Five years after Gilead Sciences Inc. gave up on momelotinib in the wake of two phase III failures in myelofibrosis, the JAK1/2 and ACVR1 inhibitor has found its way to the market in the hands of GSK plc. Branded Ojjaara, the drug gained U.S. FDA approval for use in intermediate- or high-risk myelofibrosis patients with anemia regardless of prior administration with JAK inhibitors such as Jakafi (ruxolitinib, Incyte Corp.). The approval came a day before the Sept. 16 PDUFA date, which had been extended by three months in June to allow the agency to review recently submitted data. A rare blood cancer characterized by dysregulated JAK-signal transducer and resulting in splenomegaly and progressive anemia, myelofibrosis affects roughly 25,000 people in the U.S.
Anthos’ phase II in afib hits primary endpoint and makes an early stop
Anthos Therapeutics Inc.’s phase II study of abelacimab in treating atrial fibrillation in patients at moderate to high risk of stroke has met its primary endpoint. The data monitoring committee stopped the study early as the fully human monoclonal antibody targeting factor XI/XIa reduced bleeding when compared to the standard of care direct oral anticoagulant, rivaroxaban. Abelacimab is also being evaluated in two phase III studies that are targeting the inhibition of factor XI and factor XIa. Privately held Anthos was formed in 2019 with a $250 million investment from Blackstone Life Sciences, which in-licensed the antithrombotic agent abelacimab that had been shelved by Novartis AG.
Biosimilar labeling distinctions a thing of the past?
If the U.S. FDA has its way, labeling would no longer distinguish between biosimilars and interchangeables. Instead, the agency is recommending that the labeling for both follow-ons include a “biosimilarity statement” and that the distinction between the two would reside only in the agency’s Purple Book. But that may not be the end of the matter. In releasing a new draft guidance today on biosimilar/interchangeable labeling, the FDA also is seeking comment on whether even a biosimilarity statement is needed now that the U.S. biosimilar market is on its feet, with 42 biosimilars approved, including four interchangeables that can automatically be substituted at the pharmacy.
Stargardt space percolates with varied players
Iveric Bio Inc.’s regulatory win Aug. 4 drew attention to the already hot eye-disease space, where intriguing new developments include the possibility of an oral therapy for Stargardt disease (STGD1). Belite Bio Inc. in late July finished enrollment of a phase III study with once-daily tinlarebant, a retinol binding protein 4 antagonist. Developers in STGD1 with oral small molecules include Kubota Vision Inc. (formerly Acucela Inc.) with emixustat, designed to bind to RPE-65. Also in the mix: Alkeus Inc., which raised $150 million by way of a series B round in June, and is advancing ALK-001 (gildeuretinol), a deuterated form of vitamin A.
Study identifies cause of death for Alzheimer’s neurons
By creating a new mouse model of Alzheimer’s disease that better recapitulated how the disease plays out in humans, investigators at KU Leuven have gained new insights into how amyloid plaques, tau tangles and neuronal death are related at the molecular level. The experiments, which were published in the Sept. 15, 2023, issue of Science, showed that amyloid-β caused the formation of tau tangles and that the long noncoding RNA MEG3 was upregulated during this process. This upregulation set off cell death in the form of necroptosis, a programmed form of inflammatory cell death. Blocking MEG3 expression in an inducible mouse model could prevent cell death.
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