Immutep Investors Spooked by LAG-3 Failure in Lung Cancer
Australian biotech Immutep saw its ASX shares tumble more than 88% after the independent data monitoring committee recommended halting its pivotal phase‑3 TACTI‑004 trial of the LAG‑3 inhibitor eftilagimod alfa (efti) in first‑line non‑small cell lung cancer. The trial, which combined efti with Merck’s Keytruda and chemotherapy, was stopped for futility despite earlier promising data in other indications. The setback follows a $20 million upfront licensing deal with Dr Reddy’s and threatens the company’s cash runway, though reserves now appear sufficient through 2027. The failure also dampens broader market confidence in LAG‑3‑targeted immunotherapies, which have seen mixed results from peers such as Bristol‑Myers Squibb and MSD.
Otsuka Pharmaceutical Reports OLE Study Data on Repinatrabit in Phenylketonuria
Otsuka Pharmaceutical disclosed early open‑label extension (OLE) data for repinatrabit (JNT‑517) in adolescents with phenylketonuria (PKU). A 75 mg twice‑daily regimen achieved a 67% mean reduction in blood phenylalanine by day 56, with responses observed across prior sapropterin responders, non‑responders, and a...
Eliquis Proves Safer than Xarelto for Patients with Deep Blood Clots
A head‑to‑head trial published in the New England Journal of Medicine compared Eliquis (apixaban) and Xarelto (rivaroxaban) in patients with deep‑vein thrombosis. The study found Eliquis significantly reduced major bleeding risk while maintaining equal efficacy in preventing clot recurrence. This...
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HEALTH INEQUITY: Red Cross Children’s Hospital Doctor Using Donated ‘Miracle’ Cystic Fibrosis Drug to Save Lives
South Africa’s Red Cross Children’s Hospital is using donated Trikafta, a $300,000‑a‑year cystic fibrosis therapy, to keep seven‑year‑old Jaylin Leitjies alive after his lungs were severely damaged in infancy. The drug, produced by Vertex Pharmaceuticals, is not registered with SAHPRA...
Market‑First Synthetic Biology: Validate Demand Before Scaling Science
When Erum Khan asked me why 80% of biotech companies fail at execution, I replied: "The majority of synthetic biology founders have become enamored with the tool, the technology that they developed and then they went out there with that hammer...
Flagship Startup Loses CEO to Ipsen; Ionis to Shed Nearly Six Decades of Board Experience
Alltrna's CEO Michelle Werner announced her resignation, moving to pharmaceutical giant Ipsen as its new chief executive. The biotech startup will rely on interim management while it searches for a permanent replacement. Simultaneously, Ionis Therapeutics disclosed that several long‑standing board...
Pharma Pulse: SteinCares Partners to Expand Biosimilar Access in Latin America and Eli Lilly Issues Warning Over Compounded Tirzepatide Safety
SteinCares and Shilpa Biologicals have signed a licensing deal to commercialize biosimilars across Latin America, with SteinCares handling regional registration and distribution while Shilpa oversees product development and long‑term manufacturing. The partnership targets broader patient access to cost‑effective biologics in...
FDA Approves GSK Arexvy for At‑risk Adults 18‑49
#GSK Arexvy RSV Vaccine approved by US FDA with a wider indication for Adults aged 18 to 49 who are at increased risk of Lower Respiratory Tract Disease caused by RSV.
Pilatus Biosciences Doses First Patient in PLT012 Antibody Trial
Pilatus Biosciences has begun dosing the first patient in a Phase I, open‑label trial of PLT012, its first‑in‑class anti‑CD36 monoclonal antibody, at Next Oncology in Houston. The FDA recently issued IND clearance along with orphan‑drug status for hepatocellular carcinoma and fast‑track...
Abbott Reports Positive FreeDM2 Study Results for CGM
Abbott announced that its FreeStyle Libre continuous glucose monitoring system outperformed traditional finger‑stick testing in the FreeDM2 randomised trial involving 303 UK patients with type 2 diabetes on basal insulin. After four months, CGM users achieved a larger reduction in HbA1c...
How to Optimize Your Biotech Company for Partnering, Licensing, and Business Success
Janita Good, a Fieldfisher partner with a D.Phil. in biochemistry, offers biotech leaders a roadmap for maximizing partnership and licensing value. She emphasizes initiating pharma discussions early, embedding commercialization plans into R&D, and aligning fundraising expectations with realistic exit timelines....
Hawkeye Bio Granted U.S Patent for Graphene Biosensor Platform
Hawkeye Bio announced that the USPTO granted U.S. Patent No. 12,461,102 for its pristine graphene‑based biosensor platform. The patent covers a technology that uses functionalized graphene particles and optical reporters to detect protease biomarkers with high sensitivity. The company is focusing...
