Biomedicine Daily · Wednesday, 21 October 2026
CBA FRONTIER INSIGHTS
FDA Approves First-of-Its-Kind Chronic Hepatitis B Treatment
The FDA has approved Hibsago (bepirovirsen), in combination with existing antiviral therapy, to treat certain adults with chronic hepatitis B without cirrhosis. Hibsago is the first FDA-approved antisense oligonucleotide for chronic hepatitis B.
Clinical readouts and safety
CSPC's HD1916 Meets Equivalence to Wegovy in Phase 3 Obesity Trial Published in Diabetes, Obesity and Metabolism
CSPC Pharmaceutical Group's HD1916, a chemically synthesized semaglutide injection, showed therapeutic equivalence to Wegovy in a Phase 3 obesity study published in Diabetes, Obesity and Metabolism (PMID: 42839752). The trial met its prespecified equivalence margin for weight loss versus the comparator.
Henlius Serplulimab ASTRIDE US Bridging Data to Be Presented Orally at ESMO 2026
Henlius released the ASTRIDE abstract at ESMO 2026. The US head-to-head bridging study will be presented as an oral report on serplulimab versus atezolizumab, both with chemotherapy, as first-line treatment for ES-SCLC. At a median follow-up of 14.1 months, median OS was 12.4 months with serplulimab versus 10.2 months with atezolizumab; 24-month OS rates were 32.2% and 17.6%. Safety was generally comparable between groups with no new safety signals; HANSIZHUANG had 10 studies accepted at the congress.
TRIC-I-HF: Transcatheter Tricuspid Repair Cuts 3-Year Death or Heart Failure Hospitalization Risk by 60% Versus Medication Alone
The TRIC-I-HF trial showed that transcatheter tricuspid valve repair combined with medication reduced the risk of death or heart failure hospitalization at 3 years by 60% compared with medication alone in 360 high-risk patients (HR 0.40, NCT04634266).
Lipoprotein(a)-Lowering Agent Fails to Reduce Cardiovascular Events
Pelacarsen did not reduce adverse cardiovascular events in a phase 3 trial despite lowering Lp(a) levels, according to the manufacturer's announcement.
Rapport’s epilepsy drug finds surprising success in bipolar trial
Rapport's RAP-219 met the primary endpoint in a Phase 2 trial in bipolar mania, improving scores by 3.1 points more than placebo at week 3, a statistically significant difference. The study enrolled about 250 adults with bipolar mania; the RAP-219 group improved an average of 15.4 points on the primary scale at week 3 versus 12.3 points for placebo. All 11 scale components and a secondary mania severity endpoint beat placebo, and adverse events were mild to moderate with similar rates in both groups.
Epkinly data only one of Genmab's make-or-break readouts ahead of steep sales erosion
BioSpace reports that Genmab faces the 2029 U.S. patent expiry of Darzalex, with revenue potentially falling from about $3.3 billion to nearly zero. The company still must report Phase 3 data this year for petosemtamab's LiGeR-HN1 and Rina-S's RAINFOL-02, in first-line HNSCC and platinum-resistant ovarian cancer, respectively.
Oxygen vs Air at Birth for Moderate- to Late-Preterm Infants
JAMA published a cluster randomized clinical trial comparing the safety and efficacy of starting respiratory support at birth with FiO2 0.30 versus 0.21 (air) in infants born at 32 to 35 weeks' gestation. The abstract describes the study design; specific outcome data were not provided in the feed text.
Regulatory and policy
gov.ukMHRA authorises plozasiran (Redemplo) for adults with familial chylomicronaemia syndrome
The UK MHRA authorised plozasiran (Redemplo) on 19 October 2026 for adults with familial chylomicronaemia syndrome. Original link: https://gov.uk/government/news/mhra-authorises-plozasiran-redemplo-for-adults-with-familial-chylomicronaemia-syndrome
FDA approves pralsetinib for non-small cell lung cancer with RET gene fusions
On August 9, 2023, the FDA granted regular approval to pralsetinib (Gavreto, Genentech) for adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC).
