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.
Pharmaceutical Executive Daily: FDA Expands Approval for Arexvy
A West Health‑Gallup survey reveals that over 82 million Americans have reduced daily spending to afford healthcare, highlighting deepening affordability pressures across income levels. The FDA has broadened GSK’s RSV vaccine Arexvy to cover high‑risk adults aged 18‑49, a group responsible for roughly 17,000 hospitalizations and 277,000 emergency visits each year. The expansion is backed by a Phase IIIb trial showing immune responses comparable to older adults. Meanwhile, a commentary argues that agentic AI could shift life‑sciences commercial functions from passive analytics to autonomous execution.
Search List of Extended Use Dates to Assist with Drug Shortages
The FDA has released a searchable table of extended use dates for specific drug lot numbers, based on manufacturer‑provided stability data. These extensions allow providers and patients to continue using existing stock beyond original expiration dates, helping to mitigate ongoing...
Ketamine Therapy for Chronic Pain and Substance Misuse
Recent peer‑reviewed study of 20 adults with chronic pain and substance misuse found ketamine therapy improved pain, mood, and dependence scores. The integrated treatment was delivered within a coordinated pain program, highlighting benefits of interdisciplinary care. Findings suggest ketamine can...
Money Raised by Biopharma
Researchers at INSERM uncovered hypothalamic tanycytes as a previously unknown tau‑clearing pathway, opening fresh therapeutic avenues for Alzheimer’s and related tauopathies. Merck Sharp & Dohme (MSD) announced the synthesis and pre‑clinical testing of novel PET tracers that bind α‑synuclein, aiming...
Shilpa Biologicals, mAbTree Program Targets Immune Pathway in Rare Blood Cancers
Shilpa Biologicals and mAbTree Biologics received FDA orphan drug designation for an investigational monoclonal antibody that targets an immune‑evasion pathway in essential thrombocythemia and polycythemia vera. The designation validates the novel mechanism and accelerates plans for IND‑enabling studies and first‑in‑human...
FDA Drug Safety Podcasts
The FDA’s Center for Drug Evaluation and Research has expanded its Drug Safety Podcast series, delivering concise audio briefings that coincide with official Drug Safety Communications. Recent episodes address serious liver injury linked to Ocaliva, rare hypocalcemia risks with Prolia,...
March 13 Policy Watch: FDA Streamlines Its Process for Approving Biosimilar Drugs
The FDA released draft guidance that lets biosimilar developers use foreign comparator data and, in some cases, replace clinical studies with chemical analysis, potentially cutting development costs by $20 million. Simultaneously, the agency launched the Adverse Event Monitoring System (AEMS), a...
Bacteria 4D Simulation, Safer Large Gene Insertion, uniQure Roller Coaster
The J. Craig Venter Institute unveiled a 4D, nanoscale simulation that tracks the entire life cycle of a minimal bacterial cell, marking a milestone for synthetic biology. A new gene‑editing platform designs DNA donors that dodge immune detection, enabling safer,...
Tahoe Therapeutics Builds Record Single-Cell Atlas Using Automated Pipetting Technology
San Francisco biotech Tahoe Therapeutics is building the world’s largest single‑cell atlas of cell‑chemical interactions, leveraging Integra Biosciences’ Assist Plus pipetting robot, Parse Biosciences’ Evercode scRNA‑seq kits, and its own AI analytics. Automation has increased single‑cell preparation throughput more than fivefold...
Renal Cell Carcinoma Strategic Intelligence Report
The latest ASCO GU strategic intelligence report spotlights renal cell carcinoma (RCC) as a field entering a transformative phase. Analysts highlight emerging biomarkers, novel HIF‑2α inhibitors, and evolving immunotherapy combinations as potential high‑impact developments. While these advances promise to reshape...
Federal Drug Price Reforms Are Working, Study Says
A new JAMA Internal Medicine study finds that the Inflation Reduction Act’s 2024 drug‑pricing provisions have reduced cost‑related medication nonadherence among Medicare beneficiaries. Compared with privately insured peers, seniors saw a 4.9‑percentage‑point drop overall and a 7.8‑point decline among those...