PRISM BioLab and Receptor.AI Partner to Develop a Drug Discovery Platform
PRISM BioLab has teamed with Receptor.AI to build an AI‑driven, physics‑guided platform for discovering orally available small molecules that target intracellular protein‑protein interactions, membrane proteins, and complex receptor systems. The collaboration fuses PRISM’s PepMetics technology—3‑dimensional scaffolds that mimic α‑helix and...
CHMP Backs Expanded Indications for Uplizna in NMOSD, IgG4-RD and gMG
On 11 December 2025 the EMA’s CHMP issued a positive opinion to extend Uplizna’s (inebilizumab) marketing authorisation to three additional autoimmune conditions: neuromyelitis optica spectrum disorder (NMOSD), immunoglobulin G4‑related disease (IgG4‑RD) and generalized myasthenia gravis (gMG). The recommendation designates Uplizna as monotherapy for...
Ultragenyx Reports the P-III (Enh3ance) Trial for DTX301 AAV8 Gene Therapy in OTC Deficiency
Ultragenyx announced Phase 3 Enh3ance data for its DTX301 AAV8 gene therapy targeting ornithine transcarbamylase (OTC) deficiency. At week 36, the therapy lowered 24‑hour plasma ammonia by 18% versus placebo and kept levels in the normal range, even as scavenger drug use...
Natera Launches Zenith Genomics in the US to Diagnose Rare Diseases
Natera announced the commercial launch of Zenith Genomics, a next‑generation whole‑genome sequencing (WGS) assay aimed at diagnosing rare and ultra‑rare diseases in the United States. The platform pairs standard WGS with long‑read sequencing confirmation to capture complex genomic features such...
BIG Summit Panel Breaks Down Risks and Opportunities Coming Out of DC
At the inaugural BIO BIG Summit, a Washington Policy Brief panel dissected the shifting U.S. healthcare landscape for biotech. The discussion highlighted progress on reauthorizing the Prescription Drug User Fee Act (PDUFA) and emerging pharmacy benefit manager (PBM) reforms, while...
Remembering Annette Dolphin, Who Helped Explain Gabapentin’s Effects
Annette Dolphin, a pioneering neuropharmacologist at UCL, died on 27 January at 74 after a five‑decade career that reshaped voltage‑gated calcium‑channel research. Her 2005 discovery that α2δ subunits control channel trafficking clarified the molecular basis of neuropathic pain and revealed...
Trial Finds Immunotherapy Did Not Improve Survival when Added to Chemoradiotherapy for Small Cell Lung Cancer
The NRG‑LU005 phase III trial evaluated atezolizumab combined with concurrent chemoradiation in patients with limited‑stage small‑cell lung cancer (SCLC). Adding the immunotherapy did not improve overall or progression‑free survival, with median overall survival of 31.1 months versus 36.1 months for...
Solid Eyes Accelerated Approval as DMD Gene Therapy Faces FDA Uncertainty
Solid Biosciences reported Phase I/II data for its DMD gene therapy SGT‑003, showing higher microdystrophin expression and a greater proportion of positive fibers than Sarepta’s Elevidys. The results strengthen SGT‑003’s case for accelerated FDA approval, but the pathway is clouded by...
Ditch the Darth Vader Mask for Sleep Apnea
Scientists have identified sulthiame, an old epilepsy drug, as a promising treatment for moderate‑to‑severe sleep apnea. In a German trial of 298 patients, higher doses cut breathing pauses by nearly 50% and boosted overnight oxygen levels. The findings, published in...
Scan that Makes Prostate Cancer Cells Glow Could Cut Need for Biopsies
Researchers presented PRIMARY2 trial data showing that PSMA PET/CT imaging can safely halve the number of biopsies required for men with suspected prostate cancer after a normal MRI. The molecular scan highlights aggressive cancer cells by making them glow, allowing...
AC Immune SA (ACIU) Q4 2025 Earnings Call Transcript
ACADIA Pharmaceuticals reported adjusted 2025 revenue of $1.08 billion, up 14% year‑over‑year, driven by strong performance of NUPLAZID and DAYBUE. NUPLAZID net sales reached $692 million with 15% growth, while DAYBUE posted $391 million, a 12% increase, aided by the newly approved DAYBUE...
Barinthus Biotherapeutics PLC (BRNS) Q4 2025 Earnings Call Transcript
Insmed reported a strong Q4 2025, highlighted by Brinsupri’s first full‑quarter US revenue of $144.6 million, surpassing internal benchmarks. Management set 2026 guidance of at least $1 billion for Brinsupri and projected total company revenue to more than double 2025 levels, driven...