FDA Issues Final Guidance on Prescription Drug Apps and Software
FDA issued final guidance on October 20, 2026, establishing a framework for developing, distributing, and labeling apps and software that accompany prescription drugs. The guidance distinguishes companion apps connected to a medical device (such as an app paired with an inhaler), which generally must be described on the drug's prescribing label, from standalone apps (symptom trackers, dose reminders, health coaching tools), which require labeling only if shown to be essential for safe and effective use or to provide a meaningful clinical benefit.
Most new drugs are still too costly at launch, ICER says
A report ICER published on October 20, 2026, covering 26 new drugs approved from 2022 to 2025, found that about two-thirds were priced above its cost-effectiveness thresholds at net prices. The inflation-adjusted median net price rose from $190,000 to $257,000, and nearly a quarter of the drugs assessed in 2025 had net prices above $500,000.
Regulatory Considerations for Prescription Drug Use-Related Software
FDA announced the final guidance "Regulatory Considerations for Prescription Drug Use-Related Software" (Docket FDA-2023-D-2482), establishing that drug labeling authorities apply to software outputs disseminated by or on behalf of a drug sponsor for use with a prescription drug or a drug-led drug-device combination product. The draft version was issued on September 19, 2023.
Submitting Clinical Trial Data to Evaluate the Ability to Operate a Motor Vehicle
FDA's Center for Drug Evaluation and Research has issued a final Level 2 guidance specifying the technical specifications for submitting clinical trial data used to evaluate the ability to operate a motor vehicle in support of marketing applications for drugs or biological products.
Industry and deals
Google NewsNouscom raises $135M for pivotal trial of cancer-preventing vaccine candidate - Fierce Biotech
Nouscom has completed a $135 million financing round, which will fund the pivotal clinical trial of its cancer-preventing vaccine candidate.
Research papers
Xu Ninglong Team Reports Direct Evidence in Science That Dendrites Participate in Abstract Rule Decisions in Living Animals
A team led by Xu Ninglong at the Chinese Academy of Sciences Center for Excellence in Brain Science reports in Science that, using two-photon imaging in awake mice performing a rule-switching task while simultaneously recording the apical dendrite and soma of the same neuron, dendrites encode abstract rules while somata encode sensory input. The two signals undergo nonlinear integration within the cell to drive decisions. Optogenetic inhibition of apical dendrites significantly weakened decision signals, suggesting dendritic computation is essential for flexible cognition and offering biological clues for brain-inspired multi-compartment AI architectures and for studying disorders with impaired cognitive flexibility such as obsessive-compulsive disorder.
Delocalized DNA holes imprint mutation position in mismatch-repair-deficient cancers
In mismatch repair deficient gastric, colorectal and endometrial cancers, researchers found G>T mutations within guanine runs enriched at the 5' guanine (0.86–0.87) and depleted at the 3' guanine (0.05–0.08).
Individualized Cardiac Index-Guided Hemodynamic Therapy in High-Risk Patients Undergoing Elective Major Abdominal Surgery: The PELICAN Randomized Clinical Trial.
The PELICAN multicenter, single-blind randomized controlled trial enrolled 1095 high-risk patients undergoing elective major abdominal surgery to compare individualized cardiac index-guided hemodynamic therapy with routine care. The primary composite outcome (acute kidney injury, myocardial injury, severe infection, nonfatal cardiac arrest, or death within 7 days) showed no significant difference between groups (44.3% vs 46.9%, RR 0.94, 95.1% CI 0.83-1.08, P=0.38).
Structural basis of a SWI/SNF-associated transcription pre-initiation complex
The study resolves the structural basis of a SWI/SNF-associated transcription pre-initiation complex. Cell 189, 1-18.e1-e13; October 29, 2026.
ESCRT-dependent YAP degradation by AKT blockade overcomes adaptive resistance to RAS(ON) inhibition in pancreatobiliary cancer
A study finds that AKT inhibition degrades YAP protein via an ESCRT-dependent lysosomal pathway, thereby overcoming adaptive resistance to RAS(ON) inhibition in pancreatobiliary cancer.
