The U.S. FDA gave its go-ahead for Hemgenix (etranacogene dezaparvovec-drlb), Uniqure NV’s one-time gene therapy – the first for the treatment of adults 18 and older living with hemophilia B. Patients have been waiting “maybe beyond two decades” for a new therapy, Uniqure CEO Matthew Kapusta said. Hemgenix emerged from pioneering work by St. Jude Children’s Research Hospital and the University College London.
Merck & Co. Inc. is adding to its hematology assets in the takeover of Imago Biosciences Inc. for $36 per share, an arrangement with an equity value of about $1.35 billion. Shares of Imago (NASDAQ:IMGO) took flight as Wall Street learned of the deal, ending the day at $35.59, up $18.19, or 104%. Imago is working on new drugs for myeloproliferative neoplasms and other bone marrow diseases. Lead candidate bomedemstat (IMG-7289), an orally available inhibitor of the epigenetic enzyme lysine-specific demethylase 1 (LSD1), is undergoing phase II trials for the treatment of essential thrombocythemia, myelofibrosis and polycythemia vera, along with other indications.
Shanghai Meiyue Biotech Development Co. Ltd. has identified macrocyclic compounds acting as coagulation factor XIa (FXIa) inhibitors reported to be useful for the treatment of thrombosis.
Protagonist Therapeutics Inc. has disclosed hepcidin mimetic conjugates acting as solute carrier family 40 member 1 (SLC40A1, ferroportin) ligands with extended half-life and stability.
Galecto Inc.’s late-September news from its ongoing phase IIa trial with GB-2064 in myelofibrosis hiked shares of the Boston-based firm and piqued interest in the lysyl oxidase-like 2 (LOXL2) target, where such names as Gilead Sciences Inc. and Pharmaxis Ltd. also have been busy.
X4 Pharmaceuticals Inc.’s recent stock-boosting phase Ib news with lead candidate mavorixafor fueled already-strong interest in finding an alternative therapy for neutropenia and deepened esteem for the drug’s long-known mechanism of action.
After winning the backing of European regulators, Biomarin Pharmaceutical Inc. is returning to the U.S. FDA with its hemophilia A gene therapy, valoctocogene roxaparvovec, following a rejection in August 2020. It's armed with data it hopes will assuage concerns about long-term safety and benefits.
CRISPR-based cell therapies continued to gain steam Sept. 27 with the announcements of a potentially valuable big pharma collaboration and an ambitious global regulatory push.
Synthetic cells (SCs) armed with recombinant growth factors could contribute to tissue regeneration and healing by promoting angiogenesis. This technology opens the door to its application in other therapies such as transplants that require the remodeling or formation of new blood vessels. In addition, they mark the way to produce intracorporeal biological drugs or the inhibition of the angiogenesis process itself when it comes to blocking the irrigation of a tumor.
A prespecified interim analysis revealed that Astrazeneca plc’s oral factor D inhibitor danicopan met the primary endpoint of a phase III trial as an add-on therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH) who were also taking a C5 inhibitor, but who still experienced extravascular hemolysis.
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