aTyr Pharma Announces Publication Demonstrating Efficacy of Efzofitimod in Pulmonary Sarcoidosis in the European Respiratory Journal
aTyr Pharma (Nasdaq: ATYR) announced the publication of a post hoc analysis of their Phase 1b/2a clinical trial for efzofitimod in pulmonary sarcoidosis patients in the European Respiratory Journal. The analysis showed a statistically significant difference in time-to-first-relapse for corticosteroid use between therapeutic (3.0 and 5.0 mg/kg efzofitimod) and subtherapeutic (1.0 mg/kg efzofitimod and placebo) groups.
Key findings include:
- 54.4% of patients in the subtherapeutic group relapsed following corticosteroid taper, compared to only 7.7% in the therapeutic group
- Median time-to-first-relapse in the subtherapeutic group was 126 days, while only one of 17 patients in the therapeutic group relapsed by the end of the study (p=0.017)
Efzofitimod is currently being investigated in the global pivotal Phase 3 EFZO-FIT™ study in 268 pulmonary sarcoidosis patients.
aTyr Pharma (Nasdaq: ATYR) ha annunciato la pubblicazione di un'analisi post hoc del loro trial clinico di Fase 1b/2a per efzofitimod in pazienti con sarcoidosi polmonare, pubblicata nell'European Respiratory Journal. L'analisi ha mostrato una differenza statisticamente significativa nel tempo fino al primo rilascio per l'uso di corticosteroidi tra i gruppi terapeutico (3,0 e 5,0 mg/kg di efzofitimod) e subterapeutico (1,0 mg/kg di efzofitimod e placebo).
I risultati chiave includono:
- Il 54,4% dei pazienti nel gruppo subterapeutico ha avuto una ricaduta dopo la riduzione dei corticosteroidi, rispetto al solo 7,7% nel gruppo terapeutico.
- Il tempo mediano fino alla prima ricaduta nel gruppo subterapeutico era di 126 giorni, mentre solo uno dei 17 pazienti nel gruppo terapeutico ha avuto una ricaduta entro la fine dello studio (p=0.017).
Efzofitimod è attualmente oggetto di indagine nello studio globale di Fase 3 EFZO-FIT™ su 268 pazienti con sarcoidosi polmonare.
aTyr Pharma (Nasdaq: ATYR) anunció la publicación de un análisis post hoc de su ensayo clínico de Fase 1b/2a para efzofitimod en pacientes con sarcoidosis pulmonar en el European Respiratory Journal. El análisis mostró una diferencia estadísticamente significativa en el tiempo hasta la primera recaída en el uso de corticosteroides entre los grupos terapéuticos (3.0 y 5.0 mg/kg de efzofitimod) y subterapéuticos (1.0 mg/kg de efzofitimod y placebo).
Los hallazgos clave incluyen:
- El 54.4% de los pacientes en el grupo subterapéutico recayó tras la reducción de corticosteroides, en comparación con solo el 7.7% en el grupo terapéutico.
- El tiempo medio hasta la primera recaída en el grupo subterapéutico fue de 126 días, mientras que solo uno de 17 pacientes en el grupo terapéutico recayó al final del estudio (p=0.017).
Efzofitimod se está investigando actualmente en el estudio pivotal de Fase 3 EFZO-FIT™ en 268 pacientes con sarcoidosis pulmonar.
aTyr Pharma(Nasdaq: ATYR)는 유럽 호흡기 저널에 폐사르코이드 환자를 위한 efzofitimod의 1b/2a 단계 임상 시험에 대한 후속 분석의 발표를 알렸습니다. 분석 결과, 치료 그룹(3.0 및 5.0 mg/kg efzofitimod)과 비치료 그룹(1.0 mg/kg efzofitimod 및 위약) 간의 corticosteroid 사용에 대한 첫 번째 재발까지의 시간에 통계적으로 유의미한 차이가 있음을 보여주었습니다.
주요 발견 내용은 다음과 같습니다:
- 비치료 그룹의 54.4%의 환자가 corticosteroid 감소 후 재발한 반면, 치료 그룹에서는 단 7.7%만이 재발했습니다.
- 비치료 그룹의 첫 번째 재발까지의 중앙 시간은 126일이었고, 치료 그룹의 17명 중 단 한 명만이 연구 종료 시점에 재발했습니다(p=0.017).