Learning how single cells respond to perturbations.
Molecular Cell highlights a recent Cell study by Adduri et al. that introduces State, a machine learning model able to generalize across different cellular contexts and predict how single-cell transcriptomes respond to genetic or chemical perturbations. Original link: https://doi.org/10.1016/j.molcel.2026.09.033
A Common CD36 Variant and the Genetic Landscape of Dilated Cardiomyopathy in Individuals of African Ancestry.
In a multicohort genetic association analysis of 82,623 individuals of African ancestry, the population attributable fraction of dilated cardiomyopathy for CD36 Y325* was 9.0%, exceeding the 3.7% for TTN truncating variants.
Medical XpressRepeating stretch of DNA offers a new clue to Alzheimer's susceptibility
A study in Nature Neuroscience by the Hong Kong University of Science of Technology and other institutions found that longer CCG repeats in the FAM193B promoter are linked to higher odds of Alzheimer's disease. Two Chinese cohorts totaling 1,516 patients and 1,674 cognitively normal participants showed about twice the odds for those whose longer copy contained at least 18 CCG repeats versus no more than 11. In a UK Biobank analysis of 2,674 cases and 22,996 controls of European ancestry, the association was weaker and used different thresholds.
Medical XpressDNA once dismissed as 'noise' reveals new bacterial species in prostate cancer patients
A University of East Anglia team reconstructed 249 near-complete bacterial genomes from existing sequencing data of 194 prostate cancer patients, identifying 18 previously unrecorded bacterial species. The team screened 2,176 PPCG sequencing datasets, retaining non-human reads normally filtered out as noise and rebuilding genomes through metagenomic assembly; 24 genomes matched no species on record. The study assigned provisional names to 16 of the new organisms and developed a precise DNA test for one species, but whether these bacteria contribute to prostate cancer remains unclear.
Phage-ICE tandems facilitate bacterial adaptation and antimicrobial resistance spread.
The study characterizes cotransferable units formed by the accretion of phages and ICEs in Streptococcus, and identifies a fail-safe route of spread via RecA-dependent homologous recombination when excision is impaired. The team screened 612 phage-ICE tandems across seven phyla and 135 species, including multidrug-resistant pathogens, which can cotransfer flanking DNA, remodel transcriptional profiles, and increase oxidative stress tolerance. DOI: 10.1126/sciadv.aed3416
NHE1 sustains leukemia-propagating capacity and mitochondrial fitness in T-cell acute lymphoblastic leukemia
Using a zebrafish transplantation model, the authors screened 786 FDA-approved compounds and found that amiloride markedly reduced the frequency of T-ALL leukemia-initiating cells without significantly delaying disease onset. Mechanistically, NHE1 knockdown reduced colony formation by more than 70% in T-ALL cells; transcriptomic and proteomic analyses showed decreased abundance of proteins involved in mitochondrial respiration and translation, and metabolomics revealed altered TCA cycle intermediates. In mouse xenografts, leukemia involvement in bone marrow and spleen was reduced, but overall survival did not differ significantly.
AI empowers scalable and programmable RNA design.
Functional RNA design is central to synthetic biology and RNA therapeutics. Recent AI advances leverage tertiary structure to interpret function and guide sequence generation, replacing traditional trial-and-error workflows. Experimental methods assisted by AI models continuously expand the structural data foundation, ultimately enabling scalable and programmable RNA design.
The landscape of gene loss and missense variation across the mammalian tree informs on gene essentiality
The authors developed GISMO and GISMO-mis, two evolutionary constraint metrics across 462 mammalian species, to complement assessments of gene essentiality. The two metrics provide complementary measures of gene essentiality and improve prioritization of human disease genes. The study was published in Nature Genetics, DOI 10.1038/s41588-026-02783-9, at https://www.nature.com/articles/s41588-026-02783-9.
