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.
Positive results for Revolution Medicines Inc.’s phase I studies of its lead candidate, the RAS-blocker daraxonrasib for treating pancreatic ductal adenocarcinoma, prompted the company to say it’s time for a phase III study in the aggressive cancer. Revolution said it plans to begin a global, randomized and registrational trial in first-line metastatic disease sometime in the fourth quarter of 2025.
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.
Neurosciences specialist NRG Therapeutics Ltd. is poised to put its new class of small-molecule regulators of the mitochondrial permeability transition pore to the test after closing an oversubscribed £50 million (US$67 million) series B.
Medicxi Ventures’ asset-focused investment model has scored another hit, in a $450 million deal with Servier SA, for KER-0193, an oral small molecule that is ready for phase II development in the treatment of the inherited autism spectrum disorder, fragile X.
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.
Results from the global phase III Harmoni trial presented at the International Association for the Study of Lung Cancer 2025 World Conference on Lung Cancer (WCLC) showed a statistically significant increase in progression-free survival after treatment with ivonescimab plus chemotherapy compared to chemotherapy alone, but failed to show a statistically significant benefit in overall survival (OS).
Robust efficacy, competitive tolerability and ease of administration. Those are the qualities for a potential blockbuster antiseizure medication, according to Abe Ceesay, CEO of Rapport Therapeutics Inc., which reported a successful phase IIa trial testing RAP-219 in patients with drug-resistant focal onset seizures and aims to move into a large-scale phase III program in 2026.
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.
“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.
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