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.
Cryoport Inc (CYRX) Q4 2025 Earnings Call Transcript
CVRx reported fourth‑quarter 2025 revenue of $16 million, a modest 4% year‑over‑year increase, while gross margin improved to 86% driven by higher selling prices and manufacturing efficiencies. Net loss widened to $11.9 million as SG&A and R&D expenses outpaced revenue growth, but the company ended the year with $75.7 million in cash and a revised debt facility extending to 2031. The firm launched the 2,500‑patient BENEFIT HF trial, secured CMS coverage, and benefited from the new Category I CPT code that stabilizes reimbursement. Guidance for 2026 projects $63‑67 million in revenue and continued margin expansion.
Cumberland Pharmaceuticals Inc (CPIX) Q4 2025 Earnings Call Transcript
Cumberland Pharmaceuticals reported fiscal 2025 revenue of $44.5 million, an 18% increase year‑over‑year, driven by a 31% Q4 surge to $13.7 million from Vibativ, Sancuso and the newly launched Talicia. Adjusted earnings turned positive at $1.7 million and operating cash flow rose to...
NDA and BLA Calendar Year Approvals
The FDA’s Center for Drug Evaluation and Research (CDER) has published its annual New Drug Application (NDA) and Biologics License Application (BLA) approval reports for calendar years 2015 through 2025, each linked in a consolidated list. These reports detail the...
Miriam Paramore, RxUtility
In a quick‑bite interview at the February 2026 VIVE conference, Miriam Paramore discussed RxUtility, a health‑tech platform that consolidates manufacturer coupons and cash‑price data to present consumers with the lowest possible drug price at the point of dispensing. The solution...
One PSMA Agent Outshines Competitors at Detecting Prostate Cancer Recurrence
A head‑to‑head study of 55 post‑prostatectomy patients compared two FDA‑cleared PSMA PET agents, Posluma (flotufolastat F‑18) and Pylarify (piflufolastat F‑18). Posluma demonstrated markedly lower bladder radioactivity, with a median bladder SUV of 10.9 versus 29 for Pylarify. This reduction translated into higher...
How Flatworms Keep Their Regeneration Powers on Track
Scientists have identified a molecular checkpoint that guides planarian stem cells during regeneration. The roundabout A receptor (RoboA) suppresses inappropriate pharyngeal differentiation by regulating the transcription factor FoxA, while the extracellular protein Anosmin1a partners with RoboA to fine‑tune cell identity...
FDA Adverse Event Reporting System (FAERS) Electronic Submissions
The FDA has mandated electronic submissions to the FAERS database using the ICH‑endorsed E2B(R3) standard, beginning January 16 2024 for post‑marketing drug and biologic reports and April 1 2024 for pre‑marketing IND safety reports. Companies have until April 1 2026 to transition fully, after which only...
Bysanti FDA Approval for Bipolar I and Schizophrenia Marks Vanda’s Second Win in Two Months
Vanda Pharmaceuticals received FDA approval for Bysanti (milsaperidone) to treat acute manic or mixed episodes of bipolar I disorder and schizophrenia in adults. The approval introduces a new chemical entity in the atypical antipsychotic class that is bioequivalent to Vanda’s...
FDA's Adverse Event Reporting System (FAERS)
The FDA Adverse Event Reporting System (FAERS) is a public database that collects adverse event, medication error, and product quality complaint reports for drugs and biologics after market approval. It follows the ICH E2B international safety reporting guidance and uses...
J&J Commits $1B to Next-Gen Cell Therapy Manufacturing in Pennsylvania
Johnson & Johnson announced a more than $1 billion investment to build a next‑generation cell and gene therapy manufacturing complex on a 154‑acre site in Montgomery County, Pennsylvania. Construction is slated to start in the second half of 2026, creating over...
