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. The company’s stock (NASDAQ:BBIO) rose dramatically with the approval as shares were up 20% at midday to $28.07 each.

Cassava hit hard by phase III miss in Alzheimer’s

Shares of Cassava Sciences Inc. (NASDAQ:SAVA) plummeted by 85%, or $22.54, to trade at $3.94 after the firm said top-line data from the phase III Rethink-Alz study testing simufilam in mild to moderate Alzheimer’s disease (AD) fell short of each of the prespecified co-primary, secondary and exploratory biomarker endpoints. The co-primary goals were the change in cognition and function from baseline to the end of the double-blind treatment period at week 52, assessed by the ADAS-Cog12 and ADCS-ADL scales comparing the drug to placebo. Simufilam, meant to restore the shape and function of altered filamin A, continued to show an overall favorable safety profile, Cassava said. The second phase III AD trial with the compound, Refocus-Alz, has been discontinued. Cassava will report data from both experiments later.

Newco news: Alloplex’s Suplexa reawakens immune cells to fight cancer

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 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.

In muscular dystrophies, gene therapies race the clock

Since the isolation of the gene that causes Duchenne muscular dystrophy, 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.

Healing the health divide: BioWorld reports on women’s health space

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.

BioWorld Insider podcast: Healing the health divide for women

As a new BioWorld investigative report shows, decades of research excluding women from clinical trials and investment decisions made in male-dominated boardrooms have led to half the world’s health needs being underserved. Of the drug development companies working on women's health solutions, the proportion of funding and partnering for the sector is quite small. But it’s slowly growing, as is the depth of science. Karen Carey, BioWorld’s managing editor and chief analyst, and the managing editor of Bioworld Science, Anette Breindl, join this episode of the BioWorld Insider podcast to talk about the business and the science of women’s health.

Also in the news

Akeso, Aptose, Ascentage, Autoimmunity, Biofrontera, Bioxcel, Channel, Chromocell, Cytodyn, Enveda, Evotec, Eyenovia, GSK, Halozyme, Immune-onc, Johnson, Leads, Lexicon, Merck, Oncoverity, Preveceutical, Protagonist, Replimune, Viripax