Olezarsen Doesn’t Lower Plaque Volume: Essence-TIMI 73b
Olezarsen, an antisense drug targeting APOC3, dramatically lowered triglycerides (‑64 %) and remnant cholesterol (‑72 %) in the Phase III Essence‑TIMI 73b trial, yet a 12‑month coronary CTA subanalysis showed no significant reduction in non‑calcified plaque volume versus placebo. The study involved 468 patients with moderate hypertriglyceridemia, most of whom (97 %) were already on statins and other lipid‑lowering therapies. While apoB fell modestly (‑16 %) and LDL remained unchanged, the imaging endpoint was neutral, prompting calls for a longer cardiovascular outcomes trial.
STAT+: Government Watchdog Urges FDA to Finalize Guidance for Advisory Committee Conflicts of Interest
The Government Accountability Office (GAO) reports that the FDA has still not finalized the financial conflicts‑of‑interest (COI) guidance for its advisory committees, despite a law mandating it 13 years ago. The agency also fails to publicly disclose how it evaluates...
FDA Warns Consumers Not to Purchase or Use Artri and Ortiga Products, Which May Contain Hidden Drug Ingredients
The FDA has issued a renewed public warning that over‑the‑counter products bearing the names “Artri” or “Ortiga” may contain undeclared prescription drugs such as dexamethasone, diclofenac sodium, and methocarbamol. Since April 2022, more than 30 consumers have reported severe health effects,...
The Man Who Let Deadly Snakes Bite Him for 20 Years—And the Universal Antivenom Hiding in His Blood
A Wisconsin man, Tim Friede, let venomous snakes bite him for two decades, building a unique repertoire of antitoxin antibodies. Researchers at biotech firm Centivax isolated two of these antibodies and combined them with the toxin‑blocking drug varespladib, creating a...
To Counter China, FDA Chief Wants to Speed New Drug Trial Process
FDA Commissioner Marty Makary said the agency will slash the amount of non‑safety data required to launch new drug trials in the United States. The streamlined approach aims to eliminate redundant paperwork, focusing only on safety‑related information. Makary framed the...
Tobacco Plant Altered to Produce Five Psychedelic Drugs
Scientists at Israel's Weizmann Institute have engineered tobacco (Nicotiana benthamiana) to produce five psychedelic compounds—including psilocin, psilocybin, DMT, bufotenin and 5‑methoxy‑DMT—using agroinfiltration, a transient gene‑delivery method that does not integrate DNA into the plant genome. The approach leverages nine introduced...
Pfizer, BioNTech to Pause COVID Vaccine Study Due to Low Enrollment
Pfizer and BioNTech announced the suspension of a FDA‑mandated post‑marketing study of their COVID‑19 vaccine due to insufficient participant enrollment. The trial, aimed at 25,500 adults aged 50‑64, was designed to assess safety, immune response, and efficacy against infection. Companies...
They Thought Their Hearing Was Gone Forever—Until Doctors Tried Something Radical
A 2025 Nature Medicine study showed that delivering a functional OTOF gene via an adeno‑associated virus dramatically improves hearing in patients with genetic deafness. Ten participants aged 1 to 24 across five Chinese hospitals experienced a reduction in hearing threshold...
Kailera Plots IPO to Fuel Obesity Pipeline
Kailera Therapeutics, after raising $1 billion across a $400 million Series A and $600 million Series B, is preparing an IPO to fund its obesity drug pipeline. Its lead candidate, ribupatide, has delivered up to 23.6% weight loss in Phase 3 injectable trials and 12.1% loss...
Eli Lilly Opposes Push to Pass Trump's Drug Pricing Deals Into Law, CEO Says
Eli Lilly is publicly opposing the White House’s effort to turn its voluntary “most‑favored‑nation” (MFN) drug‑pricing agreements into law. The company, along with more than a dozen peers, signed MFN deals in 2025 to align U.S. prices with those in other...
Enlivex Clears Pivotal FDA Hurdle in Knee Osteoarthritis
Enlivex has secured FDA Investigational New Drug (IND) clearance to launch a global Phase 2b trial of its immunotherapy Allocetra for moderate‑to‑severe age‑related knee osteoarthritis. The study will be randomized, double‑blind, and placebo‑controlled, building on promising Phase 1/2a data from 134 patients....
FDA Open To More Data On Rare Disease Drug Ersodetug Despite Missed Phase 3 Endpoint
Rezolute announced that the FDA remains open to reviewing additional data from its Phase 3 trial of the rare‑disease hypoglycemia drug ersodetug, despite the study missing its primary efficacy endpoint. The agency’s willingness suggests the experimental therapy could still move toward...
