Favorable FDA Pathway Should Keep Bluebird's Clinical Studies Flying Straight

During its R&D day, bluebird bio Inc BLUE provided insight into the new manufacturing process for LentiGlobin and an update on the design of the Beta-thalassemia pivotal studies.

The design should support LentiGlobin’s commercial launch for “essentially the entire moderate to severe Beta-thalassemia population, in the US and Europe by 2020,” BTIG’s Dane Leone said in a report. He reiterated a Buy rating on Bluebird, while raising the price target from $72 to $83.

US Approval

With the new manufacturing process for LentiGlobin, the company should be able to recapture the most severe Beta-thalassemia patients.

“We think that the combination of the new manufacturing process boosting drug potency along with a transfusion reduction endpoint, versus transfusion independence endpoint, greatly increases odds of clinical success,” Leone wrote. He expects the compound to receive US approval by yearend-2019.

EU Approval

Management expects the ongoing HGB-204 and ‘205 programs to support EU approval for moderate to severe Beta-thalassemia patients, with interim support from the new pivotal studies. The analyst expects EU approval by yearend-2018.

The revenue estimate for 2020 has been raised from $284 million to $744 million, reflecting ~$380 million of LentiGlobin revenues for the treatment of moderate to severe Beta-thalassemia [all genotypes] and ~$282 million of sales for the treatment of Sickle cell, Leone added.

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