At this week’s annual meeting of the American Society of Hematology, Bluebird Bio announced favorable updates related to its gene therapy for sickle cell disease (SCD).
Bluebird (BLUE) stated that the data from Group C of its ongoing Phase 1/2 HGB-206 study of its investigational LentiGlobin gene therapy (bb1111) for sickle cell disease revealed a complete elimination of SCD-related severe Vaso-Occlusive Events (VOEs).
No severe VOEs were reported through 24 months of follow-up in Group C patients who had a history of at least four severe VOEs and at least six months of follow-up.
Bluebird’s CEO David Davidson stated, “In addition to these clinical outcomes, for the first time with a gene therapy we now have patient-reported outcomes through the validated PROMIS-57 tool, showing reduction in pain intensity at 12 months after treatment with LentiGlobin for SCD. These results provide insight into the potential real-life impact LentiGlobin for SCD may offer patients.”
Explaining why Bluebird’s impressive sickle cell data sets a high bar for the field, Mizuho Securities analyst Difei Yang wrote, “Gene therapy (and gene editing) programs currently in development for sickle cell disease seek to reduce HbS (sickle hemoglobin) by either increasing production of hemoglobin A (such as bluebird bio) or fetal hemoglobin. Both approaches have the same goal, and BLUE’s approach so far has set a very high bar in our view in light of the latest safety and efficacy data.”
Yang reiterated a Buy rating on the stock with a price target of $123 (177% upside potential) and believes that the company’s LentiGlobin data in sickle cell disease has shown transformative clinical outcomes and would drive commercial adoption of the therapy, generating “meaningful value over time.” (See BLUE stock analysis on TipRanks)
The analyst feels that Bluebird’s platform remains underappreciated by the market and continues to see significant potential for value creation over time, especially considering that the company has now developed a total of three leading new medicines in both oncology (bb2121) and genetic diseases (LentiGlobin and Lenti-D).
The rest of the Street has a cautiously optimistic outlook on Bluebird, with a Moderate Buy analyst consensus based on 12 Buys and 6 Holds. With shares down 49.5% year-to-date, the average price target of $76.81 reflects an upside potential of 73% in the months ahead.
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