aTyr Pharma (NASDAQ: ATYR) misses Phase 3 endpoint, ends 2025 with $80.9M cash

Rhea-AI Filing Summary

aTyr Pharma reported fourth quarter and full year 2025 results and provided a pipeline update centered on its lead drug efzofitimod for interstitial lung diseases.

The Phase 3 EFZO-FIT study in pulmonary sarcoidosis did not meet its primary endpoint of reducing mean daily oral corticosteroid dose at week 48, but the 5.0 mg/kg dose showed clinical benefits on several pre-specified measures of symptoms and quality of life, and maintained lung function with a favorable safety profile. The company has a Type C meeting with the FDA scheduled for mid-April 2026 to review the data and determine the regulatory path forward.

aTyr’s Phase 2 EFZO-CONNECT trial in systemic sclerosis-related interstitial lung disease is on track to complete enrollment in the first half of 2026. The company also highlighted preclinical progress for ATYR0101 in fibrosis and published research on a bispecific NRP2/PLXNA1 antibody with potential anticancer activity. For 2025, aTyr generated $190 thousand in collaboration revenue, incurred $60.2 million in research and development expenses and $17.6 million in general and administrative expenses, leading to a net loss attributable to aTyr of $74.1 million, or $0.80 per share. Cash, cash equivalents, restricted cash and investments totaled $80.9 million as of December 31, 2025.

Positive

• None.

Negative

• Phase 3 primary endpoint not met: The EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis failed its primary endpoint of change from baseline in mean daily oral corticosteroid dose at week 48, creating uncertainty around the regulatory path for the lead program.

Insights

Biotech & Clinical Development Analyst

Phase 3 miss on primary endpoint is partially offset by secondary measures and FDA dialogue.

The EFZO-FIT Phase 3 trial in pulmonary sarcoidosis did not achieve its primary endpoint of steroid dose reduction at week 48, which is a key efficacy hurdle for regulators. However, the 5.0 mg/kg efzofitimod arm showed benefits on multiple pre-specified symptom and quality-of-life measures and maintained lung function, with a safety profile consistent with prior studies.

aTyr plans a Type C FDA meeting in mid-April 2026 to review the data and discuss the path forward. The actual regulatory trajectory will depend on how the FDA weighs the missed primary endpoint against these secondary outcomes and safety findings. The ongoing Phase 2 EFZO-CONNECT trial in SSc-ILD, expected to complete enrollment in the first half of 2026, provides an additional indication that could diversify the efzofitimod story.

Biotech Equity & Cash Runway Analyst

Spending rose with late-stage trials, while year-end cash modestly increased.

For the year ended 2025, aTyr reported license and collaboration revenues of $190 thousand and a net loss attributable to the company of $74.1M, compared with a $64.0M net loss in 2024. The increase reflects higher research and development spending of $60.2M, largely driven by the EFZO-FIT and EFZO-CONNECT studies.

General and administrative expenses rose to $17.6M, indicating growing overhead. Cash, cash equivalents, restricted cash and available-for-sale investments were $80.9M as of December 31, 2025, up from $75.1M a year earlier, supporting ongoing clinical and preclinical programs. Future disclosures in company filings may clarify how this cash position aligns with planned development milestones.

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): March 5, 2026

ATYR PHARMA, INC.

(Exact name of registrant as specified in its charter)

Delaware		001-37378		20-3435077
(State or other jurisdiction of incorporation)		(Commission File Number)		(IRS Employer Identification No.)
10240 Sorrento Valley Road, Suite 300 San Diego, CA				92121
(Address of Principal Executive Offices)				(Zip Code)

Registrant’s telephone number, including area code: (858) 731-8389

Not Applicable

(Former Name or Former Address, if Changed Since Last Report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligations of the registrant under any of the following provisions:

☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class	Trading Symbol(s)	Name of each exchange on which registered
Common Stock, par value $0.001 per share	ATYR	The Nasdaq Capital Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 or Rule 12b-2 of the Securities Exchange Act of 1934.

