reached alignment with the U.S. Food and Drug Administration (FDA) on the protocol for a Phase 3 study of its lead therapeutic candidate, efzofitimod, in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease. The Company expects to focus on regulatory submissions in the U.S. and Europe as it initiates study-related activities in the fourth quarter of 2026.
“We received feedback earlier this week from the FDA and we are very pleased to have reached alignment on the protocol for our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr Pharma. “This important milestone reflects the progress of our efforts to advance efzofitimod for patients with pulmonary sarcoidosis. With limited treatment options available, particularly for patients requiring chronic therapy, we believe efzofitimod has the potential to become an important new treatment option.”
The planned Phase 3 trial will 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 (FVC) at week 48 and the key secondary endpoint will be change from baseline in the King’s Sarcoidosis Questionnaire (KSQ)-Lung score at week 48.
The study design is supported by data from a subgroup analysis of the Phase 3 EFZO-FIT™ study that showed that patients with pulmonary sarcoidosis with restrictive lung disease (defined as FVC percent predicted ≤
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.
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 plans to initiate study-related activities for a global Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD, in the fourth quarter of 2026. 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, including the additional capital that we expect to be necessary for future development; timelines and plans with respect to certain regulatory and development milestones, activities and goals, including our plans to initiate study-related activities in the fourth quarter of 2026 for the planned Phase 3 study; 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, risks that the results from the planned Phase 3 study may not ultimately support FDA approval of efzofitimod in pulmonary sarcoidosis, 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
FAQ
AI-generated questions and answers. How Rhea-AI works. Not financial advice.
What will aTyr Pharma measure in its planned Phase 3 efzofitimod study?
The primary endpoint is change from baseline in forced vital capacity at week 48. The key secondary endpoint is change from baseline in the King’s Sarcoidosis Questionnaire-Lung score at week 48.
Who can enter aTyr Pharma’s planned Phase 3 efzofitimod study?
The study is intended to enroll up to approximately 372 patients with symptomatic pulmonary sarcoidosis and restrictive lung disease. Eligible patients are receiving a stable dose of ≤ 5.0 mg daily oral corticosteroid and/or a background immunosuppressant. Background treatment will remain stable throughout the study.
How does aTyr Pharma plan to fund further efzofitimod development?
Further development in the planned Phase 3 study will require additional capital. The potential sources identified are equity or debt offerings, grants, collaborations, partnerships and licensing arrangements.