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.
FDA PreCheck Pilot Program Structure
The FDA PreCheck Pilot Program is a two‑phase initiative aimed at speeding the launch of new U.S. pharmaceutical manufacturing sites. Phase 1 delivers early engagement through Pre‑Operational Reviews and the creation of a Type V Drug Master File that documents facility design, equipment qualification, and quality systems. Phase 2 leverages that repository to enable pre‑submission meetings, expedited facility assessments, and earlier inspection timelines for drug applications. The program assigns a dedicated FDA point of contact and offers flexible, milestone‑driven interactions to reduce regulatory delays.
Nipocalimab Demonstrates Sustained Disease Control Over Two Years in Generalized Myasthenia Gravis
Johnson & Johnson announced that nipocalimab (Imaavy) sustained disease control in generalized myasthenia gravis for over two years, extending efficacy beyond the initial 24‑week trial to 120 weeks. Patients showed mean reductions of 6.47 points on the MG‑ADL and 5.97...
First Non-INSTI, Tenofovir-Free Regimen DOR/ISL Gains FDA Approval for HIV
The FDA has approved Merck’s Idvysno, a once‑daily, single‑tablet combination of doravirine and islatravir, marking the first non‑INSTI, tenofovir‑free two‑drug regimen for adults with virologically suppressed HIV‑1. Phase 3 trials demonstrated non‑inferior viral suppression compared with standard three‑drug regimens, with only...
Moderna, After Losing US Funding, Rebounds to Start mRNA Bird Flu Vaccine Trial
Moderna has launched a Phase 3 trial of its mRNA‑1018 bird‑flu vaccine, enrolling about 4,000 healthy adults in the United States and the United Kingdom. The study follows the loss of a $766 million U.S. government contract, which had funded earlier development...
Neuromodulation and Nerve Ablation for Chronic Pain with NeuroOne CEO Dave Rosa — Episode 252
NeuroOne Medical Technologies, led by veteran CEO Dave Rosa, is advancing minimally invasive electrodes that combine nerve ablation, neuromodulation, and direct drug delivery for conditions like epilepsy and chronic pain. Rosa, who has spent three decades at firms such as...
FDA Expands Teplizumab-Mzwv Approval to Young Children With Stage 2 T1D
The FDA granted supplemental approval for Sanofi’s teplizumab‑mzwv (Tzield) to treat children as young as one year with stage 2 type 1 diabetes, expanding the prior 8‑year‑and‑up indication. The decision follows interim data from the PETITE‑T1D phase 4 trial, where 23 children received...
Immunotherapy in Locally Advanced HNSCC: Is There Still Room for New Agents?
In June 2025 the FDA approved MSD’s Keytruda (pembrolizumab) for resectable locally advanced head‑and‑neck squamous cell carcinoma (LA HNSCC) with PD‑L1 CPS ≥ 1, based on the KEYNOTE‑689 trial. A separate adjuvant nivolumab study (NIVOPOSTOP) showed promising results, though regulatory filing is...
STAT+: Gene Therapy Trial for Deafness Adds Evidence to Drug’s Efficacy
Researchers have reported that a gene‑therapy injection dramatically improved hearing in a Chinese clinical trial, with 90% of participants noting significant gains. The study, published in Nature, includes both children and adults, such as a 32‑year‑old who regained functional hearing....
Curve Biosciences Announces Key AI and Clinical Advancements of Whole-Body Intelligence for Chronic Diseases
Curve Biosciences announced two major milestones: its genomic AI foundation model will be presented at the International Conference on Learning Representations (ICLR) and its Whole‑Body Blood Test demonstrated strong performance in a real‑world liver cirrhosis monitoring study. The study enrolled...
Trump’s “Most‑favored‑nation” Deals Don’t Ensure Cheapest U.S. Drug
One lie worth refuting that @RobertKennedyJr keeps repeating across hours of testimony before Congress from the last week (final Senate Finance/HELP day today): The "most-favored nation" drug pricing deals that Trump crafted with 16 cos. DO NOT by any means guarantee...
The META-AF Trial
Researchers launched the META‑AF trial to evaluate metformin as an adjunct to catheter ablation in atrial fibrillation patients. The study randomizes roughly 500 participants to receive metformin or placebo beginning two weeks before ablation, with follow‑up through 12 months. Primary...
