Phase III
More than a month after failing its Friedrich’s ataxia trial, PTC Therapeutics’ vatiquinone has again missed its primary endpoint, this time in a mitochondrial disease-associated seizures study.
The company Thursday reported positive topline results for a Phase III trial of its Alzheimer’s-related agitation treatment, while also disclosing improprieties by a principal investigator.
The funds will support a trial of a tuberculosis vaccine developed by GSK that could potentially be the first new TB shot in a century.
The biopharma’s monoclonal antibody is the first to win approval for the two most common forms of generalized myasthenia gravis, a rare autoimmune and neuromuscular disorder.
FibroGen takes another hit as its antibody hopeful fails in a second indication. The biopharma says that it is cutting costs to extend its cash runway.
Regeneron envisions new dosing for Eylea’s label and it’s the moment of truth for BioMarin’s gene therapy for hemophilia A. For that and more, see inside.
The majority of ALS patients are excluded from clinical trials. Experts say using biomarkers and stratifying trial populations can expand eligibility and provide additional scientific insights.
The company reported results Friday from the KEYNOTE-811 trial in which the anti-PD-1 therapy took an important step toward FDA approval for rare esophageal tumors.
The data for Reproxalap, Aldeyra Therapeutics’ drug for treating allergic conjunctivitis and dry-eye disease, could potentially pave the way for an alternative to over-the-counter eye drops.
In the latest HEALEY trial readout, Clene’s CNM-Au8 significantly reduced plasma neurofilament light chain levels in ALS patients, which could be a strong and reliable signal of its clinical benefit.
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