Pharma Pulse: Regulatory Speed Records
Boehringer Ingelheim’s oral kinase inhibitor Hernexeos received FDA accelerated approval for first‑line HER2‑mutant lung cancer in a record‑fast 44 days, thanks to the National Priority Voucher program. The drug demonstrated a 76 % response rate, positioning it as a breakthrough in personalized...
Rethinking Idiopathic Pulmonary Fibrosis: Unmet Needs, Evolving Biology and the Future of Clinical Research
Idiopathic pulmonary fibrosis (IPF) continues to pose a severe, often late‑diagnosed respiratory challenge, with mortality exceeding many cancers. Boehringer Ingelheim’s Martin Beck highlighted the shift toward earlier detection using AI‑enhanced imaging and a broader view of IPF as a heterogeneous,...
Water Interactions Reveal How Surface Coatings Reshape Nanoparticle Drug Delivery
Arizona State University researchers quantified how water interacts with biomolecule‑coated magnetite nanoparticles, revealing that surface coatings dramatically reshape hydration energetics, immune recognition, and drug‑delivery performance. Using a calorimetry‑gas adsorption system, they measured water adsorption on particles coated with bovine serum...
Generate Biomedicines’ IPO Brings In $400M for Pivotal Tests of Severe Asthma Drug
Generate Biomedicines priced its IPO at $16 per share, raising $400 million to fund pivotal trials of its lead candidate GB‑0895. The antibody blocks the TSLP pathway and is engineered for six‑month dosing, a potential advantage over existing asthma biologics that...
Best Practices for Conducting and Reporting Pharmacoepidemiologic Safety Studies Using Electronic Healthcare Data Sets
The FDA issued a final guidance document outlining best practices for conducting and reporting pharmacoepidemiologic safety studies that rely on electronic healthcare data, such as administrative claims and electronic medical records. It prescribes how sponsors should document study design, analytical...
$24M Verdict Against Seattle Stem Cell Center Clinic in Man’s Death
Seattle Stem Cell Center was ordered to pay $24 million after a jury found the clinic liable for the 2019 death of Michael Trujillo, who suffered catastrophic bleeding following an undocumented epidural injection while on blood‑thinning medication. Evidence showed the procedure...
Alnylam Canada ULC – Receives Positive Recommendation From Canada’s Drug Agency (CDA) for the Public Reimbursement of AMVUTTRA® (Vutrisiran Injection),...
Alnylam Canada received a positive recommendation from Canada’s Drug Agency to list AMVUTTRA® (vutrisiran) for public reimbursement in adult patients with cardiomyopathy caused by wild‑type or hereditary ATTR amyloidosis. The therapy, approved by Health Canada in December 2025, expands the...
Moderna’s Dual Covid-Flu Vaccine Poised for EMA Approval on Positive CHMP Take
The European Medicines Agency’s CHMP has issued a positive opinion on Moderna’s mCombriax, a combined COVID‑19 and influenza mRNA vaccine, after a Phase III trial showed stronger immune responses than a mixed regimen of Sanofi’s flu shot and Spikevax. EMA approval...
Other News to Note for March 2, 2026
At CROI 2026, researchers spotlighted the growing neurodegenerative burden among aging people living with HIV, emphasizing heightened risks of depression and cognitive vulnerability despite long‑term antiretroviral therapy. Parallelly, the University of Southern California announced a novel series of MAPT aggregation...
In the Clinic for March 2, 2026
BioWorld’s “In the Clinic for March 2, 2026” page functions as a centralized gateway to the latest biopharma, med‑tech, and scientific content. It aggregates data snapshots, special reports, infographics, and market scorecards covering everything from GLP‑1 trends in China to mRNA vaccine...
Regulatory Actions for March 2, 2026
On March 2, 2026 BioWorld published a regulatory snapshot covering biopharma and med‑tech firms such as AS Software, Asieris, Boehringer Ingelheim, Deephealth, Eli Lilly, Ipsen, Moderna, Neurogene, Novartis, Optellum, Photocure, Regeneron, Sanofi, Sentynl, Synergy Spine Solutions and X4. The roundup highlights...