Scientists Create Plant That Produces Ayahuasca, Shrooms, and Toad Psychedelics All At Once
Scientists have genetically modified tobacco plants to biosynthesize five distinct psychedelic compounds typically sourced from psilocybin mushrooms, ayahuasca vines, and the Sonoran Desert toad. The engineered pathway, detailed in a Science Advances paper, yields measurable amounts of psilocybin, DMT, 5‑MeO‑DMT...
Key Considerations for Drug Development Pipelines in Early Phase Clinical Trials
Early‑phase clinical trials are shifting from traditional maximum‑tolerated dose (MTD) hunting to identifying an optimal biological dose (OBD) that balances safety, efficacy, and exposure. The FDA’s Project Optimus and related guidance now require dose‑optimization using all clinical data, prompting broader...
Researchers Unlock the Key to Axon Regeneration
Researchers at Icahn School of Medicine discovered that the aryl hydrocarbon receptor (AHR) acts as a molecular brake preventing axon regeneration after nerve injury. Genetic deletion or pharmacological inhibition of AHR in mouse models redirected neurons from a stress‑survival mode...
FDA Revises Recommendation on First Full Epcoritamab Dose in R/R DLBCL to Allow Outpatient Monitoring
The FDA has revised the label for epcoritamab (Epkinly) to permit outpatient monitoring of the first full 48‑mg dose in relapsed/refractory diffuse large B‑cell lymphoma (R/R DLBCL). The change follows interim EPCORE‑NHL‑6 data showing the dose can be safely administered...
Text Mining Culture Conditions and Glycosylation Relationships
Researchers at the University of Delaware and Waters have created an automated text‑mining pipeline that extracts relationships between cell‑culture conditions and protein glycosylation with 88% accuracy. The extracted data are normalized and stored in a Bioprocess Knowledge Graph, enabling a...
Peptonics Solves Cell Culture Defoaming Debacle
Researchers have demonstrated that the peptide‑based surfactant Peptonic ih‑T1010 performs on par with the industry‑standard poloxamer 188 in CHO and HEK293 fed‑batch cultures for monoclonal antibodies and AAV vectors. The new surfactant dramatically reduces foam formation, allowing manufacturers to skip...
Under the Magnifying Glass: A Wave of Drug Price Transparency
In late January and early February 2026, three federal initiatives – a Department of Labor proposed rule, the Consolidated Appropriations Act of 2026, and an FTC settlement with Express Scripts – introduced mandatory transparency reporting for pharmacy benefit managers (PBMs)....
Teva Intensifies Biosimilar Competition with FDA Approval and Dual Filing Acceptance in US and Europe
Teva Pharmaceutical Industries received U.S. FDA approval for its denosumab‑adet biosimilar, covering all approved indications of Amgen’s Prolia, including osteoporosis and bone loss in cancer patients. The company also secured simultaneous acceptance of regulatory filings with the FDA and the...
Esketamine Nasal Spray Shows Rapid, Durable Effectiveness in Treatment-Resistant Depression
New real‑world evidence from the ECHO study confirms that esketamine nasal spray delivers rapid and durable symptom relief for adults with treatment‑resistant depression. In a European‑Israel cohort of 570 patients, average treatment lasted nine months, producing mean MADRS reductions of ‑10.3...
Amgen, Zai Lab Team up on DLL3; Janux Gets $35M Milestone Payment
Amgen and China‑based Zai Lab have announced a Phase 1b clinical study that combines Amgen’s T‑cell engager Imdelltra with Zai Lab’s experimental antibody‑drug conjugate zocilurtatug pelitecan, targeting the DLL3 protein in aggressive neuroendocrine cancers. The trial will evaluate safety and early...
DNA Testing Can Help Right Racial Imbalance in Breast Cancer
Routine genomic testing with Agendia’s MammaPrint and BluePrint can narrow the long‑standing survival gap between Black and white women with early‑stage, hormone‑receptor‑positive breast cancer. In a study of more than 1,000 matched patients, Black women were twice as likely to...
Atom Swapping Arrives for 5-Membered Cyclic Ethers
Researchers at the National University of Singapore have unveiled a skeletal‑editing method that replaces the oxygen atom in five‑membered saturated cyclic ethers with nitrogen, sulfur, carbon or selenium. The protocol uses triphenylphosphine and N‑bromosuccinimide to generate a dibromo intermediate, which...
