Novo Nordisk A/S announced positive clinical data for its two hemophilia therapies and said each is shaping up to fill unmet needs in what is an increasingly competitive market.
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.
Catalyst Biosciences Inc. decided to stop developing marzeptacog alfa (MarzAA), a recombinant human coagulation factor VIIa variant for treating factor VII deficiency, and sell its hemophilia assets to focus on its complement therapeutics and protease medicines platform.
PARIS – Defymed SAS reported the formation of the Decapes consortium, which brings together the Institute of Chemistry and Processes for Energy, Environment and Health (ICPEES) and the specialist microtechnology and biomaterials service provider for med-tech companies, Statice. This French consortium is working on a minimally invasive version of Defymed's Mailpan device, which will extend the application range for this artificial pancreas to include other chronic pathologies besides type 1 diabetes.
LONDON – Hemab ApS has closed a $55 million series A to take forward treatments for bleeding and thrombosis disorders based on antibodies in-licensed from Novo Nordisk A/S, where the newco’s founder, Johan Henrik Faber, previously led hemophilia drug research.
Amsterdam-based Uniqure NV’s regulatory hitch with its highly regarded hemophilia B program had analysts questioning during a conference call what the development might mean for the company’s deal with CSL Ltd.
LONDON – After announcing its $17.4 million series A funding in the midst of pandemic in April, Genespire Srl has now gone public on the preclinical research upon which its programs will be based, presenting the data at last week’s American Society of Gene and Cell Therapy (ASGCT) annual meeting.
Aptevo Therapeutics Inc.’s chief scientific officer, Jane Gross, told BioWorld that the sale of the firm’s marketed recombinant hemophilia B therapeutic, Ixinity, to Medexus Pharmaceuticals Inc. allowed for a “cleaner message” to Wall Street. “It was a little difficult to explain having a commercial asset and an R&D pipeline,” she said.
LONDON – Dutch startup Enzyre BV is teaming up with Takeda Pharmaceutical Co. Ltd. to complete development of a home diagnostic device that aims to make it as easy for hemophiliacs to self-test their coagulation status as it is for diabetics to monitor their blood sugar levels.
Bluebird Bio Inc. and Novo Nordisk A/S plan to develop in vivo genome editing treatments for genetic diseases, including hemophilia, over the next three years.
