BioSpace News Archive

Genetown

BeiGene Presents Clinical Data on Zanubrutinib and Tislelizumab at the 25th European Hematology Association (EHA) Virtual Congress

BeiGene, Ltd. announced that data on its BTK inhibitor zanubrutinib in relapsed/refractory marginal zone lymphoma and other B-cell malignancies and its anti-PD-1 antibody tislelizumab in R/R NK/T-cell lymphomas were presented at the 25th European Hematology Association Virtual Congress, taking place on June 11-14.

June 12, 2020
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20 min read
Drug Development

Agios First-in Class PKR Activator Mitapivat Demonstrates Sustained Hemoglobin Responses in Non-transfusion-dependent α- and β-Thalassemia in Phase 2 Study

Agios Pharmaceuticals, Inc., a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, reported interim data from its ongoing Phase 2 study evaluating single agent mitapivat in non-transfusion-dependent α- and β-thalassemia.

June 12, 2020
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9 min read
Drug Development

Agios Announces Clinical Proof-of-Concept Has Been Established in Phase 1 Study of Mitapivat, First-in Class PKR Activator, in Sickle Cell Disease

Safety Profile Consistent with Previously Published Phase 2 Data for Mitapivat in Patients with Pyruvate Kinase Deficiency or Expected in the Context of Sickle Cell Disease

June 12, 2020
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8 min read
Drug Development

X4 Pharmaceuticals Presents Positive Clinical Data from Phase 2 Study of Mavorixafor in WHIM Syndrome at EHA 2020

Sustained efficacy and safety trends observed for up to 28.6 months support ongoing pivotal Phase 3 trial dosing and endpoints, including selection of the measurement of time above threshold for absolute neutrophil count as primary endpoint

June 12, 2020
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6 min read
Genetown

New Pivotal Data Demonstrate Superiority of Apellis’ Pegcetacoplan to Eculizumab in Improving Hemoglobin Levels, Independent of Prior Transfusions, in PNH

Apellis Pharmaceuticals, Inc., a global biopharmaceutical company pioneering targeted C3 therapies, presented detailed results from the Phase 3 PEGASUS study, which in January showed superiority for pegcetacoplan over eculizumab in improving hemoglobin levels in adults with paroxysmal nocturnal hemoglobinuria.

June 12, 2020
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9 min read
Genetown

Constellation Pharmaceuticals Provides Updates of MANIFEST Study for CPI-0610 and EZH2 Franchise

Spleen response rates in first-line patients at 12 weeks and 24 weeks in line with previously reported data and differentiated from standard of care

June 12, 2020
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11 min read
Genetown

CRISPR Therapeutics and Vertex Announce New Clinical Data for Investigational Gene-Editing Therapy CTX001™ in Severe Hemoglobinopathies at the 25th Annual European Hematology Association (EHA) Congress

CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 Phase 1/2 trials in transfusion-dependent beta thalassemia and severe sickle cell disease, and highlighted recent progress in the CTX001 development program.

June 12, 2020
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15 min read
EHA25Virtual: Pegcetacoplan, a Novel C3 Complement Inhibitor - Beginning of a New Era in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH)

PNH is a rare but severe and usually life-long blood disease in which the blood cells are fragile and easily destroyed by a part of the patient’s own immune system, called complement.

June 12, 2020
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1 min read
Sierra Oncology Reports Favorable Long-Term Safety and Dose Intensity Data for Momelotinib

Data presented at the 25th European Hematology Association (EHA) Virtual Congress further demonstrate the potential anemia benefits and favorable hematological safety profile of momelotinib

June 12, 2020
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7 min read
Drug Development

ALX Oncology Presents Preliminary Phase 1 Study Results of ALX148 in Combination with Rituximab in Patients with Relapsed or Refractory Non-Hodgkin Lymphoma

Preliminary Results from Fully Enrolled ALX148 Plus Rituximab Combination Cohort Presented at the 25th Congress of the European Hematology Association

June 12, 2020
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3 min read