This Day On The Street
Continue to site
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, Agios: Preview of Next Week's Important Pipeline Updates

NEW YORK ( TheStreet) -- Two members of the Biotech IPO Class of 2013 -- Bluebird Bio (BLUE - Get Report) and Agios Pharmaceuticals (AGIO - Get Report) -- will deliver important pipeline updates at the European Hematology Association (EHA) Congress on Sat. June 14. Let's preview both presentations, starting with Bluebird and its LentiGlobin gene therapy for beta-thalassemia.

Lenti what for beta who? 

LentiGlobin is gene therapy, meaning it inserts a fully functional gene for human beta-globin into the patient's own hematopoietic stem cells. The theory behind gene therapy is relatively simple: For diseases caused by single, malfunctioning or missing gene, effective treatment or even a complete cure can be achieved (theoretically) by replacing the defective gene with one that is fully functional. Gene therapy sounds simple but the execution is obviously a lot more complicated.

I won't get into the weeds explaining the process by which BlueBird produces and delivers LentiGlobin except to say a working gene for human beta-globin would allows beta-thalassemia (B-Thal) patients to produce functional beta-globin -- the largest component of hemoglobin, which carries oxygen in red blood cells. B-Thal patients suffer from anemia and iron overload. There is no currently approved cure or effective treatment, so patients require regular blood transfusions to combat the anemia. 

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. 

Next Saturday, researchers will present data on two B-thal patients treated with LentiGlobin in an ongoing phase I/II study. Key data to look for include the engraftment success rate i.e. how many "gene-therapy treated" stem cells are populating the bone marrow; improvements in hemoglobin levels; achievement of transfusion independence; and if so, how quickly from the gene therapy. 

Agios will be presenting a clinical update from an early-stage study of experimental cancer metabolism drug  AG-221 last reported at the American Association of Cancer Research annual meeting in April

Agios is developing AG-221 under a collaboration with Celgene (CELG). The companies are also working together on AG-120, a similar drug designed to block another mutated cancer-growing protein known a IDH1.

At the EHA meeting on Sat. June 14, researchers will be presented updated study results from additional patients treated with AG-221, some given higher doses of the drug. 

In April at AACR, six of 10 patients with advanced, treatment-refractory acute myeloid leukemia carrying the IDH2 mutation had objective tumor responses, including three complete remissions and two complete remissions with incomplete platelet recovery. A single patient achieved a partial response. 

Wondering how BlueBird and Agios have fared since their public equity debuts? 

BLUE Chart
BLUE data by YCharts

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.

Check Out Our Best Services for Investors

Action Alerts PLUS

Portfolio Manager Jim Cramer and Director of Research Jack Mohr reveal their investment tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
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
Stocks Under $10

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

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
14-Days Free
Only $9.95
14-Days Free
Dividend Stock Advisor

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
  • Updates with exact steps to take - BUY, HOLD, SELL
Trifecta Stocks

Every recommendation goes through 3 layers of intense scrutiny—quantitative, fundamental and technical analysis—to maximize profit potential and minimize risk.

Product Features:
  • Model Portfolio
  • Intra Day Trade alerts
  • Access to Quant Ratings
Real Money

More than 30 investing pros with skin in the game give you actionable insight and investment ideas.

Product Features:
  • Access to Jim Cramer's daily blog
  • Intraday commentary and news
  • Real-time trading forums
Only $49.95
14-Days Free
14-Days Free
AGIO $70.35 2.70%
BLUE $93.45 4.40%
AAPL $117.83 -0.17%
FB $105.28 -0.12%
GOOG $749.85 0.23%


Chart of I:DJI
DOW 17,791.81 -21.58 -0.12%
S&P 500 2,088.89 +0.02 0.00%
NASDAQ 5,122.7270 +6.5840 0.13%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs