Cogent Biosciences Reports Recent Business Highlights and Fourth Quarter and Full Year 2025 Financial Results
Cogent Biosciences announced that its lead drug bezuclastinib is on track for multiple FDA submissions in 2026, including NDAs for non‑advanced systemic mastocytosis (Non‑AdvSM), advanced SM (AdvSM) and second‑line GIST. The company reported $901 million in cash, sufficient to fund operations through 2028, while R&D and G&A expenses rose year‑over‑year, leading to a $328.9 million net loss for 2025. Positive Phase 3 data from the PEAK trial showed a 46% objective response rate and a median progression‑free survival of 16.5 months versus sunitinib alone. Cogent also highlighted pipeline progress on KRAS, ErbB2, PI3Kα and FGFR inhibitors.
DDW Highlights: 17 February 2026
In this 16‑minute DDW Highlights episode, host Bruno Quinney recaps five major drug‑discovery stories from the past week, focusing on a breakthrough study linking specific biomarkers to improved cancer survival rates and the launch of a multinational consortium tackling chronic...
FDA's New Move Undermines mRNA Flu Vaccine
https://open.substack.com/pub/pauloffit/p/weaponizing-the-fda-against-vaccines?utm_campaign=post-expanded-share&utm_medium=web A closer look at the FDA's recent kill shot against an mRNA influenza vaccine.
MAHA: Pseudoscientific Scheme Undermining Medicine for Profit
As I’ve written @BakerInstitute MAHA = a system of pseudoscience designed to undermine scientific biomedicine in America for the benefit and profits of the wellness influencer industry. It’s incredibly corrupt and endangers the health of Americans. Vaccines are just the...
Sequential Optimization of Multivariate Metal–Organic Framework Based Biocatalysis
The study introduces a sequential Latin hypercube sampling‑coupled Bayesian optimization (LHS‑BO) workflow that simultaneously designs multivariate zirconium‑based enzyme‑@‑MOF (E‑MOF) biocomposites and tunes the downstream glucose oxidase–horseradish peroxidase (GOx‑HRP) cascade. Optimized E‑MOFs ZG67 and ZH16 deliver encapsulation efficiencies above 90%, retain...
Bispecific Autoimmune Therapies Set to Transform 2024
"Dual Wielding in I&I: A Pivotal Year Ahead" - new blog - Atlas EIR Cody Tranbarger with a tour de force on the bispecific opportunities in autoimmune... https://t.co/StQJX0vgfx
Dual Wielding in I&I: A Pivotal Year Ahead
The article argues that the next breakthrough in immunology and inflammation (I&I) therapeutics will come from multi‑drug and multi‑target strategies rather than single‑target antibodies, which have hit an efficacy ceiling. It traces the evolution from early blockbuster biologics like Humira...
STAT+: Ocular’s Experimental Eye Drug Beats Low Dose of Regeneron’s Eylea in Late-Stage Trial
Ocular Therapeutix announced that its experimental drug Axpaxli met the primary endpoint in a Phase 3 trial for wet age‑related macular degeneration, maintaining vision in 74% of patients at nine months and 66% at one year after a single injection....
Size‐Transformable Supramolecular Nanoprodrugs Enable Redox Imbalance Amplification and Cholesterol Modulation to Boost Multidimensional Tumor Immunotherapy
Researchers have engineered a tumor‑targeting, tumor‑microenvironment‑responsive supramolecular nanoprodrug that switches size to enhance deep tumor penetration. The nanoprodrug simultaneously induces redox imbalance—accumulating reactive nitrogen species and depleting glutathione—to amplify ferroptosis, while depleting cholesterol to rejuvenate exhausted T cells. This multidimensional...
PH‐Responsive Nanoparticle‐Coated Calcium Phosphate Granules for Bone Cancer Therapy
Researchers have engineered β‑tricalcium phosphate (β‑TCP) granules coated with selenium‑doped mesoporous silica nanoparticles (SeMIA) linked via pH‑responsive imine–alendronate bonds. The imine linkers remain stable at physiological pH but cleave in the mildly acidic osteosarcoma microenvironment, releasing nanoparticles that selectively kill...
STAT+: Compass Says Its Psilocybin Drug Helped Patients with Severe Depression in Two Trials
Compass Pathways announced that its synthetic psilocybin therapy, COMP360, achieved statistically significant improvements in two identical Phase 3 trials for severe major depressive disorder. Both studies met primary endpoints, showing greater reduction in depression scores than placebo‑controlled psychotherapy. The data suggest...
Katerina Leftheris
Enhanced Genomics announced the appointment of Katerina Leftheris to its board of directors, tapping her extensive medicinal chemistry and machine‑learning drug‑discovery experience. Leftheris most recently served as Chief Scientific Officer at Vilya, a company focused on oral macrocycle therapeutics, and...
Haelixa Embeds DNA Authentication Into Products
Haelixa, a Swiss deep‑tech firm, introduced a DNA‑based Authenticity Service that embeds nano‑sized, brand‑unique DNA markers into luxury items such as watches, jewellery and leather accessories. The invisible marker survives resale, repair and returns, and can be verified on‑site with...
Selecting the Optimal Cell Therapy Manufacturing Platform
Developers of cell and gene therapies must choose between fully integrated turnkey platforms and modular, multi‑device systems. Integrated solutions deliver rapid time‑to‑value and simplified workflows, ideal for low‑volume autologous products, but they restrict reagent flexibility and scale. Modular platforms provide...
How to Evaluate Prospective Diagnostic Laboratory Partners for Your Patient Support Program
The pharmaceutical industry now invests over $5 billion annually in patient support programs (PSPs), which boost adherence by roughly 30 % and lower overall healthcare costs. Diagnostic laboratory testing is a cornerstone of these programs, delivering timely data that guides dosing, monitors...
CStone Secures FDA Clearance to Begin CS2009 Phase II Trial
CStone Pharmaceuticals has secured FDA clearance for its investigational new drug application, allowing the launch of a Phase II trial of CS2009, a trispecific antibody that simultaneously targets PD‑1, VEGFA and CTLA‑4. The global study uses a parallel, multi‑cohort expansion design...
ExCellThera Secures German NUB Status 1 Listing for Zemcelpro
ExCellThera and its subsidiary Cordex Biologics have secured a Status 1 listing under Germany’s NUB programme for their cell therapy Zemcelpro (dorocubicel). The therapy, which received conditional marketing authorisation from the European Commission, targets adults with haematological malignancies lacking suitable donor...
STAT+: As China’s Drug Industry Races Ahead, Its GLP-1 Race Is Accelerating Too
China’s pharmaceutical sector is rapidly expanding, now generating its own GLP‑1 drugs alongside imported treatments. Novo Nordisk reported a 5% decline in Ozempic sales in China last year, the first dip since its 2021 launch, while sales grew elsewhere. The...
GSK
#GSK Exdensur (depemokimab) approved in EU for treating "severe asthma with type 2 inflammation and chronic rhinosinusitis with nasal polyps". Follows from the SWIFT and ANCHOR phase III trials.
Biopharma Must Cope With The Fetal Bovine Serum Squeeze
Biopharma faces a tightening fetal bovine serum (FBS) market as U.S. cattle inventories hit record lows and European disease outbreaks curb supply. Prices have surged more than 300% over five years and release timelines have lengthened from weeks to months...
Red Light Therapy Shows Promise for Treating Brain Injuries
A University of Utah Health study examined red‑light photobiomodulation (PBM) as a preventive therapy for repetitive head acceleration events in collegiate football. Twenty‑six Division I athletes were randomized to active or sham transcranial and intranasal PBM over 16 weeks, receiving three...
Rare Disease Month Developments, Part 1 – The Good: RPD PRV Program Renewed, FDA Rare Disease Hub’s 2026 Strategic Agenda...
Congress renewed the Rare Pediatric Disease Priority Review Voucher (RPD PRV) program, extending it to September 30 2029 and eliminating the dual sunset dates. The FDA released its Rare Disease Innovation Hub’s 2026 Strategic Agenda, allocating $1 million in funding and outlining plans...
Plasmid Injection Triggers Immune Attack on Cancer Survival Protein
What if a physician could inject you with a plasmid that would induce an immune response against a protein needed for cancer cell survival? This novel strategy is being tested and looks promising for refractory cancers. Talking Biotech 488 ...
Exploring the Impact of Cognitive Profiles on the Response to Magnetic Seizure Therapy and Electroconvulsive Therapy
An open‑label, non‑randomized trial compared magnetic seizure therapy (MST) and electroconvulsive therapy (ECT) in 398 patients with major depressive disorder (MDD) or schizophrenia (SCZ). Both modalities produced comparable symptom reductions, but MST yielded faster re‑orientation and less decline in processing...
