Biomedicine Daily · Monday, 21 September 2026
CBA FRONTIER INSIGHTS
Ivonescimab Posts 16.9-Month PFS After Third-Generation EGFR-TKI Failure in NSCLC
Ivonescimab, a PD-1/VEGF bispecific antibody, achieved a median progression-free survival of 16.9 months in patients with locally advanced or metastatic non-squamous NSCLC whose disease progressed on third-generation EGFR-TKIs, against 4-5 months for traditional chemotherapy in this setting. China's NMPA has approved the drug for this population, establishing a new treatment standard for third-generation EGFR-TKI-resistant disease. The result anchors a day that also brought the FDA approval of Ultragenyx's gene therapy FAYUVI for Sanfilippo syndrome type A at $3.95 million per dose — the world's second most expensive drug — and a 16-policy package from China's NHSA and NHC opening medical insurance payment to stem cell drugs.
Clinical readouts and safety
Jie'entai Gets First Prescription in China for FGFR-Inhibitor-Resistant Biliary Tract Cancer
The first prescription for Jie'entai, written by Professor Zhou Jun, has been dispensed in China, moving the drug into clinical use. It is described as the first targeted therapy approved worldwide for biliary tract cancer that has progressed after FGFR inhibitor treatment, giving Chinese patients with drug-resistant advanced disease a treatment option where none existed. The shift is from no available drug to an accessible therapy.
Ivonescimab Reaches 16.9-Month PFS After Third-Generation EGFR-TKI Resistance in NSCLC
Ivonescimab, a PD-1/VEGF bispecific antibody, delivered a median progression-free survival of 16.9 months in patients with locally advanced or metastatic non-squamous NSCLC whose disease progressed on third-generation EGFR-TKI treatment, compared with 4-5 months for traditional chemotherapy in this setting. China's NMPA has approved it for this population, giving third-generation EGFR-TKI-resistant disease a new treatment standard.
Beacon lights path to FDA approval with pivotal trial win for rare vision loss gene therapy
Beacon Therapeutics' gene therapy for X-linked retinitis pigmentosa improved visual acuity in a pivotal trial, positioning the company to seek regulatory approval for the rare vision loss disease. The company plans to file based on the result, which comes in an indication where larger companies have not succeeded.
Regulatory and policy
China's NHSA and NHC Open Medical Insurance Payment Path for Stem Cell Drugs
China's National Healthcare Security Administration and the National Health Commission issued "Measures to Support High-Quality Development of Innovative Drugs", a set of 16 supporting policies covering stem cell drugs from R&D and clinical trials through to medical insurance payment. The measures give stem cell products a defined legal status as drugs and establish dual payment channels to address high treatment costs. Implementation details will follow from the agencies.
FDA Approves Ultragenyx Gene Therapy FAYUVI for MPS IIIA at $3.95 Million Per Dose
On September 17, the FDA approved FAYUVI (rebisufligene etisparvovec-hopf, development code UX111), a gene therapy from Ultragenyx Pharmaceutical, for children with mucopolysaccharidosis type IIIA, also called Sanfilippo syndrome type A. Ultragenyx set the US list price at $3.95 million per dose, second only to Lenmeldy at $4.25 million, making FAYUVI the world's second most expensive drug.
Envafolimab Plus GEMOX Enters 2026 CSCO Biliary Tract Cancer Guidelines as First-Line Grade I Recommendation
The 2026 edition of the CSCO biliary tract cancer guidelines, released on September 18, 2026 at the 26th CSCO annual meeting, adds envafolimab combined with gemcitabine plus oxaliplatin (GEMOX) as a grade I recommendation (category IA evidence) for first-line treatment of unresectable locally advanced or metastatic biliary tract malignancies in patients who can tolerate intensive chemotherapy. It is the first first-line immune combination regimen for biliary cancer based on a trial enrolling only Chinese patients, with evidence from 462 patients at 36 centers across China.
Research papers
Fudan Team Led by Yu Faxing Maps Mechanism Controlling Organ Size, Aging and Regeneration
A team led by Yu Faxing at Fudan University, with collaborators, has published work in Cell Stem Cell on the mechanisms that control organ size and govern organ aging and regeneration. The study addresses how organs stop growing once they reach a defined size and how growth programs restart after injury, questions both listed among the 125 major scientific questions selected by Science. The work points to a shared mechanistic basis linking size control, aging, and regenerative growth.
Single-Cell and Spatial Mapping With CRISPR Points to GLIS3 in IBD Fibrosis
Integrating public IBD single-cell datasets with 10X Xenium spatial transcriptomics, researchers built a map of nearly 4.3 million cells and identified an interaction circuit between FCN1+IL1B+ macrophages and inflammation-associated fibroblasts (IAFs). IAFs were enriched in the N1 and N14 pathological niches and spatially co-localized with these macrophages. Bidirectional CRISPR screens pinpointed GLIS3 as the core transcription factor driving this immune-stromal loop, providing a basis for anti-fibrotic targets in IBD.
COVID-19 vaccination around immune checkpoint inhibitor start and survival in a nationwide cohort of patients with cancer.
In a French nationwide cohort of 95,015 adults initiating immune checkpoint inhibitor therapy, mRNA COVID-19 vaccination within 100 days before or after ICI start was associated with modestly lower early all-cause mortality (pre-ICI HR 0.90 at 1-3 months; post-ICI HR 0.84 at 3 months), with no significant association after 12 months. Similar patterns for non-mRNA COVID-19 and other adult vaccines point to healthy-vaccinee effects rather than a specific mRNA vaccine benefit.
Methylation-Based ctDNA for Post-Treatment Surveillance of Non-Viral Head and Neck Cancer
A methylation-based ctDNA assay (Guardant360) detected recurrence in non-virally associated head and neck squamous cell carcinoma with 76.9% sensitivity and 94.6% specificity (AUC 0.858) in a 50-patient post-treatment cohort. Positive ctDNA preceded clinical detection in 6 of 10 recurrences by a median of 131 days and tracked with worse survival: 1-year progression-free survival was 25.0% versus 96.4%. The findings support adding methylation-based ctDNA to surveillance for this population, pending prospective validation.
Methods and resources
An AI Model Identifies Chemotherapy Benefit in Node-Negative HR+/HER2- Breast Cancer Patients from TAILORx, a Phase 3 Randomized Clinical Trial
An AI model integrating H&E pathology image features with clinical variables (Ataraxis Breast CTX) identified which intermediate-RS node-negative HR+/HER2- breast cancer patients benefit from adjuvant chemotherapy in a prespecified analysis of 6735 TAILORx patients. CTX-benefit showed a significant treatment-by-biomarker interaction (p = 0.016), with high-benefit patients gaining 5.6% in 5-year disease-free interval from chemotherapy while low-benefit patients showed none; stratifying by RS alone at a matching threshold was not significant (p = 0.20). CTX-prognostic also predicted DFI independent of RS (C-index 0.736 with endocrine therapy alone).