Shares of Ultragenyx Pharmaceutical tumbled around 47% before the opening bell on Thursday, September 3 after the drugmaker informed that the trial for its rare, neurogenetic disorder drug, apazunersen, failed to meet its goal at a later stage.
On Wednesday, the US-based biopharmaceutical firm announced results from the Phase 3 trial for apazunersen (GTX-102) in Angelman syndrome, a lifelong neurogenetic disease caused by loss-of-function of the maternally inherited gene.
Ultragenyx shares tanked 46.9% to $14.09 in pre-market by 8:43 am ET or 6:13 pm IST. The shares closed 2.79% higher at $26.53 on Wednesday.
According to Nasdaq listed drugmaker, study failed to meet its main goal of improving cognitive scores or the key Multidomain Responder Index (MDRI).
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“Based on everything we observed in the robust Phase 1/2 clinical development program and long-term extension study, we are disappointed by the result,” said Emil Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “Even more, we are disappointed for the global patient community who has invested so much in early-stage research, working to bring a first-ever treatment to their children.”
The company said it will evaluate the apazunersen programme in light of this outcome and make a decision accordingly. It will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.
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"We will maintain focus on our growing commercial business, which continues to create meaningful value, including new sources of revenue from the recent approval of GENGLYCOS for glycogen storage disease type Ia, the potential approval of UX111 for Sanfillipo syndrome, and the expansion of existing products to new territories. This strong commercial foundation will support our pipeline, while continuing toward profitability in 2027," Kakkis added.
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