Biomedicine Daily · Saturday, 5 September 2026

[177Lu]Lu-PSMA-617 in patients with PSMA-positive metastatic androgen pathway modulator-naive/sensitive prostate cancer (PSMAddition): a phase 3 randomised, controlled trial.

Adding 177Lu-PSMA-617 to androgen deprivation therapy plus an androgen receptor pathway inhibitor reduced the relative risk of radiographic progression or death by 28% in PSMA-positive metastatic APMN/S prostate cancer, meeting the primary endpoint of prolonged radiographic progression-free survival. In the phase 3 PSMAddition trial, 572 patients per arm were randomised; radiographic progression or death occurred in 24% of the 177Lu-PSMA-617 arm versus 30% of the control arm. Grade 3 or worse adverse events occurred in 51% versus 43%, with dry mouth the most common event in the 177Lu-PSMA-617 arm.

Fatal IEC-HS cases halt eight Novartis Rap-cel autoimmune trials as BMS pauses Zola-cel enrollment

Three fatal cases of immune effector cell-associated hemophagocytic syndrome (IEC-HS) have led Novartis to halt all eight of its Rap-cel trials in autoimmune and neurological diseases. On the same day, August 31, 2026, BMS voluntarily paused enrollment of new patients for Zola-cel in autoimmune indications while it evaluates transient inflammatory adverse reactions seen in its trials.

Intravenous iron to treat anaemia before cardiac surgery (ITACS): international, double blind, placebo controlled randomised trial.

Ivonescimab Beats Pembrolizumab on Overall Survival in Phase 3 HARMONi-2

Ivonescimab, Akeso's PD-1/VEGF bispecific antibody, has beaten pembrolizumab on overall survival in the phase 3 HARMONi-2 trial, according to a company announcement on September 3. The predefined OS interim analysis passed review by the independent data monitoring committee, showing statistical significance and clinical benefit. Ivonescimab is the first bispecific antibody to outperform a PD-1 monoclonal antibody on OS in a phase 3 trial, in first-line PD-L1-positive (TPS ≥1%) locally advanced or metastatic NSCLC.

FDA Approves Revolution Medicines' Daraxonrasib for Metastatic Pancreatic Cancer

On August 26, 2026, the FDA approved Revolution Medicines' Rasonque (daraxonrasib, formerly RMC-6236) for adults with metastatic pancreatic adenocarcinoma who have received at least one prior line of systemic therapy. It is the first FDA-approved targeted therapy aimed at the active-state RAS(ON) protein family. The RAS(ON) molecular glue approval marks one of the biggest clinical advances in pancreatic cancer in nearly a decade.

STAT+: Ionis wins FDA approval for first drug for Alexander disease, a rare and deadly neurological disorder

The FDA approved Ionis Pharmaceuticals' Zanvastro as the first disease-modifying therapy for Alexander disease, a rare and deadly neurological condition, for use in children and adults. In a pivotal trial, walking speed stayed stable in treated patients while the control group declined 33%, and the data hinted that treating young children may improve motor function. The drug was generally safe, with serious adverse events more common in the control group.

Ionis’ ‘breakthrough’ Alexander disease approval establishes pillar of rare disease pipeline

Ionis has secured approval for its Alexander disease treatment, a decision the company sees as establishing a pillar of its rare disease pipeline. Alexander disease is a rare genetic condition; in the case described, a toddler diagnosed after seizures and MRI changes had no prior treatment option indicated. The approval positions Ionis to build further in rare diseases.

Ionis’ ‘breakthrough’ Alexander disease approval establishes pillar of rare disease pipeline

The FDA has approved Ionis' drug for Alexander disease, a rare and often fatal neurological condition, giving the company a pillar for its rare disease pipeline. The approval follows diagnoses like that of Elise Pearson, whose seizures and MRI changes led to a genetic confirmation of the disease at under three years old. Ionis now has an approved therapy in a indication with no prior treatment named in the news.

CAFC Upholds Exelixis Cabozantinib Crystalline Salt Patent Claims, Delaying MSN Cabometyx Generic to 2030

On August 31, 2026, the US Court of Appeals for the Federal Circuit affirmed a Delaware federal court judgment, holding that claims in Exelixis patents US 11,091,439, US 11,091,440 and US 11,098,015 covering the crystalline salt cabozantinib (L)-malate, its formulations and treatment methods meet the written description requirement of 35 U.S.C. §112(a). As a result, the earliest launch of MSN's generic of Cabometyx is pushed to 2030.

