Biomedicine Daily · Saturday, 19 September 2026
CBA FRONTIER INSIGHTS
Ivonescimab First to Beat Pembrolizumab on Overall Survival in Phase 3 Lung Cancer
At the World Conference on Lung Cancer in Seoul, Akeso's ivonescimab became the first therapy to outperform pembrolizumab head to head on overall survival in a randomized phase 3 trial. In HARMONi-2, first-line advanced wild-type NSCLC patients with PD-L1 TPS of at least 1% had median OS of 30.75 months versus 22.57 months, a 27% reduction in death risk, with benefit concentrated in PD-L1-high (HR 0.58) and squamous (HR 0.65) patients. The result builds on a previously reported PFS advantage of 11.1 versus 5.8 months. Ivonescimab is the first domestic Chinese innovative drug to beat pembrolizumab in a phase 3 lung cancer trial, a milestone for PD-1/VEGF bispecific antibodies and for China's drug development sector.
Clinical readouts and safety
Ivonescimab Beats Pembrolizumab on Overall Survival in HARMONi-2, Head to Head
Ivonescimab from Akeso became the first therapy to beat pembrolizumab head to head on overall survival in a randomized phase 3 trial, with median OS of 30.8 months versus 22.6 months and a 27% reduction in death risk in HARMONi-2. Benefit concentrated in PD-L1-high (HR=0.58) and squamous (HR=0.65) patients. The same WCLC also featured two Chinese B7-H3 ADCs, HS-20093 and YL201, each cutting death risk 54% versus topotecan in relapsed small cell lung cancer.
Tam-Peli Extends Survival in Recurrent SCLC; Daraxonrasib Active in RAS-Mutant NSCLC
At the World Conference on Lung Cancer in Seoul, the B7-H3 antibody-drug conjugate Tam-Peli prolonged survival in recurrent small cell lung cancer, with median overall survival of 13.3 months versus 9.4 months (HR 0.46, P<0.001), results published simultaneously in NEJM. The RAS(ON) inhibitor daraxonrasib also showed activity in RAS-mutant non-small cell lung cancer.
AK112 Extends Overall Survival Over Pembrolizumab in HARMONi-2 Phase 3 NSCLC Trial
Ivonescimab (AK112) significantly improved overall survival versus pembrolizumab monotherapy in the phase 3 HARMONi-2 trial, raising median OS to 30.75 months from 22.57 months and reducing the risk of death by 27% in first-line advanced wild-type NSCLC with TPS at least 1%. The OS result, presented at WCLC, follows the previously reported PFS benefit, with median PFS of 11.1 months versus 5.8 months (HR=0.51). AK112 is the first domestic Chinese innovative drug to beat pembrolizumab head-to-head in a phase 3 lung cancer trial.
Fatal toxicities test autoimmune CAR T’s safety thesis
Three patients treated with Novartis' Phase II CAR T candidate rapcabtagene autoleucel (rap-cel) died after developing immune effector cell-associated hemophagocytic syndrome (IEC-HS), a rare acute inflammatory toxicity, and Novartis paused clinical development of the autoimmune program. The deaths challenge the premise that CAR T therapy offers immune reset with less toxicity in autoimmune disease than in cancer, and developers cite rapid manufacturing, construct design, and patient biology as potential determinants of IEC-HS risk.
mRNA-encoding CD19-targeting T cell engager for refractory immune thrombocytopenia
An mRNA-lipid nanoparticle-encoded CD19×CD3 T cell engager, which depletes B cells with attenuated cytokine release in preclinical models, produced sustained platelet recovery over 6 months in three patients with treatment-refractory immune thrombocytopenia. The results support mRNA-encoded T cell engagers as a way to reset autoreactive immunity in B cell-driven autoimmune disease.
Xenon pauses phase 3 depression trials after psychosis events
Regulatory and policy
FDA Approves Ultragenyx Gene Therapy Fayuvi for Children with MPS IIIA
The FDA has approved Fayuvi (rebisufligene etisparvovec), a gene therapy developed by Ultragenyx Pharmaceutical, making it the first FDA-approved therapy for children with mucopolysaccharidosis type IIIA, also known as Sanfilippo syndrome type A. The rare inherited disease progressively damages the brain and nervous system, causing gradual loss of cognitive, speech and other developmental abilities.
