As Avidity Biosciences Inc. brought the second-largest follow-on offering of the year to the market, the company also released positive early and midstage stage results of del-zota, an antibody-oligonucleotide conjugate, in treating Duchenne muscular dystrophy. Phase I/II results showed a reversal of disease progression in patients who have been continuously treated for a year, plus improvements in several functional measures.

Remynd NV has turned in positive phase IIa data for a new target in Alzheimer’s disease, showing that restoring calcium homeostasis in neurons reduces levels of pathological tau proteins, increases dopamine levels and has a positive effect on cognition in symptomatic patients.

Two months after starting the phase I/II Synrgy trial with its gene therapy, CAP-002, enrolling 12 pediatric patients with rare disease STXBP1 encephalopathy, Capsida Biotherapeutics paused the study following the death of the trial’s first patient.

Maze Therapeutics Inc. CEO Jason Coloma said the latest phase I results with MZE-782, a prospect for phenylketonuria (PKU) as well as chronic kidney disease (CKD), bolster the firm’s “conviction to move into phase II” next year in both indications.

PMV Pharmaceuticals Inc.’s rezatapopt yielded impressive overall response data in ovarian cancer, prompting the firm to aim for a potential accelerated approval filing in early 2027. The news caused the firm’s stock (NASDAQ:PMV) to jump in premarket trading, but shares ended the day at $1.27, down 20%, as investors signaled they are clearly holding out for further data confirming broader market potential for the p53-targeting small molecule.

“New explosions in biotechnology are allowing us to interrogate cancers at a very sophisticated level compared to before,” Dennis Slamon told audience members at the Global Bio Conference in Seoul, South Korea Sept. 3.

Argenica Therapeutics Ltd. saw its stock drop 56% following mixed results for its ARG-007 phase II Seancon trial in acute ischemic stroke patients undergoing endovascular revascularization, or thrombectomy.

Daiichi Sankyo Co. Ltd. and Merck & Co. Inc.’s antibody-drug conjugate (ADC) ifinatamab deruxtecan produced a confirmed 48.2% objective response rate in a phase II study of previously treated patients with extensive-stage small-cell lung cancer.

Detailed data are expected later, but partners Biontech SE and Duality Biologics Co. Ltd. are celebrating a phase III interim analysis readout demonstrating that HER2-targeting antibody-drug conjugate (ADC) trastuzumab pamirtecan hit the primary endpoint of progression-free survival in patients with HER2-positive unresectable or metastatic breast cancer who have previously received trastuzumab and a taxane-based chemotherapy.