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.
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Taisho Pharmaceutical’s vornorexant (TS‑142), marketed as Vorzzz®, received Japanese regulatory approval in August 2025 as a dual orexin‑1/2 receptor antagonist for insomnia. The drug distinguishes itself from existing DORAs through rapid absorption and a short elimination half‑life, aiming to minimize next‑day residual effects. In pivotal Phase 3 trials, once‑daily 5 mg and 10 mg doses significantly improved sleep onset latency and sleep efficiency compared with placebo. Taisho has not indicated whether it will seek approval in the United States.
Digital Nudges Amplify Lifestyle Gains for GLP‑1 Patients
Medicines plus healthy habits create outcomes that neither could achieve alone. A new @StanfordMed study published in JAMA shows digital lifestyle nudges may catalyze a critical first step toward behavior change for patients on GLP-1 medications. “Achieving your best health involves a...
FDA Slashes Advisory Panels, Threatening Drug Safety Oversight
I've covered most of the big controversies the FDA has faced over the past 25 years: Vioxx, drug-coated stents, and Aduhelm all spring to mind. In all of these cases, the FDA's advisory panel system, in which the agency calls together...
Unlocking Hidden Pocket on a Billion‑dollar Drug Target
Researchers led by Harvard chemist Christina Woo have mapped a previously unknown allosteric pocket on cereblon, the E3 ligase that underpins billions of dollars in cancer‑drug activity. The study shows that binding a small molecule to this hidden site can...
Foundation Models Set to Transform Drug Discovery
Would you like to hear an opinion on how the future of drug discovery will look like as foundation models become more capable in drug discovery tasks? Check out our dialogue and the first pilot with the foundation model genius @ramin_m_h...
AbbVie’s Amylin Candidate ‘Competitive’ in Early-Stage Trial
AbbVie announced top‑line Phase 1 multiple ascending‑dose data for its amylin analog ABBV‑295, showing 7.75‑9.79% weight loss after 12 weeks of treatment. The long‑acting compound was administered every other week then monthly, with a favorable tolerability profile and no serious adverse...
AbbVie, Gubra Post Obesity Data; Regeneron Obesity Drug Succeeds in China
AbbVie and its partner Gubra released Phase 2 data on a long‑acting amylin analogue that produced significant weight loss in obese participants, with reductions approaching double‑digit percentages and a clean safety signal. The study highlighted dose‑responsive efficacy and tolerability, positioning the...
FDA Leaders Makary and Pazdur Meet Investors in Miami
Re: Prasad, from this morning's @statnews Readout newsletter: FDA Commissioner Marty Makary is in Miami this week speaking to health care investors attending a slew of broker conferences, noted Mizuho health care strategist Jared Holz. That should make for some...
GlobalData’s Key Thoughts From World EPA Congress 2026
GlobalData Healthcare attended the World Evidence, Pricing and Access (EPA) congress in Amsterdam, where industry leaders debated mounting market instability. Sessions focused on the impact of the Most Favored Nation (MFN) policy, shifting US tariffs, rapid AI integration, and the...
GLP-1 Drugs Modulate Gene Expression via MED14 Phosphorylation
Stable GLP‑1 receptor agonists such as Exendin‑4 and Ozempic improve beta‑cell viability by modulating gene expression. Researchers at the Salk Institute discovered that these drugs induce phosphorylation of Med14, a core subunit of the Mediator transcription complex. Phosphorylated Med14 enables...
Regeneron’s Weight Loss Partner Hansoh Delivers Much-Needed Phase 3 Win in China
Regeneron’s Chinese partner Hansoh announced that its dual GLP‑1/GIPR agonist olatorepatide achieved a 19% mean weight loss in a Phase 3 trial of 604 obese or overweight adults, meeting both co‑primary endpoints. The study reported lower gastrointestinal adverse events compared with...
Bristol Myers Says Second CELMoD Succeeds in Phase 3
Bristol Myers Squibb announced that its oral CELMoD candidate mezigdomide met primary endpoints in the Phase 3 SUCCESSOR‑2 trial for relapsed or refractory multiple myeloma. The open‑label study showed a statistically significant improvement in progression‑free survival compared with the current standard...
Medidata, CRIO Boost Clinical Trials with Integration
Medidata, a Dassault Systèmes brand, has partnered with eSource specialist CRIO to automate clinical data flow from site systems into the Medidata Platform. The plug‑and‑play integration now serves over 2,500 research sites in roughly 30 countries, delivering near‑100% data accuracy and...
