Ultragenyx 的 Angelman 综合征三期 Aspire 试验失败，股价盘中暴跌后做空追击性价比不足

Ultragenyx Pharmaceutical ( RARE ) plunged more than 46% to an all-time low on Thursday amid multiple downgrades spurred by a late-stage trial setback for apazunersen, its experimental therapy for a rare, neurogenetic disorder called Angelman syndrome. In a statement late Wednesday, the Novato, California-based biopharma said that it will review its future operations, including R&D plans for apazunersen, after its Phase 3 Aspire study didn’t meet the primary endpoint in patients with Angelman syndrome. In reaction, RBC Capital Markets downgraded the stock to Sector Perform from Outperform and cut the price target to $19 from $40 per share, noting that after the trial failure it is difficult to see a catalyst that can significantly change the direction of Ultragenyx ( RARE ) shares. “Numerical data was not shared, but our read is that the drug showed no signal at all given little spin in the press release despite a very large unmet need with, on paper, plenty of regulatory flexibility,” analyst Luca Issi wrote. Baird analyst Jack Allen agreed. “Disappointingly, given the miss on both endpoints, we suspect this trial was a complete whiff," he wrote, downgrading the stock to Neutral from Outperform. Bank of America downgraded Ultragenyx ( RARE ) to Neutral from Buy and slashed its target price to $20 from $48 per share, with analyst Tazeen Ahmad noting that she was surprised by the outcome, given strong Phase 2 data for apazunersen. While Ultragenyx's ( RARE ) commercial operations provide a valuation floor for the stock, the analyst has become more conservative on its upcoming gene therapy rollouts as management plans to focus on managing expenses.