Topic: clinical trial data
Acasti still thinks its drug has a chance, pointing to the reduction in triglyceride levels in the placebo group to explain the failure.
Ultragenyx Pharmaceutical saw its shares jump around 27% in trading Friday after announcing positive top-line data out of its gene therapy trial.
Regeneron’s rare disease drug reduced 90% of new bone formation in adults with a disorder that causes the body’s soft tissues to turn into bone.
The result suggests Apellis can challenge Alexion’s dominance in paroxysmal nocturnal hemoglobinuria.
The setback wiped 74% off Novan’s value and left its cash runway looking precariously short, but the biotech plans to start another phase 3.
The failure of the JAK1 inhibitor, which wiped $2 billion off Incyte’s market cap, is another blow to efforts to expand beyond Jakafi.
The results wiped 50% off Wave’s share price, adding to losses it suffered when it dumped a Duchenne muscular dystrophy program earlier in December.
BioMarin’s vosoritide hit the mark in phase 3, helping children with achondroplasia, the most common form of dwarfism, grow more quickly than placebo.
The drug, Compass Pathways’ COMP360, came through the placebo-controlled phase 1 without setting off any safety alarms.
The data position Leo to file for approval of the Dupixent rival in atopic dermatitis next year.