Pharma News and Headlines
Pharma News and Headlines
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 over 12‑30 months without any rise in major adverse cardiac events, myocardial infarction, stroke, or stent thrombosis. The analysis, published in JACC, included patients mostly on prior PCI and showed consistent results even in high‑risk thrombotic subgroups. Authors argue the data warrant an immediate shift in guideline recommendations toward OAC‑only regimens after the acute phase.
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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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 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 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...
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...
The FDA issued draft guidance that could lower biosimilar testing requirements by permitting foreign comparator data, potentially cutting development costs by about half.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 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 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....
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...
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 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....
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 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 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...
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...
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,...
Capricor Therapeutics reported fourth‑quarter 2025 revenue of $16 million, a modest 4% year‑over‑year increase, while gross margin rose to 86% driven by higher selling prices and manufacturing efficiencies. Net loss widened to $11.9 million as operating expenses outpaced revenue growth, but the...
Vertex Pharmaceuticals reported Q4 2025 revenue of $3.2 billion, a 10% quarterly increase, and $12 billion for the full year, up 9% year‑over‑year. Gross margin reached 85.7% and non‑GAAP net income rose 24% to $1.3 billion, underscoring strong profitability. New product...
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,...
Researchers at Harvard’s Wyss Institute and Dana‑Farber unveiled DoriVac, a DNA origami‑based vaccine platform that delivers antigens and adjuvants on a self‑folding nanostructure. In pre‑clinical mouse studies and a human lymph‑node‑on‑a‑chip model, DoriVac generated antibody and T‑cell responses comparable to...
Vima Therapeutics announced an additional $40 million raise, taking its Series A funding to $100 million. The capital will support parallel Phase 2 trials of its lead candidate VIM0423 in isolated dystonia and Parkinson’s disease, expanding the startup’s focus beyond the rare movement disorder....
The FDA placed a clinical hold on Regenxbio’s RGX‑111 and RGX‑121 gene‑therapy trials after a pediatric MPS I patient developed a tumor four years post‑treatment. The case marks the first documented long‑latency cancer linked to an adeno‑associated virus (AAV) vector in...
Two pre‑clinical studies propose active clearance of β‑amyloid as a new Alzheimer’s strategy. Researchers at Washington University engineered astrocytes with chimeric antigen receptors (CARs) that engulf plaques, while another team designed bispecific peptides that ferry amyloid into cells for lysosomal...
Senator Josh Hawley introduced a bill to immediately withdraw the FDA's safety approval for mifepristone, the primary abortion medication. The legislation follows recent Supreme Court and Trump‑era reviews of the drug and cites a controversial conservative study alleging serious adverse...
Three‑year follow‑up of the AGENT IDE trial shows the Agent paclitaxel‑coated balloon (DCB) outperforms uncoated balloon angioplasty in treating in‑stent restenosis (ISR), with target‑lesion failure (TLF) rates of 32.7% versus 40.9% (hazard ratio 0.72). The advantage is driven mainly by...
Foghorn Therapeutics announced a financial update highlighting progress on its lead oncology candidate FHD-909, which remains on schedule in Phase 1 dose‑escalation for SMARCA4‑mutant NSCLC. The company also confirmed that its selective CBP and EP300 degrader programs are slated for...
Elutia Inc. reported its Q4 and full‑year 2025 results, highlighting the $88 million sale of its BioEnvelope business to Boston Scientific and a cash position of $36.4 million plus $8 million in escrow. The company submitted a 510(k) for the base biologic matrix...
Codexis reported FY 2025 revenue of $70.4 million, a 19% increase year‑over‑year, driven largely by a $37.8 million technology‑transfer agreement with Merck. Q4 revenue jumped 81% to $38.9 million, and product gross margin improved to 64% for the year. The company ended 2025 with...
The FDA announced a unified adverse‑event database that will eventually incorporate all of its existing reporting systems, including the vaccine‑side‑effect tracker jointly operated with the CDC. The new platform, called the Adverse Event Monitoring System, centralizes data from drugs, biologics,...
Researchers at BOKU University in Vienna have engineered a novel chromatography resin designed to capture secretory immunoglobulin A (IgA) at titers suitable for commercial manufacturing. The resin employs a reengineered bacterial surface ligand, analogous to Protein A, within a macropore...
Researchers at Massachusetts General Hospital, in partnership with Full Circles Therapeutics, have introduced a circular single‑stranded DNA donor platform called INSTALL that enables kilobase‑scale gene insertion without triggering the cGAS immune sensor. The method combines a short double‑stranded DNA segment...
