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+: Pharmalittle: We’re Reading About Iran War’s Impact on Pharma Supply Chain, a Pfizer Lyme Vaccine, and More
The ongoing Iran‑Israel conflict has not yet crippled global pharmaceutical supply chains, but it threatens the modest 0.3% of worldwide drug output and 0.6% of active‑pharmaceutical‑ingredient (API) production that originates in the region. Disruptions to key shipping lanes, especially the Strait of Hormuz, are forcing manufacturers in India and the EU to seek alternative routes, raising logistics costs that could eventually be passed to patients. Meanwhile, Pfizer and Valneva reported that their Lyme disease vaccine reduced infection risk by more than 70%, though the trial missed a critical statistical threshold. The companies plan to pursue regulatory approval despite the data shortfall.
Lyme Vaccine Hits 70% Efficacy, Misses Confidence Threshold
The $PFE/ $VALN Lyme vaccine was a product for which there was a lot of hope. Results today show OK efficacy. Vaccine efficacy landed at 70%; investors hoped for 80% but thought as low as 60% would be relevant. But the...
Why Pharma Is Exploring Direct-to-Employer Benefit Models
Pharmaceutical manufacturers are re‑examining traditional PBM‑centric distribution by offering direct‑to‑employer drug purchasing models. Companies like Andel charge a per‑prescription transaction fee and eliminate administrative or per‑member fees, aiming to lower drug spend for employers. Their platform centralizes prescription intake, routes...
FDA Warns of Seizure Risk with some Parkinson’s Drugs
The FDA’s recent safety alert warns that high‑dose levodopa/carbidopa regimens can provoke seizures when patients develop vitamin B6 deficiency. Fourteen post‑marketing cases, including two fatalities, involved doses exceeding 1,000 mg daily and featured focal‑onset seizures that generalized. Vitamin B6 supplementation halted...
Kali Therapeutics Partners with Sanofi for KT501 in a ~$1.2B Deal
Kali Therapeutics has signed a license agreement with Sanofi granting the French giant exclusive worldwide rights to KT501, a next‑generation tri‑specific T‑cell engager aimed at B‑cell‑mediated autoimmune diseases. The deal provides Kali with $180 million in upfront and near‑term payments and...
Oryon Cell Therapies Reports Phase 1b/2a Data Showing Sustained Motor Improvements in People with Parkinson’s Disease
Oryon Cell Therapies presented interim Phase 1b/2a data showing that its autologous dopaminergic neuron‑replacement therapy produced sustained motor improvements in Parkinson’s patients. Five participants experienced 29‑62% reductions in OFF‑state MDS‑UPDRS Part III scores over 6‑18 months, with continued gains beyond six months....
If Gene Therapies Are so Revolutionary, Why Does No One Want to Pay for Them?
Gene therapies promise one‑time cures for diseases like sickle‑cell and inherited blindness, but their price tags—often $1 million to $3 million per patient—clash with the U.S. insurance model. More than half of new cell and gene therapies face coverage restrictions because insurers...
Henlius Receives NMPA IND Clearance for HLX18 (Biosimilar, Opdivo)
Henlius announced that China’s National Medical Products Administration (NMPA) has granted IND clearance for HLX18, a biosimilar of Opdivo (nivolumab), targeting resected solid tumours. The company also reported first‑patient dosing in Phase I trials for HLX17 (Keytruda biosimilar) and HLX13...
Xilio Therapeutics Announces Pipeline and Business Updates and Fourth Quarter and Full Year 2025 Financial Results
Xilio Therapeutics reported a strong cash position of $137.5 million, extending its runway to the end of 2027, and highlighted significant pipeline advances. The company plans to file an IND for its bispecific PD‑1/masked IL‑2 candidate XTX501 in mid‑2026 and start...
Latent-Y: The Autonomous AI Agent for Drug Design at Scale
Latent Labs unveiled Latent‑Y, an autonomous AI agent that designs therapeutic antibodies from natural‑language prompts. Powered by the Latent‑X2 generative model, the platform compresses weeks of expert work into hours and can run multiple design campaigns in parallel. In three...
Pyxis Oncology Provides Business Update and Reports Fourth Quarter and Full Year 2025 Financial Results
Pyxis Oncology announced completion of target enrollment for its Phase 1 MICVO monotherapy study in recurrent/metastatic head and neck cancer, enrolling roughly 40 patients. Interim CEO Thomas Civik highlighted promising early efficacy, with a 46% objective response rate as monotherapy and a...
