Biomedicine Daily · Thursday, 15 October 2026
CBA FRONTIER INSIGHTS
FDA Approves First Therapy for BCG-Unresponsive Papillary-Only Bladder Cancer
The FDA expanded approval of Anktiva (nogapendekin alfa inbakicept-pmln), given with BCG, to adults with BCG-unresponsive, high-grade papillary-only non-muscle invasive bladder cancer, making it the first FDA-approved treatment for that disease. The decision drew on evidence from Anktiva's earlier carcinoma in situ approval, where a trial showed a majority of adults achieved a complete response and many maintained it for at least 12 months. The agency is finalizing draft guidance that would let developers use CIS study evidence to support papillary-only NMIBC approvals. The same day's coverage includes pirtobrutinib's NMPA and FDA first-line CLL/SLL approvals, new DESTINY-Breast11 residual-burden data for T-DXd-THP in HER2-positive early breast cancer, and Samsung Biologics' roughly $1.8 billion PolyPeptide tender offer clearing its key hurdle with 87.59% of shares tendered.
Clinical readouts and safety
medicalxpress.comTriple-action drug retatrutide delivers major weight loss for people with diabetes, Phase III trial finds
DESTINY-Breast11 RCB Analysis Shows T-DXd-THP Cuts Residual Burden in High-Risk HER2-Positive Early Breast Cancer
In the phase 3 DESTINY-Breast11 trial in high-risk HER2-positive early breast cancer, neoadjuvant T-DXd followed by paclitaxel, trastuzumab and pertuzumab (T-DXd-THP) achieved a pCR rate of 67.3% versus 56.3% with dose-dense doxorubicin-cyclophosphamide followed by THP, and a prespecified RCB analysis presented at ESMO BC 2026 showed an RCB-0+I rate of 81.3% versus 69.1%. The RCB index distribution shifted toward lower residual burden (Q3/Q4 boundary 1.01 vs 1.38, P=0.0059), and among patients not reaching pCR the median RCB was 1.5 versus 1.7. The findings add pathologic-response detail beyond the binary pCR endpoint to guide postoperative adjuvant decisions in an anthracycline-free regimen.
medicalxpress.comTargeted radioactive therapy may help delay spread of recurrent prostate cancer
Adding two cycles of the PSMA-targeted radioligand 177Lu-PNT2002 before SBRT cut the risk of progression or needing additional hormone therapy by 62% versus SBRT alone in 92 men with oligorecurrent hormone-sensitive prostate cancer, followed for a median of 31.9 months. At 24 months, 33% of the combination group had no detectable disease versus 12% with SBRT alone, and fewer developed widespread disease or castration-resistant prostate cancer. The phase 2 LUNAR results suggest treating microscopic disease while delaying hormone therapy.
Intermittent or Continuous Renal-Replacement Therapy in Acute Kidney Injury
Talazoparib plus enzalutamide in high-volume metastatic hormone-naive prostate cancer: a randomized phase 2 trial
Adding the PARP inhibitor talazoparib to enzalutamide improved prostate-specific antigen response in patients with high-volume metastatic hormone-sensitive prostate cancer in the randomized phase 2 ZZ-FIRST trial. Spatial transcriptomic analysis of paired biopsies showed that androgen receptor inhibition suppresses homologous recombination repair-related programs, and this suppression correlated with proliferation arrest rather than functional HRR deficiency.
medicalxpress.comScientists identify clues to making immunotherapy more effective against glioblastoma
Neoadjuvant nivolumab plus ipilimumab extended median survival to about 13.5 months versus roughly nine months with nivolumab alone in a phase 2 trial of 63 patients with recurrent glioblastoma. Five patients, about 15% of the combination group, became long-term survivors, with none on nivolumab alone. Tumor T-cell density independently predicted overall survival, and early pseudoprogression on MRI correlated with longer survival, pointing to biomarkers for patient selection.
medicalxpress.comDrug tapering noninferior to treatment continuation for rheumatoid arthritis with low disease activity
Structured tapering of biologic or targeted synthetic DMARDs was noninferior to continuation for rheumatoid arthritis patients with sustained low disease activity in a 24-week randomized trial of 171 patients assigned to tapering and 177 to continuation. Low disease activity was maintained in 88.2% of the tapering group versus 89.6% of the continuation group, a risk difference of −1.43 percentage points (95% CI, −8.9 to 5.23), meeting the noninferiority criterion. Most patients who worsened regained low disease activity after protocol-guided treatment re-escalation.
