Friday, an analyst from Stifel has increased the price target on Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares to $127 from $124, maintaining a Buy rating on the stock.
The revision comes in light of recent positive Phase 3 clinical trial data for DTX401, a treatment for glycogen storage disease type Ia (GSDIa).
The trial demonstrated a significant reduction in the need for cornstarch, a dietary supplement used to manage the condition, along with other evidence indicating a fundamental change in the patients' physiology.
This Phase 3 trial's results are seen as potentially leading to a one-time, durable treatment that could eventually eliminate GSDIa patients' lifelong dependence on cornstarch.
The Stifel analyst noted the importance of the trial's design in ensuring blinding and indicated that when all data are considered together, they present a strong case for DTX401's effectiveness.
The detailed data from the trial is expected to be presented later in 2024, with additional long-term data from the Phase 3 crossover study anticipated to further strengthen the treatment's profile as it moves toward a Biologics License Application (BLA) submission planned for 2025.
The analyst has raised the probability of success for the DTX401 program to 80%, up from the previous 70%. This adjustment in the model is the basis for the increase in the price target for Ultragenyx shares. The positive clinical outcomes have thus played a pivotal role in the upgraded financial expectations for the company's stock.
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