In yet another fail for the Duchenne muscular dystrophy (DMD) field, Taiho Pharmaceutical Co. Ltd.’s pizuglanstat (TAS-205) did not meet the primary endpoint in a phase III trial. The phase III Reach-DMD trial, a randomized, placebo-controlled, double-blind and open-label, extension study of pizuglanstat in patients with DMD showed no significant difference in the mean change from baseline to 52 weeks in the primary endpoint of time to rise from the floor in the ambulatory cohort of the study.
Tickling Wall Street’s already strong interest in the mechanism of action was Aurinia Pharmaceuticals Inc., which June 30 made public positive results from the phase I single ascending-dose study with aritinercept (AUR-200), the company’s dual inhibitor of B cell-activating factor (BAFF) and a proliferation-inducing ligand (APRIL).
A mixed bag of top-line phase II data prompted stellar stock results for Prokidney Corp. and its chronic kidney disease (CKD) and diabetes therapy, rilparencel. The results came from two arms of the study, each with its own treatment regime. The market heartily took to the results as the company’s stock (NASDAQ:PROK) closed 515% upward at $3.73 per share after starting the day under $1 each.
Accunea Ltd.’s bioanalysis technology, Renosure, can measure creatinine clearance in machine perfused kidneys, according to data presented at the recent European Society of Organ Transplantation 2025.
Apogee Therapeutics Inc. plans to enter a crowded atopic dermatitis market with established drugs, such as Dupixent (dupilumab, Regeneron Pharmaceuticals Inc. and Sanofi SA) and Ebglyss (lebrikizumab-lbkz, Eli Lilly and Co.), which are sold by much larger competitors. But the company thinks it can compete with its anti-IL-13 antibody, APG-777, thanks to the drug’s 77 day half-life that may allow it to be dosed only two to four times a year in the maintenance phase, compared to 13 to 26 injections per year for the established competitors.
Cogent Biosciences Inc. is eyeing an NDA submission to the U.S. FDA by the end of this year in the wake of positive top-line results from the registration-directed second part of the Summit phase II trial testing bezuclastinib in non-advanced systemic mastocytosis (SM). The data show clinically meaningful and highly statistically significant improvements in SM across the primary and all key secondary endpoints, including patient-reported symptoms and objective measures of mast cell burden.
Jasper Therapeutics Inc.’s otherwise upbeat data with subcutaneous briquilimab was hamstrung by apparent trouble with one lot of drug used in the phase Ib/II Beacon study in chronic spontaneous urticaria. Shares of the Redwood Calif.-based firm (NASDAQ:JSPR) closed July 7 at $3.04, down $3.73, or 55%.
The first systematic whole genome sequencing study of how chemotherapy damages healthy tissues has shown that many, but not all, of these agents cause mutations and premature aging of normal blood cells.
Exelixis Inc.’s positive phase III top-line data June 22 with zanzalintinib (zanza) in colorectal cancer had Wall Street speculating anew about the odds with the tyrosine kinase inhibitor in an important indication.
Antibiotics specialist Bioversys AG has sealed a potential CHF529 million (US$667.5 million) deal with Shionogi Co. Ltd., in which they will work together on novel ansamycins for treating multidrug-resistant lung infections caused by non-tuberculous mycobacteria.
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