Verastem reports ‘robust’ response rate in pancreatic cancer subgroup where Rasonque struggled
VS-7375, an oral selective KRAS G12D dual ON/OFF inhibitor, produced a 50% confirmed ORR in second-line metastatic pancreatic ductal adenocarcinoma at 900 mg daily, versus 24% for Rasonque in G12D patients in the Phase 3 RASolute 302 trial. In third- and fourth-line mPDAC the confirmed ORR was 36%, with 43% confirmed ORR in NSCLC at 600 mg and 33% unconfirmed ORR in third-line colorectal cancer combined with anti-EGFR therapy. Discontinuation due to adverse events was 2% across the 295-patient TARGET-D 101 trial, and Verastem will start a Phase 3 first-line mPDAC trial by year-end.
Regulatory and policy
FDA Approves First Treatment for Certain People With BCG-Unresponsive Bladder Cancer
The FDA expanded approval of Anktiva (nogapendekin alfa inbakicept-pmln), given with BCG, to adults with BCG-unresponsive, high-grade papillary-only non-muscle invasive bladder cancer, the first FDA-approved treatment for that disease. The decision relied on evidence from Anktiva's earlier approval for carcinoma in situ, where a trial showed a majority of adults achieved a complete response and many maintained it for at least 12 months. The FDA is finalizing draft guidance to let developers use CIS study evidence for papillary-only NMIBC approvals.
gov.ukMHRA authorises Onswik (insulin efsitora alfa) for adults with type 2 diabetes
NMPA and FDA Approve Pirtobrutinib for First-Line CLL/SLL Treatment
China's NMPA approved pirtobrutinib on September 28, 2026 for adult patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) across all lines, including those untreated. On October 2, 2026, the FDA followed with approval for previously untreated adults with CLL/SLL and no known 17p deletion. Pirtobrutinib is a highly selective, non-covalent (reversible) BTK inhibitor that inhibits both wild-type BTK and BTK carrying the C481 mutation without forming a covalent bond at C481.
Rare Disease Framework
The FDA, with the Reagan-Udall Foundation for the FDA, will hold a hybrid public meeting on October 28, 2026, to gather input on its Rare Disease Framework Initiative, which aims to reimagine the agency's approach to rare disease drug development and review. The framework would tailor regulatory advice and decision-making to the evidence reasonably attainable for each disease, embed the patient voice at defined milestones, leverage external expertise, and apply risk-based approaches to manufacturing review and inspections. Changes could come through regulations, guidance documents and internal processes; FDA set out its aims in a white paper released October 14, 2026.
medicalxpress.comWHO experts recommend HPV shot for girls aged 4–8
WHO's Strategic Advisory Group of Experts on Immunisation (SAGE) has recommended that countries consider routine HPV vaccination for girls as young as 4 if it boosts uptake, after confirming a single-dose schedule for girls aged 9 to 14 as the most efficient route to the 90% coverage target. Global HPV vaccine coverage stands at 33% across 165 introducing countries, while more than 95 million girls had been vaccinated by the end of 2025. SAGE also set priority groups for expanding single-dose vaccination: catch-up for women up to 25, then up to 30, routine vaccination of boys aged 9 to 14, and catch-up for males up to 20.
STAT+: Lobbyists and trade groups fight to shape how states regulate medical AI
The Federation of State Medical Boards has convened a 22-member AI workgroup, including executives from the American Medical Association, American Hospital Association, Anthropic and Elevance Health, which met in Washington, D.C., in early October. The group is drafting guidelines to help state medical boards regulate AI tools that perform clinical functions with limited or no direct physician supervision. A draft could be made public in weeks to months, open for feedback before formal adoption, and could form the basis of state legislation.
STAT+: Trump administration delays new rule that could restrict imported medicines from Canada and overseas
The Trump administration delayed by 90 days a federal rule that could stop Americans from buying prescription drugs from licensed international pharmacies in Canada and overseas. The rule, which was to take effect Oct. 22, targets the de minimis exemption that lets shipments valued under $800 pass through customs more easily, as part of a crackdown on illegal goods such as opioids. Patient advocacy groups and dozens of congressional lawmakers had urged a rethink; the delayed effective date is the next milestone.
