Bluebird Bio's LentiD 'Hits The Mark'

bluebird bio Inc BLUE presented interim data for the Phase II/III trials of its lead gene therapy LentiD in CCALD, a rare neurodegenerative disorder, at the American Academy of Neurology (AAN) meeting.

Goldman Sachs’ Salveen Richter maintained a Neutral rating on the company, with a price target of $149.

LentiD Data

Mentioning that the data was compelling, with a superior safety profile, Richter said, “Analysis Data suggests Lenti-D prevents CCALD progression given the lack of major functional disabilities (MFDs), which is the primary endpoint and most important clinical measure, in our view.”

In addition, the data revealed that the Neurological Function Score did not worsen in 16 of the 17 patients in the study, which compares favorably with natural progression of the disorder, where NFS worsens within 12 months.

“CCALD biomarkers were also positive,” Richter stated, pointing out that neuro-inflammation was found to decrease, while the Loes score stabilized.

Safety & Efficacy

According to the Goldman Sachs report, the trial data also revealed that “safety remains benign, with no graft versus host disease (GVHD) or clonal dominance.”

Also, the analyst noted that the efficacy of Lenti-D was superior to allo transplants, with a favorable safety profile, whereas allo transplants have been correlated with more than 10 percent treatment related mortality and GVHD in 45-75 percent of patients.

While this data validates bluebird bio’s platform, Richter pointed out that the next major catalyst for the stock would come at year-end 2016, when LentiGlobin data is presented in betathalassemia and sickle cell.