Paragon Offshoot Korsana to Go Public in Reverse Merger for Alzheimer's Work
Paragon Therapeutics is spinning out its Alzheimer’s-focused unit, Korsana Biosciences, via a reverse merger that will list the company on a U.S. exchange. The deal, championed by Paragon co‑founder Jonathan Violin, merges Korsana with a publicly traded shell, providing immediate...
How a 20-Year Old Asthma Drug Is Boosting Food Allergy Research
A 20‑year‑old asthma medication, Xolair (omalizumab), is now accelerating food‑allergy research, especially for peanut sensitivities. Recent clinical trials combined the drug with oral immunotherapy, cutting severe reaction rates by roughly 70 percent. The FDA has recently cleared the first oral...
IO Biotech Will File for Bankruptcy After Failure of Cancer Vaccine in Key Trial
Danish biotech IO Biotech announced it will file for Chapter 11 bankruptcy after its lead cancer vaccine failed to demonstrate efficacy in a pivotal trial. The vaccine, designed to treat aggressive solid tumors, showed no statistically significant benefit, prompting the...
Can AI Agents Automate Scientific Discovery?
Nvidia’s GTC keynote highlighted a new wave of agentic AI systems—OpenClaw, Kosmos, LabOS, Latent‑Y and Dyno Psi‑Phi—designed to automate and accelerate scientific discovery. These agents combine large‑language models, XR interfaces and robotic labs to compress months of research into days while...
STAT+: Insilico Medicine CEO on How Best to Use AI in Drug Development
Insilico Medicine, a veteran AI‑driven drug discovery firm, announced a partnership with Eli Lilly that includes a $115 million upfront payment and up to $2.75 billion in milestone‑based total consideration. The deal leverages Insilico’s generative‑AI platform to co‑develop novel therapeutics, primarily targeting metabolic...
PharmaShots Quarterly Outlook: The Forces Reshaping Biopharma in Q1 2026
Q1 2026 biopharma saw a wave of mega‑size M&A, with deals like Boston Scientific’s $14.5 billion purchase of Penumbra and Eli Lilly’s $7.8 billion acquisition of Centessa, underscoring a strategic push for precision platforms. The quarter also delivered a string of rare‑disease approvals—Zycubo,...
Portugal: Will the Life Science Sector See Upswing Amid Funding Worries?
Portugal’s life‑science sector generated €29.7 billion ($34.4 billion) in gross value added in 2024, employing over 268,000 people across 124,000 firms. The ecosystem gained visibility after hosting BIO‑Europe Spring, showcasing biotech startups, research parks like Biocant, and major deals such as BioNTech’s...
ACC 2026: Dulaglutide Promotes Coronary Plaque Stabilisation in Patients with T2D
At the American College of Cardiology 2026 meeting, researchers reported that dulaglutide, a weekly GLP‑1 receptor agonist, stabilised coronary plaques in patients with type‑2 diabetes. In a prospective randomised trial of 39 participants with intermediate coronary stenoses, dulaglutide led to...
Scientists Are Working on “Everything Vaccines”
Vaccines prove their worth when they fail, as recent flu and COVID‑19 seasons have shown. The COVID‑19 pandemic exposed how quickly a novel virus can outpace vaccine development, while the 2025 flu season suffered a mismatch when the H 3 N 2 strain...
Biopharma M&A Heats Up, Rare Diseases Win Three Approvals, Wave Crashes
Biopharma giants Biogen, Eli Lilly and Merck collectively spent over $20 billion in a single week to acquire biotech firms with approved products or promising pipelines, accounting for three of the year’s four largest deals. Merck bought Terns Pharmaceuticals for $6.7 billion, while...
Ambrosia Eyes Next-Generation Small Molecule GLP-1s With $100M Series B
Ambrosia Biosciences announced the completion of a $100 million Series B financing round to fund the development of next‑generation small‑molecule GLP‑1 oral therapies for obesity. The capital will support a Phase 1 trial of its lead GLP‑1 candidate, which leverages AI‑driven molecular design...
Rare Disease Advocacy Group Urges Trump Administration to Restore FDA Clarity
A coalition of nearly 100 rare‑disease patient groups, biotech executives and investors wrote to President Trump, Health Secretary Robert F. Kennedy Jr., Medicare administrator Mehmet Oz and FDA Commissioner Marty Makary urging the administration to restore regulatory clarity at the...
Axsome Therapeutics Acquires Global Rights to Balipodect From Takeda
Axsome Therapeutics has signed an asset purchase agreement with Takeda to acquire worldwide rights to the PDE10A inhibitor TAK‑063, branded as balipodect. The deal provides Axsome with the ability to develop, manufacture and commercialize the drug for schizophrenia and Tourette...
