“This was worse than our national election,” Eric Peterson said as he explained his vote Oct. 10 concluding that Stealth Biotherapeutics Inc.’s elamipretide is effective in treating Barth syndrome, an ultra-rare mitochondrial disease that currently affects 129 males in the U.S. Peterson, a vice provost, senior associate dean and professor at the University of Texas Southwestern Medical Center, was one of 10 members of the U.S. FDA’s Cardiovascular and Renal Drugs Advisory Committee (CRDAC) who came to that conclusion. Six others had a different opinion. Regardless of which way they voted, the panelists attested to how difficult the decision was. Read More
Ligachem Biosciences Inc., of Daejeon, South Korea, and Osaka, Japan-based Ono Pharmaceutical Co. Ltd. agreed to two antibody-drug conjugate (ADC) deals that could reach $700 million (₩943.5 billion) combined. Read More
Booster Therapeutics is ready to open up a new arm of the proteasome after raising $15 million in seed funding to advance small molecules it says can degrade multiple types of harmful proteins. Rather than tagging single disease proteins with a ubiquitin marker for degrading via 26S proteasomes, these compounds directly activate 20S proteasomes that naturally recognize disordered proteins without the need for ubiquitin tagging. Read More
Innocare Pharma Ltd.’s tyrosine kinase 2 (TYK2) inhibitor, ICP-488, met the primary endpoint in a phase II trial in Chinese patients with moderate to severe plaque psoriasis, and the results clear the way to accelerate clinical development in psoriasis and other autoimmune diseases. Read More
Virus is associated with sickness, but oncolytic virus therapies, which harness viruses to attack and kill cancer cells, may soon change the standard of treatment for cancer, including those long deemed uncurable like malignant glioma. Read More
Gene and cell therapies will drive innovation for the next 10 years, Claus Zieler, the chief commercial officer at Astellas Pharma Inc., said in this episode of the BioWorld Insider podcast. Developers are on the cusp of breakthroughs because a gene can now be replaced “and that means we can potentially cure a disease rather than intervening in a disease.” Read More
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Aurigene, Clearside, Dyne, Entrada, Innocare, Insmed, Panavance, Tevogen, Vanqua, Viatris, Viking. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Alkem, Astellas, Bio-Thera, Boehringer, Ciconia, Circle Pharma, Eli Lilly, Gedeon Richter, Gemma, Insitro, Johnson & Johnson, Nona, Overt Bio, Qinotto, Sonnet, Sumitomo, Takeda, Xortx. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Alnylam, Bayer, Biogen, Can-Fite, Capricor, Denovo, Eisai, Leads, Neurosense, Neuvivo, Novavax, Ocugen, Soleno, Teva, Zealand. Read More
