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.
Viewpoint — ‘Completely Unethical’: RFK, Jr.’s Medical Ignorance Deprives Melanoma Cancer-Sufferers of a Life-Saving Therapy
Replimune’s RP1, a promising therapy for metastatic melanoma, received an initial FDA advisory panel recommendation for approval, but the agency’s biologics chief Vinay Prasad overruled the decision and halted the drug’s launch. At a House hearing, Health and Human Services Secretary Robert F. Kennedy Jr. repeated inaccurate claims that the trial involved chemotherapy, a statement fact‑checked as false. Critics argue the FDA’s reversal reflects bias and political pressure, while oncologists cite real‑world cases where RP1 has prolonged patient lives.
Muons, Massive Waves and Restored Sight: The Winners at the ‘Oscars of Science’ – Podcast
The Breakthrough Prize, dubbed the "Oscars of science," handed out $3 million awards in physics, mathematics and life sciences at a high‑profile ceremony in Los Angeles. Jean Bennett was among the laureates, receiving the life‑sciences prize for co‑creating the first FDA‑approved gene‑augmented...
GLP-1 Weight Loss Drugs Trigger a Life-Threatening Brain Condition by Depleting Vitamin B1
A recent analysis of FDA adverse‑event reports and case studies linked 15 instances of Wernicke encephalopathy—a severe, vitamin B1‑deficiency brain disorder—to the use of GLP‑1 weight‑loss drugs such as semaglutide and tirzepatide. Most patients experienced prolonged nausea, vomiting, and rapid weight...
Accelerating Drug Discovery with Fragment Screening
Scientists at DOE’s Brookhaven National Laboratory are piloting a publicly‑available fragment‑based drug design (FBDD) platform at the NSLS‑II synchrotron. Using macromolecular X‑ray crystallography, the program couples robotics, automation and AI to screen small chemical fragments against protein targets. Early tests...
Crowdfunded Rapamycin‑Exercise Trial Shows Promising Aging Data
Five years ago, @BradStanfieldMD reached out with an idea: a crowdfunded clinical trial testing rapamycin combined with exercise in older adults. The results are now published — and Brad and I just sat down for a full 42-minute breakdown...
Perth Biotech at Cutting Edge of the Future of Medicine
Syngenis, a Perth‑based biotech, is converting its research‑grade oligonucleotide lab into Australia’s first GMP‑certified facility, enabling local production of clinical‑grade DNA and RNA strands. The move could bring back roughly AUD 160 million (about US 105 million) of annual overseas GMP work, tapping into...
US Health Secretary Kennedy Defends Trump’s Mathematically Impossible Drug Discount Claims
U.S. Health Secretary Robert F. Kennedy Jr. defended President Donald Trump’s mathematically impossible drug‑discount claims during a Senate Finance Committee hearing, insisting a drop from $600 (≈ $566 USD) to $10 represented a 600% reduction. The assertion contradicts basic percentage math, as the actual discount...
What Does DOJ’s New Corporate Enforcement Policy Mean for the FDA- and DEA-Regulated Industry?
The Department of Justice released its first department‑wide Corporate Enforcement and Voluntary Self‑Disclosure Policy (CEP), establishing a uniform framework for prosecuting corporate crime. The policy lets companies obtain a declination of charges if they voluntarily disclose misconduct, fully cooperate, remediate...
NSW Opens $96M RNA Research and Manufacturing Facility
New South Wales has launched a $96 million (≈$63 million USD) RNA research and manufacturing hub at Macquarie University’s Innovation Precinct. The state‑run facility, operated by Aurora Biosynthetics, will produce RNA‑based vaccines and therapeutics from a single site. NSW pledged an additional...
Nanomerics Secures US Patent Extending MET Platform Protection Into the 2040s
Nanomerics Ltd. has been granted a US patent that stretches the protection of its MET nanomedicine delivery platform into the 2040s. The filing secures the company’s core ocular and nose‑to‑brain drug pipelines and signals confidence in commercializing its nanotech‑based therapies.
Shingles Vaccine Cuts Dementia Risk by Half.
Have you had your shingles shot? A major 2026 study of over 300,000 people age 65 and older found that the shingles vaccine reduces the risk of dementia by up to 50%. Remarkably, men, women, and folks across age and ethnic...
