With the U.S. FDA approval of Attruby (acoramidis) for transthyretin amyloidosis with cardiomyopathy (ATTR-CM), Bridgebio Pharma Inc. is taking on an industry giant. The next-generation, oral, small-molecule stabilizer of transthyretin will take on Pfizer Inc.’s Vyndamax (tafamidis, Vyndaqel), a TTR stabilizer approved in 2019 for ATTR-CM but expected to lose exclusivity in late 2028. The approval was based on a phase III study that showed Attruby significantly reduced death and cardiovascular-related hospitalizations. The NDA, which had a Nov. 29 PDUFA date, was approved Nov. 22. Read More
The Alzheimer’s disease (AD) space took another blow along with the shares of Cassava Sciences Inc. (NASDAQ:SAVA), which plummeted by 83.8%, or $22.19, to end Nov. 25 at $4.30 after the firm unveiled top-line data from the phase III Rethink-Alz study with simufilam. The study tested simufilam in mild to moderate AD, where results fell short of each of the prespecified co-primary, secondary and exploratory biomarker endpoints. Read More
When it comes to cell therapy, Alloplex Biotherapeutics Inc. CEO Frank Borriello said he believes that autologous, personalized therapy is the only thing that makes sense. “The allure of an off-the-shelf therapy has been such a magnet. It sucked in a lot of companies into that dream, and I'm sorry to say, it hasn't really worked out for them,” he told BioWorld. Instead, Borriello said he envisioned a cell training platform that doesn’t just tweak a single immune pathway but instead harnesses multiple immune pathways to turn the tables on cancer. Read More
It’s difficult to fathom that the health of half the world’s population is underserved. But it’s a hard truth. Women’s health has traditionally focused on what Seema Kumar, the CEO of Cure, coined as the “bikini zone.” However, reproduction and breast health are a drop in the bucket when it comes to women’s health. In this multipart, in-depth series, BioWorld provides insight into the oft-overlooked space of women’s health, exposing the disparities in research and funding while looking toward future opportunities for bridging the gender gap in health care. Read More
Since the isolation of the gene that causes Duchenne muscular dystrophy (DMD), scientists have progressed in understanding the mechanisms that lead to muscular diseases that can be evident from the early stages of childhood. This has led to the development of diagnostics and therapeutics, some approved by the FDA. Read More
Karen Carey, BioWorld’s managing editor and chief analyst, and Anette Breindl, managing editor of Bioworld Science, join this episode of the BioWorld Insider podcast to talk about the business and the science of women’s health. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Chromocell, Channel, Eyenovia, Evotec, Halozyme, Lexicon, Protagonist. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Akeso, GSK, Johnson, Leads, Merck, Replimune. Read More
