Ultragenyx’s Phase 3 Aspire study for GTX-102 is fully enrolled with 129 participants. Study completion is expected in 2026, with plans for regulatory submission. The latest update is out from Ultragenyx Pharmaceutical (RARE). On July 31, 2025, Ultragenyx Pharmaceutical announced that the Phase 3 Aspire study for GTX-102, a treatment for Angelman Syndrome, is fully enrolled with 129 participants. The study, which began in December 2024, involves a 48-week trial comparing GTX-102 treatment to a sham comparator group, with primary and secondary endpoints focusing on cognitive improvements and multi-domain responses. Completion is expected in the second half of 2026, with plans to provide topline data and progress to regulatory submission. The most recent analyst rating on RARE stock is a Buy with a $61.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page. According to Spark, TipRanks’ AI Analyst, RARE is a Neutral. Ultragenyx Pharmaceutical’s overall stock score is driven by its challenging financial performance, reflected in poor profitability and cash flow, alongside bearish technical indicators and an unattractive valuation. The earnings call provided some optimism with strong revenue growth and advancement in clinical trials, but significant losses and cash usage remain concerns. Corporate events offer potential future growth catalysts, though recent regulatory setbacks highlight ongoing risks. To see Spark’s full report on RARE stock, click here. Ultragenyx Pharmaceutical Inc. operates in the biopharmaceutical industry, focusing on developing novel products for the treatment of rare and ultra-rare genetic diseases.

Ultragenyx Completes Enrollment for GTX-102 Phase 3 Study

Ultragenyx Pharmaceutical completed enrollment for its Phase 3 Aspire study of GTX-102, a treatment for Angelman Syndrome, with 129 participants. The study, which began in December 2024, is expected to complete in 2026, with plans for regulatory submission. The company's stock is under scrutiny due to financial performance and regulatory risks.