Biomedicine Daily · Tuesday, 22 September 2026
CBA FRONTIER INSIGHTS
Galleri's 140,000-Person Trial Fails to Cut Late-Stage Cancers
Grail's Galleri multicancer detection test missed its primary endpoint of reducing late-stage cancers in a UK trial that randomly assigned 140,000 residents to testing plus usual care or usual care alone, a setback for the goal of a single blood test to catch most cancers. Other developments: the FDA approved Bayer's finerenone for chronic kidney disease tied to type 1 diabetes, the first new option there in more than 30 years; Beacon's laru-zova became the first gene therapy to hit a Phase III primary endpoint in X-linked retinitis pigmentosa, with 31% of higher-dose patients gaining 15 or more letters on the LLVA scale; and Telix agreed to acquire ITM Isotope Technologies Munich in a $1.65 billion radiopharma merger with up to $700 million in contingent payouts.
Clinical readouts and safety
Hansoh TYK2 Inhibitor HS-10374 Meets Primary Endpoints in Phase III Plaque Psoriasis Trial
Hansoh Pharmaceutical's TYK2 selective allosteric inhibitor HS-10374 met the co-primary endpoints and multiple secondary endpoints in a pivotal Phase III trial in adults with moderate-to-severe plaque psoriasis. The randomized, double-blind, placebo-controlled, multicenter trial evaluated the efficacy and safety of HS-10374 in this population. Hansoh plans to file the drug for market approval.
Beacon edges ahead in XLRP gene therapy race with Phase III hit
Laruparetigene zovaparvovec (laru-zova) from Beacon Therapeutics is the first gene therapy to meet the primary endpoint in a Phase III X-linked retinitis pigmentosa trial, improving visual acuity on the low-luminescence visual acuity (LLVA) scale in the VISTA study. Responders gaining 15 letters or more included 31% of patients in the higher-dose cohort and 24.1% in the lower-dose cohort. The result raises the prospect of preventing or delaying blindness in XLRP patients.
Hansoh TYK2 Inhibitor HS-10374 Meets Co-Primary Endpoints in Phase III Psoriasis Trial
Hansoh Pharmaceutical's selective allosteric TYK2 inhibitor HS-10374 met both co-primary endpoints and multiple secondary endpoints in a pivotal Phase III trial in adults with moderate-to-severe plaque psoriasis. At week 16, the proportion of HS-10374 patients achieving PASI 75 and the proportion achieving sPGA 0/1 with at least a 2-point reduction from baseline were both significantly greater than with placebo, adding a new candidate for psoriasis treatment.
First-line FMT resolves immune checkpoint inhibitor colitis in days, MD Anderson team finds
First-line fecal microbiota transplantation (FMT) rapidly relieved immune checkpoint inhibitor-related diarrhea and colitis in a cohort from MD Anderson Cancer Center: symptom improvement began at a median of 1.5 days and 76.9% of patients achieved clinical remission. More than eight in ten patients were able to resume their anticancer therapy. The approach positions FMT ahead of high-dose steroids and biologics as initial treatment for this immune toxicity.
Oxygen vs Air at Birth for Moderate- to Late-Preterm Infants: The AIROPLANE Cluster Randomized Crossover Trial.
Starting resuscitation of 32-to-35-week neonates with Fio2 0.30 instead of 0.21 did not change the primary outcome: ongoing respiratory support when leaving the delivery room occurred in 72.6% vs 73.3% (risk difference, -0.83). In the cluster randomized crossover trial across 26 Australian maternity hospitals, 1818 newborns were enrolled, and Fio2 0.30 was associated with less escalation of delivery room support and less endotracheal ventilation beyond the delivery room (6.4% vs 9.3%).
J&J firms up Caplyta's 1st phase 3 win in bipolar-associated manic episodes
Caplyta, Johnson & Johnson's atypical antipsychotic acquired through its $14.6 billion purchase of Intra-Cellular Therapies, has passed a phase 3 trial in manic episodes associated with bipolar disorder. The result moves the drug one step closer to a broader psychiatric label, building on already strong sales. No efficacy figures were reported in the announcement.
STAT+: Alkermes’ orexin agonist shows potential to treat ADHD in early-stage trial
Alkermes' orexin agonist ALKS 7290 has shown for the first time that this drug class may benefit people with ADHD. In a randomized Phase 1 study, adults with moderate to severe symptoms, starting at a median of 39 on the Adult ADHD Investigator Symptom Rating Scale, took 50 milligrams daily; after two weeks their scores fell by 19 points, to mild symptoms, and the drug was well tolerated.
