News
BioMarin’s investigational therapy failed to elicit clinical improvements in patients with ENPP1 deficiency, while also missing key secondary endpoints of rickets severity and growth.
FEATURED STORIES
Nusano will bring a massive new radioisotope facility in Salt Lake City online by the end of the year, establishing a supply of starting materials for the next generation of radiopharmaceuticals.
Last month, Revolution Medicines’ RAS inhibitor doubled survival in a Phase 3 pancreatic cancer trial. On the biotech’s heels are Immuneering, Actuate Therapeutics, Erasca and more, looking to improve on that result with increased tolerability—and more time for patients.
The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, BioSpace reviews genetic medicines in development for the central nervous system, retinal, cardiac and neuromuscular diseases.
FROM OUR EDITORS
Read our takes on the biggest stories happening in the industry.
The Department of Health and Human Services is spinning its wheels, unable to establish steady leadership at three major divisions—the CDC and the FDA’s two primary review units.
THE LATEST
The agreement, which BMO Capital Markets called a “mild positive” for Structure, appears to address Roche’s concerns about the composition of investigational weight loss drug CT-996.
OncoNano’s platform, called ON-BOARD, packages drugs in pH-sensitive micelles that ensure their specific delivery near tumors, while also preventing systemic exposure.
Next-generation automation is closing the gap between curative science and real-world demand, enabling faster development, global consistency and broader patient access to CAR T therapies.
After a strong open to the year, the public markets suffered a six-month drought that led to biotech’s tightest IPO window in years.
Only a handful of the top pharmas have signed Most Favored Nation drug pricing deals with the White House, while smaller biotechs continue to hang in limbo.
In a year that saw advisory committees placed under a particularly bright microscope at the FDA, the agency held fewer meetings than usual and agreed with its advisors only 57% of the time, Jefferies reported.
Industry leaders are focused on the resilience of key starting material supply and the knock-on effects of automation in the new year.
The fierce rivalry between Novo Nordisk and Eli Lilly is alive and well, as the two companies are expected to face off with their new obesity pills this year.
After getting the crucial first-mover advantage with an FDA approval for a weight loss pill, Novo Nordisk looks to win the market before rival Lilly can arrive with its own oral option for obesity.
J&J paid Numab Therapeutics $1.25 billion upfront for the asset in 2024 based on the belief that its dual mechanism of action could improve on existing therapies.