Phase III
The company’s immunotherapy, in combination with chemotherapy Avastin, showed favorable progression-free survival in a late-stage hepatocellular carcinoma study.
As Vertex and CRISPR Therapeutics await the monumental potential approval of exa-cel, bluebird bio, Iovance and Rocket Pharmaceuticals wait patiently in the wings.
A failed rare disease clinical trial brings social media expertise to the pharma industry and patient recruitment: A discussion with Bryan Manning, founder of Clinical Enrollment and Two Blind Brothers
Although Kodiak Sciences initially scrapped its development of tarcocimab tedromer after late-stage failures, new data has convinced the company to give the eye drug another shot.
Following two late-stage failures, Travere Therapeutics has unveiled the results of two Phase III studies, attempting to regain Filspari’s footing in IgA nephropathy and focal segmental glomerulosclerosis.
In a late-stage study, most Huntington’s disease patients reported that their symptoms of chorea were “much improved” or “very much improved” following treatment with Neurocrine Biosciences’ VMAT2 inhibitor Ingrezza.
Following the recent Chinook Therapeutics acquisition, the Novartis experimental IgA nephropathy therapeutic atrasentan significantly reduced protein in the urine of patients compared to placebo.
Despite failing to hit the primary endpoint in the Phase III EMBARK study, the company plans to file for a label expansion for its Duchenne muscular dystrophy gene therapy Elevidys.
The Japanese pharma’s attention deficit/hyperactivity disorder candidate has shown significant symptom improvements in children and adolescents in two late-stage studies.
Subcutaneous injections of Eisai and Biogen’s Leqembi led to numerically greater amyloid removal than the intravenous version of the Alzheimer’s disease therapy, though risks of brain swelling and bleeding remained.
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