STAT+: Insmed Drug Benefits Patients with Rare, Bacterial Lung Disease, Study Shows
Insmed announced that a Phase 3 trial showed adding its inhaled antibiotic Arikayce to standard therapy significantly improved respiratory symptoms and boosted culture conversion rates in patients with newly diagnosed mycobacterium avium complex (MAC) lung infection. The study met its primary...
Avalo Therapeutics Reports 2025 Financial Results and Recent Business Updates
Avalo Therapeutics announced its 2025 financial results, reporting $98.3 million in cash and short‑term investments that should fund operations into 2028. Research and development expenses jumped to $50.1 million, driven by the Phase 2 LOTUS trial of abdakibart (AVTX‑009) for hidradenitis suppurativa. The...
'Zombie' Cells Created by Transplanting Genomes Into Dead Bacteria
Researchers have revived a dead bacterial cell by transplanting the complete genome of Mycoplasma capricolum into a chemically inactivated host, creating the first living synthetic bacterium assembled from non‑living parts. The experiment builds on the 2010 landmark where a synthetic...
STAT+: Pfizer’s Lyme Vaccine Shows Efficacy, but Misses Key Statistical Hurdle
Pfizer and Valneva’s experimental Lyme vaccine cut the risk of infection by more than 70% in a late‑stage trial, offering a promising preventive tool for a disease that affects roughly 476,000 Americans annually. The study, however, missed its primary statistical...
STAT+: Apogee Therapeutics Data Show Long-Acting Eczema Drug Induced Relief with Less Frequent Injections
Apogee Therapeutics reported that its experimental long‑acting eczema biologic, zumilokibart, achieved sustained skin‑clearance in a mid‑stage trial. Seventy‑five percent of patients receiving the drug every three months and 85 % of those dosed every six months maintained an EASI‑75 response after...
IntraBio Reports the US FDA’s sNDA Submission of Aqneursa for Ataxia-Telangiectasia
IntraBio has filed a supplemental New Drug Application (sNDA) with the U.S. FDA seeking approval of Aqneursa (levacetylleucine) for Ataxia‑Telangiectasia (A‑T). The filing is supported by a Phase III trial that met its primary and key secondary endpoints and demonstrated a...
How Do Recent Actions From FDA Provide Insight to the Agency's Enforcement Posture?
The FDA’s Rare Disease Evidence Principles (RDEP) introduce flexible trial designs, allowing sponsors to use natural‑history data and novel biomarkers as endpoints. These guidances aim to accelerate approvals for rare‑disease therapies while maintaining safety as a top priority. However, analysts...
Regulating Peptide Sales Protects Consumers over Hype
Peptides are all the rage right now, but it’s hard to see how they are different from the previous generations of snake oils. The principle that anything that makes a health claim should have to justify it with scientific evidence...
HUTCHMED Initiates P-III Trial of HMPL-760 + R-GemOx for R/R Diffuse Large B-Cell Lymphoma in China
HUTCHMED has launched a Phase III trial of HMPL‑760 combined with R‑GemOx in relapsed/refractory diffuse large B‑cell lymphoma (DLBCL) patients in China, dosing the first patient on March 20, 2026. The study will enroll approximately 240 patients who have failed first‑line therapy and...
A Targeted Nanozyme for STING Activation Improves BiTEs Therapy Outcomes in Colorectal Cancer
Researchers engineered a tumor‑targeted nanozyme, MnO2‑dsDNA@BiTE/APT, that simultaneously delivers a double‑strand DNA STING agonist and a PD‑L1/CD3 bispecific T‑cell engager. The MnO2 carrier releases Mn2+ ions, activating the STING pathway, while the surface‑bound BiTE recruits T cells to cancer cells....
Dizal Reports the P-III (WU-KONG28) Trial Results on Zegfrovy (Sunvozertinib) in EGFRm NSCLC
Dizal announced topline results from its Phase 3 WU‑KONG28 trial, comparing oral once‑daily Zegfrovy (sunvozertinib) to platinum‑based chemotherapy as first‑line treatment for advanced NSCLC with EGFR exon 20 insertion mutations. The study met its primary endpoint, demonstrating a statistically significant improvement in...
Mesoporous Silica Nanoparticles‐Based Formulations for Enhanced Oral Delivery of Peptide Drugs: A Case Study on Insulin
Researchers engineered mesoporous silica nanoparticles (MSN) to encapsulate insulin and co‑formulated them with succinylated β‑lactoglobulin into pH‑responsive tablets. Surface grafting with polyethylene glycol and phosphonate groups boosted insulin solubility by roughly 2.5‑fold and stabilized the particles in gastrointestinal fluids. The...
