On Bluebird Bio After A Glimpse Of Preliminary Lentiglobin Data

Last week, preliminary data for Bluebird Bio (BLUE)'s Lentiglobin were released as part of the abstract dump for ASH in December (we wrote at length about this, here). Bluebird also issued their third quarter financial results this morning, Wednesday.

At ASH, Bluebird will have an oral presentation (abstract) on initial LentiGlobin results from the NORTHSTAR study and a poster (abstract) with updated data from the two patients enrolled in HGB-205 (previously presented at EHA). Both of these abstracts include data as of July 31, with longer follow-up in a larger patient population to be presented at the meeting.

HGB-205 still encouraging. Bo/BE patients from EHA remain transfusion independent.
Subjects 1201 and 1202 (previously reported transfusion-independent at EHA) both have remained transfusion independent for 180 and 90 days, respectively. In addition, neither subject has had any adverse events associated with the received gene therapy. As shown below, Subject 1201 is producing 7.2 [g/dl] ßA-T87Q at day +180 (vs. 6.6 [g/dl] at ~ day +135 at EHA) and Subject 1202 is producing 6.8 [g/dl] at day +90 (vs. 4.2 [g/dl] at ~ day 60 at EHA).

Click on picture to enlarge

This update further validates the LentiGlobin drug product. Subjects are undergoing durable and increasing ßA-T87Q globin expression and remain transfusion independent 3 and 6 months from treatment.

Preliminary NORTHSTAR results may suggest dose-dependent hemoglobin response
As of July 31, 3 subjects enrolled in the NORTHSTAR study have received LentiGlobin (2 additional patient await treatment); follow-up hemoglobin expression was available in just one patient (1102, shown below) for the abstract.

Click on picture to enlarge

Subject 1102 did not achieve full transfusion-independence and received a rescue transfusion of pRBC (packed red blood cells) on day +96 because of fatigue and ultimately low hemoblogin (total Hb: 8.6 [g/dl]).

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Disclosure: One or more of PropThink's contributors are long BLUE.

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