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Efzofitimod/kg

Phase 3

Pulmonary Sarcoidosis | Small molecule | Respiratory |aTyr Pharma, Inc.|Last Updated: Jun 4, 2026

Target and mechanism

ModalitySmall molecule

Also known as efzofitimod

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment268

FDA Designations

No designations recorded

Clinical trial landscape

Efzofitimod/kg · 2 trials · 2 indications

Phase 3 1Phase 2 1
NCT05415137Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary SarcoidosisPulmonary Sarcoidosis
COMPLETED268 Analytics
PHASE3COMPLETED
Efficacy and Safety of Intravenous Efzofitimod in Patients With Pulmonary Sarcoidosis
Pulmonary SarcoidosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Change from baseline in mean daily oral corticosteroid (OCS) dose at Week 48
Baseline to Week 48
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Baseline up to Day 60

TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. AEs were defined as any untoward medical occurrence in a participant administered study drug and that does not necessarily have a causal relationship with the study drug. Worsening of a pre-existing medical condition should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. SAEs were defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required in-participant hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, a congenital anomaly/birth defect, an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in 'Reported Adverse Events' Section.

Secondary Endpoints

Change from baseline in KSQ-Lung score at Week 48
Baseline to Week 48
Steroid withdrawal rate
Baseline to Week 48
Change from baseline in absolute value of FVC at Week 48
Baseline to Week 48
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Efzofitimod 3 mg/kgEXPERIMENTAL -
Efzofitimod 5 mg/kgEXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Efzofitimod 1 mg/kgEXPERIMENTALParticipants will receive single dose of efzofitimod 1 milligrams/kilograms (mg/kg) IV infusion on Day 1.

Interventions

NameTypeDescription
Efzofitimod 3 mg/kgDRUGEfzofitimod IV infusion every 4 weeks for a total of 12 doses
Efzofitimod 5 mg/kgDRUGEfzofitimod IV infusion every 4 weeks for a total of 12 doses
PlaceboDRUGPlacebo IV infusion every 4 weeks for a total of 12 doses
Efzofitimod 1 mg/kgDRUGConcentrate for solution for infusion
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites91

Inclusion Criteria: * Documented history of pulmonary sarcoidosis for at least 6 months, supported by the following evidence: documented histologically proven diagnosis of sarcoidosis by tissue biopsy and documented evidence of parenchymal lung involvement by historical radiological evidence * Evid...

Countries:United StatesBrazilFranceGermanyItalyJapanNetherlandsSpainUnited KingdomPuerto Rico
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Recent Changes (Last 90 Days)

MEDIUMJul 5, 2026NCT05415137TRIAL_REMOVED: changed
MEDIUMJul 5, 2026NCT05415137TRIAL_REMOVED: changed
MEDIUMJul 5, 2026NCT05415137TRIAL_REMOVED: changed
HIGHJun 4, 2026NCT05415137Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHJun 4, 2026NCT05415137Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHJun 4, 2026NCT05415137Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHJun 4, 2026NCT05415137Status: ACTIVE_NOT_RECRUITING → COMPLETED

Frequently asked questions about Efzofitimod/kg

What is Efzofitimod/kg used for?

Efzofitimod/kg is an investigational small molecule being developed for respiratory conditions, including pulmonary sarcoidosis, interstitial lung disease, and severe pneumonia related to SARS-CoV-2 (COVID-19). It is administered intravenously and is currently in clinical trials, with the most advanced study being a Phase 3 trial in pulmonary sarcoidosis.

Who makes Efzofitimod/kg?

Efzofitimod/kg is developed by aTyr Pharma, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker symbol ATYR. The company is conducting clinical trials to evaluate the drug's safety and efficacy in multiple respiratory indications.

What phase is Efzofitimod/kg in?

Efzofitimod/kg is in Phase 3 clinical development for pulmonary sarcoidosis, based on a completed Phase 3 trial (NCT05415137). It is also being studied in Phase 2 trials for COVID-19 severe pneumonia and interstitial lung disease. The drug is investigational and not yet approved by regulatory authorities.

What clinical trials is Efzofitimod/kg in?

Efzofitimod/kg has been evaluated in three clinical trials. NCT04412668 was a completed Phase 2 study in COVID-19 severe pneumonia. NCT05415137 was a completed Phase 3 trial in pulmonary sarcoidosis. NCT05892614 is an ongoing Phase 2 trial in systemic sclerosis-related interstitial lung disease.

Is Efzofitimod/kg the same as efzofitimod?

Yes, Efzofitimod/kg is also known as efzofitimod, and the drug is sometimes referred to by the dose strength efzofitimod 450 mg. These names refer to the same investigational compound developed by aTyr Pharma.

Is Efzofitimod/kg FDA approved?

Efzofitimod/kg is not FDA approved. It remains an investigational drug in clinical development, with trials ongoing or completed in pulmonary sarcoidosis, interstitial lung disease, and COVID-19 severe pneumonia. Approval status has not been granted for any indication.