Protective Mutations Associated with APOE in Alzheimer’s Disease
Recent research has identified several rare APOE mutations that confer protection against Alzheimer’s disease, including the R136S variant, the APOE3‑Christchurch (R136S) allele, loss‑of‑function CASP7, and missense changes V236E and R251G. These variants mitigate tau pathology, improve amyloid clearance, and dampen...
MRNA-Packed Nanoparticles Restore Fertility in Genetically Infertile Mice and Produce Live Offspring
Researchers at Shanghai Jiao Tong University engineered a lipid nanoparticle (LNP) formulation that delivers therapeutic mRNA directly to spermatocytes in mice. By injecting mRNA encoding the wild‑type Msh5 gene, they transiently restored meiosis in mice with a genetic block, achieving...
Pre‑market Rallies Set for Tomorrow’s Data Releases
Two Major Data Readouts Coming Tomorrow: $OCUL ripped in after-hours, +26% to ~$11.16, clear positioning into retina data. $CMPS has been quietly bid into its Phase 3 TRD readout, steady accumulation with mild pre-market strength. Both leaning long into the print. Now it’s...
Free, Ad‑free Biomed Insights Reaching 200k Globally
Ground Truths are free essays and podcasts on the hot topics in biomedicine and health. Mostly things the media doesn't cover or adequately highlight. No ads or sponsors. Join ~200,000 subscribers from 212 countries https://t.co/ZgnO2Y0P4L
FDA Approval of Sibeprenlimab Signals New Era in IgA Nephropathy: Jackson Peter Kim, MD
The FDA granted accelerated approval to sibeprenlimab‑szsi (Voyxact) for primary IgA nephropathy, marking the first APRIL‑inhibiting monoclonal antibody for this condition. Interim results from the phase 3 VISIONARY trial showed a 50 % reduction in proteinuria at nine months versus 2 % with...
People Are Still Working on the Senolytic Peptide FOXO4-DRI
FOXO4‑DRI, a peptide that blocks the FOXO4‑p53 interaction, continues to be explored as a senolytic therapy. Recent preclinical work shows that injecting the peptide into aged and progeroid mice reduces endothelial cell senescence and improves aortic function. Companies such as...
FM Nirmala Sitharaman to Push Biopharma, Semiconductor Missions in Norway
Finance Minister Nirmala Sitharaman arrived in Oslo to promote India’s Biopharma SHAKTI programme and Semiconductor Missions 2.0, seeking long‑term capital from Norwegian partners. She will meet Prime Minister Jonas Gahr Store, Finance Minister Jens Stoltenberg, and startup leaders at Oslo Science Park to showcase a ₹10,000‑crore,...
Vaccine Makers Curtail Research and Cut Jobs
Federal policies driven by Health Secretary Robert F. Kennedy Jr. are creating a hostile environment for vaccine developers, prompting companies like Moderna to scale back late‑stage studies. A Texas startup canceled a new manufacturing plant, and a San Diego firm...
Silencing Growth Hormone Has Strong Effects in Mouse Brains
Researchers engineered mice lacking growth hormone receptors specifically in adipose tissue (Ad‑GHRKO) and observed striking brain benefits in aged males. Compared with control mice, the Ad‑GHRKO group showed increased neuronal activity, reduced neuroinflammation, lower tau phosphorylation, and fewer senescence markers....
B‑Cell Depletion: Rebooting Immunity to Cure Autoimmunity
"Immune Reset" Rebooting the immune system by depletion of B cells, like a reboot of a computer, to achieve cures vs autoimmune diseases https://t.co/253NtyFmCN
3D Fragments vs the Histamine H1 Receptor
Researchers at Vrije Universiteit Amsterdam built an 80‑compound, three‑dimensional fragment library and screened it against the histamine H1 receptor. Only one fragment (1a) showed activity, but iterative optimization yielded the low‑nanomolar antagonist VUF26691 with picomolar cellular potency. The campaign required...
Women Show Higher P‑tau217 Levels and Tau Deposition
p-tau217 is a breakthrough blood test for risk of developing Alzheimer's disease. Important to know there are sex differences for this biomarker, with women having higher values at baseline and more tau deposition. @JAMANeuro https://t.co/Xrr8SjsiVY https://t.co/eyDt6mgSqR
Psychedelic Shows Rapid Depression Relief in Small Trial
A psychedelic quickly reduced depression (in patients with major depression disorder) in a small, double-blind placebo randomized trial @NatureMedicine https://t.co/vvug72VGBi https://t.co/Tn58rP0ufS
2025 Q4 Job Market Report: Positive Signs Emerge for Job Seekers
The fourth quarter of 2025 marked the first quarter‑over‑quarter rise in biopharma job postings, with a 10% increase and a 4% uptick in average listings, despite a 14% year‑over‑year decline. Science/research and development roles grew 14% while clinical positions surged...
