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.
Is FDA Moving the Goalposts on 483 Responses? What the New Draft Guidance Means for Your Company
The FDA released its first draft guidance outlining how drug, biologic and veterinary manufacturers should respond to Form FDA 483 observations. The document mandates a structured response—including an executive summary, risk assessments, and detailed remediation plans—and requires identification of the response preparer with authorization letters if external counsel is used. It reinforces the 15‑business‑day submission window while allowing interim reporting for incomplete corrective actions. By tying response quality to the broader quality‑system performance, the guidance signals a more holistic enforcement approach.
Toward the Simultaneous Detection of Multiple Diseases with a Highly Cost-Effective Cell-Free DNA Methylome Test
Researchers introduced MethylScan, a low‑cost cell‑free DNA methylome sequencing assay that profiles the entire cfDNA methylome from a single blood draw. In a cohort of 1,061 individuals, the test achieved an AUROC of 0.938 for multicancer detection (63.3% sensitivity at...
Testosterone Isn’t a Magic Cure-All for Middle Age
Social media influencers have promoted testosterone therapy as a cure‑all for perimenopausal women, promising energy, sharper cognition, and a revived libido. Medical experts caution that the only proven benefit is a modest increase in sexual satisfaction for postmenopausal women, and...
Cancer Immunotherapy Works Better Earlier in the Day
Advanced Science News highlighted three breakthrough studies: a fluorescent sensor that provides real‑time detection of E. coli in catheter bags, enabling earlier intervention for urinary tract infections; a systematic analysis of lipid‑nanoparticle components that clarifies how each interacts with cells, paving...
Portugal’s Biotech Industry Is Growing Up
Portugal’s biotech sector is shedding its niche reputation, with turnover among trade‑group members more than tripling between 2016 and 2020 and over half of firms earning the majority of revenue from exports. A multi‑node cluster model spanning Cantanhede, Porto, Braga,...
FDA Reversals in Rare Disease Space Highlight Confusion Around External Controls
In 2024 the FDA signaled support for using natural‑history external controls in rare‑disease gene‑therapy trials, but later reversed that stance for uniQure’s Huntington’s therapy, demanding a sham‑surgery Phase 3 study. The agency’s guidance still encourages innovative designs, yet recent reversals for...
Viewpoint — ‘Miracle’ Peptides: Regulatory Greyzone and RFK, Jr. Propaganda Opens the U.S. to a Perilous Biohacking Experiment
Robert F. Kennedy Jr. announced on Joe Rogan’s podcast that the FDA plans to move roughly 14 experimental peptide compounds from a restricted status to availability through compounding pharmacies. While over 100 peptide drugs are FDA‑approved, the compounds marketed by...
Lean Derisking: Smart Ways to Cross Drug Development’s “Valley of Death”
API’s recent webinar highlighted practical strategies to bridge the drug‑development "valley of death," emphasizing early derisking from discovery through first‑in‑human studies. The panel stressed using AI‑driven in‑silico filters, staged in‑vitro and animal testing, and aligning preclinical models with clinical biomarkers...
Tohoku University Unveils Nanoscale Creatinine Sensor Delivering Results in 35 Seconds
Researchers from Tohoku University and the City College of New York announced a chemiresistive biosensor that quantifies creatinine in urine within about 35 seconds. The device uses a platinum‑nanoparticle polymer composite and a three‑enzyme cascade, covering a clinically relevant range...
Exercise‑linked Protein Target Could Rejuvenate Aging Blood‑brain Barrier
Scientists Find a Mechanism for How Exercise Protects the Brain 🗣️Finding drugs to trim proteins like TNAP could be a new way to rejuvenate the blood-brain barrier, even after it’s been degraded by age. “We’re uncovering biology that Alzheimer’s research has largely...
Psilocybin Single Dose Beats Nicotine Patches for Quitting
A single dose of psilocybin is more effective than nicotine patches for quitting smoking, study suggests https://t.co/APDQt1WYxp https://t.co/zDnXcUjFyG
Morning Brief Podcast: Pharma's AI Reckoning
The Economic Times podcast examined how artificial intelligence is reshaping pharma, highlighting AlphaFold’s ability to shrink protein‑structure projects from months to weeks and Lupin’s rollout of generative AI across more than 90 data repositories. Guests from PwC India, Dr. Reddy’s and...
