aTyr Pharma Stock Plummets Over 80% on Failed Sarcoidosis Trial
Phase 3 study of efzofitimod misses primary endpoint, overshadowing positive secondary data and triggering massive investor sell-off.
Shares of aTyr Pharma (ATYR) cratered on Tuesday, collapsing by more than 80% after the biotechnology company announced its pivotal Phase 3 EFZO-FIT™ study for pulmonary sarcoidosis failed to meet its primary goal. The catastrophic market reaction came despite the company highlighting some positive secondary findings, which were not enough to assuage investor fears over the drug's regulatory prospects.
The study evaluated efzofitimod, a novel immunomodulator, for its ability to help patients with pulmonary sarcoidosis reduce their dependence on oral corticosteroids (OCS). The primary endpoint was the change in the average daily OCS dose from the beginning of the study to week 48. Efzofitimod did not demonstrate a statistically significant benefit over a placebo in this crucial measure, with a p-value of 0.3313, .
In its announcement, aTyr Pharma pointed to several positive secondary outcomes. For instance, 52.6% of patients treated with the 5.0 mg/kg dose of efzofitimod achieved a complete withdrawal from steroids by the end of the study, compared to 40.2% in the placebo group. The company also noted a clinical improvement in the King's Sarcoidosis Questionnaire (KSQ)-Lung score for patients receiving the drug. However, due to the trial's hierarchical statistical design, these secondary findings are considered nominal because the primary endpoint was not met, a fact that poses a .
The market's response was swift and brutal. The stock's collapse was accompanied by a wave of analyst downgrades and has drawn the attention of at least one national shareholders' rights firm, which has into whether the company may have misled investors about the drug's potential. The higher-than-expected performance of the placebo group in tapering steroid use was a key factor in the trial's failure to achieve its primary goal.
Despite the setback, aTyr Pharma stated it plans to engage with the U.S. Food and Drug Administration (FDA) to discuss the path forward for efzofitimod. Nevertheless, the failure of the EFZO-FIT study's primary endpoint represents a major blow to the company and its lead drug candidate, creating significant uncertainty about its future.