Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) and Mereo BioPharma Group plc (NASDAQ:MREO) stocks hit their 52-week low on Monday, as per data from Benzinga Pro.
Mereo BioPharma and its partner Ultragenyx revealed results from the Phase 3 Orbit and Cosmic studies for setrusumab (UX143) in Osteogenesis Imperfecta (OI).
Osteogenesis Imperfecta (OI) includes a group of genetic disorders impacting bone metabolism. The collagen mutations in OI can result in increased bone brittleness, which contributes to a high rate of fractures.
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The companies in a press release on Monday said that neither study achieved statistical significance against the primary endpoints of reduction in annualized clinical fracture rate compared to placebo or bisphosphonates, respectively.
Both studies achieved the secondary endpoints of improvements in bone mineral density (BMD) against comparators. There was no change in the safety profile observed.
“We are surprised and disappointed by these results given the promising data from our Phase 2 study…,” said Emil Kakkis, chief executive officer and president of Ultragenyx. “We continue to explore the data to gain a deeper understanding of the findings.”
Orbit and Cosmic Results
In the Orbit study, participants experienced statistically significant and substantial improvements in BMD compared to placebo, at levels consistent with the treatment effect observed in the Phase 2 portion of the study.
These BMD changes were not accompanied by a corresponding reduction in annualized fracture rates, and there was a low fracture rate in the placebo group.
In the pediatric Cosmic study, patients had a substantially higher baseline fracture rate compared to the patients enrolled in Orbit.
In this younger patient population, meaningful improvements in BMD were associated with a reduction in annualized fracture rate for setrusumab-treated patients compared to bisphosphonate-treated patients, though the reduction did not meet statistical significance.
Ultragenyx is conducting 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 given the totality of these data.
Ultragenyx is evaluating its planned operations and will promptly define and implement significant expense reductions.
Dr. Kakkis continued, “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.”
Price Action: Ultragenyx Pharmaceutical shares were down 43.96% at $19.16 at the time of publication on Monday. The stock is trading at a new 52-week low, according to Benzinga Pro data. Mereo BioPharma Group shares were down 89.32%.
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