Today's Pharma Pulse
Ona lands $86.6M Series B, one of Spain’s biggest biotech financings
Spanish biotech Ona announced a $86.6 million Series B round, ranking among the largest venture financings 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 funds will expand Ona’s AI‑driven drug discovery platform and accelerate its pre‑clinical pipeline.
Merck, Pfizer’s Drug Combo ‘Rewrites the Standard of Care’ in Bladder Cancer
Merck and Pfizer’s Keytruda‑Padcev doublet delivered a dramatic survival advantage in the Phase 3 EV‑304 study of muscle‑invasive bladder cancer. The regimen cut the risk of disease progression, recurrence or death by 47% compared with gemcitabine‑cisplatin and lowered overall‑mortality risk by 35%. Pathological complete response rose to 55.8% versus 32.5% in the control arm, offering a chemo‑free option even for cisplatin‑eligible patients. The data pave the way for regulatory submissions and could reset the standard of care in this high‑risk indication.
Best Practices for Applying HDX-MS to FBLD
A recent open‑access study demonstrates that hydrogen‑deuterium exchange mass spectrometry (HDX‑MS) can reliably map binding sites of extremely weak fragment hits (up to 7 mM KD) against Cyclophilin D. By optimizing protein concentration at 10 µM and testing fragments at 2.5‑10 mM, the...
Big Pharma’s Next Entrant Could Come From China
Chinese drugmakers, led by Jiangsu Hengrui Pharmaceuticals, are emerging as global biotech contenders. Hengrui topped 2024 trial sponsorship, boasts a pipeline of 100 investigational drugs and 400 trials, and secured a $500 million partnership with GSK that could yield $12 billion. Analysts...
FDA Demands Sham‑controlled Trial for uniQure’s AMT‑130
$QURE AMT-130 update: Still blocked The FDA stated that it cannot agree that data from the Phase I/II studies, compared to an external control, are sufficient to provide the primary evidence of effectiveness required to support a marketing application for...
FDA Limits NTLA Gene Therapy to Mild Cases, Undermining Demand
$NTLA drug is off hold but now being restricted by the FDA to the least severe/sick patients, which would logically be those least likely to want/need an experimental gene editing therapy. Existing options will dominate. Target/disease selection is a...
Real-World Data Brings New Insights to Natural History of Disease Studies
Real‑world data (RWD) is reshaping natural history studies by delivering longitudinal, patient‑level information that was previously only obtainable through costly prospective trials. Advances in artificial intelligence and natural language processing now allow rapid extraction of structured data from unstructured sources...
MRK's Ovarian Cancer Claim Rejected by FDA, EMA
$MRK claims a survival benefit in ovarian cancer irrespective of PD-L1 status, but FDA & EMA say BS. Via @ApexOnco -> https://t.co/Cld1O7Uuvp #ESGO26
Ascendis’ Yuviwel (Navepegritide) Receives the US FDA Accelerated Approval for Achondroplasia
The U.S. FDA granted accelerated approval to Ascendis’ Yuviwel (navepegritide) for increasing linear growth in children aged two years and older with achondroplasia and open epiphyses. Approval rests on three placebo‑controlled trials and up to three years of open‑label extension...
Why It’s Critical to Close Open Steps in Cell Therapy Manufacturing
Cell therapy manufacturing is vulnerable to contamination because living cells cannot be terminally sterilized. Regulatory bodies such as EU GMP Annex 1 and the FDA now require risk‑based contamination control strategies that prioritize closed, sterile, and automated processes. Closing open steps...
Sanofi Receives CHMP Recommendation for Dupixent Expansion in Europe
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending Dupixent (dupilumab) for paediatric chronic spontaneous urticaria (CSU) in children aged 2‑11. The recommendation is based on robust Phase III data from the LIBERTY‑CUPID...
STAT+: Trump’s Drive to Get Europe to Pay More for Drugs Creates Uncertainty for Countries, Patients
President Trump has pressed European nations to raise their drug spending, arguing that higher foreign prices would allow the United States to lower its own prescription costs. While no clear price hikes have materialized yet, the demand has sparked uncertainty...
Opus Genetics Reports Phase I/II Trial Results of OPGx-BEST1 Gene Therapy
Opus Genetics presented early Phase I/II data for its OPGx‑BEST1 gene therapy targeting best vitelliform macular dystrophy and autosomal‑recessive bestrophinopathy. In a sentinel 63‑year‑old participant, the treatment was well tolerated and delivered a 12‑letter gain in best‑corrected visual acuity after...
