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.
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.
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.
Spectrum Pharmaceuticals Inc. celebrated a long-awaited win with the U.S. FDA’s approval late Sept. 9 of novel G-CSF drug eflapegrastim, cleared for use in chemotherapy-induced neutropenia nearly four years after the company first filed for regulatory approval. Despite moves this year to reduce its cash burn, Spectrum has ready to go a commercial team expected to sell eflapegrastim as well as cancer drug poziotinib, which is under FDA review with a PDUFA date of Nov. 24, 2022.
Shares in Forma Therapeutics Holdings Inc. gained 51% as Novo Nordisk A/S made a $20-per-share offer that values the firm’s equity at $1.1 billion. The stock (NASDA:FMTX) had closed at $13.40 prior to the disclosure of the bid on Sept. 1. It closed the day at $20.24, up $6.84, suggesting at least some optimistic investors believe the final price could go higher.
Access to advanced therapies proved to be a major talking point at a conference in London, following the U.S. approval of Bluebird Bio Inc.’s Zynteglo (betibeglogene autotemcel) cell-based gene therapy for beta thalassemia and its $2.8 million price tag. Regulators in Europe backed Zynteglo in 2019 but Bluebird opted to withdraw the therapy in 2021 after deciding that the complex thicket of pricing bodies in Europe was too difficult to negotiate.
RSS