EMP‑01 Shows Strong Patient‑Reported Gains in Social Anxiety
What incredible news to wake up to! Some weeks ago, @ataibeckley reported positive topline results for EMP-01 (oral R-MDMA, a patent-protected version of #MDMA) for Social Anxiety Disorder(SAD). The data reported was the reduction in symptoms as reported by the treating...
Restrictions on Obesity Drug Coverage Force Patients to Pivot
Insurance giants CVS Caremark and other carriers are pulling popular GLP‑1 obesity drugs such as Zepbound and Wegovy from many formularies, leaving millions of patients without coverage. GoodRx data shows 12 million people lost Zepbound and another 12 million lost Wegovy between...
Maze Therapeutics Secures $150 Million in Stock and Pre‑Funded Warrants Offering
Maze Therapeutics announced the pricing of a $150 million underwritten offering of common stock and pre‑funded warrants. The capital will extend the company’s runway to 2029 and fund the development of its lead candidates for APOL1‑mediated kidney disease and phenylketonuria.
Amazon Launches GLP‑1 Weight‑Loss Program, Targeting $25‑Per‑Month Market
Amazon introduced a GLP‑1 weight‑loss service through its One Medical unit, combining virtual and in‑person care with pharmacy fulfillment, pricing drugs such as Wegovy at $25 per month for insured patients, marking a strategic push into healthcare that could boost...
Kelun-Biotech to Unveil Three Oncology Trial Results at 2026 ASCO Meeting
Sichuan Kelun-Biotech Biopharmaceutical announced that it will present results from three clinical studies—sac‑TMT plus pembrolizumab, the RET inhibitor lunbotinib, and the novel ADC SKB500—at the 2026 American Society of Clinical Oncology meeting in Chicago. The disclosures underscore the company's expanding...
AI Is Spitting Out More Potential Drugs than Ever. This Start-Up Wants to Figure Out Which Ones Matter.
10x Science, a biotech AI startup founded by former Stanford researchers, announced a $4.8 million seed round led by Initialized Capital. The company’s platform combines deterministic chemistry algorithms with AI agents to automatically interpret mass‑spectrometry data, turning raw spectra into actionable...
AI‑Driven Discoveries Shift Medicine to Understanding the How
The Real Era of the Art of Medicine Begins with Artificial Intelligence. I mean, when AI discovers new treatments and runs in silico clinical trials that physicians, pharma companies, or medical innovators would never think of, our job will be understanding the...
Moderna Launches mRNA Bird Flu Trial After Funding Loss
Moderna, after losing US funding, rebounds to start mRNA bird flu vaccine trial https://t.co/cKyC9jfWAj by Kristin Jensen $MRNA
New Review Casts Doubt On Alzheimers Drugs But Is Controversial
A new Cochrane review of 17 trials involving more than 20,000 Alzheimer’s patients concludes that amyloid‑targeting monoclonal antibodies deliver only trivial cognitive benefits and carry safety risks. The analysis groups together all anti‑amyloid antibodies—including older failures—thereby diluting the modest gains...
Prelude Tx Unveils Early-Stage KAT6A Degrader
Looking through my #aacr26 collection of posters from yesterday. Prelude Tx have a KAT6A degrader (PRT13722) in early development to compete with inhibitors: https://t.co/23zBtCX8Hs
Personalized CRISPR Poised to Become Standard Care
How individualized CRISPR genome editing can go from rare, expensive use to broader accessibility and a standard of care by @UrnovFyodor and Sadik Kassim @Nature https://t.co/ddc5ASPPAK https://t.co/GOFneIyuai
Progress Against Pancreatic Cancer, Part One
Revolution Medicines reported that its RAS‑targeting small molecule daraxonrasib more than doubled overall survival for patients with metastatic pancreatic ductal adenocarcinoma, extending median survival to 13.2 months versus 6.7 months on standard chemotherapy. The drug works by stabilizing a novel...
Merck Expands AI Push; Roche Reports MS Drug Data
Merck adds to pharma’s AI push; Roche details MS drug results https://t.co/VmpWyLhP9A $MRK $RHHBY $TGTX $RCUS $GILD #biotech
FDA Approval Could Ignite Peptide Boom and Doping Grey Zone
Peptide space is going to blowup if FDA approves it. Interesting grey area for sports doping.
Immunotherapy Drug Helps Bladder Cancer Patients Avoid Major Organ Removal
A phase‑2 trial led by NYU Langone Health found that adding pembrolizumab (Keytruda) to standard chemotherapy, radiation, and surgery allowed 60% of patients with muscle‑invasive bladder cancer to avoid cystectomy for up to two years. The study, the largest of...
