— First-in-class Clinical Program Targets Cancer Metabolism in Genetically Defined Patient Population —
CAMBRIDGE, MA, USA I September 23, 2013 I Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), today announced dose administration for the first patient in a Phase 1 study of AG-221 in patients with advanced hematologic malignancies with an isocitrate dehydrogenase-2 (IDH2) mutation. AG-221 is an oral, selective, potent inhibitor of the mutated IDH2 protein, making it the first targeted therapeutic candidate to treat patients with cancers that harbor IDH2 mutations.
“Agios is a first mover in targeting dysregulated cellular metabolism to develop potentially transformative medicines. With AG-221, we are combining our deep understanding of metabolism and cancer genetics in an effort to bring a precisely targeted new treatment option to patients with IDH2-mutant hematological cancers.”
“IDH2 represents one of the most promising targets in cancer biology today,” said David Schenkein, M.D., chief executive officer at Agios. “Agios is a first mover in targeting dysregulated cellular metabolism to develop potentially transformative medicines. With AG-221, we are combining our deep understanding of metabolism and cancer genetics in an effort to bring a precisely targeted new treatment option to patients with IDH2-mutant hematological cancers.”
“Cancer drug development is advancing at a remarkable pace, and we think that patients will benefit as our therapies evolve to target specific genetic abnormalities,” said Eytan Stein, M.D., lead investigator at Memorial Sloan-Kettering Cancer Center. “By enrolling patients with the appropriate genetic background, we hope to expedite our understanding of the safety, tolerability and efficacy of AG-221.”
AG-221 is part of Agios’ global strategic collaboration with Celgene, a leading biotechnology company. Established in 2010, the goal of the partnership is to discover, develop and deliver novel disease-altering oncology therapies based on Agios’ cancer metabolism research platform.
About the Study
The Phase 1 multicenter, open-label, dose escalation clinical trial of AG-221 is designed to assess the safety and tolerability of AG-221 as a single agent administered orally twice daily in a 28-day cycle. The study is expected to only enroll subjects who have an IDH2-mutant hematologic malignancy, including acute myelogenous leukemia (AML) and myelodysplastic syndrome. Key objectives in the study include determining maximum tolerated dose, pharmacokinetics, pharmacodynamics (including inhibition of the oncometabolite 2-hydroxyglutarate, or 2-HG) and preliminary anti-tumor activity of AG-221. Disease-specific expansion cohorts will be enrolled at the maximally tolerated or biologically relevant dose. Please refer to www.clinicaltrials.gov for additional clinical trial details.
About IDH Mutations
The IDH protein is a critical metabolic enzyme in the citric acid cycle, also known as the tricarboxylic acid (TCA), or Krebs cycle. Agios’ scientists first established that the mutated forms of IDH produce a metabolite, 2-HG, which may contribute to the formation and malignant progression of various forms of cancer. Agios and its collaborators recently demonstrated that IDH1 and IDH2 mutations initiate and drive cancer growth by blocking differentiation, also referred to as maturation, of primitive cells. Agios believes that inhibition of these mutated proteins may lead to clinical benefit for the subset of cancer patients whose tumors carry these mutations.
The connection between cancer and metabolism has been the central focus of scientists at Agios, who were the first to identify the neo-activity of IDH1 mutations to produce the oncometabolite 2-HG in research published in Nature in 2009. These insights revealed the potential of IDH1 and IDH2 mutations as novel therapeutic targets in cancer. The IDH1 gene mutation was initially discovered in brain cancers in 2008 by researchers at Johns Hopkins. More recently, mutations in both IDH1 and IDH2 have been linked to hematologic malignancies including AML, one of the most common types of leukemia in adults, as well as several other cancers.
About Agios Pharmaceuticals, Inc.
Agios Pharmaceuticals is focused on discovering and developing novel drugs to treat cancer and rare metabolic genetic diseases (known as inborn errors of metabolism or IEMs) through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class lead product candidates in cancer metabolism and IEMs in clinical and preclinical development. All Agios programs focus on genetically identified patient populations leveraging our knowledge of metabolism, biology and genomics. For more information, please visit our website at www.agios.com.
