Ultragenyx’s DTX401: Promising Phase 3 Results and Commercial Viability Support Buy Rating

In a report released today, Ram Selvaraju from H.C. Wainwright reiterated a Buy rating on Ultragenyx Pharmaceutical, with a price target of $80.00.

Ram Selvaraju has given his Buy rating due to a combination of factors that highlight the promising potential of Ultragenyx Pharmaceutical’s DTX401 therapy. The recent 96-week data from the Phase 3 GlucoGene study demonstrated significant and sustained benefits for patients with glycogen storage disease type 1a (GSD1a), including a notable reduction in daily cornstarch intake and improved quality of life. These results, which surpassed the 48-week findings, indicate a durable therapeutic effect and enhanced glycemic control, with minimal safety concerns.
Moreover, the therapy’s ability to maintain glucose control with low hypoglycemia rates and improved fasting tolerance, along with its positive impact on pediatric patients in Japan, underscores its transformative potential. The absence of serious adverse events and the therapy’s regulatory designations further de-risk its approval process. Consequently, Selvaraju reaffirms his confidence in DTX401’s commercial viability and its anticipated 2026 launch as the first disease-modifying treatment for GSD1a, supporting his Buy rating and a 12-month price target of $80 per share.

Based on the recent corporate insider activity of 43 insiders, corporate insider sentiment is negative on the stock. This means that over the past quarter there has been an increase of insiders selling their shares of RARE in relation to earlier this year.