Takeda Pharmaceutical Co. Ltd.'s oral antiviral, maribavir, failed to meet the primary endpoint of noninferiority compared to Roche Holding AG’s valganciclovir in the phase III Aurora study for cytomegalovirus (CMV) infection in hematopoietic stem cell transplant recipients. The multicenter, randomized, double-blind, double-dummy, active-controlled study was to form the basis for a future label expansion for the therapy.
While new data from Arcus Biosciences Inc. and Gilead Sciences Inc. didn't do much to calm an unsteady development corridor, researcher, developers and analysts still hold out hope for the TIGIT pathway. The Arcus-Gilead randomized phase II study combining anti-TIGIT domvanalimab and anti-PD1 antibodies for treating first-line, metastatic non-small-cell lung cancer produced positive results, including improvements in median progression-free survival (PFS) and six-month landmark PFS rates vs. monotherapy. But a hefty dent in Arcus' shares provide a reminder of TIGIT’s fragility as a field, and a reminder of the phase III failure in May for Roche Holding AG unit Genentech Inc.'s anti-TIGIT immunotherapy tiragolumab, which dragged down the share value of several other class entrants. Arcus stock (NASDAQ:RCUS) dipped deeply on Dec. 20 on the new data’s release, but rallied on Dec. 21 by closing 7.5% upward at $22.15 each.
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.
“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.
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.”
After gaining U.S. FDA priority approval for the first gene therapy to treat hemophilia B, CSL Ltd. reported long-term data from the pivotal HOPE-B trial that showed a single infusion of Hemgenix (etranacogene dezaparvovec-drlb) generated elevated and sustained mean factor IX levels and reduced the rate of annual bleeding. Presented at the American Society of Hematology (ASH) annual meeting on Dec. 10, data showed 24-month results reinforced the safety of treatment, with no serious treatment-related adverse effects.
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