Magenta Therapeutics Inc., the developer of an antibody-drug conjugate intended to help prep certain relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome patients for stem cell transplant or gene therapy, has stopped dosing the drug in part of an ongoing trial after observing two cases of serious adverse events (SAEs) possibly related to the candidate.
After posting positive top-line data in August from its phase III ENHANCE-2 study of ensifentrine for treating chronic obstructive pulmonary disease (COPD), Verona Pharma plc now has positive results from the companion phase III study. The ENHANCE-1 trial of ensifentrine as a COPD maintenance treatment hit its primary and key secondary endpoints, prompting the company to say it plans to submit an NDA for the treatment to the U.S. FDA sometime in the first half of 2023.
Onward Medical NV reported clinical outcomes for the first 10 patients given therapy to regulate blood pressure using its implantable ARC technology. This pulse generator produced an immediate improvement in blood pressure regulation in all study participants.
“Far superior to what almost anyone expected,” was how H.C. Wainwright analyst Ed Arce described the top-line readout for Madrigal Pharmaceuticals Inc.’s phase III study of resmetirom, which hit both of its dual endpoints in patients with nonalcoholic steatohepatitis (NASH) and is expected to form the basis of an accelerated approval application to the U.S. FDA in the first half of 2023.
Cofactor Genomics Inc. opened the non-small-cell lung cancer portion of its study of the Oncoprism test, which predicts response to immunotherapy. The Predicting Immunotherapy Efficacy from Analysis of Pre-treatment Tumor Biopsies (PREDAPT) trial will study the test’s predictive ability in 11 cancers in total.
Guardant Health Inc. reported positive results from the ECLIPSE trial demonstrating 83% sensitivity of its DNA blood test in detecting colorectal cancer (CRC) in average-risk adults. Specificity was 90% in people without advanced neoplasia, as well as in those who had a negative colonoscopy result.
Astellas Pharma Inc. reported top-line results showing zolbetuximab, a monoclonal antibody targeting Claudin 18.2, met the primary endpoint for progression-free survival (PFS) as well as secondary endpoints for overall survival (OS) in the phase III Glow trial in CLDN18.2-positive, HER2-negative, locally advanced unresectable or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma.
Bryostatin-1 has failed another Alzheimer’s disease (AD) study. Synaptogenix Inc. released top-line data showing the protein kinase C activator missed the primary endpoint in the company’s phase II study in treating advanced AD.
When potentially dangerous high liver enzymes showed up in two asymptomatic participants during Third Harmonic Bio Inc.’s ongoing phase Ib study of the KIT inhibitor THB-001, the company decided to discontinue the clinical trial.
A preliminary assessment of Avidity Biosciences Inc.’s phase I/II study of AOC-1001 in myotonic dystrophy type 1 (DM1) showed the first-ever targeted delivery of RNA into muscle, an area previously untreatable with existing RNA therapeutics. Sarah Boyce, Avidity’s CEO, said in a Dec. 14 call that the antibody oligonucleotide conjugate’s data were unprecedented in the RNA space and in myotonic dystrophy type 1 (DM1), labeling it a “revolutionary advancement.”
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