Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech rounds
Spanish biotech firm Ona announced an $86.6 million Series B financing, ranking among the largest venture rounds in Spain’s life‑science sector this year. The round was led by Seventure Partners with participation from existing backers and new strategic investors. The funding will be used to scale Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical programs.
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 chemoradiation alone. A secondary analysis revealed that twice‑daily thoracic radiation markedly improved survival compared with once‑daily schedules. The study enrolled 544 patients across the United States and Japan, providing robust data on contemporary treatment approaches.
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...
![[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...
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...
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...
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...
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...
Pharmaceutical Executive Daily: Senate Democrats Seeking Information on MFN Pharma Deals
Senate Democrats, led by Sen. Ron Wyden, have sent letters to eleven pharmaceutical companies demanding details on their most‑favored‑nation (MFN) agreements and whether those prices generate real savings for Medicaid, with a March 23 response deadline. The inquiry follows earlier requests...
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...
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...
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...
‘Magic Mushroom’ Derivative Could Heal without Hallucinations, Sparking Hope for New Therapies
Scientists at the University of Padova synthesized fluorinated psilocin derivatives, identifying compound 4e as a lead that retains serotonergic activity while markedly reducing hallucinogenic effects in mice. In vitro assays showed 4e is a selective partial agonist at 5‑HT2A and...
Why Peptide Enthusiasts Fear Tested Vaccines
Can someone explain to me why many of the same folks who love experimenting with peptides (i.e. little to no rigorous safety data) are terrified of vaccines and other drugs that have testing... How does someone square that circle?
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...
Navigating Complexity in Emerging Biotech: Innovations, Integrations, and Initial Hurdles
Industry experts highlight three intersecting forces reshaping emerging biotech: the persistent funding and regulatory hurdles faced by early‑stage startups, the rapid migration of AI from a supportive tool to an operational backbone, and breakthrough computational methods—including quantum chemistry—that are redefining...
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...
RFK Jr Promotes Unproven Peptides, BPC‑157 Lacks Solid Data
RFK Jr is trying to get 14 peptides, without data on safety or efficacy, licensed and approved by FDA. His favorite is BPC-157. "Only three small human studies of BPC-157 exist, for instance, the largest of which is a telephone...
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
Baseimmune Announces Strategic Expansion Into Fibrosis with Lead Program Targeting Idiopathic Pulmonary Fibrosis (IPF)
Baseimmune announced a new fibrosis‑focused pipeline leveraging its computational protein design platform to create multi‑pathway immunotherapies, starting with idiopathic pulmonary fibrosis (IPF). The company aims to deliver proof‑of‑concept efficacy data for its lead IPF program in 2026‑2027, addressing the limitations...
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...
Lilly Warns GLP‑1 Knockoffs Are Dangerous, Intensifies Compounder Battle
Lilly warns GLP-1 knockoffs may be ‘dangerous,’ escalating war with compounders https://t.co/XOKLqIzAES @ByJonGardner $LLY $NVO #obesity
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...
Pierre Fabre, ATRA to Meet FDA on Ebvallo CRL Soon
Pierre Fabre and $ATRA said FDA meeting to discuss the Ebvallo CRL has been scheduled. (Likely occuring within the next month.)
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...
FDA Cracks Down on Misleading GLP‑1 Telehealth Marketing
The FDA is targeting telehealth marketing of GLP-1 drugs. Who’s prescribing them? Medical groups face ‘tricky’ questions when partners make misleading claims via @KatieMPalmer https://t.co/pf4Fwa7W7r
How Does Cocaine Rewire the Brain?
Researchers at Michigan State University used mouse models and CRISPR technology to map how cocaine rewires the ventral hippocampus‑nucleus accumbens (vHPC‑NAc) circuit. They discovered that the transcription factor ΔFosB acts as a molecular switch, accumulating with repeated cocaine exposure and...
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%,...