CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today reported a full analysis of updated data, including patient-reported outcome (PRO) measures, from its global Phase 3 ACTIVATE and ACTIVATE-T studies of mitapivat in adults with pyruvate kinase...
treatment News
Cambridge, Mass. – Rare disease company Agios Pharmaceuticals reached the primary endpoint in a Phase III study for its drug pyrukynd (mitapivat) in adults with non-transfusion-dependent alpha- or beta-thalassemia, according to the company’s announcement on Wednesday The Phase III ENERGIZE study met the primary endpoint of hemoglobin response, defined as...
CAMBRIDGE, Mass. — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the U.S. accelerated approval of mitapivat, an oral...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat genetically defined diseases, today announced positive results from its Phase 2, open-label, multicenter study of mitapivat in adults with non-transfusion dependent . Data from the study will be featured in an oral...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, announced that it has submitted a New Drug Application (NDA) for mitapivat to the U.S. Food and Drug Administration (FDA) for the treatment of adults...
CAMBRIDGE, Mass. – Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, today announced that it has submitted a Supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for TIBSOVO® (ivosidenib tablets) as a potential...
CAMBRIDGE, Mass. — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that new data on mitapivat, an oral pyruvate kinase (PK) activator, will be featured in oral and poster presentations during the 31st European Hematology Association (EHA) Congress...
GAINESVILLE, Fla., and CAMBRIDGE, Mass., – Applied Genetics Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, which include X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM), announces two important initiatives to help patients with potential interest...
GAINESVILLE, Fla. and CAMBRIDGE, Mass. – Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today reported 12-month data from its ongoing achromatopsia (ACHM) Phase 1/2 clinical trials, including data from all adult...
GAINESVILLE, Fla. and CAMBRIDGE, Mass. – Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare retinal diseases, today reported additional positive data from the ongoing X-linked retinitis pigmentosa (XLRP) Phase 1/2 clinical trial, including 12-month...