SOURCE: Agios Pharmaceuticals
Post Views: 115
— First-in-class Clinical Program Targets Cancer Metabolism in Genetically Defined Patient Population —
CAMBRIDGE, MA, USA I September 23, 2013 I Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), today announced dose administration for the first patient in a Phase 1 study of AG-221 in patients with advanced hematologic malignancies with an isocitrate dehydrogenase-2 (IDH2) mutation. AG-221 is an oral, selective, potent inhibitor of the mutated IDH2 protein, making it the first targeted therapeutic candidate to treat patients with cancers that harbor IDH2 mutations.
“Agios is a first mover in targeting dysregulated cellular metabolism to develop potentially transformative medicines. With AG-221, we are combining our deep understanding of metabolism and cancer genetics in an effort to bring a precisely targeted new treatment option to patients with IDH2-mutant hematological cancers.”
“IDH2 represents one of the most promising targets in cancer biology today,” said David Schenkein, M.D., chief executive officer at Agios. “Agios is a first mover in targeting dysregulated cellular metabolism to develop potentially transformative medicines. With AG-221, we are combining our deep understanding of metabolism and cancer genetics in an effort to bring a precisely targeted new treatment option to patients with IDH2-mutant hematological cancers.”
“Cancer drug development is advancing at a remarkable pace, and we think that patients will benefit as our therapies evolve to target specific genetic abnormalities,” said Eytan Stein, M.D., lead investigator at Memorial Sloan-Kettering Cancer Center. “By enrolling patients with the appropriate genetic background, we hope to expedite our understanding of the safety, tolerability and efficacy of AG-221.”
AG-221 is part of Agios’ global strategic collaboration with Celgene, a leading biotechnology company. Established in 2010, the goal of the partnership is to discover, develop and deliver novel disease-altering oncology therapies based on Agios’ cancer metabolism research platform.
About the Study
The Phase 1 multicenter, open-label, dose escalation clinical trial of AG-221 is designed to assess the safety and tolerability of AG-221 as a single agent administered orally twice daily in a 28-day cycle. The study is expected to only enroll subjects who have an IDH2-mutant hematologic malignancy, including acute myelogenous leukemia (AML) and myelodysplastic syndrome. Key objectives in the study include determining maximum tolerated dose, pharmacokinetics, pharmacodynamics (including inhibition of the oncometabolite 2-hydroxyglutarate, or 2-HG) and preliminary anti-tumor activity of AG-221. Disease-specific expansion cohorts will be enrolled at the maximally tolerated or biologically relevant dose. Please refer to www.clinicaltrials.gov for additional clinical trial details.
About IDH Mutations
The IDH protein is a critical metabolic enzyme in the citric acid cycle, also known as the tricarboxylic acid (TCA), or Krebs cycle. Agios’ scientists first established that the mutated forms of IDH produce a metabolite, 2-HG, which may contribute to the formation and malignant progression of various forms of cancer. Agios and its collaborators recently demonstrated that IDH1 and IDH2 mutations initiate and drive cancer growth by blocking differentiation, also referred to as maturation, of primitive cells. Agios believes that inhibition of these mutated proteins may lead to clinical benefit for the subset of cancer patients whose tumors carry these mutations.
The connection between cancer and metabolism has been the central focus of scientists at Agios, who were the first to identify the neo-activity of IDH1 mutations to produce the oncometabolite 2-HG in research published in Nature in 2009. These insights revealed the potential of IDH1 and IDH2 mutations as novel therapeutic targets in cancer. The IDH1 gene mutation was initially discovered in brain cancers in 2008 by researchers at Johns Hopkins. More recently, mutations in both IDH1 and IDH2 have been linked to hematologic malignancies including AML, one of the most common types of leukemia in adults, as well as several other cancers.
About Agios Pharmaceuticals, Inc.
Agios Pharmaceuticals is focused on discovering and developing novel drugs to treat cancer and rare metabolic genetic diseases (known as inborn errors of metabolism or IEMs) through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline across both therapeutic areas, Agios has multiple first-in-class lead product candidates in cancer metabolism and IEMs in clinical and preclinical development. All Agios programs focus on genetically identified patient populations leveraging our knowledge of metabolism, biology and genomics. For more information, please visit our website at www.agios.com.
SOURCE: Agios Pharmaceuticals
Post Views: 115
