- RTX-134 wird "eingestellt"
- neues 52 WT
- MK 283 Mio. $
RTX-134 Program Update "As a result of the decision to focus on oncology and autoimmunity, the Company is deprioritizing the RTX-134 program for the treatment of phenylketonuria (PKU) and its other rare disease programs. Multiple factors contributed to this decision, including unanticipated delays in the RTX-134 program, primarily due to continued manufacturing challenges at the Company’s contract manufacturing organization (CMO), the anticipated high cost associated with producing chronic, high-dose therapy for enzyme deficiencies and the continued momentum of the Company’s oncology pipeline. Future capital investments and improvements in manufacturing efficiency, together with enhancements to the RED PLATFORM, may enable Rubius to revisit chronic, high dose-dependent conditions in the future.
As previously announced the first patient was dosed in the Phase 1b PKU clinical trial of RTX-134 in January 2020. While there were no reported adverse events and RTX-134 administration was well tolerated, the results from the first patient were uninterpretable possibly due, in part, to the low dose of cells administered and the sensitivity of the flow cytometry assay used to detect circulating cells. As a result of the deprioritization, the current Phase 1b clinical trial in PKU will be discontinued."
https://ir.rubiustx.com/news-releases/...h-quarter-and-full-year-2019
|