![[Therapeutics] Pyruvate Kinase Activators in Hereditary Haemolytic Anaemias: Current Evidence and Clinical Potential](https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/8c90dd45d33f73eeff78aead96a79a6a.webp)
[Therapeutics] Pyruvate Kinase Activators in Hereditary Haemolytic Anaemias: Current Evidence and Clinical Potential
Hereditary hemolytic anemias affect millions worldwide and have few disease‑modifying options. Oral pyruvate kinase activators, especially mitapivat, increase glycolytic ATP production, correcting a common metabolic defect in red cells. Clinical trials have shown efficacy in pyruvate kinase deficiency, sickle cell...
Functionalized Nanoparticles Could Open the Door to Swallowable Insulin Pills
Researchers have grafted the permeation enhancer 1‑phenylpiperazine onto safe silica nanoparticles, creating a hybrid that boosts intestinal insulin absorption while eliminating toxicity. In obese, insulin‑resistant mice, oral insulin delivered with these functionalized particles lowered blood glucose for 8‑10 hours, outperforming...
Interpretive Restraint After a Well Conducted Negative Trial
A multicentre, double‑blind, placebo‑controlled trial gave women with polycystic ovary syndrome (PCOS) 4,000 IU vitamin D daily for up to 90 days before IVF. The regimen raised serum 25‑hydroxyvitamin D levels but did not increase live‑birth rates after the first embryo...
Tonix Pharmaceuticals Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights
Tonix Pharmaceuticals announced FDA approval and U.S. launch of TONMYA, the first new fibromyalgia drug in over 15 years, in August 2025 with commercial availability beginning November 17, 2025. The company reported fourth‑quarter product revenue of $5.4 million and full‑year revenue...
FDA to Recommend Additional, Earlier MRI Monitoring for Patients with Alzheimer’s Disease Taking Leqembi (Lecanemab)
The FDA is adding an earlier MRI requirement before the third Leqembi infusion to catch amyloid‑related imaging abnormalities with edema (ARIA‑E) sooner. An analysis of pharmacovigilance data revealed 101 serious ARIA‑E cases, including six deaths, many occurring before the fifth...
Reading DNA Sequence and Epigenetic Modification State in 1 Molecule
Researchers at the University of Pennsylvania have unveiled an integrated sequencing workflow that simultaneously reads DNA sequence and distinguishes cytosine modifications—5‑methylcytosine (5mC) and 5‑hydroxymethylcytosine (5hmC)—on a single molecule. The method creates a hairpin duplex, incorporates deamination‑resistant analogs on the copy...
Xplore Program 2026: A Remote Summer Fellowship in Longevity
Longevity Xplorer (LongX) has opened applications for its 2026 Xplore Program, a fully remote summer fellowship that bridges the gap between academic interest in aging and hands‑on work in longevity biotech. The nine‑week curriculum combines a month of intensive biology...
Jefferies Paints WVE Data as Win‑win‑win; Bear Sees Combo Role
Doing some reading ahead of the next wave of $WVE INHBE "body composition" 🙄 data, smirking at the Jefferies analyst set up, which basically frames 6-month data as a win-win-win for the stock price. His "bear case" "Bear case would still...
Closing the 9-Year Gap: A New Biomarker Targets the Diagnostic Delay in Axial Spondyloarthritis
Axial spondyloarthritis affects about 1.4% of adults but patients wait an average of nine years for diagnosis in North America, far longer than rheumatoid arthritis. Augurex has introduced SPINEstat, an anti‑14‑3‑η multiplex blood test that can distinguish inflammatory back pain...
Major Risk Factor for Rare Early-Onset Dementia Found
Researchers at VIB and the University of Antwerp have identified a repeat expansion in the GOLGA8A gene as a major genetic risk factor for atypical frontotemporal lobar degeneration with ubiquitin‑positive inclusions (aFTLD‑U). The expansion appears in nearly 60 % of examined...
New Advanced Tangential Flow Filtration Systems for Efficient Bioprocessing
Intech‑Hanbon has unveiled a new line of advanced tangential flow filtration (TFF) systems aimed at biopharmaceutical, biotech and life‑science manufacturers. The platforms deliver high filtration efficiency, low shear stress and precise pressure control, while supporting a broad spectrum of applications...
Seeing the Brain in a Different Light
Researchers at Kyushu University have introduced SeeDB‑Live, an isotonic optical‑clearing medium based on bovine serum albumin that renders brain tissue transparent while preserving normal neuronal function. The solution, refined after screening nearly 100 compounds, enables three‑fold brighter fluorescence imaging in...