INN-Coming: Insights on the Industry’s Latest Disclosures
The WHO’s INN proposed list 134, released in early 2026, reveals several late‑stage drug candidates that were previously hidden from public view. Notably, two NLRP3 inhibitors—abdenoflast and parunoflast—appear to map to Eli Lilly’s newly acquired Ventyx assets VTX2735 and VTX3232, both showing promising...
Why Novo Nordisk's Ireland Expansion Is Key to Fighting Off Eli Lilly
Novo Nordisk announced a €432 million ($506 million) investment to expand its Athlone, Ireland facility, increasing capacity for oral products such as the newly launched Wegovy pill. The expansion is intended to secure supply outside the United States and help the company...
Subq Inventory Expense Shifts Income, Potentially Doubles TAM
Note for $TGTX, this year has $100m in expenses building inventory for Subq - from a theoretical accounting perspective should really be capitalized (and then written-off if failed or amortized in year of sale), but in actuality gets deducted from...
CPHI Middle East 2026 | 11-13 May, Riyadh
CPHI Middle East will return to Riyadh in May 2026, building on the 2024 edition that attracted over 30,000 visitors from more than 100 countries. The event promises expanded networking opportunities, direct market access, and the latest regulatory and innovation...
Ozempic Reverses Osteoarthritis Cartilage Damage, Study Shows
I used to teach that osteoarthritis was "wear and tear" — lose weight, take painkillers, wait for a knee replacement. A study just published in Cell Metabolism proved that wrong. Semaglutide (Ozempic) didn't just reduce joint pain in osteoarthritis patients — it...
Bioxytran Reports Positive Phase 1b/2a Results for Antiviral ProLectin‑M
Bioxytran announced positive phase 1b/2a data for its oral antiviral ProLectin‑M in a randomized, double‑blind, placebo‑controlled trial of 39 mild‑to‑moderate COVID‑19 patients in India. The highest dose (16,800 mg/day) achieved viral clearance in 90% of participants by day 5 versus 20% on placebo,...
Module 2, Section 2: The Druggable Interactome
The Module 2, Section 2 lecture introduces the druggable interactome, compiling key resources that map the human druggable genome, protein expression, kinase families, transcription‑factor proteomics, GPCRs, and ion‑channel complexes. It highlights quantitative estimates—over 3,000 proteins deemed druggable and hundreds of actionable kinases—while...
4 Large-Cap Pharma Stocks to Watch as Industry Recovery Gains Steam
The large‑cap pharmaceutical sector is rebounding in 2026 after a muted 2025, buoyed by strong quarterly results, aggressive M&A, and robust pipeline activity. Zacks ranks the industry at #172, but it posted a 10.8% gain over the past year, outpacing...
Vueway Expands Pediatric Use: Alberto Spinazzi Shares Bracco Group’s Vision
The European Union has granted approval for Vueway (gadopiclenol) to be used in neonates, infants, and toddlers, marking a pivotal expansion of pediatric MRI contrast agents. Vueway delivers diagnostic‑grade imaging at half the dose of conventional macrocyclic GBCAs, thanks to...
Safety Concerns Spur Aardvark to Halt Key Prader-Willi Drug Trial
Biotech Aardvark Therapeutics announced a voluntary pause of the Phase 3 HERO trial for its Prader‑Willi drug ARD‑101 after routine safety monitoring identified reversible cardiac observations in healthy volunteers receiving supratherapeutic doses. The pause affects both the main trial and an...
Advocacy Bias Keeps Unproven DMD Therapy, Blocks HD Progress
The Duchenne muscular dystrophy patient & advocacy community keeps a gene therapy and multiple drugs on the US market, stopping the FDA from taking action, despite failed confirmatory clinical trials showing no efficacy and questionable safety. The Huntington's disease patient and...