Gubra Plots Roivant-Like Model as Partnered Obesity Drugs Take Flight
Gubra, a 300‑employee Danish biotech known for its obesity drug partnerships, announced plans to adopt a Roivant‑style incubation model. The strategy will spin out a series of focused biotech companies, each targeting niche therapeutic areas beyond obesity, such as metabolic...
Can A Single Shot Save Your Heart?
Researchers have developed a self‑amplifying RNA injection that directs skeletal muscle to produce the heart‑healing peptide Nppa, dramatically reducing scar formation in pig models of myocardial infarction. The lipid‑nanoparticle‑delivered RNA sustains protein expression for at least four weeks, far outlasting...
Eurofins CDMO Alphora – Announces Development & Implementation of AI-Powered Salt and Co-Crystal Screening Software
Eurofins CDMO Alphora has launched an AI‑powered software that predicts salt and co‑crystal formation for APIs and intermediates, developed in partnership with a local university. The machine‑learning platform is now a core component of its solid‑state screening programs, delivering highly...
De Novo Enzymes Redefine Peptide Therapeutic Production
Peptide therapeutics are powerful. But making them has always been difficult. Traditional chemical synthesis can involve long routes, poor selectivity, and significant environmental impact, while biological production is often limited by the pathways evolution happened to provide. What if we could design...
Biotechs Report Regulatory Headaches, High-Stakes Catalysts During Q4 Earnings
Q4 earnings highlighted a mixed biotech landscape, with regulatory turbulence easing for some firms while high‑stakes catalysts loom. Capricor Therapeutics is preparing to resubmit its Duchenne cardiomyopathy cell therapy after the departure of FDA CBER director Vinay Prasad, and reported...
High Altitude Survival Gene Mutation Points to Strategy for Repairing Nerve Damage
Researchers identified a high‑altitude Retsat Q247R mutation that enhances myelin formation under hypoxic stress and accelerates remyelination in mouse models. The variant boosts neuronal production of the vitamin‑A‑derived metabolite ATDR, which activates the RXR‑γ pathway in oligodendrocyte progenitors. Administering ATDR...
Immutep's LAG-3 Drug Fails Phase 3 Lung Cancer Study
Immutep’s LAG‑3 fusion protein eftilagimod‑alpha failed to improve overall survival in a Phase 3 randomized study in non‑small cell lung cancer. The trial, enrolling roughly 600 patients, did not meet its primary endpoint and showed no statistically significant benefit versus standard...
Ultragenyx’s Gene Therapy Hits Key Endpoint in Phase 3 Trial for Blood Ammonia Disease
Ultragenyx announced that its investigational gene therapy for ornithine transcarbamylase (OTC) deficiency met a primary endpoint in a Phase 3 trial involving 37 patients. The study demonstrated a statistically significant reduction in blood ammonia levels, the key marker of disease...
Rare Disease Sales To Soar to $400B+ By 2032 as Small Molecules Resurgent: Evaluate
Orphan‑drug sales are projected to exceed $400 billion by 2032, more than double the 2025 level. Small‑molecule therapies dominate the pipeline, accounting for 45% of the 20 most valuable orphan candidates, signaling a resurgence after years of biologic focus. The Inflation...
Parkinson’s Research Reaches “Pivotal” Stage, but Barriers Remain
Parkinson’s research has entered a pivotal phase, driven by deeper disease insights and advanced models such as patient‑derived iPSCs. Despite a pipeline of potential disease‑modifying therapies, funding shortfalls and outdated trial endpoints continue to impede progress. Parkinson’s UK’s Virtual Biotech...
LabConnect Expands Global Central Laboratory Infra with Wuxi, China Facility
LabConnect announced the grand opening of a new central laboratory in Wuxi, China, expanding its global network to eight sites. The facility, built with Teddy Laboratory (now Frontage Laboratories), integrates LabConnect’s technology‑driven logistics and data oversight with local expertise. It...
CBD and CBG Reverse Fatty Liver in Mice
I teach medical students that fatty liver disease (MASLD) affects 1 in 3 adults and has limited approved drug treatments. That may be changing. Hebrew University researchers found CBD and CBG -- two non-psychoactive cannabis compounds -- reversed fatty liver in mice...
