aTyr Pharma announces the publication of positive data from the Phase 1b/2a clinical study of efzofitimod for the treatment of pulmonary sarcoidosis in the journal CHEST


aTyr Pharma, Inc.

First therapy in sarcoidosis to reduce steroid load while demonstrating improvements in key physiological and quality of life measures.

EFZO-FIT™ Phase 3 global pivotal study currently recruiting.

SAN DIEGO, Nov. 09, 2022 (GLOBE NEWSWIRE) — aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company committed to first-in-class drug discovery and development from its proprietary tRNA synthetase platform, announced today the publication in the journal CHEST of positive results from a randomized, double-blind, placebo-controlled Phase 1b/2a clinical trial of its lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis, a major form of interstitial lung disease. The publication, titled “Efzofitimod for the treatment of pulmonary sarcoidosis”, is available on the journal’s website and at:

“The dose-dependent improvements in several clinically relevant endpoints seen with efzofitimod compared to placebo, along with the desirable safety and tolerability profile, are very important results,” said Daniel A. Culver, DO, chairman of the Department of Pulmonary Medicine at The Cleveland Clinic and lead author of the publication. “The ongoing EFZO-FIT™ study provides the opportunity to evaluate the steroid-sparing effects of efzofitimod in a larger clinical trial. We hope this study will lead to a safe and effective treatment that will reduce steroid burden and have a major impact on the quality of life of patients with pulmonary sarcoidosis, which is badly needed.

The study demonstrated that efzofitimod was safe and well tolerated at all doses and showed consistent dose-response on key efficacy parameters and improvements over placebo, including measures of steroid reduction, lung function, measures of sarcoidosis symptoms and inflammatory biomarkers.

“The publication of this manuscript marks the first peer-reviewed publication of clinical data on efzofitimod in a major medical journal. These data indicate that efzofitimod provides substantial benefits to patients – improving lung function and symptoms of cough, shortness of breath and fatigue – while reducing their toxic steroid load,” said Sanjay S. Shukla, MD, MS, President and CEO. of Tyre. “EFZO-FIT™ is expected to be the largest interventional study for patients with sarcoidosis to date, and we believe that efzofitimod is well positioned to be the first disease-modifying therapy to be marketed for patients with this disease. debilitating disease.”

Efzofitimod is a first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states via selective modulation of neuropilin-2 (NRP2). Efzofitimod is currently being studied in a pivotal global Phase 3 study in patients with pulmonary sarcoidosis known as EFZO-FIT™, which is supported by safety and efficacy data from the phase 1b/2a study. Efzofitimod has received orphan drug and Fast Track designations from the FDA for sarcoidosis.

Phase 1b/2a clinical trial in patients with pulmonary sarcoidosis

The Phase 1b/2a study was a randomized, double-blind, placebo-controlled, multiple-escalating dose clinical trial in 37 patients with pulmonary sarcoidosis. The trial consisted of three cohorts testing doses of 1.0 mg/kg, 3.0 mg/kg and 5.0 mg/kg of efzofitimod or placebo, given intravenously every month for six months. The primary objective of the study was to evaluate the safety, tolerability, immunogenicity, and pharmacokinetic profile of multiple doses of efzofitimod versus placebo. Secondary objectives included the potential steroid-sparing effects of efzofitimod, in addition to other exploratory efficacy assessments, such as lung function.

About Pulmonary Sarcoidosis

Sarcoidosis is an immune-mediated disease characterized by the formation of granulomas, clusters of inflammatory cells, in one or more organs of the body, mainly in the lungs. Nearly 200,000 Americans live with pulmonary sarcoidosis, and the prognosis ranges from mild, self-limiting disease to chronic, debilitating disease, with 1 in 5 cases resulting in fibrosis or scarring of the lungs, leading to permanent loss of function. lung disease and in many cases death. Current treatment options include corticosteroids and other immunosuppressive therapies, which have limited efficacy and are associated with severe side effects that many patients cannot tolerate long term.

Subscriptionut Efzofitimod

aTyr is developing efzofitimod as a potential therapeutic agent for patients with fibrous lung disease. Efzofitimod, a fusion protein composed of the immunomodulatory domain of histidyl-tRNA synthetase fused to the FC region of a human antibody, is a selective neuropilin-2 modulator that downregulates the innate and adaptive immune response in inflammatory disease states. aTyr’s primary indication for efzofitimod is pulmonary sarcoidosis, a major form of interstitial lung disease. Clinical proof of concept for efzofitimod was recently established in a placebo-controlled, multiple ascending dose Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis, which demonstrated the safety and consistent dose-response and benefit trends of efzofitimod versus placebo on key efficacy endpoints including steroid reduction, lung function, clinical symptoms, and inflammatory biomarkers. aTyr is currently leading EFZO-FITa phase 3 study of efzofitimod in patients with pulmonary sarcoidosis.

Subscriptionut aTyr

aTyr is a biotherapeutics company committed to first-in-class drug discovery and development based on its proprietary tRNA synthetase platform. aTyr’s research and development efforts focus on a recently discovered area of ​​biology, the extracellular functionality and signaling pathways of tRNA synthetases. aTyr has built a global intellectual property portfolio directed towards a potential pipeline of protein compositions derived from 20 tRNA synthetase genes and their extracellular targets. aTyr is primarily focused on efzofitimod, a clinical-stage product candidate that binds to the neuropilin-2 receptor and is designed to downregulate immune engagement in fibrotic lung disease. For more information, please visit

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 generally identified by the use of words such as “anticipates”, “believes”, “estimates”, “would expects, ‘intends’, ‘may’, ‘plans’, ‘plans’, ‘seeks’, ‘should’, ‘will’ and variations of these words or similar expressions. We intend that these forward-looking statements be covered by these safe harbor provisions for forward-looking statements and make this statement in order to comply with these safe harbor provisions. These forward-looking statements include statements regarding the potential therapeutic benefits of efzofitimod and plans for certain clinical activities. These forward-looking statements also reflect our current beliefs about our plans, intentions, expectations, strategies and prospects, which are based on information currently available to us and assumptions made by us. Although we believe that our plans, intentions, expectations, strategies and prospects, as reflected or implied by these forward-looking statements, are reasonable, we cannot guarantee that the plans, intentions, expectations or strategies will be achieved or realized. All forward-looking statements are based on our management’s estimates and assumptions which, although we believe to be reasonable, are inherently uncertain. In addition, actual results may differ materially from those described in these forward-looking statements and will be affected by a variety of risks and factors beyond our control, including, without limitation, uncertainty regarding the COVID-19 pandemic. 19, the risks associated with the discovery, development and regulation of our product candidates, the risk that we or our partners stop or delay preclinical or clinical development activities of any of our existing or future product candidates for various reasons (including difficulties or delays in recruiting patients into trials), the possibility that existing collaborations may be terminated prematurely, and the risk that we may not be able to raise additional funds necessary for our business plans and product development, as well as the risks set forth in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2022 filed with the SEC on August 15, 2022 and in our other filings with the SEC. Except as required by law, we undertake no obligation to publicly update any forward-looking statements, whether as a result of new information, future events or otherwise.

Ashlee Dunton
Director, Investor Relations and Corporate Communications
[email protected]


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