aTyr Pharma Shares Plummet After Lung Disease Drug Fails Phase III Trial

Lead: Clinical-stage biotech aTyr Pharma announced on September 15 that its experimental therapy efzofitimod missed the primary endpoint in a late-stage pulmonary sarcoidosis study, sending shares tumbling over 80% to a record low.

Nut Graf: The failed trial outcome challenges aTyr’s flagship program and intensifies pressure on the company to chart a regulatory path forward. With only this asset in clinical development, investor confidence has eroded, underscoring the urgency of forthcoming FDA discussions.

Key Trial Results

• Primary endpoint: No significant reduction in mean daily corticosteroid dose at 48 weeks (2.79 mg vs. 3.52 mg placebo; p=0.3313).
• Steroid withdrawal: 53% of efzofitimod patients off steroids vs. 40% placebo, below expectations.
• Quality-of-life: Nominal improvements in King’s Sarcoidosis Questionnaire-Lung score (p=0.0479) and combined steroid withdrawal plus KSQ-Lung responder rate (p=0.0199).

Stock Market Impact

Trading on September 15 was halted premarket; upon reopening, ATYR shares plunged to approximately $1.14-near historic lows-reflecting an over-80% decline in value.

Company Response and Next Steps

aTyr plans to engage the U.S. Food and Drug Administration to review the EFZO-FIT™ data, highlight quality-of-life benefits and explore potential regulatory pathways despite the missed primary outcome.

Expert Commentary

Piper Sandler analyst Yasmeen Rahimi noted that while the primary goal was unmet, the totality of data may still warrant a regulatory filing after further discussions with authorities.

Background on Pulmonary Sarcoidosis

Pulmonary sarcoidosis, affecting roughly 200,000 Americans, causes granuloma formation in the lungs. Current standard of care relies on high-dose steroids, which carry significant long-term risks.