“Our goal is that by the end of this call, you’ll share our conviction in the totality of the data.” So said Agios Pharmaceuticals Inc. CEO Brian Goff as the company reported top-line data from its 52-week Rise Up trial testing mitapivat in sickle cell disease showing the oral pyruvate kinase activator hit statistical significance on one of two primary endpoints and two of three key secondary endpoints. Read More
In a verbal sparring over who can deliver the lowest drug prices in the U.S., several Senate Democrats are urging President Donald Trump to immediately release the list of second-round Medicare-negotiated drug prices, instead of doing what they characterize as “ambiguous” and “opaque” pricing deals with individual biopharma companies. Read More
Success in Japan may further bolster Protara Therapeutics Inc. in its push with TARA-002 in pediatric patients with macrocystic and mixed cystic lymphatic malformations (LMs), for which the New York-based firm disclosed interim results from an ongoing phase II trial. Read More
The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of gut bacteria. Read More
At the 2025 annual meeting of the Society for Neuroscience in San Diego this week, Catherine Woolley’s plenary lecture was an unusual combination of debunking and affirming the importance of sex differences in the brain. Read More
Tempest Therapeutics Inc. entered definitive agreements approved by its board to acquire certain dual-targeting CAR T programs from Factor Bioscience Inc. and its affiliates in an all-stock transaction expected to close in early 2026. Read More
Immunotherapy-focused biotech company LTZ Therapetics Inc. and GSK plc are partnering to develop up to four potential first-in-class myeloid cell engagers using LTZ’s immune-engager platform to target blood cancers and solid tumors. Read More
Editor Annette Boyle discusses the challenges med-tech companies face in bringing innovation to market and how the industry can overcome them. Read More
Regulatory snapshots, including global drug submissions and approvals, and other regulatory decisions and designations: Bionet, Celcuity, Celltrion, Dewpoint, Grifols, Vasa. Read More