EU Launches PsyPal Trials Using Psychedelics to Ease Palliative Care Distress
On April 13, 2026, the European Union’s Directorate‑General for Health and Food Safety inaugurated the PsyPal project, a €‑funded research programme that will run clinical trials of psychedelic‑assisted therapy for patients facing psychological distress at the end of life. The...
Ex‑FDA Officials Say RFK Jr. Mischaracterizes 2023 Peptide Ban as Illegal
Former FDA officials contend that Health and Human Services Secretary Robert F. Kennedy Jr. is falsely portraying the agency’s 2023 ban on 19 peptide drugs as illegal. They warn that a reversal could legitimize unsafe, untested compounds circulating in a...
Why the US Needs a Unified, Mission-Based Strategy for Health Innovation
The United States’ decades‑old linear research model—government funding, academic discovery, private commercialization—has driven breakthroughs like the Internet and vaccines, but today market‑driven incentives are skewing biomedical innovation toward high‑profit areas such as oncology. This has left critical fields like psychiatry...
Orexin Receptor Antagonists for Major Depressive Disorder: Perspectives From a Systematic Review and Meta-Analysis
A systematic review and meta‑analysis of orexin receptor antagonists (QXR‑ANTs) in adults with major depressive disorder found a modest but statistically significant reduction in overall symptom scores (standardized mean difference –0.16) and a 52% increase in remission rates compared with...
Scientists Find Hidden Brain Cells Helping Deadly Cancer Grow
Canadian researchers have uncovered that oligodendrocytes, a type of brain support cell, actively promote glioblastoma growth by signaling through the CCR5 receptor. In laboratory models, interrupting this communication dramatically slowed tumor expansion. The team also identified Maraviroc, an FDA‑approved HIV...
Pinnacle Medicines Adds $89M for Oral Peptides With Properties of Injectable Biologics
Pinnacle Medicines announced an $89 million Series B financing, bringing its total capital to $134 million, to advance an AI‑driven platform that designs orally bioavailable peptide drugs. The startup aims to launch its lead asthma and chronic obstructive pulmonary disease program in human...
SIRT1 Activation Shows Promise for Age‑related Brain Disorders
SIRT1 Activators as Geroprotective Agents in Brain Aging: Mechanisms and Therapeutic Potential "...SIRT1 could be a promising pharmacological target for age-associated brain disorders, warranting more robust translational studies to validate these findings in humans..." https://t.co/hGVxZ4RErM
Trump Unveils 100% Pharma Tariff, Jefferies Says Indian Generics Likely Safe
U.S. President Donald Trump announced a sweeping 100% tariff on certain pharmaceutical imports, prompting alarm across the supply chain. A Jefferies analysis released days earlier warned of the move but projected that Indian generic manufacturers would remain largely exempt, limiting...
Psilocybin Slows Down Human Reaction Times and Impairs Executive Function During the Acute Phase of Use
Researchers conducted a systematic review and multilevel meta‑analysis of 13 studies, finding that psilocybin dose‑dependently slows reaction times during its acute phase. While low to medium doses cause mild delays, high doses produce moderate to severe slowing, especially in basic...
Immunome's CSO Sells 9,438 Shares Ahead of Q2 FDA Filing for Lead Therapy
Immunome's chief scientific officer, Jack Higgins, sold 9,438 shares of the company on April 2, 2026, reducing his direct stake by 30%. The sale, executed under a pre‑arranged 10b5‑1 plan, coincides with Immunome's preparation to file an NDA for its...
Cures Emerge From Incremental Advances, Not Single Breakthroughs
The media makes it seem like cures are one breakthrough away. In reality, cures come from smaller breakthroughs and improvements made by the work of thousands of scientists, drug developers and doctors. We’re witnessing this evolution in real time with...
Integrated Analysis Identifies Disulfidptosis Related Tumor Antigens and Molecular Subtypes in Hepatocellular Carcinoma for mRNA Vaccine Development
Researchers developed a disulfidptosis‑based framework for hepatocellular carcinoma (HCC) that combines molecular subtyping, mRNA vaccine design, and prognostic modeling. Analysis of TCGA‑LIHC and GEO data identified 32 disulfidptosis‑related genes that separate HCC into two subtypes with distinct survival, immune infiltration,...
India’s Commerce Secretary Urges Shift From Low‑cost Generic Hub to Global Quality Pharma Leader
India’s Commerce Secretary Rajesh Agrawal told industry leaders the country must move beyond its low‑cost image and become a globally recognised quality pharma producer. With a $60 bn domestic market and $28 bn in exports, the push aims to lift the sector...