Industry and deals
Samsung Biologics' $1.8 Billion PolyPeptide Takeover Clears Acceptance Hurdle
Samsung Biologics' all-cash tender offer for the Swiss peptide CDMO PolyPeptide Group AG at CHF 44.31 per share, valuing it at about CHF 1.46 billion (roughly $1.8 billion), cleared its key hurdle when 87.59% of shares were tendered by the October 12 deadline. The deal, the largest ever in Korea's pharma and biotech industry, extends Samsung Biologics into peptides including GLP-1 services, backed by PolyPeptide's six GMP plants. Definitive interim results come October 16, with an additional acceptance period through October 30 and completion expected by end of 2026, followed by a squeeze-out and delisting from the Swiss exchange.
Gilead Pays $1.875 Billion for Exclusive License to Vir's Three Dual-Masked Solid Tumor TCEs
Vir Biotechnology has granted Gilead an exclusive license to develop and commercialize three novel PRO-XTEN dual-masked T cell engagers (TCEs) targeting selected solid tumor antigens, under a collaboration and license agreement announced October 14 in San Francisco. The deal is worth $1.875 billion. The companies will work to discover and design the three development candidates.
Zealand pens up to $1.2B deal with Generate for AI-designed obesity, metabolic drugs
Zealand Pharma will pay Generate:Biomedicines $30 million upfront, up to $35 million in early preclinical milestones and nearly $1.2 billion in development, regulatory and commercial milestones, plus tiered royalties, to access Generate's generative AI platform for designing protein therapeutics for obesity and metabolic diseases. Generate handles research through candidate selection; Zealand leads clinical development, regulatory and commercialization. The deal follows mixed Zealand readouts for petrelintide and survodutide.
How consumer tech company Whoop is building for Medicare patients
Whoop joined Medicare's 10-year ACCESS pilot, offering its wearable, a 12-month app subscription, and clinician support at no cost to traditional Medicare patients 65 and up with high blood pressure, high cholesterol, obesity, or prediabetes. Chief medical officer Ami Bhatt, speaking at the STAT Summit in Boston, said the company must improve measures such as blood pressure, hemoglobin A1C, and LDL cholesterol to receive full payment, and is working with the National Council on Aging and AARP on personalization, with app simplification first on the roadmap.
‘Pharma bro’ Shkreli runs afoul of court’s lifetime pharma ban
The FTC has asked a judge to hold Martin Shkreli in contempt for allegedly violating his lifetime pharma industry ban, in place since 2022, by trading pharma stocks and commenting on company management via livestreams on YouTube, X and Reddit. The complaint cites trades and commentary involving dozens of companies including Cassava Sciences, Capricor Therapeutics, Amgen and Eli Lilly. The FTC wants Shkreli to divest his shares, pay a fine equal to his profits and face escalating penalties for further violations.
Research papers
Engineered TYK2 dependence enables myeloid-selective IL-10 receptor agonism
IL-10's therapeutic use is limited because its myeloid-suppressive and T-cell-activating effects cannot be separated. Using the JAK1 inhibitor VVD-118313 in bone-marrow-derived macrophages, the authors show that JAK1-JAK1 transphosphorylation only partially reduces IL-10-induced STAT3 phosphorylation, with TYK2-mediated transphosphorylation compensating; in TYK2-deficient BMDMs, VVD-118313 fully abrogated STAT3 phosphorylation and IL-10 function. In human monocytes, the TYK2 inhibitor zasocitinib similarly partially reduced IL-10-driven STAT3 phosphorylation, revealing differential TYK2 dependence that may enable myeloid-selective IL-10 receptor agonism.
Capillary-selective vasodilation through myosin inhibition halts ischaemic cascades
Myosin inhibition produces vasodilation that is selective for capillaries and halts the cascades that follow ischaemia, as reported in this work on microvascular obstruction. The approach targets the capillary level, where pericyte-mediated constriction has been linked to no-reflow after ischaemia in the brain, heart and kidney. No quantitative outcome data are reported here.
A microexon regulatory code reveals autism-linked genetic variation
An expanded map of neuronal microexons and a machine learning model of microexon splicing reveal rare human genetic variation predicted to converge on a subset of microexons in genes linked to nervous system biology and neurodevelopmental disorders. Analysing 4,790 bulk RNA-seq samples and 47,380 single nuclei from human cortex, the authors detected 1,514 previously unannotated microexons, 292 of them differentially spliced between neural and non-neural cells. The work points to non-coding variants as a possible contributor to the roughly 85% of autism cases with no identified genetic cause.
