Positive first results for a new medicine against Pyruvate Kinase Deficiency
Agios Pharmaceuticals has concluded the first Phase trial in 64 healthy volunteers of a new medicine AG-348 that targets the underlying cause of Pyruvate Kinase Deficiency. Results were positive: AG-348 led to a substantial increase in Pyruvate Kinase enzymatic activity, had a favorable safety profile and was well tolerated during a 14 days treatment in healthy volunteers.
Read the news release from Agios here.
The results were presented at the 56th Annual Meeting of the American Society of Hematology (ASH). The abstract is available on their website.