Today, a brief rundown of news from Bristol Myers Squibb, Novartis and Agios Pharmaceuticals, plus Food and Drug Administration officials who are proposing a new framework for testing CAR-T cell therapy for cancer.
Vinay Prasad and two other officials within the Food and Drug Administration office regulating many genetic medicines have outlined a stricter approval framework for the next CAR-T cell therapies developed for cancer. In an article published Monday in the Journal of the American Medical Association, the trio wrote that newer CAR-T treatments need to extend survival, or the time before a type of event occurs, in randomized, controlled trials. The control groups in those studies must also take into account the existing standard treatments, including other approved CAR-T therapies, and prove superior unless “adequately justified and discussed” with the FDA. The new protocol represents a higher approval bar for CAR-T therapies, which, historically, have been cleared based on their ability to induce responses in single-arm studies. — Ben Fidler
A combination of chemotherapy and Bristol Myers Squibb and BioNTech’s dual-acting cancer immunotherapy showed signs of efficacy in women with an aggressive breast cancer, regardless of whether their tumors expressed a key protein making them likely to respond to treatment. Phase 2 study results presented at a medical meeting Tuesday show that their therapy, pumitamig, drove responses in a confirmed 61.5% of treated participants in first- or second-line triple-negative breast cancer, and either shrank tumors or stopped them from growing in just over 92% of them. The partners, which allied on pumitamig this year in a multibillion-dollar deal, recently began a global Phase 3 study in triple-negative tumors. A different late-stage study in China should produce results in 2026. — Ben Fidler
Novartis is wagering more than $1.7 billion on a London-based startup’s drug discovery technology to find new medicines for allergy-associated conditions. Per terms of a heavily backloaded deal announced Tuesday, Relation Therapeutics will receive an upfront payment and additional research funding, plus an equity investment, for a total of $55 million. In exchange, Novartis gets access to Relation’s AI-powered platform, which the companies will use to “identify, validate, and advance potential first-in-class targets in atopic diseases driven by immune dysregulation.” In addition to this collaboration, Relation announced it has raised another $26 million from a group of investors that includes the venture capital arm of chipmaker Nvidia. — Jacob Bell
Eli Lilly said Tuesday it will spend $6 billion to build a new factory in Huntsville, Alabama, the third of four plants it pledged to construct when it committed $50 billion to reshoring its supply chain earlier this year. The facility will make active pharmaceutical ingredients for small molecule and peptide drugs when completed in 2032, including orforglipron, the obesity pill Lilly hopes will gain FDA approval in 2026. Construction will generate 3,000 jobs and when complete, 450 people will be employed at the facility. The site was selected from 300 applications, and the decision was made based on local workforce training, as well as transportation, infrastructure, zoning and incentive considerations. — Jonathan Gardner
The FDA missed a proposed deadline for granting an expanded approval to an anemia tablet from Agios Pharmaceuticals. Known scientifically as mitapivat, the drug is already sold under the brand name Pyrukynd for adults with “pyruvate kinase deficiency.” Agios has been trying to broaden its use into a spectrum of genetic blood disorders that cause anemia, with an approval verdict originally expected Dec. 7. The company said it’s “collaborating closely” with the FDA to finalize labeling and risk-mitigation materials, but, as of Monday, the agency had not yet provided a timeline for a regulatory decision. According to Marc Frahm, an analyst at TD Cowen, Agios management noted that the holdup is not due to any meaningful disagreements between the two parties, but rather “dotting Is and crossing Ts” on the finer details of a risk-mitigation program. And to Leerink Partners analyst Andrew Berens, the lack of an FDA rejection implies that “an approval is likely imminent.” — Jacob Bell
