Biomedicine Weekly · Week 38, 2026 · 09.14 — 09.20
CBA FRONTIER INSIGHTS
This issue's figures
Open a card for the chart; click again to closeIvonescimab Outlives Keytruda; B7-H3 ADCs Reshape Small Cell Lung Cancer
The World Conference on Lung Cancer in Seoul dominated this week. Akeso's PD-1/VEGF bispecific ivonescimab became the first therapy to beat pembrolizumab head to head on overall survival in a randomized phase 3 trial, posting median OS of 30.8 versus 22.6 months in first-line PD-L1-positive NSCLC. In relapsed small cell lung cancer, two parallel phase 3 trials of B7-H3 ADCs each cut the risk of death by 54% versus topotecan, with TAISHAN-302 published in the New England Journal of Medicine. Meanwhile, the FDA approved firsts in gene therapy and SMA, China's NHSA piloted pre-approval pricing for new medical technologies, and the FDA finalized a pilot designed to bring first-in-human trials back to US soil.
Ivonescimab Wins Overall Survival Head-to-Head
On this pageAkeso and Summit's PD-1/VEGF bispecific ivonescimab beat pembrolizumab on overall survival in the phase 3 HARMONi-2 trial in first-line PD-L1-positive advanced NSCLC: median OS of 30.8 versus 22.6 months, HR 0.73, P=0.009, a 27% reduction in death risk. Presented by Professor Zhou Caicun at the WCLC presidential symposium, with 234 OS events at a median follow-up of 36 months, it is the first randomized double-blind phase 3 trial positive for both PFS and OS against pembrolizumab. Benefit was most pronounced in PD-L1-high and squamous patients. A key regulatory decision point arrives in November.
Ivonescimab Beats Keytruda on Overall Survival in Phase III HARMONi-2 NSCLC Trial
Ivonescimab, a PD-1/VEGF bispecific antibody, beat Merck's Keytruda on overall survival in the HARMONi-2 study, a phase III head-to-head trial in advanced NSCLC, with median OS of 30.8 months versus 22.6 months and a 27% reduction in relative risk of death. This is the first time a PD-1/VEGF bispecific has defeated Keytruda on overall survival in a phase III head-to-head trial. Akeso and Summit Therapeutics presented the data at the World Conference on Lung Cancer.
Ivonescimab Beats Pembrolizumab on Overall Survival in HARMONi-2 Trial
Ivonescimab, Akesu Bio's PD-1/VEGF bispecific antibody, beat pembrolizumab as first-line monotherapy in PD-L1-positive non-small cell lung cancer in the HARMONi-2 trial. Median overall survival was 30.8 months versus 22.6 months with pembrolizumab, HR=0.73, P=0.009, presented by Professor Zhou Caicun at the WCLC presidential symposium. It is the first drug to beat pembrolizumab in a randomized controlled trial on two endpoints.
Ivonescimab Beats Pembrolizumab on Overall Survival in PD-L1-Positive NSCLC in HARMONi-2
Ivonescimab monotherapy as first-line treatment for PD-L1-positive non-small cell lung cancer achieved a median overall survival of more than 30 months, extending survival by over 8 months compared with pembrolizumab. The phase 3 HARMONi-2 trial, led by Professor Zhou Caicun of Shanghai East Hospital and presented at the 2026 World Conference on Lung Cancer, is the first randomized double-blind phase 3 trial to show positive results for both overall survival and progression-free survival against pembrolizumab.
Ivonescimab Beats Pembrolizumab on Overall Survival in HARMONi-2 Phase 3 Trial
Ivonescimab, a PD-1/VEGF bispecific antibody from Akeso, has delivered full overall survival results from the phase 3 HARMONi-2 trial, with a median OS of 30.8 months versus 22.6 months for pembrolizumab monotherapy in a head-to-head comparison. Reported by Caicun Zhou of Shanghai East Hospital, this makes ivonescimab the first cancer immunotherapy to achieve positive results in both progression-free survival and overall survival against pembrolizumab in a randomized, double-blind phase 3 study. A key regulatory decision point arrives in November.