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.

Item 2.02 Results of Operations and Financial Condition.

On March 5, 2026, aTyr Pharma, Inc. issued a press release announcing financial results for the year ended December 31, 2025. A copy of the press release is furnished as Exhibit 99.1 to this report and is incorporated herein by reference.

The information under this Item 2.02, including Exhibit 99.1 hereto is being furnished and shall not be deemed “filed” for the purposes of Section 18 of the Securities and Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall such information be deemed incorporated by reference into any filing under the Securities Act of 1933, as amended or the Exchange Act, except as expressly set forth by specific reference in such filing.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits

Exhibit No.		Description
99.1		Press Release, dated March 5, 2026
104		Cover Page Interactive Data File (embedded within the Inline XBRL document)

2

SIGNATURE

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

	ATYR PHARMA, INC.
	By:	/s/ Jill M. Broadfoot
		Jill M. Broadfoot
		Chief Financial Officer
Date: March 5, 2026

3

Exhibit 99.1

Contact:

Ashlee Dunston

Sr. Director, Investor Relations and Public Affairs

adunston@atyrpharma.com

aTyr Pharma Announces Fourth Quarter and Full Year 2025 Results and Provides Corporate Update

Company scheduled to meet with the FDA in mid-April 2026 to review the results of the Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis.

Phase 2 EFZO-CONNECT™ study of efzofitimod in systemic sclerosis-related interstitial lung disease (SSc-ILD) on track to complete enrollment in the first half of 2026.

Ended 2025 with $80.9 million in cash, cash equivalents, restricted cash and investments.

SAN DIEGO – March 5, 2026 – 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 2025 results and provided a corporate update.

“In 2025 we announced results from our Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease (ILD) where a significant proportion of patients develop chronic or progressive disease with debilitating symptoms despite current treatment options. This marked an important milestone, not only for the broader sarcoidosis community, but also for aTyr, as it was the Company’s largest and first Phase 3 study of a tRNA synthetase-derived therapy generated from our platform,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr.

“We are ready and look forward to engaging with the U.S. Food and Drug Administration (FDA) in mid-April to review the results of the study and determine the path forward for efzofitimod in pulmonary sarcoidosis. We plan to provide an update regarding the next steps for the program following the receipt of the official FDA meeting minutes.”

Fourth Quarter 2025 and Subsequent Period Highlights

•Announced the scheduling of a Type C meeting with the FDA in mid-April 2026 to review the results of the Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis. The Company expects to provide an update regarding the outcome of the meeting following the receipt of the official meeting minutes. EFZO-FIT™ was a Phase 3 study to evaluate the efficacy and safety of 3.0 mg/kg and 5.0 mg/kg of efzofitimod or placebo in 268 patients

with symptomatic pulmonary sarcoidosis. The study did not meet its primary endpoint of change from baseline in mean daily oral corticosteroid dose at week 48. Clinical benefit for 5.0 mg/kg efzofitimod was observed across multiple pre-specified study efficacy parameters at week 48 compared to placebo, including the King’s Sarcoidosis Questionnaire (KSQ)-Lung score (p=0.0479), Fatigue Assessment Scale score (p=0.0226), KSQ-General Health score (p=0.0197), and complete steroid withdrawal with KSQ-Lung score improvement (p=0.0196). Additionally, treatment with efzofitimod maintained lung function as measured by forced vital capacity and was well-tolerated with a safety profile consistent with prior trials conducted to date.

•On track to complete enrollment in the Phase 2 EFZO-CONNECT™ study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with limited or diffuse systemic sclerosis (SSc, or scleroderma)-related ILD (SSc-ILD) in the first half of 2026. This proof-of-concept study is a randomized, double-blind, placebo-controlled, 28-week study consisting of three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo administered intravenously monthly for a total of six doses. The study intends to enroll up to 25 patients at multiple centers in the United States. Promising interim data from the study were reported in the second quarter of 2025.

