Blood and Clotting
Taiwan-based PharmaEssentia hopes to add essential thrombocytopenia to the label for its pegylated interferon product, already approved to treat polycythemia vera.
With successful Phase II data versus active comparators for both REGN7508 and REGN9933, Regeneron hopes to position the candidates to compete against existing factor X products.
Hematologists’ concerns about hepatoxicity with hemophilia A gene therapies may limit use, but Phase III data for Pfizer’s candidate at ASH show a lower rate of ALT increases than seen with BioMarin’s Roctavian.
Takeda will pay $200m up front for rights to Phase II elritercept for anemia in patients with MDS and myelofibrosis, allowing Keros to extend its cash runway to 2028.
The US FDA approved the drug for hemophilia A and B, and while it may struggle to compete in hemophilia A against Roche’s Hemlibra, it has a big convenience advantage in hemophilia B.
Once the deal with Sanofi closes, the rare disease drug will become one of Recordati’s fastest forecast sellers.
The withdrawal amid concerns about the risk-benefit ratio reduces treatment options for sickle cell disease, but it is not expected to have much effect on the SCD market, including the gene therapies.
US FDA approval of Niktimvo for third-line chronic GVHD, a competitor to Sanofi’s Rezurock, came less than two months ahead of anticipated approval of Syndax’s leukemia drug revumenib.
The company’s giroctocogene fitelparvovec, partnered with Sangamo, could compete with BioMarin’s hemophilia A gene therapy Roctavian, but longer-term data may be needed.
2seventy bio sold full rights to a hemophilia A program and in vivo gene editing technology to partner Novo Nordisk.
The Phase III ESLIM-01 study with HUTCHMED’s Syk inhibitor sovleplenib met its primary endpoint, demonstrating a 48.4% durable response rate for chronic immune thrombocytopenia, significantly higher than placebo. Global development appears to be accelerating and a multinational dose-finding Phase Ib study has also been initiated.
Having already posted Phase III success in non-transfusion dependent patients, Agios’s Pyrukynd now has shown the ability to reduce transfusions in alpha and beta thalassemia patients.
Cargo Therapeutics has got an early boost from its abstract to be presented the European meeting, while Shattuck has suffered on safety doubts for its CD47 inhibitor.
The company expects to have data from transfusion-dependent and non-transfusion dependent patients this year, with a potential expanded US approval in 2025 for Pyrukynd.
Mim8 looks good in hemophilia A but Hemlibra’s lead is all but unassailable.
The drug maker said the program would allow for continued reimbursement when patients change insurance plans. A Sangamo-partnered gene therapy for hemophilia A is also in the works.
The company announced positive topline results from a Phase II study of mezagitamab and expects to have five programs in Phase III development this fiscal year.
Gilead said the FDA requested a partial clinical hold on the studies as well, just over a week after the drug maker said it would not develop the magrolimab further for blood cancers.
The Phase III trial of BT524 produced positive topline results in patients with acquired fibrinogen deficiency, potentially making it the first fibrinogen concentrate to win US FDA approval for that indication.
The anti-C5 inhibitor sets an example of swift global trial and approval in China while fiercer competition awaits in the US, Europe and Japan.