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Bluebird bio Presents Updated Data from Phase 2/3 Clinical Study of Lenti-D Gene Therapy for Cerebral Adrenoleukodystrophy at the 13th European Pediatric Neurology Society Congress.

M2 PHARMA-September 24, 2019-Bluebird bio Presents Updated Data from Phase 2/3 Clinical Study of Lenti-D Gene Therapy for Cerebral Adrenoleukodystrophy at the 13th European Pediatric Neurology Society Congress


- US-based gene therapy bluebird bio, Inc. (NASDAQ: BLUE) presented updated results from the clinical development programme for its investigational Lenti-D gene therapy in patients with cerebral adrenoleukodystrophy at the 13th European Pediatric Neurology Society Congress in Athens, Greece, the company said.

CALD is a rare genetic and rapidly progressive disease that can lead to severe loss of neurologic function and death.

The Phase 2/3 Starbeam study (ALD-102) is assessing the efficacy and safety of Lenti-D in boys 17 years of age and under with CALD.

Updated data from the ongoing observational study (ALD-103) of allogeneic hematopoietic stem cell transplant (allo-HSCT) in boys 17 years of age and under with CALD were also presented.

The Phase 2/3 Starbeam study has completed enrollment. All reported data below are as of April 25, 2019 and reflect a total population of 32 patients with a median follow-up time of 21.2 months (0.0 60.2 months).

Of the 32 patients who have received Lenti-D as of April 25, 2019, 15 have completed ALD-102 and enrolled in a long-term follow-up study, 14 are currently on-study, and three are no longer on-study.

The primary efficacy endpoint in the study is the proportion of patients who are alive and free of MFDs at Month 24.

MFDs are six severe disabilities commonly attributed to CALD and thought to have the most profound impact on a patient's ability to function independently, including loss of ability to communicate, cortical blindness, need for tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement.

Of those patients who have or would have reached 24 months of follow-up and completed the study, 88 % (N=15/17) continue to be alive and MFD-free in a long-term follow-up study. The 14 patients currently on study have less than 24 months of follow-up and have shown no evidence of MFDs.

The longest follow-up of the additional 14 patients was 20.4 months.

Three out of the 32 treated patients did not or will not meet the primary efficacy endpoint; two patients withdrew from the study at investigator discretion, and one experienced rapid disease progression early on-study resulting in MFDs and death.

Secondary and exploratory efficacy outcomes included: changes in neurologic function score, a 25-point score used to evaluate the severity of gross neurologic dysfunction across 15 symptoms in six categories; resolution of gadolinium enhancement, an indicator of active inflammation in the brain; and change in Loes score, an MRI measurement of white matter changes in CALD.

Of the 32 patients treated, 30 had stable NFS following treatment with Lenti-D, defined as NFS 3 from baseline. Loes scores generally stabilised within 12-24 months and GdE+ enhancement resolved in most patients following Lenti-D treatment.

The primary safety endpoint is the proportion of patients who experience acute (>=Grade 2) or chronic graft-versus-host disease by Month 24.

GvHD is a condition that may occur after an allo-HSCT, where the donated cells view the recipient's body as foreign and attack the body.

No events of acute or chronic GvHD have been reported post-Lenti-D treatment and there have been no reports of graft failure, cases of insertional oncogenesis, or replication competent lentivirus.

The safety profile of Lenti-D is generally consistent with myeloablative conditioning with busulfan and cyclophosphamide, the standard preparative regimen completed prior to HSCT.

Three adverse events have been deemed potentially related to treatment with Lenti-D and include BK-mediated viral cystitis (N=1, grade 3) and vomiting (N=2, grade 1); all three resolved using standard measures.

Allo-HSCT has been successfully used to treat CALD but comes with risks, including graft failure, acute and chronic GvHD, and death, as well as infection as a result of the immune suppression required post-transplant.

The ongoing observational study, ALD-103, is designed to assess safety and efficacy outcomes of this treatment option in boys 17 years of age and younger with CALD.

The study measures CALD disease-related outcomes in four patient cohorts: early disease 1 (N=21; Loes

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Publication:M2 Pharma
Article Type:Clinical report
Date:Sep 24, 2019
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