Eli Lilly Surges to #1 Pharma Sales in 2025
Another testimony of Eli Lilly’s increasing dominance in the BioTech and Pharma sector - compared to other Big Pharma companies - such as $PFE $JNJ $NVS $NVO $BMY $ABBV $MRK $SNY and others, can be seen in this excellent @NatRevDrugDisc...
Some Spiny Mouse Species Are Long-Lived in Addition to Displaying Exceptional Regeneration
Researchers found golden spiny mouse (Acomys russatus) lives longer and retains regenerative abilities compared to its sister species. In a non‑pathogen‑free setting, aged A. russatus showed minimal frailty, reduced inflammaging, and preserved thymic structure beyond four years. Transcriptomic analysis revealed youthful...
Brain Atlas Maps Epigenetic Changes Associated with Aging in Mice
Scientists at the Salk Institute released the most comprehensive single‑cell atlas of epigenetic aging in the mouse brain, profiling over 200,000 cells across eight regions and 36 cell types with methylation, chromatin conformation, and spatial transcriptomics. The map uncovers cell‑type...
STAT+: Cancer Cells Can ‘Barf’ Proteins Onto Their Cell Surface. That May Create New Targets for Immunotherapies
Researchers at UCSF discovered the Src kinase, traditionally an intracellular signaling protein, displayed on the outer membrane of malignant cells. The finding, published in Science, showed surface Src was absent from healthy donor tissue, suggesting a tumor‑specific marker. This unexpected...
Beyond the Safety Check: Why First-in-Human Trials Demand a New Approach in 2026
Phase I trials have evolved from simple safety checks into data‑rich, adaptive studies that integrate biomarker strategies, exposure‑response analyses, and formulation optimization. Regulators now expect sponsors to justify Phase II dosing decisions, making early pharmacodynamic and target‑engagement data essential. Emerging biotechs face...
Biotech Recovery: Fundamentals Meet Opportunity
Small- and mid‑cap biotechnology has rebounded sharply, climbing over 35% year‑to‑date in 2025 after a 20% early‑year dip and a 60% drawdown during the COVID‑era bubble. The rally is fueled by stronger clinical data, successful product launches, heightened M&A activity,...
No Evidence of Health Risks From Genetically Modified Crops Found
South Korean researchers from Seoul National University and Chosun University published a paper showing no scientific link between genetically modified (GM) crops and chronic diseases, confirming earlier findings from the 2016 National Academies report. Their analysis of PubMed literature and...
Low‑dose Rapamycin Eases Fatigue and PEM in ME/CFS
Low-dose rapamycin alleviates clinical symptoms of fatigue and PEM in ME/CFS patients via improvement of autophagy: a pilot study Myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS) is a complex, multisystem disorder characterized by profound fatigue… 👉 “Low-dose rapamycin effectively reduced PEM and other key...
Sterile PES Filters Prove Effective for Algae Work
YES. My dumb idea of using sterile PES filters and/or their parchment slip covers for algae work seems to be workinggg. Gonna try to genegun these and tranafer the filter onto selective media once a recovery period passes. Was looking...
The Many Dangers of 7-Ketocholesterol
Researchers led by Matthew O’Connor have published a comprehensive review of 7‑ketocholesterol (7KC), an oxidized cholesterol formed by reactive oxygen species. The paper details how 7KC accumulates in atherosclerotic plaques, transforms macrophages into inflammatory foam cells, and exerts neurotoxic effects...
High‑fat Diet Pushes Gut Bacteria to Brain via Vagus
How to get bacteria to move from the gut into the brain? —eat a high-fat diet —vagus nerve is the conduit —in the mouse model https://t.co/BI6HIbVgPl
New Cancer Interception Strategy Regresses Pancreatic Precancers
🆕 @ScienceMagazine An exciting new approach vs pancreatic cancer: "cancer interception" Considered ubiquitous, the pancreas in healthy adults has hundreds of PanINs of microscopic premalignant cancer that can be regressed in the experimental model https://t.co/erVtthSMjS https://t.co/2xHIYdIPrx
Pfizer to Close Ignite, Its R&D Services Unit for Small Biotechs
Pfizer announced the shutdown of its Ignite unit, a dedicated R&D services platform that partnered with early‑stage biotech companies. Ignite offered fee‑based scientific, regulatory, and strategic support, helping dozens of startups advance drug candidates. The closure aligns with Pfizer’s cost‑discipline...
FDA Reform Essential for Real Longevity Breakthroughs
I love the longevity proponents on X But we WON'T get true longevity therapeutics if we don't reform the FDA or find a way around them. The FDA is still a quagmire of regulatory rot that has to be reformed to get...