Scientist with Rare FUS ALS Mutation Enrolls in Preventive Gene Therapy Trial
Jeff Vierstra, a 41‑year‑old scientist, has been receiving experimental spinal infusions that silence a rare FUS mutation for three years, making him the first known person to undergo gene‑targeted therapy before any ALS symptoms. The treatment, run at Columbia University’s...
The Peptide Economy vs the Healthcare AI Economy: Which Side of the Trade Matters More
The essay contrasts the rapidly expanding peptide economy—led by GLP‑1 and next‑generation obesity drugs—with the burgeoning healthcare‑AI sector, arguing they are interdependent rather than competing. Peptide revenues are projected to surpass $200 billion annually by 2030, while AI revenues sit at...
Daratumumab Boosts Revlimid Efficacy in Smoldering Myeloma
#EAonc EAA173 - Daratumumab to Enhance Therapeutic Effectiveness of Revlimid in Smoldering Myeloma (DETER-SMM) - PI: @nsc_natalie https://t.co/VtBMJUjI5X Activated: Apr 30, 2019 #mmsm @eaonc #NCT03937635 @VincentRK @mweissmdphd https://t.co/fFVUqNssyX
Phase 3 Trial Tests Daratumumab
.@SWOG S2213 Ph3 RCT Dara-VC Induction Followed by ASCT or Dara-VCD Consolidation & Daratumumab Maintenance in Pts w/ Newly Diagnosed AL Amyloidosis [Activated: 12/1/23] https://t.co/OizUfJCc2c #mmsm #bmtsm https://t.co/ClwxVAhSy0
Study Finds Single Psilocybin Dose Reshapes Personal Values for Up to Three Months
Researchers at King’s College London reported that a single dose of psilocybin produced lasting changes in participants' core values, including greater appreciation for life and self‑acceptance, measured up to 85 days after the experience. The findings link acute feelings of...
Eli Lilly’s New GLP‑1 Pill and Retatrutide Threaten Novo Nordisk’s Obesity Lead
Eli Lilly’s newly approved oral GLP‑1 obesity drug, orforglipron, hits U.S. pharmacies on April 6, offering a pill without food‑timing restrictions. Coupled with phase‑3 data showing its experimental triple‑agonist retatrutide can cut 28.7% of body weight, Lilly is poised to overtake...
Microfluidic Chip Review Highlights Faster Path to Cancer Immunotherapy Success
Researchers at Southern University of Science and Technology Hospital published a review in Cancer Biology & Medicine showing that microfluidic chips can model the tumor immune microenvironment more faithfully than traditional assays. The paper argues the technology could accelerate drug...
Congress Must Reform Broken IND Process for FIH Trials
Great to see FDA & the administration prioritizing FIH (First in Human) reform. The current IND process is broken & has shifted FIH trials overseas. Countries like Australia have relied on a more flexible, notification-based pathway for years. Now Congress...
Eli Lilly’s Retatrutide Targets Novo Nordisk’s GLP‑1 Stronghold with 28.7% Weight Loss Data
Eli Lilly’s sub‑cutaneous triple‑hormone agonist retatrutide shaved an average 28.7% of body weight in a phase‑3 trial, positioning the drug as a direct challenge to Novo Nordisk’s GLP‑1 portfolio. The move comes as Lilly also rolls out its oral GLP‑1 pill orforglipron,...
Urea‑Activated Nanocarrier Enables Targeted SGLT2 Inhibition for Metabolic and Kidney Disease
A team led by Ren, Gao and Yun introduced a urea‑activated nanocarrier that releases SGLT2 inhibitors only where urea is elevated, delivering precise metabolic rescue in animal models of cardiovascular‑kidney‑metabolic syndrome. The approach promises to cut systemic side effects and...
Levetiracetam Blocks Amyloid Beta via SV2a‑dependent APP Modulation
Levetiracetam prevents Aβ production through SV2a-dependent modulation of APP processing in Alzheimer’s disease models "...supporting the hypothesis that protein accumulation is a relevant pathogenic event in amyloid pathology..." https://t.co/bRqoZP1Qyf @ScienceTM
Low‑Dose ATG Extends Honeymoon Phase in Type 1 Diabetes, Study Finds
A Lancet‑published trial of 117 children and young adults shows that a low dose of antithymocyte globulin (ATG) can preserve pancreatic beta‑cell function for a year, extending the disease’s “honeymoon” phase while cutting side‑effects by more than half. The finding...
