With a $128 million series A financing, Diagonal Therapeutics Inc. launched to develop its lead program using agonist antibodies for treating, among other indications, the rare disease hereditary hemorrhagic telangiectasia. The antibodies are designed to activate a receptor complex in the TGF-β superfamily genetically impaired in patients with the bleeding disorder. Diagonal also is developing a treatment for the orphan disease pulmonary arterial hypertension.
Disc Medicine Inc. CEO John Quisel said that top-line phase II findings from the study called Aurora with bitopertin in erythropoietic protoporphyria are “hard for us to interpret. This package of data is something that we’re going to have to sort through,” and the Watertown, Mass.-based firm expects to talk with the U.S. FDA about next steps in the second half of this year.
Voydeya (danicopan), from Alexion, Astrazeneca Rare Disease, racked up its second global approval as the U.S. FDA greenlit it as an add-on therapy for treating extravascular hemolysis in adults with paroxysmal nocturnal hemoglobinuria (PNH), a crowded market with several already approved treatments and more in development.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of Pfizer Inc.’s Emblaveo (aztreonam-avibactam), an antibiotic combination that would offer a new option to patients with serious bacterial infections caused by multidrug-resistant gram-negative bacteria.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending approval of Pfizer Inc.’s Emblaveo (aztreonam-avibactam), an antibiotic combination that would offer a new option to patients with serious bacterial infections caused by multidrug-resistant gram-negative bacteria. If approved, Emblaveo would be among the first beta-lactam/beta-lactamase inhibitor combos cleared for use in Europe.
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.”
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