aTyr Pharma Announces Second Positive DSMB Review for Efzofitimod in Phase 3 EFZO-FIT™ Study in Pulmonary Sarcoidosis
aTyr Pharma has announced that an independent Data and Safety Monitoring Board (DSMB) has recommended the continuation of its Phase 3 EFZO-FIT™ study without modifications based on a second interim analysis. The study evaluates the efficacy and safety of their lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis.
Efzofitimod aims to provide a safer, non-steroidal treatment option compared to current corticosteroid treatments, which carry significant side effects. The global study includes 264 participants across multiple countries and focuses on steroid reduction as the primary endpoint, with secondary endpoints assessing lung function and sarcoidosis symptoms.
The DSMB’s positive review supports efzofitimod's favorable safety profile. aTyr Pharma continues to explore efzofitimod's potential in treating interstitial lung diseases (ILD), offering hope for safer, more effective treatment options.
- DSMB recommends continuation of EFZO-FIT™ study without modifications.
- Favorable safety profile of efzofitimod supported by second interim analysis.
- Phase 3 study aims to enroll 264 subjects globally.
- Primary endpoint is steroid reduction, promising a safer alternative to current treatments.
- Potential non-steroidal treatment option for pulmonary sarcoidosis with fewer side effects.
- Global study includes multiple centers in the US, Europe, Japan, and Brazil.
- No efficacy data provided in the interim analysis.
- Significant reliance on future DSMB reviews for continued positive assessments.
- High competition in the market for ILD treatments.
- Enrollment and study completion timelines not specified, potential delays in reaching the market.
Insights
The second positive DSMB review for efzofitimod in aTyr Pharma's Phase 3 EFZO-FIT™ study highlights its favorable safety profile, which is significant in the context of pulmonary sarcoidosis treatment. Current treatments often involve oral corticosteroids that come with notable side effects and toxicity, making a non-steroidal option like efzofitimod highly appealing. The DSMB's endorsement to continue without modifications implies efzofitimod is well-tolerated, a critical factor for regulatory approval and future patient compliance.
In the broader context of interstitial lung diseases (ILDs), which include pulmonary sarcoidosis, the development of a first-in-class biologic immunomodulator is promising. Efzofitimod's mechanism, targeting neuropilin-2 to modulate activated myeloid cells and resolve inflammation without immune suppression, is innovative and could potentially revolutionize ILD treatment. The study's design, which includes a forced steroid taper and evaluation of both steroid reduction and lung function, suggests a comprehensive approach to demonstrating both symptom relief and broader health benefits. Investors should monitor upcoming DSMB reviews and final trial results closely.
The DSMB's recommendation to continue the EFZO-FIT™ study without modifications is a positive signal for aTyr Pharma from a financial perspective. This news suggests that efzofitimod is progressing steadily through Phase 3 trials, which are typically the most expensive and critical phase before a drug can seek regulatory approval. A smooth advancement through this phase minimizes unexpected costs and delays.
Given the high unmet need and the potential market size for ILD treatments, successful Phase 3 trials could significantly increase aTyr Pharma's valuation. Investors should consider the trial's primary endpoint, steroid reduction, which if met, could position efzofitimod as a preferred alternative to current corticosteroid treatments. However, it's important also to consider the competitive landscape and potential regulatory hurdles. For now, this news appears to boost confidence in the company's development pipeline.
With the ongoing EFZO-FIT™ study reflecting positive interim safety outcomes, the market potential for efzofitimod in the treatment of pulmonary sarcoidosis looks increasingly promising. The current reliance on oral corticosteroids for managing this condition, which can lead to significant side effects, underscores a substantial gap in the market that efzofitimod might fill if proven effective and safe.
The study's global reach, enrolling patients across the United States, Europe, Japan and Brazil, indicates a strategic approach to capture a diverse patient population, enhancing the generalizability of the trial results. Given the unmet need for safer treatments for ILDs, the successful development of efzofitimod could position aTyr Pharma advantageously in the market, potentially leading to strong sales and market penetration. Investors should keep an eye on further updates and final trial results to assess the commercial viability of this therapeutic candidate.
Independent data and safety monitoring board (DSMB) recommends continuation of study without any modifications.
Findings further support favorable safety profile of efzofitimod.
SAN DIEGO, May 14, 2024 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE) (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 that an independent data and safety monitoring board (DSMB) recommended that the ongoing Phase 3 EFZO-FIT™ study of its lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis could continue without any modifications after a second pre-planned, interim analysis.
“This second DSMB review for EFZO-FIT™ builds upon the favorable safety profile seen with efzofitimod to date. We expect to conduct additional DSMB reviews as we progress throughout the study,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. “Current standard of care for patients with pulmonary sarcoidosis includes oral corticosteroids, which can incur significant side effects and toxicity. Efzofitimod has the potential to be a safe, non-steroidal treatment option for these patients, which is greatly needed.”
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 intends to enroll up to 264 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) and www.efzofit.com.
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,” 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, the timing and results of future DSMB reviews, and our expectations with respect to the conduct, timing, enrollment, 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 |
FAQ
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