Efzofitimod는 현재 268명의 폐사르코이드 환자를 대상으로 한 글로벌 주요 3상 연구 EFZO-FIT™에서 조사되고 있습니다.
aTyr Pharma (Nasdaq: ATYR) a annoncé la publication d'une analyse post hoc de leur essai clinique de Phase 1b/2a pour efzofitimod chez des patients atteints de sarcoïdose pulmonaire dans le European Respiratory Journal. L'analyse a montré une différence statistiquement significative dans le temps jusqu'à la première rechute pour l'utilisation de corticostéroïdes entre les groupes thérapeutiques (3,0 et 5,0 mg/kg d'efzofitimod) et subthérapeutiques (1,0 mg/kg d'efzofitimod et placebo).
Les principales conclusions comprennent :
- 54,4% des patients du groupe subthérapeutique ont fait une rechute après la réduction des corticostéroïdes, contre seulement 7,7% dans le groupe thérapeutique.
- Le temps médian jusqu'à la première rechute dans le groupe subthérapeutique était de 126 jours, tandis qu'un seul des 17 patients du groupe thérapeutique a rechuté à la fin de l'étude (p=0,017).
Efzofitimod fait actuellement l'objet d'une étude de Phase 3 EFZO-FIT™ à l'échelle mondiale portant sur 268 patients atteints de sarcoïdose pulmonaire.
aTyr Pharma (Nasdaq: ATYR) hat die Veröffentlichung einer post-hoc Analyse ihrer klinischen Phase 1b/2a Studie zu efzofitimod bei Patienten mit pulmonaler Sarkoidose im European Respiratory Journal bekannt gegeben. Die Analyse zeigte einen statistisch signifikanten Unterschied in der Zeit bis zur ersten Rückfall in Bezug auf die Verwendung von Kortikosteroiden zwischen den therapeutischen Gruppen (3,0 und 5,0 mg/kg efzofitimod) und subtherapeutischen Gruppen (1,0 mg/kg efzofitimod und Placebo).
Wichtige Ergebnisse umfassen:
- 54,4% der Patienten in der subtherapeutischen Gruppe erlitten einen Rückfall nach der Reduzierung von Kortikosteroiden, im Vergleich zu nur 7,7% in der therapeutischen Gruppe.
- Die mediane Zeit bis zum ersten Rückfall in der subtherapeutischen Gruppe betrug 126 Tage, während nur einer von 17 Patienten in der therapeutischen Gruppe bis zum Ende der Studie einen Rückfall erlitt (p=0.017).
Efzofitimod wird derzeit in der globalen entscheidenden Phase 3 EFZO-FIT™-Studie bei 268 Patienten mit pulmonaler Sarkoidose untersucht.
- Statistically significant difference in time-to-first-relapse for corticosteroid use between therapeutic and subtherapeutic groups
- Only 7.7% of patients in the therapeutic group relapsed compared to 54.4% in the subtherapeutic group
- Efzofitimod received orphan drug designation in the U.S., E.U. and Japan for sarcoidosis
- Efzofitimod received Fast Track designation in the U.S. for pulmonary sarcoidosis
- Ongoing global pivotal Phase 3 EFZO-FIT™ study with 268 pulmonary sarcoidosis patients
- None.
Insights
This publication presents compelling evidence for efzofitimod's efficacy in treating pulmonary sarcoidosis, a challenging form of interstitial lung disease. The post hoc analysis of the Phase 1b/2a study reveals statistically significant differences in time-to-first-relapse between therapeutic and subtherapeutic groups.
Key findings include:
- Only
7.7% of patients in the therapeutic group relapsed, compared to54.4% in the subtherapeutic group - Median time-to-first-relapse was 126 days in the subtherapeutic group, while only 1 of 17 patients in the therapeutic group relapsed by study end (p=0.017)
These results suggest efzofitimod's potential as a steroid-sparing treatment, addressing a critical need in sarcoidosis management. The ongoing Phase 3 EFZO-FIT™ study (NCT05415137) with 268 patients will be important in confirming these promising results. If successful, efzofitimod could represent a paradigm shift in sarcoidosis treatment, offering disease modification without the long-term toxicity associated with corticosteroids.
The publication of these positive results in the European Respiratory Journal is a significant milestone for aTyr Pharma (NASDAQ: ATYR). It strengthens the company's position in the competitive biotech landscape and potentially increases the likelihood of successful Phase 3 trials and eventual FDA approval.
Key financial implications:
- Increased investor confidence may lead to improved stock performance
- Enhanced potential for partnerships or licensing deals
- Possible increase in market cap, currently at
$131,127,423
The global sarcoidosis treatment market is projected to reach
Post hoc analysis of Phase 1b/2a study demonstrated statistically significant difference in time-to-first-relapse for corticosteroid use in therapeutic (3.0 and 5.0 mg/kg efzofitimod) vs subtherapeutic (1.0 mg/kg efzofitimod and placebo) groups.
