Patients with restrictive lung disease demonstrate clinically meaningful benefit in FVC and improvements in multiple PROs for 5.0 mg/kg efzofitimod compared to placebo.
Company submitted protocol to FDA in June 2026 for planned Phase 3 study of efzofitimod in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease.
SAN DIEGO, July 15, 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 the Company will present a subgroup analysis of the Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease, at the World Association of Sarcoidosis and Other Granulomatous Disorders (WASOG) 2026 Congress, which is scheduled to take place July 15 – 17, 2026, in Porto, Portugal.
“This subgroup analysis of patients from EFZO-FIT™ with restrictive lung disease presents clear evidence that those treated with efzofitimod experienced a clinically meaningful benefit in lung function and positive trends of improvement in multiple patient-reported outcomes (PROs) while removing steroids,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr Pharma. “Following our recent interactions with the U.S. Food and Drug Administration (FDA), last month we submitted a protocol for a planned Phase 3 study in pulmonary sarcoidosis patients with restrictive lung disease (defined as forced vital capacity, or FVC, percent predicted ≤ 80% with a normal FEV1/FVC ratio), utilizing FVC as the primary endpoint of the study and the King’s Sarcoidosis Questionnaire (KSQ)-Lung score as the key secondary endpoint. We believe the approach of investigating efzofitimod in this defined patient population where FVC is the relevant measure of lung function is a well-informed plan to demonstrate the potential for efzofitimod to improve the lives of these patients.”
Details of the poster presentation appear below. The poster will be available on the aTyr website once presented.
Title: Evaluating Efzofitimod in a Subset of Sarcoidosis with the Restrictive Phenotype
Authors: Vis Niranjan, Pavithra Ramesh, Sanjay Shukla, Nelson Kinnersley, Daniel A. Culver. RxMD, aTyr Pharma, Octa Consulting Services, Cleveland Clinic.
Poster Number: PO123
Session: Controversies in Sarcoidosis Treatment and Disease Progression
Date and Time: Thursday, July 16, 2026, at 1:15pm WEST
Location: Porto, Portugal
EFZO-FIT™ included 264 pulmonary sarcoidosis patients with all lung phenotypes who were enrolled and treated (intent-to-treat, or ITT). The post hoc analysis included a subset of 44 patients with prespecified restrictive lung disease (FVCpp ≤ 80%, FEV1/FVC ≥ 0.7). The change from baseline at week 48 for FVC and PROs was analyzed using a random coefficient regression model (RCRM) to supplement the mixed model for repeated measures (MMRM) analysis performed for the ITT data. The baseline characteristics were generally balanced in the restrictive patients compared to the ITT population, however mean FVC was expectedly lower. Overall, the magnitude of steroid reduction in the 5.0 mg/kg efzofitimod arm was similar to placebo. In the 5.0 mg/kg efzofitimod arm, the placebo adjusted week 48 change from baseline for FVC by RCRM was 123.8 ml. The placebo adjusted week 48 change from baseline by RCRM showed improvement for the 5.0 mg/kg efzofitimod arm versus placebo for KSQ-Lung, KSQ-General Health, Fatigue Assessment Scale, and the Leicester Cough Questionnaire. Furthermore, efzofitimod was generally well tolerated in the restrictive subgroup, similar to the ITT. The findings suggest that further investigation of efzofitimod in patients with restrictive lung disease is warranted.
About the EFZO-FIT™ study
EFZO-FIT™ was a global Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis with all lung phenotypes. The 52-week study consisted 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 (264 enrolled and treated) with pulmonary sarcoidosis at multiple centers in the United States, Europe, Japan and Brazil. The trial design incorporated a forced steroid taper. The primary endpoint of the study was steroid reduction at week 48. Secondary endpoints included measures of sarcoidosis symptoms and lung function at week 48.
About Pulmonary Sarcoidosis
Pulmonary sarcoidosis is an inflammatory disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body. Approximately 160,000 Americans are diagnosed with pulmonary sarcoidosis and the prognosis ranges from benign and self-limiting to chronic, debilitating disease, permanent loss of lung function and death. Current treatment options include corticosteroids and other immunosuppressive therapies, which have limited efficacy and are associated with serious side-effects that many patients cannot tolerate long-term.
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, the potential therapeutic benefits and applications of efzofitimod, our timelines and plans with respect to certain development activities and goals, the proposed design of our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis, including the success of the targeted endpoints and strategy to focus on a more limited patient population in demonstrating the potential for efzofitimod to improve lives, and our interpretation of the results of the 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 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 or 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 DunstonSr. Director, Investor Relations and Public [email protected]