Epifrontier Therapeutics Inc. has been awarded a grant of up to $32 million in nondilutive funding from the Japan Agency for Medical Research and Development (AMED) to advance the clinical development of EPF-001 (RK-701), a first-in-class G9a inhibitor being developed for sickle cell disease and β-thalassemia.
What one analyst called an “intriguing” overall survival signal in phase III results has Karyopharm Therapeutics Inc. planning to meet with the U.S. FDA about a would-be sNDA filing for Xpovio (selinexor) in myelofibrosis (MF).
On the heels of China’s approval of Sino Biopharmaceutical Ltd.’s rovadicitinib, Sanofi SA is now inlicensing the first-in-class dual JAK/ROCK inhibitor in a deal worth more than $1.4 billion.
On the heels of China’s approval of Sino Biopharmaceutical Ltd.’s rovadicitinib, Sanofi SA is now inlicensing the first-in-class dual JAK/ROCK inhibitor in a deal worth more than $1.4 billion.
China’s National Medical Products Administration has approved Sino Biopharmaceutical Ltd.’s rovadicitinib, branded as Anxu, for first-line treatment of adults with intermediate- or high-risk primary myelofibrosis, as well as post polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis.
On the average, humans – and pigs, and deer, and birds – who live at high altitudes have better blood glucose control than their counterparts near sea level. In work published in the Feb. 19, 2026, issue of Cell Metabolism, investigators have linked this phenomenon to red blood cells that directly take up and metabolize glucose from the blood under low oxygen conditions.
Agios Pharmaceuticals Inc. is preparing to present a mixed bag of phase III Rise Up data to the U.S. FDA in hopes of “full approval” for mitapivat in sickle cell disease (SCD), which would make it its third indication in rare hematology.
The pressure to replace animal testing with human-relevant assays that are more predictive of human-drug responses has now reached a tipping point, and there is a movement toward greater acceptance of these potentially more translatable tests.
Sunshine Lake Pharma Co. Ltd. has divulged heterocyclic fused ring compounds acting as pyruvate kinase PKLR activators potentially useful for the treatment of anemia, abetalipoproteinemia, hemolysis, hereditary spherocytosis, sickle cell disease and thalassemia.
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-cells-in-blood-flow.webp?height=488&t=1772563584&width=650 "Primary myelofibrosis (PMF) cells in blood flow")
