This Day On The Street
Continue to site
ADVERTISEMENT
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

Studies Presented At 19th European Hematology Association Annual Congress Evaluate Activin Receptor Ligand Trap Programs In Beta-Thalassemia

Celgene Corporation (NASDAQ:CELG) and Acceleron Pharma Inc. (NASDAQ:XLRN) today announced interim data from two studies of candidates from the companies’ joint development program in beta-thalassemia. The data presented at the 19 th European Hematology Association (EHA) annual congress show activity for two programs, sotatercept and ACE-536, in patients with both transfusion dependent and non-transfusion dependent beta-thalassemia. There is currently no drug approved to treat beta-thalassemia. Both sotatercept and ACE-536 have been granted orphan drug designation for beta-thalassemia by the United States Food and Drug Administration (FDA).

Sotatercept

In the first study, which was highlighted as part of the EHA press briefing on June 13, sotatercept, a novel activin type IIA receptor fusion protein, was evaluated in transfusion-dependent (TD) and non-transfusion-dependent (NTD) beta-thalassemia patients.

A total of 32 patients were treated in this dose-finding study in which sotatercept was administered subcutaneously once every 3 weeks at doses of 0.1 (n=8), 0.3 (n=9), 0.5 (n=8), or 0.75 (n=7) mg/kg. Of these patients, 22 were non-transfusion dependent and 10 were transfusion dependent.

The mean baseline hemoglobin level for the NTD patients was 8.7 g/dL (range 6.1-10.7), 8.3 g/dL (range 6.0-9.5), 8.2 g/dL (range 6.4-9.3) and 8.8 g/dL (range 7.9-9.6) in the 0.1, 0.3, 0.5 and 0.75 mg/kg groups, respectively. Among these patients, there was a dose-dependent increase in hemoglobin.
  • The proportion of patients achieving a maximum hemoglobin increase of ≥ 1.0 g/dL above baseline was 0%, 67%, 83% and 100% in the 0.1, 0.3, 0.5, and 0.75 mg/kg dose groups, respectively.
  • The proportion of patients achieving a maximum hemoglobin increase of ≥ 2.0 g/dL above baseline was 0%, 0%, 33% and 50% in the 0.1, 0.3, 0.5 and 0.75 mg/kg dose groups, respectively.

In TD patients, there were dose dependent reductions in transfusion burden.
  • The proportion of patients achieving ≥ 20% reduction in transfusion burden was 0%, 33%, 50% and 67% in the 0.1, 0.3, 0.5, and 0.75 mg/kg dose groups, respectively.
  • The proportion of patients achieving ≥ 50% reduction in transfusion burden was 0%, 0%, 0% and 33% in the 0.1, 0.3, 0.5, and 0.75 mg/kg dose groups, respectively.

Of these 32 patients, 25 remain on treatment, with the longest duration of treatment > 78 weeks (patient still on study). Three treatment-related adverse events of grade 2 or higher occurred, leading to treatment discontinuation. In the 0.1 mg/kg group, one TD patient experienced worsening grade 3 bone pain and one NTD patient experienced grade 2 phlebitis. In the 0.5 mg/kg group, one patient with a history of ventricular extrasystoles experienced a grade 3 adverse event of ventricular extrasystoles.

1 of 4

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
Options Profits

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

Product Features:
  • Actionable options commentary and news
  • Real-time trading community
SYM TRADE IT LAST %CHG

Markets

DOW 17,683.58 -46.53 -0.26%
S&P 500 2,068.76 -8.02 -0.39%
NASDAQ 4,991.94 -17.2740 -0.34%

Free Reports

Top Rated Stocks Top Rated Funds Top Rated ETFs