Immutep Investors Spooked by LAG-3 Failure in Lung Cancer
Australian biotech Immutep saw its ASX shares tumble more than 88% after the independent data monitoring committee recommended halting its pivotal phase‑3 TACTI‑004 trial of the LAG‑3 inhibitor eftilagimod alfa (efti) in first‑line non‑small cell lung cancer. The trial, which...
Otsuka Pharmaceutical Reports OLE Study Data on Repinatrabit in Phenylketonuria
Otsuka Pharmaceutical disclosed early open‑label extension (OLE) data for repinatrabit (JNT‑517) in adolescents with phenylketonuria (PKU). A 75 mg twice‑daily regimen achieved a 67% mean reduction in blood phenylalanine by day 56, with responses observed across prior sapropterin responders, non‑responders, and a...
Eliquis Proves Safer than Xarelto for Patients with Deep Blood Clots
A head‑to‑head trial published in the New England Journal of Medicine compared Eliquis (apixaban) and Xarelto (rivaroxaban) in patients with deep‑vein thrombosis. The study found Eliquis significantly reduced major bleeding risk while maintaining equal efficacy in preventing clot recurrence. This...
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HEALTH INEQUITY: Red Cross Children’s Hospital Doctor Using Donated ‘Miracle’ Cystic Fibrosis Drug to Save Lives
South Africa’s Red Cross Children’s Hospital is using donated Trikafta, a $300,000‑a‑year cystic fibrosis therapy, to keep seven‑year‑old Jaylin Leitjies alive after his lungs were severely damaged in infancy. The drug, produced by Vertex Pharmaceuticals, is not registered with SAHPRA...
Market‑First Synthetic Biology: Validate Demand Before Scaling Science
When Erum Khan asked me why 80% of biotech companies fail at execution, I replied: "The majority of synthetic biology founders have become enamored with the tool, the technology that they developed and then they went out there with that hammer...
China Biotech Receives FDA Approval for First Cell Therapy Trial
With FDA go ahead, a China #biotech notches a first in cell therapy testing https://t.co/QorwJmou8w by @realJacobBell $XENE $BHVN
Pharma Pulse: SteinCares Partners to Expand Biosimilar Access in Latin America and Eli Lilly Issues Warning Over Compounded Tirzepatide Safety
SteinCares and Shilpa Biologicals have signed a licensing deal to commercialize biosimilars across Latin America, with SteinCares handling regional registration and distribution while Shilpa oversees product development and long‑term manufacturing. The partnership targets broader patient access to cost‑effective biologics in...
Cancer Vaccines Show Promise for Hard-to-Treat Tumors
Cancer vaccines are showing marked efficacy vs refractory cancers and are going to be part of the future Rx armamentarium. Beyond that, ultimately, for prevention in high-risk individuals. A new stellar review @NatureMedicine https://t.co/PhtdZDtSBl https://t.co/okmwCD8AyB
Amyloid Antibody Therapy Triggers 100 Microhemorrhages in APOE4 Patient
100 brain microhemorrhages in a patient with cerebral amyloid angiopathy and 2 copies of APOE4 after amyloid antibody therapy https://t.co/ASpVuijFNW https://t.co/hdtwtv1usq
PsiQuantum and National Cancer Center Japan Partner on Quantum Computing
PsiQuantum has signed a research agreement with Japan's National Cancer Center to explore utility‑scale quantum computing for oncology drug discovery. The partnership will focus on developing fault‑tolerant quantum algorithms and clinically relevant applications using PsiQuantum's Construct platform. It also brings...
LAG‑3 Checkpoint Suffers Another Trial Setback, Focus Shifts
NEW: Another failure in the clinic in the world of LAG-3, a target once seen as possibly the next major checkpoint inhibitor after PD-1 and CTLA-4. My latest on Immutep's Phase 3 disappointment, as LAG-3 focus now shifts to a...
Biotech Layoffs Hit Evotec, Vistagen; Immutep Shares Plunge
Evotec, Vistagen lay off staff; Immutep shares collapse on study failure https://t.co/OikmCdJ6sg $EVO $VTGN $IMMP - 81% $RARE $ATRA
Pilatus Biosciences Doses First Patient in PLT012 Antibody Trial
Pilatus Biosciences has begun dosing the first patient in a Phase I, open‑label trial of PLT012, its first‑in‑class anti‑CD36 monoclonal antibody, at Next Oncology in Houston. The FDA recently issued IND clearance along with orphan‑drug status for hepatocellular carcinoma and fast‑track...