Menarini signs $771M deal to take Gan & Lee GLP-1 to European market

Menarini Group will pay Gan & Lee Pharmaceuticals €62 million ($72 million) up front under a deal worth up to $771 million to register and commercialize Gan & Lee's GLP-1 bofanglutide in Europe. The Italian company takes the Chinese insulin maker's drug to the European market. Registration and commercialization work under the collaboration follows.

Superluminal’s $60M series B to drive first clinical study: Finance Report

Superluminal Medicines has raised a $60 million series B round to support its first drug candidate's move into clinical testing. BVF Partners led the crossover syndicate, with Deep Track Capital and Perceptive Advisors joining as new investors and RA Capital Management, the seed and series A leader, participating. The funding is intended to carry the company's lead candidate into the clinic.

Excess intracellular lactate represses mRNA translation and tumor progression through lactate-tRNA charging.

Excess intracellular lactate represses mRNA translation because alanyl-tRNA synthetase 1 charges tRNAs with lactate instead of amino acids; slower lactyl transfer than alanine transfer modifies non-cognate tRNAs and broadly compromises elongation fidelity. Aggressive tumors elevate lactate transporters to limit intracellular lactate and evade this repression. Pharmacological blockade of monocarboxylate transporters restored lactate accumulation, re-established translational repression, and impaired tumor progression in mice.

Brønsted-basic small molecules restore GTP hydrolysis in KRAS-Q61 mutants

A UC Berkeley team led by Ziyang Zhang reports a strategy to reactivate GTP hydrolysis in KRAS-Q61 mutants rather than inhibit KRAS. Brønsted-basic small molecules deliver a basic group to the catalytic site to substitute for the mutated glutamine 61 residue, restoring hydrolysis and driving KRAS inactivation. The work appears in Nature Chemical Biology.

The dystonia-associated Torsin1A sustains CLCC1 function in membrane fusion of the nuclear envelope for NPC biogenesis.

Torsin1A, the AAA+ ATPase mutated in DYT1 dystonia, works by sustaining the function of CLCC1, a conserved membrane protein. In Drosophila germ cells and human cells, loss of Torsin or CLCC1 impaired nuclear envelope membrane fusion and nuclear pore complex biogenesis, and CLCC1 overexpression rescued the defects caused by Torsin loss. The findings point to modulating CLCC1 expression as a therapeutic prospect for DYT1 dystonia.

Rewiring the ribosome to translate proteins encoded in its own RNA.

Bacterial ribosomes can translate proteins encoded within their own RNA, shown by engineering a chimeric messenger-ribosomal RNA (mrRNA) in which a protein-coding sequence was appended to 16S rRNA. The mrRNA assembles into a small subunit forming Ribo-M, which translates mrRNA-encoded proteins; translation is abolished by small-subunit mutations or antibiotics, showing cis translation. Embedding mrRNA in a ribosome with tethered subunits produced Ribo-MT, uniting encoding, decoding and peptide synthesis in one RNA scaffold, with implications for orthogonal protein production and RNA world models.

Prediction of maternal and infant outcomes from longitudinal electronic health records with a Mother-Child AI agent

An LLM-based clinical assistant that orchestrates multiple tools, applied to sequential electronic health record data, can forecast maternal and infant conditions. The work suggests a window of opportunity to enhance risk-stratified care for mothers and infants, published in Nature Medicine.

Transferable genetic toolsets for nonmodel gut Clostridia enable in vivo reversible control of metabolite production.

Transferable genetic toolsets now work across diverse nonmodel gut Clostridia, combining strong constitutive promoters with an inducible, tunable promoter system and CRISPR-Cas gene deletion. Applied in mice, the system achieved targeted and reversible control of microbiota production of trimethylamine and deoxycholic acid, metabolites implicated in host lipid metabolism and disease. This enables causal linking of microbiota genes to host physiology and therapeutic engineering of the microbiota.

STAT+: After failure of Angelman syndrome drug, experts see hope in other experimental therapies

Ultragenyx reported earlier this week that its experimental drug for Angelman syndrome, a rare genetic disease causing severe developmental delays, failed a late-stage trial. Experts such as University of North Carolina researcher Mark Zylka say the outcome does not condemn the mechanism, and Ultragenyx, Ionis and Oak Hill Bio continue developing their own antisense treatments for the condition. Their programs could also inform genetic medicines for other neurological disorders involving intellectual disability.