Researchers Explore New Approach to Multivirus Drug Development
Researchers at Stanford Medicine, led by Shirit Einav, are pioneering a host‑targeted antiviral strategy that disables human enzymes essential for viral replication rather than attacking the virus directly. Their recent Nature Communications paper describes a small‑molecule, RMC‑113, which halted replication...
Chicken Gene-Editing Advance Opens Path to Drug-Producing Eggs
University of Missouri researchers used CRISPR to insert a gene cassette into the chicken housekeeping gene GAPDH, overcoming epigenetic silencing that has hampered stable transgenic poultry. The inserted reporter stayed active for months of cell division, proving continuous expression. This...
FDA Notification Regarding Dehydrated Alcohol
The FDA has issued a formal notice telling companies to stop distributing any dehydrated alcohol products that are not the FDA‑approved Ablysinol from Belcher Pharmaceuticals. Ablysinol, approved on June 21, 2018, is the only legally marketed dehydrated alcohol in 1 mL and 5 mL...
Paxlovid Showed No Hospitalization Benefit in Vaccinated Seniors
In 2 randomized, open-label trials of Paxlovid there was lack of evidence of reduced hospitalizations among the participants, who were age 50+ with coexisting conditions, and who were vaccinated. The endpoint was very low (<1.2%) in the treatment and control...
Fenebrutinib Shows Promise, Yet Safety Concerns Loom
Relevant to $TGTX was the Roche $RHHBY fenebrutinib trial results recently announced. Excellent efficacy, but two cases of Hy's Law. Both resolved, but a patient might not be so lucky next time. Hard for me to see this gaining widespread...
CMS Delays Part D Portion of BALANCE Model on Expansion of GLP-1 Access
On April 21, the Centers for Medicare & Medicaid Services announced that the Medicare Part D component of the Better Approaches to Lifestyle and Nutrition for Comprehensive Health (BALANCE) Model will be delayed until 2027 pending additional evaluation and data collection....
TGTX Shows Consistently Low Relapse Rates Across Trials
This very low relapse rate in this $TGTX Phase IV observational trial is consistent with that seen in their 6-year extension study: https://t.co/Shvb8h8VRr
Washington Post Publishes AHA Letter in Response to Anti-340B Editorial
The American Hospital Association (AHA) President Rick Pollack wrote a letter to the Washington Post defending the 340B drug pricing program after an editorial called for its repeal. Pollack argues the program is vital for delivering free or low‑cost medications,...
GDUFA III Drug Master File (DMF) Review Enhancements
The FDA’s GDUFA III reauthorization adds a mechanism for early review of Type II API drug master files up to six months before an ANDA or prior‑approval supplement is filed. A draft guidance explains how DMF holders can request a “DMF prior...
Mabwell’s Nectin‑4 ADC Shows Pseudoprogression in Cervical Cancer Case Report
Mabwell announced that a case report on its Nectin‑4‑targeting ADC 9MW2821, published in The New England Journal of Medicine, documented a rare hepatic pseudoprogression in an advanced cervical cancer patient. The report highlights dramatic tumor marker decline and lesion resolution,...
KUKA FLEX CBD May Be Harmful Due to Hidden Drug Ingredient
The FDA issued a safety alert after laboratory testing revealed that KUKA FLEX CBD, sold online and in some stores for joint pain, contains the prescription NSAID diclofenac, which is not disclosed on the label. Diclofenac can trigger cardiovascular events, serious gastrointestinal...
Curcuflex May Be Harmful Due to Hidden Drug Ingredients
The U.S. Food and Drug Administration has warned consumers to avoid Curcuflex, a joint‑pain product sold online and in some stores, after laboratory testing revealed it contains the prescription drugs dexamethasone and diclofenac, neither of which appear on the label....
Top Protein Degrader Session at AACR26 Highlights Await
Excellent protein degrader session today at #AACR26. Third speaker was all Do Not Post (sorry @HartungIngo !) so some highlights from the other three speakers coming up...
RM Joe May Be Harmful Due to Hidden Drug Ingredients
The FDA has issued a safety alert that the joint‑pain product RM Joe, sold online and in some stores, contains undeclared dexamethasone phosphate and diclofenac. Laboratory testing confirmed the hidden prescription‑drug ingredients, which can trigger severe steroid‑related and NSAID‑related side effects....
