Agios Pharmaceuticals Inc. is preparing to present a mixed bag of phase III Rise Up data to the U.S. FDA in hopes of “full approval” for mitapivat in sickle cell disease (SCD), which would make it its third indication in rare hematology.
A world-first pediatric mRNA cancer vaccine trial is launching in Australia that could open new possibilities for children with aggressive brain tumors. The Paedneo-Vax trial, funded by Canada’s Providence Therapeutics Holdings Inc., in combination with the Australian Government and philanthropic donors, is the first multisite pediatric study to evaluate individualized mRNA vaccines designed specifically for each child's cancer.
Venture capital firms and research institutions have launched the European Life Science Coalition in a bid to strengthen Europe’s VC ecosystem by mobilizing more private and public investment.
Calla Lily Clinical Care Ltd.’s strategic collaboration with Merck KGaA to support the development of Callavid, its intravaginal drug delivery platform, is a “huge validation” of the work the company is doing, Lara Zibners, co-founder and chairman of Calla Lily, told BioWorld.
After two decades of work with Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy, PTC Therapeutics Inc. withdrew its bid for approval of the compound upon hearing from the U.S. FDA. Regulators told Warren, N.J.-based PTC that the data package for Translarna is “unlikely to meet the agency's threshold of substantial evidence of effectiveness.”
Biopharma dealmaking continued its momentum into 2026, with January logging $31.16 billion in total value. That figure outpaced January 2025’s $28.63 billion and exceeded the 2025 monthly average of $24.38 billion. In addition, January 2026 ranks as the highest first month in the past eight years.
A circuit formed by tumor, immune and nervous systems triggers cancer cachexia and anorexia, the excessive loss of weight, muscle and fat experienced in some cancer types. A new study is the first showing these three actors of a triangle interaction that initiates and feeds the process.
Undeterred by all the unsuccessful biopharma suits challenging the U.S. Medicare price negotiations, Abbvie Inc. filed its own complaint Feb. 11 in the U.S. District Court for the District of Columbia, seeking to undo CMS’ selection of Botox in the third round of the negotiations.
RSS
