LONDON – Targed Biopharmaceuticals BV has raised €39 million (US$44.2 million) in a series A financing that will enable it to take its targeted clot busting drug Microlyse into clinical development. The first-in-class product consists of urokinase, a serine protease involved in the conversion of inactive plasminogen to active plasmin, linked to a nanobody targeted at von Willebrand factor, the blood glycoprotein that plays a key role in hemostasis.
The FDA’s approval of Agios Therapeutics Inc.’s Pyrukynd (mitapivat) for treating hemolytic anemia marks a turnaround from nearly a year ago. That’s when Agios sold its commercial, clinical and research-stage oncology portfolio to privately held Servier Pharmaceuticals LLC to concentrate on rare diseases.
Edigene Inc. has extended a long-running partnership with an academic lab at the Chinese Academy of Medical Sciences and Peking Union Medical College with the goal of co-developing hematopoietic stem cell regenerative therapies and technology for the treatment of inherited blood disorders, as well as the discovery of biomarkers to improve quality control of stem cell production.
Sierra Oncology Inc. plans an NDA in the second quarter of this year for momelotinib (MMB), thanks to data from the phase III Momentum study with the drug in myelofibrosis (MF) patients who are symptomatic and anemic after treatment with an approved JAK inhibitor. Momentum, which pitted momelotinib against the androgen danazol, met all of its primary and key secondary endpoints. MMB inhibits JAK1/2 and ACVR1/ALK2. Shares of San Mateo, Calif.-based Sierra (NASDAQ:SRRA) closed Jan. 25 at $22.68, up $7.17, or 46%.
Having unveiled more data from the ongoing, global phase III Gener8-1 study with Roctavian (valoctocogene roxaparvovec, also known as valrox), Biomarin Pharmaceutical Inc. remains on track to file a regulatory submission with the FDA in the second quarter of this year for the gene therapy to treat adults with severe hemophilia A. The EMA is already reviewing a marketing authorization application.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology annual meeting, including: Century, Forma.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology annual meeting, including: Beyondspring, Coimmune, Geron, JW, Novartis, Oncolytic, Oncternal, Oric, Oryzon, Ose, Pfizer, Poseida, Precigen, Rheos, Roche, Rocket, Sangamo, Sanofi, Seagen, Secura, Sierra, Syndax, Talaris, Tessa, TG, Viracta, Wugen, Xencor.
DUBLIN – Fans of “His Dark Materials” and the “Book of Dust,” Philip Pullman’s series of fantasy novels set in Oxford, U.K. and more remote corners of the earth, are familiar with alethiometers. In the hands of a highly select number of characters, these complex, compass-like devices can interrogate a mysterious substance called “dust” – an elementary particle associated with consciousness – and provide trusted answers to the most crucial questions.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Hematology annual meeting, including: 2seventy, Actinium, Agios, Allogene, Antegene, Apellis, Aptose, Ascentage, Atar, Bantam, Bioinvent, Daiichi, Harpoon, Iaso, Innocare, Innovent, Janssen, Karyopharm, Keros, Kymera, Lantern, Legend, Magenta, Mereo, Mustang, Nektar, Neximmune, Nextcure, Nkarta.
Two trials presented at the annual meeting of the American Society of Hematology (ASH) showed that treatment with the antithrombin inhibitor fitusiran reduced the bleeding rate in patients with severe hemophilia by roughly 90%, regardless of whether they had type A or B, and whether they had developed antibodies to recombinant clotting factors.