P‑tau 217 Blood Test Outperforms Total P‑tau 217 For
The breakthrough blood test p-tau 217 for risk and diagnosis of Alzheimer's disease is more accurate than total p-tau 217, in a head-to-head comparison using PET imaging https://t.co/faILpVr9Ua https://t.co/lzUgFnFUP5
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...
GLP‑1 Weight‑Loss Drugs Work Better for Women
A systematic RCT analysis of GLP-1 drugs show they are more effective in women than men for weight loss https://t.co/cycwi4nnN5 https://t.co/KOl0FgKaZD
Roche's MS Drug Shows Promise, Approval Still Uncertain
Roche pill succeeds in another MS study, but approval questions linger https://t.co/JY8uZCI5iA @ByJonGardner $RHHBY $SNY
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...
FDA Demands Extra Study for UniQure's Huntington Gene Therapy
UniQure says FDA wants another study of Huntington’s gene therapy https://t.co/1pgFDoE2V9 by @realJacobBell $QURE - 35% #GeneTherapy #Huntingtonsdisease
AI Pathology for Cancer Markers Hindered by Shortcut Learning
AI of whole slide images for cancer molecular markers is not ready for clinical use due to confounding and biases ("shortcut learning"), supported by multiple examples @natBME https://t.co/zG8uVIxrJf https://t.co/hQ0tjiapIJ
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...
Exercise‑derived Muscle Vesicles Boost Brain Microglia, Improve Cognition
A new mechanism for improved cognitive function from exercise in the Alzheimer's disease model Skeletal muscle EC vesicles interact with the brain and rev up microglia function https://t.co/eZu14YVasY
Aardvark Pauses Pivotal Prader‑Willi Trial over Safety
Safety concerns spur Aardvark to halt key Prader-Willi drug trial https://t.co/PWkOuDMIvo $AARD - 52% $SLNO
Vaccine Pioneer Warns: We’re Heading Downhill.
Stanley Plotkin, known as the “godfather of vaccines,” in an interview with @HelenBranswell: “All I can say is that I’m beginning to regret having lived so long — because we’re going downhill.” https://t.co/A2zcoP2Ghd
Antibody Plus Ozempic Drives Significant Weight Loss
New @NatureMedicine A randomized trial of antibody vs activin type II receptors with or without semaglutide (Ozempic). The antibody, bimagrumab, promotes muscle growth. Marked weight loss with the combination https://t.co/XUK93GTAmA
Veteran Vaccine Pioneer Warns of Eroding Confidence
Stanley Plotkin had a major hand in the development of a number of vaccines in use today; he designed the rubella vaccine. He remembers the world before widespread use of vaccines & knows what's coming as vaccine policy is rewritten...
GENB CEO Describes Emotional Whirlwind Going Public
2March: How did @generate_biomed CEO @mike_nally describe his whirlwind of emotions as his company went public? Read what he and CFO Jason Silvers, MD, JD said about $GENB plus updates on $CASIF, $NVAX, $PMVP & $SRPT in my latest StockWatch...
Future: Real-Time Immune Monitoring Like Glucose Tracking
Someday we'll be able to track our immune system like we do glucose Today @NatBME https://t.co/HFcFiUeyFg Previously @ScienceMagazine https://t.co/EcgBdwGxcd https://t.co/SpoZ5bLtjx
Flare's GU26 Targets Undruggable PPAR‑γ, Yet Caveats Remain
Who's heard of Flare Therapeutics? #GU26 late-breaker shows promise in hitting "undruggable" PPAR-γ target, with a few major caveats. Via @ApexOnco -> https://t.co/1GepvywtL0
Europe Braces for Fallout of Trump's Drug Pricing Plan
“Nobody wants to feel cornered:" Europe braces for the fallout of Trump's drug pricing plan https://t.co/HZjZH0mdCU
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...