FDA Approves FAYUVI, First Gene Therapy for Children with MPS IIIA
The FDA has approved FAYUVI, the first gene therapy for children with Sanfilippo syndrome type A (MPS IIIA), an ultra-rare, fatal lysosomal storage disease that mainly affects the central nervous system. FAYUVI is a one-time intravenous infusion that uses a modified, non-infectious AAV9 vector to deliver functional copies of the SGSH gene to patients' cells. The therapy had received orphan drug, fast track and breakthrough therapy designations from the FDA.
Ultragenyx wins first approval for MPS IIIA, closing a bumpy four-year path
FDA granted Ultragenyx traditional approval for rebisufligene etisparvovec, now Fayuvi, the first treatment for Sanfilippo syndrome Type A (MPS IIIA). CBER approved the gene therapy on clinical evidence that treated children maintained or improved cognitive function versus an untreated natural-history cohort, rather than on CSF heparan sulfate as a surrogate endpoint. The outcome exceeds the accelerated approval Ultragenyx had sought in its original application and resubmission, while CBER's biomarker stance remains unresolved.
NMPA Approves Eli Lilly's RET Inhibitor Selpercatinib Tablets in China
China's NMPA has approved Eli Lilly's selpercatinib tablets, a 5.1-class new drug, for marketing in China, as shown on the agency's website on September 18, 2026. This is the first approval of the tablet formulation of selpercatinib in China. Selpercatinib is the world's first approved highly selective RET inhibitor.
FDA Approves Bayer's Kerendia for Type 1 Diabetes-Related Chronic Kidney Disease
The US FDA has approved Bayer's Kerendia (finerenone tablets) to reduce urinary albumin-to-creatinine ratio (UACR) in adults with chronic kidney disease (CKD) associated with type 1 diabetes. Announced on 17 September 2026, this is the first new treatment option FDA has approved for T1D-related CKD in more than 30 years, and the third indication for finerenone since its first approval in 2021. Around 20% to 30% of type 1 diabetes patients have CKD, and about a quarter of them progress to end-stage renal disease.
Eli Lilly's Selpercatinib Tablet Formulation Approved in China
Eli Lilly's selpercatinib has been approved in China in tablet form, the first domestic approval of this formulation. Selpercatinib, the first highly selective RET kinase inhibitor approved worldwide and able to cross into the central nervous system, was first approved by the US FDA in May 2020 and entered China in September 2022 as a capsule for RET fusion-positive advanced or metastatic NSCLC, RET-mutant medullary thyroid cancer and RET fusion-positive radioactive-iodine-refractory thyroid cancer in adults and patients aged 12 and older.
TACE plus dual Hengrui regimen enters CSCO primary liver cancer guideline 2026 at grade I
TACE combined with Hengrui's "dual ai" regimen has been added to the CSCO Primary Liver Cancer Guideline 2026 as a grade I recommendation with 1A-class evidence. The recommendation rests on the CARES-336 trial, described as China-originated phase III evidence with international presentation. The guideline update was dated 2026-09-18.
Ascentage's APG-2575 Proposed for Breakthrough Therapy With Acalabrutinib in Frontline CLL/SLL
Ascentage Pharma's APG-2575 (lisatocla) has been proposed for breakthrough therapy designation in China, in combination with acalabrutinib for previously untreated adult patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL). Lisatocla is an orally administered selective Bcl-2 inhibitor developed by Ascentage, described as the second worldwide and first in China to show clear efficacy and reach pivotal registration-stage trials as a Bcl-2 inhibitor. The combination regimen showed an overall response rate of 97.7%.
EMA’s CHMP backs eight new medicines in September
EMA's CHMP issued positive opinions for eight new medicines at its September meeting, including two small molecules for ovarian cancer, a potential first daily oral therapy for some adults with hidradenitis suppurativa, and a GLP-2R agonist for short bowel syndrome that FDA previously rebuffed, plus two biosimilars and two generics. Among them is Lifyorli relacorilant, Corcept Therapeutics' selective glucocorticoid receptor antagonist, recommended with nab-paclitaxel for adults with platinum-resistant, high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer who have had one to three prior systemic regimens. EU approval decisions from the European Commission follow.