Researchers at Biohub identified a three‑amino‑acid cocktail—methionine, arginine and serine—that dramatically improves lipid nanoparticle (LNP) delivery of therapeutic mRNA and CRISPR components. Co‑administering the supplement boosted protein expression up to 20‑fold and raised gene‑editing rates from roughly 25% to nearly...
Johns Hopkins researchers have engineered a streamlined biodegradable polymeric nanoparticle that delivers mRNA to T cells, prompting them to generate CD19‑CAR receptors that target disease‑causing B cells. In mice, a single intravenous dose eliminated 95% of circulating B cells within...
VIDO swiftly responded to COVID‑19 by designing a subunit vaccine candidate within days of the SARS‑CoV‑2 genome release, isolating the virus, and establishing animal models that enabled a Phase 1 human trial by early 2021, making it the first Canadian university...
Costco announced a partnership with digital health platform Sesame and fertility network IVI RMA North America to offer members dramatically reduced prices on fertility medications, with discounts of up to 80 percent. The program guides members through a digital intake,...
The FDA’s acting CDER director, Dr. Tracy Beth Hoeg, is reportedly moving to hire Dr. Adam Urato, a maternal‑fetal specialist who is petitioning the agency to add boxed warnings about alleged pregnancy risks of antidepressants. Urato’s claims rely on limited...
Biogen reported Phase Ib data for its antisense oligonucleotide salanersen, showing substantial neurofilament light chain reductions and new motor milestones in children with spinal muscular atrophy (SMA) who had previously received onasemnogene abeparvovec. The study of 24 participants demonstrated a roughly...
Lonza has broadened its agreement with Genetix Biotherapeutics to increase manufacturing capacity for ZYNTEGLO, the sole FDA‑approved gene therapy for transfusion‑dependent beta‑thalassemia. Production will continue at Lonza’s Houston, Texas, dedicated cell‑and‑gene therapy site, with provisions to scale up for future...
The U.S. Food and Drug Administration approved leucovorin, a synthetic vitamin B9, solely for cerebral folate deficiency, a rare genetic disorder. Earlier this year, President Trump and HHS Secretary Robert Kennedy Jr. promoted the drug as an autism cure, prompting a surge in...
Eli Lilly’s chief executive David Ricks announced a strategic push to sustain the company’s recent $1 trillion revenue milestone by accelerating AI‑driven drug discovery and manufacturing. He highlighted a new partnership with Nvidia to embed generative‑AI models across the R&D pipeline, aiming...
Eli Lilly announced a $3 bn, ten‑year investment to expand its Chinese supply chain for the oral GLP‑1 weight‑loss drug orforglipron. The plan adds oral solid‑dosage capacity in Beijing, expands the Suzhou injection hub, and includes a $200 m CDMO partnership with Pharmaron....
Researchers have engineered an antibody‑drug conjugate that simultaneously triggers HIV’s entry‑facilitating conformational shift and binds the newly exposed epitopes. In vitro tests showed the dual‑action molecule achieved seven‑ to ten‑fold greater neutralization than the antibody alone and outperformed separate mixtures...
The FDA has granted approval for leucovorin, a folinic‑acid formulation, to treat a rare metabolic disorder despite the absence of new clinical trial data. The decision leans on decades of off‑label use and historical safety records rather than prospective studies....
Life Biosciences has secured FDA IND approval for ER-100, the first partial epigenetic reprogramming therapy to enter human trials. The gene‑therapy delivers OCT‑4, SOX‑2 and KLF‑4 to retinal ganglion cells via a single intravitreal injection, aiming to reset age‑related epigenetic...
Astellas Pharma leveraged the Trump administration’s push for lower U.S. drug prices to argue for higher reimbursement for its eye drug Izervay in Japan, winning a more generous price level. Meanwhile, a review of FDA adverse event reports found that...
The FDA is rolling out the Adverse Event Monitoring System (AEMS), replacing the legacy FAERS platform and consolidating reporting across drugs, biologics, devices, cosmetics, food, tobacco, and veterinary products. AEMS introduces standardized electronic submissions, AI‑driven redaction and analytics, and a...
GoodRx announced a partnership with Viatris to slash prices on 17 of the latter’s established brand drugs, offering up to 85% off cash prices and as little as $0‑$4 for eligible commercially insured patients. The discounts are available at more...
Researchers at ETH Zurich have introduced a boron‑based ligation strategy that overcomes the concentration barrier in chemical protein synthesis. By masking potassium acyltrifluoroborates (KATs) with chiral zwitterionic complexes, the team achieved efficient peptide coupling at micromolar levels, far lower than...
UCB announced results from its Phase III BE BOLD trial comparing Bimzelx (bimekizumab) to risankizumab in 553 adults with active psoriatic arthritis. The study met its primary endpoint, showing Bimzelx superior in achieving ACR50 responses at week 16. Bimzelx is...
