STAT+: Trump Is Getting More Credit than Biden for Efforts to Lower Drug Prices
A recent KFF poll shows 41% of Americans believe the Trump administration’s policies will lower prescription drug costs, outpacing awareness of the Biden‑era Medicare price‑negotiation law. Support is sharply partisan—79% of Republicans versus 11% of Democrats share this view. Only 31% of respondents were familiar with the Medicare negotiation program passed under the Inflation Reduction Act. The data suggest Trump receives more public credit for drug‑price efforts despite limited exposure to the newer Democratic legislation.
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....
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...
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...
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...
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[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...
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 Requests Removal of Suicidal Behavior and Ideation Warning From Glucagon-Like Peptide-1 Receptor Agonist (GLP-1 RA) Medications
The FDA has asked manufacturers to strip the suicidal ideation and behavior warning from the labeling of three GLP‑1 receptor agonists—Saxenda, Wegovy and Zepbound—used for weight loss. A comprehensive safety review, including a meta‑analysis of 91 placebo‑controlled trials involving 107,910...
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...
Apixaban Bests Rivaroxaban in New DOAC Comparison
A head‑to‑head trial (COBRRA) of 2,700 acute VTE patients found apixaban significantly lowers bleeding risk compared with rivaroxaban, while maintaining similar efficacy in preventing recurrent clots. Patients received standard dosing regimens for three months, with apixaban showing a 3.3% composite...
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...
Electronic Registration and Listing Compliance Program
The FDA’s Electronic Registration and Listing Compliance Program (eDRLS) safeguards the accuracy of drug registration and listing data that underpin inspections, electronic prescribing, and reimbursement systems. While automated validation rules catch many errors, the program also employs manual surveillance to...
Search for Pharmaceutical Quality Documents
The FDA has released an online searchable table of 249 pharmaceutical quality‑related guidances, MAPPs, and compliance programs. Recent entries include the Active Pharmaceutical Ingredient (API) Process Inspection compliance program (Sept 2025) and guidance on outsourcing facility inspections (Jan 2025). Topics span drug...
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...
FDA’s Labeling Resources for Human Prescription Drugs
The FDA provides a comprehensive suite of labeling resources aimed at industry staff who develop human prescription‑drug labeling, including DailyMed, Drugs@FDA, FDALabel, and Medication Guides. These tools grant access to over 140,000 labeling documents and detailed regulatory histories. The agency...
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...
Propanc Advances PRP Toward Clinical Trials for Pancreatic Cancer
Propanc Biopharma announced that its proenzyme therapy PRP is moving toward a Phase Ib first‑in‑human study for advanced pancreatic cancer. The company highlighted preclinical data showing more than 85 % tumor‑growth inhibition and secured FDA orphan‑drug designation. A validated pharmacokinetic assay developed...
In the Clinic for March 12, 2026
BioWorld’s March 12, 2026 clinic roundup aggregates a suite of data snapshots and special reports spanning biopharma, medical technology, and emerging therapeutic areas. The collection links to analyses on mRNA vaccine research, China’s GLP‑1 market, CAR‑T developments, and the med‑tech outlook for...
LLNL-Led Study Uses Machine Learning, Veterans’ Health Records to Identify ALS Drug-Repurposing Candidate
Lawrence Livermore National Laboratory and partners applied causal‑inference machine learning to electronic health records of more than 11,000 U.S. veterans with amyotrophic lateral sclerosis. The analysis, published in The Lancet Digital Health, identified 27 existing medications that correlate with longer...
A Simpler Form of DNA May Be Key to Non-Viral Gene Therapy, Study Suggests
Researchers have identified a streamlined DNA construct that could replace viral carriers in gene‑therapy applications. The study demonstrates that this minimalist DNA format delivers therapeutic genes with efficiency comparable to adeno‑associated viruses while eliciting a weaker immune response. Production of...
Patient Education
The FDA emphasizes that generic medications are as safe and effective as brand‑name drugs while offering substantial cost savings. Over 90% of prescriptions in the United States are filled with generics, reflecting broad market acceptance. To address consumer questions, the...