Modelling mouse embryogenesis from chemically induced totipotent stem cells
A mouse embryo model built from chemically induced totipotent-like stem cells progresses through key developmental milestones, including gastrulation, embryonic axis formation, haematopoiesis and early organogenesis. The work extends embryo modelling to a starting cell type with totipotent-like properties. It provides a system for studying development from the earliest stages onward.
Sexual dimorphism in epidermal stem cell dynamics
Long-term intravital imaging of ear skin epidermis shows the interfollicular epidermis is not maintained by a single equipotent compartment: basal cells pass through an intermediate transit-amplifying-like state with limited proliferative potential before differentiating. Tracking labelled clones over 5 months, combined with single-cell expression profiling and biophysical modelling, reveals stochastic but locally coordinated stem cell renewal. Sister-cell events show temporal coordination strongly enriched in female mice, and functional experiments tie this to sex hormone regulation of basal cell dynamics.
Resident Macrophages Feed Pancreatic Cancer Selenium Transporter to Evade Ferroptosis
Resident tissue macrophages in pancreatic cancer supply a selenium transporter protein to tumor cells, protecting them from ferroptosis and supporting an invasive EMT state, a study from Bai Yong Shen and Florent Ginhoux at Shanghai Jiao Tong University's Ruijin Hospital published in Cell shows. The work identifies RTM-Selenop as an axis of metabolic support in the tumor microenvironment and positions Mrc1 as a reliable marker distinguishing resident tissue macrophages from monocyte-derived macrophages, which have different transcriptional profiles.
SPARCS Screen of About 70 Million Cells Recovers Macroautophagy Genes and Golgi Control of STING Signaling
SPARCS, a spatially resolved CRISPR screening method, identifies abnormal cells from microscopy images, recovers them, and traces the associated genes and protein changes. Applied to roughly 70 million cells, it recovered nearly all core macroautophagy genes and uncovered regulation of STING signaling by Golgi pH state, revealing mechanisms conventional screens tend to miss.
Structural basis of ligand recognition and gating in mosquito IRs
Cryo-EM structures of the IR8a-IR75k carboxylic acid receptor from the malaria mosquito Anopheles stephensi, captured in four states including apo, ligand-bound open, and nonanoic acid- and octanoic acid-bound forms, reveal how ligand specificity is set by alkyl chain length and carboxylate geometry. Ligand binding closes IR75k's bilobed ligand binding domain, and the change propagates through conserved mechanical linkers to dilate the pore, a mechanism corroborated by molecular dynamics simulations and electrophysiology. The work provides an atomic-resolution view of mosquito host detection and a basis for rational design of mosquito control strategies targeting host-seeking behavior.
Intrinsic physicochemical activity at microplastic–water interfaces
Commodity microplastics are not chemically inert at water interfaces: they spontaneously generate reactive oxygen species. This intrinsic physicochemical activity challenges the prevailing paradigm of plastic inertness and carries implications for human health and the environment. The work reframes microplastics as active chemical participants rather than passive contaminants.
medicalxpress.comBigger data reveal the second, third and fourth switches controlling a gene
Analysis of 14,324 RNA sequencing samples from TOPMed yields tens of thousands of high-resolution e/sQTL signals, showing that most genes carry multiple independent regulatory switches whose second, third and fourth controls elude smaller datasets. Of 10,611 GWAS signals linked to a specific gene, roughly two-thirds involved such secondary switches. About one in six whole-blood signals came from variants rare or absent in European ancestry, and only 1.3% appear in the four large European-based eQTL resources.
Artificial intelligence-enabled clinical phenotyping of human brown fat
AI-enabled quantification of brown adipose tissue (BAT) activity in humans links higher BAT activity to a healthier metabolic profile and lower rates of psychiatric disease. The same approach identified existing pharmacologic agents that could be repurposed to activate BAT in humans. The work provides a scalable way to phenotype BAT and connect it to clinical outcomes and drug targets.
Citron kinase activity controls alternative splicing events that drive prostate cancer growth.
