Functional Concept
FDA Approves Revuforj, a First-in-Class Menin Inhibitor, for Relapsed or Refractory Acute Leukemia with KMT2A Translocation
FDA approval, Revuforj, Menin inhibitor, Acute leukemia, KMT2A translocation, Relapsed or refractory, Syndax Pharmaceuticals
Rona’s Sanofi-Sourced siRNA Demonstrates Early Efficacy in Dyslipidaemia Treatment
siRNA, dyslipidaemia, Rona, Sanofi, RNA interference, lipid disorders, therapeutic development
Boston Pharmaceuticals Achieves Phase 2 Success with Efimosfermin Alfa for MASH Treatment, Paving Way for Pivotal Trials
Efimosfermin alfa (BOS-580), Metabolic dysfunction-associated steatohepatitis (MASH), Non-alcoholic steatohepatitis (NASH), Phase 2 clinical trials, FGF21 analogue, Liver disease treatment, Pivotal trials
BeiGene Rebrands as BeOne Medicines, Emphasizing Global Unity Against Cancer
BeiGene, BeOne Medicines, Global Oncology, Cancer Treatment, Name Change, Strategic Growth
FDA Approves PTC Therapeutics’ Kebilidi, the First Gene Therapy Delivered Directly to the Brain for AADC Deficiency
PTC Therapeutics, Kebilidi, Gene Therapy, AADC Deficiency, FDA Approval, Brain-Delivered Therapy, Rare Genetic Disorder
GUBamy, a Weekly Amylin Agonist, Shows Promising Weight Loss and Tolerability in Phase 1 Trial
Amylin agonist, Obesity treatment, Weight loss, Phase 1 trial, GUBamy, Long-acting amylin agonist
FDA Denies Full Approval for Ocaliva in Primary Biliary Cholangitis, Accelerated Approval Stands
FDA, Ocaliva, Intercept Pharmaceuticals, primary biliary cholangitis, accelerated approval, rare liver disease
FDA Issues Second Clinical Hold on Kezar’s Zetomipzomib Program Within Two Months
Kezar Life Sciences, Zetomipzomib, FDA Clinical Hold, Lupus Nephritis, Autoimmune Hepatitis, Clinical Trials
Metsera Secures $215 Million Series B Funding Following Promising Phase 1 Weight Loss Data
Metsera, Series B funding, weight loss, obesity treatment, GLP-1 drugs, NuSH peptides
Gilead Exceeds COVID-19 Drug Sales Projections, Faces $1.75 Billion Loss on Trodelvy Trial Failure
Gilead, COVID-19, Veklury, Trodelvy, drug sales, clinical trial failure