This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here

Bluebird Bio Higher on Strong Gene Therapy Study Results

By: Adam Feuerstein | 06/16/14 - 07:28 AM EDT

Bluebird Bio (BLUE) shares are up 53% to $40 in Monday's pre-market trading in reaction to the strongly positive results presented Saturday from the company's gene therapy against the blood disorder beta-thalassemia

What? You were enjoying your weekend and not sitting at your computer reading about clinical trial results? That's crazy. If you're trying to catch up this morning, I wrote about the Bluebird data on Saturday. Here's why investors are reacting happily:



Two patients with a serious, inherited blood disorder have been able to stop blood transfusions following a single treatment with an experimental gene therapy developed by Bluebird Bio (BLUE), according to new but preliminary data presented Saturday. 

The two patients reported on today have beta-thalassemia (B-thal), a disease caused by a missing or defective gene which prevents oxygen-carrying hemoglobin from functioning properly. The BlueBird therapy, known as LentiGlobin, replaces the defective gene with one that is fully functional.

Beta-thalassemia patients suffer from chronic anemia and typically require regular and lifelong blood transfusions. Following a single infusion of Bluebird's LentiGlobin gene therapy, the two B-thal patients started producing functional hemoglobin and within 10 and 12 days, respectively, were able to halt blood transfusions. The patients have now been blood-transfusion independent for approximately 6 and 3 months, respectively, according to Bluebird Chief Medical Officer David Davidson. The data are being presented Saturday at the European Hematology Association annual meeting.

"Following the [gene] transplant, we're seeing near normal levels of hemoglobin," said Davidson. "These early results far exceeded our expectations."

Bluebird only has limited data on two patients and the study, conducted in France, continues with seven B-thal patients expected to enroll and be treated. Bluebird has begun another Lentiglobin B-Thal study in the U.S., which will enroll 15 patients. The company expects to report updated results from the Lentiglobin studies at the end of the year. 

Bluebird went public last year partly on the back of some encouraging proof of concept data showing a first-generation gene therapy approach was feasible for B-thal patients. LentiGlobin is a second-generation gene therapy designed to be more potent -- and hopefully more effective. The results presented today suggest Bluebird's improved gene therapy is leading to more clinical benefit for patients.

The two patients entered the study with the "major" or Beta E/Beta 0 genotype of B-thal, which required them to undergo monthly blood transfusions to deal with chronic anemia. Following the Lentiglobin infusion, patient 1 produced 6.6 g/dl of "marked" beta-globin at 4.5 months, a measure of functional hemoglobin produced by the working gene inserted by LentiGlobin. Patient 2 produced 4.2 g/dl of "marked" beta-globin at 2 months. In both cases, LentiGlobin is producing more functional hemoglobin and at a faster rate than Bluebird's first generation gene therapy. 

With two weeks, both patients were transfusion independent, which is the most important measure of clinical benefit in B-thal.

Bluebird estimates there are about 15,000 patients with B-thal in the U.S. and Europe, a majority of which have the major genotype which requires regular blood transfusions and would be candidates for LentiGlobin. 


J.P. Morgan analyst Cory Kasimov wins the award for best research note headline with this gem: "Bluebird Bio: I see BLUE, and It Looks Glorious"

Kasimov:


We weren't expecting to quickly bring back additional references to our initiation title for the company that is essentially the polar opposite of "Old School," but Saturday's presentation of initial data from the Phase 1/2 HGB-205 study evaluating BLUE's gene therapy LentiGlobin in beta-thalassemia patients at the European Hematology Association (EHA) Congress in Milan exceeded even the most optimistic expectations and seems more than worthy of the description. We were bullish going into the event and are no doubt pleased by the early and high-level production of corrected AT87Q-globin observed (demonstrates that the gene therapy is indeed doing what it's supposed to be doing). However, what's of far greater surprise is that the first two patients treated in this clinical trial not only became transfusion independent but did so very rapidly. Based on this rather striking (albeit early) data, we anticipate BLUE shares could be sharply higher on Monday.


Bluebird is holding a conference call at 8 am ET.

Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.

To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.

Markets

DOW 17,279.74 +13.75 0.08%
S&P 500 2,010.40 -0.96 -0.05%
NASDAQ 4,579.7890 -13.6380 -0.30%

Adam's Tweets

Brokerage Partners

Select the service that is right for you!

COMPARE ALL SERVICES
Action Alerts PLUS

Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
  • Weekly roundups
Real Money Pro

All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.

Product Features:
  • Real Money + Doug Kass Plus 15 more Wall Street Pros
  • Intraday commentary & news
  • Ultra-actionable trading ideas
Trifecta Stocks

Trifecta Stocks analyzes over 4,000 equities weekly to find the elite 1% of stocks that pass rigorous quantitative, fundamental and technical tests.

Product Features:
  • Model portfolio
  • Trade alerts
  • Recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
Stocks Under $10

David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
  • Weekly roundups
Quant Ratings

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
  • Upgrade/downgrade alerts
Dividend Stock Advisor

Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Alerts when market news affect the portfolio
  • Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
Breakout Stocks

Bryan Ashenberg, using sophisticated stock screening and fundamental research, identifies potentially explosive small and mid-cap stocks.

Product Features:
  • Model portfolio
  • Small-cap and mid-cap focus
  • Intraday trade alerts
  • Weekly roundups
Options Profits

Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • 100+ monthly options trading ideas
  • Actionable options commentary & news
  • Real-time trading community
  • Options TV
Top Rated Stocks Top Rated Funds Top Rated ETFs