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.
STAT+: Virginia Lawmakers Push a New Approach to a Prescription Drug Affordability Board
Virginia lawmakers are proposing a prescription‑drug affordability board that will use Medicare’s annually negotiated drug list as its benchmark. The board would also impose upper payment limits, creating a ceiling on what insurers pay for those medicines. This strategy would make Virginia the first state to target every Medicare‑selected drug and to have authority to set payment caps, surpassing the nine states with existing boards. Advocates argue the model could drive deeper price reductions than current state efforts.
Pharmaceutical Executive Daily: Novo Nordisk Commits $506 Million to Ireland Manufacturing Site
Novo Nordisk announced a $506 million investment to expand its manufacturing site in Ireland, aiming to boost capacity for diabetes and obesity therapies. The expansion addresses rising global demand and enhances supply‑chain resilience. The announcement coincides with industry focus on measuring...
SpotSee-Controlant Collaboration Addresses Shipment Monitoring Gaps in Temperature-Sensitive Supply Chains
SpotSee and Controlant have launched a strategic partnership that combines SpotSee’s WarmMark QR temperature indicators with Controlant’s real‑time IoT monitoring devices and cloud analytics. The joint solution creates a layered, end‑to‑end visibility platform that captures environmental data during transit and...
Intralesional Cemiplimab Shows Promise as a Nonsurgical Alternative for Early-Stage CSCC
A phase‑1 pilot of low‑dose intralesional cemiplimab in early‑stage cutaneous squamous cell carcinoma (CSCC) demonstrated rapid tumor regression with visual objective response rates of 66.7%–75% and pathologic complete responses of 58.3%–66.7%. Safety was favorable, with no grade ≥ 3 events and minimal...
Tirzepatide Delivers up to 21% Weight Loss, Dose‑dependent
Tirzepatide: In the SURMOUNT-1 trial, weight reduction percentages were dose-dependent: 5 mg: ~15.0% weight reduction. 10 mg: ~19.5% weight reduction. 15 mg: ~20.9% weight reduction (Jastreboff et al., 2022). https://www.nejm.org/doi/10.1056/NEJMoa2206038 https://www.gatlan.com/ @GatlanHealth 
FDA Approves Navepegritide for Children With Achondroplasia
The FDA granted accelerated approval to navepegritide (Yuviwel), a once‑weekly CNP prodrug, for children aged two years and older with achondroplasia and open growth plates. In the phase 2b ApproaCH trial, the drug raised annualized growth velocity to 5.89 cm/year, a 1.49 cm/year...
New Clinical Data Highlight Povorcitinib’s Potential to Achieve High-Threshold Lesion Clearance in HS
New post‑hoc analyses of the phase 3 STOP‑HS1 and STOP‑HS2 trials show that oral povorcitinib, a selective JAK1 inhibitor, delivers rapid, high‑threshold lesion clearance in patients with severe hidradenitis suppurativa. In the 75 mg arm, up to 57% of participants achieved complete...
Kyowa Kirin Abandons Touted Eczema Drug Following Safety Review
Kyowa Kirin announced it will cease all clinical trials of rocatinlimab, an anti‑OX40 antibody once touted as a potential eczema blockbuster, after a safety review identified emerging malignancy signals. The review, conducted with former partner Amgen, uncovered a confirmed and...
Novel Mechanism for Parkinson’s Is Linked to ATP Deficiency
Scientists at Ludwig Maximilian University discovered that loss of the DJ-1 protein triggers ATP deficiency in human dopaminergic neurons, leading to reduced VMAT2 levels and impaired dopamine vesicle loading. The resulting dopamine oxidation fuels accumulation of pathological α‑synuclein species, a...
For Patients With mCRPC, Results With Pluvicto in Real-World Settings Keep Pace With Clinical Trials
Real‑world evidence from Duke’s PRECISION platform shows Pluvicto (Lu‑177 vipivotide tetraxetan) delivers a median progression‑free survival of 13.5 months in PSMA‑positive metastatic castration‑resistant prostate cancer (mCRPC) after androgen‑receptor pathway inhibitor (ARPI) therapy, matching the pivotal PSMAfore trial. Patients who received Pluvicto...
Pharma Pulse: FDA Grants Priority Review to Rusfertide and Expands Approval for Novo Nordisk’s Sogroya
The FDA granted priority review to Takeda’s rusfertide, a first‑in‑class hepcidin mimetic for polycythemia vera, after Phase III trials more than doubled response rates. A regulatory decision is expected in Q3 2026. The agency also expanded Novo Nordisk’s once‑weekly Sogroya to treat children...
