aTyr Pharma Announces Fourth Positive DSMB Review for Efzofitimod in Phase 3 EFZO-FIT™ Study in Pulmonary Sarcoidosis
aTyr Pharma announced a fourth positive safety review by an independent data and safety monitoring board (DSMB) for its Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis patients. The DSMB recommended continuing the study without modifications, reinforcing the therapeutic candidate's favorable safety profile.
The global Phase 3 study involves 268 subjects across multiple centers in the United States, Europe, Japan, and Brazil. The 52-week trial includes three parallel cohorts randomized to receive either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo, administered intravenously monthly for 12 doses. The study's primary endpoint focuses on steroid reduction, with secondary endpoints measuring lung function and sarcoidosis symptoms.
Efzofitimod is a first-in-class biologic immunomodulator targeting interstitial lung disease (ILD). The company expects to report topline results in Q3 2025.
aTyr Pharma ha annunciato un quarto positivo controllo di sicurezza da parte di un comitato indipendente di monitoraggio dei dati e della sicurezza (DSMB) per il suo studio di Fase 3 EFZO-FIT™ su efzofitimod in pazienti con sarcoidosi polmonare. Il DSMB ha raccomandato di continuare lo studio senza modifiche, rafforzando il profilo di sicurezza favorevole del candidato terapeutico.
Lo studio globale di Fase 3 coinvolge 268 soggetti in diversi centri negli Stati Uniti, in Europa, in Giappone e in Brasile. La sperimentazione di 52 settimane include tre coorti parallele randomizzate a ricevere 3.0 mg/kg o 5.0 mg/kg di efzofitimod o un placebo, somministrati per via endovenosa mensilmente per 12 dosi. L'obiettivo primario dello studio si concentra sulla riduzione degli steroidi, con obiettivi secondari che misurano la funzione polmonare e i sintomi della sarcoidosi.
Efzofitimod è un immunomodulatore biologico di prima classe che mira alle malattie polmonari interstiziali (ILD). L'azienda prevede di riportare i risultati preliminari nel terzo trimestre del 2025.
aTyr Pharma anunció una cuarta revisión positiva de seguridad por parte de un comité independiente de monitoreo de datos y seguridad (DSMB) para su estudio de Fase 3 EFZO-FIT™ sobre efzofitimod en pacientes con sarcoidosis pulmonar. El DSMB recomendó continuar el estudio sin modificaciones, reforzando el perfil de seguridad favorable del candidato terapéutico.
El estudio global de Fase 3 involucra 268 sujetos en múltiples centros en los Estados Unidos, Europa, Japón y Brasil. El ensayo de 52 semanas incluye tres cohortes paralelas randomizadas para recibir 3.0 mg/kg o 5.0 mg/kg de efzofitimod o placebo, administrados por vía intravenosa mensualmente durante 12 dosis. El objetivo primario del estudio se centra en la reducción de esteroides, con objetivos secundarios que miden la función pulmonar y los síntomas de la sarcoidosis.
Efzofitimod es un inmunomodulador biológico de primera clase que se dirige a la enfermedad pulmonar intersticial (ILD). La empresa espera informar los resultados preliminares en el tercer trimestre de 2025.
aTyr Pharma는 폐 사르코이드증 환자를 대상으로 한 efzofitimod의 3상 EFZO-FIT™ 연구에 대해 독립적인 데이터 및 안전성 모니터링 위원회(DSMB)로부터 네 번째 긍정적인 안전성 검토를 발표했습니다. DSMB는 연구를 수정 없이 계속 진행할 것을 권장하며, 치료 후보의 유리한 안전성 프로필을 강화했습니다.
이 글로벌 3상 연구는 미국, 유럽, 일본 및 브라질의 여러 센터에서 268명의 피험자를 포함합니다. 52주간의 시험은 efzofitimod 또는 위약을 각각 3.0 mg/kg 또는 5.0 mg/kg 투여받는 세 개의 병렬 코호트로 무작위 배정됩니다. 연구의 주요 목표는 스테로이드 감소에 중점을 두고 있으며, 부차적인 목표는 폐 기능과 사르코이드증 증상을 측정합니다.
Efzofitimod는 간질성 폐 질환(ILD)을 겨냥한 최초의 생물학적 면역 조절제입니다. 회사는 2025년 3분기에 주요 결과를 보고할 것으로 예상하고 있습니다.
aTyr Pharma a annoncé une quatrième évaluation positive de la sécurité par un comité indépendant de surveillance des données et de la sécurité (DSMB) pour son étude de phase 3 EFZO-FIT™ sur l'efzofitimod chez des patients atteints de sarcoïdose pulmonaire. Le DSMB a recommandé de poursuivre l'étude sans modifications, renforçant ainsi le profil de sécurité favorable du candidat thérapeutique.
L'étude mondiale de phase 3 implique 268 sujets dans plusieurs centres aux États-Unis, en Europe, au Japon et au Brésil. L'essai de 52 semaines comprend trois cohortes parallèles randomisées pour recevoir soit 3,0 mg/kg soit 5,0 mg/kg d'efzofitimod ou un placebo, administrés par voie intraveineuse mensuellement pendant 12 doses. L'objectif principal de l'étude se concentre sur la réduction des stéroïdes, avec des objectifs secondaires mesurant la fonction pulmonaire et les symptômes de la sarcoïdose.
