Wave Life Sciences Ltd. made public favorable data from the phase II Forward-53 study with WVE-N531, an exon skipping oligonucleotide for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. 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. WVE-N531 also continued to prove safe and well-tolerated, Cambridge, Mass.-based Wave said. Shares (NASDAQ:WVE) were trading at $9.90, up 46 cents.

Acrivon focuses ACR-368 work on endometrial cancer

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. Investors, however, might have focused more on the company’s announcement that it was officially deprioritizing work with the compound in ovarian and bladder cancers. Shares of Acrivon (NASDAQ:ACRV) were trading down about 38% at midday.

Disease-modifying Alzheimer's drugs are ‘drivers of change’

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. As a comparator, think about how GLP-1 agonists have shifted the world view of obesity, suggested Lenny Shallcross, executive director of the World Dementia Council. “Treatments profoundly change the way people think about a disease. Sure [Alzheimer’s therapies] have got off to a modest start, but they are fundamental drivers of change,” he told the council’s annual meeting in London, March 25.

Senate votes in Makary, Bhattacharya at FDA and NIH

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 Centers for Disease Control and Prevention.

Ten days of normal survival of a pig liver in a human being

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. “This time… we just collected the liver from the gene-modified pig and then transplanted the liver to the brain-dead human being, in another place, not in the original place … because that original liver of the human being still existed,” co-senior author Lin Wang told BioWorld. Wang is a professor at Xijing Hospital, Fourth Military Medical University in China.

Also in the news

Alumis, Aprea, Astrazeneca, Axsome, Benitec, Cassava, Character Biosciences, CNS, Cytonics, Entero, Ernexa, Eterna, Fairjourney, Genelux, Immunogenx, Kaken, Kezar, Mural, Nanoscope, Neuronos, Newsoara, Oncotelic, Palatin, Recode, Relmada, Regor, Sanofi, Scholar Rock, SOLVE FSHD, Syncromune, Tolremo, Transcripta, Traws, Trigone, Twostep