Ultragenyx Pharma stock plunges as setrusumab study fails interim analysis

Published 10/07/2025, 12:34
Ultragenyx Pharma stock plunges as setrusumab study fails interim analysis

Investing.com - Ultragenyx Pharmaceuticals (NASDAQ:RARE) stock plummeted approximately 25% in after-hours trading from its current price of $41.44 after announcing its Phase 3 Orbit study for setrusumab failed to meet predetermined interim analysis thresholds. The company, which maintains a market capitalization of $3.9 billion, has seen its shares trade between $29.59 and $60.37 over the past 52 weeks.

The company, along with its partner, revealed Thursday that the study evaluating setrusumab for Osteogenesis Imperfecta (OI) in patients aged 5-25 years will proceed to its final analysis after patients have been on therapy for at least 18 months, with results expected by year-end 2025.

The trial did not clear the predetermined p-value threshold of less than 0.01 required for the second interim analysis, necessitating continuation to the final analysis phase rather than an early conclusion.

Canaccord Genuity maintained its Buy rating and $136.00 price target on Ultragenyx despite the setback, with the firm characterizing the development as an "incremental negative" relative to expectations. This target represents the highest among analyst projections, which range from $39 to $136, with the overall analyst consensus remaining strongly bullish at 1.35 (where 1 is Strong Buy and 5 is Strong Sell).

The research firm suggested the steep stock decline was "way, way overdone," noting that its discounted cash flow analysis values Ultragenyx’s base business of currently approved products plus collaboration and license revenue at approximately $40 per share. For deeper insights into Ultragenyx’s valuation and financial health metrics, access the comprehensive Pro Research Report available exclusively on InvestingPro.

In other recent news, Ultragenyx Pharmaceutical (TADAWUL:2070) Inc. announced that its Phase 3 Orbit study for setrusumab in osteogenesis imperfecta will proceed to a final analysis. The study did not meet the statistical significance threshold in its second interim analysis, which required a p-value below 0.01. Despite this, the Data Monitoring Committee confirmed the drug’s acceptable safety profile, allowing the study to continue. Analysts from BofA Securities, Goldman Sachs, and Citi have maintained their Buy ratings on Ultragenyx, with price targets of $80, $82, and $110, respectively. BofA Securities noted a 60% probability of success for the program, while Citi estimated an 85% probability of success at final analysis. The final analysis, expected around the end of 2025, will apply a revised statistical threshold of p

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