Alzheimer's Drugs Show Minimal Benefit, Review Reveals Deeper Issues
Alzheimer’s drugs offer little benefit, major review finds – and the reasons go deeper than the science https://t.co/cnMPAEQumY https://t.co/wtg8llquON
FDA Criticizes Replimune's Melanoma Trial Design
Some observers, including @US_FDA, have criticized @Replimune for the design of the trial that led to the decision not to grant accelerated approval of their melanoma treatment. I asked the company's Chairman about that view. Watch the full interview: https://t.co/XYVmSeodLv https://t.co/OO8eXTZOXh
Yeicob Ácido Hialurónico May Be Harmful Due to Hidden Drug Ingredients
The FDA has warned that Yeicob Ácido Hialurónico, marketed for joint pain, contains undeclared dexamethasone phosphate and diclofenac. Laboratory testing confirmed these prescription‑grade drugs are hidden in the supplement’s label. The agency advises consumers to stop using the product and...
The BioPharm Brief: Early Design Risks, Oncology Signals, and a Biosimilars Power Move
The BioPharm Brief highlights three pivotal biopharma trends: early‑stage process design flaws can cripple cell and gene therapy scale‑up, leronlimab demonstrates early clinical and biomarker activity in metastatic colorectal cancer, and Amneal Pharmaceuticals’ acquisition of Kashiv BioSciences creates a vertically...
STAT+: At AACR, Talk of Chinese Biotech, Oncology’s Comms Issue, and More
Revolution Medicines highlighted two key updates at the AACR meeting: promising frontline pancreatic cancer data for its RAS inhibitor daraxonrasib and the introduction of a novel compound, RM-055. RM-055 is described as a catalytic inhibitor that can strip a phosphate...
FDA Warns Device Manufacturers of Nitrosamine Impurities that Could Cause Cancer
The FDA’s Center for Devices and Radiological Health has issued a warning to manufacturers of drug‑device combination products about the presence of nitrosamine impurities, specifically 1‑methyl‑4‑nitrosopiperazine, detected in rifampin‑impregnated devices. The agency highlighted that catheters, cardiac‑implant envelopes, and other devices...
CVS Claims It’s on the Verge of Losing All Its Pharmacies in Tennessee
CVS Health warns that the Tennessee Fair Rx Act, now passed by the state Senate, would force it to shut down all more than 100 retail pharmacies and 25 MinuteClinic locations in the state. The bill bars companies that own...
An Experimental New Drug for Stiff Person Syndrome Restores Mobility
Researchers at Kyverna Therapeutics reported that a single infusion of their experimental CAR‑T cell therapy, miv‑cel, dramatically improved mobility in patients with stiff person syndrome (SPS). In a Phase II trial of 26 participants, walking speed increased and eight of twelve...
BioMérieux Unveils BIOFIRE SPOTFIRE Molecular Testing Solution for Biopharma
bioMérieux has launched BIOFIRE SPOTFIRE, a molecular testing system aimed at biopharma quality control. The instrument delivers mycoplasma results in less than an hour, leveraging automated workflows, touchscreen operation, and barcode scanning. Designed to be compact and stackable, it integrates with...
Ocrelizumab in PPMS. Complementing Regulatory History with a Decade of Clinical Evidence
Roche’s ocrelizumab (Ocrevus) is the only FDA‑approved disease‑modifying therapy for primary progressive multiple sclerosis (PPMS). Since its 2017 approval based on the ORATORIO trial, a decade of data—including the 2025 O’HAND study and ten‑year extension analyses—has confirmed robust efficacy across...
Pharmaceutical Executive Daily: Idvynso Receives FDA Approval
The FDA granted approval to Idvynso, a new oral therapy for HIV‑1 infection in adults, citing trial data that showed sustained viral suppression and a safety profile on par with existing regimens. The drug adds to a shifting HIV market...
Why Early Process Design Is Key to Cell and Gene Therapy Success
At INTERPHEX 2026, Charles River’s Dr. Alan Smith warned that cell and gene therapy developers often prioritize rapid first‑in‑human studies at the expense of scalable manufacturing. He highlighted recurring gaps where insufficient early‑stage process design forces costly redesigns during later trials or...