•Presented a poster related to its investigational new drug candidate, ATYR0101, at the Keystone Symposia on Fibrosis: Cross Organ Pathology and Pathways to Clinical Development. The poster demonstrated that subcutaneous delivery of ATYR0101 yielded a comparable pharmacokinetic and immunogenicity profile, which is favorable to other delivery methods, while reducing lung inflammation. The findings presented in the poster suggest the ability of ATYR0101 to potentially resolve the cycle of chronic inflammation and fibrosis utilizing a novel mechanism and further support a compelling therapeutic profile for patients suffering from fibrosis. The poster is available on the Company’s website.

•Published an article demonstrating the generation of a functional neuropilin-2 (NRP2)/plexinA1 (PLXNA1) bispecific antibody in the Journal of Biological Chemistry. The publication, entitled, “A bispecific antibody designed to act as a NRP2/PLXNA1 agonist mimics anticancer activity of SEMA3F,” demonstrates that the bispecific antibody selectively mimics the beneficial aspects of semaphorin 3F (SEMA3F)/NRP2 signaling while avoiding potentially cross-toxic reactivity, serving as a basis for a novel anticancer therapy. The publication is available on the Company’s website and at: https://www.jbc.org/article/S0021-9258%2825%2902908-4/fulltext.

Year Ended 2025 Financial Highlights and Cash Position

•Cash & Investment Position: Cash, cash equivalents, restricted cash and available-for-sale investments as of December 31, 2025, were $80.9 million.

•R&D Expenses: Research and development expenses were $60.2 million for the year ended 2025, which consisted primarily of costs for the Phase 3 EFZO-FIT™ and Phase 2 EFZO-CONNECT™ studies and research and development costs for the Company’s preclinical product candidates.

•G&A Expenses: General and administrative expenses were $17.6 million for the year ended 2025.

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. In addition to the global Phase 3 EFZO-FIT™ study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD, efzofitimod is also being investigated 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 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 “aims,” “anticipates,” “believes,” “can,” “designed,” “expects,” “hopes,” “intends,” “look toward,” “may,” “plans,” “potential,” “project,” “suggest,” “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 the potential therapeutic benefits and applications of efzofitimod and ATYR0101; the potential for efzofitimod to improve patient quality of life across multiple disease related health outcomes in pulmonary sarcoidosis; the expected size of, and number of patients to be enrolled in the Phase 2 EFZO-CONNECT™ study; timelines and plans with respect to certain development activities and development goals, including the occurrence and timing of our meeting with the FDA to review the results of the

Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis as well as our expectations with respect to the outcome of that meeting, the timing of our update for that meeting and next steps for the development of efzofitimod in pulmonary sarcoidosis; and our expectation that our Phase 2 EFZO-CONNECT™ study of efzofitimod in patients with SSc-ILD will complete enrollment in the first half of 2026. 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, 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 risks inherent in using the results from the EFZO-FIT™ study to pursue FDA approval for efzofitimod in pulmonary sarcoidosis, the risk of delays in our clinical trials, risks associated with the discovery, development and regulation of our existing or future 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 (including difficulties or delays in patient enrollment in planned clinical trials), 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.

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,
		2025				2024				2025				2024
Revenues:
License and collaboration agreement revenues		$	—			$	—			$	190			$	235
Total revenues			—				—				190				235
Operating expenses:
Research and development			10,891				12,228				60,219				54,372
General and administrative			3,905				3,592				17,598				13,777
Total operating expenses			14,796				15,820				77,817				68,149
Loss from operations			(14,796	)			(15,820	)			(77,627	)			(67,914	)
Total other income (expense), net			832				852				3,504				3,892
Consolidated net loss			(13,964	)			(14,968	)			(74,123	)			(64,022	)
Net loss (gain) attributable to noncontrolling interest in Pangu BioPharma Limited			1				1				5				(1	)
Net loss attributable to aTyr Pharma, Inc.		$	(13,963	)		$	(14,967	)		$	(74,118	)		$	(64,023	)
Net loss per share, basic and diluted		$	(0.14	)		$	(0.18	)		$	(0.80	)		$	(0.86	)
Shares used in computing net loss per share, basic and diluted			98,010,084				82,724,659				92,985,359				74,261,265