Weekly Reads: 1st FDA-Approved CIRM Supported Therapy, Asymmetric Histone Inheritance, Stem Cell Retraction
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I (LAD‑I) in children without a matching bone‑marrow donor. This marks the first FDA‑approved product directly funded by California’s Institute for Regenerative...
Re: RSV Vaccination Programme Expanded to 3 Million More Older People
The UK health authorities have announced an expansion of the RSVpreF (Abrysvo) vaccination programme to include an additional three million adults aged 60 and older. Clinical trial data published in the New England Journal of Medicine confirm the vaccine’s ability...
Dr. Vinay Prasad Said He Would Deliver New COVID Vaccine RCTs. He Failed and Should STFU.
Pfizer and BioNTech announced they are halting a U.S. phase‑III trial of their updated COVID‑19 vaccine aimed at adults 50‑64 because enrollment fell far short of the planned 25,000‑30,000 participants. The companies said the decision was unrelated to safety or...
Dr Sorcha O'Connor: The PsilOCD Study Investigating Low-Dose Psilocybin for OCD (#532)
In this episode, host Stuart Ralph talks with Dr. Saoirse O'Connor, a neuroscientist who led the PsilOCD study—the first controlled European trial of low‑dose psilocybin for obsessive‑compulsive disorder. The conversation covers the science of psilocybin, the study design (a 10 mg...
U.S. Drug Tariffs Seen as Sparing Taiwan Prices, Supply
The United States has issued an executive order imposing up to 100% tariffs on patented drugs manufactured abroad, while offering a 20% rate for firms that relocate production to the U.S. Taiwan officials say the measure will not raise drug...
Wake Forest Launches $5.7M MORPH Study on ‘Movement Medicine’ for Older Adults
Wake Forest School of Medicine has started a five‑year, $5.7 million NIH‑funded MORPH trial to evaluate a low‑impact “movement medicine” protocol for seniors with osteoarthritis. The remote program uses wearables, mobile apps and weekly video groups to encourage frequent, gentle activity...
GLP‑1 Drugs Cut Heart Risk, Lower Dementia, Spark New Trials
Semaglutide and tirzepatide have shown a 20% drop in major cardiovascular events, a 24% slowdown in kidney disease progression, and a 33% lower Alzheimer’s diagnosis rate. The FDA’s expanded Wegovy label and a wave of new brain‑health trials are turning...
Machine Learning Links Spironolactone to Reduced Fibrosis Risk
Spironolactone and Fibrosis in Heart Failure Risk: Machine Learning Analysis of HOMAGE Trial Plasma Proteomics https://t.co/Ho2RL0RhO4 https://t.co/cHkogagDTt
REGENXBIO Hit with Securities Fraud Class Action; Lead Plaintiff Deadline Set for April 14
Rosen Law Firm has filed a securities‑fraud class action against gene‑therapy company REGENXBIO, alleging false statements about its RGX-111 candidate. Investors who bought shares between Feb. 9 2022 and Jan. 27 2026 must file by April 14 2026 to serve as lead plaintiff.
Real-World Safety of Second-Line Diabetes Drugs in Elderly
A 2026 Nature Communications study examined real‑world safety of second‑line diabetes drugs in patients 65 and older after metformin. Using electronic health records and claims data, the researchers compared sulfonylureas, DPP‑4 inhibitors, SGLT2 inhibitors and GLP‑1 agonists with propensity‑score matching....
GLP‑1s Massively Marked up; LLMs Priced Near Cost
One observation here: GLP-1s are priced at maybe 25-50x production cost (not counting R&D). LLM usage is priced at perhaps 1x inference cost (not counting training). In any case, two great innovations that are rapidly catching on.
China Competition Will Accelerate US Pharma, Cut Red Tape
This is great to see. Biotech and pharma competition with China is going to force us to sharpen and accelerate processes in the US, and cut red tape. And we'll get the benefit of Chinese drugs as well. Win/win.
SPRY Therapeutics Secures FDA Label Expansion for Needle‑Free Epinephrine Spray
SPRY Therapeutics (ARS Pharmaceuticals) received FDA approval on April 3 to remove age limits on its Neffy 1mg needle‑free epinephrine nasal spray, allowing use by anyone weighing 33 lb or more. The move opens the product to a broader pediatric and...