Ivonescimab Beats Pembrolizumab on Overall Survival in First-Line PD-L1-Positive Advanced NSCLC
Ivonescimab significantly extended overall survival versus pembrolizumab as first-line treatment for PD-L1-positive advanced NSCLC, meeting the prespecified statistical bar for significance. At a median follow-up of 36 months as of August 20, 2026, 234 overall survival events had occurred and median overall survival reached 30.8 months. Prespecified subgroup analyses showed a consistent survival benefit, most pronounced in patients with high PD-L1 expression.
B7-H3 ADCs Set New Bar in Relapsed SCLC
On this pageTwo parallel phase 3 trials of B7-H3 antibody-drug conjugates reported back to back at WCLC. TAISHAN-302 (Tam-Peli/YL201) and ARTEMIS-008 both hit their primary OS endpoint against topotecan with identical hazard ratios of 0.46 (95% CI 0.35-0.62), with TAISHAN-302 published simultaneously in NEJM. Tam-Peli cut death risk 54% and progression risk 71%, with median OS of 13.3 versus 9.4 months (P<0.001). The same meeting also featured HS-20093 cutting death risk 54% in this setting, and the RAS(ON) inhibitor daraxonrasib showed activity in RAS-mutant NSCLC.
TAISHAN-302 Trial Sets New Benchmark for Second-Line Treatment of Relapsed Small Cell Lung Cancer
The TAISHAN-302 study, led by Professor Zhang Li's team, was presented at the WCLC plenary session and published in the New England Journal of Medicine. It tested a drug named only in Chinese in the source material as second-line therapy for relapsed small cell lung cancer, a setting where nearly all patients relapse after first-line platinum-based chemotherapy with immunotherapy and effective options are scarce. The report names no efficacy figures.
Parallel Phase III Trials Show B7-H3 ADCs Cut Death Risk in Relapsed Small Cell Lung Cancer
Two Phase III trials of B7-H3 antibody-drug conjugates in second-line relapsed small cell lung cancer, TAISHAN-302 and ARTEMIS-008, both hit their primary overall survival endpoint against topotecan, and TAISHAN-302 was published simultaneously in the New England Journal of Medicine. Reported back to back at WCLC 2026, the trials yielded identical OS hazard ratios of 0.46 (95% CI 0.35-0.62), providing independent, replicable Phase III evidence that B7-H3 ADCs beat current standard treatment in this setting.
B7-H3 ADC Tam-Peli Cuts Death Risk 54% in Relapsed Small Cell Lung Cancer Phase III Trial
The B7-H3-targeted ADC Tam-Peli (YL201) improved overall survival, progression-free survival and objective response rate in patients with relapsed small cell lung cancer versus topotecan hydrochloride, according to phase III TAISHAN-302 data presented at WCLC. The drug reduced the risk of death by 54% and the risk of disease progression or death by 71%. The results position the first B7-H3 ADC to break through in second-line treatment of this disease.
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.
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.
FDA Approvals: Firsts in Gene Therapy, SMA, CKD
On this pageThe FDA granted Ultragenyx traditional approval for Fayuvi (rebisufligene etisparvovec), a one-time intravenous AAV9 gene therapy delivering functional SGSH genes and the first treatment for Sanfilippo syndrome type A, based on treated children aged 2-5 maintaining or improving cognitive scores versus natural-history controls. Scholar Rock's apitegromab (Isembyld), the first muscle-targeted SMA therapy, was approved September 11 for patients aged 2 and older on SMN2-targeted therapy. Bayer's finerenone (Kerendia) was approved for CKD related to type 1 diabetes, the first new therapy for this population in over 30 years and its third US approval.
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.
FDA Approves First Gene Therapy for Pediatric Patients with Sanfilippo Syndrome Type A
The FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time intravenous AAV9 gene therapy from Ultragenyx Pharmaceutical, as the first treatment for pediatric patients with mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A. In an open-label, single-arm multicenter study, treated patients aged 2 to 5 maintained or improved cognitive scores versus an untreated historical control. Patients receive corticosteroids from one day before infusion through at least eight weeks after, with warnings including thrombotic microangiopathy.
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.
Scholar Rock scores long-awaited FDA approval, the first for muscle loss in SMA
FDA Approves Scholar Rock's Apitegromab (Isembyld), First Muscle-Targeted SMA Therapy
Scholar Rock's apitegromab, brand name Isembyld, received FDA approval on September 11 as the first muscle-targeted therapy for spinal muscular atrophy. It is indicated for SMA patients aged 2 and older who are receiving SMN2-targeted therapy, and works by inhibiting myostatin production to promote muscle growth. US shipments begin within days of approval.
