H.C. Wainwright reduced its price target on Ultragenyx Pharma by half to $25 from $50, maintaining a Buy rating after the company’s Phase 3 Aspire study for apazunersen (GTX-102) failed to meet its primary and key secondary endpoints in Angelman syndrome.
The trial, which evaluated cognitive and multidomain responder outcomes, did not achieve statistically significant changes from baseline in either measure. The drug’s safety profile remained consistent with earlier Phase 1/2 data, though baseline comparability between treated and control groups did not support efficacy signals, according to the analyst note.
Ultragenyx’s shares were trading at $26.53 at the time of the update, just above H.C. Wainwright’s new target. The firm had previously assigned a 50% probability of success to the asset but noted its financial models did not assume regulatory approval or market entry in 2027, leaving income statement projections unchanged.
The setback prompted broader analyst downgrades and target reductions. Canaccord maintained its Buy rating but cut its target to $37 from $83. Wells Fargo kept an Overweight rating while lowering its target to $18 from $50. JPMorgan downgraded Ultragenyx to Neutral and reduced its target to $36 from $80. Baird and Evercore ISI both downgraded to Neutral and In Line, respectively, with targets slashed to $16 from $40 and $34.
The company, which reported levered free cash flow of negative $489 million over the last twelve months, said it would evaluate the apazunersen program and implement significant expense reductions to support its commercial operations. Ultragenyx has not provided a timeline for its decision on the drug’s future.
