Ultragenyx Pharmaceutical (RARE) Stock Forecast & Price Target

Ultragenyx Pharmaceutical Inc. is positioned for potential growth, driven by its innovative pipeline and the promising long-term data from the DTX401 program in glycogen storage disease type 1a. The recent 96-week data indicates a durable and transformative impact, providing considerable de-risking for regulatory approval and highlighting the therapy’s significant commercial potential. Furthermore, the company anticipates a path towards GAAP profitability by 2027, underlining a positive outlook supported by operational adjustments and multiple near-term catalysts to enhance share value.

Ultragenyx Pharmaceutical Inc reported a 3Q25 revenue of $159.9 million, falling short of both internal expectations and market predictions, indicating continued financial challenges as a development-stage company with no imminent path to profitability. Key risks contributing to the negative outlook include potential clinical trial failures, competition impacting sales projections, and various regulatory and patent challenges, all of which could hinder the company’s growth trajectory and market position. Additionally, the significant underperformance in R&D expenses, which were $216 million compared to estimates of $166 million, raises concerns about financial management and resource allocation, thereby amplifying long-term profitability risks.