aTyr Pharma Announces Last Patient Visit in Phase 3 EFZO-FIT™ Study of Efzofitimod in Patients with Pulmonary Sarcoidosis
Rhea-AI Summary
aTyr Pharma (NASDAQ:ATYR) has announced the completion of the last patient visit in its Phase 3 EFZO-FIT™ study for efzofitimod in pulmonary sarcoidosis patients. The study, which is the first-ever global Phase 3 and largest interventional sarcoidosis trial, enrolled 268 subjects across multiple centers in the US, Europe, Japan, and Brazil.
The 52-week study evaluates two dosing regimens (3.0 mg/kg and 5.0 mg/kg) against placebo, administered monthly for 12 doses. The trial's primary endpoint focuses on steroid reduction, with secondary endpoints measuring sarcoidosis symptoms and lung function. Topline results are expected in Q3 2025.
Efzofitimod, a first-in-class biologic immunomodulator, targets interstitial lung disease by selectively modulating activated myeloid cells through neuropilin-2, potentially offering a safer alternative to current steroid-based treatments.
Positive
- First-ever global Phase 3 and largest interventional study for sarcoidosis treatment
- Novel mechanism of action that may reduce steroid burden without immune suppression
- Broad international study coverage across US, Europe, Japan, and Brazil
- Significant enrollment of 268 subjects completed
Negative
- Results not yet available - success of the treatment still uncertain
- Complex 52-week study duration may impact patient retention and data quality
News Market Reaction
On the day this news was published, ATYR declined 8.23%, reflecting a notable negative market reaction. Argus tracked a peak move of +137.5% during that session. Argus tracked a trough of -5.0% from its starting point during tracking. Our momentum scanner triggered 36 alerts that day, indicating elevated trading interest and price volatility. This price movement removed approximately $57M from the company's valuation, bringing the market cap to $632M at that time.
Data tracked by StockTitan Argus on the day of publication.
Topline results expected in the third quarter of 2025.
SAN DIEGO, July 22, 2025 (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 the last patient has completed their last visit in the Company’s Phase 3 EFZO-FIT™ study of its lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis, a major form of interstitial lung disease. The Company expects to report topline results from this study in the third quarter of 2025.
“Completing this first-ever global Phase 3 and largest interventional study conducted for patients with sarcoidosis to date is a major accomplishment for aTyr and the broader sarcoidosis community,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “Current treatment options for sarcoidosis are limited and rely heavily on the use of oral corticosteroids, which often have significant side effects. This study provides an opportunity to evaluate a treatment such as efzofitimod that may have the potential to reduce steroid burden and improve quality of life in patients with this chronic disease. We thank the patients, investigators, patient advocacy organizations and our partner Kyorin Pharmaceutical Co., Ltd. for helping to make this study possible, and we look forward to sharing topline results later this quarter.”
About the EFZO-FIT™ Study
EFZO-FIT™ is a global Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. The 52-week study consists of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously once a month for a total of 12 doses. The study enrolled 268 subjects with pulmonary sarcoidosis at multiple centers in the United States, Europe, Japan and Brazil. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction. Secondary endpoints include measures of sarcoidosis symptoms and lung function. More information is available at www.clinicaltrials.gov (NCT05415137).
About Efzofitimod
Efzofitimod is a first-in-class 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. aTyr is currently investigating efzofitimod in the global Phase 3 EFZO-FIT™ study in patients with pulmonary sarcoidosis, a major form of ILD, and in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related 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 first-in-class 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,” “designed,” “could,” “can,” “expects,” “intends,” “may,” “plans,” “potential,” “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 potential therapeutic benefits and applications of efzofitimod; timelines and plans with respect to certain development activities (such as the timing of receipt of topline data) of our Phase 3 EFZO-FIT™ study; and certain development goals. 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 regarding geopolitical and macroeconomic events, risks associated with the discovery, development and regulation of efzofitimod, the risks associated with clinical trials generally, the risk that we or our 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 existing 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
When will aTyr Pharma (ATYR) release the Phase 3 EFZO-FIT trial results?
What is the primary endpoint of aTyr's Phase 3 EFZO-FIT trial?
How many patients were enrolled in aTyr's Phase 3 EFZO-FIT study?
What dosing regimens are being tested in aTyr's Phase 3 EFZO-FIT trial?
How does aTyr's efzofitimod work in treating pulmonary sarcoidosis?
