After hearing two conflicting presentations of the safety and efficacy of Geron Corp.’s imetelstat, the U.S. FDA’s Oncology Drugs Advisory Committee (ODAC) voted 12-2 March 14 that the drug’s benefit outweighed its risks as a treatment for transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes in patients who have failed or no longer respond to erythropoiesis stimulating agents (ESAs), or who are not eligible for ESA treatment.
Although Geron Corp.’s imetelstat met its primary and key secondary endpoints in a phase III study, the U.S. FDA is questioning the magnitude and durability of the effect of the first-in-class telomerase inhibitor as a second-line treatment of transfusion-dependent anemia in adults with low- to intermediate-1 risk myelodysplastic syndromes. The agency’s concerns resulted in more than an 12% stock tumble March 12 after the FDA released its briefing document two days ahead of an Oncology Drugs Advisory Committee meeting, in which the panel will be asked to vote on whether imetelstat’s benefits outweigh its risks.
Iron regulates the metabolism of hematopoietic stem cells (HSCs) and acts as a genetic control of their fate, preserving their identity and regenerative capacity during tissue maintenance and repair. A group of scientists at Albert Einstein College of Medicine has described the key components of a molecular pathway that iron regulates. “What we are proposing here with this mechanism is that iron serves like a switchboard and a sensor,” senior author Britta Will told BioWorld. Will is at the Department of Oncology, the Cell Biology Department, and the Ruth and David Gottesman Institute for Stem Cell Research and Regenerative Medicine at Albert Einstein College of Medicine.
In a paper published on Feb. 21, 2024, in Science Translational Medicine, researchers from the Versiti Blood Research Institute described how they successfully controlled bleeding for months in hemophilia A models using a siRNA therapy that targeted plasminogen.
Hun-taek Kim founded Tiumbio Co. Ltd. in 2016 after spending more than two decades at a major chemical and life science firm, SK Chemicals Co. Ltd. “The prospects for our three major assets are very bright, and the probability of failure is low,” CEO Kim told BioWorld. “We’re looking for a breakthrough in rare diseases – to develop new treatments for [niche] markets with large unmet demand.”
In a recent study, researchers from Technische Universität Dresden and affiliated organizations evaluated the novel small-molecule inhibitor of the CBP/p300 HAT domain, A-485, as a potential therapeutic candidate for the treatment of hematological cancers and other pathologies related to blood leukocyte compartment.
The European Commission approved two therapies for progressive, genetic diseases: Biogen Inc.’s Friedreich’s ataxia drug, Skyclarys (omaveloxolone), and Crispr Therapeutics AG’s CRISPR/Cas9 gene therapy for sickle cell disease and transfusion-dependent beta-thalassemia, Casgevy (exagamglogene autotemcel, exa-cel).
Bleeding of unknown cause is a group of rare disorders that are still difficult to accurately diagnose. A case report on a patient with hematoma in the perineal region after her first delivery was presented.
The rare hereditary fibrinogen disorder hypodysfibrinogenemia is characterized by fibrinogen defects, which can cause thrombotic and hemorrhagic phenotypes that are not always predicted by routine coagulation tests. Researchers from Academic Hospital Maastricht aimed to characterize the genetic profile of a family with hypodysfibrinogenemia and predict bleeding and/or thrombotic phenotypes in asymptomatic family members using innovative testing.
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