Real-world, digital-first delivery of GLP-1 receptor agonist therapy in women.
A prospective observational study in 8,127 women in the UK examined initiation of GLP-1 receptor agonist therapy (99.8% tirzepatide) through a digital program incorporating mandatory biochemical monitoring, weekly video follow-up and clinician-guided dose titration.
Methods and resources
Engineered human Argonaute 2 for targeted C-to-U RNA base editing
Researchers fused human AGO2 with APOBEC3A and designed guide sequences that induce RNA circularization, creating the C-to-U RNA base editor ALTER. In an EGFP Y66H reporter system and at endogenous targets including PPIB, SMARCA4, ACTB and TYMS, ALTER-4 showed editing activity comparable to or higher than the dCas13-derived RESCUE-S and CURE-S, and lowering transfection dose reduced off-target editing by about 25-fold.
Regulatable in vivo gene expression via adaptamers
Bryant et al. developed compact RNA switches called adaptamers, which use an FDA-approved drug to activate endogenous RNA-editing mechanisms and control gene expression without introducing exogenous proteins. In mice, controllable expression of a metabolic hormone reversed obesity, offering a simpler route to drug-controllable gene therapy.
Multiancestry conditional and joint analysis applied to GWAS summary statistics
Manc-COJO is a multiancestry conditional and joint analysis method for GWAS summary statistics. It integrates data from multiple ancestries to identify independent genetic associations, reduces false positives from single-ancestry analysis, and supports fast, flexible conditional and joint analysis in diverse populations. The paper was published online in Nature Genetics on 20 October 2026, DOI: 10.1038/s41588-026-02799-1.
arxiv.orgrecAMPLIFY: Large-Scale Pretraining of Recursive Protein Language Models
The authors adapt recursive pretraining to masked protein language models with recAMPLIFY, trained on the same 2T tokens as AMPLIFY-350M. The model has 155M parameters, 56% fewer than AMPLIFY-350M, and improves CASP contact recovery and three of four supervised tasks, with fold accuracy up 22.5%.
arxiv.orgExplore or Confirm? LLM Agents Misallocate Measurements Under Noise
The authors introduce the Wellocate benchmark, testing five language-model agents on a generated landscape and two real protein datasets, GB1 and PhoQ, to see how they allocate a fixed measurement budget between exploring and confirming. Three agents over-confirmed, and one on the generated landscape over-trusted single readings. Giving the over-confirmers a list of untried candidates markedly improved the final picks of two models on real protein data, while deferring re-tests to a final round recovered only about a quarter of the loss from wrong picks.
Opinion and commentary
Will this sleep-disorder drug be the next big blockbuster?
Takeda's Orzeyful (oveporexton) became the first orexin receptor agonist approved by the FDA for narcolepsy, and many researchers think the class could expand beyond sleep disorders, much like GLP-1 drugs. The article traces orexin's independent discovery in the 1990s by teams at Scripps and UT Southwestern, and cites analysts forecasting a global narcolepsy drug market exceeding $6.4 billion by 2031.
China picks first researchers for 15-year mega grants
The National Natural Science Foundation of China has selected its first cohort of 44 young researchers, who will receive funding for up to 15 years under a '5+5+5' phased arrangement. The group includes 9 researchers from outside China; their names and projects have not been released, and no short-cycle, short-term target assessments will be conducted over the 15 years. The initial five-year grant is 4 million yuan (US$596,000), rising to 8 million yuan and then 16 million yuan in the second and third phases.
Also today
28 events that did not make today's daily, one table per category, one row per event. Open a category to expand.
RegulatoryChina's First Auditory Brainstem Implant Approved by NMPA · dostarlimab · Enable Injections Announces Approval in Japan for CirCLIQ® Injector based on enFuse® Onody Delivery · FDA Approves Once …5 ▾▴
Filings, acceptances, designations, approvals and withdrawals at a regulator.