BioMarin Receives FDA Approval for Palynziq Use in Adolescents
BioMarin’s enzyme substitution therapy Palynziq received FDA approval for use in adolescents aged 12 and older with phenylketonuria (PKU). The decision was based on Phase III PEGASUS trial results showing 44.4% of participants lowered blood phenylalanine below guideline levels, with a...
How America’s Former Steel Heartland Secured a $3.5bn Eli Lilly Facility
Eli Lilly announced a $3.5 billion life‑sciences manufacturing complex in Lehigh Valley, Pennsylvania, slated to open in 2031. The facility will produce the company’s next generation of weight‑loss drugs and employ roughly 850 engineers, scientists and technicians. The deal, the largest life‑sciences...
18 UK Biotech Companies You Should Know About
The article profiles 18 UK biotech firms spanning oncology, immunology, regenerative medicine and AI‑driven drug design, highlighting recent milestones such as Amphista’s $30 million upfront deal with BMS, Autolus’ FDA and EU approvals for obe‑cel, and Isomorphic Labs’ $600 million raise to...
X4 Pharmaceuticals’ Xolremdi (Mavorixafor) Receives the CHMP Positive Opinion for WHIM Syndrome
X4 Pharmaceuticals’ Xolremdi (mavorixafor) received a positive opinion from the European CHMP, recommending approval under exceptional circumstances with an EC decision expected in Q2 2026. The recommendation is based on the global Phase III 4WHIM trial involving 31 patients aged 12 and...
Ipsen Reports the CHMP Positive Opinion for Ojemda (Tovorafenib) for R/R BRAF-Altered Pediatric Low-Grade Glioma (pLGG)
European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a positive opinion recommending conditional approval of Ipsen’s Ojemda (tovorafenib) as monotherapy for pediatric low‑grade glioma with BRAF fusions, rearrangements or V600 mutations. The recommendation is based on...
FDA Action Alert: BMS, GSK, Aldeyra and More
The FDA will render decisions this March on five high‑profile drug applications, including two delayed from last year. Bristol Myers Squibb seeks a psoriatic arthritis label for Sotyktu, backed by Phase 3 data showing a 54.2% ACR20 response. Aldeyra, Rhythm, GSK...
What’s Your Cold Chain Testing Telling You?
Altor is hosting a free webinar on March 12, 2026 to teach cold‑chain professionals best practices in thermal testing. The session will cover how nuanced variables affect test outcomes and which questions to ask to validate designs. Speakers include Altor’s Director of...
Quanterix Corp (QTRX) Q4 2025 Earnings Call Transcript
Quanterix (QTRX) reported FY 2025 revenue of $16.1 million, down $11 million from the prior year, driven by steep declines in collaboration and contract‑manufacturing income. R&D spending fell modestly to $140.7 million, while SG&A rose to $65.5 million due to higher professional fees for...
Xeris Biopharma Holdings Inc (XERS) Q4 2025 Earnings Call Transcript
Xeris Biopharma reported record Q3 2025 product revenue of $74.1 million, a 40% year‑over‑year increase driven primarily by Recorlev’s 109% revenue jump. The company posted its first quarterly net income and an adjusted EBITDA of $17.4 million, reflecting strong operating leverage and...
Uniqure NV (QURE) Q4 2025 Earnings Call Transcript
UniQure reported a regulatory setback as the FDA now deems the Phase 1/2 external‑control data for AMT‑130 insufficient for a Biologics License Application, creating uncertainty around the U.S. filing timeline. Despite this, the high‑dose AMT‑130 achieved a statistically significant 75% slowdown...
Nuvation Bio Inc (NUVB) Q4 2025 Earnings Call Transcript
Nuvation Bio reported $7.7 million net product revenue for Iptrozi in Q3 2025, driven by 204 new patient starts and payer coverage expanding to over 80% of U.S. lives. Clinical data showed an 89% overall response rate and a median...
Urogen Pharma Ltd (URGN) Q4 2025 Earnings Call Transcript
UroGen Pharma reported over $127 million in cash at the end of Q4 2025, affirming sufficient liquidity to reach profitability if current plans hold. The company highlighted steady month‑over‑month growth in Zasturi enrollment, with community treatment share now around 35‑40% and institutional...
