Atyr Pharma shares fell 1.5% in volatile premarket trade on Monday, with investors on edge ahead of topline results from its pivotal Phase 3 EFZO-FIT trial of efzofitimod. The data is expected any day and will be presented at the European Respiratory Society conference.

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The 52-week global study, which completed its last patient visits in July, enrolled 268 pulmonary sarcoidosis patients across 85 centers in nine countries. Patients were randomized to monthly intravenous doses of efzofitimod at 3 mg/kg, 5 mg/kg, or placebo. 

The primary endpoint is steroid reduction from baseline to week 48, with secondary endpoints including symptom relief and lung function. The trial design incorporated a forced steroid taper, a key measure for assessing clinical benefit.

Analysts have flagged the readout as a potential inflection point. Jefferies lifted its price target to $17 from $9 last month, citing encouraging earlier-stage data and estimating a 60% probability of statistical success. H.C. Wainwright reiterated a ‘Buy’ rating with a $35 target, Cantor Fitzgerald reaffirmed ‘Overweight’ citing strong statistical power to show steroid reduction, and Leerink Partners maintained an ‘Outperform’ rating with a $16 target.

CEO Sanjay Shukla said completing EFZO-FIT, the largest interventional study ever conducted in sarcoidosis, was a milestone for patients who currently rely heavily on corticosteroids despite serious side effects. 

The company is also studying efzofitimod in systemic sclerosis-related interstitial lung disease in its ongoing Phase 2 EFZO-CONNECT trial.

Atyr reported second-quarter results in August with cash, equivalents and investments of $83.2 million as of June 30. Subsequent to the quarter, it raised about $30.7 million through an at-the-market offering. 

The company said the balance is sufficient to fund operations for a year beyond the EFZO-FIT readout. R&D expenses were $15.4 million in the quarter, driven by trial and manufacturing costs, while G&A expenses totaled $4.9 million.

On Stocktwits, retail sentiment was ‘bullish’ amid ‘extremely high’ message volume.

One user described the range of possible outcomes as a “bear case” of $2 if the data disappoints, a “base case” of $10–$15 on decent results, and a “bull case” of $25–$35 if topline data are strong enough to spark a squeeze.

Another user said they had bought $2 call contracts to limit downside risk, noting that the position would cover losses if the stock fell toward $1.50 while leaving room to benefit if a short squeeze drove the shares higher.

Atyr Pharma’s stock has risen 66.6% so far in 2025.

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