Leerink Partners Reiterates Outperform Rating on Ultragenyx Pharma (RARE)

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Ultragenyx Pharma (RARE) Provides Update on Stage 1 Cohorts in Phase 1/2/3 Cyprus2+ Study

October 3, 2024 4:31 PM EDT

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. Multiple responders have completely tapered off of standard-of-care treatment with responses seen in all three dose cohorts. The company plans to enroll an additional cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off of... More