aTyr Pharma Announces Scheduling of FDA Type C Meeting to Discuss Efzofitimod Program in Pulmonary Sarcoidosis

aTyr Pharma (Nasdaq: ATYR) said the FDA accepted a Type C meeting request to discuss efzofitimod for pulmonary sarcoidosis, scheduled for mid-April 2026.

Rhea-AI Summary

aTyr Pharma (Nasdaq: ATYR) said the FDA accepted a Type C meeting request to discuss efzofitimod for pulmonary sarcoidosis, scheduled for mid-April 2026. The company will review Phase 3 EFZO-FIT results and report meeting minutes afterwards.

The Phase 3 study enrolled 268 patients; it did not meet the primary endpoint of corticosteroid dose reduction at week 48, but 5.0 mg/kg efzofitimod showed statistically significant benefits on multiple patient-reported outcomes and maintained lung function, with a safety profile consistent with prior trials.

Positive

• FDA accepted a Type C meeting for mid-April 2026 to review efzofitimod
• Phase 3 EFZO-FIT enrolled 268 patients, a sizable symptomatic pulmonary sarcoidosis cohort
• 5.0 mg/kg efzofitimod showed statistically significant improvements on multiple endpoints (KSQ-Lung p=0.0479)
• Efzofitimod maintained lung function (forced vital capacity) and was well tolerated

Negative

• Phase 3 EFZO-FIT did not meet its primary endpoint of corticosteroid dose reduction at week 48
• Regulatory path remains uncertain pending the FDA Type C meeting outcome and official meeting minutes

Argus Feb 3 session

-1.03% close to close Open Argus

Details

News Market Reaction – ATYR

On Feb 3, the day this news came out, ATYR closed 1.03% below the previous close.

Data tracked by StockTitan Argus for the Feb 3 session.

Key Figures

EFZO-FIT sample size: 268 patients Efzofitimod dose: 5.0 mg/kg KSQ-Lung p-value: p=0.0479 +3 more

EFZO-FIT sample size

268 patients

Phase 3 EFZO-FIT study in pulmonary sarcoidosis

Efzofitimod dose

5.0 mg/kg

Dose showing clinical benefit across multiple efficacy parameters

KSQ-Lung p-value

p=0.0479

Improvement in KSQ-Lung score at week 48 vs placebo

Fatigue scale p-value

p=0.0226

Fatigue Assessment Scale improvement at week 48 vs placebo

KSQ-General Health p-value

p=0.0197

KSQ-General Health score improvement at week 48 vs placebo

Steroid withdrawal + KSQ-Lung p-value

p=0.0196

Complete steroid withdrawal with KSQ-Lung improvement vs placebo

Historical Context

5 past events · Latest: Nov 10

5 events

1. Nov 10

   Conference appearance

   24h Move

   -3.3%

   Piper Sandler healthcare conference participation and investor outreach plans.

2. Nov 06

   Earnings and update

   24h Move

   -5.3%

   Q3 2025 results and reiteration that EFZO-FIT™ missed its primary endpoint.

3. Sep 30

   Clinical data details

   24h Move

   -9.3%

   Additional EFZO-FIT™ findings with missed primary endpoint but secondary benefits.

4. Sep 15

   Topline Phase 3

   24h Move

   -83.2%

   Topline EFZO-FIT™ results showing primary endpoint not met despite secondary signals.

5. Sep 05

   Inducement grants

   24h Move

   -2.7%

   Nasdaq Rule 5635(c)(4) inducement stock option grants to new employees.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

phase 3, phase 2, forced vital capacity, interstitial lung disease, +3 more

7 terms

phase 3 medical

"EFZO-FIT™ was a Phase 3 study of efzofitimod in 268 patients..."

Phase 3 is the late-stage clinical testing step for a new drug or medical treatment, where the product is given to large groups of patients to confirm effectiveness, monitor side effects, and compare it to standard care. Successful Phase 3 results are often the final scientific hurdle before regulators decide on approval and market launch—like passing a final exam before graduation—and can sharply change a company's valuation and future revenue prospects.

phase 2 medical

"efzofitimod is also being investigated in the Phase 2 EFZO-CONNECT™ study..."

Phase 2 is the mid-stage clinical trial where a new drug or treatment is tested in a larger group of patients to see if it works and to keep checking safety after initial human testing. Think of it as a field test that proves whether a product actually delivers its promised benefit. Investors watch Phase 2 closely because its results strongly influence a medicine’s chances of reaching the market, the size of its potential sales, and the company’s valuation.

forced vital capacity medical

"treatment with efzofitimod maintained lung function as a measure of forced vital capacity..."

