Drug Development
Ionis and Ultragenyx are competing to develop oligonucleotide treatments for Angelman syndrome, but will Neuren’s peptide catch up?
FEATURED STORIES
In the wake of Pfizer’s voluntary market withdrawal of the popular sickle cell disease therapy, BioSpace looks at five investigational drugs currently making their way through the pipeline.
Artificial intelligence won’t replace people in biopharma, but it is infiltrating every step of drug development, including in some ways that aren’t so obvious.
Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market. However, recent years have seen a number of high-profile market withdrawals and failed confirmatory trials.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
Both vibostolimab and favezelimab have had disappointing runs leading up to their termination, sustaining several late-stage failures.
The FDA has put a stop to U.S. initiation of PepGen’s Phase II trial for its Duchene muscular dystrophy treatment. The company faced the same hurdle for an earlier neuromuscular candidiate in 2023.
Even as Biogen and Eisai’s Leqembi and Eli Lilly’s Kisunla slowly roll out onto the market, experts question the efficacy of these anti-amyloid antibodies and the amyloid hypothesis overall.
Novartis, Biogen, Takeda and Novo Nordisk are all betting on advances in the molecular glue degraders space, collectively investing billions in hopes of treating cancer, Alzheimer’s disease, cardiometabolic disease and more.
Some 90% of investigational drugs fail—and success rates are even more dire in the neuro space. Here, BioSpace looks at five clinical trial flops that stole headlines over the past 12 months.
Following news of RSV lower respiratory tract infections in infants immunized with Moderna’s investigational RNA vaccines, FDA advisors said the trial investigators should continue the study, while keeping an eye out for further safety signals.
Incyte is abandoning its ALK2 blocker zilurgisertib, which it was trialing for myelofibrosis-associated anemia, while iTeos will deprioritize the development of inupadenant after it failed to meet the biotech’s clinical bar in a Phase II study of metastatic non-small cell lung cancer.
Ocaliva recently failed to secure the FDA’s traditional approval for primary biliary cholangitis due to safety concerns.
The EMA approved a kidney disease–related label expansion for the blockbuster GLP-1 drug after a study showed reduced risk of death by 20%.
Pfizer, facing increasing pressure from Novartis, is touting a Phase III win for Ibrance as the first clinical evidence supporting the CDK4/6 inhibitor class’ use in patients with a specific type of breast cancer.