10x Science: The Founders Who Built the Field Are Now Building the Platform
10x Science is launching an AI‑driven platform that automates molecular characterization of biologic drugs, a step traditionally performed manually by PhD scientists using outdated software. The company’s founders—two Stanford‑trained researchers with Nobel‑linked publications and a veteran YC entrepreneur—bring deep scientific...
Marijuana to Be Reclassified Alongside Ketamine, Steroids
"The new categorization would put it in line with ketamine and steroids." well it's a start
FDA Clinical Trials Training Modules
The FDA’s Center for Drug Evaluation and Research has released new clinical‑trial training modules that capture the agency’s latest regulatory intelligence, emerging guidance, and real‑world compliance experience. The curriculum serves as a global benchmark, helping organizations demonstrate regulatory excellence and...
Relaxing the Rules: How Claim Type Supported Patent Validity for Teva Pharmaceuticals’ “Headache Patents”
The Federal Circuit reversed a lower‑court ruling that invalidated Teva Pharmaceuticals’ “headache patents,” holding that the method‑of‑treatment claims satisfied written description and enablement requirements. The court emphasized that the claims were directed to using humanized mouse antibodies, not the antibodies...
Pharma-Backed Petition Urges FDA to Overhaul Rejection Letters
New Pharma-backed petition calls on FDA to reform how it releases drug rejection letters, says current process is unlawful - https://t.co/XLwCxt1FY6
Study Finds Rapamycin May Undermine Exercise Gains in Seniors
An international team led by Brad Stanfield reported that a weekly 6 mg dose of rapamycin blunted the functional gains from a 13‑week home exercise program in 40 sedentary adults aged 65‑85. The placebo group outperformed the rapamycin group on chair‑stand,...
Scientists Found a Chemical That Could Help Regrow Your Hair. It Might Be In Your Pantry.
Researchers from China and Australia have created a dissolving microneedle patch that combines minoxidil with stevioside, a natural sweetener from the Stevia plant, to improve hair‑loss treatment. The stevioside acts as a solubilizing agent, enhancing minoxidil's water solubility and skin...
Dayspring Pharma’s CG2001 Foam Hits Primary Endpoint in Phase II AGA Trial of 110 Chinese Men
Dayspring Pharma announced that its CG2001 foam achieved the primary efficacy endpoint in a 30‑week Phase II study of 110 Chinese men with androgenetic alopecia. The combination of 5% minoxidil and 0.075% finasteride delivered roughly 50% greater hair‑growth results versus historic...
PK/PD Crucial for Next-Gen ADC Development
Wonderful tour de force on ADCs by @raffcolo highlighting the importance of PK/PD going forward with new formats https://t.co/nUpBNNoVil
GLP‑1 Weight‑Loss Drugs Face Safety Scrutiny as Oral Pills Expand in India
Clinicians caution that GLP‑1 injectables such as Ozempic and Wegovy can trigger gastroparesis and nutritional deficits in patients with chronic disorders, even as one in eight Americans now uses them. At the same time, a wave of oral GLP‑1 tablets...
Leronlimab Shows Early Clinical and Biomarker Activity in Metastatic Colorectal Cancer at AACR 2026
CytoDyn presented early Phase 2 results showing that leronlimab combined with TAS‑102 and bevacizumab markedly reduced circulating tumor DNA in metastatic colorectal cancer patients. CCR5 expression was confirmed in all prescreened tumors, and median ctDNA fell about 70% by week two...
Veeva Systems Beats Q4 Forecast, Posts $836M Revenue, Highlights Life‑Science SaaS Momentum
Veeva Systems reported Q4 FY2026 revenue of $836 million, topping guidance and pushing full‑year revenue to $3.195 billion. The results were driven by R&D Cloud adoption and expanding Vault CRM, while the company forecast 13% subscription‑revenue growth for FY2027.
We Need More Radioactive Drugs. Can We Make Them From Nuclear Waste?
A new wave of radiopharmaceutical cancer treatments is driving unprecedented demand for radioisotopes, prompting companies to extract them from legacy nuclear waste. Researchers at the UK National Nuclear Laboratory are refining radioactive lead from stored waste, while firms like Belgium’s...