Harnessing Immune System: New Frontiers in Cancer Therapy
The new Huberman Lab episode is out: Avoiding, Treating & Curing Cancer With the Immune System | Dr. Alex Marson 0:00 Alex Marson 2:21 Diseases & Current Biological Landscape; AI & Computational Tools 5:56 Immune System, Innate vs Adaptive Immune System 10:55 Thymus, T...
Bristol Myers Squibb Announces Positive Phase 3 Results From the SUCCESSOR-2 Study of Oral Mezigdomide in Relapsed or Refractory Multiple...
Bristol Myers Squibb reported positive interim Phase 3 results from the SUCCESSOR‑2 study, showing that oral mezigdomide combined with carfilzomib and dexamethasone (MeziKd) significantly extended progression‑free survival versus carfilzomib‑dexamethasone alone in relapsed or refractory multiple myeloma. The trial marks the first...
Lilly Issues Statement on CMS BALANCE Model for GLP-1 Drugs
Eli Lilly announced that the CMS CMMI BALANCE Model will cover its GLP‑1 obesity drugs—Zepbound, Mounjaro and, if approved, orforglipron—under Medicare Part D starting Jan 1 2027. After meeting the deductible, beneficiaries will pay a $50 monthly out‑of‑pocket cap, while pre‑deductible cost sharing is...
Bridging Innovation and Clinical Reality: Shalabh Gupta, CEO of Unicycive Therapeutics, on the Future of Biotech
Shalabh Gupta, CEO of Unicycive Therapeutics, argues that biotech can benefit from tech‑driven design thinking, rapid prototyping, and disciplined capital allocation. He highlights how the company repurposed automotive battery‑shrinkage technology to create Oxylanthanum carbonate, a kidney‑disease candidate now under FDA...
Xenon To Seek Approval of First-in-Class Epilepsy Drug After Exceeding Phase 3 Expectations
Xenon Pharmaceuticals reported that its Phase 3 X‑TOLE2 trial of azetukalner, a novel Kv7 potassium channel opener, achieved a 53.2% reduction in focal onset seizures at the 25 mg dose, far exceeding expectations and representing the highest placebo‑adjusted efficacy recorded in a...
Genome Editing for Biopharmaceutical Manufacturing
Chinese hamster ovary (CHO) cells remain the backbone of biopharmaceutical manufacturing, but traditional gene‑editing tools struggle with low knock‑in efficiency. Transposase‑based platforms such as Leap‑In and piggyBac now provide high‑efficiency, multi‑copy integration and can handle large DNA cargos up to...
Selectivity in Cells May Vary
The Chemical Probes Portal’s >30‑fold selectivity rule, originally based on cell‑free assays, is challenged by a new open‑access J. Med. Chem. study comparing DiscoverX kinase panels with NanoBRET cellular profiling. Researchers found that most inhibitors appear less potent in living...
Associated Press Cannot Explain Bewildering Reporting on FDA’s Tracy Hoeg and Antidepressant Risks
The Associated Press published a story alleging that FDA senior regulator Dr. Tracy Hoeg was pushing a petition from Dr. Adam Urato to add pregnancy‑risk warnings to antidepressants and that their personal relationship constituted a conflict of interest. The author,...
RNA Editing Startup Tacit Therapeutics Launches with $19M for Brain Diseases
Tacit Therapeutics, an RNA‑editing startup focused on neurological disorders, announced its launch backed by a $19 million financing round. The capital, led by Andreessen Horowitz and DCVC, will fund the development of ADAR‑based therapeutics targeting diseases such as ALS, Huntington’s, and...
Module 2, Section 3: Target Validation
The module on target validation walks through how phenotypic and target‑based strategies intersect in immune‑focused drug discovery. It highlights recent literature on TYK2 pseudokinase stabilization as a mechanism to block T‑cell signaling, and cites Icotrokinra and Deucravacitinib as successful examples....
Novo Nordisk Expands U.S. Patient Access to FDA-Approved Semaglutide Medicines Through Hims & Hers
Novo Nordisk announced a partnership with telehealth provider Hims & Hers, allowing U.S. consumers to purchase FDA‑approved Ozempic and Wegovy at the same self‑pay prices as other digital platforms. The agreement eliminates compounded GLP‑1 offerings and enables patients to transition...
Two AstraZeneca Drugs To Be Scrutinized in First FDA Cancer Advisory Panel in 9 Months
The FDA’s Oncologic Drugs Advisory Committee will convene on April 30 to evaluate AstraZeneca’s oral SERD camizestrant for first‑line HR‑positive, HER2‑negative breast cancer and its AKT inhibitor Truqap for metastatic hormone‑sensitive prostate cancer. Camizestrant’s Phase 3 SERENA‑6 trial reported a 56% reduction...