Regulatory and policy
FDA Approves Bayer's Finerenone for Type 1 Diabetes-Related Chronic Kidney Disease
On September 17, 2026, Bayer announced that the FDA approved finerenone (Kerendia) for chronic kidney disease associated with type 1 diabetes in adults. It is the third US indication for finerenone and the first new FDA-approved treatment option in more than 30 years for CKD related to type 1 diabetes, a setting where treatment options have long been limited, adding drug therapy evidence targeting kidney progression risk.
NMPA Approves Suzhou Zhonghua's Vweilening Sacubitril/Valsartan 100 mg Tablets
Suzhou Zhonghua Pharmaceutical's Weilening, a 100 mg sacubitril/valsartan sodium tablet, has won NMPA approval in China for adults with chronic heart failure with reduced ejection fraction (NYHA class II-IV, LVEF of 40% or less). The company positions the dual-target drug as addressing an unmet need in standard treatment for heart failure, the end stage of cardiovascular disease.
FDA Updates Regulations to Advance Innovative Alternatives to Animal Testing
The FDA issued a direct final rule on September 21, 2026 clarifying that non-animal methods, such as human cells, organs-on-chips and computer models, can be used where appropriate for safety testing of human drugs and biological products before trials in people. The rule replaces terms like "animal tests" with "nonclinical tests" and "nonclinical studies," as defined under FDORA 2022, and does not eliminate animal studies or change evidentiary standards. The FDA also launched a database with 25 examples of New Approach Methodologies from its review materials, and the public may comment; significant adverse comments would lead the agency to withdraw the rule and proceed via a companion proposed rule.
PDUFA VIII aims to remove CMC bottlenecks, shore up FDA staffing
FDA and industry have negotiated a new PDUFA agreement, dated September 21, 2026, intended to speed patient access to new drugs and cut costs for developers and the agency by raising the share approved in a single review cycle. The deal targets CMC bottlenecks and FDA staffing, leaves past performance goals unchanged and adopts incremental process reforms. Negotiated while FDA was under political and institutional stress, it includes largely symbolic America First provisions.
IntraBio's Aqneursa scores expansion, now the first treatment for rare ataxia
White House plans to put NIH grants under political control: reports
President Donald Trump's senior officials met in the Oval Office on Friday to discuss plans for an executive order creating an external panel with the power to reject National Institutes of Health grants. The move would tighten White House control over NIH funding decisions. Next step is whether the executive order is issued as discussed.
STAT+: Lilly’s breast cancer combo wins approval
The FDA approved Eli Lilly's combination drug treatment for patients with an advanced form of breast cancer, reported Sept. 21, 2026. The decision gives Lilly a new option in the advanced breast cancer setting. Full details of the approval are available to STAT+ subscribers.
Industry and deals
STAT+: Radiopharma companies merge after one receives surprise FDA rejection
Telix Pharmaceuticals will acquire all shares of ITM Isotope Technologies Munich SE in a merger worth at least $1.65 billion, with up to $700 million more payable to ITM shareholders if ITM's lead drug meets regulatory and sales goals. The deal combines two radiopharmaceutical companies and creates a well-resourced player in a field dominated by Novartis, which launched two radioactive isotope cancer treatments in 2018 and 2022.
Telix to Acquire Germany's ITM Isotope Technologies Munich for About $1.65 Billion
Telix Pharmaceuticals has agreed to acquire Germany-based ITM Isotope Technologies Munich SE for about $1.65 billion on a cash-free, debt-free basis, according to a September 21 Reuters report. ITM shareholders are expected to receive about $1.25 billion in Telix shares priced at $11.84 per share. After completion, existing Telix shareholders will hold about 76.3% of the combined company and ITM shareholders about 23.7%.
Analysts ‘extremely bullish’ as Telix beams into Novartis’ territory with $1.6B ITM acquisition
Telix-ITM merger aims to create radiopharma juggernaut: Deals Report
Telix Pharmaceuticals is acquiring German radiopharma company ITM Isotope Technologies Munich in an all-stock deal valuing ITM at $1.65 billion on a cash- and debt-free basis. ITM shareholders will receive about $1.25 billion in Telix shares and are eligible for $700 million in additional payouts. The combined company gains global scale, manufacturing capabilities and an oncology product that could soon reach the market.
Research papers
Liquid biopsy for early detection of pancreatic ductal adenocarcinoma.
A blood-based microRNA assay combined with CA19-9, called PANXEON, detected early-stage pancreatic ductal adenocarcinoma in a prospective multicenter study of 1,785 individuals from four countries. The miRNA signature reached an AUC of 88.6% and sensitivity of 83.8% for early-stage PDAC; combined with CA19-9, sensitivity was 86.8% for stage I-II disease, with false-positive rates of 3.2% in low-risk and 15.6% in high-risk controls. It also detected high-grade dysplasia in 64.3% of individuals with high-risk pancreatic cysts.
