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- Investors appear to lack enthusiasm over interim data released today by bluebird bio ( BLUE -4.8%) from a Phase 2/3 clinical trial assessing gene therapy candidate, Lenti-D, in pediatric patients with cerebral adrenoleukodystrophy (CALD), a rare inherited disorder linked to the X chromosome characterized by the buildup of fats in the brain. The results are being presented today at the 68th Annual Meeting of the American Academy of Neurology in Vancouver, BC.
- The trial, called The Starbeam Study, has enrolled 17 boys up to the age of 17 with CALD. All have been treated with Lenti-D and have had at least six months of follow-up. Eight patients have had 12-24 months of follow-up. According to clinicaltrials.gov, the estimated study completion date is August 2018.
- The primary endpoint is the proportion of patients with no major functional disabilities (MFDs) at month 24 post treatment. MFDs are six components (e.g. loss of communication, cortical blindness, tube feeding) of a scoring system called neurological function score (NFS), that measures clinical deficits across 15 functional domains. The presence of any of the six represents a profound negative impact on patients' lives.
- The data show all 17 patients remain free of MFDs, with 16 experiencing stable NFS. 14 had stable Loes scores (measure of quantifying demyelination and atrophy in CALD patients using brain MRI). Lenti-D's safety profile was consistent with myeloablative (lowering of the number of blood-forming cells in the bone marrow) conditioning.
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