Redefining ‘First‑in‑Class’ Amid Faster Competing Therapies
How do we define “first in class” when there are others further ahead? This example is from Atheron Therapeutics w/ a CCNE1 degrader #aacr26 https://t.co/s22biREUrt
CDC Vaccine Efficacy Report on Halved Winter Hospitalizations Shelved
The #CDC report that showed the #Covid vaccine halved the risk of a recipient needing emergency care or hospitalization this winter has been shelved by HHS, @bylenasun reports. https://t.co/tCNx8LSIKn
Lilly, Novo Dip as Proposed Medicare Coverage for GLP-1 Pilot Thrown Off Balance
The Centers for Medicare & Medicaid Services (CMS) has indefinitely postponed its BALANCE financing model, a pilot intended to expand Medicare coverage for GLP‑1 obesity drugs. The delay triggered a roughly 2% drop in Eli Lilly’s share price and a 4%...
Expanding the Toolkit: Non-Statin Therapies, Broader Populations, and Closing the Treatment Gap
The 2026 ACC/AHA guideline now gives strong, Class I recommendations for non‑statin therapies—bempedoic acid, PCSK9 antibodies, ezetimibe and inclisiran—for patients who cannot tolerate statins. It endorses combination regimens, such as a moderate‑intensity statin plus ezetimibe, which the RACING trial showed matches...
INBRAIN Neuroelectronics Completes Patient Recruitment for First-in-Human Study Evaluating Its Graphene Cortical Interface
INBRAIN Neuroelectronics announced that patient recruitment is complete for its first‑in‑human trial of a graphene‑based cortical interface. Ten patients were enrolled, and eight underwent surgery without any peri‑operative device failures, yielding complete datasets. The study, run with the University of...
MSD-Backed Ray Locks in $125m to Back Eye Drug Pipeline
Ray Therapeutics announced a $125 million Series B round, led by Janus Henderson Investors with participation from Merck’s venture arm, MRL Ventures and Novo Holdings. The capital will fund late‑stage clinical work and commercial preparation for RTX‑015, its lead gene‑therapy candidate for...
Immunotherapy Offers Hope in Avoiding Bladder Removal for Cancer Patients
A new immunotherapy regimen combining checkpoint inhibitor pembrolizumab with standard chemoradiation has demonstrated a high rate of bladder preservation in patients with muscle‑invasive bladder cancer. In a multinational Phase III trial of 560 participants, 68% of patients avoided cystectomy at...
Amid US Ordeal, Moderna Wins EU Approval for Flu/COVID-19 Combo Shot
Moderna received European Commission approval for its mCOMBRIAX vaccine, a combined flu and COVID‑19 shot targeting adults 50 and older across all 27 EU members plus Iceland, Liechtenstein and Norway. The combo pairs Moderna’s next‑generation COVID vaccine mNEXSPIKE with the...
An ‘AI Scientist’ Can Tackle Drug R&D. What Does that Mean for Pharma?
AI agents are moving from analytical tools to autonomous coworkers in pharma, with Owkin’s K Pro platform acting as an “AI scientist” that can answer complex research questions in hours rather than weeks. The system pulls together literature, gene‑expression data, and...
New Data Build Case for Roche's Oral BTK Drug for MS
Roche reported that its oral BTK inhibitor fenebrutinib dramatically reduced relapse rates and MRI lesions in two phase 3 FENhance trials for relapsing multiple sclerosis, outperforming Sanofi's Aubagio. The drug cut annualised relapse rates by 51.1% and 58.5% and lowered inflammation...
Regeneron Reports P-III (NIMBLE) Trial Data on Cemdisiran for Generalized Myasthenia Gravis (gMG)
Regeneron presented Phase III (NIMBLE) data showing that subcutaneous cemdisiran 600 mg every 12 weeks significantly improved symptoms in adults with generalized myasthenia gravis (gMG) and anti‑AChR antibodies. Over 24 weeks, the cemdisiran arm reduced MG‑ADL scores by 4.5 points versus 2.2...
New Study Reveals CRISPR Enzyme that Responds to Human DNA Methylation
A collaborative team from Wageningen University & Research and the Van Andel Institute has identified a CRISPR-associated enzyme that senses DNA methylation, a key epigenetic mark distinguishing cancer cells from normal tissue. The enzyme selectively binds to methylated human DNA,...
