bluebird bio Inc (NASDAQ:BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, announced that interim data from the ongoing Phase 2/3 Starbeam Study (ALD-102) evaluating the Lenti-D™ product candidate in pediatric patients with cerebral adrenoleukodystrophy (CALD) will be presented at the American Academy of Neurology (AAN) Annual Meeting during the Clinical Trials Plenary Session. The data will be highlighted in an oral presentation by Florian Eichler, M.D., Director of the Leukodystrophy Service at Massachusetts General Hospital for Children.

“CALD is a rare and deadly disease, and currently the only available treatment option is allogeneic hematopoietic stem cell transplant, which is often challenging for patients without a matched sibling donor,” said David A. Williams, M.D., president of Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and the principal investigator of the Starbeam study. “It is exciting to see a potential autologous treatment option for these patients. While these study results are early, they are encouraging, and we look forward to further understanding the impact that treatment with Lenti-D gene therapy could have on patients as these data evolve.”

Interim Data Summary

The Starbeam Study is a global, multi-center study assessing the efficacy and safety of an investigational gene therapy in boys up to 17 years of age with CALD. As of March 31, 2016, 17 patients with CALD had received Lenti-D drug product. All patients had at least six months of follow up, with eight patients having between 12 and 24 months of follow up.

The primary efficacy endpoint for the Starbeam Study is the proportion of patients with no major functional disabilities (MFDs) at 24 months post treatment. MFDs are six components of the neurological function score (NFS) that, if present, would have a profound negative impact on patients’ lives: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence and complete loss of voluntary movement. Secondary endpoints include Loes score (a method for quantifying demyelination and atrophy in patients with CALD using brain MRI), NFS (a scoring system assessing clinical deficits across 15 functional domains), gadolinium enhancement on MRI (an indicator of neuroinflammation), and safety.

Data from these 17 patients as of March 31, 2016, that will be reported at AAN include:

  • All patients remain free of MFDs.
  • Sixteen of 17 patients had NFS stabilization (change of <3 points and an absolute NFS ≤ 4).
    • Two patients have an NFS of 1, due to the occurrence of stuttering in one patient, and episodic urinary incontinence in another patient.
    • One patient had an early, rapidly progressive course and has an NFS of 5, reflecting deficits in speech, vision, difficulty walking and running, and episodes of urinary incontinence.
  • Fourteen of 17 patients had a stable Loes score (change of ≤ 5 points or an absolute Loes Score ≤ 9).
  • Sixteen of 17 had resolution of gadolinium enhancement by month six. Re-emergence of diffuse contrast enhancement was seen in five patients at month 12. Of those five patients, the two with at least 18 months of follow up showed resolution of gadolinium enhancement at month 18.
  • The safety profile of Lenti-D treatment appears consistent with myeloablative conditioning with one possibly drug-related serious adverse event (Grade 3 BK-mediated viral cystitis) and one possibly drug-related adverse event (Grade 1 tachycardia). Both resolved with standard measures.
  • Integration site analyses have demonstrated polyclonal reconstitution in all subjects without evidence of clonal dominance to date.

“While the interim results from the Starbeam study are early, with only three of the 17 patients having completed the study thus far, we are pleased to see evidence of neurologic and radiographic stabilization of CALD,” said David Davidson, M.D., chief medical officer, bluebird bio. “All patients are free of MFDs and most have had no progression in NFS. Brain MRI is the primary tool to quantify disease activity, and it is encouraging that most patients in the study have had stabilization of Loes score and resolution of gadolinium enhancement. We look forward to sharing additional data from this trial as the results mature, and would like to express our gratitude to the study investigators, and especially to the patients and families who are participating in this trial.”

David Williams receives research support from bluebird bio for research related to sickle cell disease, and the company has licensed intellectual property from Boston Children’s Hospital for technology related to gene therapy for hemoglobinopathies that Williams co-invented. (Original Source)

Shares of bluebird bio are up nearly 5% in $50.1o . BLUE has a 1-year high of $197.35 and a 1-year low of $37.40. The stock’s 50-day moving average is $47.06 and its 200-day moving average is $60.86.

On the ratings front, bluebird bio has been the subject of a number of recent research reports. In a report issued on April 8, Goldman Sachs analyst Salveen Richter maintained a Buy rating on BLUE, with a price target of $149, which implies an upside of 212.2% from current levels. Separately, on April 4, Leerink Swann’s Michael Schmidt reiterated a Buy rating on the stock and has a price target of $45.

According to, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Salveen Richter and Michael Schmidt have a total average return of 28.9% and 3.6% respectively. Richter has a success rate of 66.7% and is ranked #65 out of 3812 analysts, while Schmidt has a success rate of 51.6% and is ranked #1050.

The street is mostly Bullish on BLUE stock. Out of 6 analysts who cover the stock, 5 suggest a Buy rating and one recommends to Hold the stock. The 12-month average price target assigned to the stock is $88.67, which represents a potential upside of 85.8% from where the stock is currently trading.