Key Challenges and Strategies for Conducting Ex‑US Trials
"Ex-US Clinical Trials: Tribulations, Preparations, and Expectations" - new From The Trenches blog from @ArthurTzianabos, CEO of Lifordi and seasoned drug developer, on the considerations for ex-US clinical trials... https://t.co/IZ6RUVKrZj
Correct Lung Target, Yet IMMP’s
Lung (not head & neck) was the right choice for $IMMP to pursue in phase 3, but it still failed. Stock -89%. Sory via @APEXONCO -> https://t.co/Y1ilGP5Aqq
The Role of AI in Large-Scale Drug Manufacturing: Current Applications and Future Trends
Artificial intelligence is reshaping large‑scale pharmaceutical manufacturing by automating process design, real‑time control, and supply‑chain management. Machine‑learning models can simulate optimal temperature, pressure and reactant levels, accelerating scale‑up and reducing waste. AI‑driven advanced process control continuously adjusts bioreactor conditions, while...
FDA Approves GSK Arexvy for At‑risk Adults 18‑49
#GSK Arexvy RSV Vaccine approved by US FDA with a wider indication for Adults aged 18 to 49 who are at increased risk of Lower Respiratory Tract Disease caused by RSV.
STAT+: Trump Is Getting More Credit than Biden for Efforts to Lower Drug Prices
A recent KFF poll shows 41% of Americans believe the Trump administration’s policies will lower prescription drug costs, outpacing awareness of the Biden‑era Medicare price‑negotiation law. Support is sharply partisan—79% of Republicans versus 11% of Democrats share this view. Only...
How to Optimize Your Biotech Company for Partnering, Licensing, and Business Success
Janita Good, a Fieldfisher partner with a D.Phil. in biochemistry, offers biotech leaders a roadmap for maximizing partnership and licensing value. She emphasizes initiating pharma discussions early, embedding commercialization plans into R&D, and aligning fundraising expectations with realistic exit timelines....
PRISM BioLab and Receptor.AI Partner to Develop a Drug Discovery Platform
PRISM BioLab has teamed with Receptor.AI to build an AI‑driven, physics‑guided platform for discovering orally available small molecules that target intracellular protein‑protein interactions, membrane proteins, and complex receptor systems. The collaboration fuses PRISM’s PepMetics technology—3‑dimensional scaffolds that mimic α‑helix and...
CHMP Backs Expanded Indications for Uplizna in NMOSD, IgG4-RD and gMG
On 11 December 2025 the EMA’s CHMP issued a positive opinion to extend Uplizna’s (inebilizumab) marketing authorisation to three additional autoimmune conditions: neuromyelitis optica spectrum disorder (NMOSD), immunoglobulin G4‑related disease (IgG4‑RD) and generalized myasthenia gravis (gMG). The recommendation designates Uplizna as monotherapy for...
Ultragenyx Reports the P-III (Enh3ance) Trial for DTX301 AAV8 Gene Therapy in OTC Deficiency
Ultragenyx announced Phase 3 Enh3ance data for its DTX301 AAV8 gene therapy targeting ornithine transcarbamylase (OTC) deficiency. At week 36, the therapy lowered 24‑hour plasma ammonia by 18% versus placebo and kept levels in the normal range, even as scavenger drug use...
Natera Launches Zenith Genomics in the US to Diagnose Rare Diseases
Natera announced the commercial launch of Zenith Genomics, a next‑generation whole‑genome sequencing (WGS) assay aimed at diagnosing rare and ultra‑rare diseases in the United States. The platform pairs standard WGS with long‑read sequencing confirmation to capture complex genomic features such...
BIG Summit Panel Breaks Down Risks and Opportunities Coming Out of DC
At the inaugural BIO BIG Summit, a Washington Policy Brief panel dissected the shifting U.S. healthcare landscape for biotech. The discussion highlighted progress on reauthorizing the Prescription Drug User Fee Act (PDUFA) and emerging pharmacy benefit manager (PBM) reforms, while...
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...
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...