Top-line results from BrainStorm’s NurOwn Phase III ALS study show the cell therapy missed the primary efficacy endpoint, but clinically meaningful responses were seen in a pre-specified subgroup which investigators considered potentially a significant step forward in therapy.
BrainStorm Cell Therapeutics Inc.’s adult stem cell therapy, NurOwn, has missed the primary efficacy endpoint in top-line results from a Phase III study in amyotrophic lateral sclerosis (ALS), disappointing investors, although clinical investigators involved in the study were encouraged by the results.
The study included more severely affected patients than other ALS studies, and the US Food and Drug Administration will review...
The updated US FDA label modifies dose titration based on TRAILBLAZER-ALZ 6, a study that showed a more gradual rise in dosing levels reduced ARIA-E incidence without reducing amyloid clearance.
The company is expecting US FDA approval of the drug in a bigger indication, major depressive disorder, later this year.
Still holding out hope after multiple rejections for the tyrosine kinase inhibitor.
Psychedelics appear to be in vogue after years of being relegated to alternative medicine. But investor pushback after the highly anticipated release of Phase III trial data by Compass Pathways reveals an increasingly high bar for development.
While companies such as Eli Lilly and Roche have faced setbacks to their fibroblast activation protein-targeting oncology therapies, Actithera believes its radiopharmaceuticals platform will prove more successful.
The company is expecting US FDA approval of the drug in a bigger indication, major depressive disorder, later this year.
Relaxing CAR-T restrictions may not be an access panacea because of patient fragility and lack of payer incentive. Conversely, the ACIP’s recent recommendation on Thiomersal-containing flu vaccines is probably the thin edge of a more restrictive wedge.