FDA to Unify Agency’s ‘Fragmented’ Safety Surveillance System
The FDA launched the Adverse Event Monitoring System (AEMS), a unified dashboard that merges seven previously fragmented safety databases into a single platform. Four databases are already live, with the remaining three slated for integration by May, covering drugs, vaccines,...
FDA to Unify Agency’s ‘Fragmented’ Safety Surveillance System
The FDA has launched the Adverse Event Monitoring System (AEMS), a unified platform that merges seven previously fragmented safety‑surveillance databases. Four of those databases are live today, with the remaining three slated for integration by May 2026. The agency processes...
Reviews of Pediatric Studies Conducted Under BPCA and Pediatric Assessments Conducted Under PREA From 2012 – Present
The FDA has compiled reviews of pediatric studies conducted under the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) from 2012 to present. A total of 88 products were studied under BPCA, 544 under PREA,...
The Hepatitis B Birth Dose Trial That Triggered the WHO Alarm
The World Health Organization issued a formal warning on 13 February 2026 about a proposed randomized trial in Guinea‑Bissau that would withhold the hepatitis B birth‑dose vaccine from roughly half of 14,000 newborns. The study seeks to measure non‑specific effects...
Oral Anticoagulation Alone Best for Stable CAD Patients With AF: Meta-Analysis
A meta‑analysis of six randomized trials involving 5,924 stable CAD patients with atrial fibrillation found that oral anticoagulant (OAC) monotherapy reduced cardiovascular mortality by 31% and major bleeding by 54% compared with OAC plus a single antiplatelet. The benefit persisted...
PharmaShots Magazine-March-2026 Edition
AI is reshaping clinical trial oversight by moving from periodic checks to continuous, real‑time intelligence. Advanced analytics and machine learning now power predictive, risk‑based monitoring that aggregates data from decentralized sites, wearables, ePROs and multiple clinical systems. This integration enables...
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Venomous Snakes Represent a Serious Public Health Problem. Scientists Are Biting Back With a Groundbreaking Antidote
Snakebite envenoming kills over 125,000 people each year and leaves three times as many disabled, while current horse‑derived antivenoms trigger severe allergic reactions in nearly half of patients. The high cost—up to $100,000 per course—and limited hospital access leave rural...
BridgeBio Builds Case for Early 2027 Launch of Dystrophy Drug
BridgeBio Pharma reported that its small‑molecule candidate BBP‑418 produced a 1.8‑fold increase in the α‑dystroglycan biomarker in a Phase 3 FORTIFY trial for limb‑girdle muscular dystrophy type 2I/R9, with effects sustained through 12 months. The trial enrolled 81 patients and also showed...
Aplagon Doses First Patient in P-IIa (HEALING) Trial of APAC for Peripheral Arterial Occlusive Disease/Chronic Limb Threatening Ischemia
Aplagon Therapeutics has administered the first dose of its intravenous APAC candidate to a patient in a Phase 2a (HEALING) trial for peripheral arterial occlusive disease leading to chronic limb‑threatening ischemia in Finland. The study will enroll approximately 42 CLTI...
Bridging Promise and Evidence in Psychedelic Medicine
Jacobs and colleagues present a state‑of‑the‑art review of psilocybin and MDMA‑assisted therapies, highlighting their potential for treatment‑resistant depression and PTSD. The authors emphasize the distinct, session‑based paradigm that leverages acute neurobiological changes to produce lasting clinical benefits. However, they also...
New FDA Guidance Could Elevate Pharma’s Biosimilar Market
The FDA issued draft guidance that could lower biosimilar testing requirements by permitting foreign comparator data, potentially cutting development costs by about half. Over 80 biosimilars have been approved in the U.S. since the first approval 11 years ago, yet...
