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.
Intra‐Articular Injectable Hydrogel Microsphere‐Based Drug Delivery System for Osteoarthritis Treatment
Osteoarthritis incidence is climbing as populations age and obesity spreads, intensifying health and economic pressures. Conventional treatments merely decelerate disease progression and often entail systemic side effects or surgical risks. Emerging hydrogel microsphere platforms create a biomimetic joint microenvironment, offering precise, localized drug release with high biocompatibility. This review outlines OA‑induced joint changes, essential microsphere attributes, fabrication techniques, modification strategies, therapeutic applications, and the hurdles that must be overcome for clinical translation.
Out-Of-Pocket’s 2025 Predictions | Out-Of-Pocket
Out‑Of‑Pocket’s 2025 outlook forecasts a turning point for several health‑care segments. Obesity drugs such as GLP‑1s are expected to become cost‑effective as pricing pressure and outcomes‑based contracts expand access. AI models will split, with healthcare‑specific versions emphasizing explainability, security and...
Editing Away Autoimmunity at the HLA Source
In this episode, Daniel Levine interviews Richard Freed, CEO of Rheumagen, about the pivotal role of HLA genes in autoimmune diseases and the company’s innovative gene‑editing approach to cure them. Freed explains how a single amino‑acid change at a conserved...
Senescent Cells Fuel Atherosclerosis; Senolytics Offer Hope
The Role of Cellular Senescence and SASP in the Pathogenesis of Atherosclerosis and the Therapeutic Potential of Senolytic Strategies in Cardiovascular Diseases "Cellular senescence and SASP drive chronic inflammation and vascular dysfunction central to atherosclerosis pathogenesis, with senescent endothelial cells, VSMCs,...
No RCTs for BPC‑157: Patent Dead, Profit Absent
Correct. There will likely be no RCTs on BPC. Unless a new variant can perform better and be patented and Rx’d… and THATs happening.
MariMed Inc (MRMD) Q4 2025 Earnings Call Transcript
CorMedix reported Q4 2025 revenue of $128.6 million, propelled by DEFENCATH ($91.2 M) and the first full‑quarter contribution from the Melinta acquisition ($37.4 M). Full‑year pro forma revenue reached $401.3 million, matching prior guidance, with DEFENCATH net sales totaling $258.8 million. Adjusted EBITDA for the...
Verrica Pharmaceuticals Inc (VRCA) Q4 2025 Earnings Call Transcript
Veru Inc. reported a $23.4 million public offering that lifted cash and working capital to $33 million and $29.7 million respectively, while narrowing its quarterly net loss to $5.3 million ($0.26 per share). The company received FDA feedback confirming two regulatory pathways for its...
Viant Technology Inc (DSP) Q4 2025 Earnings Call Transcript
Vanda Pharmaceuticals reported 2025 revenue of $216.1 million, up 9% year‑over‑year, driven primarily by Fanapt’s 24% sales increase and a successful bipolar launch. The company secured FDA approval for tradipitant (Nirius) to prevent motion‑sickness vomiting, with a commercial rollout slated for...
FDA Leucovorin Approval Raises Concerns About Reliance On Case Reports
The FDA granted approval for leucovorin calcium tablets to treat a rare genetic disorder that impairs folate transport to the brain. Unlike typical approvals, the agency relied primarily on a handful of patient case reports rather than a sponsor‑run clinical...
STAT+: GOP Senator Is Investigating the FDA over Rejections of Rare Disease Drugs
Sen. Ron Johnson (R‑Wis.) announced an investigation into the FDA’s rejections of rare‑disease treatments, requesting the agency’s complete response letters for drugs targeting ataxia, Sanfilippo syndrome and similar conditions. Johnson argues the FDA’s cited deficiencies are often “nitpicky,” suggesting an...
Biologics and Biosimilars Landscape 2025: IP, Policy, and Market Developments
The FDA approved 18 biosimilars in 2025, spanning six therapeutic areas and marking a surge in interchangeable designations to over 20. A wave of denosumab biosimilars and first‑in‑kind interchangeable products such as Poherdy® and Omlyclo® highlighted market diversification. BPCIA litigation...