Citron (CIT) kinase drives prostate cancer growth by phosphorylating the RNA splicing regulator THRAP3, controlling its binding to pre-messenger RNA targets and producing transcripts that stimulate growth of prostate cancer cell lines, xenografts, patient-derived organoids, and PDX-derived organoids. An antisense oligonucleotide blocking THRAP3 recruitment to a CIT-dependent RNA binding site prevented production of these transcripts and inhibited growth of cell lines and PDX-derived organoids across prostate cancer stages and lineages, but not of benign cells. The work identifies CIT-mediated alternative splicing as a therapeutic strategy in treatment-resistant prostate cancer.
ALIX-dependent small extracellular vesicles from tissue-resident mesenchymal cells regulate pancreatic β cell proliferation in obesity.
Deleting ALIX in PDGFRα+ mesenchymal progenitors of mice blocks small extracellular vesicle production and disrupts metabolism. In high-fat diet-induced obesity, these knockout mice gained less weight and had smaller subcutaneous adipocytes, yet showed impaired glucose tolerance with reduced pancreatic β cell area and smaller islets despite normal insulin sensitivity. Conditioned-media sEVs from knockout mesenchymal stem cells lost the ability to promote β cell proliferation, identifying mesenchymal sEVs as regulators of β cell proliferation in obesity.
Engrafting human cortical organoids.
Xenocortication, a transplantation approach described by Kaganovsky and colleagues, integrates human cortical organoids into the mouse brain by grafting human induced pluripotent stem cell-derived cortical organoids into the cortical cavity of neonatal immunodeficient 'apallial' mice, in which most of the neocortex and hippocampus had been genetically depleted. Magnetic resonance imaging showed the grafts grew approximately 4.7-fold between 2 and 3 months after transplantation and accounted for nearly 92% of the combined cortical tissue volume. The approach addresses limited intracranial space and interactions with existing neural circuits that restrict organoid growth and integration after transplantation into rodents.
Methods and resources
De novo design of cysteine proteases
Designed cysteine proteases from scratch using a computational design pipeline built on RFdiffusion2 active-site scaffolding, ProteinMPNN sequence design, and AlphaFold 3 prediction. Candidate enzymes were characterized by crystallography, biochemical kinetic assays, and mammalian-cell imaging, extending the de novo enzyme design approach to the cysteine protease class.
Computational design of metalloproteases
RFdiffusion was used to design proteins de novo with water-activating zinc-based active sites positioned next to a peptide substrate binding pocket. The resulting proteases were highly active and programmable, with the authors pointing to potential applications in therapeutics, biotechnology and bioremediation.
MAGIC: an international network for evaluating generative artificial intelligence in global health.
The authors establish MAGIC, an international network for evaluating generative artificial intelligence in global health. The network brings together collaborators across institutions in Asia, Europe, Africa and the Americas, including groups at Duke-NUS Medical School, Harvard, Tsinghua and the World Health Organization Regional Office for South-East Asia, with a focus on the ethics, regulation and standardization of AI for health.
youtube.comMartin Steinegger interview: organizing the protein universe with AI structure prediction at billion scale
In a CSHL interview, AlphaFold2 and ColabFold co-author Martin Steinegger describes organizing the protein universe at scale using AI-predicted 3D structures rather than sequences. AlphaFold DB holds about 240 million structures and BioHub has released 1.1 billion metagenomic predictions; clustering the universe by domain combinations yields roughly 2 to 3 million groups for finding distant homologs, functional motifs and enzymes.
Opinion and commentary
As White House shifts focus toward AI and tech, U.S. science leaders defend role of the American university
MIT enrolled nearly 500 fewer graduate students this fall, a 20% drop from 2024, after a $100 million cut in federal research awards, President Sally Kornbluth said at the STAT Summit in Boston. She and Science journals editor-in-chief Holden Thorp defended the 80-year-old model of federally funded university research as the Trump administration shifts priorities toward AI and technology, backed by $6 billion in new national research investments. Forecasts put China on track to surpass U.S. research spending in two to three years, with nine of the top 10 Nature Index institutions now in China.
Editorial Expression of Concern: Thyroid hormone inhibits lung fibrosis in mice by improving epithelial mitochondrial function
Nature Medicine editors issued an Editorial Expression of Concern on 14 October 2026 about a 2017 paper reporting that thyroid hormone inhibits lung fibrosis in mice by improving epithelial mitochondrial function. The concerns involve similar-looking blots in Figs. 3n and 4a and overlapping images in Fig. 5e. Authors located one correct blot and replicated Fig. 5e data, but could not locate the original data for Fig. 4a; several authors agree with the expression, Farida Ahangari does not, and the rest have not responded.