ATYR PHARMA INC.
Condensed Consolidated Balance Sheets
(in thousands)
		December 31,				December 31,
		2025				2024
Cash, cash equivalents, restricted cash and available-for-sale investments		$	80,922			$	75,076
Other receivables			873				1,736
Property and equipment, net			4,263				4,850
Operating lease, right-of-use assets			5,524				5,817
Financing lease, right-of-use assets			596				1,192
Prepaid expenses and other assets			825				8,159
Total assets		$	93,003			$	96,830
Accounts payable and accrued expenses		$	13,682			$	13,715
Current portion of operating lease liability			836				711
Current portion of financing lease liability			630				541
Long-term operating lease liability, net of current portion			10,308				11,144
Long-term financing lease liability, net of current portion			259				887
Total stockholders’ equity			67,288				69,832
Total liabilities and stockholders’ equity		$	93,003			$	96,830

FAQ

What were aTyr Pharma (ATYR) key financial results for 2025?

aTyr Pharma reported 2025 license and collaboration revenues of $190 thousand, research and development expenses of $60.2 million, and general and administrative expenses of $17.6 million. Net loss attributable to aTyr was $74.1 million, or $0.80 per share.

How much cash did aTyr Pharma (ATYR) have at year-end 2025?

As of December 31, 2025, aTyr Pharma held $80.9 million in cash, cash equivalents, restricted cash and available-for-sale investments. This balance supports its ongoing Phase 3 and Phase 2 trials and preclinical programs in fibrosis and inflammation.

What were the main outcomes of aTyr Pharma’s EFZO-FIT Phase 3 trial?

The EFZO-FIT Phase 3 study in pulmonary sarcoidosis did not meet its primary endpoint of steroid dose reduction at week 48. However, 5.0 mg/kg efzofitimod showed benefits on several pre-specified symptom and quality-of-life measures and maintained lung function with a favorable safety profile.

When will aTyr Pharma meet the FDA about efzofitimod in sarcoidosis?

aTyr Pharma is scheduled for a Type C meeting with the FDA in mid-April 2026 to review EFZO-FIT Phase 3 results. The company plans to provide an update on next steps after receiving the official meeting minutes.

What is the status of aTyr Pharma’s EFZO-CONNECT Phase 2 study?

The EFZO-CONNECT Phase 2 study evaluates efzofitimod in systemic sclerosis-related interstitial lung disease. It plans to enroll up to 25 patients and is on track to complete enrollment in the first half of 2026, following promising interim data reported earlier.

What additional pipeline progress did aTyr Pharma (ATYR) report?

aTyr highlighted preclinical data on ATYR0101, showing favorable pharmacokinetics and reduced lung inflammation with subcutaneous delivery. It also published research on an NRP2/PLXNA1 bispecific antibody that mimics beneficial SEMA3F signaling, supporting potential anticancer applications.

How did aTyr Pharma’s 2025 expenses compare between R&D and G&A?

In 2025, aTyr Pharma incurred $60.2 million in research and development expenses, mainly for EFZO-FIT and EFZO-CONNECT, and $17.6 million in general and administrative expenses. This shows spending was heavily concentrated on advancing clinical and preclinical programs.

Filing Exhibits & Attachments

2 documents

Press Releases

• EX-99.1 EX-99.1 194.5 KB

Other Documents

• EX-101 XBRL TAXONOMY EXTENSION SCHEMA WITH EMBEDDED LINKBASES DOCUMENT 25.4 KB