Incyte’s Lung Cancer Expansion Bid Thwarted by Issues at Novo’s Catalent-Acquired Site
The FDA rejected Incyte’s supplemental application to add non‑small cell lung cancer to Zynyz’s label, citing compliance failures at Novo Nordisk’s Catalent‑owned Indiana manufacturing plant. The agency’s complete response letter pinpointed inspection findings at the site as the sole approvability...
Hims & Hers Stock Price Is Surging Today. A Surprising Deal with Novo Nordisk Is the Reason Why
Shares of Hims & Hers Health surged after the telehealth firm announced a partnership with Novo Nordisk to sell the Danish drugmaker’s GLP‑1 weight‑loss products, including Wegovy. The deal reverses a recent legal clash in which Novo threatened litigation over...
The First KRAS Drugs Have Been Sluggish on the Market. Will the Next Generation Fare Better?
Amgen’s Lumakras earned accelerated FDA approval in 2021 as the first KRAS‑targeted therapy, but sales have been modest, reaching $92 million in Q4 2025 with only an 8% year‑over‑year increase. A second G12C inhibitor, Krazati, entered the market in 2022, narrowing Amgen’s...
Roche's Oral SERD Flunks Phase 3 Breast Cancer Test
Roche’s oral SERD giredestrant failed to meet its primary endpoint in the phase 3 persevERA trial, showing no significant improvement over letrozole when combined with palbociclib in first‑line HR‑positive, HER2‑negative advanced breast cancer. The company now limits U.S. filing to ESR1‑mutated...
Drug‑Safety Shock Hits Ipsen; Cut Exposure, Go Defensive
Macro: drug-safety shock; Key factor: SYMPHONY‑1 found secondary hematologic malignancies; Risk: regulatory re‑rating and pipeline impairment; Insight: trim Ipsen (IPN) exposure; favor defensive pharma 📉 — Viktor Kopylov, PhD, CFA. More insights: t.me/si14Kopylov
UniQure Sparks Gene Therapy Rally After Prasad Departs
UniQure leads genetic medicine biotech rally after news of Prasad’s exit https://t.co/zu1CtJfj29 by @realJacobBell $QURE $RGNX $ATRA $LXEO #GeneTherapy
Photothermally Triggered Intratumoral In Situ Drug Synthesis: A Smart Nanoplatform for NIR‐Controlled Precise Activation of Antitumor Precursors
Researchers have engineered an iron‑doped mesoporous silica nanoplatform (FOBA) that, when exposed to 808 nm near‑infrared light, uses the Y6 photothermal converter to heat and trigger a PEG gate, creating a transient solvent that enables in‑situ synthesis of the cytotoxic agent...
Multivitamin Modestly Slows Epigenetic Aging; Cocoa Doesn’t
Just published @NatureMedicine A daily multivitamin (MVM) slowed epigenetic aging in a randomized trial after 2 years; effect was small (~2 months) and not seen with cocoa extract supplement (vitamin was Centrum Silver) https://t.co/snOMNsTzW7 https://t.co/a6MhMuRhJb
FMT Triples the Boost to Cancer Immunotherapy
Not just replication, but triplication of the benefit of revving up cancer immunotherapy with FMT (Yes💩) https://t.co/rXyQdO74FJ
Aisa Pharma Reports Positive Results for AISA-021
Aisa Pharma announced positive Phase II data for AISA‑021, a once‑daily calcium channel blocker, in systemic sclerosis‑associated Raynaud’s phenomenon (SSc RP). The double‑blind, placebo‑controlled RECONNOITER trial enrolled 64 patients and showed a 22.1% reduction in weekly Raynaud attacks and a 155% placebo‑adjusted...
FDA's Turbulent Leadership and Shift Away From Advisory Panels
Two terrific #FDA related pieces by @lizzylawrence.bsky.social & @matthewherper.bsky.social. Matt's is a look at lessons to be drawn from Vinay Prasad's chaotic tenure; Lizzy's delves into the FDA's turning away from adcomms. https://t.co/gC5GNTiPm1 & https://t.co/ygvylHi0uz
Five Key Lessons From Vinay Prasad’s FDA Tenure
. @matthewherper is so damn reasonable. Smart, too. 5 lessons from Vinay Prasad’s turbulent tenure at the FDA https://t.co/R65vPj5ep5
GSK Licenses Liver Disease Drug to Italy’s Alfasigma for up to $690 Million
British drugmaker GSK has licensed worldwide rights to its experimental liver‑disease drug linerixibat to Italy’s Alfasigma for up to $690 million. The agreement includes a $300 million upfront payment, a $100 million tranche upon U.S. approval, $20 million upon European and UK clearance, and...