NMN Restores NAD, Shields Heart via SIRT3
According to ITOA, NAD declines with age, causing epigenetic changes & disease susceptibility In 2010, we showed NAD-dependent enzyme, SIRT3, protects the heart 🐁 New study says NAD precursor NMN protects the heart via SIRT3 in mice fed a Western diet https://www.sciencedirect.com/science/article/pii/S0022316626000842
OpenADMET Releases Massive CYP Assay Datasets for Community AI
OK. Here it is. Over the last year, the @OctantBio and OpenADMET team have been hard at work developing scalable, quantitative, data-rich, and low-cost methods for assessing CYP reactivity and inhibition. The interplay between building a data engine and building...
Fresh Formats, a Debut Event Director, and a New Viennese Venue for LogiPharma 2026
LogiPharma 2026 will debut in Vienna’s Austria Center, moving from France after four years. Under new event director Ben Sharples, the conference introduces interactive panels, Oxford‑style debates and hands‑on training through the LogiPharma Academy. The program splits into two interconnected...
Ginkgo's 2025 Revenue Far Misses $7.6B Promise
Ginkgo reported full year 2025 revenue of $170M at the end of last week. When it went public at a $15B valuation in 2021, they guided to investors that they’d be adding $7.6B in new business PER YEAR in 2025...
Drug Discovery Bottleneck? Cell-Free Platform Screens Peptides Faster, Even in Harsh Conditions
Researchers at the Innovation Center of NanoMedicine unveiled PL‑display, a cell‑free platform that immobilizes individual peptides on magnetic beads for rapid screening. The method delivers over ten‑fold efficiency gains versus traditional cell‑based displays and can operate under high‑temperature, high‑salt, or...
GoodRx Launches Employer Program to Help Subsidize High-Cost Brand Drugs
GoodRx introduced GoodRx Employer Direct, a new service allowing employers to directly subsidize the manufacturer‑sponsored price of high‑cost brand medications such as GLP‑1 drugs without adding them to health‑plan formularies. The model, first piloted with retailer Hy‑Vee, lets employers contribute...
🎧 What Investors Get Wrong About Hims & Hers, Ep. 2 with Paul Cerro
In this episode, the hosts dissect why investors misunderstand Hims & Hers' (HEMS) gross margin potential, emphasizing that as a compounder without its own API manufacturing, HEMS cannot achieve margins higher than drug manufacturers. They illustrate how HEMS' margin trajectory...
Weekly Reads: Longevity Hype Vs. Cool Research, Losing Your Y, FDA Maelstrom
This week’s coverage juxtaposes longevity hype with emerging science, highlighting David Sinclair’s bold claim that aging could be reversed within 10‑20 years, a new Nature paper confirming adult hippocampal neurogenesis, and research showing men lose the Y chromosome as they...
Hidden Ingredient in Ozempic and Wegovy Tablets Raises New Gut Health Questions
Researchers at Adelaide University conducted the first in‑vivo study of salcaprozate sodium (SNAC), the absorption enhancer used in oral semaglutide tablets such as Ozempic and Wegovy. Over a 21‑day period in rats, repeated SNAC exposure reduced fibre‑degrading gut bacteria, lowered...
Welireg Poised to Broaden Reach per GU26 Data
$MRK Welireg could be about to expand its reach at last. #GU26 data via @ApexOnco with relevance to $RCUS -> https://t.co/k59284INdK
Miami Preps for Biotech Week; BiotechTV Covers Barclays Conference
Looks like Miami is getting ready for biotech week (the week after next). Note: BiotechTV will be broadcasting from the Barclays conference March 10 & 11. https://t.co/ToZH0EgOVz
Epic's First Pharma Deal
Epic Systems announced its first pharmaceutical customer, Eli Lilly, for the newly launched Epic Discovery platform. The deal marks the inaugural sale of a Health Grid product to a life‑sciences firm, allowing Lilly to streamline clinical trial creation, site identification, activation,...
In China, Phase 2 Study of Relma-Cel in R/R MCL Finds Durable Responses
A phase‑2 trial of relmacabtagene autoleucel (relma‑cel), a CD19‑directed CAR‑T therapy, enrolled 59 Chinese patients with relapsed/refractory mantle‑cell lymphoma after BTK‑inhibitor failure. The study reported a 71.2% overall response rate and a 59.3% complete response rate, with median time to...
IL11 Deficiency Causes Female Infertility, Low Weight, Osteoporosis
But note IL11 knockout mice are infertile (females) and have low body weight and some osteoporosis. Still interesting angle though.
