LONDON – The EMA recommended approval of Apellis Pharmaceuticals Inc.’s C3 complement inhibitor, Aspaveli (pegcetacoplan), to treat paroxysmal nocturnal hemoglobinuria, but has diverged from the FDA, excluding treatment-naïve patients adding its use should be restricted to those who have failed to respond to C5 inhibitor drugs.
In the year’s second biggest M&A deal, Merck & Co. Inc. will take over pulmonary and hematologic specialist Acceleron Pharma Inc. for $11.5 billion. The acquisition brings Merck a pair of potential blockbuster drugs, one of them already marketed. There is sotatercept, in development for treating pulmonary hypertension (PH), and also Reblozyl (luspatercept-aamt), the first and only erythroid maturation agent approved in the U.S., Europe and Canada for treating anemia in certain blood disorders.
Shares of Protagonist Therapeutics Inc. (NASDAQ:PTGX) fell 62% to $17.53 on Sept. 17 after it reported finding malignant skin tumors in a mouse model test of its most advanced candidate, rusfertide for blood disorders. After the company notified the FDA, the regulator put the program on a clinical hold, leading dosing of patients in all ongoing trials of rusfertide to be halted for now. The development could impact Protagonist's ability to start phase III testing of the candidate in polycythemia vera (PV) early in 2022, as well as efforts to expand its development to a third indication beyond PV and hereditary hemochromatosis (HH) by the end of this year, as it has planned.
Disc Medicine Inc. closed a $90 million series B round to move its two lead assets into clinical trials in patients next year. Bitopertin, an oral inhibitor of glycine transporter 1 (GlyT1), is entering a phase II trial in patients with erythropoietic porphyrias (EPPs), a set of rare genetic disorders caused by mutations that disrupt heme synthesis. Disc-0974, an antibody directed against hemojuvelin, will enter a phase II trial in myelofibrosis patients with transfusion-dependent anemia. The molecule, which Disc Medicine in-licensed from North Chicago-based Abbvie Inc., is currently undergoing a phase I trial in healthy volunteers.
Facing inevitable challenges ahead beyond peak performance for its wildly successful cystic fibrosis franchise, Vertex Pharmaceuticals Inc. is continuing to look to the future, announcing Aug. 24 it licensed rights to CRISPR gene-editing technology from Arbor Biotechnologies Inc. Co-founded by Feng Zhang and David Walt, Arbor's tech could figure into new Vertex cell therapies for diabetes, hemoglobinopathies and other diseases.
Inmagene Biopharmaceuticals Co. Ltd. has licensed rights from Kissei Pharmaceutical Co. Ltd. to develop and commercialize the spleen tyrosine kinase (Syk) inhibitor fostamatinib for all potential indications in China.
China’s NMPA has given Belief Biomed Inc. the official go-ahead to start testing its investigational gene therapy, BBM-H901, for the potential treatment of hemophilia B in the country, marking the first time an I.V. gene therapy for a rare disease has been approved in China. The company plans to advance the phase I/II trial for the candidate shortly, it said.
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