Biomedicine Daily · Wednesday, 23 September 2026

CagriSema Beats Tirzepatide in Phase III and Meets Primary Endpoint in REDEFINE 9

Novo Nordisk's CagriSema beat tirzepatide 5 mg head-to-head in the phase III REIMAGINE 5 study in adults with type 2 diabetes whose blood glucose remained inadequately controlled on metformin or an SGLT2 inhibitor. The parallel phase III REDEFINE 9 study in people with obesity or overweight met its primary endpoint. If approved, CagriSema would be the first GLP-1 plus amylin combination therapy worldwide.

Amgen's Dazukibart Succeeds in Phase 3 Trial for Systemic Sjögren's Syndrome

Amgen's dazukibart met its primary goal in a Phase 3 trial in moderate-to-severe systemic Sjögren's syndrome, delivering a statistically significant and clinically meaningful improvement in systemic disease activity. The topline result supports the drug as a potential new, highly differentiated treatment option for patients with the debilitating autoimmune disease.

Trastuzumab plus nivolumab with gemcitabine and cisplatin as first-line therapy for HER2-positive unresectable biliary tract cancer: a phase 1b/2 trial

A phase 1b/2 trial tested trastuzumab combined with nivolumab, gemcitabine and cisplatin as first-line therapy for HER2-positive unresectable biliary tract cancer. The regimen showed encouraging clinical response rates, and HER2 expression was associated with clinical benefit. The results support HER2 status as a marker guiding trastuzumab-based treatment in this population.

Performance and safety of a multi-cancer early detection test: the PATHFINDER 2 study

The PATHFINDER 2 study evaluated the safety and performance of a blood-based multi-cancer early detection test in an intended-use population of more than 35,000 participants aged 50 years or older. It reports outcomes on how the test performs when used prospectively in people without a prior cancer diagnosis. The findings add interventional evidence on safety and performance in the intended-use setting.

Celldex monoclonal antibody rebounds with pair of phase 3 wins for chronic skin condition

Stereotactic Body Radiotherapy vs Moderately Hypofractionated IMRT for Localized Intermediate-Risk Prostate Cancer Research Summary

A trial compared stereotactic body radiotherapy (SBRT) with moderately hypofractionated intensity-modulated radiation therapy (MH-IMRT) in patients with localized intermediate-risk prostate cancer, measuring urinary and bowel quality of life and disease-free survival. The study was designed to test whether SBRT is superior to MH-IMRT on these endpoints. No results are reported here.

FDA Approves Ionis Antisense Drug Zanvastro as First Alexander Disease Therapy

The FDA approved Ionis Pharmaceuticals' Zanvastro (zilganersen, development code ION373), an antisense oligonucleotide, for treating Alexander disease in children and adults. It is the first FDA-approved therapy for the disease and the first disease-modifying therapy directly targeting accumulation of the causal protein. The approval came on September 3, 2026, nearly three weeks ahead of its PDUFA date of September 22, 2026.

STAT+: HHS unveils new initiatives to reduce animal testing in drug development

For the second time this year, HHS announced strategies to shift drug development toward human-based research methods and reduce animal testing. The FDA issued a final rule allowing alternatives to animal testing when 'appropriate' for determining the safety of drugs and biologics, replacing terms like 'animal tests' with 'nonclinical tests.' The NIH will provide $88 million for 10 infrastructure projects encouraging methods such as cell-based assays, organs-on-a-chip, and computer models.

Tariff guidance leaves companies in the dark

The Trump administration released a Federal Register notice spelling out which drugs qualify for exemptions from tariffs on patented pharmaceuticals, one week before the tariffs are scheduled to take effect. The notice leaves critical questions about the exemption process unanswered. The Department of Commerce, seeking emergency approval from the Office of Management and Budget to collect exemption requests, acknowledged that an inability to issue decisions before the 100 percent tariff rate takes effect creates uncertainty for companies' tariff liability.

Iambic maintains momentum from string of Big Pharma partnerships by aiming for IPO

Iambic Therapeutics, a San Diego AI drug discovery biotech, has filed with the SEC to go public after raising a $100 million series B in 2023 and a similar round in November 2025. IPO proceeds would chiefly fund IAM1363, an oral brain-penetrant HER2 inhibitor in a phase 1 study in advanced HER2-altered solid tumors, with a registrational trial planned for early 2027. The company had $207.9 million in the bank at the end of June and partnerships including a Takeda deal worth up to $1.7 billion.

RAF1 scaffold integrity shapes chemogenetic degradation outcomes in KRAS-driven lung cancer models

Pharmacological degradation of RAF1 via a dTAG-RAF1 mouse model proved efficient and systemically well tolerated but, unlike genetic Raf1 ablation, did not produce tumor regression in KRAS-driven lung adenadenocarcinoma. The N-terminal FKBP12F36V tag perturbed the RAF1 interactome, including scaffold links to apoptotic regulators, blunting the degradation phenotype. Scaffold integrity therefore determines chemogenetic fidelity, and degraders need functional validation, not just target elimination.

Targeting Polθ Helicase with RP-3467: Preclinical Validation and Clinical Response in a gBRCA1 ovarian cancer patient with an acquired PARPi resistance mutation in TP53BP1

RP-3467, a selective small-molecule inhibitor of DNA polymerase theta, suppresses Polθ enzymatic activity, abrogates microhomology-mediated end joining, and induces synthetic lethality in homologous recombination-deficient models. Combined with PARP inhibitors olaparib or rucaparib, it produced sustained tumor regression across multiple xenograft and patient-derived xenograft models without added systemic toxicity. In a phase 1 trial (NCT06560632), a heavily pretreated germline BRCA1-mutant ovarian cancer patient with a TP53BP1 loss-of-function mutation achieved a confirmed partial response to RP-3467 plus olaparib by RECIST v1.1.

Genomic foundation models extend clinicopathologic and transcriptomic prognostication in soft tissue sarcoma

Evo2, a genomic foundation model, was used to embed short variants, fusions and rearrangements from 102 patients with soft tissue sarcoma and 129 TCGA-SARC cases into patient-level genomic representations for Cox survival modelling. Adding Evo2 to SARCULATOR raised the OS C-index from 0.620 to 0.770 in the study cohort and from 0.595 to 0.716 in TCGA-SARC; in TCGA-SARC it also improved CINSARC-based OS prediction from 0.476 to 0.653. These descriptive point estimates support altered tumor sequence context as an independent prognostic layer for sarcoma risk stratification.

Large-scale esophageal cancer screening through noncontrast computed tomography and artificial intelligence

Esophageal cancer detection through noncontrast computed tomography is enhanced by a new artificial intelligence tool called Esophageal AI-Guided malignant Lesion Evaluation. In a large-scale study, the tool achieved high sensitivity and specificity across diverse settings.

Blood proteomics of menopause map to brain aging and dementia risk

Menopause is associated with hormone-linked molecular changes in blood proteomics that correlate with poorer cognitive outcomes later in life. Analysis of proteomic data from nearly 15,000 women across several cohorts identified molecular signatures that map to brain aging and dementia risk, and these signatures might inform selection of therapeutic targets to improve brain health.

The novel transcripts we keep rediscovering

A Nature Biotechnology commentary argues that transcript annotations should not be split into 'known' versus 'novel' categories. It proposes instead a multidimensional framework for interpreting transcript diversity that weighs technical credibility, contextual prevalence, reproducibility, molecular consequence and phenotypic relevance. The piece was published online on 22 September 2026.