L'efzofitimod est un immunomodulateur biologique de première classe ciblant les maladies pulmonaires interstitielles (ILD). La société prévoit de communiquer les résultats préliminaires au troisième trimestre 2025.
aTyr Pharma hat eine vierte positive Sicherheitsüberprüfung durch ein unabhängiges Daten- und Sicherheitsüberwachungskomitee (DSMB) für seine Phase-3-Studie EFZO-FIT™ zu efzofitimod bei Patienten mit pulmonaler Sarkoidose angekündigt. Das DSMB empfahl, die Studie ohne Änderungen fortzusetzen und stärkt damit das günstige Sicherheitsprofil des therapeutischen Kandidaten.
Die globale Phase-3-Studie umfasst 268 Probanden in mehreren Zentren in den Vereinigten Staaten, Europa, Japan und Brasilien. Die 52-wöchige Studie beinhaltet drei parallele Kohorten, die randomisiert entweder 3,0 mg/kg oder 5,0 mg/kg efzofitimod oder ein Placebo erhalten, das monatlich über 12 Dosen intravenös verabreicht wird. Der primäre Endpunkt der Studie konzentriert sich auf die Reduzierung von Steroiden, während sekundäre Endpunkte die Lungenfunktion und die Symptome der Sarkoidose messen.
Efzofitimod ist ein biologischer Immunmodulator der ersten Klasse, der auf interstitielle Lungenerkrankungen (ILD) abzielt. Das Unternehmen erwartet, die vorläufigen Ergebnisse im dritten Quartal 2025 zu berichten.
- Fourth consecutive positive DSMB safety review
- No safety concerns identified in Phase 3 trial
- Study proceeding as planned without modifications
- Large-scale global trial with 268 patients enrolled
- Final trial results still pending until Q3 2025
- Efficacy data not yet available
Insights
The fourth positive DSMB review for aTyr's Phase 3 efzofitimod trial represents a significant de-risking event in the drug's development pathway. With 268 patients enrolled and approximately three-quarters through this 52-week study, the continued clean safety profile strengthens efzofitimod's potential as a chronic maintenance therapy for pulmonary sarcoidosis.
This safety milestone is particularly important considering efzofitimod's mechanism as a first-in-class biologic immunomodulator that selectively targets activated myeloid cells through neuropilin-2. The drug's potential advantage lies in resolving inflammation without broad immune suppression – a critical differentiation from current standard-of-care oral corticosteroids, which carry substantial side effects with long-term use.
The trial's primary endpoint of steroid reduction directly addresses a major unmet need in sarcoidosis treatment. Current steroid-based therapies often require trade-offs between symptom management and side effect burden, making efzofitimod's potential steroid-sparing effect especially valuable.
With topline data expected in Q3 2025, aTyr is approaching a pivotal catalyst. Success would position efzofitimod as potentially the first FDA-approved therapy specifically for pulmonary sarcoidosis, addressing a condition with treatment options and establishing aTyr's tRNA synthetase platform as a viable source of novel therapeutics.
The continued safety clearance for efzofitimod highlights the promising profile of this novel immunomodulatory approach. Unlike broad immunosuppressants, efzofitimod's selective targeting of activated myeloid cells through neuropilin-2 represents a precision medicine approach to interstitial lung disease treatment.
The successful completion of a fourth DSMB review is particularly notable given the 52-week treatment duration – significantly longer than many immunomodulator trials. This extended safety profile is important for pulmonary sarcoidosis, a chronic condition where treatment safety over time is paramount.
The trial's forced steroid taper design is methodologically robust, directly testing efzofitimod's ability to maintain disease control while reducing corticosteroid dependency. This design element addresses the core clinical challenge in sarcoidosis management – how to control inflammation while minimizing the substantial morbidity associated with long-term steroid use.
Beyond sarcoidosis, the continued safety profile supports aTyr's parallel development in systemic sclerosis-related ILD (EFZO-CONNECT™ study), suggesting potential applicability across multiple fibrotic lung conditions with shared immunopathology. This positions efzofitimod as a potential platform therapy for a spectrum of interstitial lung diseases unified by dysregulated myeloid cell activity and fibrotic progression.
No safety concerns identified by independent data and safety monitoring board (DSMB), which recommends continuation of study without any modifications.
SAN DIEGO, March 06, 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 the outcome of a fourth, pre-planned interim safety analysis conducted by an independent data and safety monitoring board (DSMB) for the ongoing Phase 3 EFZO-FIT™ study of the Company’s lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis. The DSMB recommended that the study continue without any modifications.
“The positive outcome from this fourth DSMB review for EFZO-FIT™ further reinforces our confidence in the favorable safety profile that we have seen for efzofitimod to date,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “As we look to position efzofitimod as a potential chronic, maintenance therapy for patients with pulmonary sarcoidosis that can reduce or eliminate the use of oral corticosteroids, we are pleased to see no safety concerns arise as the study progresses. We look forward to reporting topline results from EFZO-FIT™ in the third quarter of this year.”
EFZO-FIT™ is a global Phase 3 randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a 52-week study consisting 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 lung function and sarcoidosis symptoms. More information on the EFZO-FIT™ study is available at www.clinicaltrials.gov (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™ 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 "anticipate," “believes,” “designed,” “could,” “can,” “expects,” “intends,” “may,” “plans,” “potential,” “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 clinical development for efzofitimod, including the potential of efzofitimod to be a potential chronic, maintenance therapy for patients with pulmonary sarcoidosis that can reduce or eliminate the use of oral corticosteroids, and the timing and results of EFZO-FIT™. 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 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
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