FDA awards orphan drug designation to VPO-227 to treat cholera

Oct. 24, 2022

VPO-227 is a small molecule with a novel mechanism of action, which blocks the cystic fibrosis transmembrane conductance regulator (CFTR) ion channel.

BioWorld Science Gastrointestinal Infection FDA Orphan drug

Today's news in brief

BioWorld

BioWorld briefs for Dec. 16, 2025.

Today's news in brief

BioWorld MedTech

BioWorld MedTech briefs for Dec. 16, 2025.

CTAD 2025: Diagnosing semaglutide’s failure in Alzheimer’s trials

BioWorld

A little over a week after announcing that the Evoke and Evoke+ studies failed to show that oral semaglutide could slow cognition decline in patients with...

Apollo’s APL-4098 shows potent antileukemic effects

BioWorld Science

Apollo Therapeutics Ltd. has developed APL-4098, a small-molecule general control nonderepressible 2 (GCN2) inhibitor for the potential treatment of AML.

Illustration of brain with electrical activity background

ABS-1230 controls seizures in KCNT1-driven severe epilepsy

BioWorld Science

Mutations in the KCNT1 gene produce gain-of-function effects that lead to overactivation of the potassium channel and consequent disruption of normal neuronal...