Making a MASH Hit: PNPLA3 and the Rise of Genotype-Driven Therapies
The lipid serine hydrolase PNPLA3, especially its I148M mutant, has emerged as a genetically validated driver of MASLD/MASH, prompting a wave of genotype‑focused drug programs. RNA‑based modalities—Arrowhead’s GalNAc‑siRNA ARO‑PNPLA3 and AstraZeneca/Ionis’ GalNAc‑ASO AZD2693—are in clinical trials aiming to lower mutant...
Why some Extracellular Vesicles Work Better: A Safer Path for Protein and Gene Delivery
Researchers compared two major extracellular vesicle (EV) biogenesis pathways and found that vesicles generated from cell‑surface protrusions via the I‑BAR protein MIM deliver functional proteins and genome‑editing enzymes far more efficiently than conventional endosome‑derived, CD63‑associated EVs. Using live‑cell imaging, they...
LLM-Edited Radiology Reports Boost Patient Understanding, Preserve Accuracy
When the LLM rewrites the radiology report, patient understanding is increased and clinical accuracy is maintained. @LancetDigitalH @curtlanglotz @smrabd https://t.co/q2UHebBQG7 https://t.co/IwZAx59brY
Respecting the Meticulous Skill Behind Biolab Hardware Design
Started my day with a peer review duty for Hardware X. Another cool build landed on my desk and it's delightful to see folks really take the minutiae and nuance of designing a tool seriously. People often make wild assumptions...
ML‑Predicted Insulin Resistance Identified as Risk Factor in 12 Cancers
Researchers at the University of Tokyo applied a machine‑learning tool, AI‑IR, to estimate insulin resistance in half a million UK Biobank participants. The analysis revealed insulin resistance as a significant risk factor for twelve distinct cancer types, providing the first...
AI Subtyping Boosts HR+/HER2‑ Breast Cancer Therapy
Precision treatment with artificial intelligence assisted subtyping enhances therapeutic efficacy in HR+/HER2− breast cancer: The LINUXtrial https://t.co/UF4Lt3g5QK https://t.co/LWuJYD2qGh
Essential Bioinformatics Curriculum: Top Courses to Master
10 courses for my dream bioinformatics curriculum: 1. Unix Commands with Greg Wilson https://t.co/83VHvTqbMt 2. statistics and R with Rafael Irizarry https://t.co/ofYSIiChoW https://t.co/1GogvjAbAA
Roche Trial Offers Hope to Patients with Rare Kidney Disease
Roche announced that its anti‑CD20 antibody Gazyva (obinutuzumab) met the primary endpoint in the phase 3 MAJESTY trial for primary membranous nephropathy (pMN). The study showed significantly higher complete remission rates at two years compared with the immunosuppressant tacrolimus, while maintaining...
‘Just Agree to It:’ Pazdur Said He Was Told To Cosign FDA’s Reduced Trial Requirements
Richard Pazdur, longtime FDA oncology leader, resigned after being pressured to endorse a controversial policy that would reduce required drug approval trials from two to one. He said Commissioner Marty Makary breached the traditional independence between the commissioner’s office and...
Increased O-GlcNAc Transferase Expression as an Approach to Improving Function in the Aging Brain
Age‑related decline in O‑GlcNAc Transferase (OGT) activity contributes to neurodegenerative disorders such as Alzheimer’s, Parkinson’s and ALS. Traditional approaches aim to raise O‑GlcNAc levels by inhibiting O‑GlcNAcase, but recent research highlights transcriptional control of OGT as a more direct therapeutic...
Novartis CEO Vasant Narasimhan on Transforming a 250-Year-Old Company
In this episode, a16z partner Jorge Conde interviews Novartis CEO Vasant Narasimhan about reshaping the 250‑year‑old conglomerate into a focused medicines company, a move he estimates will unlock $180 billion of value. Narasimhan outlines Novartis’s three platform pillars—cell and gene therapies,...
Inflammatory Glycogen Produced by Gut Microbes Contributes to Neurodegeneration
Researchers have identified inflammatory glycogen produced by gut microbes as a driver of age‑related neurodegeneration, especially in ALS and frontotemporal dementia linked to C9ORF72 mutations. In germ‑free mice lacking C9ORF72, colonization with glycogen‑producing Parabacteroides merdae triggered monocytosis, blood‑brain barrier breakdown,...