SAN DIEGO, Oct. 02, 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 publication of a post hoc analysis of the Phase 1b/2a clinical trial of its lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis, a major form of interstitial lung disease, in the European Respiratory Journal. The publication, entitled, “Therapeutic Doses of Efzofitimod Demonstrate Efficacy in Pulmonary Sarcoidosis,” is available on the Journal’s website and at: https://doi.org/10.1183/23120541.00536-2024.
“Oral corticosteroids are considered first-line therapy in patients with sarcoidosis. However, while they may help improve symptoms, long-term use is often associated with significant toxicity and reduced quality of life,” said Ogugua Ndili Obi, M.D., M.P.H., M.Sc., Associate Professor of Medicine and Clinical Director of the Sarcoidosis Program at the Brody School of Medicine at East Carolina University and lead author of the paper. “Many patients find it difficult to taper and/or maintain reduced steroid doses, as symptoms often flare or disease remains refractory, so the low relapse rate seen for the efzofitimod therapeutic group and a significant difference in time-to-first-relapse in this 24-week study is impressive. The ability of a therapy such as efzofitimod to maintain disease control while decreasing or discontinuing steroid use entirely would be clinically important and very meaningful to patients.”
The publication reports findings from a post hoc analysis of a pre-specified endpoint in the Phase 1b/2a randomized, double-blind, placebo-controlled, 24-week study of efzofitimod in 37 patients with pulmonary sarcoidosis receiving oral corticosteroid treatment who underwent a forced steroid taper in the first 8 weeks of the study. In this pooled analysis, the time-to-first-relapse was significantly shorter in the subtherapeutic group (1.0 mg/kg efzofitimod and placebo) than in the therapeutic group (3.0 and 5.0 mg/kg efzofitimod). The median time-to-first-relapse in the subtherapeutic group was 126 days, whereas only one of 17 patients in the therapeutic group had relapsed by the end of the study (p=0.017). Furthermore,
“We continue to publish data from our Phase 1b/2a study that further demonstrate the efficacy of efzofitimod in pulmonary sarcoidosis patients and positions this first-in-class immunomodulator as a promising new treatment option that can reduce or avoid steroid-related toxicity,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “We believe we are on the cusp of a paradigm shift in the treatment for sarcoidosis, where patients may have the opportunity to receive clinically validated therapies that can treat their underlying disease without incurring added harm.”
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. Efzofitimod is currently being investigated in the global pivotal Phase 3 EFZO-FIT™ study in 268 pulmonary sarcoidosis patients (NCT05415137). Efzofitimod has received orphan drug designation in the U.S., E.U. and Japan for sarcoidosis and Fast Track designation in the U.S. for pulmonary sarcoidosis.
Phase 1b/2a Clinical Trial in Patients with Pulmonary Sarcoidosis
The Phase 1b/2a study was a randomized, double-blind, placebo-controlled, multiple-ascending dose clinical trial in 37 patients with pulmonary sarcoidosis. The trial consisted of three cohorts testing doses of 1.0 mg/kg, 3.0 mg/kg and 5.0 mg/kg of efzofitimod or placebo, dosed intravenously every month for six months. The primary objective of the study was to evaluate the safety, tolerability, immunogenicity and pharmacokinetic profile of multiple doses of efzofitimod compared to placebo. Secondary objectives included the potential steroid-sparing effects of efzofitimod, in addition to other exploratory assessments of efficacy, such as lung function. (NCT03824392)
About Pulmonary Sarcoidosis
Sarcoidosis is an immune-mediated disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body, predominantly in the lungs. Almost 200,000 Americans live with pulmonary sarcoidosis and the prognosis ranges from benign and self-limiting to chronic, debilitating disease, with 1 in 5 cases resulting in fibrosis, or scarring, of the lungs, which causes permanent loss of lung function and in many cases death. Current treatment options include corticosteroids and other immunosuppressive therapies, which have limited efficacy and are associated with serious side effects that many patients cannot tolerate long-term.
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,” “can,” “expects,” “intends,” “may,” “plans,” “potential,” “will,” “would,” 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 benefits and therapeutic application of efzofitimod as compared to the potential benefits and drawbacks of the current standard of care for sarcoidosis, timelines and plans with respect to certain development activities (such as the timing of data from clinical trials) and our publishing of additional data, the potential benefits of efzofitimod to be a new treatment option that can reduce or avoid steroid-related toxicity 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, risks associated with clinical trials and their resulting data 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 | |
| Director, Investor Relations and Public Affairs | |
| adunston@atyrpharma.com |
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