US Weight Loss Drugmakers Slash Prices in Fight to Win Customers
U.S. weight‑loss drugmakers are slashing GLP‑1 prices to win cash‑pay customers as insurers balk at coverage. Eli Lilly reduced Zepbound’s monthly cost by $50‑$100, bringing it to $299, while Wegovy now sells for $149 a month, far below its $1,600 launch...
Powering the Next Wave of Cell Therapy: From iPSC-Derived Cells to In Vivo Reprogramming
Induced pluripotent stem cells (iPSCs) are being engineered into diverse therapeutic cell types, while in vivo reprogramming aims to convert resident cells directly within patients, eliminating traditional cell‑manufacturing steps. Both strategies depend on precise recombinant growth factors, cytokines, extracellular matrix proteins...
Gene Therapies for Hearing Loss Strike an Encouraging Note in Embattled Modality
Gene‑therapy candidates for hereditary hearing loss are gaining traction as safety concerns ease with localized delivery. Regeneron’s DB‑OTO and Eli Lilly’s AK‑OTOF have each demonstrated clinically meaningful hearing improvements in early‑stage trials, positioning them as frontrunners for the first approved deafness...
9 Months In, FDA’s New Priority Voucher Program Still Clouded With Uncertainty
The FDA’s National Priority Voucher (CNPV) program, launched in June 2025, promises to cut drug review times from 10‑12 months to just one or two months for products that meet defined national priorities. Early successes include Johnson & Johnson’s Tecvayli/Darzalex...
Open‑source Super‑intelligent Model Accelerates Drug Discovery
Very important paper from our & @liquidai teams - deeply honored to have published with the geniuses @ramin_m_h & @xanamini . Take the super flexible model with high density of intelligence, post-train and fine-tune for basic...
FDA OKs Fecal Transplants, Yet Access Declines
Paradox: The FDA approved some fecal transplants for C. difficile. But accessing treatment got harder. https://t.co/AFFyCGPeKW
DEA Fine Tunes Power of Attorney and DEA-222 Requirements
On March 20, 2026, the DEA issued a technical amendment to its 2019 final rule that clarifies who may execute and revoke Power of Attorney documents for DEA Form 222 and who may sign the form itself. The amendment aligns...
Phase 3 Lyme Vaccine Shows Mixed Preliminary Results
The news is mixed in the preliminary results from a Phase 3 trial of a needed #Lyme disease vaccine, @matthewherper reports. https://t.co/bcbfeXchk1
CtDNA Drives Next‑stage Risk Stratification in TNBC
From Prognosis to Action: Circulating Tumor DNA and the Next Phase of Risk Stratification in Triple-Negative Breast Cancer [Mar 18, 2026] Schneider & @StoverLab @JCO_ASCO https://t.co/6jPBxQe9QP #bcsm #PrecisionMedicine #LiquidBiopsy https://t.co/MOG73iTcNl
NADPH Oxidase-1 Suppression Prolongs the Antidepressant-Like Effect of Ketamine
Researchers introduced K‑4, a novel AMPA‑receptor positive allosteric modulator, which produced rapid and sustained antidepressant‑like effects in treatment‑resistant depression rat models. Bulk RNA‑seq revealed that K‑4 markedly down‑regulated NADPH oxidase‑1 (NOX‑1) in the medial prefrontal cortex and lateral habenula. Pharmacological...
New Guidelines Add Testing, Therapy for ESR1‑mutated Metastatic Breast Cancer
Guideline Update Provides New Testing & Tx Recx for Pts w/ ER-Positive, HER2-Negative Met Breast Cancer w/ ESR1 Mutations [May 17-18, 2023] @ASCO Daily News - https://t.co/3ZVrEkQEN5 @DrHBurstein et al. @JCO_ASCO https://t.co/68e7D0CSgU #bcsm #PrecisionMedicine #LiquidBiopsy https://t.co/KBx2hq2BDW
Low‑
Lenalidomide + high-dose dexamethasone (RD) vs lenalidomide + low-dose dexamethasone (Rd) as initial therapy for newly diagnosed multiple myeloma: an open-label RCT [10/22/2009] @VincentRK et al. @TheLancetOncol https://t.co/hXxlFINejC #NCT00098475 #EAonc E4A03 #mmsm #caxtx #ctsm https://t.co/3Pa2TRMntk
ENAVATE Sciences Boosts Zenas BioPharma Stake to $142 M, Highlighting Obexelimab
ENAVATE Sciences GP, LLC bought an additional 157,800 Zenas BioPharma shares for an estimated $5.19 million, taking its position to $142.3 million (28.1% of ENAVATE’s reportable AUM). The move signals strong conviction in Zenas’s Obexelimab pipeline as the company prepares a BLA...
