Phase II
The German biopharma company on Tuesday announced ambitious pipeline plans that include starting 10 new Phase II and III trials over the next 12 to 18 months.
In a Phase I/II study, Ultragenyx’s antisense oligonucleotide GTX-102 led to significant cognitive, behavioral, sleep and gross motor improvements in patients with the rare genetic disorder Angelman syndrome.
Investors were disappointed in data from a mid-stage study of Enlivex Therapeutics’ Allocetra cell therapy for the treatment of sepsis. The Israeli company is considering a follow-on trial in sepsis caused by urinary tract infection.
Data presented at the American Association for Cancer Research’s annual meeting sparked hope that we could be getting closer to treating pancreatic cancer, glioblastoma and other particularly difficult tumors.
GLP-1 treatments are all the rage in this space, but they aren’t the only approach in development. The pipeline assets highlighted here offer a differentiated approach, potentially increasing efficacy or reducing side effects.
On the heels of withdrawing Relyvrio from the U.S. and Canadian markets, Amylyx is now charting a path in Wolfram syndrome with promising interim Phase II data for its lead asset AMX0035.
Metabolic dysfunction-associated steatohepatitis experts welcomed Madrigal’s Rezdiffra as an “important first,” but there are more mid- to late-stage therapies showing promising results.
Merck and Kelun-Biotech’s antibody-drug conjugate achieved a 22% objective response rate and 80.5% disease control rate in heavily pretreated patients with gastric or gastroesophageal junction cancer.
Novo Nordisk and Ionis Pharmaceuticals unveiled promising respective data at the American College of Cardiology’s Annual Scientific Session, while Boehringer Ingelheim and Eli Lilly’s Jardiance missed the endpoint in a myocardial infarction study.
The biotech’s personalized neoantigen vaccine for colorectal cancer did not demonstrate a molecular response difference from the control arm. Gritstone blames trial protocol and plans for a Phase III study.
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