aTyr Pharma Announces Last Patient Visit in Phase 3 EFZO-FIT鈩� Study of Efzofitimod in Patients with Pulmonary Sarcoidosis
aTyr Pharma (NASDAQ:ATYR) has announced the completion of the last patient visit in its Phase 3 EFZO-FIT鈩� study for efzofitimod in pulmonary sarcoidosis patients. The study, which is the first-ever global Phase 3 and largest interventional sarcoidosis trial, enrolled 268 subjects across multiple centers in the US, Europe, Japan, and Brazil.
The 52-week study evaluates two dosing regimens (3.0 mg/kg and 5.0 mg/kg) against placebo, administered monthly for 12 doses. The trial's primary endpoint focuses on steroid reduction, with secondary endpoints measuring sarcoidosis symptoms and lung function. Topline results are expected in Q3 2025.
Efzofitimod, a first-in-class biologic immunomodulator, targets interstitial lung disease by selectively modulating activated myeloid cells through neuropilin-2, potentially offering a safer alternative to current steroid-based treatments.
aTyr Pharma (NASDAQ:ATYR) ha annunciato il completamento dell'ultima visita del paziente nel suo studio di Fase 3 EFZO-FIT鈩� per efzofitimod nei pazienti con sarcoidosi polmonare. Lo studio, che rappresenta il primo studio globale di Fase 3 e il pi霉 grande trial interventistico sulla sarcoidosi, ha arruolato 268 soggetti in diversi centri negli Stati Uniti, Europa, Giappone e Brasile.
Lo studio della durata di 52 settimane valuta due regimi posologici (3,0 mg/kg e 5,0 mg/kg) rispetto al placebo, somministrati mensilmente per 12 dosi. L'end point primario dello studio 猫 la riduzione degli steroidi, mentre gli end point secondari misurano i sintomi della sarcoidosi e la funzionalit脿 polmonare. I risultati principali sono attesi nel terzo trimestre del 2025.
Efzofitimod, un immunomodulatore biologico di prima classe, agisce sulle malattie polmonari interstiziali modulando selettivamente le cellule mieloidi attivate tramite neuropilina-2, offrendo potenzialmente un'alternativa pi霉 sicura ai trattamenti attuali a base di steroidi.
aTyr Pharma (NASDAQ:ATYR) ha anunciado la finalizaci贸n de la 煤ltima visita del paciente en su estudio de Fase 3 EFZO-FIT鈩� para efzofitimod en pacientes con sarcoidosis pulmonar. El estudio, que es el primer ensayo global de Fase 3 y el mayor ensayo intervencionista de sarcoidosis, incluy贸 a 268 sujetos en m煤ltiples centros de EE. UU., Europa, Jap贸n y Brasil.
El estudio de 52 semanas eval煤a dos reg铆menes de dosificaci贸n (3.0 mg/kg y 5.0 mg/kg) frente a placebo, administrados mensualmente durante 12 dosis. El objetivo principal del ensayo es la reducci贸n de esteroides, mientras que los objetivos secundarios miden los s铆ntomas de sarcoidosis y la funci贸n pulmonar. Se esperan resultados principales en el tercer trimestre de 2025.
Efzofitimod, un inmunomodulador biol贸gico de primera clase, se dirige a la enfermedad pulmonar intersticial modulando selectivamente las c茅lulas mieloides activadas a trav茅s de neuropilina-2, ofreciendo potencialmente una alternativa m谩s segura a los tratamientos actuales basados en esteroides.
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aTyr Pharma (NASDAQ:ATYR) a annonc茅 la fin de la derni猫re visite des patients dans son essai de phase 3 EFZO-FIT鈩� portant sur l鈥檈fzofitimod chez des patients atteints de sarco茂dose pulmonaire. Cet essai, qui est le premier essai mondial de phase 3 et le plus grand essai interventionnel sur la sarco茂dose, a inclus 268 sujets r茅partis dans plusieurs centres aux 脡tats-Unis, en Europe, au Japon et au Br茅sil.
Cette 茅tude de 52 semaines 茅value deux sch茅mas posologiques (3,0 mg/kg et 5,0 mg/kg) compar茅s 脿 un placebo, administr茅s mensuellement pour 12 doses. Le crit猫re principal porte sur la r茅duction des st茅ro茂des, tandis que les crit猫res secondaires mesurent les sympt么mes de la sarco茂dose et la fonction pulmonaire. Les r茅sultats principaux sont attendus au troisi猫me trimestre 2025.
Efzofitimod, un immunomodulateur biologique de premi猫re classe, cible la maladie pulmonaire interstitielle en modulant s茅lectivement les cellules my茅lo茂des activ茅es via le neuropiline-2, offrant potentiellement une alternative plus s没re aux traitements actuels 脿 base de st茅ro茂des.
aTyr Pharma (NASDAQ:ATYR) hat den Abschluss des letzten Patientenbesuchs in seiner Phase-3-Studie EFZO-FIT鈩� f眉r Efzofitimod bei Patienten mit pulmonaler Sarkoidose bekanntgegeben. Die Studie, die die erste globale Phase-3-Studie und die gr枚脽te interventionelle Sarkoidose-Studie ist, rekrutierte 268 Probanden an mehreren Standorten in den USA, Europa, Japan und Brasilien.
