Stock Soars as bluebird bio Gives Sneak Peek at Promising Sickle Cell Data
Published: May 26, 2015
May 21, 2015
By Riley McDermid, BioSpace.com Breaking News Sr. Editor
Shares of closely-watched gene therapy company Bluebird Bio are up more than 15 percent in morning trading Thursday, after the company released an abstract of data that shows its sickle cell anemia drug, LentiGlobin, has kept two patients transfusion free for 14 and 11 months, respectively.
Bluebird released the data from a February test as part of an abstract it will present in June at the European Hematology Association (EHA) in Vienna, Austria.
On Feb. 2, 2015, Bluebird announced that the FDA had granted LentiGlobin BB305 Breakthrough Therapy designation, which is used to expedite the development and review of a potential drug candidate that is expected to be used to treat a serious or life-threatening diseases.
In the case of LentiGlobin BB305, initial data last fall from an ongoing Phase I/II Northstar (HGB-204) and HGB-205 studies looked at eight patients with beta-thalassemia that were treated with LentiGlobin. In the first four patients, treatment resulted in sufficient hemoglobin production to decrease the need for transfusion support among the patients. That news sent shares of the company up 70 percent the day it was announced, as the market looked eagerly for signs that Bluebird’s LentiGlobin BB305 could be a panacea for blood diseases.
Today’s data had a similar effect on the company’s share price and had executives eager to trumpet the results and explain what they mean, which is that one of the patients treated has no had to seek hospitalization for his sickle cell and has even started producing anti-sickling properties.
“The early data included in our abstract provide further validation for our approach and important insights into the safety and mechanism of action of LentiGlobin in both beta-thalassemia and sickle cell disease,” said David Davidson, chief medical officer for Bluebird.
“As noted in the abstract, we are pleased to report that the two patients with beta-thalassemia major, on whom we first reported last year at EHA, remained transfusion independent at 14 and 11 months post-transplant,” he said. “In addition, it is very encouraging that the patient with sickle cell disease is increasing production of HbAT87Q, which has anti-sickling properties, and has not had a post-treatment hospitalization for a sickle cell disease-related event. At EHA we will present further follow up data on all three subjects.”
That news had both analysts and Wall Street investors cheering, because it shows LentiGlobin is on track.
“Our recent deep dive on LentiGlobin combined with this update keep us confident that BLUE is on the cusp of a dramatic breakthrough for many sickle cell disease patients and we await further updates,” wrote Joshua Schimmer, a biotech analyst for Piper Jaffray, in a note to investors.
Bluebird’s LentiGlobin BB305 extracts blood stem cells and then infuses them with a working version of the malfunctioning gene that had caused the disease. People with the disease must undergo monthly blood transfusions in order to survive—but if Bluebird’s therapy continues to be successful, they may now be freed from that burden. The number of people affected is not huge, but is certainly significant: Around 40,000 babies world-wide and between 1,000 and 3,000 in the U.S. are born with the condition each year.
The new therapy is so effective, Wall Street is champing at the bit to see it be rushed into later-stage trials to bring it to market more quickly. Some analysts have long said Bluebird has a “robust” proof of concept for the therapy so far, said Schimmer.
“The abstract notes the peripheral blood T87Q vector copy number is an impressive 2.4. This should be a good marker for what's happening in the bone marrow, meaning that a majority of cells have a copy of the corrected gene,” he wrote in his note. “This also means that BLUE's busulfan myeloablation regimen was effective in establishing chimerism with the LentiGlobin cells. And this also means that as the patient continues to be weaned from transfusions, the corrected cells should be easily able to ramp up production and eliminate sickling and its consequences.”
The results of this data, though hardly extensive, are apparently promising enough for the FDA and the EMA to consider conditional approval for use of the drug.
Bluebird generated a lot of buzz earlier this year when its chief executive told an audience at a closely watched industry event held by J.P. Morgan that the company expects data on its potentially-disease ending sickle cell treatment by the end of 2015.
Nick Leschly, CEO of Bluebird, also said that LentiGlobin BB305 keeps track of production a key marker in keeping down the cost of clinical trials and measuring data.
Leschly made the comments at the J.P. Morgan Healthcare Conference in January in San Francisco and is the oldest and largest conference of its type. It includes 300 of the largest biotech, healthcare and biopharma companies presenting their top-line data and estimates to 4,000 eager bankers, analysts, institutional investors, hedge funds and journalists. He presented the following milestones for Bluebird in the next two years:
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