Today, Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) revealed promising clinical activity and metabolic improvements in the Phase 1/2/3 Cyprus2+ study of its investigational gene therapy, UX701, for treating Wilson disease.
The first stage of the study included 15 patients across three dosing cohorts, with six patients successfully discontinuing standard-of-care treatments. These patients showed normalized levels of non-ceruloplasmin bound copper (NCC) and increased ceruloplasmin-copper activity, indicating improved copper metabolism and ATP7b function.
Ultragenyx plans to add a fourth cohort with a higher dose of UX701 and an optimized immunomodulation regimen for enhanced treatment efficiency. The company aims to have most patients stop standard-of-care treatment before moving to the second stage of the trial.
The Cyprus2+ study is structured in three stages to evaluate safety, efficacy, and treatment optimization, with a focus on long-term follow-up.
InvestingPro Analysis
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) is making significant progress with its gene therapy for Wilson disease, supported by strong financial metrics and market performance. RARE has reported a 35.75% increase in quarterly revenue as of Q2 2024, demonstrating growth that aligns with ongoing research efforts like the UX701 program.
InvestingPro data shows that 8 analysts have raised their earnings forecasts, indicating positive expectations following the latest clinical results. RARE has also delivered a 38.15% price total return over the last three months, reflecting investor confidence in the company's pipeline.
It's worth noting that RARE is currently not profitable, typical for biopharmaceutical firms in the development phase. Investors should consider additional tips from InvestingPro for a deeper financial analysis of RARE.
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