Updated results for Phase II/III Starbeam study of Lenti-D gene therapy in cerebral adrenoleukodystrophy.- bluebird bio.

bluebird bio announced updated results from the Phase II/III Starbeam study (ALD-102) of its investigational Lenti-D gene therapy in boys 17 years of age and under with cerebral adrenoleukodystrophy (CALD), and initial data from ALD-103, the ongoing observational study of outcomes from allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys 17 years of age and under with CALD. The Starbeam study has met its enrollment goal.

A ll reported data are as of April 25, 2018 and reflect a total study population of 31 patients. Of these 31 patients, 29 have received Lenti-D and the median follow-up for all treated patients was 34 months (0.4 � 54 months). As previously reported in the New England Journal of Medicine (October 2017), of the 17 patients treated with Lenti-D who completed 24 months of follow-up, 15 patients (88 percent) were alive and MFD-free. One patient withdrew from the study, and one died following rapid development of MFDs post-treatment. All patients who were MFD-free at 24 months (n=15) continued to be MFD-free. The median follow-up for the initial group (n=17) was 41.4 months (13.4 - 54.0 months).

An additional 12 patients have received Lenti-D in the Phase II/III Starbeam study. While these patients have not reached the primary endpoint of 24-month follow-up, there have been no MFDs reported as of April 25, 2018. The median follow-up for this additional cohort of patients is 4.2 months (0.4 � 11.7 months).

The primary safety endpoint of the Phase 2/3 Starbeam study is the proportion of patients who experienced grade of at least 2 acute graft-versus-host disease (GvHD) or chronic GvHD by 24 months post-treatment. No acute or chronic GvHD has been reported post-Lenti-D treatment as of April 25, 2018. There has been no graft failure, insertional oncogenesis or replication competent lentivirus detected. The safety profile of Lenti-D is generally consistent with myeloablative conditioning with busulfan and cyclophosphamide. Three adverse events (AE) have been deemed potentially related to treatment with Lenti-D and include BK-mediated viral cystitis (grade 3), tachycardia (grade 1), and vomiting (grade 1). The data were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2018 Symposium.