Merck Steps up as 'Meaningful Competitor' To Gilead with HIV Pill Approval
Merck received FDA approval for Idvynso, a once‑daily, single‑pill regimen for virologically suppressed HIV‑1 adults, slated for U.S. launch on May 11, 2026. The drug demonstrated non‑inferior viral suppression versus continued Biktarvy use in two pivotal trials. Analysts see Idvynso...
FDA Approves MSD’s Once-Daily HIV Combo Idvynso
Merck’s Idvynso (doravirine/islatravir) received FDA approval, marking the first tenofovir‑free, non‑INSTI two‑drug regimen for HIV. The decision follows Phase III trials that demonstrated non‑inferior viral suppression compared with Gilead’s three‑drug standard Biktarvy. Idvynso targets virologically suppressed patients without prior treatment failure...
Judge Delays Purdue Pharma Sentencing, Clearing $7 B Settlement Path
U.S. District Judge Madeline Cox Arleo postponed Purdue Pharma's criminal sentencing to allow opioid victims to attend in person. The delay clears the path for a $225 million forfeiture and a $7 billion settlement from the Sackler family, which will fund government...
Ipsen’s Ojemda (Tovorafenib) Receives Conditional Approval for R/R BRAF-Altered Pediatric Low-Grade Glioma (pLGG) in the EU
Ipsen’s oral kinase inhibitor Ojemda (tovorafenib) received conditional approval from the European Commission for treating pediatric low‑grade glioma (pLGG) with BRAF fusions, rearrangements or V600 mutations. The indication applies to patients six months or older who have progressed after at...
Ractigen Therapeutics Shows 81% NfL Drop in Phase I ALS Trial
Ractigen Therapeutics announced that a single intrathecal dose of its siRNA drug RAG-17 cut plasma neurofilament light chain by 81% in a Phase I study of SOD1‑ALS patients, while showing a clean safety profile. The data, presented at the 2026...
Top Drugs at Risk of Supply Shortages: Report
The U.S. Pharmacopeia’s new vulnerable medicines report flags 100 drugs—half of which depend on a single‑country key starting material—as prone to supply shocks. While only 30 of these are in active FDA shortage, the list highlights injectables, ICU sedatives, IV...
Rapid Blood Infection Test Fails to Improve Survival
A large, open‑label randomized trial of 899 patients with gram‑negative bacteremia compared rapid antimicrobial susceptibility testing directly from positive blood cultures to standard sub‑culture testing. The rapid approach delivered susceptibility results in about 7.5 hours versus 44 hours for the conventional method,...
New Nanomedicine Approach Boosts Chemotherapy And Immune Activity In Pancreatic Cancer
A preclinical mouse study published in Advanced Science demonstrates that photoactivatable multi‑inhibitor liposomes (PMILs) can deliver irinotecan directly to pancreatic ductal adenocarcinoma (PDAC) tumors when activated by light. The regimen combines minocycline‑mediated DNA‑repair inhibition, photodynamic priming, and localized chemotherapy, boosting...
CovAngelo Accurately Models Reaction Barriers for Covalent Drug Discovery
BEIT introduced CovAngelo, a layered QM/QM/MM platform that accurately predicts activation barriers for covalent inhibitors. By combining classical molecular mechanics, quantum‑mechanical embedding (ECC‑DMET), and high‑level quantum chemistry focused on the bond‑forming event, the method captures subtle electronic and environmental effects...
The Great Unbalding. Fallen Follicles, Rise! (NY Mag)
Scientists at Pelage Pharmaceuticals unveiled PP405, a novel drug that reprograms dormant hair‑follicle stem cells to regrow thick hair on balding scalp. Early Phase 2a data released in June 2024 showed rapid regrowth in areas previously considered irreversibly lost, sparking intense...
New Study Published Evaluating PharmaSens All-in-One Insulin Patch Pump
PharmaSens AG announced that the Journal of Diabetes Science and Technology published data from the first clinical feasibility study of its niia all‑in‑one insulin patch pump, which combines insulin delivery with continuous glucose monitoring. The single‑arm trial enrolled 18 adults...
BioAegis and Prenosis Partner for Inflammatory Disease Therapies
BioAegis Therapeutics has teamed up with AI‑focused startup Prenosis to accelerate precision‑medicine approaches for inflammatory diseases. The partnership will analyze biospecimens from BioAegis’s Phase II BTI‑203 trial of recombinant human plasma gelsolin in 600 ARDS patients across Europe, Canada and the...