Undermanned VRBPAC Meets to Pick Flu Vaccine Strains
The #VRBPAC committee is scheduled to meet Thursday to advise #FDA on #flu vaccine strain selection. VRBPAC is supposed to have at least 15 members. It currently has 7 voting members & no chair. It has met only twice since...
TopTown SuperDNA Aims to End Aging by 2028
We will solve aging or will die trying. Check out the TopTown SuperDNA project. It is no longer a concept - projected launch in Q3 2028. Fully-integrated residential-biotechnology-clinical infrastructure is key to longitudinal AI-powered drug discovery. https://t.co/LK5XaTci29
Key Factors Contributing to Uncertainty in Moderna's mRNA Vaccine Review
The FDA initially refused to review Moderna’s mRNA‑based flu vaccine, prompting surprise among industry observers. After a White House meeting, the agency reversed course and granted Moderna a Type A meeting, effectively resetting the review process. Lanton notes this regulatory flip‑flop...
RLMD's Strong Bladder Cancer Data Hits IBRX Valuation
The $RLMD bladder cancer data (phase 2) reported this morning look very good. Another competitive blow to $IBRX particularly at its bloated valuation. https://t.co/wYThfwIpG8
QURE-FDA Turbulence Highlights Market Volatility, MRNA Settlement Relief
9March: What a roller-coaster ride with $QURE and @US_FDA ! I sum up a wild week--plus $MRNA's could've-been-worse settlement with $ABUS & $ROIV (w/ insights from CEO Matt Gline) and updates from $DAWN and $TNGX in my latest StockWatch for...
ENHERTU® Granted Priority Review in the U.S. as Post-Neoadjuvant Treatment for Patients with HER2 Positive Early Breast Cancer
Daiichi Sankyo and AstraZeneca have received FDA priority review for ENHERTU® as a post‑neoadjuvant therapy in HER2‑positive early breast cancer. The decision follows the DESTINY‑Breast05 phase 3 trial, which showed a 53% reduction in invasive disease‑free survival events versus trastuzumab‑emtansine (T‑DM1). Three‑year...
First Evidence of Cancers’ Unique Nuclear Metabolic Fingerprints
Researchers identified more than 200 metabolic enzymes physically attached to chromatin, revealing a distinct "nuclear metabolic fingerprint" for each cell type and cancer. Approximately 7% of all chromatin‑bound proteins were metabolic, including unexpected oxidative‑phosphorylation components. The fingerprint varied by tumor...
Safety Concerns Prompt Ipsen to Pull Tazverik From Market
Ipsen is voluntarily withdrawing its EZH2 inhibitor Tazverik worldwide after an interim safety analysis in the phase 1b/3 SYMPHONY‑1 trial linked the drug to secondary hematologic malignancies. The pull‑back includes terminating all ongoing Tazverik studies and recalling the product in markets...
GSK Secures $300M Upfront for Linerixibat Deal
#GSK to get $300m upfront and $100m upon US FDA approval plus $20m for EU approval and up to $270m sales based payments from Alfasigma who will acquire rights to Linerixibat, a bile acid transporter inhibitor, currently under development.
Navigating the PPQ Process: Proven Strategies to Safeguard Quality for Cell and Gene Therapies
Process Performance Qualification (PPQ) is the final validation step before commercial manufacturing of cell and gene therapies, but its complexity often triggers delays and compliance risks. The article highlights three proven strategies—early master‑plan development, continuous quality improvement, and data‑driven analytics—to...
Roche’s Big-Hope Breast Cancer Drug Fails in Crucial First-Line Trial
Roche’s oral breast‑cancer therapy, touted as a potential blockbuster, missed its primary endpoint in the pivotal Phase 3 persevERA trial. The study evaluated the drug as a first‑line treatment for hormone‑receptor‑positive, HER2‑negative metastatic breast cancer and enrolled more than 1,200 patients...
kyron.bio Announces Strategic Partnership with Servier to Advance Precision Glycosylation in Antibody Therapeutics
Kyron.bio announced a strategic partnership with Servier to glycoengineer a Servier‑selected antibody using its precision glycosylation platform. Servier will fund the research and retain an option to pursue further development based on the results. The collaboration builds on Kyron.bio’s 2024...