Bayer’s Kerendia secures FDA nod as first drug in 30 years for Type 1 diabetes-associated kidney disease
FDA Approves Bayer's Kerendia for Type 1 Diabetes-Related Chronic Kidney Disease, First New Therapy in Over 30 Years
The US FDA has approved Bayer's Kerendia (finerenone) for chronic kidney disease associated with type 1 diabetes, making it the first entirely new therapy for this population in more than 30 years. The approval is finerenone's third in the US, following July 2021 approval for reducing cardiorenal risk in adults with CKD related to type 2 diabetes and July 2025 approval for symptomatic chronic heart failure with LVEF of at least 40%. Finerenone is now the only mineralocorticoid receptor antagonist approved for CKD related to both type 1 and type 2 diabetes.
HER2-Targeted ADCs Advance Across Tumors
On this pageIn the phase 3 DESTINY-Lung04 trial in first-line HER2-mutant advanced NSCLC, trastuzumab deruxtecan delivered median PFS of 14.3 versus 8.3 months for pembrolizumab plus chemotherapy, with ORR of 70% versus 44.5% — but preliminary OS trended against it, with median OS of 29.3 versus 33.1 months and a 15% higher death risk. China's NMPA approved T-DXd plus pertuzumab on August 11, 2026 as the first first-line ADC regimen for HER2-positive metastatic breast cancer, based on DESTINY-Breast09. Chia Tai Tianqing's HER2 bispecific ADC TQB2102 hit its primary endpoint in a phase 3 HER2-low breast cancer trial.
Trastuzumab Deruxtecan Extends Median PFS to 14.3 Months as First-Line Therapy in HER2-Mutant Advanced NSCLC
In the phase III DESTINY-Lung04 trial, first-line trastuzumab deruxtecan (Enhertu) gave a median progression-free survival of 14.3 months in patients with HER2-mutant advanced non-small cell lung cancer, versus 8.3 months with pembrolizumab plus chemotherapy. It is the first HER2-targeted therapy to show superiority over standard treatment in this setting.
Enhertu Extends PFS in DESTINY-Lung04 but Early Overall Survival Signals 15% Higher Death Risk
Enhertu posted strong progression-free survival in the DESTINY-Lung04 trial presented at WCLC 2026: median PFS of 14.3 months, 6 months longer than control, with a 37% reduction in risk of disease progression or death and an ORR of 70% versus 44.5%. Preliminary overall survival did not favor the drug: 115 deaths (50.7%) on Enhertu versus 98 (43.2%) on control, median OS 29.3 versus 33.1 months, a 15% higher risk of death.
NMPA Approves T-DXd Plus Pertuzumab for First-Line HER2-Positive Metastatic Breast Cancer
China's NMPA approved on August 11, 2026 the combination of trastuzumab deruxtecan (T-DXd) with pertuzumab as first-line treatment for adults with unresectable or metastatic HER2-positive breast cancer. The approval rests on the global DESTINY-Breast09 trial. The regimen challenges the long-standing escalation model in which patients start on trastuzumab plus pertuzumab with a taxane and switch to an ADC only after progression.
NMPA Approves T-DXd Plus Pertuzumab as First-Line Treatment for HER2-Positive Advanced Breast Cancer
China's NMPA approved trastuzumab deruxtecan (T-DXd) combined with pertuzumab on August 11, 2026 as first-line treatment for adults with unresectable or metastatic HER2-positive breast cancer. It is the first first-line ADC regimen approved in HER2-positive metastatic breast cancer in China. The approval rests on the global DESTINY-Breast09 study, in which Chinese patients made up close to a quarter of enrolment and the Chinese subgroup showed efficacy and safety better than the global population.
Chia Tai Tianqing HER2 Bispecific ADC TQB2102 Hits Primary Endpoint in Phase III HER2-Low Breast Cancer Trial
Chia Tai Tianqing's HER2 bispecific ADC TQB2102 has hit its prespecified primary endpoint and key secondary endpoints in the Phase III TQB2102-III-01 trial, per an interim analysis judged by an independent data monitoring committee. The trial tested the drug in HER2-low recurrent or metastatic breast cancer. The company says TQB2102 balances efficacy and safety through three core technical features.
