Hematopoietics

Kadmon Announces Positive Topline Results of Pivotal Trial of Belumosudil in Chronic Graft-Versus-Host Disease

NEW YORK, NY / ACCESSWIRE / May 21, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced positive topline results from the primary analysis of ROCKstar (KD025-213), the pivotal trial evaluating belumosudil (KD025) in patients with chronic graft-versus-host disease (cGVHD) who have received at least two prior lines of systemic therapy. Belumosudil (KD025) achieved clinically meaningful …

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Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies

BOSTON–(BUSINESS WIRE)–Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, today announced positive topline results from its Phase 3 clinical study evaluating the safety and efficacy of omidubicel, an investigational advanced cell therapy in development as a potential life-saving …

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New Data Demonstrates Andexxa Can Provide a Net Cost Reduction for the Treatment of Intracranial Hemorrhage Associated with Oral Factor Xa Inhibitors

SOUTH SAN FRANCISCO, Calif., March 5, 2020 /PRNewswire/ — Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced the presentation of new data demonstrating that using Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo] to treat patients with intracranial hemorrhage (ICH) associated with the oral Factor Xa inhibitors apixaban or rivaroxaban is projected to …

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Astex Announces FDA Accepts for Priority Review its NDA for Oral C-DEC for the Treatment of MDS and CMML

PLEASANTON, Calif.–(BUSINESS WIRE)–Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Japan, today announced that the U.S. FDA has accepted for Priority Review its NDA for oral C-DEC (cedazuridine and decitabine) as a treatment for adults with previously untreated intermediate- and high-risk MDS including CMML. …

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FDA Grants Fast Track Designation to Imago’s Bomedemstat for Treatment of Essential Thrombocythemia

SAN FRANCISCO–(BUSINESS WIRE)–Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the development of bomedemstat (IMG-7289) for the treatment of essential thrombocythemia (ET), a bone marrow disease associated with high …

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Akari Therapeutics Initiates Pivotal Phase 3 Trial of Nomacopan in Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic Microangiopathy

NEW YORK and LONDON, Dec. 23, 2019 (GLOBE NEWSWIRE) — Akari Therapeutics, Plc (Nasdaq:AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, announces that a U.S. Food and Drug Administration (FDA) investigational new drug application (IND) …

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FDA Grants Breakthrough Therapy Designation to Bristol-Myers Squibb’s Orencia to Help Prevent Acute Graft-Versus-Host Disease

PRINCETON, N.J.–(BUSINESS WIRE)–Bristol-Myers Squibb Company (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for ORENCIA® (abatacept) for the prevention of moderate to severe acute graft-versus-host disease (GvHD) in hematopoietic stem cell transplants from unrelated donors. There are no approved therapies for the prevention of …

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Omeros Reports Positive Data Across Primary and Secondary Endpoints in Pivotal Trial of HSCT-Associated Thrombotic Microangiopathy Patients Treated with Narsoplimab

SEATTLE–(BUSINESS WIRE)–Omeros Corporation (Nasdaq: OMER) today announced positive data from its pivotal clinical trial of the company’s novel investigational complement inhibitor narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a frequently lethal complication of HSCT. These preliminary data were recently provided to FDA as part of …

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CRISPR Therapeutics and Vertex Announce Positive Early Results for its Investigational CTX001 for Severe Hemoglobinopathies

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 19, 2019 (GLOBE NEWSWIRE) — CRISPR Therapeutics (NASDAQ: CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. One patient with transfusion-dependent …

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Vertex and Molecular Templates Collaborate to Discover and Develop Novel Targeted Conditioning Regimens to Enhance Hematopoietic Stem Cell Transplants

BOSTON and AUSTIN, Texas, Nov. 18, 2019 (GLOBE NEWSWIRE) — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and Molecular Templates, Inc. (Nasdaq: MTEM; “Molecular Templates” or “MTEM”) today announced that the two companies have entered into a strategic research collaboration to discover and develop novel targeted conditioning regimens that may enhance the …

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