JPMorgan has downgraded Ultragenyx Pharma Inc. to Neutral from Overweight, citing the failure of the Phase 3 ASPIRE trial of GTX-102 for Angelman Syndrome to meet primary or key secondary endpoints.
The bank also lowered its price target to $36 from $80, reducing its December 2027 target. Ultragenyx’s shares were trading at $26.53 at the time of the announcement, reflecting a market capitalization of approximately $2.61 billion.
JPMorgan estimated the commercial business’s fundamental value in the mid-$20s per share range, noting that the company’s gross profit margin over the last twelve months was negative 20.78%. The downgrade follows the trial setback, which raises questions about the near-term viability of GTX-102.
Despite the setback, Ultragenyx retains other pipeline catalysts. UX111 for MPS IIIA remains on track for a Prescription Drug User Fee Act (PDUFA) decision later this month, with a high likelihood of approval. Additionally, the FDA granted accelerated approval for Genglycos, a gene therapy for glycogen storage disease type Ia, ahead of the initial 2026 decision date.
Other analysts have also adjusted their outlook. Baird and Evercore ISI both lowered their price targets to $16 and shifted their ratings to Neutral from Outperform. Wells Fargo, however, raised its target to $50 while maintaining an Overweight rating, though it expressed skepticism about the GTX-102 trial’s prospects. H.C. Wainwright reiterated its Buy rating with a $50 target following the Genglycos approval.
