Entering text into the input field will update the search result below

Agios' AG-348 successful in mid-stage study in rare congenital anemia; shares up 9% premarket

Jun. 13, 2016 9:20 AM ETAgios Pharmaceuticals, Inc. (AGIO) StockBy: Douglas W. House, SA News Editor
  • Agios Pharmaceuticals (NASDAQ:AGIO) jumps 9% premarket on increased volume in response to its announcement of positive results in a Phase 2 clinical trial, DRIVE-PK, assessing AG-348 in patients with pyruvate kinase deficiency (PKD), a rare and potentially debilitating inherited anemia. The data were presented at the 21st Congress of the European Hematology Association in Copenhagen.
  • As of the March 27 data cutoff, 18 transfusion-dependent PKD patients were treated with AG-348 twice daily for up to six months. 50% (n=9/18) showed rapid and sustained increases in hemoglobin greater than 1.0 g/dL (range: 2.3 - 4.9 g/dL), including 69% (n=9/13) of patients with at least one missense mutation (a type of mutation in one DNA base pair that results in the substitution of one amino acid for another in the protein made by the gene). About 80% of PKD patients carry at least one missense mutation.
  • DRIVE-PK includes two arms of 25 subjects each, one receiving 50 mg of AG-348 twice daily and the other 300 mg twice daily. Of the nine patients who responded with an increase in hemoglobin at least 1.0 g/dL, four were in the 50 mg arm and five in the 300 mg arm. The median time to the increase was 1.9 weeks. None of the five patients with two non-missense mutations achieved the increase in hemoglobin.
  • PKD presents like hemolytic anemia, characterized by the accelerated destruction of red blood cells. There are no approved therapies to treat the underlying cause of the disorder.

Recommended For You

More Trending News

About AGIO Stock

SymbolLast Price% Chg
Market Cap
PE
Yield (TTM)
Rev Growth (YoY)
Prev. Close
Compare to Peers

Related Stocks

SymbolLast Price% Chg
AGIO--
Agios Pharmaceuticals, Inc.