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ASH Preview - Cellectis Steals The Limelight

Unexpected revelation of data from a single patient treated with Cellectis’s allogeneic CAR-T construct has allowed the French company to steal the early limelight in yesterday’s unveiling of abstracts for the ASH conference in Orlando.

But the relatively muted reaction- Cellectis ADRs closed up 18%- might suggest that lessons are being learnt about placing undue reliance on tiny studies. A case in point is Bluebird Bio, whose stock had surged 62% on four-patient beta thalassaemia data at last year’s ASH; the unveiling of an update to this trial in Orlando marked Bluebird, off 22% yesterday, as the first big loser of ASH.

Not that Bluebird’s latest data cut, from the Northstar trial of its Lentiglobin gene therapy, is bad – far from it. But it comes down to baseline expectations; after the company’s market cap breached $7bn, and with some in the market touting that it had a “cure” on its hands, things could only really go downhill.

The group’s ASH presentation details four patients who have a less severe form of beta-thalassaemia, in whom Lentiglobin has prompted production of beta-globin, and who remain transfusion-free for over 90 days. So far so good (Bluebird sings sweet music to the ears of investors, December 9, 2014).

But the problem is with three other patients, with the beta-0/beta-0 form of the condition in which almost no beta-globin is produced: two have received a transfusion and one remains transfusion-dependent. The market must now dial back any expectation of a cure for all beta-thalassaemia forms.

Surprise

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