Clinical research
To protect the central nervous system, the blood-brain barrier bars entry to around 98% of molecules—but approaches like Roche’s trontinemab could spell new hope in Alzheimer’s and beyond.
The company’s immunotherapy, in combination with chemotherapy Avastin, showed favorable progression-free survival in a late-stage hepatocellular carcinoma study.
The two mid-stage flops—one in major depressive disorder and another in focal onset seizures—involve drug candidates from partnerships with Xenon Pharmaceuticals and Takeda, respectively.
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
A combination of Gilead Sciences’ domvanalimab and Arcus Biosciences’ zimberelimab, along with chemo, has shown an “encouraging” overall response rate in a mid-stage study.
Although it hit primary and secondary endpoints in a Phase II study, Ventyx Biosciences concluded that the results weren’t strong enough to face the stiff competition for the indication.
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.
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