Also today
22 events that did not make today's daily, one table per category, one row per event. Open a category to expand.
ClinicalBNT113 · Admilparant · ARO-MAPT · Enhanced CAR T …11 ▾▴
Trial starts, early and mid-stage data, and results that did not make the daily.
- BNT113UpdateBioNTech's BNT113 combined with pembrolizumab (Keytruda) failed to reach progression-free survival, a co-primary endpoint, in the phase II/III AHEAD-MERIT trial (NCT04534205) in HPV16-positive recurrent or metastatic head and neck squamous cell carcinoma. The independent data monitoring committee recommended continuing the study pending final analysis of the other co-primary endpoint, overall survival. The miss comes nine months after the vaccine drew high industry expectations.
- AdmilparantUpdateBMS has announced top-line results from a Phase 3 trial of Admilparant in idiopathic pulmonary fibrosis (IPF), with safety presented as the main highlight. The disease affects about 14 million people worldwide, carries a median survival of 3 to 5 years after diagnosis and a five-year survival rate of about 45%, and has been treated for decades with only pirfenidone and nintedanib. No efficacy figures were included in the announcement.
- ARO-MAPTUpdateA subcutaneously administered RNA interference drug, Arrowhead's ARO-MAPT, crossed into the brain of healthy volunteers and sharply reduced tau, a protein implicated in Alzheimer's disease. In a trial of about 64 people, the highest dose, 12.8 mg/kg on days one and 15, cut tau by an average of 73% at day 105, with reductions as high as 85%. Arrowhead said the medicine could be injected once every three months, and the results support a platform for delivering RNA drugs past the blood-brain barrier.
- Enhanced CAR TUpdate
- leniolisibUpdateLong-term leniolisib was generally well tolerated in 37 patients with activated phosphoinositide 3-kinase delta syndrome treated for a median of 4.3 years. Annual infection rates (P = 0.0005), respiratory infection rates (P = 0.0017) and immunoglobulin replacement therapy use (P = 0.0095) all declined per year of treatment, with clinically meaningful gains in 36-Item Short Form Survey general health scores. This extends the efficacy and safety evidence for leniolisib in APDS well beyond the pivotal trials.
- DuracyteUpdateDuracyte, a Rice University spinout based in Houston, is developing subcutaneous implants of engineered human cells that continuously secrete biologics in the body for up to a year, with the aim of replacing repeated infusions and injections. The work has drawn roughly $120 million in non-dilutive funding. The company plans to start its first clinical trial in the first half of next year, testing an implant that produces the checkpoint inhibitor ipilimumab in patients with peritoneal cancer.
- VRB-101 (ecnoglutide)UpdateVerdiva Bio has discontinued a Phase 2 trial of oral ecnoglutide (VRB-101) as a maintenance therapy for overweight and obesity, a business decision not based on safety, with eight patients enrolled at cancellation. The trial had enrolled patients with at least 10% weight loss after six months of subcutaneous semaglutide or tirzepatide. Verdiva, which licensed the drug from Sciwind Biosciences for $70 million upfront plus over $2.4 billion in milestones, will continue developing VRB-101 for other weight-loss indications, as monotherapy and with its two amylin analogs. In a separate 163-patient Phase 2 head-to-head trial in China, subcutaneous ecnoglutide produced 12.8% average weight loss at 20 weeks versus 9.5% for semaglutide.
- FDA Clears BailiUpdateThe FDA has cleared a global Phase III trial (NCT07869836) of Baili-Bio's T-Bren, the candidate's first global multicentre Phase III study. SystImmune Inc., the company's wholly owned overseas subsidiary, is the global lead sponsor and handles protocol design, site selection, data management, drug supply and regulatory communications. It is Baili-Bio's second self-developed, independently initiated and led global Phase III trial; T-Bren is a core pipeline asset to which the company holds full worldwide rights.FDA
- New study examines GLPUpdate
- J&J’s disappointing Q3, RevMed’s subgroup data, BMS’ ‘mixed’ results, Caribou’s cutbackUpdate
- NiktimvoUpdate
Deals and financingRoche licenses Rona Therapeutics' ALK7 RNAi candidate for obesity in deal worth up to $1.16 billion · Soufflé Therapeutics · Pfizer pens $500M Enodia pact to join Lilly in emerging protein degrader field · J&J earnings set ‘negative tone’; Shkreli vs. the FTC …6 ▾▴
Licensing, M&A and financing that did not make the daily.
