Blood and Clotting
The Phase III portion of the Phase II/III RISE UP study in sickle cell disease met the hemoglobin response primary endpoint, but not the sickle cell pain crisis reduction co-primary endpoint nor a secondary endpoint on reducing fatigue.
The company said in its third quarter earnings that it would focus on areas that better foster growth for the company as the hemophilia A treatment only brought in $3m in Q3.
Deal Snapshot: CSL secures exclusive option to acquire VarmX, depending on results for a Phase III trial for its lead compound, VMX-C001, a reversal agent for Factor Xa inhibitors.
The drugmaker reported a negative Phase III study for inclacumab, after a partial clinical hold for osivelotor and pulling Oxbryta from the market.
Analysts see the new strategy as a way to address pressure from the Trump administration’s most favored nation drug pricing proposals.
CMS said that 33 US states plus the District of Columbia and Puerto Rico had joined a program whereby the agency negotiates outcomes-based contracts on their behalf.
Roctavian shows durable bleed control and safety over five years, but high costs and access hurdles limit its commercial outlook.
The drugmaker announced positive topline results for the drug in hemophilia A and B patients with inhibitors, on top of the existing approval for patients without inhibitors.
First results for a first-in-class mutCALR-targeted therapy in essential thrombocythemia presented at EHA point to a lucrative future for the early-stage product.
Takeda/Protagonist are awaiting 52-week data to confirm the results from the VERIFY trial of the drug in polycythemia vera.
The Belgium-based firm will take the first-in-class drug, based on tick saliva, targets inflammation without increasing bleeding, into a Phase IIb trial is in preparation, supported by a new series B funding round.
CFO François Roger tells Scrip the French drugmaker is flexible geographically on making investments but its spend in the US has risen regardless of the threat of tariffs.
The small interference RNA therapeutic can be used by patients regardless of inhibitor status.
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.