Insulet Reports EVOLUTION 2 Study Results for FCL Automated Insulin Delivery System in Type 2 Diabetes
Insulet presented data from its EVOLUTION 2C feasibility study of a fully closed‑loop (FCL) automated insulin delivery system for adults with type 2 diabetes at ATTD'26. In the 24‑patient trial, the final algorithm raised time‑in‑range (TIR) to an average 68%,...
Bavarian Nordic and SII Sign Agreement for Chikungunya Vaccine Production
Bavarian Nordic has signed a manufacturing agreement with the Serum Institute of India to transfer full technology for its chikungunya vaccine, CHIKV VLP (branded Vimkunya). The deal replaces an earlier partnership with Biological E and builds on the companies' existing mpox‑vaccine collaboration....
Lilly Warns of Impurity Risk in Certain Compounded Forms of Mounjaro and Zepbound
Eli Lilly warned that compounded versions of its GLP‑1 drugs Mounjaro and Zepbound, when mixed with vitamin B12, may contain unsafe impurities. The alert follows growing off‑label compounding as demand outpaces supply. Lilly’s notice urges clinicians and patients to verify product sources...
From Two Trials to One, Sponsors Face a Higher Standard
The FDA announced that a single pivotal trial can now satisfy efficacy requirements for new drug applications, replacing the previous two‑study mandate. This change raises expectations for data depth, quality, and risk‑based management throughout the trial lifecycle. Guests Oxana Iliach...
Gesynta Pharma Doses First Patient in Phase II NOVA Trial
Gesynta Pharma has begun dosing the first patient in its Phase II NOVA trial, evaluating oral vipoglanstat for endometriosis. The double‑blind, placebo‑controlled study will enroll about 190 women across Europe and compare two dose levels against placebo over four menstrual cycles....
Lilly Issues Public Warning About Tirzepatide Compounded with B12
Eli Lilly has issued a public warning after testing revealed a significant impurity formed when tirzepatide is compounded with vitamin B12. The impurity’s health effects are unknown, and the company has alerted the FDA and urged patients to consult physicians....
George Medicines Partners with Ahngook Pharmaceutical to Commercialize GMRx2 in Korea
George Medicines has signed an exclusive licensing and supply agreement with South Korea’s Ahngook Pharmaceutical to bring its triple‑combination antihypertensive pill, GMRx2, to the Korean market. The single‑pill formulation blends telmisartan, amlodipine and indapamide in three dose strengths, aiming for...
C2N Diagnostics Partners with BeauBrain Healthcare to Offer PrecivityAD2 Blood Test for Alzheimer’s Disease in South Korea
C2N Diagnostics has signed a partnership with BeauBrain Healthcare to introduce its PrecivityAD2 blood test for Alzheimer’s disease in South Korea, targeting patients aged 50 and older with mild cognitive impairment or dementia. Clinical studies published in JAMA and npj...
Tigecycline-Induced Hypoglycemia in Critically Ill Patients with Severe Infections: A Retrospective Cohort Study
A retrospective cohort of 169 critically ill patients receiving tigecycline showed significant reductions in blood glucose at three daily time points. Hypoglycemia occurred in 11.2% of patients and was linked to a nearly four‑fold increase in 28‑day mortality (OR 3.83). Larger...
Ancient Mushroom, Modern Medicine: Paul Stamets Says Agarikon Mycelium May Be Key to Fighting Viral Pandemics
Mushroom mycologist Paul Stamets presented data indicating that Agarikon (Fomitopsis officinalis) mycelium possesses broad antiviral properties. In two placebo‑controlled trials, a combined Agarikon‑turkey‑tail extract reduced COVID‑19 vaccine side effects, sustained antibody titers, and accelerated recovery in hospitalized patients. The research,...
Nektar Therapeutics (NKTR) Q4 2025 Earnings Call Transcript
Nektar Therapeutics reported strong Q4 2020 results, highlighting robust progress on its cytokine platform. The IL‑2 agonist BEMPEG demonstrated a median progression‑free survival of 30.9 months in melanoma and is now enrolled in six registrational trials across melanoma, renal, bladder,...