- Roche licenses Rona Therapeutics' ALK7 RNAi candidate for obesity in deal worth up to $1.16 billionUpdateRona Therapeutics granted Roche an exclusive global license on October 13, 2026 to an RNAi candidate targeting ALK7 (ACVR1C), developed on its APOLLO extra-hepatic delivery platform, for the treatment of obesity and metabolic diseases. The deal carries potential total payments of up to $1.16 billion plus tiered royalties on global sales. Roche takes on worldwide development and commercialization rights.
- Soufflé TherapeuticsUpdateBoston-based RNA therapy company Soufflé Therapeutics closed an oversubscribed $135 million Series B round on October 13, led by Juvia Group with new investors including TCG Crossover Management, BB Biotech, Fidelity Management & Research Company, Balyasny Asset Management and a sovereign wealth fund. The funds will support its cell-specific RNA delivery platform and pipeline development as most oligonucleotide drugs remain limited to liver targeting. The company aims to move its RNA-targeted delivery therapies toward the clinic.
- Pfizer pens $500M Enodia pact to join Lilly in emerging protein degrader fieldUpdate
- J&J earnings set ‘negative tone’; Shkreli vs. the FTCUpdate
- Kaléo ← BARDAUpdate
- Stryker pens deal with CMR Surgical to advance roboticUpdate
OtherFDA Approves Teva's WELTRUZA, a Once · BMS Seeks Chinese Approval of Mavacamten in Pediatric Obstructive Hypertrophic Cardiomyopathy with P · GSK Plans Cuts to More Than Twelve Hundred Jobs Across Belgian Production Sites · Argatroban Injection …5 ▾▴
Everything else with a concrete event that did not make the daily.
- FDA Approves Teva's WELTRUZA, a OnceUpdateThe US FDA has approved Teva's WELTRUZA (olanzapine) extended-release injectable suspension for treating adults with schizophrenia. It is the first and currently only olanzapine long-acting injectable given as a once-monthly subcutaneous injection for that indication, requiring no loading dose, no oral supplementation and no post-injection monitoring. The approval rests on the pivotal SOLARIS study.FDA
- BMS Seeks Chinese Approval of Mavacamten in Pediatric Obstructive Hypertrophic Cardiomyopathy with PUpdateBristol Myers Squibb has filed a marketing application in China for mavacamten capsules in pediatric obstructive hypertrophic cardiomyopathy, submitted on October 10 under registration category 2.4 in five strengths of 1mg, 2.5mg, 5mg, 10mg and 15mg. The CDE has placed the application in its priority review procedure. Mavacamten is described as the first and only approved cardiac myosin inhibitor, and the filing would extend its use beyond adults to younger patients.NMPA
- GSK Plans Cuts to More Than Twelve Hundred Jobs Across Belgian Production SitesUpdateGSK said on Wednesday that it plans a restructuring across its production sites in Belgium that could affect more than twelve hundred employees in total. The company informed unions and a special works council of the plan, according to its statement and a report in Belgium's L'Echo, and the unions have not yet responded publicly. Consultations over the restructuring are the next step.
- Argatroban InjectionUpdatePar Health USA is voluntarily recalling 22 lots of Argatroban Injection 250 mg/2.5 mL (100 mg/mL) to the hospital/user level after particulate matter identified as cellulose, polystyrene, polyethylene terephthalate, stopper material and possibly stainless steel was detected. The affected lots, distributed nationwide to wholesalers and hospitals between December 30, 2024 and May 5, 2026, carry a risk of pulmonary emboli, blood vessel occlusion and vein wall inflammation. No adverse events have been reported; distributors and hospital pharmacies should stop use and return inventory through Inmar, Inc.FDA
- PhRMAUpdatePhRMA has sued the US Department of Health and Human Services and CMS and their leaders in Washington, DC, asking a court to block the Medicare GLOBAL programme, which CMS plans to implement in 2027 to apply most-favored-nation pricing in Medicare Part B. PhRMA seeks to void all of the programme's provisions, even though as few as four companies may ultimately be affected. CMS announced the plan as a high-profile drug-pricing commitment aimed at older voters.