Canaccord Genuity has reduced its price target on Ultragenyx Pharma by 55% to $37 from $83, while maintaining a Buy rating, after the company’s Phase 3 Aspire trial for its experimental therapy GTX-102 in Angelman syndrome missed both primary and key secondary endpoints.
Ultragenyx’s stock closed at $26.53 on Friday, down from the revised target but still above the $16–$36 price targets set by other firms. Wells Fargo lowered its target to $18 from $50 with an Overweight rating, JPMorgan cut its target to $36 from $80 and downgraded the stock to Neutral, Baird set a target of $16 down from $40 with a Neutral rating, and Evercore ISI reduced its target to $16 from $34 with an In Line rating.
The trial failure centered on the primary endpoint, a change from baseline in Bayley-4 cognitive raw score, and the key secondary endpoint, net response on the Multidomain Responder Index, both of which were not met. The company reported no statistically significant differences between treated and control groups in either measure, undermining the drug’s efficacy claims.
Ultragenyx is tracking toward approximately $750 million in revenue for the year, having generated $717 million in the trailing twelve months. The company continues to face negative free cash flow, burning through $489 million in the period, according to InvestingPro data.
The setback raises questions about the near-term commercial prospects for GTX-102, which had been positioned as a potential treatment for Angelman syndrome, a rare neurodevelopmental disorder. Ultragenyx has not indicated whether it will pursue additional trials or regulatory pathways based on the current data.
