- Agios Pharmaceuticals (AGIO -9%) slumps on slightly higher volume in response to its announcement of updated results from a Phase 2 clinical trial, DRIVE PK, assessing AG-348 for the treatment of patients with pyruvate kinase (PK) deficiency. The data were presented at the European Hematology Association Annual Meeting in Madrid.
- As of the March 27 data cutoff, 48% (n=25/52) of all 52 treated patients experienced a maximum increase in hemoglobin (Hb) of greater than 1.0 g/dL from baseline. Data reported in December 2016 showed 58% (n=15/26) achieved the maximum increase in Hb. In May 2016, the percentage was 68% (n=13/19).
- On the safety front, the most frequent mild/moderate adverse events (AEs) in the treatment group were headache, insomnia and nausea. Three patients discontinued treatment: chest discomfort/pleural effusion, pharyngitis/nausea and anemia. Five participants experienced serious AEs: withdrawal hemolysis followed by anemia (n=1), anemia (1), osteoporosis (1), hypertriglyceridemia (1) and pharyngitis (1).
- PK is an enzyme that plays a key role in glycolysis, the conversion of glucose into lactic acid. PK has several tissue-specific isoforms (PKR, PKL, PKM1, PKM2). Inherited mutations in PKR enzymes cause a deficit in cellular energy within red blood cells which leads to the clinical manifestations of PK deficiency.
- The rationale for AG-348, an oral activator of PKR, is the restoration of metabolic function and the correction of the underlying defect in red blood cells in PK deficiency sufferers.
- The company intends to advance AG-348 into Phase 3 development in H1 2018.
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