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Agios Pharma (AGIO) to complete Phase 2 study of AG-348 in Patients With Pyruvate Kinase Deficiency in Q3 2017

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Additional Relevant Details PK deficiency disease understanding is still evolving. The disease typically presents during early infancy with jaundice and severe anemia, which can require immediate life-saving intervention via replacement of the infant’s entire blood system with a donor’s blood, referred to as an exchange transfusion. The current standard of care for PK deficiency is supportive, including blood transfusions, splenectomy, chelation therapy to address iron overload and/or interventions for other treatment- and disease-related morbidities. Currently, there is no approved therapy to treat the underlying cause of PK deficiency.
Additional Relevant Details Update on June 24, 2017: Safety DataA safety analysis conducted for all 52 treated patients as of the data cut-off shows that AG-348 continues to be well tolerated.
  • The majority of treatment-related adverse events (AEs) were Grade 1-2; the most frequent were headache, insomnia and nausea.
  • Three patients experienced treatment related AEs leading to discontinuation: chest discomfort/pleural effusion (n=1), pharyngitis/nausea (n=1) and anemia (n=1).
  • Five patients experienced drug-related serious adverse events: withdrawal hemolysis followed by anemia (n=1), anemia (n=1), osteoporosis (n=1), hypertriglyceridemia (n=1) and pharyngitis (n=1).
    ° Grade 4 hypertriglyceridemia at week 24 resolved upon AG-348 discontinuation (patient had Grade 1 hypertriglyceridemia at baseline).
  • Preliminary measurements of testosterone in men suggest aromatase inhibition by AG-348 with the majority of testosterone changes remaining within the normal range. Longer follow-up is required to assess clinical significance.
Efficacy DataIn the efficacy analysis of all 52 treated patients, 25 patients overall and 24 of 42 patients with at least one missense mutation achieved rapid, robust and sustained Hb increases from baseline of >1.0 g/dL as of the data cut-off.
  • In patients who had Hb increases of >1.0 g/dL, the mean maximum Hb increase was 3.5 g/dL (range 1.1-5.8 g/dL).
  • The median time to a Hb increase of >1.0 g/dL was 10 days (range 7–141 days).
  • Median baseline Hb in patients who experienced a maximum Hb increase of >1.0 g/dL was 9.7 g/dL (range 7.5–12.3 g/dL) vs. 8.0 g/dL (range 6.5–10.1 g/dL) in patients who did not experience the increase.
  • In patients with a Hb increase of >1.0 g/dL improvements in hemolysis associated parameters were observed:
    ° An increase in haptoglobin and decrease in lactate dehydrogenase (LDH) were observed in the first weeks of dosing.
    ° Rapid decreases in reticulocytes were observed.
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Related Keywords Phase 2 Study, Ag-348, Pyruvate Kinase Deficiency, Pk Deficiency, Open Label