Protective Effects of Gypenosides on LDL-Induced Myocardial Injury Through the miR-223/NLRP3 Axis in Hyperlipidemia
A cross‑sectional analysis of 19,862 Chinese adults linked elevated LDL‑C to higher glucose, BMI, blood pressure, white‑blood‑cell count and triglycerides, especially among middle‑aged and older men. Parallel in‑vitro experiments showed native LDL directly impairs H9C2 cardiomyocyte viability, proliferation, migration and...
Estonian Validfor Raises $1.2 Mililion Pre-Seed to Cut Pharma Validation Timelines From Months to Weeks
Estonian compliance startup Validfor secured a $1.2 million pre‑seed round led by DOMiNO Ventures, with participation from Curiosity VC and angels. The company is building an AI‑native, agentic digital validation platform that promises to shrink pharma, biotech and medtech validation cycles...
IO Shuts Down Following Regulatory Roadblocks
Danish biotech IO Biotech announced it will wind down operations and file for Chapter 7 bankruptcy after regulatory setbacks. The FDA rejected its biologics license application for the cancer vaccine Cylembio in September, citing insufficient data. A Phase 3 trial combining Cylembio...
EMA Seeks Input on Virtual Alternative to Animal Test
The European Medicines Agency has released a draft qualification opinion that would allow virtual control groups (VCGs) to replace rats in dose‑range‑finding toxicology studies. The proposal, submitted by Synapse Research and five pharma partners under the VICT3R consortium, is open...
Medical Podcasts
Medical Design Briefs released a series of podcasts on April 1 2026 highlighting emerging trends in drug delivery. The episodes cover AI‑driven personalized medicine in oncology, sustainability challenges for insulin pens and other devices, intra‑arterial platforms that target solid tumors, and wearable...
Medical Podcasts
Medical Design Briefs released a series of April 2026 podcasts spotlighting emerging drug‑delivery trends. Episodes feature First Ascent Biomedical’s AI‑driven platform that personalizes oncology therapy, MGS engineers discussing greener insulin‑pen designs, RenovoRx’s intra‑arterial delivery system that targets solid tumors, and...
Clinical Trial For Brain Cancer Treatment Has Promising Results
A novel glioblastoma treatment combining oral 5‑ALA with low‑intensity ultrasound has shown promising early results, extending median survival by over 14 months in a phase 1 trial for recurrent patients. The approach sensitizes tumor cells to ultrasound, allowing diffuse targeting of...
FDA, After Turbulent Year, Leaves Drugmakers Guessing on Its Direction
The FDA’s leadership turmoil has intensified under Commissioner Marty Makary, with the agency cycling through multiple heads of its CDER and CBER centers in just over a year. Public‑facing comments from senior officials have sparked sharp stock moves, most notably...
Will Pfizer’s Lyme Disease Gamble Pay Off or Set the Space Back?
Pfizer and French partner Valneva are seeking FDA approval for a 6‑valent OspA Lyme disease vaccine after a late‑stage trial showed more than 70% efficacy, though the study missed its primary statistical endpoint due to low infection rates. The candidate...
GSK Reports the NMPA Approval of Exdensur (Depemokimab) for Severe Asthma
GlaxoSmithKline’s biologic Exdensur (depemokimab) received approval from China’s National Medical Products Administration as an add‑on maintenance therapy for patients aged 12 and older with severe eosinophilic asthma. The approval is based on Phase III SWIFT‑1 (382 participants) and SWIFT‑2 (380 participants)...
Oric to Advance Prostate Cancer Drug to Phase 3, but Combo Choice Raises Doubts
Oric Pharmaceuticals announced that its PRC2 inhibitor will move into a registrational Phase 3 trial for prostate cancer after reporting encouraging safety and disease‑control signals in a Phase 1b study. The company plans to evaluate the drug both as a...
Frontier Medicines Grants Ex-China Rights of FMC-220 to LG Chem
Frontier Medicines has granted LG Chem an exclusive global license to develop and commercialize its covalent p53 Y220C activator FMC-220 outside Greater China, while retaining full rights within that region. LG Chem will manage regulatory filings, global clinical development, and...
Patients Know Best and Heartfelt Technologies Collaborate to Accelerate Clinical Trial Recruitment
Patients Know Best teamed with Heartfelt Technologies to automate recruitment for the Innovate UK‑funded HF‑TRACK trial, enrolling 16 participants in just four weeks—a 135% increase over the trial’s average rate. The partnership leveraged PKB’s coded health‑record data to display a...