aTyr Pharma Announces Third Positive DSMB Review for Efzofitimod in Phase 3 EFZO-FIT™ Study in Pulmonary Sarcoidosis
aTyr Pharma announced positive results from the third pre-planned interim safety analysis of its Phase 3 EFZO-FIT™ study for efzofitimod in pulmonary sarcoidosis patients. The independent Data and Safety Monitoring Board (DSMB) reviewed data from all 268 enrolled patients and recommended continuing the study without modifications.
The EFZO-FIT™ study is a global Phase 3 randomized, double-blind, placebo-controlled trial evaluating efzofitimod's efficacy and safety. The 52-week study includes three parallel cohorts randomized to receive either 3.0 mg/kg, 5.0 mg/kg of efzofitimod, or placebo monthly for 12 doses. The trial's primary endpoint is steroid reduction, with secondary endpoints measuring lung function and sarcoidosis symptoms.
aTyr Pharma ha annunciato risultati positivi dalla terza analisi intermedia di sicurezza pre-pianificata del suo studio di Fase 3 EFZO-FIT™ per efzofitimod in pazienti affetti da sarcoidosi polmonare. Il Comitato Indipendente di Monitoraggio dei Dati e della Sicurezza (DSMB) ha esaminato i dati di tutti i 268 pazienti arruolati e ha raccomandato di continuare lo studio senza modifiche.
Lo studio EFZO-FIT™ è un trial globale di Fase 3, randomizzato, in doppio cieco e controllato con placebo che valuta l'efficacia e la sicurezza di efzofitimod. Lo studio di 52 settimane include tre coorti parallele randomizzate a ricevere rispettivamente 3.0 mg/kg, 5.0 mg/kg di efzofitimod o placebo mensilmente per 12 dosi. L'endpoint primario del trial è la riduzione degli steroidi, con endpoint secondari che misurano la funzione polmonare e i sintomi della sarcoidosi.
aTyr Pharma anunció resultados positivos del tercer análisis interim preplaneado de seguridad de su estudio de Fase 3 EFZO-FIT™ para efzofitimod en pacientes con sarcoidosis pulmonar. El Comité Independiente de Monitoreo de Datos y Seguridad (DSMB) revisó los datos de los 268 pacientes inscritos y recomendó continuar el estudio sin modificaciones.
El estudio EFZO-FIT™ es un ensayo global de Fase 3, aleatorizado, doble ciego y controlado con placebo que evalúa la eficacia y seguridad de efzofitimod. El estudio de 52 semanas incluye tres cohortes paralelas aleatorizadas para recibir ya sea 3.0 mg/kg, 5.0 mg/kg de efzofitimod, o placebo mensualmente durante 12 dosis. El objetivo primario del ensayo es la reducción de esteroides, con objetivos secundarios que miden la función pulmonar y los síntomas de la sarcoidosis.
aTyr Pharma는 폐 사르코이드증 환자에 대한 efzofitimod의 3상 EFZO-FIT™ 연구의 세 번째 사전 계획된 중간 안전성 분석에서 긍정적인 결과를 발표했습니다. 독립적인 데이터 및 안전성 모니터링 위원회(DSMB)는 268명의 등록된 환자에 대한 데이터를 검토한 후 연구를 수정 없이 계속 진행할 것을 권장했습니다.
EFZO-FIT™ 연구는 efzofitimod의 효능과 안전성을 평가하는 전 세계적인 3상 무작위 이중 맹검 위약 대조 시험입니다. 52주 연구는 각각 3.0 mg/kg, 5.0 mg/kg의 efzofitimod 또는 위약을 12회 투여하는 3개의 평행 코호트로 무작위 배정됩니다. 시험의 주요 목표는 스테로이드 감소이며, 부차적인 목표는 폐 기능과 사르코이드증 증상을 측정합니다.
aTyr Pharma a annoncé des résultats positifs de la troisième analyse intermédiaire de sécurité préalablement planifiée de son étude de Phase 3 EFZO-FIT™ pour efzofitimod chez des patients atteints de sarcoïdose pulmonaire. Le Comité Indépendant de Surveillance des Données et de la Sécurité (DSMB) a examiné les données des 268 patients inscrits et a recommandé de poursuivre l'étude sans modifications.
L'étude EFZO-FIT™ est un essai mondial de Phase 3, randomisé, en double aveugle et contrôlé par placebo évaluant l'efficacité et la sécurité d'efzofitimod. L'étude de 52 semaines comprend trois cohortes parallèles randomisées pour recevoir soit 3,0 mg/kg, 5,0 mg/kg d'efzofitimod, soit un placebo mensuel pendant 12 doses. Le principal critère de jugement de l'essai est la réduction des stéroïdes, avec des critères secondaires mesurant la fonction pulmonaire et les symptômes de la sarcoïdose.
aTyr Pharma hat positive Ergebnisse aus der dritten vorgeplanten interimistischen Sicherheitsanalyse seiner Phase-3-Studie EFZO-FIT™ für efzofitimod bei Patienten mit pulmonaler Sarcoidose bekannt gegeben. Das unabhängige Data and Safety Monitoring Board (DSMB) hat die Daten aller 268 eingeschriebenen Patienten überprüft und empfohlen, die Studie ohne Änderungen fortzusetzen.
Die EFZO-FIT™-Studie ist eine globale Phase-3-Studie, die randomisiert, doppelblind und plazebokontrolliert ist und die Wirksamkeit und Sicherheit von efzofitimod bewertet. Die 52-wöchige Studie umfasst drei parallele Kohorten, die randomisiert entweder 3,0 mg/kg, 5,0 mg/kg efzofitimod oder monatlich über 12 Dosen ein Placebo erhalten. Der primäre Endpunkt der Studie ist die Steroidreduktion, während sekundäre Endpunkte die Lungenfunktion und die Symptome von Sarcoidose messen.
- Third consecutive positive DSMB safety review for Phase 3 trial
- Study successfully enrolled 268 patients across multiple global centers
- Trial proceeding without required modifications, indicating favorable safety profile
- None.
Insights
Independent data and safety monitoring board (DSMB) review includes all 268 patients who have been enrolled in the study and recommends continuation of study without any modifications.
SAN DIEGO, Dec. 10, 2024 (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 third, 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.
“We are pleased to report yet another positive safety review for efzofitimod, which includes all 268 patients that have been enrolled in our global pivotal Phase 3 EFZO-FIT™ study,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “Safety is paramount when looking to provide a disease modifying treatment for a chronic condition such as pulmonary sarcoidosis, where reducing or replacing a toxic standard of care such as oral corticosteroids could be highly meaningful and improve quality of life for patients.”
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 an improved treatment for pulmonary sarcoidosis over the standard of care and our expectations with respect to the conduct, 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
Director, Investor Relations and Public Affairs
adunston@atyrpharma.com
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