- China's First Auditory Brainstem Implant Approved by NMPAUpdateChina's NMPA has approved the innovative device registration application for the auditory brainstem implant (ABI) from Zhejiang Nurotron Biotechnology. It is the first ABI developed in China and the country's first product to perform electrical stimulation and neural signal extraction in the brainstem region.NMPA
- dostarlimabUpdateThe FDA has granted accelerated approval to dostarlimab for previously untreated stage II/III MSI-H/dMMR locally advanced rectal cancer. Of 154 patients, 110 no longer required surgery, radiotherapy, or chemotherapy. The approval gives some patients who would otherwise need a permanent stoma an option to avoid surgery, radiotherapy, and chemotherapy.FDA
- Enable Injections Announces Approval in Japan for CirCLIQ® Injector based on enFuse® Onody Delivery Update
- FDA Approves OnceUpdateFDA
- FDA Approves Updated COVIDUpdateFDA
Clinicalbitopertin · Aulin Biotech Recombinant Staphylococcus aureus Vaccine Shows 73.25% Protective Efficacy in Late · emvistegrast · ABBV-466 …16 ▾▴
Trial starts, early and mid-stage data, and results that did not make the daily.
- bitopertinUpdateDisc Medicine (NASDAQ:IRON) announced that GlyT1 inhibitor bitopertin met both co-primary endpoints in the Phase III APOLLO trial in erythropoietic protoporphyria (EPP). Compared with placebo, whole-blood protoporphyrin IX (PPIX) levels fell 41.4% (p<0.0001) and average monthly pain-free light exposure time at month six increased 12.2 hours (p=0.001); shares rose nearly 40% after hours.FDA
- Aulin Biotech Recombinant Staphylococcus aureus Vaccine Shows 73.25% Protective Efficacy in LateUpdateAulin Biotech (688319.SH) says the final report of the late-stage clinical study of its recombinant Staphylococcus aureus vaccine shows protective efficacy of 73.25% against S. aureus infection within 42 days after closed fracture surgery. The vaccine completed late-stage unblinding in July 2026, and the company recently received the final report.
- emvistegrastUpdateGilead Sciences reported that in the Phase 2 SWIFT trial of emvistegrast in ulcerative colitis, the 200 mg group achieved a 12-week clinical response rate of 66% versus 37% for placebo. Clinical remission rates were 31% and 8%, respectively. The source calls it the first small-molecule alpha-4-beta-7 inhibitor to show efficacy in Phase 2, and Gilead plans to start Phase 3 in 2027.
- ABBV-466UpdateAbbVie presented TARGET-CD Phase 2 data for ABBV-466 as a late-breaking abstract at UEGW 2026: endoscopic remission at Week 12 was 30.2%, versus 21.3% for risankizumab monotherapy and 18.0% for trosunilimab monotherapy. At Week 24 the gap widened to 40.5% versus 22.4% and 20.0%.
- Visen Pharma Reports 182UpdateVisen Pharma reported 182-week long-term data from the phase 3 PaTHway China trial of palopegteriparatide. 95.6% of patients remained off conventional therapy, mean eGFR rose 13.6 mL/min/1.73 m² from baseline, and 75.7% of patients had an eGFR increase of at least 5 mL/min/1.73 m².
- Lilly's Mirikizumab Phase 3 SHINEUpdateLilly announced that the Phase 3 SHINE-2 study of mirikizumab in ulcerative colitis patients aged 6 to 17 met its primary endpoint of clinical remission. More than 80% of patients responded to induction therapy, and half of those responders achieved clinical remission at one year. Lilly plans to file the data with global regulators to support expanding mirikizumab's indication to pediatric ulcerative colitis.
- Hengrui's PDUpdateHengrui Medicine's PD-L1/TGF-βRII bispecific antibody fusion protein relafenlip alfa (SHR-1701), combined with CAPOX as first-line treatment for unresectable locally advanced or metastatic HER2-negative gastric/gastroesophageal junction adenocarcinoma, was published in the Journal of Clinical Oncology as the phase 3 RELIGHT study.
