ORLANDO, Fla. - Chimeric antigen receptor T (CAR T) cells have proved to be a major advance in treating patients with refractory B-cell malignancies so far. But, often, in approaching those issues "we solve one and we create another," Stephen Schuster, a doctor at the Abramson Cancer Center at the University of Pennsylvania in Philadelphia, told reporters at the 61st American Society of Hematology (ASH) annual meeting Saturday.
The “remarkably appealing” route of administration and every-three-month dosing put Ferring Pharmaceuticals SA’s nadofaragene firadenovec (rAd-IFN/Syn3, also known as Instiladrin) in strong position for approval in high-grade Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC).
News of a partial FDA clinical hold for pediatric patients after safety reports from an ongoing trial testing palovarotene in fibrodysplasia ossificans progressiva (FOP) identified cases of early growth plate closure sent shares of Ipsen SA (Paris:IPN) falling 13.6% Friday.
Despite some encouraging top-line data from Sage Therapeutics Inc.’s phase III Mountain study of SAGE-217 on depressive symptoms in adults with major depressive disorder (MDD), the company, the market and analysts were taken aback at the trial’s failure to hit its primary endpoint.
SAN DIEGO – Following up on its October announcement that it would file for FDA approval of beta-amyloid-targeting aducanumab, Biogen Inc. presented the final dataset for the phase III Emerge and Engage studies at the 12th Clinical Trials on Alzheimer’s Disease Meeting.
SAN DIEGO – Three months ago, Acadia Pharmaceuticals Inc. said Nuplazid (pimavanserin), its serotonin inverse agonist and antagonist that preferentially targets the 5-HT2A receptor, met the primary endpoint during an interim look at the phase III Harmony study in patients with dementia-related psychosis (DRP).
In the summer of 2016, when Victoria, British Columbia-based Aurinia Pharmaceuticals Inc. offered phase IIb results with its calcineurin inhibitor voclosporin in lupus nephritis (LN), Wall Street ignored the otherwise-positive results and zeroed in on the trial’s death rate: 13 casualties across three arms of the 265-subject Aura-LV study.
A pivotal trial of Omeros Corp.'s narsoplimab, an investigational therapy for an occasional but serious complication of hematopoietic stem cell transplant (HSCT), found that just one dose was enough to deliver a beneficial effect in more than half the study's high-risk participants, meeting the study's primary endpoint.
New top-line results from a phase III study of Ardelyx Inc.'s tenapanor – recently approved in irritable bowel syndrome – confirmed the drug can also help dialysis-dependent chronic kidney disease patients with hyperphosphatemia achieve reduced serum phosphorus levels vs. placebo.
Protagonist Therapeutics Inc. stock (NASDAQ:PTGX) nosedived 47% on Tuesday after the company released preliminary results from its phase II open-label study of PTG-300, an injectable hepcidin mimetic to treat patients with transfusion-dependent beta-thalassemia.
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