Policy, Platform and Repurposing Shifts
On this pageChina's NHSA published a notice on September 15 piloting pre-approval pricing for high-level new medical technologies, letting tertiary hospitals apply from the start of clinical research with real-world value assessment embedded. The FDA finalized its Expedited IND Pilot to speed first-in-human trials in the US, citing China's over 7,100 registered Phase 1 trials in 2024, about 39% of the global total, and estimating 6-12 months of savings. Also this week: FDA signaled willingness to approve psychedelics on 12-week data before redosing is settled; a gene-edited pig liver functioned 11 days after orthotopic transplant into a brain-dead recipient; AI-designed protein assemblies delivered RNA orders of magnitude more efficiently; and levetiracetam showed survival benefit in diffuse midline glioma via GABAergic synapse dampening.
China's NHSA to Trial Pre-Approval Pricing Mechanism for High-Level New Medical Technologies
On September 15, China's National Healthcare Security Administration published a notice (document Yibao Fa [2026] No. 23) establishing a trial pre-approval system for medical service pricing of high-level new technologies and products. The mechanism moves the pricing acceptance stage earlier so that regulatory approval for clinical use links directly to charging authorization, and it pairs this with constraints on consumable prices, conditional charging and value assessment to filter routine iterative products and guard against misuse. Companies will be pushed to prepare clinical and economic evidence in parallel from early development stages.
China's NHSA Pilots Pre-Approval Pricing Pathway for New Medical Technologies and Products
China's National Healthcare Security Administration issued a notice on September 15 establishing a pilot pre-approval pricing system for high-level new technologies and products, allowing hospitals to apply for pricing pre-approval from the start of clinical research rather than after regulatory approval. Under the system, a tertiary hospital applies directly to provincial medical insurance departments, and once the first hospital files, others need not repeat the process. Approved technologies can then convert pre-approval into formal pricing through new, conditionally added, or compatible price items, with real-world value assessment embedded throughout.
FDA Finalizes Expedited IND Pilot to Reclaim First-in-Human Trials from China and Australia
On September 15, 2026, the FDA published the final design of its Expedited IND Pilot, which pairs selected sponsors with Qualified Research Institutions to submit IND components on a rolling basis during Pre-IND review. The agency explicitly cited China and Australia, noting FIH trials in the US can take up to two years while both countries move faster; HHS data cited show China's share of global Phase 1 trials passed the US in 2021, with over 7,100 registered trials in 2024, about 39% of the global total. The 30-day statutory review window and Clinical Hold authority are unchanged; FDA estimates trimming unnecessary submission content alone could save 6 to 12 months, and pilot experience may inform a formal QRI qualification system.
FDA could approve psychedelics before redosing is resolved
FDA officials from CDER say the agency is prepared to approve psychedelic therapies for chronic psychiatric disorders based on 12-week efficacy data, before sponsors have settled how long effects last or when patients need redosing. The position appears in a New England Journal of Medicine article and clarifies what the agency's final guidance for psychedelic drug development, released in July, left open. Companies profiled include Compass Pathways, Cybin, Ataibeckly and Eli Lilly.
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.
Creating bottom-up RNA transfer vehicles from synthetic protein assemblies.
AI-designed protein assemblies combined with natural protein domains yielded more than 100 bottom-up RNA transfer vehicles with unique sizes and shapes, surpassing the RNA transfer efficiency of widely used delivery vehicles by several orders of magnitude. Tropism was programmed with computationally designed peptide binders, enabling delivery of therapeutically relevant cargo RNAs across cellular models. One vehicle's in vivo biodistribution was mapped in a mouse at near-single-cell resolution, confirmed as safe, and used for a gene-editing treatment strategy for Duchenne muscular dystrophy in patient-derived cells and a pig.
Levetiracetam therapeutically targets GABAergic synapses in diffuse midline glioma
The antiseizure drug levetiracetam is associated with longer survival and reduced tumor growth in diffuse midline glioma, but not in hemispheric high-grade glioma. In experimental models and patient cohort data, the effect arose from selectively dampening GABAergic synaptic signaling, acting independently of the drug's canonical SV2A-mediated antiseizure mechanism. This points to a tumor-specific therapeutic use for an already available drug.
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