FDA Drug Competition Action Plan
The FDA’s Drug Competition Action Plan (DCAP) continues to drive generic drug competition by streamlining standards for complex products, closing loopholes that allow brand‑name companies to delay approvals, and improving the overall ANDA review process. Aligned with GDUFA III, the plan...
Europe’s Pharma Trade with the US Remains Critical Despite Tariff Turmoil
U.S. pharmaceutical tariffs on EU imports have risen to 15% after a brief 10% measure, reviving trade tensions despite a prior MFN deal. Europe is pursuing alternative markets, notably a new EU‑India free‑trade agreement that could eliminate up to 11%...
Ruxolitinib Cream Shows Strong Efficacy and Reassuring Safety in New Analyses
New integrated safety data from 20 trials confirm that ruxolitinib 1.5% cream delivers a low incidence of serious infections, cardiovascular events, thromboembolic events, and malignancies across atopic dermatitis, vitiligo and other inflammatory skin conditions. A phase 3b trial in adults with...
Newly Added Guidance Documents
The FDA has published a batch of newly added guidance documents, spanning drug exclusivity, post‑approval safety reporting, real‑world data studies, Bayesian trial methods, and patient‑preference research. Ten documents are highlighted, with six in draft form and four finalized, dated between...
Webinar: The Future Is Collaborative: Transforming Clinical Trials
A MEDSIR‑hosted webinar titled “The Future is Collaborative: Transforming Clinical Trials” examined how collaborative‑initiated trials and investigator‑initiated trials (IITs) are reshaping oncology research. Speakers Dr. Javier Cortés and Dr. Antonio Llombart‑Cussac, both leading breast‑cancer oncologists, discussed their roles in recent...
From Prompt to Pill: Researchers Propose AI-Driven Path To ‘Pharmaceutical Superintelligence’
A team of researchers proposes "pharmaceutical superintelligence," an AI‑driven pipeline that can turn a plain‑language request into a preclinical drug candidate and even outline clinical trial plans. The framework stitches together generative chemistry models, multi‑agent reasoning, and robotic labs into...
MaxLife Technologies Inc. Dba Maxlife - 721453 - 02/20/2026
The FDA issued a warning letter to MaxLife Technologies for false and misleading claims on its website about compounded semaglutide and tirzepatide products. The company labeled the drugs as “generic compounded medication” and implied FDA approval and that MaxLife was...
Synthetic Gene Medicines May Disrupt DNA Repair
Researchers at Karolinska Institutet reported that phosphorothioate antisense oligonucleotides (ASOs) directly bind key DNA‑repair enzymes, forming nuclear condensates that trigger a false DNA‑damage response. The effect was observed at concentrations commonly used in laboratory assays, though clinical doses reach lower...
'Dismal' Survival Demands Change in Bile Duct Cancer Therapy
An international panel of 147 oncology leaders, including Prof. John Bridgewater, issued a consensus calling for sweeping reforms in the diagnosis, treatment, and research of cholangiocarcinoma (bile duct cancer). The disease remains deadly, with three‑quarters of patients dying within a...
Nine Companies Advancing Biotech in Philadelphia
Philadelphia’s biotech ecosystem, long known for CAR‑T breakthroughs, is diversifying into obesity, metabolic disease, gene editing, and solid‑tumor immunotherapies. Nine local companies illustrate this shift, from Alveus Therapeutics’ $197 million Series A obesity program to Cabaletta Bio’s autologous CD19 CAR‑T for autoimmune...
Key Biosimilars Events of February 2026
February 2026 saw a surge of biosimilar activity, with key regulatory approvals for products such as Sandoz Enzeevu (Eylea), Accord Filkri (Neupogen), and STADA Gotenfia (Simponi) across the US, Canada and the EU. Companies forged new licensing and commercialization deals that extend biosimilar...
Quell Takes New Treg Into Clinic After Transplant Study Halt
Quell Therapeutics has launched the phase 1/2 CHILL trial of its autologous CAR‑Treg therapy QEL‑005 in rheumatoid arthritis and systemic sclerosis, after pausing the liver‑transplant program QEL‑001. The multi‑center study will enroll patients in the UK, Germany and Spain, with read‑out...
Boehringer Ingelheim Axes MASH Pact With OSE After Disappointing Mid-Stage Data
Boehringer Ingelheim has terminated its metabolic dysfunction‑associated steatohepatitis (MASH) collaboration with OSE Immunotherapeutics after an exploratory Phase 2 trial failed to meet its primary endpoint. The original 2018 deal included a €15 million upfront payment and up to €1.1 billion in milestone potential,...
