Topline data from Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis expected in the third quarter of 2025.
Fourth positive DSMB review for efzofitimod in Phase 3 EFZO-FIT™ study reinforces its favorable safety profile.
Publication in Science Translational Medicine validates efzofitimod’s mechanism of action.
Company to host conference call and webcast today, March 13th at 5:00 p.m. EDT / 2:00 p.m. PDT.
SAN DIEGO, March 13, 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 fourth quarter and full year 2024 results and provided a corporate update.
“2024 was an important year for aTyr, as we completed enrollment in our global pivotal Phase 3 EFZO-FIT™ study, which is the largest interventional study ever to be conducted in pulmonary sarcoidosis, a disease where incidence and prevalence are rising yet there are still limited treatment options,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “The stage is set for 2025 to be a potential milestone year as we look toward reporting topline data for EFZO-FIT™ in the third quarter. Leading into this pivotal readout, we are very pleased with the execution of the study to date and the continued favorable safety profile demonstrated for efzofitimod, including our most recent positive DSMB review, which recommended the study continue as designed. Efzofitimod is poised to potentially become a safe and effective alternative to the current standard of care and improve patients’ lives.”
Fourth Quarter 2024 and Subsequent Period Highlights
Year Ended 2024 Financial Highlights and Cash Position
Conference Call and Webcast Details
aTyr will host a conference call and webcast today at 5:00 p.m. EDT / 2:00 p.m. PDT to discuss its financial results and provide a corporate update. Interested parties may access the call by registering here in order to obtain a dial in, personalized passcode and webcast information. Links to a live audio webcast and replay may be accessed on the aTyr website Events page at: https://investors.atyrpharma.com/events-and-webcasts. An audio replay will be available for at least 90 days following the event.
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 “aims” “anticipates,” “believes,” “can,” “designed,” “expects,” “intends,” “look toward,” “may,” “plans,” “potential,” “project,” “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 interactions with the FDA relating to the SAP for the Phase 3 EFZO-FIT™ study, including the measurement of steroid reduction; the expected size of, and number of patients to be enrolled in, the EFZO-CONNECT™ study; the skin assessments and expected number of patients to be included in the interim data for the EFZO-CONNECT™ study; the potential therapeutic benefits and applications of efzofitimod; expectations regarding our financial guidance and the sufficiency of our cash runway; and timelines and plans with respect to certain development activities and development goals, including the potential filing of a BLA for efzofitimod in pulmonary sarcoidosis and our expectation that our Phase 3 EFZO-FIT™ study of efzofitimod in patients with pulmonary sarcoidosis will report topline data in the third quarter of 2025 and expectation that our Phase 2 EFZO-CONNECT™ study will report interim data in the second quarter of 2025. 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, risks related to our reliance on third-party partners and the potential that such partners may not perform as anticipated, the fact that NRP2 and tRNA synthetase biology is not fully understood, uncertainty regarding the ultimate long-term impact of evolving macroeconomic and geopolitical conditions, the risk of delays in our clinical trials, risks associated with the discovery, development and regulation of our product candidates, including the uncertainty of related costs and regulatory filings and the risk that results from clinical trials or other studies may not support further development, the risk that we may cease or delay preclinical or clinical development activities for any of our existing or future product candidates for a variety of reasons, the fact that our collaboration agreements are subject to early termination, 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 |
| ATYR PHARMA INC. | |||||||||||||||
| Condensed Consolidated Statements of Operations | |||||||||||||||
| (in thousands, except share and per share data) | |||||||||||||||
| Three Months Ended | Years Ended | ||||||||||||||
| December 31, | December 31, | ||||||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||||||
| Revenues: | |||||||||||||||
| License and collaboration agreement revenues | $ | — | $ | — | $ | 235 | $ | 353 | |||||||
| Total revenues | — | — | 235 | 353 | |||||||||||
| Operating expenses: | |||||||||||||||
| Research and development | 12,228 | 12,755 | 54,372 | 42,293 | |||||||||||
| General and administrative | 3,592 | 3,204 | 13,777 | 12,979 | |||||||||||
| Total operating expenses | 15,820 | 15,959 | 68,149 | 55,272 | |||||||||||
| Loss from operations | (15,820 | ) | (15,959 | ) | (67,914 | ) | (54,919 | ) | |||||||
| Total other income (expense), net | 852 | 1,198 | 3,892 | 4,522 | |||||||||||
| Consolidated net loss | (14,968 | ) | (14,761 | ) | (64,022 | ) | (50,397 | ) | |||||||
| Net (gain) loss attributable to noncontrolling interest in Pangu BioPharma Limited | 1 | 1 | (1 | ) | 8 | ||||||||||
| Net loss attributable to aTyr Pharma, Inc. | $ | (14,967 | ) | $ | (14,760 | ) | $ | (64,023 | ) | $ | (50,389 | ) | |||
| Net loss per share, basic and diluted | $ | (0.18 | ) | $ | (0.25 | ) | $ | (0.86 | ) | $ | (0.94 | ) | |||
| Shares used in computing net loss per share, basic and diluted | 82,724,659 | 59,261,219 | 74,261,265 | 53,606,488 | |||||||||||
| ATYR PHARMA INC. | |||||||
| Condensed Consolidated Balance Sheets | |||||||
| (in thousands) | |||||||
| December 31, | December 31, | ||||||
| 2024 | 2023 | ||||||
| Cash, cash equivalents, restricted cash and available-for-sale investments | $ | 75,076 | $ | 101,650 | |||
| Other receivables | 1,736 | 2,436 | |||||
| Property and equipment, net | 4,850 | 5,531 | |||||
| Operating lease, right-of-use assets | 5,817 | 6,727 | |||||
| Financing lease, right-of-use assets | 1,192 | 1,788 | |||||
| Prepaid expenses and other assets | 8,159 | 2,521 | |||||
| Total assets | $ | 96,830 | $ | 120,653 | |||
| Accounts payable and accrued expenses | $ | 13,715 | $ | 15,088 | |||
| Current portion of operating lease liability | 711 | 831 | |||||
| Current portion of financing lease liability | 541 | 497 | |||||
| Long-term operating lease liability, net of current portion | 11,144 | 12,339 | |||||
| Long-term financing lease liability, net of current portion | 887 | 1,428 | |||||
| Total stockholders’ equity | 69,832 | 90,470 | |||||
| Total liabilities and stockholders’ equity | $ | 96,830 | $ | 120,653 |