Public Citizen To Push Compulsory Licensing If Pfizer Withholds Drugs From France
Public Citizen announced it will encourage compulsory licensing of generic drugs if Pfizer follows through on its January threat to withhold medicines from France unless the country raises drug prices. The consumer group’s stance turns a pricing dispute into a...
Study Suggests Long Non-Coding RNA Has Potential as New Class of Genetic Medicine
Researchers at the University of Toronto have engineered synthetic long non‑coding RNA (lncRNA) molecules that can dampen inflammatory responses. The study, led by PhD student Janice Pang and biologist Omar Khan, demonstrates that these engineered lncRNAs reduce cytokine release in...
CDMO Neuland Labs Expects Commercial Production Facility to Be Operational by Summer
Neuland Laboratories announced that the first module of its new commercial peptide manufacturing facility will be operational by summer on its 17‑acre Bonthapally campus in India. The module adds 6,370 L of solid‑phase and liquid‑phase peptide synthesis capacity, ranging from 250 L...
Lundbeck CEO Talks Drug Pricing, Protecting Biotech and European Needs
Lundbeck chief executive Charl van Zyl warned that Europe’s fragmented drug‑pricing regime threatens biotech sustainability and patient access. He cited recent remarks by HHS Secretary Robert F. Kennedy Jr. suggesting Europeans already pay higher prices than Americans. Van Zyl called...
FDA Clears New Formulation of Lantheus' PSMA Imaging Agent
The FDA has cleared Pylarify TruVu, a reformulated version of Lantheus’ PSMA‑targeting radiopharmaceutical piflufolastat F‑18. The new formulation improves stability at higher radioactive concentrations, enabling larger batch production and broader distribution. Lantheus plans a Q4 2026 commercial launch with a rolling geographic...
Phase III Data Support Johnson & Johson’s Teclistamab as Second-Line Therapy for RRMM
Johnson & Johnson has filed a Type II variation with the European Medicines Agency to add teclistamab, a bispecific T‑cell‑redirecting antibody, as a second‑line monotherapy for relapsed/refractory multiple myeloma. The Phase III MajesTEC‑9 trial, enrolling 614 patients, demonstrated a 71 % reduction in...
Pharmaceutical Executive Daily: FDA Expands Approval of Leucovorin
The FDA has expanded approval of leucovorin calcium tablets as the first therapy for cerebral folate transport deficiency linked to FOLR1 gene variants. The decision relied on published case literature, showing 87‑89% of patients achieved clinical improvement. In oncology, AstraZeneca...
Weight‑loss Meds Cut Alcohol Use in Half of Users
Study shows nearly half of weight loss participants reduced alcohol use after starting anti-obesity medication (GLP-1 agonists, metformin, naltrexone). https://jamanetwork.com/journals/jamanetworkopen/fullarticle/2827069 https://www.gatlan.com/ @GatlanHealth
Regulatory Pharmaceutical Fellowship Program
Several U.S. universities have launched two‑year medication safety fellowships that rotate participants through academic training, a major pharmaceutical company, and the FDA. Butler University offers two tracks with Regeneron and Eli Lilly, Purdue partners with AbbVie, and Rutgers collaborates with Pfizer....
Peptides in the Pipeline: How Companies Are Overcoming the Early-Stage Challenges
Peptide therapeutics are booming, with more than 2,000 candidates in discovery and pre‑clinical stages and projected global sales of $91.4 bn by 2025. Companies like Orocidin faced severe synthesis and purification hurdles for their QR‑01 peptide, but Syngene’s custom low‑loading resin...
Johns Hopkins Leads $24M Multinational Consortium to Find Hepatitis B Cure
Johns Hopkins Medicine is heading a five‑year, $24 million NIH‑funded Hepatitis B and HIV Cure Consortium that brings together research teams from the United States, Brazil, India, Senegal and Uganda. The first year will enroll 450 participants co‑infected with HIV and chronic...
Efgartigimod Effective in Treating Juvenile Myasthenia Gravis
A multicenter retrospective study of 17 Chinese patients with juvenile myasthenia gravis found that weekly efgartigimod 10 mg/kg for four weeks produced rapid and substantial clinical improvement. Clinically meaningful improvement was observed in 70.6% of patients by week 1 and 91.7% by...