Personalized Therapy Improves Outcomes in Residual TNBC
BRE12-158: A Postneoadjuvant, Randomized Phase II Trial of Personalized Therapy Versus Treatment of Physician's Choice for Patients With Residual Triple-Negative Breast Cancer [Dec 15, 2021] Schneider et al. Radovich @JCO_ASCO https://t.co/oJm6BJrMtA #bcsm #PrecisionMedicine
Genetic Links to Aromatase Inhibitor Musculoskeletal Side Effects
Genome-Wide Associations [GWAS] and Functional Genomic Studies of Musculoskeletal Adverse Events in Women Receiving Aromatase Inhibitors [Sep 20, 2010] Ingle et al. @DrWeinshilboum @JCO_ASCO https://t.co/1NJcXkWy8c #bcsm #Supponc
Eli Lilly and Company (LLY) Expands Zepbound Access as Pricing Reforms Boost Long-Term Growth Outlook
Eli Lilly announced on March 16 that its obesity treatment Zepbound will be sold directly to consumers for $299 a month for the 2.5 mg dose through LillyDirect and major pharmacy chains. A new savings card locks that price nationwide for the KwikPen...
Circulating DNA/Cells Predict Recurrence Post‑Chemo in TNBC
Assoc of Circulating Tumor DNA & Circulating Tumor Cells After Neoadjuvant Chemotherapy w/ Disease Recurrence in Pts w/ Triple-Negative Breast Cancer: BRE12-158 RCT [Jul 9, 2020] Radovich et al. @JAMAOnc https://t.co/XxH9kTT9d5 #bcsm #cactc #NCT02101385 https://t.co/kgV0yM2I1f
Pfizer's Lyme Phase 3 Cut by One‑Third Over Contractor Issues
Pfizer's phase 3 for its Lyme trial was supposed to be 18k patients -- but issues with a contractor at the start of the trial cut the size by a third, and @ky_lahucik + I reported a long time ago...
FDA Drug Safety Communication: Revised Recommendations for Celexa (Citalopram Hydrobromide) Related to a Potential Risk of Abnormal Heart Rhythms with...
The FDA issued a Drug Safety Communication clarifying that citalopram (Celexa) should not be prescribed above 40 mg daily because higher doses significantly prolong the QT interval and can trigger fatal Torsade de Pointes. The label now mandates a maximum of...
P7C3 Boosts NAD Enzyme, Improves Mouse Alzheimer Model
P7C3 is an activator of an enzyme that makes NAD called NAMPT. Cool that it’s working in a 🐁 model of AD 👏
China Transforms From Free Rider to Drug Innovator
New @nberpubs: "From Free Rider to Innovator: The Rise of China's Drug Development" https://t.co/cQpEqRC1jy https://t.co/pSL5kCzlnO
Pfizer Lyme Vaccine Effective yet Misses Crucial Statistical Benchmark
Pfizer’s Lyme vaccine shows efficacy, but misses key statistical hurdle There is a set up for drama here. $Valn $pfe Valneva stock is down 14% in premarket trading. https://t.co/WN0KOl4Nqh via @statnews
Only Seven Proven Longevity Compounds After Massive Mouse Trials
7 Drugs. 30,000 Mice. 20 Years. The Only Longevity Compounds With Real Evidence. https://t.co/BzdoDZeicb @agingroy https://t.co/uM5QMpIAS6
Stem Cell Dose Boosts Walk Test, Cuts Frailty
Randomized phase 2b dose-escalation trial of stem cell therapy with laromestrocel for aging frailty 🌟Performance on the 6-minute walk test improved in a dose-response fashion 🌟Improved 6-minute walk test distance correlated with patient-reported outcomes 🌟The percentage of study subjects classified as frail decreased...
Allogeneic Stem Cells Safely Boost Function in Frail Adults
Allogeneic Mesenchymal Stem Cells Ameliorate Aging Frailty: A Phase II Randomized, Double-Blind, Placebo-Controlled Clinical Trial “Treated groups had remarkable improvements in physical performance measures and inflammatory biomarkers, both of which characterize the frailty syndrome. Given the excellent safety and efficacy...
Single Stem Cell Dose Boosts Strength, Reverses Frailty
Stem cell therapy shows promise for reversing aging-related frailty in new clinical trial 🗣️A clinical trial reports that a single dose can significantly improve physical strength and key signs of aging in older adults with frailty. https://t.co/5mw5r64imb https://t.co/uI88QpTEIa
FDA Staff Hear Peptide Plan via Rogan Podcast
“At least two career staffers at the FDA whose work includes compounding drugs said they learned of the health secretary’s plans to take action on peptides only from his appearance on Rogan’s podcast.” https://t.co/vpTDqstpDZ