Wave Life Sciences Ltd. plans to file with the U.S. FDA for accelerated approval of WVE-N531, an exon skipping oligonucleotide for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping, a decision based on favorable data from the phase II Forward-53 study. The analysis was conducted after 48 weeks of treatment with 10 mg/kg of the drug dosed every two weeks. Forward-53 achieved all trial goals, turning up sustained exon skipping, muscle concentrations, and dystrophin restoration through 48 weeks and a 61-day tissue half-life that supports giving the DMD therapy once a month. Read More
Acrivon Therapeutics Inc. provided updated phase II data for checkpoint kinase candidate ACR-368, highlighting promising response rates for biomarker-positive patients with endometrial cancer and raising the possibility of an accelerated pathway in the second-line setting. Read More
The first disease modifying therapies for Alzheimer’s may have limited utility in some senses, but they will be a force for change, providing momentum and altering the way governments as payers, and health systems as carers, think about the disease. Read More
The U.S. Senate has approved the nominations of two key members of the Trump administration, Marty Makary as FDA commissioner and Jay Bhattacharya as NIH director. While these are two of the most critical appointments for the Trump administration, the Senate still has two other important appointments in queue, including the directors of the Centers for Medicare & Medicaid Services and the CDC. Read More
Transplanting an animal organ into a human is now a closer reality following the successful xenotransplantation of a genetically modified pig liver into a patient diagnosed with brain death in China. The operation was intended to evaluate organ function over a 10-day period. This is a complex experimental trial that did not involve removing the patient's liver and still requires further study. However, the positive preclinical results suggest this strategy could save the lives of those waiting for a human organ, at least in certain cases. Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Astrazeneca, Axsome, Cassava, CNS, Genelux, Kezar, Mural, Nanoscope, Newsoara, Oncotelic, Traws. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Alumis, Entero, Eterna, Ernexa, Immunogenx, Kaken, Relmada, Regor, SOLVE FSHD, Transcripta, Trigone, Twostep. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Genelux, Palatin, Recode, Sanofi, Scholar Rock. Read More
