After shares plunged more than 42% following the Phase 3 setbacks for setrusumab, Wells Fargo’s Benjamin Burnett maintained an overweight rating and lowered his target to $45, still implying over 128% upside from the prior close. His research note cited statistically significant gains in bone mineral density and forecasted an improved risk–reward ahead of forthcoming FDA feedback. By mid-afternoon trading, this positive reassessment helped the stock rally more than 13%, with average daily volume jumping tenfold compared to the three-month average.

Ultragenyx reported that neither the Orbit (159 patients across 11 countries) nor the Cosmic (69 patients across 7 countries) study achieved the primary goal of reducing annualized fracture rates versus placebo or bisphosphonates. Both trials did demonstrate strong statistical improvements in bone mineral density—secondary endpoints that mirrored positive trends seen in Phase 2. In response, management announced plans for substantial expense reductions while conducting deeper analyses of other bone-health and clinical measures to assess next steps for the program.

Ultragenyx completed the rolling submission of its Biologics License Application to the FDA for DTX401, an AAV8 gene therapy for Glycogen Storage Disease Type Ia, based on data from 52 treated patients with up to six years of follow-up. Phase 3 GlucoGene results showed meaningful and clinically significant reductions in daily cornstarch requirements, stable glycemic control and improved fasting tolerance, translating into quality-of-life gains on the Patient Global Impression of Change scale. DTX401 holds Rare Pediatric Disease, orphan drug, Fast Track and RMAT designations from the FDA, positioning it as the first potential therapy to address the underlying genetic defect in this ultra-rare condition.