- efgartigimodUpdateargenx announced that Phase 3 ALKIVIA data for efgartigimod in autoimmune myositis will be presented as a late-breaking oral presentation (#LB24) at ACR Convergence 2026.
- TedopiUpdate
- 9MW2821Update
- pemvidutideUpdate
- pegcetacoplanUpdate
- A daily probiotic powder taken for six months improved memory and thinking scores in older adults wiUpdateA 24-week double-blind RCT in 87 participants from the Korean GARD cohort showed that the three-strain probiotic KL-P301 (Bifidobacterium animalis subsp. lactis, Limosilactobacillus fermentum, and Levilactobacillus brevis) significantly reduced plasma pTau181 (p<0.001) and improved CDR scores in older adults with early memory problems.
- Initial HIV Therapy for Adults and TreatmentUpdateA randomized clinical trial in treatment-naive adults with HIV compared a regimen of doravirine, lamivudine, and tenofovir disoproxil fumarate against a regimen of dolutegravir, emtricitabine, and tenofovir alafenamide for effects on weight gain and viral suppression. The current material is a summary-level description and does not report specific weight change or virologic suppression results.
- FabhaltaUpdate
- Weight Loss in Older Adults With Persistent Atrial FibrillationUpdate
Deals and financingRoche and Hansoh Strike ADC Strategic Collaboration and License Deal Worth Over $15.5 Billion · Merck KGaA to Acquire UK siRNA Company Silence Therapeutics for About $1.3 Billion in Cash · METiS Pharmaceuticals and Eli Lilly Strike AI · Jinbo Bio Completes 1 Billion Yuan Private Placement to Yangshengtang as Funds Verified …7 ▾▴
Licensing, M&A and financing that did not make the daily.
- Roche and Hansoh Strike ADC Strategic Collaboration and License Deal Worth Over $15.5 BillionUpdateHansoh Pharmaceutical and Roche entered a new ADC strategic collaboration and license agreement on October 19. Hansoh will receive a $400 million upfront payment and a $110 million one-year payment, with potential milestones exceeding $15 billion and a total deal value over $15.5 billion. The source says the deal sets a record for the highest potential total value of a China ADC out-licensing transaction. When Roche licensed Hansoh's CDH17 ADC HS-20110 a year ago, the upfront payment was $80 million; this upfront payment rose sharply.
- Merck KGaA to Acquire UK siRNA Company Silence Therapeutics for About $1.3 Billion in CashUpdateMerck KGaA of Darmstadt, Germany, announced on October 19 an all-cash acquisition of UK-based Silence Therapeutics for approximately $1.3 billion, offering $20 per ADS, a 130% premium over the October 16 closing price. The deal is expected to close in the first quarter of 2027, subject to shareholder and regulatory approvals.
- METiS Pharmaceuticals and Eli Lilly Strike AIUpdateMETiS Pharmaceuticals (7666.HK) announced on October 19, 2026 a strategic collaboration with Eli Lilly and Company on novel LNP delivery solutions. The agreement includes an upfront payment of over ten million US dollars and milestone payments of up to 1.1 billion US dollars. The collaboration is based on METiS's AiLNP platform under its NanoForge system, exploring tissue-specific delivery technologies suited to multiple therapeutic modalities.
- Jinbo Bio Completes 1 Billion Yuan Private Placement to Yangshengtang as Funds VerifiedUpdateJinbo Biochemical's roughly 1 billion yuan private placement of shares to Yangshengtang completed capital verification and payment on October 16. Yangshengtang also committed to buying at least another 1 billion yuan of shares on the secondary market within 18 months after the placement. The issue price was 277.22 yuan per share for 3.6072 million shares; the company earlier cut the fundraising cap from 2 billion yuan to 1 billion yuan, dropping a planned 850 million yuan for working capital, to focus proceeds on its humanized collagen FAST database R&D platform.
- Siemens Healthineers ← ParadUpdate
- TCGX ← TriGemXUpdate
- Immune Boost Capital ← OrthrUpdate