We recently published 10 Stocks on Fire Ahead of 2026. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the best performers on Tuesday.
Ultragenyx rebounded by 15.52 percent on Tuesday to close at $22.78 apiece as investors resorted to bargain-hunting following the previous day’s steep 46 percent fall, dragged by disappointing clinical trial results for its brittle bone disease treatment.
In a statement on Monday, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) said that two of its phase 3 clinical trials to test the efficacy of setrusumab failed to achieve statistical significance in reducing annual clinical fracture rate as compared to placebo and bisphosphonates.
Both studies, however, achieved the secondary endpoints of improvements in bone mineral density (BMD) against comparators.
“We are surprised and disappointed by these results, given the promising data from our Phase 2 study and the lack of approved treatment options available to patients with OI (Osteogenesis Imperfecta) who live with significant pain, disability, and disease burden,” said Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) President and CEO Emil Kakkis.
“We continue to explore the data to gain a deeper understanding of the findings,” he added.
The company would conduct additional analyses on the data across both studies, including on other bone health and clinical endpoints beyond fractures, to assess next steps for the program.
“While we are disappointed by these results, we continue to build our commercial revenue from four approved products and prepare for a transformational year ahead with potentially two near-term gene therapy launches and a pivotal Phase 3 readout in Angelman syndrome,” Kakkis said.
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Disclosure: None. This article is originally published at Insider Monkey.