Die 52-w枚chige Studie bewertet zwei Dosierungsschemata (3,0 mg/kg und 5,0 mg/kg) im Vergleich zu Placebo, die monatlich 眉ber 12 Dosen verabreicht werden. Der prim盲re Endpunkt der Studie ist die Steroidreduktion, sekund盲re Endpunkte messen Sarkoidose-Symptome und Lungenfunktion. Die Hauptresultate werden im dritten Quartal 2025 erwartet.
Efzofitimod, ein neuartiger biologischer Immunmodulator, zielt auf interstitielle Lungenerkrankungen ab, indem es aktivierte myeloide Zellen selektiv 眉ber Neuropilin-2 moduliert und somit m枚glicherweise eine sicherere Alternative zu den derzeitigen steroidbasierten Behandlungen bietet.
- First-ever global Phase 3 and largest interventional study for sarcoidosis treatment
- Novel mechanism of action that may reduce steroid burden without immune suppression
- Broad international study coverage across US, Europe, Japan, and Brazil
- Significant enrollment of 268 subjects completed
- Results not yet available - success of the treatment still uncertain
- Complex 52-week study duration may impact patient retention and data quality
Insights
aTyr's completion of Phase 3 sarcoidosis trial represents crucial near-term catalyst with potential to transform treatment landscape.
The completion of patient visits in aTyr's Phase 3 EFZO-FIT鈩� study represents a significant milestone in the clinical development of efzofitimod for pulmonary sarcoidosis. This achievement marks the culmination of the largest interventional study ever conducted for this indication, with 268 patients enrolled across multiple international sites.
Pulmonary sarcoidosis represents a substantial unmet medical need. Current treatment relies heavily on corticosteroids, which carry significant side effect burdens including weight gain, mood disturbances, osteoporosis, and metabolic dysfunction. The primary endpoint of steroid reduction is thus clinically meaningful and patient-centered.
Efzofitimod's mechanism as a tRNA synthetase-derived immunomodulator that selectively targets activated myeloid cells via neuropilin-2 represents a novel therapeutic approach. This mechanism potentially offers inflammation resolution without broad immunosuppression鈥攁 crucial differentiation from current options.
The 52-week study design with monthly intravenous dosing incorporates a forced steroid taper protocol, which provides a rigorous test of efzofitimod's steroid-sparing effects. The inclusion of secondary endpoints measuring sarcoidosis symptoms and lung function will provide comprehensive efficacy data.
With topline results expected this quarter, this represents an imminent catalyst for aTyr. Success could position efzofitimod as the first disease-modifying therapy specifically developed for sarcoidosis, potentially transforming the treatment paradigm for this challenging interstitial lung disease.
Topline results expected in the third quarter of 2025.
SAN DIEGO, July 22, 2025 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: ATYR) (鈥渁Tyr鈥� or the 鈥淐ompany鈥�), 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 last patient has completed their last visit in the Company鈥檚 Phase 3 EFZO-FIT鈩� study of its lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis, a major form of interstitial lung disease. The Company expects to report topline results from this study in the third quarter of 2025.
鈥淐ompleting this first-ever global Phase 3 and largest interventional study conducted for patients with sarcoidosis to date is a major accomplishment for aTyr and the broader sarcoidosis community,鈥� said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. 鈥淐urrent treatment options for sarcoidosis are limited and rely heavily on the use of oral corticosteroids, which often have significant side effects. This study provides an opportunity to evaluate a treatment such as efzofitimod that may have the potential to reduce steroid burden and improve quality of life in patients with this chronic disease. We thank the patients, investigators, patient advocacy organizations and our partner Kyorin Pharmaceutical Co., Ltd. for helping to make this study possible, and we look forward to sharing topline results later this quarter.鈥�
About the EFZO-FIT鈩� Study
EFZO-FIT鈩� is a global Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. The 52-week study consists of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously once a month for a total of 12 doses. The study enrolled 268 subjects with pulmonary sarcoidosis at multiple centers in the United States, Europe, Japan and Brazil. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction. Secondary endpoints include measures of sarcoidosis symptoms and lung function. More information is available at (NCT05415137).
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鈩� study in patients with pulmonary sarcoidosis, a major form of ILD, and in the Phase 2 EFZO-CONNECT鈩� 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鈥檚 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鈥檚 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 .
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," 鈥渂elieves,鈥� 鈥渄esigned,鈥� 鈥渃ould,鈥� 鈥渃an,鈥� 鈥渆xpects,鈥� 鈥渋ntends,鈥� 鈥渕ay,鈥� 鈥減lans,鈥� 鈥減otential,鈥� 鈥渨ill,鈥� 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 potential therapeutic benefits and applications of efzofitimod; timelines and plans with respect to certain development activities (such as the timing of receipt of topline data) of our Phase 3 EFZO-FIT鈩� study; 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, 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 regarding geopolitical and macroeconomic events, risks associated with the discovery, development and regulation of efzofitimod, the risks associated with clinical trials generally, 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
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FAQ
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