Syntara Ltd.’s stock plummeted 52% on news that the FDA recommended an additional phase IIb trial following a type C meeting about the company’s clinical development plan for lysyl oxidase inhibitor amsulostat (SNT-5505, formerly PXS-5505) in myelofibrosis.
Hebei Yiling Medicine Research Institute Co. Ltd. has described piperidine indole derivatives targeting complement factor B (CFB) reported to be useful for the treatment of inflammatory disorders, and hematological, neurodegenerative and autoimmune diseases.
Agios Pharmaceuticals Inc. said the patients’ deaths tied to anemia therapy Pyrukynd (mitapivat) that were announced in a securities analyst’s Aug. 4 note to investors hasn’t changed the drug’s already established benefit-risk profile on the drug’s U.S. prescribing information.
A total of 33 states, plus the District of Columbia and Puerto Rico, agreed to participate in the U.S. CMS’ voluntary, outcomes-based program aimed at helping state Medicaid programs cover high-priced cell and gene therapies, starting with therapies for sickle cell disease.
It may be a year later than originally planned, but Regeneron Pharmaceuticals Inc. is set to commercialize Lynozyfic (linvoseltamab) in the U.S. following FDA approval for use in adults with relapsed and refractory multiple myeloma. A bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3, Lynozyfic was cleared under the accelerated approval pathway, with continued approval contingent upon a confirmatory trial.
Hemab ApS recently presented preclinical data on its human monoclonal antibody against von Willebrand factor (vWF), HMB-002, for the treatment of von Willebrand disease.
Novo Nordisk A/S recently presented data regarding their antibody fragment Inno-8 for the potential treatment of hemophilia A. Inno-8 was characterized in vitro alone or in combination with hemostatic agents used for treating hemophilia A.
Bispecific antibodies mimicking coagulation factor VIIIa (FVIIIa) have yielded successful results in the treatment of hemophilia A, but there remain some issues in regard to their production.
“The lack of therapeutic precision in treatment of myeloid malignancies is in sharp contrast with the fact that myeloid cancers represent the perhaps best characterized cancers of all at the cellular, molecular, and genetic levels,” Johanna Olweus told her audience at the Friday plenary session of the European Hematology Association 2025 Annual Congress.
“The lack of therapeutic precision in treatment of myeloid malignancies is in sharp contrast with the fact that myeloid cancers represent the perhaps best characterized cancers of all at the cellular, molecular, and genetic levels,” Johanna Olweus told her audience at the Friday plenary session of the European Hematology Association 2025 Annual Congress.
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