Forma Therapeutics, Inc.

Novo Nordisk Healthcare Ag

Patrick F Kelly

Susan Ashwell

Wei Lu

Alan Collis

Duncan Walker

Blythe Thomson

Jeff Davis

David R Lancia, Jr

Anna M Ericsson

Zhongguo Wang

Gary Gustafson

Neal Green

Gary Marshall

Lorna Mitchell

Madhu Mondal

Sanjeev Forsyth

Maria Ribadeneira

Patricia Schroeder

David R Lancia

Lu Wei

The compound (S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one, or a pharmaceutically acceptable salt thereof, is useful to increase the affinity of hemoglobin for oxygen. Methods and compositions for the treatment of a hemoglobinopathies are provided herein, including certain pharmaceutical compositions for activating PKR.

Activating pyruvate kinase R

Patients diagnosed with a cancer harboring an IDH-1 mutation can be treated by the administration of a therapeutically effective amount of a pharmaceutical composition comprising Compound 1, a selective inhibitor of 2-HG production from mIDH-1 enzymes including the R132 mutations R132C, R132H, R132L, R132G, and R132S.

Inhibiting mutant isocitrate dehydrogenase 1 (mIDH-1)

Methods of treating patients diagnosed with cancer harboring an IDH-1 mutation are provided, including the therapeutic administration of a certain inhibitor of a mutant IDH-1 as a single agent, or in combination with azacitidine (AZA).

Inhibiting mutant IDH-1

Methods of treating patients diagnosed with AML or MDS harboring mutant IDH-1 include detecting an IDH1 mutation and the therapeutic administration of an inhibitor of a mutant IDH-1 as a single agent, or in combination with azacitidine (AZA) or cytarabine.

Treating patients harboring an isocitrate dehydrogenase 1 (IDH-1) mutation

Inhibiting mutant isocitrate dehydrogenase 1 (mlDH-1)

Treating patients harboring an isocitrate dehydrogenase-1 (IDH-1) mutation

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Concord, MA, United States of America

Patrick F Kelly