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.

The funding is in place for a phase IIb trial of its lead gut microbiome live biotherapeutic, after MRM Health NV closed a €55 million (US$64 million) series B round.

Phase III results from Sanofi SA’s study of amlitelimab in treating atopic dermatitis met the primary and key secondary endpoints, but investors took a step back.

Roivant Sciences Ltd.’s Immunovant Inc. unit unveiled phase II data in Graves’ disease with batoclimab, whetting appetites on Wall Street for the results with next-generation prospect IMVT-1402 from two potentially registrational trials that are enrolling now.

There have been numerous improvements in the treatment of cardiovascular disease since the European Society of Cardiology (ESC) first met in 1950, but unmet medical need remains and the science continues to advance, as delegates heard at the 75^th annual meeting in Madrid, Spain, Aug. 29-Sept. 1.