DDW Highlights: 3 March 2026
In this episode, Bruno Quinney highlights four major developments: a long‑acting injectable HIV regimen (cabotegravir + rilpivirine) that cut virological failure risk by nearly half versus daily oral therapy; SolasCure’s ORES wound gel, which accelerated debridement 22‑fold and healing seven‑fold in chronic...
Lynk Pharmaceuticals Reports Topline P-III Trial Data on Zemprocitinib in Atopic Dermatitis
Lynk Pharmaceuticals announced topline Phase III data for its oral JAK inhibitor zemprocitinib in 356 patients with moderate‑to‑severe atopic dermatitis. Both the 12 mg and 24 mg doses met the co‑primary endpoints at week 16, delivering 38‑46 percentage‑point improvements in EASI‑75 and roughly 30 percentage‑point gains...
Animal Drugs: Strengthening Federal Incentives Could Help Address Unmet Animal Health Needs
The GAO report reveals that, despite a conditional approval pathway introduced in 2004 and expanded in 2018, FDA‑approved animal drugs for minor species and niche uses remain scarce. From FY2018 through FY2025, only 13 drugs received conditional approval, all targeting...
FDA Doubts QURE Gene Therapy Benefit, Leaks to Media
New reporting from me taking you inside the $QURE - FDA Huntington's gene therapy imbroglio. I spoke to a senior FDA official late yesterday, who told me agency reviewers "are not convinced there’s any therapeutic benefit of the product. If...
STAT+: FDA Is ‘Not Convinced’ UniQure’s Huntington’s Therapy Has Benefit, Senior Official Says
The FDA concluded that UniQure’s experimental gene therapy for Huntington’s disease has not demonstrated therapeutic benefit based on existing clinical data. Reviewers said they are not persuaded by the evidence, prompting the agency to block the company from submitting a...
AI‑Discovered Gal‑3 Blocker Cuts Inflammation, Boosts Memory in Mice
Alzheimer’s research is increasingly looking beyond plaques and tangles to the brain’s immune response. A study published in Elsevier's Pharmacological Research journal used AI to screen 1.6M compounds for a Galectin-3 (Gal-3) blocker that could cross the brain barrier. In Alzheimer’s-like...
Prime Medicine Pursues Approval After Two-Patient Gene Editing Trial
Prime Medicine $PRME to seek approval for gene editing treatment after two-patient trial https://t.co/nBnrUzqSCN via @Jasonmmast
Innovent Receives the NMPA Approval for Jaypirca to Treat R/R Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma (CLL/SLL)
Innovent’s pirtobrutinib, marketed as Jaypirca, received Chinese NMPA approval for adults with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma who have undergone at least one prior therapy, including a BTK inhibitor. The decision follows the phase‑III BRUIN CLL‑321 trial,...
Pierre Fabre Pushes to Revive US Cell Therapy Approval
Pierre Fabre seeks to revive US approval chances for spurned cell therapy https://t.co/gJeklT8asd @ByJonGardner $ATRA $QURE $RGNX
FDA Guidance Boosts Biotech, HHS Calls It Fake
Did the FDA’s individualized therapy guidance put a spike in the heart of a startup biotech? Fake news, says the HHS. Yeah, it did, says an insider. Exclusive from @RLCscienceboss and @Jared_Whitlock https://t.co/IN9o4VIp03
The Role of the cGAS-STING Interaction in the Age-Related Inflammation of the Brain
The cGAS‑STING pathway, a DNA‑sensing immune circuit, becomes aberrantly activated in the aging brain as mitochondrial and nuclear DNA escape into the cytoplasm. This chronic activation drives low‑grade neuroinflammation, contributing to neurodegenerative diseases such as Alzheimer’s, Parkinson’s and ALS. Preclinical...
LLM Medical Research Overwhelmingly Lacks Real Clinical Data
A new systematic review of LLMs in medicine @NatureMedicine @ekoermann https://t.co/npvDeqUvel “Despite thousands [4,609] of publications since late 2022, only a small fraction use real clinical data and just 19 randomized trials exist.”
Integrating Omics Data, Not Algorithms, Is Pharma’s Biggest Challenge
The hardest problem in pharma data science isn't the algorithm. It's getting three different omics datasets to agree with each other. That's exactly what we'll dig into at #ddpeast26 on June 10 in Waltham. I'm joining as a panelist and roundtable moderator....