The amount of air a person can forcefully breathe out after taking the deepest breath possible; think of it as how much air you can squeeze out of a balloon in one hard blow. It matters to investors because it’s a common, objective measure used in clinical trials and patient monitoring for respiratory drugs, devices and treatments—changes in this number can signal whether a therapy works, affecting regulatory approval, sales and company value.

interstitial lung disease medical

"Efzofitimod is a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD)..."

A group of lung conditions that cause inflammation and scarring of the thin tissue between the air sacs, which makes it harder for oxygen to pass into the blood; imagine the lungs’ fine filters becoming stiff and less effective. Investors care because reports of interstitial lung disease can affect a drug’s safety profile, trigger regulatory warnings or label changes, and shift demand for treatments or create liability risks that influence a company’s valuation.

neuropilin-2 medical

"therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation..."

Neuropilin-2 is a protein on the surface of cells that acts like a docking station or traffic director, helping signals guide the growth of blood vessels, lymphatic vessels and nerves. In drug development it matters because altering neuropilin-2’s activity can influence tumor growth, spread and wound healing, so it is a target for therapies and a potential biomarker; investors watch related research progress as an indicator of therapeutic potential and commercial opportunity.

trna synthetase medical

"Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells..."

tRNA synthetase is an enzyme that matches each building block of proteins (an amino acid) to its correct carrier molecule called transfer RNA (tRNA), ensuring proteins are assembled correctly in cells. Think of it as a factory worker who puts the right part onto the right delivery tray before assembly; if it fails, proteins can be made incorrectly. Investors watch these enzymes because they can be drug targets, biomarkers of disease, or sources of genetic disorders that affect a company’s therapeutic or safety profile.

immunomodulator medical

"Efzofitimod is a novel biologic immunomodulator in clinical development..."

An immunomodulator is a medicine or therapy that changes how the immune system behaves—either boosting its ability to fight infections and cancer or calming it to reduce harmful inflammation. Investors watch these products because their success in clinical trials, safety and side-effect profiles, and regulatory approvals can open large markets or limit sales; think of them as tools that tweak the body’s defense system, with outcomes that strongly affect a drug maker’s future revenue and stock value.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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

SAN DIEGO, Feb. 03, 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 U.S. Food and Drug Administration (FDA) has accepted the Company’s meeting request to discuss its lead therapeutic candidate, efzofitimod, for the treatment of pulmonary sarcoidosis. The Type C meeting is scheduled for mid-April 2026.

“We look forward to meeting with the FDA in mid-April to review the results of our Phase 3 EFZO-FIT™ study and determine the path forward for efzofitimod in pulmonary sarcoidosis, a major form of interstitial lung disease,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr Pharma. “We expect 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 of efzofitimod in 268 patients with symptomatic pulmonary sarcoidosis. While 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 study efficacy parameters at week 48 compared to placebo, including improvement in change from baseline for 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 a measure of forced vital capacity and was well-tolerated with a safety profile consistent with prior trials conducted to date.

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 "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 the potential therapeutic benefits and applications of efzofitimod; and timelines and plans with respect to certain development activities and 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. 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 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 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 Dunston

Sr. Director, Investor Relations and Public Affairs

adunston@atyrpharma.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What is the purpose of aTyr Pharma's mid-April 2026 FDA Type C meeting for ATYR?

The meeting will review Phase 3 EFZO-FIT results and discuss next steps for efzofitimod regulatory strategy. According to the company, the discussion will focus on interpreting the trial data and determining the path forward for pulmonary sarcoidosis.

Did the Phase 3 EFZO-FIT study for efzofitimod meet its primary endpoint in 2026?

No, the study did not meet the primary endpoint of mean daily oral corticosteroid dose change at week 48. According to the company, secondary measures showed clinical benefit for 5.0 mg/kg on several patient-reported outcomes.

Which efficacy measures showed benefit for efzofitimod in the EFZO-FIT Phase 3 study (ATYR)?

Efzofitimod 5.0 mg/kg showed significant improvement in KSQ-Lung, Fatigue Assessment Scale, KSQ-General Health, and steroid withdrawal with KSQ-Lung improvement. According to the company, these signals were statistically significant versus placebo at week 48.

How many patients were enrolled in the EFZO-FIT Phase 3 trial and what was the safety outcome?

EFZO-FIT enrolled 268 symptomatic pulmonary sarcoidosis patients and treatment was well tolerated. According to the company, the safety profile was consistent with prior efzofitimod trials and no new safety signals were reported.

What should investors expect after the ATYR FDA Type C meeting in April 2026?

Investors should expect an update after receipt of official meeting minutes detailing FDA feedback and next regulatory steps. According to the company, they will provide a public update regarding the meeting outcome following the minutes.