Eli Lilly escalates oral SERD battle with FDA approval for Inluriyo, Verzenio combo
Industry and deals
Research papers
Gene-Edited Pig Liver Transplanted Into Brain-Dead Human Maintains Function for 11 Days
A team at Air Force Medical University led by Dou Kefeng transplanted a gene-edited pig liver orthotopically into a brain-dead human recipient and maintained liver function for 11 days. The result extends earlier auxiliary metabolic support from engineered pig livers to full orthotopic pig-to-human liver xenotransplantation, which has remained challenging because of the liver's immunological and physiological complexity.
TRI-611, a selective, brain-penetrant molecular glue degrader of ALK.
TRI-611, a brain-penetrant molecular glue degrader, bridges the ALK kinase domain and the CRL4 adaptor CRBN through a degron interface distinct from the kinase active site, yielding proteome-wide selectivity. It degrades all ALK fusion protein forms tested, including wild-type and TKI-resistant versions, driving regression of subcutaneous and intracranial tumour models of ALK-positive NSCLC, alone and synergistically with ALK TKIs. It is described as the first clinical-stage molecular glue degrader targeting an oncogenic gene fusion.
Mutational constraints on RSV F and its neutralization by antibodies.
Deep mutational scanning of RSV F pseudovirus measured how nearly all mutations affect cell entry and neutralization by IgG and Fab forms of nirsevimab, clesrovimab and other key antibodies. A biophysical model explains why resistance to nirsevimab is more common in subtype B than A strains, combining bivalent IgG buffering with lower Fab potency against subtype B. The measurements enable real-time surveillance, showing resistant strains have arisen sporadically but are presently rare.
γδ T cells modulate anti-tumor immunity in small cell lung cancer
γδ T cells infiltrate small cell lung cancer and can kill tumor cells through multiple therapeutic approaches, including MHC-independent recognition via BTN2A1. High levels of γδ T cell infiltration predict improved outcomes with αPD-L1 therapy in SCLC, pointing to these cells as a target for immunotherapy.
γδ T cells BiTE back at small cell lung cancer
Ng et al. characterize cytotoxic γδ T cells in small cell lung cancer and target them with a bispecific T cell engager (BiTE) that engages Vδ2+ cells. The approach is designed to overcome unsuccessful checkpoint blockade immunotherapy by bypassing low MHC-I expression and ineffective CD8+ T cells. The work points to an alternative effector cell for immunotherapy in this cancer.
A universal visual foundation model for computational cytopathology
CROWN is a universal visual foundation model for computational cytopathology, benchmarked on more than 200 real-world cytology tasks across cohorts, including lymph node metastasis and cervical screening datasets. It gives cytopathology a single pretrained model that can be evaluated across many clinical cytology settings rather than task-specific systems.
ABO2203: mRNA Encoding CD19×CD3 Bispecific Tested Against Refractory Immune Thrombocytopenia
ABO2203 is an in vivo T-cell engager approach: an mRNA encoding a CD19×CD3 bispecific antibody, developed for refractory immune thrombocytopenia. It aims to deliver B-cell-directed bispecific therapy without the manufacturing burden, lymphodepletion and genomic-integration risks of CAR-T, and without the short half-life and continuous infusion required by protein TCEs such as blinatumomab. The work targets patients who respond inadequately to glucocorticoids, TPO receptor agonists, rituximab, BTK inhibitors or splenectomy.
Opinion and commentary
Inflection points and transitions in Alzheimer's disease.
Researchers propose that Alzheimer's disease does not progress as a linear cascade from amyloid-β accumulation to Tau pathology and neurodegeneration, but through discrete molecular, cellular and network phases organized around biological inflection points. At these thresholds, stress-driven loss of homeostasis produces qualitative shifts in cellular behavior, and altered tissue states spread as a mosaic across the brain. The framework, published in Nature, identifies amyloid-associated Tau phosphorylation as a pivotal inflection point and reframes Alzheimer's as a disorder shaped by biological thresholds, timing and erosion of homeostatic resilience.