Patent Term Extension: Challenges with Defining and Claiming Approved Biologics
Patent term extension (PTE) offers up to five extra years of exclusivity for FDA‑approved drugs, but biologics present a unique hurdle because the law hinges on defining the “active ingredient.” Unlike small molecules, biologics are large, variable structures, making it...
T‑cell Engagers Offer New Autoimmunity Treatment Opportunity
T-cell engagers for autoimmunity. Listen to Ken Song describe the opportunity as CEO of San Diego-based Candid Therapeutics. Sponsored by @AlphaSenseInc & Dash Bio. https://t.co/Ct2viH00c4
APGE Should Drop OX40 Bispec, It's Just a Black Box
$APGE should can the OX40 bispec IMO. You’re basically just tacking on a black box with that MOA at this point…
CMS Extends Deadline for MFN Pricing Pilot Scheme
The U.S. Centers for Medicare & Medicaid Services (CMS) has pushed back the deadline for manufacturers to join its GENEROUS pilot, extending the initial cut‑off to April 30 while keeping the final deadline at June 30. The voluntary program applies a Most‑Favoured...
Industry Leaders Announce World’s First Microphysiological Systems Industry Association
Eight leading European microphysiological systems companies have launched the Industry Alliance for Microphysiological Systems (IAMPS), the world’s first trade association dedicated to MPS technologies. IAMPS will represent organ‑on‑chip, organoid and related NAM developers, aiming to harmonize standards, promote data sharing,...
Merck and Eisai Present P-III (LITESPARK-011) Trial Data on Welireg + Lenvima in Advanced Renal Cell Carcinoma (RCC) at ASCO...
Merck and Eisai disclosed Phase III LITESPARK-011 results showing that the oral combination of Welireg (belzutifan) and Lenvima (lenvatinib) outperformed cabozantinib in 747 patients with advanced renal cell carcinoma who progressed after anti‑PD‑1/PD‑L1 therapy. At a median follow‑up of 29...
Dermatology Trials by the Numbers: Key Trends and Benchmarks for 2026
Dermatology research is booming, with more than 16,000 trials completed, ongoing, or planned by the end of 2025. The Asia‑Pacific region now hosts roughly 8,000 studies, a five‑fold increase over the past decade, matching North America and Europe combined. Low...
Cryo-EM Reveals New Aspects of CRISPR-Cas Biology
Researchers at Vilnius University used cryogenic electron microscopy to map eleven CRISPR‑Cas protein complexes, including three variants of a newly described Cas9‑Cas1‑Cas2‑Csn2 supercomplex. The study shows that Cas9, traditionally viewed only as a DNA‑cutting enzyme, also directs the selection and...
Clinically‐Relevant Static Magnetic Field Induces Release of Encapsulated Molecules From Magnetoliposomes
Researchers demonstrated that a clinically‑available 1.5 T static magnetic field can trigger the release of encapsulated molecules from magnetoliposomes (MLs). The study used citric‑acid‑stabilized Fe₃O₄ nanoparticles, with and without a chitosan coating, and monitored structural changes via SAXS and DLS. Fluorescence...
A Strong Dose of Digital for U.K. Pharma and Life Sciences
Earlier this year the UK government pledged £82.6 million to AI‑focused drug‑discovery firms, underscoring its ambition to lead the global digital health transformation. Major players such as AstraZeneca are launching dedicated health‑tech units like Evinova to accelerate clinical‑trial design, while the...
How North Carolina Attracted Amgen and Roche To Become a Next-Gen Obesity Drug Production Hotspot
North Carolina’s Holly Springs is becoming a hub for next‑generation obesity drugs as Amgen and Roche commit more than $3.5 billion to build manufacturing capacity. Amgen’s $1.55 billion rollout includes a new drug‑substance plant and a second facility slated for 2032, while Roche’s...
#596: Why Do Omega-3 Trials Show Mixed Results?
Omega‑3 supplementation trials produce mixed results due to differences in dose, population risk, and chosen endpoints. High‑dose EPA/DHA studies in secondary‑prevention cohorts with elevated triglycerides, such as REDUCE‑IT, show significant cardiovascular benefit, whereas lower‑dose primary‑prevention trials like VITAL and ASCEND...
Navigating the FDA After the Storm To Advance Drug Candidates
After a wave of senior departures and a 3,500‑position cut, the FDA entered 2026 with a largely inexperienced workforce, raising uncertainty for drug developers. The agency’s new AI guidance, released in January, outlines best practices but emphasizes human oversight and...