A Causal Multi-modal AI Model Stratifies Residual Risk and Identifies Candidates for Treatment Escalation in Node-Positive HR+/HER2- Early Breast Cancer
Ataraxis Breast CTX, a causal multi-modal AI model combining clinical variables with H&E histopathology, stratified residual recurrence risk in 556 node-positive HR+/HER2- breast cancer patients from the UNIRAD trial. Five-year disease-free survival was 93% in the low-risk group versus 80% in the high-risk group, and CTX-benefit predicted everolimus benefit in an exploratory multivariable analysis (interaction p = 0.01). The model may inform selection of patients for adjuvant treatment escalation.
Decidual-Like Natural Killer Cells Are Enriched in Melanomas and Acquire CD9 Through Melanoma Cell Contact
Melanomas harbor decidual-like NK cells with a CD56 Bright CD16 - CD49a + CD9 + phenotype, a population rare or absent in peripheral blood from patients with melanoma and healthy donors. The cells appear in tumors across sex, melanoma subtype, and disease site, and tumor CD9 expression correlated with intratumoral dl-NK frequency (Pearson rho = 0.40, p = 0.056). Co-culture showed direct contact with CD9-expressing melanoma cells induces CD9 acquisition by NK cells, suggesting an immunologic parallel between melanoma and the maternal-fetal interface.
Gut-immune-PVAT axis is involved in ethanol-induced abdominal aortic dysfunction via IL-17RA, TLR4, and FPR1 signaling
Chronic plus binge ethanol exposure in male C57BL/6J mice caused leaky gut, systemic inflammation, and abdominal aortic and perivascular adipose tissue (PVAT) dysfunction, linked to microbiota-derived fMLP and LPS, M1 macrophage polarization, Th17 expansion, and FPR1 upregulation. Smooth muscle FPR1 deletion prevented the vascular hyporesponsiveness, and the PVAT-dependent alterations were absent in germ-free, Rag1-/-, IL-17RA-/-, or TLR4-/- mice. The work positions PVAT as a central integrator of gut microbiota-derived signals in alcohol-related vascular injury.
Weighing the Odds: Body Mass Index and Recurrence-Free Survival in Early-Onset Colorectal Cancer
BMI at diagnosis shows a U-shaped association with recurrence-free survival in early-onset colorectal cancer, with the lowest hazard at BMI 27 and the highest at BMI below 20 or above 30 (LRT p=0.02). In 1,459 curative-intent surgery patients, no such association appeared in average-onset colorectal cancer (LRT p=0.15), and the cohorts differed significantly (LRT p=0.046). The findings suggest the obesity paradox may be specific to EOCRC.
Limited added benefit of seasonal influenza vaccination before A(H5) vaccination in mice and ferrets challenged with A(H5N1)
Seasonal influenza vaccination given before A(H5) vaccination did not reliably improve protection against clade 2.3.4.4b A(H5N1) in mice and ferrets. Seasonal vaccine induced antibodies to seasonal haemagglutinins but none detectable against A(H5), and did not consistently boost A(H5)-directed antibody responses. Priming improved survival in one of three lethal mouse challenge experiments, with no effect in the other two or in ferrets.
Incremental Predictive Value and Representation of Transcriptomic Features for Immunotherapy Response in Advanced Urothelial Carcinoma
Knowledge-guided tumor microenvironment gene-set features outperform data-driven transcriptome-wide gene selection for predicting atezolizumab response in advanced urothelial carcinoma. In 298 IMvigor210 patients, they improved AUPRC by 0.058 over clinical variables, but gains shrank to +0.011 beyond TMB and PD-L1, and data-driven selection was unstable (Jaccard 0.227) with AUPRC 0.150 lower. The pattern replicated in an external cohort (+0.128 vs +0.019).
Opinion and commentary
[Correspondence] Multicancer detection tests: holy grail or cautionary tale?
Grail's Galleri trial, which randomly assigned 140,000 UK residents to usual care plus multicancer detection testing or usual care alone, failed to meet its primary endpoint of reducing late-stage cancers. Results were presented at the ASCO scientific meeting in June 2026. The outcome sets back the aspirational goal of a single blood test able to identify most cancers.
A consensus-based guideline for personalized bacteriophage therapy
Nature Medicine has published a consensus-based guideline, online on 21 September 2026, giving practice-oriented recommendations for safe and standardized clinical use of personalized bacteriophage therapy. The guideline is intended to guide clinicians applying phage therapy tailored to individual patients. Adoption in clinical practice follows publication.