Paper on Target Trial Emulation Overlooks Confounding by Indication
How is it that a paper that claims to show how to do target trial emulation does not address confounding by indication and its ramifications for data collection? https://t.co/dzGZZdpBoc #Statistics
Analysis Finds Efficiencies, Savings of Using a Single Bispecific for DLBCL and FL
A new analysis quantifies the operational and financial benefits of using Genmab’s epcoritamab, a dual‑indication bispecific antibody, for both relapsed/refractory diffuse large B‑cell lymphoma (DLBCL) and follicular lymphoma (FL). In a community‑practice model of 100 patients, the study projects 3,110...
Accelerating CAR Engineering Shows Promising Cancer Immunotherapy
Some very good news about engineering our cells vs cancer (accelerating the CAR). A short thread 1. For background, a new 5★ review on cancer immunotherapy @Cancer_Cell https://t.co/1Qz2rCs8I5 https://t.co/sJ6BwaXyjk
Single-Cell Atlas Links Marrow Immune Dysregulation to Myeloma Outcomes
A single-cell atlas characterizes dysregulation of the bone marrow immune microenvironment associated with outcomes in multiple myeloma https://t.co/5t0M2eX0fB https://t.co/p3kv8LEzrC
Mixed Immune Signature Identified in Chronic Hand Eczema
A phase‑2b trial enrolling 94 adults with chronic hand eczema (CHE) without etiologic pre‑selection uncovered a mixed immune signature spanning type 2, type 3 and type 1 pathways. Dupilumab, an IL‑4Rα antagonist, delivered a 59.8% mean improvement in modified Total Lesion Symptom Score...
GLP-1 News Galore; Top 100 Venture Investors; Doug Ingram to Step Down; and More
This week’s Endpoints Weekly highlighted a surge of GLP‑1 developments, including new trial data and expanded indications that reinforce the class’s dominance in obesity and diabetes treatment. The newsletter also released its annual Top 100 venture investors list, showing a notable...
EMA’s CHMP Recommends Three New Orphan Drugs, Rebuffs Two FDA-Approved Programs
The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended six new medicines, including three orphan drugs already approved by the FDA—Ojemda for pediatric low‑grade glioma, Palsonify for acromegaly, and Xolremdi for WHIM syndrome. The agency also gave a...
Emerging Models Point to a New Operating System for Rare Disease Innovation
BioCentury’s cookie policy details five categories of cookies—strictly necessary, functional, marketing, advertising, and analytics—each serving distinct purposes on its website. Strictly necessary cookies support authentication, registration, and navigation, while functional cookies enable personalization of services. Marketing and advertising cookies help...
![[Review] Autosomal Dominant Polycystic Kidney Disease](https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/e5c772f073be33a83118f46a1ac91f13.webp)
[Review] Autosomal Dominant Polycystic Kidney Disease
Autosomal dominant polycystic kidney disease (ADPKD) remains the most common hereditary cause of chronic kidney disease, imposing substantial morbidity, mortality, and healthcare costs worldwide. Recent advances in molecular genetics and high‑resolution imaging have sharpened diagnostic criteria and enabled more accurate...
![[Comment] Safeguarding Genomic Integrity in Pluripotent Stem-Cell Therapies](https://hixhlmpcokxhartfkpyi.supabase.co/storage/v1/object/public/images/thumbnails/fba52b2aab9c591bf0e059f57acce9ea.webp)
[Comment] Safeguarding Genomic Integrity in Pluripotent Stem-Cell Therapies
Human pluripotent stem cells (hPSCs) are emerging as a transformative platform for Parkinson’s disease, with recent phase I/II trials showing successful engraftment of hESC‑ and iPSC‑derived dopaminergic neurons. Yet extensive data reveal that cultured hPSCs frequently acquire recurrent genetic lesions—most...
STAT+: Minnesota Report Shows Large Hospitals Continue to Dominate the 340B Drug Discount Program
Minnesota’s Department of Health reports that hospitals and clinics in the state earned at least $1.34 billion in 2024 from the 340B drug discount program. Participants received $3.045 billion in discounted medicines but paid $1.53 billion plus $165 million in administration fees. The largest...
The Science of Controlling Drug Release in Implants with Ultrasonic Spray Coating
Ultrasonic spray coating is emerging as a core engineering discipline for drug‑eluting implants, with parameters such as drug‑to‑polymer ratio, nitrogen carrier‑gas flow, nozzle height, and spray power dictating coating thickness, profile, and elution behavior. The article explains how variations in...