Baird downgraded Ultragenyx Pharmaceuticals Inc to Neutral from Outperform and cut its price target to $16 from $40, citing the failure of the Phase 3 Aspire study of GTX-102 in Angelman syndrome.
The pivotal trial, which evaluated the experimental therapy apazunersen, missed both its primary and key secondary endpoints, including the Bayley-4 cognitive raw score and the Motor Development Rating Instrument. The results were disclosed after the market close, with Ultragenyx’s stock closing at $25.81 on Tuesday.
Evercore ISI followed suit, lowering its rating to In Line from Outperform and reducing its price target to $16. The firm noted the trial’s outcome as a setback for the company’s pipeline. Canaccord, however, raised its price target to $83 while maintaining a Buy rating, reflecting a more optimistic outlook on Ultragenyx’s prospects.
H.C. Wainwright reiterated its Buy rating with a $50 price target, while Wells Fargo increased its target to $50 and maintained an Overweight rating. The divergence in analyst views underscores the uncertainty following the trial failure.
Ultragenyx’s market capitalization stood at $2.61 billion ahead of the announcement. The company has faced financial strain, reporting negative free cash flow of $489 million, described as a rapid cash burn. Earlier this month, Ultragenyx received accelerated FDA approval for its gene therapy GENGLYCOS, a treatment for glycogen storage disease type Ia, covering patients aged eight and older. The approval was granted ahead of the expected decision date, providing a rare positive offset to the trial setback.
