Bluebird Bio (BLUE) released on September 5 its updated data from the Phase 2/3 Starbeam study evaluating the investigational Lenti-D gene therapy for the treatment of cerebral adrenoleukodystrophy (or CALD) in boys aged 17 years or less.
The above chart shows details about the investigational Lenti-D gene therapy.
About the study
The Phase 2/3 Starbeam study met its enrollment goal with the data reported by Bluebird Bio as of April 25. Thirty-one patients were studied, and 29 of them received Lenti-D gene therapy. The median follow-up for all those patients was 34 months.
The primary efficacy endpoint reflects the proportion of patients who are alive and free from major functional disabilities at month 24 since the beginning of the treatment. The company reported that of the 17 patients treated with Lenti-D, 15 were alive and free from major functional disabilities during the 24-month follow-up. The results were published in the New England Journal of Medicine in October 2017.
The secondary efficacy endpoints included Neurologic Function Score (or NFS) and gadolinium enhancement (or GdE). NFS quantified the severity of neurologic dysfunction based on the 15 symptoms. Of the 15 patients studied at the end of 24 months on April 25, 2018, 14 patients reported an NFS score of less than or equal to one, while one patient reported an increase in the NFS score from one to two. GdE was also assessed through magnetic resonance imaging in patients following the transplant at an interval of six months for up to 24 months, and then at an interval of 12 months. Of the15 patients studied at the end of 24 months, 14 patients reported negative GdE.