Google Cloud secures £400 mln U.K. Ministry of Defence sovereign cloud deal
Investing.com - H.C. Wainwright has reiterated its Buy rating and $80.00 price target on Ultragenyx Pharma (NASDAQ:RARE), currently trading at $31.51, following positive 96-week data from the Phase 3 GlucoGene study of DTX401 in glycogen storage disease type 1a (GSD1a). According to InvestingPro data, the stock appears undervalued, with analyst targets ranging from $34 to $128 per share.
The new data demonstrated progressive and durable benefits beyond the pivotal 48-week readout, with patients achieving approximately 61% mean reduction in daily cornstarch intake compared to 41% at 48 weeks. Two-thirds of patients eliminated at least one nighttime dose, resulting in uninterrupted sleep and quality-of-life improvements. The $3.03B market cap company has shown strong revenue growth of 26.77% over the last twelve months, maintaining a healthy liquidity position with a current ratio of 2.45.
Glycemic control was maintained with low rates of hypoglycemia, more time in the euglycemic range, and improved fasting tolerance. A separate open-label Japanese pediatric cohort saw complete elimination of daily cornstarch by 24-36 weeks while preserving glucose control.
Safety profiles remained consistent with prior reports, showing manageable hepatic adverse events and no adeno-associated viral vector-related toxicities. The longer-term results provide risk mitigation for the DTX401 program by confirming both durability and real-world benefits.
H.C. Wainwright views DTX401 as well-positioned for a 2026 launch as the first disease-modifying therapy in GSD1a with meaningful commercial potential, supporting its 12-month price target of $80 per share. InvestingPro subscribers can access additional insights, including 8 more key ProTips and a comprehensive analysis of Ultragenyx’s financial health and valuation metrics in the exclusive Pro Research Report.
In other recent news, Ultragenyx Pharmaceutical Inc. reported stronger-than-expected financial results for the second quarter of 2025. The company achieved an earnings per share of -$1.17, surpassing analysts’ forecasts of -$1.30. Additionally, Ultragenyx’s revenue reached $166 million, exceeding the anticipated $161.97 million. In terms of product development, Ultragenyx announced positive long-term data from its Phase 3 study of DTX401, an AAV gene therapy for glycogen storage disease type Ia (GSDIa). Patients treated with DTX401 showed a significant 61% reduction in daily cornstarch intake at 96 weeks. The company also initiated a rolling Biologics License Application submission to the U.S. FDA for this gene therapy, targeting completion by the fourth quarter of 2025. These developments highlight Ultragenyx’s ongoing progress in both financial performance and therapeutic advancements.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.