BioNTech Co-Founders Launch New mRNA Company
BioNTech announced it will create an independent biotech company dedicated to next‑generation mRNA technologies. The spin‑out will be led by co‑founders Ugur Sahin and Özlem Türeci, who will assume management by the end of 2026. BioNTech will contribute core mRNA assets in...
CSL Breaks Ground on $1.5B Illinois Immunoglobulin Plant Expansion
CSL‑Behring broke ground on a $1.5 billion expansion of its Kankakee, Illinois manufacturing complex, slated to be operational by 2031. The project will add at least 300 pharmaceutical positions and roughly 800 construction jobs, with the state offering more than $200 million...
Agomab IPO Raises $200M to Fund Fibrosis Therapies
Agomab Therapeutics NV closed its Nasdaq IPO, raising roughly $200 million by selling 12.5 million American Depositary Shares at $16 each. The capital will fuel its fibrosis‑focused pipeline, beginning with ontunisertib, an oral TGFβ‑ALK5 inhibitor for fibrostenosing Crohn’s disease that achieved its...
NX-1607
Nurix Therapeutics has launched NX-1607, the first orally bioavailable small‑molecule inhibitor of the immune regulator CBL‑B, into a Phase 1a/1b trial for advanced cancers. CBL‑B modulates activation of T, B and NK cells, and NX-1607 locks the protein in an inactive...
A Dose of Psilocybin Helps Smokers Quit in New Study
Researchers at Johns Hopkins found a single dose of psilocybin dramatically increased smoking cessation rates compared with nicotine patches. In a randomized trial of 82 smokers, 17 of 41 participants who received psilocybin remained abstinent after six months versus four...
First-of-Its-Kind Vaccine Protects Children From Deadly E. Coli Infections
Scientists announced ETVAX, the first oral vaccine that targets enterotoxigenic *E. coli* (ETEC) in children, after a large‑scale trial in The Gambia. The study involved 4,936 infants aged six to 18 months and demonstrated a 48% reduction in moderate‑to‑severe ETEC...
STAT+: Novo Nordisk Is Warned by the FDA for Failing to Report Side Effects Tied to GLP-1 Drugs
Novo Nordisk received an FDA warning letter on March 5, 2026 for failing to report suspected side effects of its GLP‑1 medicines. The violations were uncovered during a 2025 inspection of the company’s facilities and were described as “serious.” The agency warned...
US Vaccine Manufacturers Set to Lose as RFK Pushes for Weaker Vaccine Mandates
The Trump administration, led by Health Secretary Robert F. Kennedy Jr., plans to eliminate over a third of the CDC childhood vaccine schedule, targeting seven of eleven vaccines. The United States, which produces roughly 36% of global vaccine output, could...
Building for Decades: Dubai’s Long Game in Life Sciences
Dubai is positioning itself as a long‑term hub for healthcare and life sciences through two complementary free‑zone clusters – Dubai Healthcare City (DHCC) and Dubai Science Park (DSP). DHCC is undergoing a $1.3 bn expansion that will create six tightly linked...
FDA Sets Scope for Attempt To Reduce Manufacturing-Related Approval Rejections
The FDA has defined the scope of pre‑submission facility meetings to curb manufacturing‑related complete response letters that delay drug approvals. It agreed to cover prior production‑site inspections, novel process elements and supply‑chain node strategies, while rejecting topics such as alternative...
Desert to Discovery: Five Hot Biotechs in Arizona
Arizona's biotech sector is booming, with $3.7 billion invested over the past seven years and a growing pipeline of innovative companies. Nectero Therapeutics secured a $96 million Series D to develop a fast‑track, breakthrough‑designated endovascular treatment for abdominal aortic aneurysms, while Humabiologics obtained...
STAT+: Pharmalittle: We’re Reading About a U.S. Senator Probing FDA, BioNTech Founders Leaving, and Much More
BioNTech co‑founders Ugur Sahin and Özlem Türeci will leave the company by year‑end to launch an unnamed mRNA‑focused venture, with BioNTech retaining a minority stake and licensing key technology. The split allows BioNTech to concentrate on its late‑stage cancer pipeline...
