aTyr Pharma Advances Development of Lead Therapeutic Candidate ATYR1923 with Announcement of “efzofitimod” as Nonproprietary Name

Efzofitimod expected to enter into a registrational trial for pulmonary sarcoidosis in 2022

SAN DIEGO, Jan. 13, 2022 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways, today announced that the United States Adopted Names (USAN) Council and the World Health Organization’s (WHO) International Nonproprietary Name (INN) Expert Committee have selected the nonproprietary name efzofitimod for ATYR1923, a novel immunomodulator targeting neuropilin-2 (NRP2), in clinical development for pulmonary sarcoidosis, a major form of interstitial lung disease.

“aTyr is working to develop a new class of medicines based on extracellular tRNA synthetase biology, and the assignment of the nonproprietary name efzofitimod for ATYR1923 is an important step as we continue to advance this potentially disease-modifying immunomodulator to its next stage of development,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “Efzofitimod is engineered from a naturally occurring splice variant of histidyl-tRNA synthetase, which is enriched in lung tissue and we believe plays a role in the natural regulation of the immune system. By restoring immune balance through selective modulation of NRP2, efzofitimod is the first tRNA synthetase-derived and NRP2-targeting therapy to demonstrate clinical activity in patients. We look forward to initiating a registrational trial of efzofitimod in our lead indication, pulmonary sarcoidosis, this year.”

The USAN Council and WHO are responsible for selecting simple, informative and unique nonproprietary (generic) drug names. Going forward, aTyr will use efzofitimod in place of ATYR1923.

About Efzofitimod

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

About aTyr

aTyr is a biotherapeutics company engaged in the discovery and development of innovative medicines based on novel biological pathways. aTyr’s research and development efforts are concentrated on a newly discovered area of biology, the extracellular functionality and signaling pathways of tRNA synthetases. aTyr has built a global intellectual property estate directed to a potential pipeline of protein compositions derived from 20 tRNA synthetase genes and their extracellular targets. aTyr’s primary focus is efzofitimod, a clinical-stage product candidate which binds to the neuropilin-2 receptor and is designed to down-regulate immune engagement in inflammatory lung diseases. 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 usually identified by the use of words such as “anticipates,” “believes,” “estimates,” “expects,” “intends,” “may,” “plans,” “projects,” “seeks,” “should,” “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 statements regarding potential therapeutic benefits and applications of efzofitimod; timelines and plans with respect to certain development activities (such as the timing of additional clinical trials and planned interactions with regulatory authorities); 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 or strategies 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 the COVID-19 pandemic, risks associated with the discovery, development and regulation of our product candidates, the risk that we or our partners may cease or delay preclinical or clinical development activities for any of our existing or future product candidates 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.



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