Deaminet 2026: Breakthroughs in Base Editing, Deaminase Biology, and Therapeutic Translation
Deaminet 2026 showcased accelerating advances in base and prime editing, highlighted by rapid Addgene distribution growth and new mechanistic insights such as PE6d’s heightened processivity and ABE8e’s dimer‑driven efficiency. Novel off‑target detection platforms like beCasKAS and Inrich‑seq revealed far more unintended...
BioNTech Founders Launch New Biotech, Eye FDA Acceptance
Sticking with the science. I find that really impressive. BioNTech founders Uğur Şahin and Özlem Türeci are leaving the biotech they founded to start a new one. Maybe by the time they have something for the clinic, the FDA will...
Melatonin Shields Mitochondria, Offering Neurodegenerative Therapy
Melatonin as a Guardian of Mitochondria: Mechanisms and Therapeutic Potential in Neurodegenerative Diseases https://t.co/kp4LZEbZzN https://t.co/boWzW7fcjM
China Is Going After the World's Most Expensive Drugs: Endpoints Signal
China is actively targeting ultra‑expensive gene therapies, challenging the notion that these treatments are immune to price competition. Recent regulatory approvals and centralized procurement initiatives have forced manufacturers to negotiate steep discounts on therapies that once commanded multi‑million‑dollar price tags....
Leucovorin Approved for Limited Autism Subgroup, Not Mass Treatment
Last fall the administration was talking about leucovorin as a potential treatment for large numbers of people with #autism. Today's approval suggests the evidence supports its use in a much smaller group of people, @rosebroderick_ reports. https://t.co/NlJR8KlaqP
BioNTech Founders Leave to Launch New mRNA Venture
BioNTech founders to step down and helm new mRNA startup https://t.co/yjvsVRQH2D by @Lilah_Alvarado $BNTX - 20%
Xenon Scores Epilepsy Win; FDA Revives Duchenne Cell Therapy Review
Xenon hits a ‘home run’ in epilepsy; FDA restarts review of Duchenne cell therapy https://t.co/l40xYeW2Xh $XENE $CAPR + 15% $REGN $PFE $ABBV $GSK #biotech
BBIO, ALNY Surge on Pfizer Tafamidis Patent Win
$BBIO $ALNY moving higher on apparently good news re: Pfizer's tafamidis patent defense. If you have access to Umer Raffat's research, he has all the details.
Psilocybin Plus Therapy Boosts Quit Rates Sixfold
🚨 @NPR spotlights our new JAMA Network Open study: One psilocybin dose + therapy gives 6X better odds of quitting smoking vs. nicotine patch + therapy. Game-changer for tobacco smoking addiction?
Blood Test Predicts Alzheimer's up to 25 Years Early
Predicting Alzheimer's disease up to 25 years in advance of any symptoms with the p-tau217 biomarker blood test, among 2,766 women mean age 70 https://t.co/aOActAUN27 https://t.co/Bxl2YZxnlf
DNA Repair Mutations Predict Response to Chemo‑Immunotherapy in Pancreatic Cancer
DNA Repair gene alterations and efficacy from gemcitabine and nab-paclitaxel with/without durvalumab and tremelimumab in metastatic pancreatic ductal adenocarcinoma https://t.co/v39bOreJwu
FDA Adds Six Former Members, Total Nine Voters
Correction: I took down a post about Thursday's #VRBPAC meeting because it contained an error. #FDA has named 6 temporary voting members (not 2), which will bring the number for the meeting to 9. The temps are all former members....
FDA Accepts CAPR's Deramiocel Resubmission, Decision Aug 22
$CAPR resubmission of the deramiocel application accepted by the FDA. New decision date: Aug. 22. https://t.co/mWSyyXg8di
CRISPR's Natural Blueprint Powers Gene‑Therapy Breakthroughs
How CRISPR works (in nature) and how it’s being leveraged as a tool for gene editing to cure and prevent diseases. Dr Alex Marson MD PhD of UCSF on the Huberman Lab podcast out now. https://t.co/fiL48JHxSL
GSK Secures Approval Yet Refuses to Produce Drug
$GSK is ostensibly the "sponsor" that won the approval but is once again saying it won't produce the drug.